[Senate Hearing 114-661]
[From the U.S. Government Publishing Office]



                                                        S. Hrg. 114-661

                              RIGHT TO TRY

=======================================================================

                                HEARING

                               before the

                              COMMITTEE ON
               HOMELAND SECURITY AND GOVERNMENTAL AFFAIRS
                          UNITED STATES SENATE

                    ONE HUNDRED FOURTEENTH CONGRESS


                             SECOND SESSION

                               ----------                              

   CONNECTING PATIENTS TO NEW AND POTENTIAL LIFE SAVING TREATMENTS, 
                           FEBRUARY 25, 2016

  EXPLORING A RIGHT TO TRY FOR TERMINALLY ILL PATIENTS, SEPTEMBER 22, 
                                  2016

                               ----------                              

        Available via the World Wide Web: http://www.fdsys.gov/

                       Printed for the use of the
        Committee on Homeland Security and Governmental Affairs
        
        
        
        




                           RIGHT TO TRY--2016
                           
                           
                           
                           




                                                        S. Hrg. 114-661
 
                              RIGHT TO TRY

=======================================================================

                                HEARING

                               before the

                              COMMITTEE ON
               HOMELAND SECURITY AND GOVERNMENTAL AFFAIRS
                          UNITED STATES SENATE

                    ONE HUNDRED FOURTEENTH CONGRESS


                             SECOND SESSION

                               __________

   CONNECTING PATIENTS TO NEW AND POTENTIAL LIFE SAVING TREATMENTS, 
                           FEBRUARY 25, 2016

  EXPLORING A RIGHT TO TRY FOR TERMINALLY ILL PATIENTS, SEPTEMBER 22, 
                                  2016

                               __________

                               __________

        Available via the World Wide Web: http://www.fdsys.gov/

                       Printed for the use of the
        Committee on Homeland Security and Governmental Affairs
        
        
        
        
        
        
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        COMMITTEE ON HOMELAND SECURITY AND GOVERNMENTAL AFFAIRS

                    RON JOHNSON, Wisconsin Chairman
JOHN McCAIN, Arizona                 THOMAS R. CARPER, Delaware
ROB PORTMAN, Ohio                    CLAIRE McCASKILL, Missouri
RAND PAUL, Kentucky                  JON TESTER, Montana
JAMES LANKFORD, Oklahoma             TAMMY BALDWIN, Wisconsin
MICHAEL B. ENZI, Wyoming             HEIDI HEITKAMP, North Dakota
KELLY AYOTTE, New Hampshire          CORY A. BOOKER, New Jersey
JONI ERNST, Iowa                     GARY C. PETERS, Michigan
BEN SASSE, Nebraska

                    Keith B. Ashdown, Staff Director
                  Christopher R. Hixon, Staff Director
                   Satya P. Thallam, Chief Economist
                    Daniel P. Lips, Policy Director
              Joshua P. McLeod, Professional Staff Member
              Gabrielle A. Batkin, Minority Staff Director
           John P. Kilvington, Minority Deputy Staff Director
 Katherine C. Sybenga, Minority Chief Counsel for Governmental Affairs
         Brian F. Papp, Jr., Minority Professional Staff Member
    Lynn Sha, Senior Health Policy Advisor, Office of Senator Carper
                     Laura W. Kilbride, Chief Clerk
                   Benjamin C. Grazda, Hearing Clerk
                   
                   
                            C O N T E N T S

                                 ------                                
                                                                   Page

                      THURSDAY, FEBRUARY 25, 2016

Opening statements:
    Senator Johnson..............................................     1
    Senator Carper...............................................     3
    Senator Ernst................................................    22
    Senator Ayotte...............................................    28
Prepared statements:
    Senator Johnson..............................................    35
    Senator Carper...............................................    37

                               WITNESSES

Darcy Olsen, President and Chief Executive Officer, Goldwater 
  Institute......................................................     4
Laura McLinn, Indianapolis, Indiana (accompanied by Jordan 
  McLinn)........................................................     6
Diego Morris, Phoenix, Arizona...................................     8
Joseph V. Gulfo, M.D., Executive Director, Rothman Institute of 
  Innovation and Entrepreneurship, Farleigh Dickinson University.    10
Nancy Goodman, Executive Director, Kids v Cancer.................    12

                     Alphabetical List of Witnesses

Goodman, Nancy:
    Testimony....................................................    12
    Prepared statement...........................................   187
Gulfo, Joseph V., M.D..:
    Testimony....................................................    10
    Prepared statement with attachments..........................    94
McLinn, Laura:
    Testimony....................................................     6
    Prepared statement...........................................    88
Morris, Diego:
    Testimony....................................................     8
    Prepared statement...........................................    91
Olsen, Darcy:
    Testimony....................................................     4
    Prepared statement with attachment...........................    39

                                APPENDIX

McLinn Fact Sheet................................................   191
Letters referenced by Senator Johnson............................   193
Patient Access to Investigational Therapies (FDA Factsheet)......   209
Statements submitted for the Record from:
    Abigail Alliance for Better Access to Developmental Drugs....   210
    Americans for Safe Access....................................   214
Responses to post-hearing questions for the Record from:
    Ms. Olsen....................................................   235

                      THURSDAY, SEPTEMBER 22, 2016

Opening statements:
    Senator Johnson..............................................   237
    Senator Carper...............................................   239
    Senator Paul.................................................   257
    Senator Lankford.............................................   272
Prepared statements:
    Senator Johnson..............................................   281
    Senator Carper...............................................   282

                               WITNESSES

Matthew Bellina, Lieutenant Commander, U.S. Navy (Retired).......   241
Hon. Ian C. Calderon, Majority Leader, State Assembly, State of 
  California.....................................................   243
Hon. Jim Neely, D.O., Member, House of Representatives, State of 
  Missouri.......................................................   246
Richard Garr, Former President and Chief Executive Officer, 
  Neuralstem, Inc................................................   247
Andrew McFadyen, Executive Director, The Isaac Foundation........   249
Eric and Frank Mongiello.........................................   251
Peter Lurie, M.D., M.P.H., Associate Commissioner for Public 
  Health Strategy and Analysis, Food and Drug Administration, 
  U.S. Department of Health and Human Services...................   266

                     Alphabetical List of Witnesses

Bellina, Matthew:
    Testimony....................................................   241
    Prepared statement...........................................   284
Calderon, Hon. Ian C.:
    Testimony....................................................   243
    Prepared statement...........................................   286
Garr, Richard:
    Testimony....................................................   247
    Prepared statement...........................................   296
Lurie, Peter, M.D., M.P.H.:
    Testimony....................................................   266
    Prepared statement...........................................   307
McFadyen, Andrew:
    Testimony....................................................   249
    Prepared statement...........................................   299
Mongiello, Eric and Frank:
    Testimony....................................................   251
Neely, Hon. Jim:
    Testimony....................................................   246
    Prepared statement...........................................   292

                                APPENDIX

Chart submitted by Senator Johnson...............................   313
Individual Patient Expanded Access Investigational New Drug 
  Application (IND) (FDA Form)...................................   314
Patient Access to Investigational Therapies (FDA Factsheet)......   315
Responses to post-hearing questions for the Record from:
    Mr. Calderon.................................................   317
    Mr. Neely....................................................   318
    Mr. Garr.....................................................   319
    Mr. Lurie....................................................   320


    CONNECTING PATIENTS TO NEW AND POTENTIAL LIFE-SAVING TREATMENTS

                              ----------                              


                      THURSDAY, FEBRUARY 25, 2016

                                     U.S. Senate,  
                           Committee on Homeland Security  
                                  and Governmental Affairs,
                                                    Washington, DC.
    The Committee met, pursuant to notice, at 10:02 a.m., in 
room SD-342, Dirksen Senate Office Building, Hon. Ron Johnson, 
Chairman of the Committee, presiding.
    Present: Senators Johnson, Lankford, Ayotte, Ernst, Sasse, 
Carper, McCaskill, Heitkamp, and Peters.

             OPENING STATEMENT OF CHAIRMAN JOHNSON

    Chairman Johnson. Good morning. This hearing will come to 
order.
    I just want to thank all of the witnesses and everybody in 
the audience for attending what I think is a really important 
hearing. The title of the hearing is: ``Connecting Patients to 
New and Potential Life-Saving Treatments.'' We are going to be 
talking to Darcy Olsen, who has certainly been at the forefront 
of trying to pass--and successfully passing--legislation called 
``Right to Try'' in States and trying to take a look at that on 
a Federal basis.
    From a personal standpoint, I do not expect anybody to 
understand my story, but our first child, our daughter, Carey, 
was born with a pretty serious congenital heart defect. And, 
her first day of life, modern medicine saved her life with a 
procedure.
    Eight months later, when her heart was the size of a small 
plum, they rebaffled the upper chamber of her heart. And so her 
heart operates backward today, but she is 32 years old and she 
has lived a perfectly normal life.
    When we were going through that, the term ``medical 
practice'' always kept running through my mind, because I never 
thought of it in terms of what it really meant--medical 
practice--that there is nothing certain. There is no therapy, 
there is no procedure, and there is no drug that performs 
exactly the same with every person. Every person is different. 
And so, the advancement of medicine really does rely on the 
expertise of individual doctors working with patients, patients 
that should have the freedom to be able to try things, 
particularly, when we know the end result.
    So this hearing is about allowing patients that freedom--
and it is such common sense. Just let people try.
    But then, when you start getting past the obvious 
conclusion--that, well, people should have the right to make 
these decisions themselves--you start getting into the 
problems, the legitimate problems and concerns of whether it is 
the responsibility of the Food and Drug Administration (FDA) or 
the companies that are producing the drugs or the procedures--
and there are legitimate concerns. It gets complex pretty fast.
    But, at the heart of this issue, it really is about people. 
And I would ask that my written opening statement be entered in 
the record, without objection.\1\
---------------------------------------------------------------------------
    \1\ The prepared statement of Senator Johnson appears in the 
Appendix on page 35.
---------------------------------------------------------------------------
    We also have a binder of 200 letters submitted to this 
Committee in favor of us holding this hearing. I normally do 
not spend a whole lot of time on an opening statement, but I 
just wanted to read at least one letter. This comes from Tim 
Wendler of Waukesha, Wisconsin. He is the husband of the late 
Trickett Wendler, who I did meet back in 2014. She had 
amyotrophic lateral sclerosis (ALS). And Tim writes:
    ``My wife, Trickett Wendler, passed on March 18, 2015, 
after a 2-year battle with amyotrophic lateral sclerosis (ALS). 
When she was first diagnosed, we scoured the country to find 
the best doctors, medicine, treatments, etc. The only treatment 
drug prescribed for my wife was the exact same drug that was 
prescribed to her father over 20 years ago. Literally no 
progress. I will tell you that once we were out of options, our 
desire to transform the possibilities became the singular focus 
of our life. I have three small children with the hereditary 
gene that predisposes them to this intolerant disease. That is 
why it is infinitely important to provide patients with 
alternatives where none exist. The `Right to Try' provides 
something that doctors, drug manufacturers, legislators, etc.--
cannot provide: hope.''
    Sadly, my wife did not have this option, but I write to you 
today to implore to you the importance of providing hope where 
none exists. I pray every day that progress is made to find a 
cure and that my children are not given the same drug that 
their mother and grandfather were given. But if none is made, I 
pray that my children will have the `Right to Try.'
    Now, I read that one because, when I did meet Trickett, 
this was, quite honestly, shortly after I met you, Darcy, and 
was made aware of what you were trying to do with ``Right to 
Try.'' And without prompting, without being asked, I made the 
statement, ``I know about this initiative, I am fully 
supportive of `Right to Try,' '' and tears started streaming 
down her cheeks.
    That has an effect. And the purpose of this hearing now is 
to put a face--a human face--so that we in Congress can make 
some intelligent decisions here--so that we can try and break 
through the legitimate concerns of whether it is the FDA or the 
drug manufacturers and give people, give patients the freedom 
and the access so that they have hope. And that is really what 
this hearing is all about.
    I again thank the witnesses for very thoughtful testimony. 
It will be powerful testimony. And I just want to thank my 
colleagues for coming because I think that this is one of the 
most important hearings that we will hold.
    With that, Senator Carper.

              OPENING STATEMENT OF SENATOR CARPER

    Senator Carper. Thank you. Thank you so much, Mr. Chairman. 
Thank you for bringing this together this morning. I appreciate 
your willingness to open a conversation about an issue that is 
important to all of us, especially to Americans that are 
seeking access to potentially life-saving treatment and new 
medical innovations for themselves or members of their 
families.
    I want to thank young Jordan for bringing his Mom today to 
be one of our witnesses, so that she can tell your story. I 
want to thank Ms. Olsen, Mr. Morris, Dr. Gulfo, and Ms. Goodman 
for being with us, especially for those of you who are going to 
share your own stories with us today.
    One of the things that the Chairman does as we start a 
hearing, before the witnesses speak, he swears in the 
witnesses--and this is going to be interesting to see how young 
Jordan, age five, handles that. It will be one for the books. 
Jordan, we are glad that you are here. All I can say is that 
when my boys were 5 years old, there was no way in the world 
that I would have put them at this table. I know--excuse me--
six. My sheet here says five. But there is no way that I would 
have done that. [Laughter.]
    They would have wrecked the hearing. But you seem to be 
very well behaved--better than some of us.
    Today, we are going to hear from patients, we are going to 
hear from their loved ones, and we are going to hear from 
others about potential opportunities, hopefully, to improve 
access to medical breakthroughs and to life-saving medical 
treatments. These folks and their families have faced some of 
the most difficult and challenging circumstances and decisions 
that just about anybody could face, and they deserve our 
compassion and they deserve our understanding. And they 
certainly are going to get our attention, today.
    Speaking as a dad, as a husband, as a brother, and as a 
son, it is important that we learn from our witnesses' 
experiences so that we in Congress can maybe work better 
together with the Executive Branch, with patient groups, with 
industry, and with other stakeholders to ensure that all 
Americans can gain access to safe and effective life-saving 
treatments as quickly as possible.
    Simply put, the development of new medicines is, as we 
know, a long, complex, and risky process--an expensive process. 
For individuals with life-threatening conditions and for their 
loved ones, safe and effective treatments cannot come quickly 
enough.
    As we will hear from some of our witnesses today, the path 
for patients and their physicians to access innovative, new 
treatments may not always be clear. Reforms may be needed to 
make sure that patients, their families, and their doctors have 
the information that they need to explore new treatment 
options.
    The U.S. Food and Drug Administration, which is charged 
with ensuring that the drugs available to American consumers 
are safe and effective, has given an extraordinary level of 
attention to the requests of patients with life-threatening 
conditions. In fact, I am told that they have approved more 
than 99 percent of requests for emergency treatments.
    Despite these high approval rates, I understand that the 
FDA believes that more can be done and that they are 
continuing, at the FDA, to work to improve patient access to 
these experimental medical treatments.
    I hope that we can help with some of those efforts and 
continue to work closely with the patients, with health care 
providers, with the pharmaceutical industry, and with the FDA 
to ensure that all patients and their families can access safe 
and reliable treatments as quickly as possible.
    Again, we are delighted that you are all here, and it is 
just a real special treat to see young Jordan at 6 years old--
Jordan, when is your birthday?
    Chairman Johnson. May 15.
    Senator Carper. May 15. All right. Well, good, a day to 
remember. Thank you very much. Welcome, everybody.
    Chairman Johnson. So almost seven--and, by the way, he is a 
great writer and a good speller--a lot further progressed than 
I was in kindergarten, trust me.
    I do want to explicitly ask consent to enter those letters 
for the record\1\ and I also want to let all of the Members 
know that we will distribute those to your offices as well.
---------------------------------------------------------------------------
    \1\ The letters referenced by Senator Johnson appears in the 
Appendix on page 193.
---------------------------------------------------------------------------
    As Senator Carper said, it is the tradition of this 
Committee to swear in witnesses, so if you will all rise and 
raise your right hand. Do you swear the testimony you will give 
before this Committee will be the truth, the whole truth, and 
nothing but the truth, so help you, God?
    Ms. Olsen. I do.
    Ms. McLinn. I do.
    Master McLinn. I do.
    Mr. Morris. I do.
    Ms. Goodman. I do.
    Dr. Gulfo. I do.
    Chairman Johnson. Thank you. Please be seated.
    Our first witness is Darcy Olsen. Ms. Olsen is the 
president and Chief Executive Officer (CEO) of the Goldwater 
Institute in Phoenix, Arizona and the author of the book ``The 
Right to Try.'' She is a graduate of Georgetown University and 
New York University. She is also a recipient of numerous awards 
for her work in public policy, including a 2014 Bradley Prize. 
Ms. Olsen.

  TESTIMONY OF DARCY OLSEN,\2\ PRESIDENT AND CHIEF EXECUTIVE 
                  OFFICER, GOLDWATER INSTITUTE

    Ms. Olsen. Thank you, Chairman Johnson, Ranking Member 
Carper, and other Members of the Committee. Thank you for being 
here today.
---------------------------------------------------------------------------
    \2\ The prepared statement of Ms. Olsen appears in the Appendix on 
page 39.
---------------------------------------------------------------------------
    As I was preparing my testimony for you on Monday, I 
received a call from an old friend of mine, who called to tell 
me that she had recently received an ALS diagnosis--or what a 
lot of people know as Lou Gehrig's disease. And, although it 
has only been a few weeks since the diagnosis, her 
deterioration has been so rapid that they have already called 
in hospice to plan for those final days. And, as her physicians 
explained to her, ALS is 100 percent fatal, and there really 
are no treatments. And this goes, of course, Chairman Johnson, 
to what you were saying. The same medicines that they were 
using 20 years ago are what they are using today.
    But what Hazel's doctors really mean is that, in their 
toolbox of approved medicines, there is nothing left, because 
the truth is that right now, pending before the FDA, there are 
a dozen treatments to treat ALS and there is possibly even a 
cure. And in my book, ``The Right to Try,'' I tell the story of 
a man named Ted Harada--and we call Ted ``Lazarus'' because he 
is the first known survivor of Lou Gehrig's. He was very 
fortunate to get into a clinical trial where he received a 
treatment that reversed his ALS symptoms. Today, it is 7 years 
later, and he is swimming with his kids, he is walking 5 
kilometer (5k) races, and he has seen no decline at all in his 
respiratory function. Ted and 31 other Americans have been 
lucky enough to try this cutting-edge therapy. But, in the 
years since that clinical trial began, 24,000 other Americans 
with Lou Gehrig's disease have passed.
    The problem is the FDA's extremely long and archaic process 
for approving treatments--especially for people with terminal 
diseases. It takes on average 15 years to bring one of these 
new drugs to market.
    During the course of writing my book, someone who has 
become a friend of mine, Jenn McNary--she is the mother of two 
brothers with Duchenne's Muscular Dystrophy (DMD)--said to me, 
``By the time this drug that we need is on the market, we are 
going to lose an entire generation of boys.'' Fifteen years of 
delay at the FDA is an entire generation of boys lost. It is 
unethical not to give these boys a chance at life.
    It is also unconstitutional. In America, today, terminal 
patients have the right to hasten their deaths through ``Right-
to-Die'' laws, but they do not have the right to try to fight 
to live. So you can get drugs to die, but not if you want to 
save your life.
    And that is why the Goldwater Institute designed what we 
call ``Right-to-Try'' laws. And, as of today, 24 States--
including 7 of the home States that members of this panel 
represent--have adopted the ``Right to Try.'' And that is 
passing on a 99-1 vote margin. Under these laws, if you have a 
terminal diagnosis and the FDA-approved treatments are not 
working for you, you have the right to try to save your life by 
taking some investigational medicines that are under study at 
the FDA, but may still be 10 years, or even 15 years, away from 
getting that final green light.
    These ``Right-to-Try'' laws are not a panacea. They will 
not help every patient. But, at least they move us in the right 
direction. I know of 28 patients that are being treated under 
the laws at this time and I know that lives, in fact, are being 
saved.
    There are two key reforms that you can move in Congress 
that will help millions access some of these life-saving drugs.
    The first is that Federal law should clearly let doctors 
prescribe drugs to terminal patients after they have passed 
``Phase 1'' safety testing. Terminal patients simply do not 
have time to wait for efficacy tests.
    Second, an estimated 30 percent of the newest advances in 
medicine are first available overseas--as you will hear in 
Diego's story. These drugs, that have already received the 
green light from countries like Germany and Japan, should be 
available to patients here in the United States. This would 
bring proven life-saving treatments to patients in America, 
now.
    If you were on a sinking ship--or your spouse or your 
child--would you pass on the only available lifeboat because 
the government had not certified it yet?
    As a society, we can and we should debate the best ways to 
make better and stronger lifeboats. But there is no argument 
for withholding the lifeboats that we do have from people who 
are drowning. So, please, help us loosen the ropes and let us 
get the lifeboats in the water.
    Thank you.
    Chairman Johnson. Thank you, Ms. Olsen.
    Our next witness is 6-year-old Jordan McLinn, who brought 
along his Mom, Laura McLinn. Ms. McLinn is the owner of Indy 
Learning Center, founded in 2005, and the mother of three--
including Jordan. She received her bachelor's degree in 
elementary education from the University of Indianapolis and a 
master's degree in education from Indiana University. She is a 
former junior high school and high school math teacher.
    Ms. McLinn and Jordan.

      TESTIMONY OF LAURA MCLINN,\1\ INDIANAPOLIS, INDIANA 
                 (ACCOMPANIED BY JORDAN MCLINN)

    Ms. McLinn. Thank you, Chairman Johnson and Ranking Member 
Carper. I really appreciate you allowing us to be here today.
---------------------------------------------------------------------------
    \1\ The prepared statement of Ms. McLinn appears in the Appendix on 
page 88.
---------------------------------------------------------------------------
    I am going to let Jordan just say a word here, and then, if 
it is OK, I am going to just send him out with Erica to play, 
but I want to make sure that he has a turn to speak here. Go 
quickly. What do you want to say?
    Master McLinn. Please say yes for the drug.
    Ms. McLinn. OK.
    Master McLinn. Watch this, Daddy.
    Chairman Johnson. I think he is, Jordan. [Laughter.]
    Thank you, Jordan. We appreciate it.
    [Applause.]
    Ms. McLinn. About a year ago around this time, Jordan and I 
were in the State of Indiana, and we were testifying together 
for the ``Right-to-Try'' legislation, and Jordan stood there, 
and he said to them, ``Please say yes.''
    This year, he is a year older, and the things that I am 
going to tell you I do not, necessarily, want his ears to hear 
this time, so I want to start by just telling you how special 
Jordan is. You see him. He is six. He is running around here 
like he owns the place. He sat right up there in that chair. 
And, that is what he does. He is really smart. He plays. He 
hugs me. He climbs.
    But, believe it or not, he is in a fight with the clock--
and he is declining. At just 6 years old, he has Duchenne 
Muscular Dystrophy, just like you heard about earlier, about 
Jenn's boys. Jordan has the same disease. It is 100 percent 
fatal. Most boys live to be around 20. Some make it a little 
longer; some do not make it that long.
    So, he is in the physical decline phase now, which means 
that he is losing function--and, with muscle disease, when that 
starts to happen, you just do not get that back. It is a little 
different from maybe cancer and some other diseases where, 
maybe when you are on your deathbed you can take a medication, 
and then, maybe, you can live a long, full life.
    For Jordan, it does not work that way. We cannot wait 15 
years and then give him these drugs that are coming up through 
the pipeline for him. There are exon-skipping drugs that are 
working, that exist. So, for the first time in the history of 
this disease, there is something that can help Jordan.
    When I was in Ohio testifying for ``Right to Try,'' I 
actually talked with them about changing the language in their 
bill there because they defined ``terminal'' as, I believe, 
maybe 6 months or a year to live. Well, Jordan's disease is 
terminal, and when he is at the point where maybe he has 6 
months to live, it is going to be too late for him to receive 
the treatments that are existing, that are coming up.
    So what I would like to say to you today is that Jordan--
there is a drug there. Jordan needs it now. I do not know the 
best way for him to get that. When we testified for ``Right to 
Try'' and it was passed in the State of Indiana, we were given 
kind of a whole new layer of hope--kind of like a backup plan. 
But, ideally, we want Jordan to get this drug because the FDA 
approves it, because it works. We do not want them to wait 15 
years to approve it because we do not have that kind of time.
    Back in 2012, you passed the Food and Drug Administration 
Safety and Innovation Act (FDASIA). The President signed that 
into law in 2012. And prior to that, I am sure that you 
remember when acquired immunodeficiency syndrome (AIDS) was 
very scary and everyone was so afraid that so many people were 
going to die--and the FDA acted very quickly and they started 
approving things quickly. And they should have. And now you do 
not hear that much about it, right? Because there are so many 
drugs out there that are helping patients with human 
immunodeficiency virus (HIV) and AIDS.
    Well, in 2012, the President signed FDASIA. FDASIA says 
that, ``Hey, this should not just apply to something like that. 
It should also apply to rare disease.'' So you gave the FDA the 
authority to say, ``It is OK to do a smaller trial, it is OK to 
do accelerated approval, and it is OK to do that for these 
boys, like Jordan, that have rare diseases.'' We might not find 
500 people to do a trial for a disease like what Jordan has. 
There might not be that many patients out there. But, right 
now, at the end of May, the FDA has a decision to make about an 
exon-skipping drug. They are going to decide yes or no. They 
are going to say yes or no to Jenn's sons, who are on this drug 
that you heard about earlier.
    In this trial, there are 12 boys. So that is a small group, 
right? The FDA does not like that. But there are 12. There are 
12 in this trial. The 4-year data showed that 10 of those 12 
boys are still walking. In the external control group that had 
13 boys--the same ages, with the same mutation, and receiving 
the same steroid regimen--one of the 13 is still walking after 
14 years. That should tell everyone in this room something, 
right? That drug is working for these boys.
    But, we are not getting a good feeling right now from the 
FDA that we are going to hear yes at the end of May. We are 
hopeful. It is the right thing to do. They have the authority 
to say yes. You have given them that power. So, I am not asking 
even for any new legislation--although I believe in ``Right to 
Try'' and I think that there is definitely a place for that. It 
might not work for Jordan today, but there are lots of cancer 
patients and ALS patients that ``Right to Try'' works for. I 
believe in that.
    But I also believe that the FDA needs to use the power that 
they already have been given, by you, to say yes and give 
accelerated approval to drugs that are working, that are safe, 
and that are effective. And boys like Jordan--all of these boys 
deserve that chance.
    So I urge you to urge the FDA to use the tools and the 
power that you have given them to say yes.
    Chairman Johnson. Thank you, Laura, and, obviously, we hope 
that they say yes.
    Ms. McLinn. Thank you.
    Chairman Johnson. Our next witness is Diego Morris. Diego 
is a 15-year-old honor student at Brophy College Preparatory 
and previously a student and student body vice president at All 
Saints Episcopal Day School. He is also a cancer survivor and, 
at the age of 13, was honorary chairman of the ``Right to Try'' 
initiative in Arizona. Diego.

         TESTIMONY OF DIEGO MORRIS,\1\ PHOENIX, ARIZONA

    Mr. Morris. Good morning, Mr. Chairman and Members of the 
Committee. Thank you for inviting me to testify. I am 
incredibly honored to be with you today.
---------------------------------------------------------------------------
    \1\ The prepared statement of Mr. Morris appears in the Appendix on 
page 91.
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    Four years ago, I was a typical 11-year-old boy. I was 
playing two sports at the time--baseball and soccer. One 
morning, I woke up with pain on the outside of my left knee. I 
thought that it was just a typical sports injury. I continued 
to play in my games, and I did everything as usual for a few 
days. But the pain would not go away and it was causing me to 
limp. My mom took me to the pediatrician--and thank goodness my 
doctor knew immediately that something was not right. She sent 
me to an orthopedic surgeon the following day for an X-ray. The 
doctor told my mom that he believed I had osteosarcoma, a rare 
type of bone tumor, just by looking at my X-ray.
    Everything happened quickly after that appointment. My 
parents consulted with many of their physician friends about 
what we should do next. My parents took the advice of our close 
family friends. He is a radiation oncologist and she is a 
pediatrician. They told my parents that I needed to have a 
biopsy as soon as possible at a premier research institution.
    Just 3 days after my trip to the pediatrician, we were on 
our way to St. Jude Children's Research Hospital (St. Jude) in 
Memphis, Tennessee. We never stopped hoping that I did not have 
cancer. After a long week of different types of tests and 
scans, they performed a biopsy. We knew the surgeons would be 
looking at a quick type of analysis that they perform in the 
operating room. If the surgeons determine that it is cancer at 
that point, they go ahead and place a port in the patient's 
chest. When I had barely come out from anesthesia, I asked my 
parents, ``Do I have a port?'' They answered, ``Yes.'' The 
three of us cried--and my life was never the same again.
    After many conversations with physicians, we decided that I 
should start chemotherapy back in Phoenix. My parents came to 
the conclusion that, if I would receive the exact same pre-
surgery chemotherapy in Phoenix, I should be home and in my own 
bed as much as possible, surrounded by the family and friends 
who love me. I received chemotherapy for 10 weeks at Phoenix 
Children's Hospital before returning to St. Jude for limb 
salvage surgery. I am so grateful that the surgeons were able 
to save my leg and completely remove the tumor. They inserted a 
significant titanium device in my leg, which partially replaced 
my femur and my knee.
    After surgery, the analysis of the tumor indicated that the 
necrosis, or the amount of the tumor killed by the initial 
chemotherapy, unfortunately, was only 50 percent. The doctors 
were hoping to see at least 80 percent necrosis. This meant 
that I would have to have a very aggressive plan of treatment. 
I needed a total of 21 rounds of chemotherapy, with some of the 
strongest chemotherapy drugs.
    Thank goodness that my parents' physician friends never 
stopped doing research on every available treatment for me. 
They told my parents about a drug called Mifamurtide. 
Mifamurtide is an immune therapy drug that has improved 
survival rates for children with osteosarcoma. My parents were 
excited about the drug, but they quickly realized that it was 
not approved in the United States. Mifamurtide was available in 
so many countries all over the world, they were astonished that 
it was not available in America. The trials for Mifamurtide had 
actually been started by physicians in the United States. My 
parents flew to Mexico City with our friend who is a 
pediatrician to see the results of Mifamurtide on their 
osteosarcoma patients. The doctors there showed them their 
findings and told my parents that I was welcome in their 
hospital to receive Mifamurtide.
    The clock was ticking. In order to have Mifamurtide immune 
therapy, I had to start it at the exact same time as my post-
surgery chemotherapy--just 10 weeks after undergoing surgery at 
St. Jude. My parents never gave up hope that they could get 
this treatment in the United States. They contacted our 
Congressman, the FDA, the drug manufacturer, and anyone who 
they thought could help us find a way. They even spoke with the 
lead physicians for the U.S. trials at the University of Texas 
MD Anderson Cancer Center (MD Anderson) and Memorial Sloan 
Kettering Cancer Center (Sloan Kettering). The doctor at Sloan 
Kettering explained Mifamurtide and answered all of my parents' 
questions. My parents told him that they were not looking for 
guarantees--just hope.
    I will never forget my parents and their friends explaining 
to me and my brother that we were going to move to London so 
that I could have Mifamurtide treatment along with my 
chemotherapy. We were so upset with my parents, at first, but, 
ultimately, we accepted that this might help to save my life. 
Our entire family left our home in Phoenix, Arizona and moved 
5,000 miles away.
    My chemotherapy treatment was brutal, and I was in the 
hospital more often than not. My dad was always exhausted and 
hated not being with us. My mom was exhausted, too, going back 
and forth between the hospital and home to take care of my 
brother and I.
    But, there is no place like home. I felt so isolated. I 
missed my friends, my home, my dog, and my school.
    My family and I were very fortunate to have the resources 
to relocate to another country. Most people do not have that 
option. When my family and I returned to the United States, we 
all agreed that we would do anything to help other families to 
not have to go through what we did to get this treatment--or, 
worse, to not have a promising treatment at all. So, when the 
Goldwater Institute asked me to serve as honorary chairman of 
the ``Right-to-Try'' campaign in Arizona, I jumped at the 
opportunity. I am grateful to Darcy Olsen and the people at the 
Goldwater Institute for giving me the chance to do something 
positive with my terrible experience. I am grateful to be 
alive, and I am grateful to be here with your esteemed 
Committee today.
    I hope and pray that we can make it easier for Americans to 
have faster access to critical medical treatment. Please help 
us give Americans a better chance to save their own lives and 
those of their loved ones. No guarantees--just hope.
    Thank you very much.
    Chairman Johnson. Diego, I have to just quickly ask now, 
have you been judged cancer-free?
    Mr. Morris. Yes.
    Chairman Johnson. For how long?
    Mr. Morris. It has been about 3 years.
    Chairman Johnson. OK. Thank you for your testimony.
    Mr. Morris. Thank you.
    Chairman Johnson. Our next witness is Dr. Joseph Gulfo. Dr. 
Gulfo is executive director of the Rothman Institute of 
Innovation and Entrepreneurship at Fairleigh Dickinson 
University, at which he is spearheading the Initiative for 
Patient-Centered Innovation. He is also visiting scholar at the 
Mercatus Center of George Mason University and the author of 
``Innovation Breakdown.'' Dr. Gulfo received his Doctor of 
Medicine (M.D.) from the University of Medicine and Dentistry 
of New Jersey and his Master of Business Administration (MBA) 
from Seton Hall University. He was responsible for the approval 
of an antibody for prostate cancer, a bladder cancer drug, and 
a medical device for melanoma detection. Dr. Gulfo.

  TESTIMONY OF JOSEPH V. GULFO, M.D.,\1\ EXECUTIVE DIRECTOR, 
ROTHMAN INSTITUTE OF INNOVATION AND ENTREPRENEURSHIP, FAIRLEIGH 
                      DICKINSON UNIVERSITY

    Dr. Gulfo. I would also like to note that I submitted a 
fuller, written statement, a book excerpt, and other writing to 
the record that I would like to be submitted.
---------------------------------------------------------------------------
    \1\ The prepared statement of Dr. Gulfo and attachments appears in 
the Appendix on page 94.
---------------------------------------------------------------------------
    Chairman Johnson. It will be entered. Thank you.
    Dr. Gulfo. Thank you very much.
    Well, thank you very much for inviting me to participate in 
this hearing. As Chairman Johnson and my fellow witnesses 
eloquently explained, ``Right to Try'' is an especially 
important concern for patients and their families--particularly 
those for whom no approved options exist and who are terminally 
ill.
    The ``Right-to-Try'' debate also vividly illustrates 
several foundational principles contained in my research about 
the proper role of the FDA for the 21st-Century medical 
ecosystem, which apply to patients at all stages of their 
disease--not just terminally ill or those with no good choices.
    I have six brief points to make.
    First, the FDA has substituted its statutory mission to 
promote health with a new, misperceived duty to protect health, 
virtually at any cost. When terminal patients have no other 
options, what is the FDA protecting when it prohibits access to 
experimental treatment? The potential harms of the FDA's 
approach to protecting health are not limited to terminal 
patients, however. It has serious implications for all who 
suffer disease--and it is having a chilling effect on medical 
innovation.
    Second, benefit versus risk is a private health decision 
requiring the consideration of the individual's needs. The 
FDA's reliance on the benefit versus risk to the average 
patient is not an acceptable substitute. ``Right-to-Try'' 
patients seeking experimental therapies have a right to make 
these decisions, and patients and their doctors know far more 
about their specific circumstances than the FDA ever could. Of 
course, the same is true for all patients at all stages of 
disease.
    Third, the FDA's restated mission to protect, rather than 
to promote health has necessarily made it judge new drugs and 
devices not on safety and effectiveness--as specified in the 
law--but, rather, on clinical utility, benefit versus risk, and 
long-term outcomes, including survival. This truly stifles 
medical innovation by necessitating larger and larger and 
longer and longer trials that are extremely expensive. The net 
result is that many compounds, which could possibly help 
patients, are not even developed--and many that are obviously 
safe, effective, and could be helpful actually fail in these 
kinds of trials because the studies are improperly structured. 
It is simply impossible to control for all of the variables 
that modulate long-term outcomes.
    Fourth, increasingly the drugs that are developed, even by 
large companies, are geared toward narrow, niche, and orphan 
populations, where benefit versus risk is a low bar because 
there are no other products available. In 2014, 40 percent of 
all new drug approvals were for orphan claims. In 2015, the 
number jumps to 48 percent.
    Fifth, the very fact that we are here, today, to discuss 
this magnificent movement is testimony to the sophistication of 
today's medical ecosystem and marketplace. That which we may 
have needed the FDA to do just a few years ago is unnecessary 
today. With the Internet and rapid communications among 
patients and among physicians, knowledge is shared at a 
lightning pace. Patients have easy access to timely, high-
quality information that allows them to make excellent 
treatment decisions with their doctors. Today's reality could 
not have been anticipated 20-plus years ago, when the FDA began 
to move away from safety and effectiveness as the rightful 
basis for approval.
    My final point is that medical innovation is fragile 
because most of it occurs in small start-up companies. I have 
been responsible for the development and approval of products 
for prostate cancer, bladder cancer, and melanoma. As such, I 
have worked very closely with all three of the FDA centers--
drugs, biologics, and devices--in the development of novel 
products. The two things that start-up companies need the most 
are investment and regulatory certainty. Two companies that I 
ran no longer exist because the FDA changed the criteria for 
approval after we performed large studies that achieved all of 
the endpoints to which the FDA agreed before we started. In 
both circumstances, the FDA told us that safety and 
effectiveness were not enough--rather, clinical utility and 
evidence concerning long-term outcomes were required. This made 
investment disappear and rendered it impossible for us to 
launch the products and bring them to doctors and patients 
effectively after, ultimately, obtaining the approval with no 
new data in either case.
    In summary, the need for the ``Right-to-Try'' movement is 
emblematic of the FDA's ``protect health at all costs'' 
ideology. This has made the FDA assume a role that it was never 
intended to have--being arbiters of benefit versus risk, 
clinical utility, and long-term outcomes--and that has brought 
the FDA into private health decision-making. We need the FDA to 
return to its public health mission of promoting health by 
making approval decisions on the basis of safety and 
effectiveness.
    Thank you.
    Chairman Johnson. Thank you, Dr. Gulfo.
    Our final witness is Nancy Goodman. Ms. Goodman is the 
executive director of Kids v Cancer, which she founded in 
memory of her son, Jacob, a victim of pediatric cancer. Her 
group focuses on encouraging pediatric cancer research and 
pediatric, rare disease drug development. Ms. Goodman.

   TESTIMONY OF NANCY GOODMAN,\1\ EXECUTIVE DIRECTOR, KIDS V 
                             CANCER

    Ms. Goodman. Thank you, Chairman Johnson, Ranking Member 
Carper, and Members of the Committee for inviting me here 
today. I am honored to testify before you about how to connect 
patients, and children in particular, to new and potentially 
life-saving treatments.
---------------------------------------------------------------------------
    \1\ The prepared statement of Ms. Goodman appears in the Appendix 
on page 187.
---------------------------------------------------------------------------
    I am the executive director of Kids v Cancer. But, more 
importantly, I am the mother of Jacob, who was a beautiful, 
normal, loud, difficult, and brilliant little boy at 8 years 
old, who was diagnosed with brain cancer. Jacob suffered 2 
years of profound neurological impairments and unmanaged pain 
before he died at age 10.
    I have been working at Kids v Cancer now since Jacob died 7 
years ago, and, frankly, I do not even like speaking about him 
in public anymore. It is too raw. But, I brought a photo so 
that you can emotionally connect that way.
    The fact is that the drugs that were used to treat Jacob 
were 40 years old, and so for this reason, I launched Kids v 
Cancer to focus on changing the landscape of pediatric cancer 
research and to make it possible for children to get access to 
novel treatments.
    When Jacob was at the end stage of his cancer, I contacted 
eight separate companies, who were in the process of adult 
clinical trials for brain cancer drugs, to request access to 
these unapproved drugs for Jacob. Finding the right person to 
speak with was very difficult. Sometimes I contacted the CEO, 
others times the Chief Medical Officer (CMO) or the head of 
business development. For one company, it was a friend of my 
cousin. It was all very ad hoc. Of the eight companies, six did 
not ever respond to my request and made no determination. Two 
formally reviewed it and declined. And then, Jacob died.
    So the purpose of the ``Right-to-Try'' laws is to help 
patients get access to drugs that they would not otherwise get 
access to. That is a serious problem, and it is a goal that I 
share. But I think that we need to take a broader approach to 
this problem, and that has been the focus of Kids v Cancer.
    The fact is that children with cancer seek compassionate 
use access to drugs because there are almost no clinical trials 
available to them of these same unapproved drugs. Kids have to 
wait until drugs are approved, when they have cancer, before 
they get access to them in a clinical trial setting.
    So our first step at Kids v Cancer was to incentivize 
companies to develop drugs specifically for pediatric cancers 
and other pediatric rare diseases. In 2012, Congress passed the 
Creating Hope Act as part of the Prescription Drug User Fee Act 
(PDUFA). That has created nearly $800 million in incentives--at 
no cost to the taxpayer--for companies that get a new drug for 
pediatric rare disease approved by the FDA. The Creating Hope 
Act is doing what everyone hoped: stimulating companies to turn 
advances in science into treatments for pediatric cancer.
    The medical challenges are hard enough to overcome. Kids 
with cancer should not be disadvantaged by the lack of economic 
incentives for companies to develop drugs for tiny markets. The 
Creating Hope Act is up for renewal as part of the 21st Century 
Cures Act passed by the House, and I urge the Senate to pass it 
as well. And I am also proud to say that several of the drugs 
developed for Duchenne Muscular Dystrophy are benefited and 
supported financially by this legislation.
    Our second step with Kids v Cancer was to make new drugs 
being developed for adult cancers available for kids as well. 
In 2003, Congress passed the Pediatric Research Equity Act 
(PREA), which requires companies developing drugs for adults to 
conduct pediatric trials on such drugs where they could benefit 
children. The problem is that PREA has not kept up with the 
science. PREA only requires clinical trials if the children 
have the same ``indication''--that is, if children have the 
same kind of cancer. But now, we know that children do not get 
breast cancer or prostate cancer, but the mechanism in these 
cancers might be evident in pediatric cancers such as 
neuroblastoma or medulloblastoma--the type of brain cancer that 
my son had.
    We have proposed the Kids Innovative Drugs (KIDS) 
Initiative, a modest change to PREA that would update PREA to 
take into account these new scientific developments and ensure 
that drugs being developed for adults, which could have 
relevance to pediatric cancers, are tested on children as well. 
And I urge Congress to take up and pass the KIDS Initiative as 
soon as possible.
    We are exploring other ideas related to eligibility for 
trials as well. For example, why do almost all adult cancer 
trials use 18 years of age as the minimum age of eligibility 
without any discussion of the scientific or medical rationale 
for this cutoff?
    And that brings me to ``Right-to-Try'' laws. Yes, when it 
comes to seeking compassionate use access to unapproved drugs, 
the paperwork is onerous and the process is time-consuming. In 
response, Kids v Cancer is launching a ``Compassionate Use 
Navigator.'' We are working to better inform physicians on how 
to apply for compassionate use applications for their pediatric 
cancer patients with drug companies, the FDA, and their 
hospitals. We hope to provide point of contacts (POCs) for drug 
companies. We will post the FDAs new, expanded access form and 
we will work with the Institutional Review Boards (IRBs) of the 
hospitals where the children are treated. We will offer to 
counsel physicians, personally, on specific applications. In 
addition, we will collect information about the efforts and the 
outcomes of pediatric cancer compassionate use applications.
    The ``Compassionate Use Navigator'' is not the whole 
solution to the challenge of connecting children with cancer to 
new treatments. However, it will give parents of dying children 
more time with their kids. It will lessen the burden that their 
physicians have as they apply for compassionate use 
applications. And, we hope that it will encourage more 
physicians of kids with cancer to apply for compassionate use.
    In addition, Kids v Cancer supports the Andrea Sloan 
Compassionate Use Reform and Enhancement (CURE) Act to have 
drug companies make available to the public their policies on 
requests for compassionate use access, including the minimum 
criteria for approving requests and the time needed to make a 
decision. I urge the Senate to pass the Andrea Sloan CURE Act 
as part of the 21st Century Cures Act bill as well.
    But, from my personal experience and from working with 
dozens of other families, my sense is that the fundamental 
problem is not the FDA, but, rather, it is the incentive facing 
companies. Even though the FDA approves virtually all 
compassionate use applications that it receives, and even 
though it has indicated that an adverse reaction to a drug 
provided for compassionate use will not adversely affect a 
company's application for that drug's approval, companies 
remain risk averse--and they would rather not provide such 
drugs.
    But, even if one could change that, the results would be 
one-off anecdotes. We cannot afford to take an ad hoc approach 
to addressing pediatric cancers and pediatric rare diseases. We 
need to address the lack of access that seriously ill children 
have to novel, unapproved drugs not only by one-off 
compassionate use applications, but also in clinical trials. 
That is why initiatives such as the Creating Hope Act and the 
KIDS Initiative are so important--they give companies 
compelling economic reasons to create new drugs for kids and 
require them, where appropriate, to study pediatric uses of 
drugs that are being developed for adults. And that makes it 
easier for children to get access to drugs that they need to 
survive and live happier, healthier lives.
    We need more, however, than anecdotes. We need to change 
the landscape of pediatric cancer, and we need to ensure that 
children with rare diseases--life-threatening diseases--like 
Jacob, Diego, and Jordan, will have access to new and 
potentially life-saving treatments.
    Thank you.
    Chairman Johnson. Thank you, Nancy. Obviously, we are sorry 
for your loss.
    I do want to go right to you--and possibly Dr. Gulfo will 
also answer the question. Why is it that kids are not involved 
in clinical trails? I mean, how else do you test these things? 
You kind of asked the question. Do you not have the answer? Do 
you have suspicions? Or, maybe, we will push it over to Dr. 
Gulfo to answer that question.
    Ms. Goodman. I have my theories as well as to why companies 
are reluctant to undertake pediatric clinical development. 
First of all, the markets are small. It is very tough to 
develop a drug for a very rare disease, whether it be pediatric 
medulloblastoma or Duchenne's Muscular Dystrophy. Accrual rates 
are small. It is just challenging to put a drug together, and 
what that means is that the drug that you develop has to really 
be very good--and that is difficult.
    Chairman Johnson. So, is that a company decision or is that 
an FDA decision?
    Ms. Goodman. That is a business decision.
    Chairman Johnson. A business decision. So, there is no law 
that prevents children from being involved in clinical trials?
    Ms. Goodman. Well, that is correct. And for that reason, 
Congress passed the Creating Hope Act in 2012.
    The second reason that I think it is difficult is that 
companies maximize return on investment (ROI) over the long run 
through blockbuster drugs, and undertaking a pediatric trial is 
just a distraction for them if their goal--for which they have 
fiduciary responsibility to shareholders--is long-run return on 
investment. There are two laws that Congress has passed to 
address this problem. One is PREA, which I discussed, and the 
other is the Best Pharmaceuticals for Children Act (BPCA). The 
problem with PREA is that it does not apply to cancer. PREA 
requires companies to undertake certain pediatric trials and 
BPCA does provide a carrot, an incentive for companies to 
provide such trials.
    The challenge with BPCA is that it is entirely optional. 
Companies do not have to undertake these trials until the time 
that it makes the best business decision, the most sense for 
them, from a business perspective--which is the very end of 
their exclusivity period. So the National Organization for Rare 
Disorders (NORD) has estimated that that is 9\1/2\ years after 
approval of the drug for adult indications.
    Chairman Johnson. Dr. Gulfo, do you have anything to add to 
that?
    Dr. Gulfo. Yes, I will add. Cancer is a disease of the 
elderly. I mean, I teach cancer biology, and in order to 
accumulate the mutations that you need, you need to live a long 
time. So, I could not agree more with what Nancy said. So, it 
is a very small market for pediatric cancers. I think that that 
is a critically important thing. The other is that----
    Chairman Johnson. But, I just want to--because, one of the 
things in your testimony that you talked about was the 
innovation occurring in orphan diseases--limited use targeted. 
So, can you explain that for me?
    Dr. Gulfo. You took the words out of my mouth. As we 
continue to see companies going for these ultra niche claims, 
you are going to get to the population sizes for pediatric 
cancer. So, I actually think that we are going to see more of 
that.
    It is also very hard to do studies in children--very hard. 
The level of approvals that you need and the way that they are 
looked at--the way that they are looked at by the IRBs and 
things, it is very difficult because you have patients who are 
young and who could, potentially, live a very long time. So, it 
is harder to do. The market is not there. But, I agree with 
you, as this progress is pushed to more and more niche claims, 
you will see more and more childhood cancer studies.
    Chairman Johnson. With my questions, I am really going to 
be talking about the impediments. I think that I said earlier 
``legitimate''--I will say ``understandable'' problems. And I 
thought that your testimony was really pretty interesting. Part 
of the problem has been things, like congressional hearings, 
where we call members of the FDA up here, and if it has not 
been perfect--let us face it, life is full of risk. There is no 
such thing as a risk-free society. Members of Congress beat up 
on the FDA, and so, they become even more risk averse. So, I 
want to concentrate on that.
    Darcy, I do want to talk to you, though, because you talked 
about Ted Harada, but you mentioned 31 other people with that 
same exact drug. I am afraid that I know what the result was 
for the other 31. Did they pass?
    Ms. Olsen. To my knowledge, most of them actually did quite 
well.
    Chairman Johnson. So, they extended their life, but, I 
mean, have they all passed or----
    Ms. Olsen. No. No, they have not all passed.
    Chairman Johnson. OK. So we have more than one Lazarus 
then.
    Ms. Olsen. Yes, we do.
    Chairman Johnson. OK.
    Ms. Olsen. And so, for that particular treatment, they are 
in Phase II and III. They are doing all kinds of tests. But 
even someone like Ted--it is interesting, when you talk about 
wanting to get into clinical trials, Ted actually no longer 
qualifies for the next clinical trial. So, if the treatment 
wears off for him, he could still die from----
    Chairman Johnson. And he had access to this when?
    Ms. Olsen. About 7 years ago. But now, he is disqualified 
for the next----
    Chairman Johnson. I cannot imagine having a family member 
with ALS, knowing that 7 years ago somebody was surviving this, 
and not having access to it. I mean, Doctor, can you explain 
that? Can anybody explain? Again, I am not beating up on 
anybody. Please explain why something that could be that great 
a breakthrough, in a disease that we all know the end state of, 
is not made available. Why don't we just rush that? Please 
explain that.
    Dr. Gulfo. I cannot. I mean, are the trials still ongoing?
    Ms. Olsen. Yes.
    Dr. Gulfo. And he does not meet the entry criteria?
    Ms. Olsen. Yes.
    Dr. Gulfo. Right, so the entry criteria would need to be 
amended for him, and there is a mechanism in place for 
compassionate use in order to do that. But, I think that what 
people are asking here is, why do we need to go through so many 
hoops?
    Chairman Johnson. Ms. McLinn, you were talking about some 
drugs that really address--maybe not specifically your son's 
condition, but certainly would tie into a potential cure as 
well. Talk about that, the impediments to compassionate use. 
What are you finding to be the barriers?
    Ms. McLinn. Well, first of all, it does apply to Jordan, 
and it is kind of confusing, but the drug that is up for 
accelerated approval is a drug that 13 percent of boys with 
Duchenne are--they can benefit from this drug. The drug that 
Jordan needs is the very next one in the pipeline. In fact, it 
works the same way. The chemical backbone is the same. It is 
made by the same company. But we need approval for this one in 
order for Jordan's to move forward.
    Chairman Johnson. And it just addresses a different part of 
the gene? Is that the----
    Ms. McLinn. That is right.
    Chairman Johnson. So how many children--how many young 
boys--has this actually helped, to date, that have had--and how 
do they get access to it? Was it through a clinical trial? Was 
it through compassionate use? Was it a one-off?
    Ms. McLinn. Only through a clinical trial, not through 
compassionate use at all. So, there were 12 boys in the 
original trial for the drug that skips Exon 51, but they are 
now doing confirmatory trials. I cannot give you an exact 
number, I am sorry, but there are a lot more boys now who are 
receiving that drug.
    Chairman Johnson. Is it tens? Is it hundreds?
    Ms. McLinn. Hundreds? Hundreds.
    Chairman Johnson. Hundreds--but there are thousands with 
the disease.
    Ms. McLinn. Oh, yes. Yes. And the thing is that 1 in 3,500 
boys have Duchenne, and so, within that 1 in 3,500, 13 percent 
of boys can take this drug that I was speaking about--that the 
FDA is going to say yes or no to. And then, 8 percent of boys 
have the mutation that Jordan has and 8 percent have another 
mutation. And then, it just keeps going down the line there. 
But we are talking still about a lot of boys that are waiting 
on this treatment. It exists. And I will tell you that there 
are boys in Europe who are receiving the drug that Jordan 
needs. I know one of them, personally. He is jumping. That does 
not happen. Jordan cannot jump. This does not happen in this 
disease. These drugs are working. So, there are boys on this 
drug in Europe--just not here, yet.
    Chairman Johnson. So just real quickly, before I turn it 
over to Senator Carper, Dr. Gulfo, is there any rationale for 
not--I mean, again, is there any rationale for not allowing 
parents, and their children with this disease, to have access 
to this drug now?
    Dr. Gulfo. Well, there is rationale not to approve it, 
but--until there is proper evidence. Now, my whole testimony is 
about defining what ``proper'' is, OK? But having them have 
access--absolutely, they should have access to it. These 
children, as you said earlier, their diseases are terminal. It 
is terrible.
    Chairman Johnson. But, again, I do want to get the other--
the companies, themselves, are concerned about having an 
adverse result----
    Dr. Gulfo. Absolutely.
    Chairman Johnson [continuing]. In a nonclinical trial, or 
whatever, and having that affect the ability to--and we all 
ought to be concerned about that, because, if there is a drug 
that could be helpful and it gets, basically, kiboshed because 
of one of these compassionate use----
    Dr. Gulfo. Right. So, why would the company do all of the 
things that you need to do to show that it is safe to give to 
the child in that case, OK, when it is just pure risk? Because 
if an adverse event were to happen--and adverse events happen--
it will directly impact the development program and hurt many 
other children. I mean, you look back at it, this could really 
hurt the availability of this drug for many others. So, I could 
not agree more with what Nancy said--although the right things 
are said, it is not in practice. There was a company whose 
product was put on clinical hold because of an adverse event 
that happened in a compassionate use setting. The company's 
name is CytRx. And that just should not happen--and that sent 
the message throughout the whole industry that there the risks 
involved in trying to be good citizens.
    Chairman Johnson. ``Catch-22'' is running through my mind 
here, as we are talking about this.
    Dr. Gulfo. Yes.
    Chairman Johnson. Senator Carper.
    Senator Carper. Mr. Chairman, one concern that I think that 
we are hearing here, today, is about a lack of access to 
information about which programs are available to help people 
who are facing these life-threatening conditions. Since the FDA 
is not with us today to provide information on how their 
current processes work, I just want to ask unanimous consent 
for the fact sheet\1\ that I have here, on patient access to 
investigational therapies, from the FDA, be included in the 
hearing record.
---------------------------------------------------------------------------
    \1\ The fact sheet referenced by Senator Carper appears in the 
Appendix on page 209.
---------------------------------------------------------------------------
    Chairman Johnson. Without objection.
    Senator Carper. Thank you.
    Thank you all for joining us today. It is wonderful 
testimony. And, Ms. McLinn, I walked back into the anteroom, 
behind me, to get a cup of coffee, and there spread out on the 
floor were all kinds of toys and a little boy having a good 
time with Erica, who is 22 years of age. And, I had a nice chat 
with them, and he seems to be in good hands, with you and with 
Erica.
    I have a question for the whole panel that I am going to 
ask you to think about--and then I am going to come back and 
ask it at the end--but I am going to telegraph my pitch. Then I 
am going to ask Nancy Goodman a couple of questions, and then 
we will come back to the whole panel. But, the question that I 
want you all to think about, in the meantime, is that there 
seems to be a general consensus that there are some 
improvements that are needed in the current process that 
patients use to gain access to clinical trials and to 
unapproved drugs. And, here is what my ask is: What actions do 
you think that Congress should take--that Senator Johnson and 
myself and our colleagues should take--either through 
legislation or through partnering with the administration, to 
improve patient access to new and potentially life-saving 
treatments? So think about that. And, while you are thinking, I 
am going to ask Ms. Goodman a couple of questions.
    Ms. Goodman, in your testimony--I think that it was at the 
end of your testimony--you noted that you do not necessarily 
believe that the FDA is a fundamental problem for those trying 
to gain access to new treatments but, rather, there are 
challenges that companies who are developing these new 
treatments face when they receive requests from an individual 
patient. Could you just elaborate on this for a moment? Does 
the FDA assist individuals who are trying to contact companies 
and seeking medical treatments? Go ahead, please.
    Ms. Goodman. So, I think that it is best that I just answer 
this from my personal experience. I am not an FDA expert nor 
have I worked at a drug company.
    When I was applying for compassionate use access for Jacob, 
the two companies who declined our requests stated that they 
had not started drug development in children and that they were 
just concerned about what the implications of the pediatric 
trial would be.
    At that time, I was like any other parent with a terminally 
ill kid. I did not know a lot of people at the FDA. I did not 
know how to contact people. Since that time, however, I have 
formed Kids v Cancer, and I am grateful that I do have an 
opportunity to speak with people at the FDA. And, I have to say 
that, though the systems could be better developed, I have 
found officials at the FDA to be very interested in helping 
families gain access to compassionate use access to drugs.
    For example, a year and a half ago, there was a little boy, 
Josh Hardy, who was seeking access to an adenovirus drug on a 
compassionate use access basis. It was a matter of life and 
death for him. The entire pediatric cancer community rallied 
around him. There was social media and traditional media. Kids 
v Cancer was not the leader, but we did participate. And, 
ultimately, my understanding is that the reason that he got 
access to the drug is that an official at the FDA heard a story 
on the Cable News Network (CNN) and she called the CEO of the 
relevant company and said, ``What is going on?'' And she 
offered to craft, with the CEO, a solution.
    So, my personal experience is that they are really very 
interested in helping--and we need to give them the support 
and, maybe, the legislative authority to do more. To that 
extent, I support the Andrea Sloan CURE Act, which would do 
that.
    Senator Carper. All right. Thank you.
    Changing gears just a little bit here, I think that you 
launched something called the ``Compassionate Use Navigator'' 
through Kids v Cancer.
    Ms. Goodman. That is correct.
    Senator Carper. Could you just walk us briefly through how 
that navigator works for patients and for their families?
    Ms. Goodman. Absolutely. So, the ``Compassionate Use 
Navigator'' will be launched in April, and so, I want to talk 
to you about our plans. The first steps that we are building 
now on our website are a lot of information for physicians, so 
that they just have an easier time understanding what it is 
that they need to do to undertake a compassionate use 
application. It is very difficult for physicians, even, to 
figure out the necessary steps right now. We will be talking 
with them first about, for example, how to find a point of 
contact in the company to reach. If they have difficulty doing 
that or if they would like our assistance, we will take that 
responsibility, and we will do our best. We will help them 
write the letter to the company. We have an excellent person 
who we just brought on board, Elena Gerasimov, who will be 
reviewing the letters and providing support. Doctors may be 
wonderful scientists and weak writers. That should not be a 
reason that a kid does not get a drug.
    Then, we will be working with the physicians as they 
approach the FDA. We will be posting the FDA's short form on 
our website and working with the physician--and we intend to 
use that form when we approach the FDA.
    And then, third, the IRBs of the hospitals need to be 
involved. Sometimes, the IRBs are slow. I have found that 
whenever I call the IRB officials and leave a nice voicemail on 
their machine, they decide within about 30 minutes.
    So, that is what we intend to do, and then we intend to 
document all of our experiences, so that the whole community 
that is interested in this issue will have some information 
about what is happening with respect to compassionate use 
applications in the pediatric sphere.
    Senator Carper. All right. Well, thank you. I am----
    Ms. Olsen. Senator Carper, I do not mean to be rude and 
interrupt, but could I just shed a slightly different 
perspective on compassionate use?
    Senator Carper. Sure, please.
    Ms. Olsen. I really appreciate what Nancy is trying to do 
with compassionate use, because it speaks to a fundamental 
problem, which is that the FDA says that they approve 99 
percent of requests, but that is because you basically have to 
get to the top of the Himalayas to be able to present your 
request. The truth is that less than 1 percent of patients who 
are terminally ill in this country will ever get through the 
compassionate use program.
    There is a principle at stake as well, which is that you 
should not have to beg the Federal Government for permission to 
save your life. This is America, and compassion should be the 
rule, not the exception to the rule. And, in working on my 
book, we interviewed Janet Woodcock, is head of Center for Drug 
Evaluation and Research (CDER) and is acting director of CDER's 
Office of New Drugs (OND), and asked this question to the FDA: 
``Do you think that it would be a good thing if tens of 
thousands of patients with terminal illnesses were able to 
access these investigational medicines that we are talking 
about? '' And, the first words out of her mouth were, ``It 
would be another burden on the health care system.''
    A little bit of a different perspective. In Europe, they 
have tackled this. For 25 years, they have had compassionate 
use. They have it writ large. The problem in America is that 
companies do not have an incentive to participate. They have 
overcome that, in Europe, by granting provisional access, so 
say for these DMD drugs where they have trials going, they open 
it up to all of the kids, and then they monitor, in real time, 
what is going on so that they get better data. The kids get 
access right away, the companies can charge a nominal fee, and 
they have an incentive to participate because the regulatory 
steps become more clear.
    So there is a way to do this. We are 25 years behind.
    Senator Carper. Thank you.
    Just very briefly, Ms. Goodman, would you like to respond 
to that--to what Darcy has said? Just very briefly.
    Ms. Goodman. So I do not have experience with the European 
system, and I will defer to Darcy on that. And, my only 
question is: What environment could we create where companies 
truly would be comfortable if they put a kid on a trial and the 
child died? Even if a company did not have to report that 
adverse event to the FDA, it gets reported in the newspapers. I 
think that the company would still be concerned. So, I do not 
know as much about this piece of it as Darcy does, but I am not 
sure that I understand, from the company's perspective, how 
this really takes care of their concerns. And so, that is why, 
from my vantage point--at Kids v Cancer we have focused, first, 
on process with respect to compassionate use--it is only part 
of the answer--and, second, getting companies to start 
developing more interesting drugs in the first place. I just 
want to give an example.
    In the cancer space, there is a new kind of drug called PD-
1 inhibitor drugs. It looks like these might be curative for 
patients with melanoma. It is very exciting. And, if you do 
combination therapies, the response rates are 70 percent in 
adult trials. It is really very exciting.
    There are 220 PD-1 inhibitor trials that are listed on 
``clinicaltrials.gov.'' This morning, I went to take a look at 
how many are available for kids right now that are open for 
enrollment. Three. How many are combination therapies? None.
    So, I just want to provide this as an example of how, from 
my perspective, I think, the goal is to create reasons for 
companies to start pediatric trials--get the denominator as big 
as possible.
    Senator Carper. All right. My time has expired, and maybe 
we will have a second round, and I can ask----
    Chairman Johnson. We will.
    Senator Carper [continuing]. That question that I 
telegraphed a few minutes ago. Thank you.
    Chairman Johnson. Senator Ernst.

               OPENING STATEMENT OF SENATOR ERNST

    Senator Ernst. Thank you, Mr. Chairman. And, thank you to 
all of you for--many of you traveled a long ways to get here. 
We are glad to have you with us today.
    Earlier this morning, I hopped on Facebook--and I was 
checking it out--and this is a picture of one of my best 
friend's sons, and he was diagnosed with leukemia several years 
ago. And he has just taken the last of his chemotherapy pills. 
He has gone through quite an extensive treatment over the past 
number of years. And so after church on Sunday, we are all 
going to celebrate the fact that he is still with us. 
Fortunately, his family was able to use approved drugs and 
treatments. There are many families that do not have access to 
those drugs. So, we are lucky. And there are a lot of families 
that are not as lucky.
    And so, this is an important topic--and I know all of you 
have struggled with this issue, and we will continue to 
struggle with this issue, I think, as we work through this 
particular situation.
    Diego, it is great to have you here.
    Mr. Morris. Thank you.
    Senator Ernst. It is great to have you here.
    So we have spent a lot of time visiting about this, and I 
was astounded when I got the statistic--it came from the Energy 
and Commerce Committee during the House's consideration of the 
21st Century Cures Act, and that statistic is that 95 percent 
of rare diseases have no recognized treatment--so, Nancy, what 
you were speaking on just a little bit ago. And, I think that 
we all agree that gaining access to treatments is important and 
discovering those treatments is important. And, I do hope that 
our colleagues on the Senate Health, Education, Labor and 
Pensions (HELP) Committee can continue working on this and 
parallel medical innovation legislation that helps us to 
streamline processes, so that we can cut some of the red tape 
to get to these cures and to make sure that we are not 
hampering innovation that is available out there.
    For this panel, it is an important time, as we have seen 
development of new technology, and drugs that can cure 
illnesses and improve the quality of life. And, although, 
today, we have spent a lot of time talking about the FDA's 
regulatory processes, especially for children, we also need to 
think ahead as well. And so, I would like to just step out 
there a little bit ahead. When we do have those life-saving 
treatments in place and they are FDA-approved, can any of you 
speak to ways that we can break down the regulatory burdens 
that exist at the Centers for Medicare and Medicaid Services 
(CMS) that affect how quickly our Medicare recipients can start 
receiving these therapies? There seem to be barriers, not just 
for our youngsters, but also for those toward the end of life. 
Is there anyone that can speak to that? Dr. Gulfo, you look 
like you are thinking very hard about it.
    Dr. Gulfo. Yes, I am. I am not an expert in that area, but 
to my understanding, Medicare patients cannot be denied 
approved drugs for approved claims.
    Senator Ernst. For approved----
    Dr. Gulfo. So, the issue is the approved claims part of it. 
Now, in pediatric cancers, there is a tremendous amount of off-
label use. I think that it is as high as 60 percent, because 
the drugs get approved for adults--like PD-1 inhibitors are 
approved for adults--and then it will be tried in pediatrics 
off-label. So, I think that that is something that you could 
look at--that, if approved drugs are used on-claim, they cannot 
be denied. It is when they are used off-claim, I think, that 
that could be an issue to look at.
    Senator Ernst. Very good. Any other thoughts? I know 
probably not your specific area, but----
    Ms. Goodman. Senator Ernst, I think that that is an 
excellent question, and, again, with respect to children, one 
challenge is that, when drugs are approved, that is when 
pediatric oncologists have the opportunity to undertake 
pediatric trials. And then, the problem is that, because these 
are rare-disease drugs, the way that they get developed is by 
companies looking forward to the premium pricing of these 
drugs--and they price them very high. And then, pediatric 
oncologists cannot afford to buy them to undertake pediatric 
studies.
    So, again, they have to wait until companies decide to 
provide them a clinical supply of the drug for free--and that 
decision occurs when it is the right business time for that 
company--not when it is the right time for the kids. That is 
why we only have three 
PD-1 inhibitor trials for all pediatric cancers at this time. 
It is not the right business time for these companies to 
undertake these trials. And, again, that is why we are asking 
Congress to take up the KIDS Initiative to reform PREA, so that 
there are certain times when, if it is appropriate, companies 
should undertake pediatric trials.
    Senator Ernst. I appreciate that very much, and, again, my 
friend's son and my friend's family, they have been through a 
lot of stress already with approved drugs. I cannot imagine the 
stressors on families as they try and get into clinical trials 
and so forth. So, I appreciate it. I think that we need to have 
further discussion on how we streamline the process and make it 
easier for folks to get those life-saving drugs.
    So, thank you, Mr. Chairman, very much. I appreciate it. 
And, thank you to all of our witnesses.
    Chairman Johnson. Thank you, Senator Ernst.
    As soon as Senator Ayotte comes back, I will turn it over 
to her, but I want to keep going back to the impediments. There 
was one--Nancy, you talked about, in one instance, you 
contacted the FDA, and that official at the FDA contacted the 
CEO and crafted a solution. As briefly as possible, what was 
the crafted solution? How did they break through? Because I 
would think that that would be sort of the model of what we are 
trying to work----
    Ms. Goodman. Yes.
    Chairman Johnson. I mean, it should not take a gargantuan 
public relations campaign to have an official at the FDA say, 
``Let us break through.'' So what was the key? In other words, 
what did the FDA allow a company to do, so that it was not risk 
averse to actually involve that person in a trial?
    Ms. Goodman. The FDA suggested what is called an ``expanded 
access trial'' for that company. So, the FDA and the company 
crafted a new trial with 20 or 30 children--but it would be a 
clinical trial. The company would not only be obligated to 
report adverse outcomes, but could also collect efficacy data 
and submit that to the FDA, too. And because it is a trial, the 
company has certain additional controls over how the drug is 
administered and who administers it. So, it was a solution that 
was good for both.
    And, I think that the question of when we use expanded-
access trials is really important--and also when companies 
should be required to inform the FDA that someone has come and 
asked for the drug. The fact is that companies do not have to 
report that information to anyone right now, and so, in this 
particular case, 300 people had asked the company for this 
drug. It is a drug to fight the adenovirus and other viral 
infections. And so, in this case, the drug really will keep 
people alive or not based on whether it is provided. And the 
FDA did not know.
    Chairman Johnson. Well, I am quite sympathetic with private 
sector businesses having kind of an adverse reaction to what 
the FDA can do to them--and trial lawyers as well. There is an 
awful lot of incentive--or disincentive for doing this, and 
that is what we are trying to break through. What are the 
impediments?
    Darcy, do you have a comment?
    Ms. Olsen. Yes, I would just like to add--I mean, I feel 
like some of this conversation about the FDA is like, saying, 
``Let us put some fresh paint on an old jalopy,'' and what we 
need to be doing is getting people to the moon. And to Senator 
Ernst's question, for the elderly, but also for pediatric 
medicines, I mean, the simplest straight line is to adopt 
reciprocity with Europe. I mean, 30 percent of the advances are 
out overseas. It is--in Diego's case, that saved his life. The 
medicine that saved his life--Europe's like gold prize for the 
greatest advance in childhood medicine--and it has been over 20 
years and it is still not approved here. This DMD drug is 
approved in Europe.
    I mean, for goodness' sake, why is our market just this? 
Why do we not open it up to these countries? This child, that 
you were talking about, would have had access to those things. 
They are proven. They are proven, they are tested, and they are 
on the market.
    So, that is what we need to be talking about. Let us get to 
the moon. That is your real answer.
    Chairman Johnson. To me, that sounds like a no-brainer. Let 
us take the first steps here that are just so incredibly common 
sense. What has been the resistance to it, though? I mean, it 
is just so common sense. Why have we not done that?
    Ms. Olsen. Well, that is a good question. I do not know all 
of the politics. There is a bill, S. 2388, right now that 
allows for reciprocal improvement--or, excuse me--approval.
    Chairman Johnson. Who is opposed to it? Literally, because 
I cannot imagine any human being taking a look at any one of 
these instances--whether it is Jacob, whether it is Jordan, or 
whether it is Diego--and not doing everything possible, on an 
individual basis. And yet, then collectively where is the 
resistance? Where is it coming from?
    Ms. Olsen. My understanding is that the FDA wants total 
control over all of the drugs in the U.S. market. And, they 
want to do the regulatory process from beginning to end--even 
if that means an additional 15 or 20 years of studies. I think 
that that is where the problem is. And, when the lead official 
tells you that access to these experimental medicines would be 
another burden on the health care system, I think that that 
gives you some insight, that sometimes----
    Chairman Johnson. Are they talking about costs? That it is 
going to cost us money to save people's lives? Is that what you 
take from that comment?
    Ms. Olsen. Yes, it is about cost and systems instead of 
patients. And so, I think that that is the short answer. That 
is where the opposition comes from--but Congress can fix that.
    Chairman Johnson. Dr. Gulfo, I want you to comment on that, 
but also I want to go to your sixth point--because it is true. 
Innovation is fragile.
    Dr. Gulfo. Yes.
    Chairman Johnson. Where are the breakthroughs going to come 
from? Listen, I believe in the National Institutes of Health 
(NIH) and the Center for Disease Control (CDC). Government can 
fund basic science and research. But, I also believe in the 
private sector and in innovators--individuals coming up with an 
idea. I supplied packaging in the medical device industry. 
There are so many practicing surgeons or physicians that have a 
concept and the idea for a medical device, and it is that 
little moment of enlightenment, ``Oh, if I could only do 
this.''
    So, we have to foster that. We cannot crush that. So, just 
speak to what I was just talking about. Where is that 
impediment? Where is the resistance to something that is so 
common sense--to take those first steps? And then, talk a 
little bit more about the fragility of innovation.
    Dr. Gulfo. Sure. So, I could not agree more with what Darcy 
is saying about reciprocity. The FDA is afraid of companies 
shopping for the least regulatory-burdensome market and that 
that would be automatically approved in the States. So, it is 
nationalistic thinking there.
    Chairman Johnson. Well, is it nationalistic or is it the 
agency wanting control?
    Dr. Gulfo. Yes, I am sorry. Agency.
    Chairman Johnson. OK.
    Dr. Gulfo. The other thing that I will say is, I could not 
agree more with Darcy as well--look, I believe in a strong FDA. 
I think that we need an FDA. But, it takes 100 hours for a 
compassionate use process to be undertaken by a doctor. So the 
FDA came out and said that they are going to reduce that to----
    Chairman Johnson. 100 hours of a doctor's time.
    Dr. Gulfo. Yes, 100 hours.
    Chairman Johnson. Of the practicing physician trying to 
save lives--it is taking him 100 hours.
    Dr. Gulfo. Right. So, the FDA said, ``We will have a new 
policy; they can do it in 45 minutes.'' It has not been 
implemented. So, if the FDA were so helpful in the example that 
Nancy gave, and you have the guidance document written, why is 
it not implemented? I know that you know a lot about that.
    Ms. Olsen. It has been a year, so it has taken them a long 
time to do that. But, I think the whole conversation about 
compassionate use is slightly misguided because it is not just 
Laura's son that needs compassionate use. It is every single 
boy who has DMD. Right? We need to bring these things to 
market. We need to get them to market faster. That is what 
Europe's compassionate use system essentially is. It is called 
``provisional access.'' So, once they go through safety testing 
and a little bit of efficacy testing, these drugs would be 
available in the United States. And because people could buy 
them, the companies would not be trying to give them away after 
spending $1.5 billion to develop them. So, they have more 
incentive to participate. And that is the problem in this 
country.
    Chairman Johnson. And your point also was just one of 
freedom.
    Ms. Olsen. Yes.
    Chairman Johnson. We are in America. This should be the 
land of the free and the home of the brave. It should be up to 
patients--not the government to tell you what you can and 
cannot do--past a certain threshold. And, again, with what you 
are trying to do, you certainly have certain threshold levels 
of approval, either within the FDA clinical trial process or in 
terms of approval overseas.
    Ms. Olsen. Correct, yes. So there is still basic safety 
with the ``Right to Try,'' and overseas it is safety plus a 
little bit more, but there--instead of getting access at 15 
years, you might get it at two, three, or four, when you are 
getting some results, because people--they do not have time to 
wait. And I think that Laura's point on that--we cannot wait 
until Jordan is 18. He needs that, today, to live a full life. 
And there are solutions. A lot of these things are available 
overseas, and so, we need to think a little bit bigger than 
just making it a little bit easier for people to apply for 
compassionate use. Compassionate use should be the rule in this 
country. Do we want to be a country where we have the right to 
die when we are terminally ill? Fantastic. But what about those 
who want to fight?
    Chairman Johnson. Dr. Gulfo, as I recall--as I interpreted 
your testimony--the FDA was really set up almost with the 
presumption of approval, correct? I mean, if it is safe, 
basically, we are going to presume to allow doctors--
physicians, who are pretty highly trained, are concerned about 
their patients, and are probably more concerned about an 
individual patient than somebody here in Washington, D.C.--that 
they have that ability to do so. Can you speak to that?
    Dr. Gulfo. First of all, I am flattered that you read my 
written testimony because that is exactly right. The law is set 
up for the FDA to not approve if--not approve if. So, the bias 
in the writing of the law was that we want innovative drugs, we 
want to promote health, and we want to----
    Chairman Johnson. But over time----
    Dr. Gulfo. So, it should be a reason not to--not a reason 
to promote. It should be a reason not to. It is like, when I 
played baseball, I was pretty good. My father said, ``You are 
pretty good, but a pretty good hitter you go up to the plate 
saying, `I will swing if it is a strike.' And, a really good 
hitter goes up to the plate and says, `I am swinging unless it 
is a ball.' '' And that is what we want. We want the FDA 
swinging unless it is a ball. OK? And so that is what we want.
    Now, for little companies, back to your point--yes, the 
engines of innovation. I do not run little companies anymore, 
but to add something that can just be pure risk, to do 
something that could put us on clinical hold, and to want to 
give a dying child something if we did not do all the kinds of 
work we are supposed to do to do that and did not get the right 
IRB approval and the whole bit, we are setting ourselves up for 
ruining the company--ruining the prospects of the product going 
forward. So there are tremendous disincentives to companies to 
try to help where they can.
    Chairman Johnson. Because we have not mentioned this yet, 
but, right now, we are talking about the disincentives for, 
maybe, having the FDA, really stop the approval process. We 
have not even talked about the trial lawyers and the liability 
issues here as well, which, Laura, I want to kind of go back to 
you. I would imagine that you would sign any waiver of 
liability toward any company, correct?
    Ms. McLinn. Of course I would.
    Chairman Johnson. I would. Have you ever met a family 
member who would not?
    Ms. McLinn. No.
    Chairman Johnson. So the liability issue should be really 
off of the table, correct?
    Ms. McLinn. Correct.
    Chairman Johnson. By the way, is it largely? Does it really 
end--no, it is not.
    Dr. Gulfo. No.
    Chairman Johnson. OK. Nancy, I know that you wanted to 
weigh in on one of these issues.
    Ms. Goodman. Well, thank you, Senator. Look, I think that 
you are asking exactly the right questions. And the piece of it 
that, again, I want to focus on is that drug development takes 
a long time. It takes 10 years or more. And, the question that 
we have been asking is: Once we know that there is an 
interesting drug under development, how do we get more patients 
that are dying on that drug? That is a very important question.
    But, I want to go back to the first question, which is: How 
do we get companies to develop more exciting drugs? The 
Creating Hope Act, which we put together, creates financial 
incentives for companies to do that. It is going to markup on 
March 9 in the HELP Committee, and I hope the Senate will 
consider reauthorizing it on a permanent basis. Companies need 
long-term assurances that this incentive will be there for all 
10 years of its development. I am concerned that a short 
renewal period will not create the proper incentive.
    Chairman Johnson. The free market provides an awful lot of 
incentives. Doctors, themselves, want to create the cures. I am 
not sure government is going to be able to dictate a proper 
incentive better than what the free market actually does. So, 
from my standpoint, how do we get rid of the impediments? How 
do we reduce the disincentives? Because I think that there are 
plenty of incentives, just from a standpoint of humanity and 
compassion and doctors trying to cure disease and stuff. That 
is a huge incentive. And the question is--it does not take 10 
years to really develop a drug. You can have a breakthrough. 
You can come up with the chemistry of it. The reason that it 
takes 10 years is the approval process and, now, the $2.6 
billion is the latest cost.
    Dr. Gulfo. It is really about what I said about shifting 
the standard. When the standard is truly safety--we do not want 
to give toxic stuff, right? And, we want to know how you can 
administer the drug safely. And, effectiveness. Effectiveness 
should be the activity of the drug--the pharmacodynamic 
activity. The FDA has taken that to unrealistic endpoints. They 
want to see survival endpoints. They want to see these 
endpoints that take tremendous trials and tremendously long 
follow-up. And to me, that is the real problem. Again, I could 
not agree more with Darcy. I wrote an editorial about this. The 
answer to ``Right to Try'' is getting drugs approved faster. 
That is the answer.
    Chairman Johnson. So, let me ask, in terms of a company, is 
there any legitimate disincentive, other than having an adverse 
effect and having the FDA just say, ``OK, this drug is 
ended,''--or the trial lawyers? I mean, if you are doing a 
scientific study and you are doing a clinical trial and all of 
a sudden you are starting to provide this drug for people that 
are not in this very controlled study, is there a legitimate 
scientific concern about harming the results of the trial, in 
terms of the information that you are getting?
    Dr. Gulfo. Actually, the answer is that there is no benefit 
to doing it, because I cannot dose enough of these one-off 
patients to get a claim in that. Right? So you need large--to 
do a study, you need a lot of patients. OK? So the companies 
pick breast cancer or they pick prostate cancer--a great 
example. However, it might be a drug that is focused on a 
particular mutation where the same mutation is shared with some 
childhood cancers. What would be great--and I would love to see 
us get there--is if we are not approving drugs on the basis of 
cancer type, but we are approving drugs on the basis of the 
genotype of the cancer. And then, they could be instantly 
applied in other places. Then you could do a basket approval. 
You could bring all sorts of patients, age groups, and certain 
disease types into one trial. FDA is not there yet.
    Chairman Johnson. Well, I will turn it over to Senator 
Ayotte, but, first, would it be advantageous for clinical 
trials, for gathering information, and for approving safety and 
efficacy, if you just had more of these drugs available to 
people who want to use them? Or would that actually harm your 
ability to get information? Do you know what I am asking?
    Dr. Gulfo. The more that are available, the more that are 
approved, and the more that are in the hands of the doctors, 
the more discoveries that we get.
    Chairman Johnson. OK.
    Dr. Gulfo. The more where a doctor observes, ``Wow, when I 
give it to this patient''----
    Chairman Johnson. That would be my assumption, so OK, good.
    Dr. Gulfo. Yes.
    Chairman Johnson. Senator Ayotte, if you are ready.

              OPENING STATEMENT OF SENATOR AYOTTE

    Senator Ayotte. Thank you. I want to thank you, Chairman 
and Ranking Member. And, certainly, thank all of you for being 
here today. Before I left, I got to hear almost all of your 
testimony, and it was very compelling and so important.
    I have had constituents that--there was a young girl who 
passed away from a rare form of cancer, and her family came to 
me and certainly wanted the opportunity for compassionate use 
through the FDA--and really they got the runaround. It was very 
difficult. And, as you think about the difficulty that--all of 
the other things that families are dealing with under those 
circumstances.
    I know that there has been a lot of discussion today about 
the incredible work that you are doing, Ms. Goodman, trying to 
help families navigate this issue. But, it seems to me that the 
FDA--does anyone have a sense of when the FDA--they put out 
this draft a year ago to make this process more simplified and 
to make it easier for families, because there is such a 
runaround. And you are all so engaged in this. Have you heard 
anything from the FDA--Doctor, I saw you shake your head--about 
what they are waiting for? This is a year. We are talking about 
families that are struggling and every minute matters to them. 
And so, it is really troubling to me that they have not issued 
final guidance. Does anyone have a sense--have we heard 
anything on this?
    Dr. Gulfo. I know just what you are talking about. It was 
an effort, to reduce the 100 hours that it takes to put in an 
application, into a 45-minute process. And we are all waiting 
with you. It is written. I believe that a draft guidance 
document was written, and it has not been implemented. But, I 
think that Darcy knows more about that than I do.
    Ms. Olsen. I just know who you can talk to at the FDA--who 
is in charge of that. It is Dr. Peter Lurie. They promised over 
a year ago that they were going to make it a 45-minute process, 
and, I think that, for people who work in this field, it is not 
a big surprise that they have not finished that form.
    But I will say this: Even once that form is finished, it is 
not going to solve all of the problems. It will be a little bit 
easier, but, we attached it to my testimony--just issued an 
investigational report on the compassionate use process which 
really goes through the disincentives that the companies face 
and why they do not participate. And that will not change just 
with a shorter form. That is going to require some of the 
bigger reforms, like they have done in Europe. And, of course, 
reciprocity would be very helpful to get drugs here today.
    Senator Ayotte. I hope----
    Chairman Johnson. Really quick, Senator Ayotte, just so 
you----
    Senator Ayotte. Yes, I hope that we can follow up on that.
    Chairman Johnson. No, we have already--as part of this 
hearing, we sent out an oversight letter asking that specific 
question, along with other things. So, we will certainly--as 
soon as we get feedback from the FDA, we will give you the 
answer.
    Senator Ayotte. Good. I hope so, because this is really 
just awful. I appreciate what you have said today, and, Ms. 
Olsen, you really put it well. How are we taking this decision-
making away from families who are in a position where it is a 
life-or-death situation for them? And what are we trying to 
protect them from by not allowing them to make their own 
decisions? It is really hard to understand. I understand if it 
is not a situation where there is a life-or-death situation, 
but let us face it, if we all put ourselves in the shoes--if we 
put ourselves in your shoes, Ms. McLinn--it is hard to do, but, 
I know that, as a mother, I would want to fight and do 
everything that I could, and I would not want to leave any 
stone unturned when it came to my son. And, I happen to have an 
8-year-old, Jacob, by the way, and so I am hoping that we can 
take this issue up in this Committee, because I think that you 
deserve more focus on research--that is critical, and that is 
something that obviously I am very supportive of. We need more 
focus, though, on the things that we know are working--to give 
you access to--and the fact that, Diego, you had to move and 
your family had to move overseas to get drugs that have been 
available overseas--you are right, not every family can do 
that. But, you should not have to do that.
    So, we have to be able to do something about it. Honestly, 
it is common sense and we need the FDA to also start putting 
themselves in the shoes of the people who they are there to 
serve. The FDA is there to serve all of us. The FDA is there to 
make sure that people can be protected, but not from 
themselves. It is about letting them make decisions. I hope 
that we can come up with a really strong, bipartisan consensus 
with some of the feedback that you have given us today.
    And, I want to thank all of you for coming here. This has 
been incredibly moving, and you have really distilled this down 
to some concrete actions that we can take as Senators that can 
make a difference. So, we look forward to working with you on 
all of that, and we are so glad that you have come here today. 
Thank you.
    Chairman Johnson. Thank you, Senator Ayotte. Senator 
Carper.
    Senator Carper. Given what Senator Ayotte just said, I may 
have missed this, but, while you were out of the room, I have 
been in and out of the room, too. We are trying to do the rest 
of our schedules and be here to listen to the testimony. But, 
the question that I asked--remember, I telegraphed a question. 
I said that I am going to telegraph my pitch. The question that 
I was going to ask was: What do you think that those of us who 
are sitting over here on this side of the dais can do, either 
through legislation or, maybe, through partnering with the 
Administration, to improve patient access to new treatments or 
to new therapies? And, if you could just briefly address that 
for me, that will pretty much be it. Do you want to go first, 
Ms. Olsen?
    Ms. Olsen. Thank you, and I----
    Senator Carper. Give us a short to-do list--one thing, 
maybe, one thing. And we will ask everybody to just give us one 
good idea.
    Ms. Olsen. OK. Reciprocity, which we talked about, with 
European countries, and since I prepared for your first 
question, which was 2----
    Senator Carper. Go ahead.
    Ms. Olsen. For these drugs that are being developed for 
people with life-threatening, terminal illnesses, we need 
provisional access, which means that, as soon as they know that 
something is working, they let people go ahead and put it on 
the market and study it, until it gets the final green light. 
That will solve the problem of companies not participating.
    Senator Carper. OK. Thank you.
    Ms. McLinn. Thank you for asking this question. I wanted to 
come here today and tell you guys exactly what you could do to 
help Jordan--to help my son--and that is all I have thought 
about since I was invited to come here to testify. And, the 
truth is that I do not know and I cannot give you an exact 
answer, and that is really hard for me to say because I am used 
to problems just having--I am a math teacher. I am used to a 
problem just having a cut-and-dry answer.
    Senator Carper. My guess is that you are a pretty good 
teacher.
    Ms. McLinn. What is that?
    Senator Carper. My guess is that you are a pretty good 
teacher.
    Ms. McLinn. Thank you. But, yesterday, I had the 
opportunity, and I was actually in Speaker Ryan's office, and I 
spoke with his chief of staff. I told him, ``In 2012, when the 
President signed FDASIA, that had a lot of support.'' And, I 
asked him, ``Can you get the President of the United States to 
go to the Advisory Committee (AdCom) meeting and say, `Hey, I 
signed this. The Congress said that we want you to do this. Can 
you do this?' '' And I know that that is an outlandish request, 
but you asked what you can do. So, I would like to see a 
physical presence by Congress at an AdCom meeting, or with the 
FDA. I would like to see you speak with them, directly, and 
say, ``We want you to use the tools that we have already given 
you. This is legislation that already exists.'' And, I want 
someone from Congress to stand up and say, ``Will you please do 
this?''
    Senator Carper. All right. Good. Thank you.
    Diego, before you speak, let me just say that we have a lot 
of witnesses before this panel. You are one of the youngest, 
and I want to say that you are one of the best. You did a great 
job.
    Mr. Morris. Thank you.
    Senator Carper. Would you like to answer my question, 
please? Give us some good advice, please, before we adjourn.
    Mr. Morris. I think that the compassionate use process 
needs to be expedited. Particularly, when I was going through 
treatment, the drug that I had in London needed to be taken 
while I was doing the chemotherapy. And, the compassionate use 
program would have taken far too long, so we did not even apply 
because we were advised that it would take too long. The 
process needs to be expedited, because, in some cases, it needs 
to be faster.
    Senator Carper. All right. Thank you, Diego. Dr. Gulfo.
    Dr. Gulfo. Yes, three things, I think, and I appreciate the 
question.
    Senator Carper. Sure.
    Dr. Gulfo. First, I think that the FDA has to feel loved. 
We all need to love the FDA, and not do fire-alarm, knee-jerk 
oversight, if you will, when things do not go well. So, I think 
that that does, as I wrote in my----
    Senator Carper. I call those ``gotcha'' hearings.
    Dr. Gulfo. Yes, OK. And so, even when the FDA gets it 
right--look at the case of Avandia. They still get beaten up 
for it, and that is just wrong.
    Senator Carper. That is a good point.
    Dr. Gulfo. And, that makes them retrench, and it is 
terrible.
    That, combined with really letting them know that we want 
them to promote health, OK? Sure, protecting is a part of 
promoting, but we want them to promote health. We do not expect 
them to guarantee absolute safety for all patients and all 
drugs.
    And, then, my third wish would be to get the FDA back to 
focusing on safety and effectiveness--not on these other 
outcomes.
    Now, in my written testimony, I have a proposal for that. 
You can have four categories of the nature of the evidence, and 
one of the categories could be those longer-term outcomes. Fine 
with me. But, I think that if you get the FDA back to promoting 
and focusing on safety and effectiveness, and not getting the 
heck beat out of them when things go wrong--because things do 
go wrong--I think that we could do a lot.
    Senator Carper. Great. That is great advice, thank you.
    And, Nancy, one more, please?
    Ms. Goodman. Thank you, Senator Carper.
    The first solution, again, is that we need to reauthorize 
the Creating Hope Act, so that companies have incentives to 
develop pediatric rare disease drugs, and I hope that the 
Senate will move on that.
    Second, when companies are developing drugs for adults, we 
need to give companies incentives and requirements for them to 
just test them in kids, and update the Pediatric Research 
Equity Act, so that it protects children with cancer.
    And, maybe, we would ask companies to explain why they have 
minimum ages--a minimum age of eligibility for their adult 
trials at 18. Maybe, ask them to explain whether there are 
medical and scientific rationales for not lowering it, so that 
kids with terminal illnesses can get access to these drugs.
    And, finally, it is only a partial solution, but it is a 
very important one. I think that it would be terrific if 
Congress could pass the Andrea Sloan CURE Act to start 
improving the compassionate use process.
    Thank you.
    Senator Carper. OK. Thank you.
    Mr. Chairman, Cole, I am going to ask you to come over here 
just for a second. For a number of years, a young man from 
Delaware named Cole Hamstead has come to Washington. He brings 
his Mom with him every time. Laura and Cole have been a vital 
part of the work of the National Hemophilia Foundation (NHF). 
He is 10 years old and a wonderful young man. And, I showed a 
picture to your son, to Jordan over here, of Cole 3 years ago 
with me and explained to Jordan that, in that picture, Cole was 
then just the same age that Jordan is today. In response, 
Jordan was nice enough to offer to let Cole play with his toys 
as sort of a sign of welcome.
    Ms. McLinn. Good job, buddy.
    Senator Carper. These are two brave young men, courageous 
young men, who face adversity in their lives and have found, 
through the help, love, and support of a lot of other people, 
some good. And, I just want to say, to those of you who 
continue to lead a good fight--and to those who have taken 
adversity, Nancy, in the loss of your own son--to make sure 
that good things happen for a lot of other young people in our 
country, thank you. You are doing the Lord's work. God bless 
you.
    Ms. Goodman. Thank you.
    Senator Carper. Thank you.
    Chairman Johnson. Thank you, Senator Carper.
    Again, I want to just thank all of the witnesses. I think 
that this has been a wonderful hearing--powerful testimony. I 
appreciate your taking the time and answering our questions.
    I am going to--I think, with consent--we are going to have 
an honorary Chairman close out the hearing. So, I am going to 
switch chairs. I wish that I had a little bit of a fancier 
sign. And, I have a couple of things to say. I am going to 
switch chairs here, and then we will let you gavel it out.
    Senator Carper. Mr. Chairman.
    Chairman Johnson. I have to say a few magic words here.
    This hearing record will remain open for 15 days until 
March 11, at 5 p.m. for the submission of statements and 
questions for the record.
    This hearing is adjourned.
    [Jordan McLinn bangs gavel.]
    Chairman Johnson. There we go. Thank you.
    [Applause.]
    [Whereupon, at 11:43 a.m., the Committee was adjourned.]

                            A P P E N D I X

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          EXPLORING A RIGHT TO TRY FOR TERMINALLY ILL PATIENTS

                              ----------                              


                      THURSDAY, SEPTEMBER 22, 2016

                                     U.S. Senate,  
                           Committee on Homeland Security  
                                  and Governmental Affairs,
                                                    Washington, DC.
    The Committee met, pursuant to notice, at 10:04 a.m., in 
room SD-342, Dirksen Senate Office Building, Hon. Ron Johnson, 
Chairman of the Committee, presiding.
    Present: Senators Johnson, Paul, Lankford, Ayotte, Ernst, 
Sasse, Carper, McCaskill, Tester, and Peters.

             OPENING STATEMENT OF CHAIRMAN JOHNSON

    Chairman Johnson. Good morning. This hearing of the Senate 
Committee on Homeland Security and Governmental Affairs is 
called to order. I want to welcome all of our witnesses and all 
of our audience members.
    This is the second hearing we are holding on issues related 
to a bill I introduced called ``Right to Try,'' and I will ask 
unanimous consent (UC) to have my written statement entered in 
the record.\1\ I will keep my comments brief because I want to 
show about a 
3\1/2\-minute video as my true opening statement.
---------------------------------------------------------------------------
    \1\ The prepared statement of Senator Johnson appear in the 
Appendix on page 281.
---------------------------------------------------------------------------
    On Saturday, I attended a Walk for Amyotrophic Lateral 
Sclerosis (ALS) in Appleton, Wisconsin. I think that so many of 
us first heard about ALS as Lou Gehrig's disease, and then 
later, on Tom Watson's caddy. And, in my own personal 
experience, a member of our Lourdes High School family, Doug 
Perzorski, was diagnosed with ALS. His wife, Meg, and their 
children--unfortunately, we lost Doug a couple of years ago.
    And, then as a U.S. Senator, a couple of years ago, I met a 
young mother of three, Trickett Wendler, and I had just met 
with the Goldwater Institute, and I knew of their efforts 
trying to pass ``Right-to-Try'' bills in States like California 
and elsewhere. Just by mentioning my interest and my support 
for ``Right to Try,'' tears started streaming down Trickett's 
face.
    On Saturday, I met a number of patients fighting ALS, and 
their families fighting with them. There was a family--and 
three of the siblings were suffering from Familial ALS (FALS). 
One had had it for 15 years--it is a slightly different 
condition when it is that kind of hereditary ALS.
    We have Matt Bellina. We also have Frank Mongiello, who 
provided such incredible testimony when we had our press 
release on this, and so, Frank, welcome to you. In a nutshell, 
this effort--if it were up to me, I would not call it ``Right 
to Try.'' I would call it ``Right to Hope.''
    And so, what I would like to do now is offer a 3\1/2\-
minute video by Dr. Ebrahim Delpassand. I consider Dr. 
Delpassand a whistleblower. He is a courageous doctor. He is 
board-certified in nuclear medicine. He has a residency at 
Baylor, where he had his residency at Baylor College of 
Medicine, and was formerly at the University of Texas MD 
Anderson Cancer Center (MD Anderson) for 12 years, where he was 
the chief of clinical nuclear medicine. In 2003, he joined the 
private oncology center Excel Diagnostics. He is an adjunct 
professor at the University of Texas Medical Branch--a highly 
qualified doctor.
    In 2005, he began investigation for a new drug to treat 
neuroendocrine cancer--carcinoid cancer. A newer version was 
later available, which meant there was another approval process 
from 2007 to 2010. In August 2010, he began treating patients 
with a therapeutic agent called Lu-177 Octreotate. In March 
2015, he treated 143 of the 150 patients allowed. He requested 
the Food and Drug Administration's (FDA's) permission to expand 
the trial and add another 100 patients. By this point, the 
manufacturer had completed its multicenter trial and was in the 
follow-up phase to observe the progression of the condition. In 
other words, the clinical trials had completed enrollment. The 
FDA cited commercialization concerns in denying the request.
    So, I would like to just play the video now.
    [Videotape shown.]
    Now, I consider Dr. Delpassand a hero. I consider him a 
whistleblower. He is taking great risk operating under Texas' 
``Right-to-Try'' law, trying to save his patients' lives, but 
also giving and offering his patients hope.
    Now, I spoke with the representatives of the ALS community, 
here. Obviously, Dr. Delpassand is dealing with cancer 
patients. We also have Jordan and Laura McLinn. Jordan has 
Duchenne Muscular Dystrophy (DMD). Now, I was overjoyed on 
Monday to find out that the FDA finally approved a drug to give 
these little boys with DMD their right to try--their right to 
hope. So, that is really what this is all about.
    Now, next week, I will be asking the U.S. Senate to approve 
the ``Right-to-Try'' bill under a unanimous consent request. We 
have cleared it on our side--and, obviously, one of the goals 
of this Committee is to provide powerful testimony and to 
convince every U.S. Senator, if they are not willing to 
cosponsor it--I would certainly love their cosponsorship of 
this very simple bill that just allows these State ``Right-to-
Try'' bills to operate and that allow heroes like Dr. 
Delpassand to give their patients hope. If my Democratic 
colleagues and other Republican colleagues that have not 
cosponsored it are not willing to cosponsor it, please do not 
object so we can pass this bill and so we can give these 
patients and their families the right to hope.
    With that, I will turn it over to Senator Carper.

             OPENING STATEMENT OF SENATOR CARPER\1\

    Senator Carper. Thank you so much Mr. Chairman and thank 
you for calling this hearing today. I appreciate your 
willingness to continue a conversation about what we all know 
is an important issue that is critical for Americans seeking 
access to potentially lifesaving treatment. I also want to 
thank you and your staff for the ongoing work that we are 
engaged in to try and move the ball forward and to find ways to 
help patients gain expedited access to experimental therapies, 
including through a forthcoming Government Accountability 
Office (GAO) report on these issues. I especially want to thank 
our witnesses. It is a great honor to welcome you here. I 
especially want to say to Matt Bellina, Lieutenant Commander 
(Retired), an A-6 pilot, Navy salutes Navy this morning. And, I 
am delighted to see you here. It is a real treat to meet you.
---------------------------------------------------------------------------
    \1\ The prepared statement of Senator Carper appears in the 
Appendix on page 282.
---------------------------------------------------------------------------
    I also want to single out, if I could, Andrew McFadyen's 
wife, Ellen, and their son, Isaac, who is being treated for 
Mucopolysaccharidosis (MPS), and his brother, whose name is 
Gabriel. Gabe, it is nice of you to be here with your mom and 
dad.
    And, to all of our other witnesses that are here, thank you 
for joining us. It is great to see you.
    Before I begin my formal statement, I just want to take a 
moment and mention my appreciation for the Chairman for sharing 
the video that we have just seen and I look forward to learning 
more about this physician's experience. My understanding is 
that, in what would seem to be similar situations, the FDA has 
approved over 99 percent of patient applications for expanded 
access to these new experimental treatments. In fact, I 
understand the FDA has even granted drug approvals based solely 
on expanded access data, even for drugs that may be in Stage II 
trials--or even in Stage I trials. The FDA, as you know, by 
law, is precluded from discussing the details of any drug under 
review, but, if the doctor that we have just heard from today 
were here, I would ask him why he did not appear to use the 
expanded access program, which has worked quickly and 
efficiently for so many patients and their doctors. With that 
in mind, I would like to ask unanimous consent that we place an 
FDA fact sheet\2\ on expanded access, along with the recently 
updated application form, in the record.
---------------------------------------------------------------------------
    \2\ The FDA Fact sheet submitted by Senator Carper appear in the 
Appendix on page 314.
---------------------------------------------------------------------------
    Chairman Johnson. Without objection.
    Senator Carper. Thank you.
    My understanding is the application form for expedited 
access is a form that previously took hours to complete, I have 
heard as much as 10 hours--maybe even more. The expedited 
updated application form, I am told, takes 45 minutes or so, 
which has been one of the barriers for folks to actually take 
advantage of this opportunity.
    But, today we are going to have an opportunity to hear from 
the FDA, State representatives, patients, loved ones, and other 
advocates on ways we could improve access to experimental 
medical treatments--something we all want to do. These 
individuals and their families have faced some of the most 
difficult and painful challenges anyone could face. They 
deserve to be heard--and they deserve better access to 
experimental treatments.
    We will also have an opportunity today to review the 
Chairman's legislation, S. 2912, which he has alluded to, the 
Trickett Wendler ``Right-to-Try'' Act. I appreciate the intent 
of Senator Johnson's bill--I think we all do--and I, certainly, 
support expanding access to experimental therapies for 
terminally ill patients.
    We must keep in mind, however, that there is, already, what 
I understand to be an effective framework in place at the FDA 
for giving patients access to experimental drugs while those 
drugs are still being tested. The agency has given an 
extraordinary level of attention to the requests of patients 
with life-threatening conditions. In fact, I am told it has 
approved, as I said earlier, more than 99 percent of requests 
for emergency treatment between 2010 and 2015. The agency has 
also taken constructive steps to greatly simplify its 
application process and further improve and streamline 
patients' access to experimental treatments.
    Despite the high approval rates and ongoing reforms, I 
understand that the FDA believes that more can be done and is 
continuing to work to further improve patient access to 
experimental treatments--and we applaud that. I hope to learn 
about some of those steps today, as well as some additional 
ideas for how to ensure that all patients in need have the 
information and the resources needed to access experimental 
medicines.
    For terminally ill patients and their loved ones, safe and 
effective treatments cannot come quickly enough. That is why we 
need to do everything we can to give patients, doctors, and the 
companies that make these drugs the tools they need to 
participate in clinical trials, utilize the FDA's expanded 
access programs, and develop new treatments as safely, 
effectively, and quickly as possible. I hope that our Committee 
can help with these efforts and work with patients, health care 
providers, the pharmaceutical industry, and the FDA to ensure 
that all patients and their families can access safe and 
effective treatments as quickly as possible.
    Again, I want to thank our witnesses and their families for 
joining us today, and for your willingness to share your 
stories and put forward possible solutions to these challenging 
issues.
    Thank you so much.
    Chairman Johnson. Thank you, Senator Carper.
    I will note that the video that we played is just a 
condensed version of a 20-minute video that we will enter into 
the record and that will be available on our website. Dr. 
Delpassand wanted to 
be here--but he had a medical emergency, I guess, with his 
mother--so he could have answered those questions.
    It is the tradition of this Committee to swear in 
witnesses, so if you will all rise and raise your right hand. 
Do you swear the testimony you will give before this Committee 
will be the truth, the whole truth, and nothing but the truth, 
so help you, God?
    Mr. Bellina. Yes.
    Mr. Calderon. Yes.
    Mr. Neely. Yes.
    Mr. Garr. Yes.
    Mr. McFadyen. Yes.
    Chairman Johnson. Thank you. Please be seated.
    Our first witness is Matthew Bellina. Mr. Bellina is a 
former U.S. Navy aviator, retiring at the rank of Lieutenant 
Commander. He received his commission in 2005 after graduating 
from Virginia Polytechnic Institute and State University 
(Virginia Tech). In April 2014, he was diagnosed with ALS. He 
is a husband and a father of two boys, with another on the way. 
Mr. Bellina.
    Senator Carper. And, if any of your family members are here 
and you would like to introduce them, please do. And, that is 
for all of the witnesses.

  TESTIMONY OF MATTHEW BELLINA,\1\ LIEUTENANT COMMANDER, U.S. 
                         NAVY (RETIRED)

    Mr. Bellina. Thank you so much, Senators. And, thank you 
for having me. I would like to introduce my family. My kids are 
the boys that were making noise while you were speaking--and I 
apologize.
---------------------------------------------------------------------------
    \1\ The prepared statement of Mr. Bellina appears in the Appendix 
on page 284.
---------------------------------------------------------------------------
    Senator Carper. I would have been afraid, when my boys were 
their ages, to have brought them to a hearing like this. 
[Laughter.]
    Mr. Bellina. Yes, it was risky, but I did not want them to 
miss it.
    Senator Carper. They are doing great.
    Mr. Bellina. It is Caitlin, JP, and Kip siting back there.
    Senator Carper. Would you all raise your hands? Would you 
raise your hands, please, kids? There you go.
    Mr. Bellina. And there is an unnamed baby on the way as 
well.
    Senator Carper. That is good.
    Chairman Johnson. Go ahead, Matt.
    Mr. Bellina. Thank you, Senator.
    So, last night, when I was laying in bed, I was 
uncharacteristically nervous about this. Senator Carper, as 
somebody who has worked in naval aviation, you can understand. 
We are not usually nervous about things. But, I felt a great 
burden to speak on behalf of all terminal patients. How do you 
do that? And, then, a kind of calm washed over me because I 
realized that this is not a class or a group of people that we 
can all lump together. We are talking about individuals, and 
they have individual needs, individual hopes, and individual 
dreams. And, I do not have the right to speak on their behalf 
or to make decisions for them. And, neither does anybody else 
in this room.
    I think the first thing I want to clarify is that I am 
proud of the FDA's ``Gold Standard.'' I am proud of what the 
FDA does. I am proud that American medicine is the best in the 
world. And, I would never, ever want them to relax those 
standards for any reason. I think that that is an important 
distinction. We are not trying to undermine the FDA.
    This is a situation where we are asking for a very specific 
carve-out for a very small exception that does not 
necessarily--well, it does not cost us any money. I am not 
asking to build a wall. I am talking about making an exception 
for people that really need it.
    I am so happy, Senator Carper, that you mentioned 
compassionate use. I would like to commend the FDA. I think 
that they have done an excellent job of doing everything, 
within their regulatory power, to approve things. But, 
unfortunately, the data has a bit of a sample bias. I think 
there were 1,000 approved this year. There were 12,000 in 
France, if that gives you any idea. There were not that many 
people applying, and I think that it has a lot to do with the 
reporting requirement. No pharmaceutical company in their right 
mind would give me a drug compassionately, because, if I have 
an adverse event--which, I am an ALS patient, let us face it, I 
probably will--they have to report that to the FDA, and it is 
going to jeopardize their standing and their trial.
    We are dealing, in this case, with heterogeneous diseases. 
There is not a single cause. ALS is a perfect example. There 
are, I think, 39 known genetic mutations for ALS. That only 
makes up 10 percent of the overall cases. The other 90 percent 
are sporadic--and they are largely veterans. We do not know 
why. People who have worked in aviation are about eight times 
as likely to get ALS.
    So, when you run an FDA trial, as we are doing them now, 
you are throwing, at best, 400 people into a room together, 
giving them a small molecule, and hoping to get a result. Well, 
the data is kind of garbage in, garbage out. We are not getting 
good data. And, we need to move toward personalized medicine, 
in these cases.
    I feel that, ``Right to Try'' may not be, exactly, the end 
goal, but it is a first down. We are moving the marker toward 
doctors, patients, and pharmaceuticals being able to look at 
patients as individual people. And, I think that is a really 
important step. It is an ideological thing. I have heard the 
argument that we will risk side effects. But, as you can tell, 
I am struggling here. At some point in the near future, I am 
going to suffocate under the weight of my chest. So, what 
difference, at this point, does it make for me to have a side 
effect?
    And so, I think that, when you are talking about toenail 
fungus or psoriasis--yes, we need to be concerned about that. 
But, where I am sitting--it is a totally different story.
    The other thing some bioethicists have talked about is, 
``false hope.'' Do we want to give terminal patients ``false 
hope? '' Well, I do not really think there is such a thing, 
because right now we have no hope, so it is either hope or no 
hope. This bill, it does not do everything, but it moves us in 
the direction of having a little bit of hope. And, I think that 
it is very consistent with what the FDA is doing right now. I 
commend them. The Sarepta approval was fantastic. But, we need 
to open things up for the market to be able to kind of correct 
for itself and allow the pharmaceutical companies to not have 
to worry about negative action against them if they are trying 
to do something good.
    Chairman Johnson. Thank you, Matt.
    Mr. Bellina. Thank you.
    Chairman Johnson. Our next witness is Assemblyman Ian 
Calderon. Assemblyman Calderon is the majority leader in the 
California State Assembly, where he was first elected to 
represent the 57th Assembly District in November 2012.
    He serves as the Chair of the Select Committee on Youth and 
California's Future and Co-Chair of the Legislative Technology 
and Innovation Caucus. He has previously worked at Hurley 
International and is a field representative for the California 
Assembly. Assemblyman Calderon.

TESTIMONY OF THE HONORABLE IAN C. CALDERON,\1\ MAJORITY LEADER, 
              STATE ASSEMBLY, STATE OF CALIFORNIA

    Mr. Calderon. Thank you, Chairman Johnson, Ranking Member 
Carper, and Members of the Committee for inviting me here 
today. I am honored to testify before you on the ``Right to 
Try'' for terminally ill patients.
---------------------------------------------------------------------------
    \1\ The prepared statement of Mr. Calderon appears in the Appendix 
on page 286.
---------------------------------------------------------------------------
    As Majority Leader of the California State Assembly, I am 
fortunate to work on a variety of public policy issues every 
year. This year, alone, I have sent bills to the Governor 
dealing with issues ranging from ensuring that financial 
literacy is part of the high school curriculum to setting 
minimum fines for piracy violations. While each bill I work on 
is a piece of policy I believe in strongly, my work on ``Right-
to-Try'' legislation, over the last 2 years, has truly given me 
purpose as an elected official. The fight to allow terminally 
ill patients to seek investigational drugs and treatments not 
yet approved by the FDA is something I am immensely proud to be 
a part of in California. And, I thank you for giving me the 
opportunity to talk about it today.
    In January 2015, many of the policy conversations in 
California centered around ``Death with Dignity.'' If you 
recall, this was mere months after Brittany Maynard, the young 
woman diagnosed with brain cancer, had moved from California to 
Oregon in order to utilize Oregon's ``Death-with-Dignity'' law. 
While researching Oregon's law and its possible application in 
California, it struck me that this conversation needed to 
include policy prescriptions to make it easier for these 
terminally ill patients to fight to save or extend their lives. 
It was then that I came across the ``Right-to-Try'' movement, 
and subsequently introduced Assembly Bill 159. For me, ``Right 
to Try'' was a logical companion to ``Death with Dignity.'' I 
never saw the two issues as incompatible. I did not want to 
limit the options for those diagnosed with a terminal illness 
only to death--albeit a more controlled one. I felt strongly 
that, if we were going to pass ``Death with Dignity,'' and thus 
make it easier for terminally ill patients to die in 
California, that we should also make it easier for these 
terminally ill patients to fight to live, by giving them access 
to potentially lifesaving drugs and treatments that have been 
deemed safe--but are not yet approved by the FDA.
    As the first iteration of California's ``Right-to-Try'' 
legislation made its way through the legislative process, I had 
the privilege of meeting David Huntley. David was a professor 
emeritus at San Diego State University, an accomplished Ironman 
triathlete, and an, obviously, loved husband and father. David 
was also diagnosed with ALS, more commonly known as Lou 
Gehrig's disease. It is a death sentence given our current lack 
of understanding of the disease--but there are ways to combat 
the speed at which it progresses and the pain that it causes. 
Shortly after his diagnosis, David learned that there was a 
promising new drug called GM604 that was still in the clinical 
trial process at the FDA and, thus, had not yet been approved. 
He sought access to this drug, but was denied. So, David spent 
the latter part of his life fighting to give patients, like 
himself, a chance. David agreed to fly up to Sacramento in 
April of last year to testify with me before the California 
Assembly Health Committee. This was the first committee hearing 
on the ``Right to Try'' in California. David's testimony and 
clear understanding of the pitfalls of the current experimental 
drug access paradigm were instrumental in getting us past the 
first legislative hurdle.
    It was evident that David was in a tremendous amount of 
pain--yet he was determined that he be there for the ``Right-
to-Try'' legislation and that it pass in his home State. Just 3 
months after testifying, on July 4, 2015, David Huntley 
succumbed to ALS and passed away. He came to Sacramento to 
testify for a measure he knew would be too late to help 
himself, but that would ensure that future terminally ill 
patients have the access to potentially lifesaving medication 
that he had been denied. That kind of selflessness is rare--and 
I will never forget his dedication.
    With David's help, ``Right to Try'' passed the Assembly 
Health Committee. But, it still faced intense scrutiny from 
five more committees in the State Assembly and the State 
Senate. Though this was only last year, it was early in the 
``Right-to-Try'' movement. The bill went through a rigorous 
public hearing process, where we sought to improve upon the 
``Right-to-Try'' legislation that had been introduced in other 
States. Each committee, in concert with the myriad of 
stakeholder groups--and in deference to concerns that felt 
unique to California--included amendments to the legislation. 
Throughout the Committee process, we worked on--and eventually 
added--several amendments to alleviate concerns about having 
proper oversight patient protections. We added Institutional 
Review Board (IRB) oversight of a physician's recommendation, 
in order to ensure that patients are fully aware of the 
potential side effects of any investigational drug they may 
consume. We also added the requirement that a consulting 
physician confirm the primary physician's diagnosis that the 
patient is terminally ill as well as inserted reporting 
requirements into the California Department of Public Health 
(CDPH) to further increase oversight. And, similar to the 
difference I see in Senator Johnson's ``Right-to-Try'' bill 
versus the House's version, we clarified that the legislation 
would not create a private cause of action against the 
prescribing physician or drug manufacturer--and this was 
instrumental in removing the opposition of the California 
Medical Association (CMA).
    While we were not able to completely remove all opposition 
to California's ``Right-to-Try'' bill, through the public 
hearing process, we did work to address many of these concerns, 
without compromising the strong intent of the bill. When my 
``Right-to-Try'' bill reached the Governor's desk last year, I 
was satisfied that, due to its strong patient protections and 
robust oversight requirements, it was one of the most 
comprehensive ``Right-to-Try'' pieces of legislation in the 
country.
    The Governor ultimately vetoed the bill. And, in his 
message, he acknowledged that the FDA was in the process of 
streamlining its Expanded Access application and wanted to 
grant the agency the time to do so--with the hope that this new 
application would make the process unnecessary. Thirty one 
other States have passed ``Right-to-Try'' legislation. I am 
happy to have been a part of the impetus that spurred the FDA 
to streamline the application. However, these new regulations--
announced in June of this year--only deal with streamlining the 
physician's portion of the application. This is an improvement, 
but the process does nothing to shorten the manufacturer's 
portion and the data required by the application or to reduce 
the 30 days the FDA has to decide. A thousand people are 
approved each year by the FDA to get access to these drugs, but 
considering the fact that 564,000 Americans are expected to die 
from cancer, alone, this year, the small number of people 
navigating the FDA's Expanded Use program speaks to the 
program's failure to actually help terminally ill patients 
obtain access to lifesaving treatment. These patients do not 
have the luxury to wait for an onerous, bureaucratic process. 
These are the reasons why ``Right-to-Try'' legislation is so 
important, and, in the midst of a battle with a life-
threatening illness, it is much easier for a patient to deal 
with their own doctor rather than a large and impersonal 
government agency.
    At the beginning of this year, I reintroduced the bill in 
California. Fortunately, with all of the protections we added 
last year, it sailed through the legislature. It now sits on 
the Governor's desk, where I am hopeful it merits a signature, 
adding California as the 32nd State to enact this legislation.
    Members, I thank you for giving me the opportunity to share 
my effort to bring ``Right to Try'' to California. I applaud 
the effort on the Federal level to do the same nationwide. I 
hope the Federal Government does not stop there. We need 
Federal legislation that expedites the FDA's drug approval 
process. It should not take, in today's technology age, 10 to 
15 years to approve a new drug that will produce lifesaving 
treatments. In the meantime, I commend the Federal effort to 
encourage the States to essentially adopt methods to work 
around the FDA.
    ``Right to Try,'' at its core, is simple, and it speaks to 
a very basic human right. If your parent, your child, or even 
you are faced with a terminal illness, there should be a 
process in place for you to seek potentially lifesaving 
treatments--and the government should not impede that. This 
bill received unanimous bipartisan support in the California 
State Assembly--all Democrats and all Republicans--and it is 
extremely important to pass it on the Federal level because it 
gives us, as a State, the opportunity to have these laws on the 
books while also, at the same time, freeing up the drug 
manufacturers, allowing them to give the drug to these 
terminally ill patients.
    Members, thank you for your time.
    Chairman Johnson. Thank you, Assemblyman Calderon.
    Our next witness is State Representative Jim Neely. 
Representative Neely represents the 8th State House District in 
the Missouri House of Representatives. He was first elected in 
November 2012. He is also a physician at Cameron Region Medical 
Center and a veteran of the United States Army. He also 
previously served for over a decade on the Cameron School 
Board. Representative Neely.

TESTIMONY OF THE HONORABLE JIM NEELY, D.O.,\1\ MEMBER, HOUSE OF 
               REPRESENTATIVES, STATE OF MISSOURI

    Dr. Neely. Good morning. Thank you. Mr. Chairman and 
Members of the Committee, again, my name is Jim Neely. I am a 
physician, State representative, and, more importantly, a dad. 
I want to go on record in support of S. 2912 because terminally 
ill patients do not have time to wait for the FDA to improve 
investigational treatments.
---------------------------------------------------------------------------
    \1\ The prepared statement of Mr. Neely appears in the Appendix on 
page 292.
---------------------------------------------------------------------------
    I have been practicing medicine for over 30 years. Over 
that time, I have seen patients with medical conditions and 
issues of life that have been very challenging to deal with, 
from multiple myeloma, multiple sclerosis (MS), ALS, to 
acquired immunodeficiency syndrome (AIDS).
    In 1985, I had my first AIDS patient. I was practicing 
medicine in Florida at the time. My first AIDS patient, he was 
debilitated, frail. He was hopeless. A year later, he died. He 
was not eligible for any clinical trials. They did not approve 
the first antiretroviral (ARV) agent until a year later.
    Throughout my medical career, I have been troubled by laws 
that restrict suffering patients' access to investigational 
treatment. As a physician, my practice is guided by evidence. I 
understand the importance of our clinical trial system. 
However, terminally ill patients deserve the option to try 
investigational treatments after they have exhausted all 
approved treatment options and they are no longer eligible for 
clinical trials.
    As I began considering the issues as a State legislator, my 
daughter Kristina was diagnosed with Stage IV colon cancer. She 
had four children at the time and was pregnant with the fifth, 
which severely limited her eligibility for clinical trials. 
From a research perspective, I understand the importance of 
studying uniform groups of patients, but there has to be room 
for compassion for patients like our daughter.
    Before she passed away, just last year, she was adamant, I 
quote, treatments ``should not be up to somebody that has no 
involvement in my care.'' I believe this bill goes a long way 
to providing more options for terminally ill patients and their 
physicians.
    A friend of mine, Ross Nichols, testified on our bill, in 
Missouri, on ``Right to Try.'' It passed unanimously out of the 
Senate and out of our House. It passed 152-1. The one who voted 
no never votes yes. This gentleman, Ross, he had glioblastoma 
(GBM), the most aggressive--the most common brain tumor. Ross 
knew that the experimental treatments he was likely to receive 
would not save his life. But he still enrolled in clinical 
trials. He explained, ``My number one job right now is being a 
dad. I will do whatever I can do to extend that.'' Ross told 
the committee that he was testifying for the bill because he 
wanted people in Missouri to have access to the same treatments 
that were available to him at the research institutions that he 
could afford to travel to. He was right.
    Many people fighting for their life cannot afford to spend 
what could be their final months traveling across the country 
in order to receive investigational treatments. I am glad Ross 
was able to travel and receive treatments that gave him hope, 
but he should not have had to travel. Ross passed away in 
February 2015. I am glad his hope for other patients lives on 
with this bill.
    Rick Suozzi, father of the late Kim Suozzi, also came to 
testify in support of our ``Right-to-Try'' bill in Missouri. 
His daughter was diagnosed with glioblastoma at the age of 21 
in her final semester at Truman State University. Kim knew her 
diagnosis was a death sentence, but she went to extraordinary 
lengths for a small chance to survive. She traveled to the top-
notch cancer institutes like Dana Farber Cancer Institute, 
UCLA, Duke, the University of Texas MD Anderson Cancer Center 
(MD Anderson). Kim enrolled in three trials in the last 6 
months of her life. She was no longer eligible for clinical 
trials. She lied to research doctors about her treatment 
history in order to make herself eligible. Can any of us blame 
her?
    My time is up. I thank you for giving me the opportunity. I 
believe government should create opportunities for people to 
care for each other. This bill knocks down some of those 
barriers. I appreciate the opportunity to be here today. It is 
an honor. Thank you. This is a bill for people. Thank you.
    Chairman Johnson. Thank you, Dr. Neely. We are so sorry for 
your loss, but thank you for your testimony.
    Our next witness is Richard Garr. Mr. Garr is the co-
founder of Neuralstem, Inc., which develops therapies involving 
brain and spinal cord stem cells, including a treatment for ALS 
currently in clinical trials. He was previously president and 
Chief Executive Officer (CEO) of the company. Prior to 
Neuralstem, he was an attorney at Beli, Weil, & Jacobs, the B&G 
Companies, and the Circle Management Companies. He is also the 
founder of the First Star Foundation, The Starlight Foundation 
Mid-Atlantic chapter, and a past honorary chairman of the Brain 
Tumor Society. Mr. Garr.

   TESTIMONY OF RICHARD GARR,\1\ FORMER PRESIDENT AND CHIEF 
              EXECUTIVE OFFICER, NEURALSTEM, INC.

    Mr. Garr. Thank you. I would like to thank the Committee 
for this opportunity to testify in support of this Act.
---------------------------------------------------------------------------
    \1\ The prepared statement of Mr. Garr appears in the Appendix on 
page 296.
---------------------------------------------------------------------------
    As president and CEO of a biopharmaceutical company 
developing treatments for currently incurable diseases, as a 
member of the advisory board that helped craft the model 
``Right-to-Try'' act which has been making its way through the 
States, and as the father of a son diagnosed with a brain 
tumor, I have been involved in the scientific, FDA regulatory, 
business, legislative, and patient advocacy arenas germane to 
this issue for over two decades. 
S. 2912 is a good bill that will provide hope and comfort to 
many patients diagnosed with fatal diseases and it will 
accelerate the effort to find cures for currently incurable 
diseases.
    There are issues--and I would like to spend my limited time 
here, today, talking about them.
    An issue we hear is that this is going to foist unsafe 
medicines on an unsuspecting public. Nothing could be further 
from the truth. This bill relies on FDA oversight. As you know, 
any drug that can be administered under this has to have gone 
through an FDA Phase I trial and has to continue to be in the 
FDA queue, which is a very important point. So, if a company 
pulls a drug out of the FDA process for any reason--safety or 
otherwise--it is no longer eligible to be administered under 
this Act.
    I also think it needs to be pointed out that, companies do 
not develop drugs for incurable diseases without spending an 
enormous amount of time and money. This is not an undertaking 
you go into lightly. At Neuralstem, it took us at least $50 
million and 10 years just to get into our first trial for ALS. 
So, in addition to the FDA safety data, which you get from the 
trials and which are required for a doctor to administer this 
drug, there is always a large body of pre-clinical safety 
data--such as animal data, in vitro, and in vivo data. And so, 
there is a lot of safety data for these drugs.
    I would also--as other people have said here--like to 
debunk the myth that this is somehow an anti-FDA bill. Nothing 
could be further from the truth. The heart of this bill is the 
safety net that continuing FDA oversight provides--an 
oversight, as everyone has said, that is universally 
acknowledged as the gold standard for the world. And, I would 
point out here that even proponents of the ``Right-to-Try'' 
movement, such as the Goldwater Institute, who probably believe 
that ``Right to Try'' is based on a constitutional principle 
and disagree with the idea of Federal preemption, have insisted 
on the FDA oversight principle of safety in all of the 31 Acts 
so far that have gotten through the States. This is not an 
anti-FDA bill.
    I would like to address your issue, Senator Carper, 
briefly. We did two trials for ALS. We will be starting a 
third, hopefully, in the not too distant future--but, probably, 
in Japan for regulatory reasons. So, we treated 40 patients--15 
in the first trial. It took us 18 months to transplant those--
to do those 15 surgeries. We had at least--and I am just 
thinking of my own personal emails at night--600 people who 
asked if they could have it on a compassionate use basis. It 
cost us $150,000 per patient--and that is without charging 
anything for ourselves--for each patient in that trial. So, the 
reason that the FDA's compassionate use vehicle, or tool, is 
not applicable is that is a tool, but it is a tool for a very 
specific use--and maybe about 1,000 people a year can do it.
    In a case like ours--and I would tell you that all of the 
products that are being developed for incurable diseases are 
not cheap pills. They are all cell therapy or gene cell, 
monoclonal antibodies. It is very expensive technology. And, 
the fact is we could not do any compassionate use--we could not 
possibly meet the need. We did not have the money and we did 
not have the time or the resources. Maybe GlaxoSmithKline can 
do it for a cancer drug. But, they are not developing these 
drugs. The biotechnology industry is developing these 
experimental medicines. And so, another part of this that has 
to be addressed--and the States will have to do it--is you have 
to let the companies charge for this--and you cannot do that 
under your existing compassionate use guidelines with the FDA. 
There are very strict limits on cost. And, actual cost for our 
product--for me to send our cell guy up to Charles River Labs 
in Pennsylvania, to follow the cells, to do what we do, and to 
ship them to the surgeon has nothing to do with the $50 million 
that it costs us to get to that point. And so, there is 
something that just does not work there.
    I see that I am running out of time, so I do want to 
mention one last thing, and that is--as you are hearing from 
everybody here, this is a ``Right-to-Try'' Act, not a ``Right-
to-Cure'' Act. And, what the doctors who deal with the patients 
and the caregivers will tell you, almost across the board in 
all of these diseases--not just ALS--is that people with fatal 
diagnoses--and this bill only applies to drugs for fatal 
diseases. People with fatal diagnoses have a sense of 
hopelessness and their caregivers do also. And, many of them 
feel that they have lost control of their lives. They want to 
go down fighting. They want to help research. No one who gets 
an experimental drug--even if we are all hoping that it can 
work--we do not develop drugs that we do not think are going to 
work. But, the fact is, over 90 percent of them fail--even 
those that get to late-stage trials.
    So, the industry is meticulous in educating patients about 
the realities of what is going to happen. And, they still want 
to go through it. Yes, they have hope and they want to help the 
research--and this will accelerate research. And so, I think it 
is also an extremely important part of these ``Right-to-Try'' 
Acts that you give people back some control of their lives in a 
place where science is telling them they have lost control and 
that is why they have this fatal diagnosis.
    Thank you for the time.
    Chairman Johnson. Thank you, Mr. Garr.
    Our final witness is Andrew McFadyen. Mr. McFadyen is the 
executive director of The Isaac Foundation, a nonprofit focused 
on funding and supporting research into finding a cure for 
Mucopolysaccharidoses (MPS), a rare and progressive disease 
affecting his eldest son. As part of his work, he is a member 
of the New York University (NYU) Langone Medical Center Working 
Group on Compassionate Use and Pre-Approval Access. He is also 
an eighth grade teacher in Kingston, Ontario and a guest 
lecturer at Queen's University Faculty of Education. Mr. 
McFadyen.

TESTIMONY OF ANDREW MCFADYEN,\1\ EXECUTIVE DIRECTOR, THE ISAAC 
                           FOUNDATION

    Mr. McFadyen. Thank you. And, I want to actually begin by 
just welcoming my kids, Isaac and Gabriel. They are my 
inspiration in all that I do. And, I wanted to use a little bit 
of my time as well to recognize you, Matthew. Your story is 
incredibly heartbreaking. Your family is just beautiful. And, 
you are an inspiration to me and I am glad that you are here.
---------------------------------------------------------------------------
    \1\ The prepared statement of Mr. McFayden appears in the Appendix 
on page 299.
---------------------------------------------------------------------------
    So, good morning. I am the executive director of The Isaac 
Foundation, an organization based in Canada that is dedicated 
to providing advisory and support to patients dealing with a 
wide range of disorders and needing access to rare disease 
treatments. Our work pushes international boundaries, with the 
bulk of our efforts taking place in Canada and the United 
States. I am also a member of the NYU Working Group on 
Compassionate Use and Pre-Approval Access, where we are making 
a concerted effort to improve and address the issues around 
access to experimental medications.
    I am very proud to say that at The Isaac Foundation we have 
never, ever been unsuccessful in gaining access to rare disease 
treatments for children in Canada. And, our work, directly, 
with pharmaceutical companies here in the United States is 
helping countless patients see similar results as well.
    Now, as I stated, my organization is incredibly dear to me 
because it is named after my son--my hero and the bravest 
person I know--Isaac McFadyen, who is here behind me today and 
who suffers from MPS Type VI.
    When Isaac was diagnosed, we were told that he was going to 
live a life of pain and suffering. Every bone, tissue, organ, 
and muscle in his body--with the exception of his brain--would 
be ravaged by his disease until he eventually succumbed to the 
condition--probably in his early to late teens.
    For 10 years, he has battled--we have battled together--to 
stave off the inevitable. And, we have been lucky. In 2006, we 
were able to gain a new life-prolonging treatment--one that was 
approved here by the FDA, but not by Health Canada--to fight 
his disease. Isaac is now 12 years old--and the 12 that we see 
today is very different than the 12 we were told to prepare 
for.
    So, I fully understand the world that our families are 
living in and I understand the unbearable burden that a 
terminal diagnosis brings to a family. I understand because I 
live each and every day staring down the mortality of my son. I 
understand because I have walked this lonely road searching for 
hope when all seemed lost. I have been there--and I am still 
there each and every day as I continue to work tirelessly to 
find a cure for Isaac before the clock runs out.
    Now, as I said, Isaac is one of the lucky ones. 
Unfortunately, little Jack Fowler in the picture here is not. 
Jack was diagnosed with a version of MPS similar to the type 
that Isaac has--except Jack has the tragic misfortune of having 
his disease attack his brain. The disease will progress rapidly 
and steal everything that makes Jack Fowler Jack Fowler--his 
mobility, his words, his thoughts--his everything--before 
finally taking Jack Fowler away from us.
    Now, there is a drug that can prevent this from happening, 
but Jack barely missed out on qualifying for the clinical trial 
and he was not able to obtain the drug under the FDA's expanded 
access program because, despite his physician, the hospital, 
the hospital review board, and the FDA all supporting his case, 
Shire Pharmaceuticals blocked access to the drug. Today, hope 
is all but lost for his parents as they watch their son slip 
away.
    But, how can hope be lost for Jack Fowler when he lives in 
a State where ``Right-to-Try'' legislation is in place? The 
Goldwater Institute has done a marvelous job of promoting 
``Right-to-Try'' laws as being the last chance for people to 
extend their lives. Very pointedly, they claim that ``Right-to-
Try'' legislation ``restores lifesaving hope back to those who 
have lost it.'' This utopian vision of access to medications 
for millions of Americans who need them is laudable. However, 
the cruel reality with ``Right-to-Try'' laws is that it will 
not grant patients the immediate access to the treatments they 
desperately need--the and it never has.
    Looking past the myths that ``Right-to-Try'' proponents 
state ad nauseam and looking past this legislation's potential 
to create an unequal access to medication, the simple fact is 
that this legislation, crafted to give people like Jack Fowler 
his fair shot of simply being alive, does not work. Although 
over 183 million Americans, including Jack Fowler, are 
currently living within the boundaries governed by ``Right-to-
Try'' laws, providing them with, as the Goldwater Institute 
claims, immediate access to the medical treatments they need, 
there continues to be no concrete evidence of a patient ever 
receiving a life-saving medication under ``Right-to-Try'' 
legislation that they otherwise would not have received under 
the FDA's expanded access program.
    In truth, ``Right to Try'' is a misnomer and provides 
nothing to patients in need, except a misguided belief that 
help has arrived. A more apt title would be ``Right to Ask'' 
because this is the only entitled right the legislation 
actually gives patients.
    There are ways forward. Over the past few months, companies 
like Janssen and BioMarin are crafting new approaches while 
working within existing FDA programs and guidelines to 
expeditiously and fairly provide access to those in need--and 
it is working.
    So, recognizing that we are all here to work toward the 
same outcome, I hope that we can move now and that we can keep 
moving until everyone in this room and everyone throughout the 
country can look the parents of Jack Fowler in the eyes and 
tell them that help is on the way, that help is here, and that 
Jack will get to realize his simple dream of being alive.
    Thank you.
    Chairman Johnson. Thank you, Mr. McFadyen. And welcome, 
Isaac.
    I have been told that Frank Mongiello is in the audience 
and he would like to make a brief statement. If we can assist 
Frank, get him up to a microphone. We have some staff coming 
down.

             TESTIMONY OF ERIC AND FRANK MONGIELLO

    Mr. Eric Mongiello. So, first I would like to start off by 
reading a quote from Mahatma Gandhi, and it----
    Chairman Johnson. And you are Frank's son? Can you just 
introduce yourself quickly?
    Mr. Eric Mongiello. I am Eric Mongiello. This is my dad 
right here, Frank Mongiello.
    So, starting with this quote: ``You may never know what 
results come of your actions, but if you do nothing, there will 
be no results.''
    Basically, what is that meaning? We do not have the luxury 
of time to wait around until this bill gets passed. Every day 
is another day off my dad's life, and others with terminal 
illnesses. Me, personally, I do not want to have to grow up 
without a father. I do not want my little brother and my 
siblings to have to grow up without him. And, with the ``Right 
to Try,'' this will give us hope. We have no hope right now. 
And, this is very important to me because, as long as we have a 
fighting chance, then I know whatever happens, whether it helps 
or not, that at least we tried, and at least we did something 
instead of sitting by the side and waiting for the inevitable. 
Thank you.
    Chairman Johnson. Thank you. Frank.
    Mr. Frank Mongiello. Thank you very much for having this 
hearing.
    My ALS is progressing every day [unclear]. We do not have 
the luxury of time.
    All we are asking for here is a basic inalienable right to 
live every [unclear]. I do not want to die, but I want to be 
able to fight for my life, and if that means taking a drug that 
might not be proven by the FDA, I am OK with that.
    It is my life. I should have the right to fight for it. 
Thank you.
    Chairman Johnson. Thank you, Frank.
    Frank made a statement when we had a press conference on 
this. And, Frank has a beautiful family and he is just asking 
for that right to hope.
    Mr. Garr, I will start with the questioning. Can you 
respond a little bit to Mr. McFadyen? Why would Shire block 
access to--can you just kind of explain that?
    Mr. Garr. I am not from Shire, so I will not speak for 
them.
    Chairman Johnson. I understand, but I mean, can you----
    Mr. Garr. In general, sure, and I would like to address 
this claim, which is that nobody has been treated with ``Right 
to Try.'' The reason no one has been treated with ``Right to 
Try'' is that this bill has not passed, right? As you put your 
industry hat on, all of the issues that have to be addressed, 
in terms of whether the FDA can use negative information to 
withhold your approval in the regular approval process--that is 
the reason, right? This bill, specifically, prohibits the FDA 
from doing that. When you design a trial, you eliminate as much 
of the variables as possible to match your evidence--your pre-
clinical evidence--and you create an experiment. Correct? And 
then, when you go to ``Right to Try'' or compassionate use, you 
start treating people, as the doctor said, that are outside of 
that.
    And so, it is very difficult, as a company, after you have 
just spent 10 years and $1 billion, or whatever it is Shire 
spent on their drug, to get to a point and then say, ``OK, we 
know that if this goes into patients that do not meet the 
inclusion criteria, there could be problems.'' So, they have an 
obligation to their shareholders to get their drug approved. 
That is probably what they told themselves. I do not think I 
agree with it, but that is what the obligation is. And, no 
company, I will tell you--and he confirmed that, right? No 
company will feel comfortable going through this. When you go 
through the compassionate use process with the FDA, that 
limited tool, you have some protection. Under the current State 
``Right-to-Try'' Acts, you have none.
    Chairman Johnson. And, that is basically the reason. You 
have the 31 States that have passed this, but because you have 
no protections at the Federal level, anybody like Dr. 
Delpassand is taking a huge risk. That is why I call Dr. 
Delpassand a real hero. He is taking an enormous risk with his 
career treating these patients and giving them hope. So, we 
really need the Federal law in order for the State laws to 
actually kick in. And, that is really, I think, what the 
strategy was--to show the support for ``Right to Try'' in the 
States-- but until the Federal Government acts, the State laws 
have not been effective.
    Mr. Garr. Absolutely.
    Chairman Johnson. Assemblyman Calderon, first of all, what 
party affiliation are you?
    Mr. Calderon. I am a Democrat.
    Chairman Johnson. And, again, this had very strong 
bipartisan support. You made the statement--and this is in 
terms of the simplicity of what we are asking for here--
patients have the right to die in certain jurisdictions, and 
yet they do not have the ``Right to Try.'' Where is the logic 
in that?
    Mr. Calderon. I do not know. That is why I felt, once I got 
the bill to the Governor's desk, that it would merit a 
signature--because of the State at the time dealing with the 
``Death-with-Dignity'' law. However, the Governor vetoed and we 
have been working with his office and we are hopeful that we 
will get a signature this year.
    Chairman Johnson. Can somebody explain, again, the 
statistics of 99 percent of expanded use being approved--1,000 
patients versus how many would be, potentially, looking at 
this? Matt, I see----
    Mr. Bellina. Senator Johnson, if I could weigh in on that. 
So, before you apply for compassionate use, as a terminal 
patient, you go and talk to your doctor and say, ``What do you 
think about this thing?'' And, they look at you as a person, it 
is between you and your doctor--it is your body. And, they say, 
``Yes, I think that could help you,'' or, ``No, there is not 
enough data,'' or whatever.
    If they think it is a good idea, the next thing you are 
going to do is call the company, and you are going to say, 
``Hey, you are in a Phase IIb trial with this drug, and I want 
to try it.'' And, I am going to tell you, from personal 
experience, about 50 times, you are not going to get a 
response.
    Chairman Johnson. So, you personally, have made those 
calls?
    Mr. Bellina. I have made those calls, personally. And, I 
cannot get into an FDA trial because I do not meet exclusion 
criteria. I have had the disease for too long. So, there is 
literally no way for me to get these drugs. As Mr. Garr said, I 
mean, they are not going to risk giving me a drug and risk me 
having an adverse event. They would have to report that to the 
FDA. They have already spent--who knows--maybe $100 million at 
this point.
    Chairman Johnson. Does anybody have any kind of statistic 
regarding , how many Matts are out there, that have made those 
50 calls, in comparison to the 1,000 successful applications of 
expanded use or expanded access?
    Mr. Bellina. Senator, I think that is, again, something we 
will never know simply because it never gets to the government 
at that point. You are talking about a gmail account to a gmail 
account. This is not under any kind of regulation. These are 
just people reaching out to people in these companies--and 
there is no way to quantify that.
    Chairman Johnson. Yes.
    Mr. Calderon. Senator, if I may, I was elected to the 
California State Assembly when I was 27 years old. I am 30 now. 
I am the youngest majority leader in the history of the State 
of California. But, I am also a Millennial, and part of my 
generation--everybody is trying to say, ``Well, how do we get 
them engaged? How do we get them to care about our political 
process?'' A lot of it is just our perception that government 
just has this inability to reach these common sense ideas. Why 
would you not allow someone who is terminally ill the ability 
to try and fight to save their own life? It is beyond logic to 
me.
    Chairman Johnson. So, having fought this successfully 
legislatively, what was the primary argument against--I mean, 
in the end, you knocked down all of those walls because you 
passed this almost unanimously, correct?
    Mr. Calderon. Absolutely.
    Chairman Johnson. Just one person voted no.
    Mr. Calderon. And, in terms of the opposition, the problem 
was more, a question of whether there was liability. All that 
my bill did, really, was give people the ability to try and ask 
for the drug. That is all the intent of the original bill was. 
But, given the opposition, we added all of these patient 
protections and oversight. By the time the bill got to the 
Governor, the reason why the Governor vetoed it is because, 
within the expanded access, the FDA was working on an expedited 
way to get access to those drugs.
    But, look, let us be honest. If I had a terminal illness, I 
would want to live. I am probably not going to want to deal 
with 
the FDA, because how do you figure that out? I do not even 
know--I am authoring the bill in California. I do not even know 
where I would go. I guess I would just go online and google how 
I would even apply for the program. But, contrast that with the 
State having a law on the books where you can just talk to your 
own doctor and say, ``Hey, Doc, I am terminally ill. I want to 
fight to live. I do not care what it takes. Give me anything 
that is going to allow me to stay on this Earth longer.'' Or, 
if it is for my kids or a parent.
    Chairman Johnson. But, you were able to overcome the 
objections, and our bill also overcomes those same objections, 
basically?
    Mr. Calderon. Absolutely.
    Chairman Johnson. Just very quickly, Dr. Neely--a similar 
situation. What objections--why did anybody oppose this in 
Missouri?
    Mr. Neely. In the State of Missouri, nobody had any issues. 
Everybody was all on board. It was about taking care of people. 
I think, at our level, it is all about taking care of people. 
And, I think that is what we have done.
    I might add we had--one of my staff physicians at Cameron 
came down with ALS in 2014 right after Missouri passed their 
``Right-to-Try'' legislation, and I was telling him about it, 
and the trials that were going on for ALS in the San Francisco 
area. Again, he was in his early 70s. He had been practicing 
medicine for 40 years. He did not want to have to deal with the 
government.
    Chairman Johnson. OK. Senator Carper, are you ready?
    Senator Carper. I am.
    Chairman Johnson. Senator Carper.
    Senator Carper. Thank you again. Matt, gosh, about 15 years 
ago, my brother-in-law was diagnosed with ALS, and so, we lived 
what you are going through.
    My mother passed away about 8 years ago--Alzheimer's. Her 
mother, her grandmother, her sister. My guess is everyone on 
this panel could tell a story. Everyone.
    Andrew, let me ask you a question. I do not know how 
familiar you are with the changes that have been made in the 
approval process at the FDA to expedite access to experimental 
treatments. But, my understanding is that there had been a lot 
of criticism, in the past, of the FDA. The FDA has been 
responsive to those criticisms. Could you just walk us through 
some of that? And, I will ask Dr. Lurie the same question 
later. But, are you familiar with that?
    Mr. McFadyen. Yes, I am, actually. I understand that they 
have made changes. They released guidance, in June, talking 
about issues that could take place--adverse events and how 
impact clinical trials. At the same time, they updated their 
application form. It has been said--I have heard it often--that 
it takes 100 hours to fill out this form--and that is just not 
the case. It takes 100 hours for the company, before they apply 
to the FDA, to fill out those forms. The old form, it did take 
a few hours to do. We went through it with Jack Fowler. We 
looked at it long and hard with Jack Fowler. I did it, myself, 
and it did not take that long.
    The new changes in place are looking at about 45 minutes 
for those forms to be filled out, and, it is not filled out by 
the patient. It is filled out by the doctor. It is the exact 
same thing. You do go to your doctor, and you ask to have 
access to this drug. The doctor sends it to the FDA and it 
needs to be sponsored by the companies.
    The same holds true for ``Right to Try.'' You go to your 
doctor, you ask, and you have to get the company on board to do 
this. So, even if this passes expeditiously at the Federal 
level, nothing really changes with respect to access when it 
comes to the companies agreeing or not agreeing to provide. 
And, I know the FDA has done a really good job of getting that 
form changed. It took a little while, but that application 
process is changed.
    I also want to note that they did release, in that 
guidance, some information for companies, so that companies 
were not as adverse to providing these drugs for patients. And, 
they said very categorically in that guidance that adverse 
events that take place will not have a bearing on the clinical 
trial process. To support that, they released an audit, and in 
that audit, they were able to show that expanded access was not 
derailing clinical trials by any stretch of the imagination.
    There are still things that they could do. They still have 
a ways to go. I would recommend that they do another audit and 
look at the drugs that have passed and have a look at which 
adverse events took place during those clinical trials for 
compassionate use, as the drug was being passed, and how those 
adverse events really impacted the discussion--the approval 
process discussion. And, our hunch with our group at NYU 
Medical Center is that the people making these decisions 
understand that the people accessing these drugs under 
compassionate use, expanded access are the sickest of the sick. 
They are not your healthy sick people that get into the trials 
and they take that into account when they are looking at 
approving these drugs.
    Senator Carper. So, let me see if I understand this. It is 
a question I just asked of my staff. If the FDA approved 
anybody who applied, who filled out this form, whether it is 45 
minutes or whatever it takes to fill it out--and I understand 
it is filled out not by the patient and not by the patient's 
family, but by the patient's physician. Is that correct?
    Mr. McFadyen. That is right.
    Senator Carper. So, the notion that--and I think it was Dr. 
Neely who said these words, and I think he was quoting his 
daughter, ``Treatment should not be up to somebody who has no 
involvement in my medical treatment.'' But, the person who 
actually submits and signs, fills out and completes the 
document that goes to the FDA is the physician of the patient. 
Is that right?
    Mr. McFadyen. That is right.
    Senator Carper. OK. So, let me see if I understand this. 
The FDA could approve access to experimental drugs to everybody 
who applies, whether it is in Stage I, Stage II, or Stage III. 
And, that does not mean that a patient is going to have access 
to those treatments. OK. I understand you are saying that is 
correct. So, then, if this legislation does not really get at 
the heart of the problem and the changes that the FDA has 
made--and I think they are significant and in the right 
direction--do not completely solve the problem, what should we 
do legislatively? I think you may have said this, but I just 
want to ask you to say it again. If this does not work--this 
legislation does not work--and if the FDA reforms are 
insufficient, what do we need to do? Or, is it something that 
maybe is beyond----
    Mr. McFadyen. Well, I think, first and foremost, we need to 
pass the 21st Century Cures Act. That will pave a quicker 
pathway----
    Senator Carper. You say the 21st Century Cures Act?
    Mr. McFadyen. That is right, yes.
    Senator Carper. Is that Senator Alexander's legislation?
    Mr. McFadyen. I believe so.
    Senator Carper. I think so, yes.
    Mr. McFadyen. I think that needs to be passed. I also think 
at the same time we need----
    Senator Carper. And, tell us why.
    Mr. McFadyen. Well, it is going to pave the way to have 
access to drugs approved a lot quicker. At the same time, I 
think we need to pass the Andrea Sloan CURE Act where--that 
forces companies to actually state online their expanded 
access, compassionate use policies--the criteria for inclusion. 
It will also force companies to have one point of contact at 
the company for somebody like Matthew to call and say, listen, 
I do not want a gmail address. I want--at Johnson & Johnson's 
(J&J's) website--and I want that one person to get back to me, 
immediately. If an application for compassionate use with a 
company is denied, it forces the company to explain exactly 
why. It actually lifts a level of secrecy at the company level.
    At the FDA, they really need to do a better job of 
communicating to the pharmaceutical industry exactly what 
adverse events do to the clinical trial process. In our study 
at NYU, we have seen that it does not actually impact it. But, 
Matthew is right. Only 1,200 or 1,300 applications land at the 
FDA to be signed off on, and although 99 percent of those are 
approved, that is still only a small representation of the 
people that need access.
    So, if ``Right-to-Try'' legislation gets passed at the 
Federal level, just as it has at State levels, we are not going 
to see the dramatic shift tomorrow that we hope to see. We just 
are not.
    I talked to a lot of industry folks before I came down 
here. I spent 16 hours a day for the past week talking to an 
unbelievable amount of companies throughout the United States--
companies that I deal with seeking access as well as companies 
that I have never talked to before. And, they are as afraid of 
adverse events outside of the clinical trial setting taking 
place under expanded access as they are under ``Right-to-Try'' 
legislation. And, although the FDA cannot hold adverse events 
against the company and against the clinical trial because they 
do not have to be reported to them, there is a very good chance 
that adverse events under ``Right to Try'' will still be 
reported. It will be reported in the media. Companies that 
trade publicly have to include reports about anything that 
could impact the approval or the development of a new drug to 
the U.S. Securities and Exchange Commission (SEC). Oftentimes, 
those reports fall into shareholders' hands, which fall into 
the general public's hands, which get reported in the media.
    And so, it is a very real issue for them under both ``Right 
to Try'' and expanded access. And so, what we really need to do 
is work with these companies to ask how we can come together to 
make the changes that we need in order to provide access for 
our patients, in the now, so that we can have true help.
    Senator Carper. Thank you so much.
    Chairman Johnson. Senator Paul.

               OPENING STATEMENT OF SENATOR PAUL

    Senator Paul. I would like to start with a question for the 
Chairman. You said that there are plans to try to have this 
pass by unanimous consent?
    Chairman Johnson. I would like to do that next week.
    Senator Paul. OK. And, you have no objections on our side 
of the aisle?
    Chairman Johnson. That is what we are hearing.
    Senator Paul. OK. And----
    Chairman Johnson. Maybe I do.
    Senator Paul. No. Have you heard any public objections on 
the other side of the aisle?
    Chairman Johnson. No, I have not.
    Senator Paul. OK.
    Chairman Johnson. But, I do not have a whole lot of 
cosponsors. I have one.
    Senator Paul. Right. But, I think it is important to know 
that that is coming up, and I think it is important to know 
that our process is somewhat secretive, in the sense that 
people do not have to reveal when they are blocking legislation 
like this. But, it can be pushed, and people can inquire who 
the opponents are, and I think if people are going to oppose 
it, I think they ought to publicly oppose it and, if they have 
reasons, put those reasons forward. But, I think that the holds 
or the blocking of this should not be secret. And, I think 
every attempt should be made to make sure that that is an open 
process and that we know who wants to stop the legislation.
    Chairman Johnson. That is why I will go down to the floor 
of the Senate next week and ask for unanimous consent. And, 
hopefully, whoever has an objection will come down and tell us 
what the objection is.
    Senator Paul. Right. With that being said, I think that 
there are a couple of things to think about if we want to place 
our faith in the FDA doing things in an expeditious way.
    The recent controversy over the epinephrine auto-injector 
(EpiPen) costing $600, well, there has been a generic out there 
that applied in 2009. So, 7 years later, we have not gotten an 
approval, and it is over, I think, frivolous sort of 
complaints. Basically, it injects the drug. It injects the 
right dose. It works. And, yet the FDA is saying, ``Well, it is 
not identical to the EpiPen device.'' And so, for technical 
reasons, it is being held up. At least that is what I have 
heard in the press--7 years. So, I think we should not place a 
lot of faith in some sort of expeditious process. In fact, I 
think the whole FDA process needs to be overhauled, not just 
for people who are terminally ill but for everything. We do a 
miserable job at the length of time of of trying to approve 
drugs in our country. So, if we are going to wait back and say, 
``Oh, well, everything is just swimmingly well, just be 
patient,'' I think that is a big mistake.
    I think the biggest part of the debate here, though, is 
over whether the compassionate use program works. And, I think 
that needs to continue to be driven home, whether it works or 
it does not work. And, I have learned a lot today about whether 
it works or it does not work. I think the fact that we have 
patients calling drug companies and they are not eligible but 
are not showing up in any statistic, it is not measurable, 
maybe, how large the number of ineligible patients is.
    My political director's sister has pulmonary fibrosis. Her 
drug is not approved here. She actually is in a clinical trial 
because she lives in New York. But, if she lived in Bowling 
Green, Kentucky, my guess is it would be a little bit harder to 
get into one of these trials because we do not have a major 
university. But, the drug she is using in her trial has been on 
the market for 10 years in Japan. Why in the world do we not 
use drugs that have been on the market to the general public? A 
Phase III trial might have 1,000 patients. How many people live 
in Japan? A hundred million people? It is a rare disease, but 
maybe there are 10,000 people in Japan using the drug. Why are 
we not using that data? We give the FDA the option to use the 
data, but they choose not to. And, this is the problem. We come 
to this legislation--it comes forward, and I will try to put a 
word in that the FDA should or shall do this, and everybody 
will say, ``Well, let us just give them the option of using it, 
let us trust the FDA to do the right thing.''
    Well, no, the FDA does what they have always done--and that 
is slow-ball and slow-roll things, and so, we have instance 
after instance--we do not have like thousands of instances of 
the FDA saying, ``We got it done''. We have thousands of 
instances of it taking 5 and 10 years.
    But, we also have to look at the whole FDA process. The 
EpiPen has a 38-year patent. Why in the world would we do that? 
They tweak their patent on the device. OK, it delivers it 
slightly faster, quicker, or differently, the needle is 
slightly longer--and we give them another 10 years. The EpiPen 
is 38 years. They got the approval in 1987. The injector was 
actually approved in the 1970s. And, their patent is not going 
to run out until 2025.
    The other thing I have learned, today--or was reinforced 
for me is--Matthew talking about the individualization of 
treatment. We may be entering into an age, if we are not 
already there, where we are never going to have 1,000 people 
get the same treatment because it is going to be individualized 
to their exact genetic disorder if it is some gene that is 
wrong in his body. Maybe the treatment is going to be specific 
to him. There are never going to be 1,000. But, ultimately, the 
way I look at this issue is, look, we live in a free country. 
My goodness, shouldn't people be free to try? And, will it 
always work? No. But, I mean, ultimately, if you have ALS, my 
guess is that you also want trials to go on. It is not like you 
are against trials to go on to see what actually works. But, if 
we prevent the trials and we prevent people from trying, we 
will never know.
    So, I commend the Chairman for putting this forward and 
wish you the best of success.
    Chairman Johnson. Thank you, Senator Paul. I do want to 
point out that Senator Donnelly and Senator Manchin are the two 
Democrat cosponsors we have.
    Mr. Garr, you talked about the ability of ``Right to Try'' 
to accelerate research. Can you just kind of speak to that? 
And, I want to keep this relatively short and then I will give 
Senator Carper a chance. I want to ask that question. But, then 
I also want to get you guys thinking about this. Where is the 
real harm? Obviously, Mr. McFadyen, you are not for this, but 
what is the harm of this? I am not saying it is a panacea. I am 
not saying it is going to work great. You have the problem of 
determining the cost of these things--incredibly expensive. 
But, what is the harm in doing this, in terms of--versus the 
benefit of giving people hope? But, again, talk about the 
accelerated research.
    Mr. Garr. Sure. We did, as I said, 15 patients in the first 
trial and 25 patients in the second trial. If we had delivered 
this drug through a ``Right-to-Try'' opportunity in any of the 
States that passed it, it would have been the exact same 
surgeons doing the exact same surgery with the exact same 
product. In fatal diseases, there are no healthy volunteer 
trials, right? So, every patient, every bit of data you get is 
important. And what I would also say is that no company looks 
at a drug and says, ``Gee, my market for this is the label I 
get from the trial,'' right? The very specific narrow group. 
No. Everyone looks at the development and says, ``OK, this is 
how we can create a trial and get this passed. And then, how do 
we expand the label? '' Right?
    And so, every pharmaceutical company--all of your companies 
in Delaware, right?--all have the same mantra, today, which is, 
``Fail fast.'' Right? They do not want to spend $2 billion in 
10 years to find out it does not work. Right? More data is 
always better. More data is always better. It always 
accelerates research.
    And, I would also just like to point out that there is a 
relatively easy answer. In Japan--and we have moved all of our 
cell therapy there--they adopted a law where, if you have a 
fatal disease, you can do a trial, and as long as it passes the 
safety test and shows some reason to believe it could work--not 
statistically significant efficacy. The trials are not powered 
to show efficacy. But, they have to show safety. They then give 
you a transitional approval. You can then commercialize for 5 
years--even 7 years--while you continue to do trials and 
continue to collect data. That gives access to everybody who 
wants it with a minimum safety net of safety established. And, 
I will tell you, it has taken the whole cell therapy industry 
and turned the map upside down and pushed everyone to Japan. 
Right?
    Now, the 21st Century Cures Act is kind of a kinder, 
gentler, and weaker model of that, but it does not come right 
out and do what the Japanese did. That is the way you solve 
this problem if you really want to.
    Chairman Johnson. And, of course, with ``Right to Try'', 
you only get drugs that have passed Phase I, which is the 
safety standard.
    Mr. Garr. Very safe.
    Chairman Johnson. Assemblyman Calderon, you have been 
trying to jump in here.
    Mr. Calderon. Thank you, Senator. I think there needs to be 
an important distinction made when it comes to access, because 
we are looking at access, right now, in terms of being able to 
get the drug--but that is not necessarily the case. You also 
have to look at access in terms of time. If you are given a 
diagnosis of a year to live with a terminal illness, well, 
right now the new expedited process, all they did was shorten 
the time for the application from 100 hours to 45 minutes. But, 
there is still a 30-day waiting period. And, now say that 
application is returned with a misspelling. Maybe they forgot 
to fill something out. You send it back, and the 30 days starts 
all over again. If you are given a year to live, 30 days is 
your life. And, the point is that with my bill in the 
legislature--in the assembly--you could do that within a week. 
That is why State laws, in terms of ``Right to Try'', are so 
important.
    Mr. McFadyen. And, can I just address that? I am sorry to 
interrupt. It is not 30 days for the FDA to get these 
applications back. Sometimes, it is just overnight. Sometimes, 
for compassionate use, expanded access, it is done over the 
telephone. So, it is not 30 days. They are not going to care 
about a misplaced ``I'' or an ``I'' before an ``e.'' That is 
not how this works. It does get done in a very rapid fashion.
    Chairman Johnson. And yet, can you explain why Dr. 
Delpassand could not expand his own trial to patients that 
needed the therapy?
    Mr. McFadyen. No, with a 3-minute video I cannot.
    Chairman Johnson. Go and look at the 20-minute video.
    Mr. McFadyen. I will. I most certainly will.
    Chairman Johnson. Give him a call.
    Mr. McFadyen. But, what I will say is that he is in the 1 
percent that did not get to move forward. So, when 99 percent 
do, there is access through that. It is not the FDA that we 
continue to vilify here. It really is the matter of the 
companies not providing access.
    Chairman Johnson. Matt, do you have something?
    Mr. Bellina. Yes, Senator, thank you. We are all hovering 
around something--a great example that I really think 
illustrates what we are all talking about here. There is a drug 
out of Japan that has been approved for ALS for about 2 years 
now. It is called Radicut. And, to the credit of the FDA, the 
company applied for a new drug application (NDA) based on 
Japanese data. You have about 10 years of trials and 2 years of 
market. And, the FDA accepted the NDA----
    Senator Carper. I am sorry, NDA?
    Mr. Bellina. New drug application. Sorry, Senator.
    Senator Carper. Thank you.
    Mr. Bellina. So, the company, Mitsubishi Tanabe 
Pharmaceuticals, asked for a 6-month review out of respect to 
the FDA. The FDA came back and said, ``We will look at it, but 
we need 10 months.'' This is a drug that has been essentially 
in humans for about 12 years and, in that 10 months, we are 
going to lose about 5,000 ALS patients. Excuse me. So, you can 
see--I am sorry. I get a little emotional about that. That drug 
should be, in my opinion, already in a Phase IV market study, 
and it really illustrates----
    Senator Paul. And, how far are we into that? How far are we 
into that 10-month cycle?
    Mr. Bellina. We are about 4 weeks in, Senator.
    Chairman Johnson. Does anybody else want to quickly comment 
before I turn it over to Senator Carper?
    [No response.]
    Senator Carper.
    Senator Carper. Mr. Garr, you mentioned that the expanded 
access program at the FDA limits what a company can charge 
patients for treatment. How do ``Right-to-Try'' laws ensure 
that patients can afford their treatments?
    Mr. Garr. Yes. All of the issues around cost and access are 
unresolved on this, but they are no different than they are for 
approved drugs. Right? So, in other words, how do we know that 
everybody can afford Genentech's new vaccine? I mean, we have 
the exact same issues for these unapproved drugs that we have 
for approved drugs. So, yes, there are issues. They are all 
resolvable. There are people in this industry who just spend 
all day, every day working on those answers. I cannot tell you 
right now what our therapy would have cost an ALS patient. I 
know what it costs us to do a trial, right? I cannot tell you 
that. But, the fact is that whether we are doing it under a 
compassionate use, where we have to give it to them basically 
without charging, we have to try and get the surgeons not to 
charge, and we have to try and get the anesthesiologist not to 
charge, and go through that whole process--or if we figure out 
what everybody is going to charge for a particular thing, that 
is no different than it will be when it becomes an approved 
drug.
    Senator Carper. I am going to ask you to hold it right 
there. What help, Mr. Garr, should be offered to patients and 
to pharmaceutical companies to ensure that patients can afford 
these drugs, and then, that companies can sustainably provide 
the treatments that patients are seeking?
    Mr. Garr. I think there are two basic things that have to 
happen. First, companies have to be able to charge for it--and, 
yes, there will be companies that still will not do it. There 
will be companies that will not offer their drugs. There is 
nothing in this Act which forces companies to offer their drug 
under compassionate use or under ``Right to Try.''
    And, the second thing is there are, I believe, the last 
time I looked at it, about a dozen States that have already 
passed laws that require insurance companies to cover 
experimental treatments for certain types of cancer. So, if you 
are in a cancer trial in Kansas, for instance--right?--and it 
is at least a Phase II trial and you have to pay for it, it is 
just like it was an approved drug, in terms of the insurance 
company. I think that is the second part of the answer. Again, 
the market has to work. The insurance industry, we have all 
kinds of people working on drug pricing and approvals 
throughout this entire industry. And, I would add that we are 
an industry that is 100 percent based on the presumption that 
all of these issues can be addressed. People may not like the 
way they are addressed. Not everybody is happy with the 
answers. But, in the end, who gets drugs, what they are paid 
for, and who pays for them--we work that out for every approved 
drug on the market. So, there is no reason we cannot work that 
out for unapproved drugs also.
    Senator Carper. Thank you very much.
    Mr. McFadyen, you have worked on behalf of patients. You 
mentioned patients and families. As a patient advocate, I just 
want to thank you for what you do. And also, Ellen, for your 
support as well, as his wife, trying to help individuals gain 
access for rare disease treatments. Have you ever worked with a 
patient who has received drugs under a ``Right-to-Try'' law?
    Mr. McFadyen. No, not at all. And, that is what we continue 
to say, that there is no concrete evidence of patients ever 
receiving a medication under ``Right to Try'' in the United 
States that they otherwise would not have received under 
expanded access laws. 
And, there are 137 million people that have access--supposed 
access--under ``Right to Try,'' and there is no evidence that 
it is working--that it is moving forward. So, in my personal 
experience, no.
    I can tell you that, the disease families that I 
represent--the rare disease folks--many of them are watching 
the live feed right now, and we are all united in the belief 
that, should legislation be passed, today, the landscape for 
them looking at access to medications, tomorrow, will not have 
changed. It is not the programs that are the issue right now. 
It is the companies and their fears under expanded access and 
adverse events--but also under ``Right to Try.''
    Senator Carper. Just a follow-up question, if I could, Mr. 
McFadyen. What has been your experience, if any, in working 
with the FDA on behalf of patients seeking access to 
experimental medicines?
    Mr. McFadyen. Yes, I have actually had a very good working 
relationship with the FDA--they have been very responsive to 
our needs. Richard Klein is the patient support liaison 
director at the FDA, and he returns emails--actually, I was 
shocked at how quickly I got responses from them, at first, and 
I am not anymore. They get information back to us very quickly. 
They help walk us through situations where we need information 
and that sort of thing. They are currently putting in place 
sort of a personal concierge service to help physicians fill 
out that 45-minute form to make sure that it is done right so 
that they can have that on-the-phone conversation review--an 
overnight review type of thing. And, from what I understand, as 
well, your next witness, Dr. Lurie, is seen within the patient 
advocacy community as somebody who is a patient advocate at 
heart with the FDA and who really wants to try and advance 
access to medications for my kids--for our patients--as fast as 
possible.
    Senator Carper. I understand that Johnson & Johnson 
recently established a new program to help patients secure, I 
think, number one, information to experimental treatments but 
also access to those experimental treatments. I do not know if 
you are familiar with their program, but if you are, could you 
talk about it, please?
    Mr. McFadyen. Yes, actually, Johnson & Johnson looked at 
this issue of access to medications long and hard and 
understood that things needed to move forward quickly. And so, 
what they put in place was something they called ``CompAC.'' 
They take all of the expanded access and compassionate use 
requests for one certain drug. It is a trial program right now 
that has just run its course and it is going to be expanding 
soon. And, they take those applications, and they send them on 
to a group at NYU Medical Center that looks at these 
applications. The applications are free of bias, so 
decisionmakers in that 10-person committee, which is made up of 
medical ethicists, researchers, physicians, and that sort of 
thing--Art Caplan leads that group. They look at those 
applications, and they make a recommendation back to J&J on 
whether a patient should be approved or denied access to that 
experimental drug.
    Last year, alone, in the 6 months that it was on, I think 
they took in 100 or so applications. Sixty two of them were 
approved. Sixty of them were recommended by the committee to 
move forward back to J&J, and after that recommendation for 60, 
two more got in a little bit more medical information to the 
company, and they were approved. The other ones that were not 
approved were not approved because the patients had not 
exhausted all other avenues of available treatments.
    And so, they used this program to make it extremely 
expedited and free from bias from pharmaceutical eyes based on 
costs, etc. And, they allowed others to recommend for them. It 
could be a pivotal turning point for companies, with respect to 
access to medications, because if this is a model that they can 
use and use successfully--a big company like J&J--and they are 
going to expand this process in the very near future for other 
drugs and that sort of thing. That is what we really need to 
do.
    Taking that example, I know BioMarin Pharmaceuticals just 
announced an early access program for a disease that I actually 
deal with quite extensively, Batten disease. Batten disease is 
one of the worst childhood diseases I have ever seen. Over the 
course of a year or two, patients lose all mobility, all access 
to their extremities, and they enter a vegetative state and 
pass away very quickly. BioMarin, after Phase I-II, opened up 
an early access program. They are trying to get as much of the 
drug out as is feasibly possible for these patients, and they 
are taking a similar path forward to J&J by allowing the 
investigators to have their own committee and their own set of 
ethics and criteria. So, they are not making the decisions 
anymore--and I am seeing this happen more and more with 
pharmaceutical companies looking at the problems that currently 
do exist with access to medications.
    Senator Carper. Thank you so much.
    Chairman Johnson. Senator Paul.
    Senator Paul. I just have a really quick question. I wanted 
to reemphasize something Mr. Garr said and make sure I 
understand it. Under the compassionate use program, no money 
can change hands--not even for costs or anything?
    Mr. Garr. No, there are mechanisms for recouping some 
costs, but the way they define cost is very different than the 
way a company thinks of it. A good example is ours. We 
literally have to send a cell technician up to Charles River 
Lab in Pennsylvania, have him work there for a day, do what he 
has to do, and ship the cells somewhere else, right? That is a 
very tiny part of the opportunity costs we lose when our Chief 
Science Officer (CSO) has to spend 24 hours being a cell 
technician.
    Senator Paul. Right. And, I guess the only other point I 
would like to make, quickly, is that, if you allow for 
profitability, I am all for that because profitability does 
drive innovation. There was an economist after World War II, 
Joseph Schumpeter, and he put it this way: ``The miracle of 
capitalism is not that queens have silk stockings, but that 
factory girls do.'' But, the way that is driven is, initially, 
only the queen may be able to afford it. And so, allowing money 
to go into the development of drugs, both through individuals 
or through companies, is a good thing. When calculators first 
came out, no poor person could afford them. Now, you are 
virtually given a calculator with your phone, basically. When 
most things are innovated--Lasik surgery came out, it was very 
expensive. Only the rich could afford it. But within years, if 
you allow capitalism to work, the price comes down. But, the 
driving force of innovation is allowing capitalism and pricing 
to work. And so, I think that is an important distinction 
between what we are talking about here and the compassionate 
use program--and a limitation of the compassionate use program.
    Thank you.
    Chairman Johnson. Thank you, Senator Paul. I would also 
point out, if you are talking about costs, it would be nice if 
it did not cost, on average, $2.6 billion to bring a drug 
successfully to market.
    Just really quickly, if anybody has a final--OK, but 
quickly.
    Mr. Calderon. Thank you, Senator Johnson. Just three quick 
points.
    In terms of the 30 days of the application process--the new 
expedited application process with the FDA--it is 30 days if 
the FDA has any questions of the physician or the manufacturer.
    In terms of statistics regarding ``Right-to-Try'' 
legislation, today, any statistics that you would gather from 
``Right-to-Try'' laws, today would be, I believe, inappropriate 
to use because, when I introduced this bill last year, there 
were less than 10 States that had this on the books. Now, there 
are over 30. So, you would not have any properly measurable 
data to look at.
    Chairman Johnson. And, until the Federal legislation 
provides that overall protection, they are not going to work. 
It does not surprise me at all. There are not very many people 
like Dr. Delpassand who are willing to take that risk.
    Mr. Calderon. Right. And, in terms of costs, well, what is 
the other alternative, having to move to another country in 
order to get access to these drugs? And so, in terms of costs, 
well, yes, it is going to be more expensive even if you have to 
move to another State that has ``Right-to-Try'' laws on the 
books. It is better to stay in your own State to have that 
opportunity to do it at home.
    Chairman Johnson. Quickly, anybody else? Mr. Garr. Oh, I am 
sorry. Mr. Neely.
    Mr. Neely. Yes, I think, just after this access, this is an 
avenue that we need. Patients need it and the doctors need it. 
Thank you.
    Chairman Johnson. Matt.
    Mr. Bellina. Yes, Senator. I think that Andrew has done a 
really good job of laying out the obstacles, and I think there 
really is a lot of work that is going to need to be done even 
after the passage of this legislation. But, I think that you 
brought up a great point. What is the overt risk? What is the 
downside of this legislation? I think, you don't not try for a 
first down because it is not in the end zone--if anybody is 
into football. So, that would be my one question.
    Chairman Johnson. Thank you, Matt.
    Just very briefly.
    Mr. McFadyen. It will be very brief. Senator Paul is no 
longer here, but I did want to address the idea of access. With 
all due respect, my son can wait until he can save up and 
afford a calculator. He cannot afford $200,000-a-year or 
$300,000-a-year drugs that can be charged under ``Right-to-
Try'' legislation. Direct costs are direct costs and providing 
access should not be about making money.
    Chairman Johnson. Again, I just want to thank all of the 
panelists and I appreciate your testimony. With that, we will 
call our next panel, Dr. Lurie. Thank you.
    Senator Carper. Thank you all.
    [Pause.]
    Chairman Johnson. Dr. Lurie, it is our tradition to swear 
in witnesses, so if you will please rise? Do you swear the 
testimony you will give before this Committee will be the 
truth, the whole truth, and nothing but the truth, so help you, 
God?
    Dr. Lurie. I do.
    Chairman Johnson. Please be seated.
    Our next witness is Dr. Peter Lurie. He is the Associate 
Commissioner for Public Health Strategy and Analysis in the 
Office of the Commissioner at the Food and Drug Administration. 
Prior to that, Dr. Lurie was Senior Advisor in the Office of 
Policy and Planning. Before coming to the FDA, he was deputy 
director of Public Citizen's Health Research Group. He had an 
earlier academic career at the University of California, San 
Francisco (UCSF), and the University of Michigan (UM). Dr. 
Lurie.

     TESTIMONY OF PETER LURIE, M.D., M.P.H.,\1\ ASSOCIATE 
COMMISSIONER FOR PUBLIC HEALTH STRATEGY AND ANALYSIS, FOOD AND 
   DRUG ADMINISTRATION, U.S. DEPARTMENT OF HEALTH AND HUMAN 
                            SERVICES

    Dr. Lurie. Good morning. Mr. Chairman, Ranking Member 
Carper, and Members of the Committee, I am Peter Lurie, as you 
have heard, with the Office of Public Health Strategy and 
Analysis at the FDA. Thank you for the opportunity to be here 
to discuss expanded access to investigational products.
---------------------------------------------------------------------------
    \1\ The prepared statement of Mr. Lurie appears in the Appendix on 
page 307.
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    As a physician, I have personally witnessed the suffering--
and we have heard much about that today in really heart-
wrenching testimony--and I have witnessed the dilemmas facing 
patients and their families when they are confronted with 
serious or life-threatening conditions and have limited 
treatment options. In these circumstances, investigational 
products may be their only hope and the FDA recognizes that. In 
many instances, patients with life-threatening diseases are 
more willing to accept the risks associated with 
investigational products than other patients would--especially 
if they have no other available options.
    And, that is why, for over two decades, the FDA has had in 
place a system to help patients gain access to investigational 
products. And, it is functioning well. The treating physician 
must first approach the pharmaceutical company. If the company 
agrees to the physician's request, the physician can then apply 
to the FDA for permission to proceed. Should they do so, they 
are highly likely to be allowed to proceed. The FDA has 
authorized more than 99 percent of single patient expanded 
access requests between 2010 and 2015. And, despite what we 
have heard, emergency requests are usually granted immediately 
over the telephone and non-emergency requests are processed in 
a median of 4 days. The 30 days means that the application can 
proceed without the FDA if we exceed 30 days. It does not take 
30 days. It takes either the same day for emergencies or a 
median of 4 days for non-emergencies.
    Now, access to investigational products requires the active 
cooperation of the treating physician, the FDA, and the 
industry. It appears that pharmaceutical companies turn down 
considerably more applications from physicians than does the 
agency. Mr. McFadyen spoke to this when he gave you the data 
from the Johnson & Johnson experience. They have turned down 98 
applications for one drug in a 6-month period. Now, the FDA has 
turned down 66 applications--fewer, for all of the drugs that 
are out there, among the thousands that it has received. So, 
they have turned down 98. We have turned down only 66. And so, 
on this, I think Mr. Garr's testimony was very much on point as 
well.
    Now, we continue to work avidly to improve the expanded 
access program because everything can be improved. The FDA 
established an expedited telephone process for daytime and 
after-hours emergency requests for expanded access--and you 
have heard about the success of that program. In 2009, we 
revised the application regulations to make the process and the 
responsibilities of physicians clearer.
    In June 2016, in response to feedback from physicians that 
completing the two expanded access forms was time-consuming, 
the FDA developed and released a new simple form for individual 
patient expanded access--and here it is. And, I am proud to 
say, I led the group that pulled this together. The form is 
estimated to take only 45 minutes to complete and requires just 
a single attachment, whereas, the previous form required up to 
eight.
    At the same time that we put out this new form, we released 
step-by-step instructions on how to complete it and two 
additional guidances--one of which addressed the charging 
issue. Simultaneously, we revamped our expanded access website 
and we produced fact sheets for physicians and patients.
    However, even patients with serious or life-threatening 
conditions require protection from unnecessary risks, 
particularly because, in general, the products that they are 
seeking through expanded access are unapproved--and may never 
be approved. Moreover, the FDA is concerned about the ability 
of unscrupulous individuals to exploit such vulnerable 
patients--and we see this. Thus, with every request, the FDA 
must determine that the potential patient benefit from the 
investigational drug justifies the potential risks, in the 
context of the disease, to be treated. And, that is why, even 
as we permit more than 99 percent of applications to proceed, 
we make meaningful changes to about 11 percent of them, 
generally to ensure patient safety, including changes in 
dosing, safety monitoring, and informed consent.
    The FDA's expanded access process strikes a careful balance 
between helping to facilitate patient access to investigational 
therapies and the need to protect patients and promote the 
public health through science-based regulations--as Mr. Bellina 
points out. Upsetting this balance has the potential to expose 
patients to unreasonable risks and stymie the development of 
medical products that could benefit us all.
    To sum up, the FDA's expanded access program allows almost 
all applications to proceed. It improves many of the 
applications that we receive and it does so expeditiously. At 
the same time, it protects vulnerable patients from potential 
harm from drugs that may not be effective and from exploitation 
by unscrupulous individuals. It also maintains the integrity of 
the clinical trials process because, in the end, the very best 
way to hasten access for patients to safe and effective drugs--
for the largest number of patients, not just those in expanded 
access programs--is to get the drug approved.
    That concludes my comments. I am happy to take any 
questions you may have.
    Chairman Johnson. Thank you, Dr. Lurie.
    You talked about undue risk. It is true, under this bill, 
that these drugs will have already passed Phase I, which has 
basically certified the safety of the drug. Can you reconcile 
those? What is the added risk if the FDA has already said it is 
a safe drug?
    Dr. Lurie. We have not, sir. At the end of Phase I, we have 
only seen a few dozen patients being treated. And so, although 
we have some preliminary information about safety, we are far 
from certain that this is a safe drug. We still have Phase II 
to go through, where we gather additional safety information, 
followed by Phase III, in which we get still more. So, Phase I 
is a very long way from establishing effectiveness and it is 
also a long way from establishing safety.
    Chairman Johnson. Well, you never completely establish 
safety. I mean, even once a drug is totally approved, you are 
going to continue to do that monitoring.
    Dr. Lurie. Certainly, but there is a world of difference 
between a couple of dozen patients and the many hundreds to 
thousands who will, ultimately, be exposed during Phase III 
trials.
    Chairman Johnson. Can you explain why Dr. Delpassand was 
not able to increase the number of patients he was able to 
treat? Now, here is a situation where the manufacturer has 
actually agreed to provide the drug.
    Dr. Lurie. Right.
    Chairman Johnson. I would assume--it seems to be the real 
stumbling block in whether it is expanded access or ``Right to 
Try'' that--because companies do not have protections against 
adverse effects--they do not have liability protection. I want 
to talk a little bit about the restrictions, in terms of being 
able to charge patients. But do you know what is pulling off, 
in terms of down there in Texas?
    Dr. Lurie. I really cannot speak to that, sir. I have only 
seen the video for the first time this morning, so I cannot 
speak to the details of that. And, as I think you understand, 
these are pre-market drugs and the FDA is restricted in its 
ability to be able to discuss them.
    Chairman Johnson. Can you understand just the human 
frustration, though, when you have a doctor apparently, again, 
working with patients, having access to a drug, treating, 
getting up to his limit of 150 patients, and having another 
almost 100 patients there that he thinks can benefit--and, I am 
sorry, bureaucrats in Washington, D.C., saying, ``No, we are 
not going to let those extra 100 patients have that hope'' ? 
Can you speak to that?
    Dr. Lurie. Yes, well, in principle, I could understand 
that. I cannot speak to his situation, but I can speak to the 
general situation, which is that, 99 percent of the time, when 
a doctor tries to get access for their patients, they succeed. 
And, the times that they do not, I must tell you, are really 
outlier situations. I mean, those are the situations where we--
meaning our medical reviewers, who know as much as anybody 
about the drug, right? They know more than the doctor because 
we have seen the information coming in, perhaps from foreign 
countries, and unpublished results--we see that stuff. So, we 
may have additional information, and so, based on that, we may 
believe the drug is unsafe. Based on that, we may believe that 
there is no reasonable hope that the drug will work. Or, we may 
know that the company is not making the drug in a way that is 
safe for patients.
    Those are the outlier reasons. That is 1 percent, right? 
Most of the time they are going through, but, when they do not, 
it is usually a fairly flagrant thing because we do not 
exercise that authority very often.
    Chairman Johnson. What is the FDA's intention, in terms of 
how it deals with someone like Dr. Delpassand, who is operating 
under Texas' ``Right-to-Try'' law? Is there going to be any 
enforcement action against doctors that have the courage to do 
that?
    Dr. Lurie. Yes, well, Senator I cannot really speak to what 
enforcement action we might take. We are a science-based 
organization--and we are interested in data. And, to the extent 
that data is generated--and that it might come to our 
attention--we are interested in the data that there might be.
    Chairman Johnson. In your testimony, you use the phrase 
``exploit vulnerable patients.'' Can you talk about what you 
mean by exploiting vulnerable patients--people, like Matt, who 
are trying to get access to a drug to give themselves some 
hope?
    Dr. Lurie. Yes. Look, we understand that patients, like 
Matt, are looking for hope. We understand that. We understand 
how desperate they can be. But, it is that very desperation 
that makes them vulnerable to exploitation. All someone needs 
to do----
    Chairman Johnson. Can I say, who are you--who is the FDA--
to make that decision for them?
    I am sorry. Who are you to tell Matt that you are going to 
protect him from exploitation when he has no further hope?
    Dr. Lurie. Well, Senator, with respect, I feel like that is 
the very responsibility with which this Congress has charged 
us. I think we have been asked to look after patients--and we 
feel that that is exactly the responsibility that we are 
exercising.
    Chairman Johnson. I have no further questions.
    Senator Carper. I want to dwell on the last question that 
the Chairman asked. In your testimony, I thought I heard you 
say the words ``protect patients against unscrupulous 
individuals who might want to take advantage of those people.'' 
Do you have any examples of that you might be able to share 
with us, either on the record or here, today?
    Dr. Lurie. Well, Senator, as I said, the problem is that we 
cannot go into specific applications. But, I think that someone 
just has to take a look at the Internet and see the kinds of 
things that are being hawked there, and how often they focus on 
very desperate patients, to realize that this is a very real 
issue.
    Senator Carper. Alright. Thank you. Matt raised, earlier, 
when he was testifying--I think it was Matt who mentioned an 
ALS drug that had been approved, in Japan, maybe a couple of 
years 
ago--2 years ago--and that there was an effort to see if that 
drug could be made available here. I think, initially, the FDA 
said it needed 6 months to look at this--6 months--and then, 
they said, ``No, we need 10 months.'' Could you just talk about 
6 months as opposed to 10 months--and, when you have some 
country, like Japan, that has been allowing a drug like this to 
be used for a couple of years? What is the approval process 
like at the FDA? How can we expedite that? Or can we?
    Dr. Lurie. OK, so I think there are two issues. First is 
the 6 months versus the 10 months. So, those are goals that are 
in what is called our ``user fee legislation.'' They are the 
product of negotiation between the FDA and industry. And, there 
is a priority review process and a standard review process. The 
priority review is for those that would be expected to show a 
particular benefit--and that is 6 months. Otherwise, it is 10 
months. About half go through each. That is the rough 
proportion between the two. That is legislation, in the end, 
that is ratified by the Congress--so we follow those. We have 
to get 90 percent--best we can--90 percent of the applications 
for priority review in 6 months and for standard review in 10 
months. And, we have met those goals now for a number of years.
    So, that is the way it works. There is a definition. The 
FDA has guidance on it that explains the difference between the 
two. And, I expect that we did our best to follow that guidance 
in deciding how to assign that particular drug.
    Now, with respect to the issue of Japan or foreign 
countries, in general, I think, there is an old story about how 
the FDA is slow and how drugs are on the market in foreign 
countries and American patients cannot get access to them. That 
is a very old story and, frankly, an outdated story. It just 
simply is not true.
    Now, 57 percent of all drugs in the last several years that 
have come on the market have come on the market in this country 
first. Now, there are about 200 countries in this world, and--
--
    Senator Carper. That is over half of all of the drugs?
    Dr. Lurie. Yes, over half of them. They are in 200 
countries and one country is first most of the time.
    Senator Carper. And, that is us?
    Dr. Lurie. And, that is us. And, just to be colloquial 
about it, if you go back to the Olympic Games and you look at 
how the Americans did, everybody said, ``Well, what a great 
success. The Americans brought home this treasure trove of gold 
medals,'' which they did--and everybody is proud of that. But, 
they only won 12 percent of the gold medals. I mean, the FDA is 
doing better than that.
    Senator Carper. OK. Thank you.
    Dr. Lurie, let me just ask--the FDA has released 
information to clarify that outcomes in expanded access cases 
are not treated the same way outcomes from clinical trials are 
treated. Do adverse events from expanded access cases 
ultimately affect the final outcome of the FDA's review of that 
drug? The FDA has approved almost 100 percent of expanded 
access applications, as you have mentioned. But has the FDA 
ever put a hold on an ongoing clinical trial as a result of an 
adverse event from a patient using expanded access? And, how 
quickly were these holds resolved?
    Dr. Lurie. OK. So, let me answer that question sort of 
generally and then specifically.
    The general answer is that our folks--our medical officers, 
who are experts at reviewing drugs--they understand that the 
expanded access situation is very different from the 
conventional clinical trial situation. The patients who are in 
it are different in expanded access. They are more likely to be 
sick. They are more likely to have other conditions. They are 
more likely to be on other drugs. There is no comparison 
group--right?--in general, in the expanded access program as 
well.
    So, we know that any particular adverse event that comes in 
should be treated, in most cases, as an anecdote and should be 
afforded the appropriate weight, which, frankly, is not a very 
high weight, because it is just not--it is so atypical. And so, 
for that reason, I think that people should place some trust in 
the fact that medical officers can distinguish between these 
two very different sets of circumstances.
    Now, we know that people are worried about it. We have 
heard this concern raised several times during the previous 
panel. And, in fact, in one of the three guidances that I 
mentioned we released back in June, we addressed this question, 
specifically, in the so-called question and answer (Q&A) 
guidance. It is one of the three. Question 25 is about exactly 
this question. We explain how the FDA's medical officers 
understand the context and will apply the appropriate weight. 
Now, that is the general answer.
    The specific answer is that we took this question seriously 
enough that we tried to gather some actual data. We went back 
10 years and we looked at all of the applications that had come 
in. There were about 11,000 of them. And, they were--I am 
simplifying, slightly, here--for about 1,000 drugs--1,033, to 
be exact. And, we asked ourselves how many times had an adverse 
event that occurred in expanded access resulted in what we call 
a ``clinical hold'' on an application--right?--an ongoing 
manufacturer's application. Out of 1,033, over 10 years, we 
found two. OK? They were partial holds. Once the issues were 
resolved, then the program was allowed to continue.
    So, we do understand this issue. We would not overweight 
these anecdotal pieces of information. But, we need to have 
that information all the same. OK?
    What we do not want is a circumstance where the FDA does 
not receive an adverse event report. Or, if they receive it, 
they are forced to ignore it. I mean, for a scientific agency, 
it is just terrible for us to have, perhaps, in rare cases, 
valid scientific information and then, have to ignore it. Let 
us say the drug comes on the market, and then, after approval, 
when thousands of people start to get the drug, that very same 
adverse event now occurs and we knew about it back then, but we 
could not address it? I mean, we will probably be right here 
before this Committee again.
    Senator Carper. Thank you. If this legislation is not--it 
is well intentioned, very well intentioned legislation.
    Dr. Lurie. Yes, sir.
    Senator Carper. If this is not the answer to this 
challenge, this conundrum that we face, what should we do that 
we have not done?
    Dr. Lurie. Well, I think that some people have talked about 
some transparency on the part of the pharmaceutical industry. I 
do think that would be helpful. Johnson & Johnson has been a 
leader. I think more and more we are seeing companies put their 
expanded access policies, either in general or with respect to 
particular drugs, on their websites. I think those things 
really help. We are, as some others mentioned, exploring the 
possibility of a navigator to help folks get through the 
process.
    As was also said, we have folks who are committed to doing 
that--who are dedicated to just that--day in and day out. And, 
they hold people's hands through this process. They understand 
which companies have drugs that are available through expanded 
access. They understand which parts of the FDA have 
jurisdiction over that drug. They understand what form needs to 
be filled out and how to do it. And, they hold your hand 
through the whole process.
    So, we are reforming ourselves, internally, to make things 
better. The form is the biggest symbol of that, to be sure. 
And, I should say, parenthetically, 100 hours--I mean, as was 
pointed out, the form was kind of a repurposed form. It had 
been for commercial purposes before. And, our form indeed said 
100 hours. But, nobody believed that it actually took the 100 
hours. I mean, do you know any doctor--or does your doctor ever 
spend 2\1/2\ weeks on you? Right? A hundred hours? It is 
inconceivable. So, that was never happening. OK? But now, all 
the same, we took the criticism seriously. And, to the extent 
that that misinformation had dissuaded people from applying, we 
have gone and reformed the form. It took a bunch of work. It is 
now done and people are starting to use it.
    Senator Carper. Mr. Chairman, I would just say that the 
folks at the FDA have a hard job. A hard job. Thank you for 
doing it.
    Chairman Johnson. Senator Lankford.

             OPENING STATEMENT OF SENATOR LANKFORD

    Senator Lankford. Dr. Lurie, thank you for being here. Let 
me pick up where you just left off there with the 100 hours for 
the form. It is my understanding that the 100 hours was not the 
time needed to complete the form, rather, it was the time 
needed to gather the information required for the form. The 
form was pretty straightforward, just as a checklist, but the 
form implied that it would take 100 hours to gather the 
information needed for the form. Is that accurate or not 
accurate?
    Dr. Lurie. That is right. Under the Paperwork Reduction Act 
(PRA), when we make these estimates, the estimate that now 
says--on the back of this form, 45 minutes, it is not just, 
literally, filling it out. It is all of the associated 
materials, all of the attachments, and so on and so forth. And, 
I want to tell you, I took a look at that form. I am a 
physician and I work at the FDA. And, I thought, if you are a 
medical doctor (M.D.) out there, who is working hard and 
dealing with expanded access on a frequent basis, it was pretty 
intimidating. I agree with that. And, that is why we pulled 
together this group--exactly to address that concern.
    Senator Lankford. So, the 45 minutes is not just completing 
the form. It is gathering information----
    Dr. Lurie. Oh, it is everything.
    Senator Lankford. It is everything required.
    Dr. Lurie. It is everything. And, whereas before, there was 
a lot of additional information to collect----
    Senator Lankford. Right, because it was just a two-page 
form before as well.
    Dr. Lurie. Right.
    Senator Lankford. So the form is not longer or shorter. The 
difference is the amount of information required to get in it?
    Dr. Lurie. It is, in fact, shorter, in the sense that there 
used to be 26-odd fields and it is down to 11 fields.
    Senator Lankford. Right.
    Dr. Lurie. What we did was we took all of the information 
that was in the attachments and we brought them right into the 
form. So, it is now one-stop shopping essentially. The only 
thing that you now need to attach--and there used to be eight 
attachments. The only thing you now need to attach is the 
letter from the company. Right? And, of course, you have to get 
that first--and we have already heard, repeatedly, about the 
difficulties that doctors sometimes have in securing that, from 
the companies, for their patients.
    Senator Lankford. OK. Talk to me about the internal 
conversation about acceptable risk. This has been one of the 
conversations to say at what point, as you go through the 
studies--one, two, and three, and the whole process--is the 
number, the percentage, there, of risk. So, help us understand 
that better.
    Dr. Lurie. Well, certainly there is no way to discuss it, 
in percentages per se, but what someone can say is that people 
at the FDA understand that risk is something that varies 
according to the severity of the illness and the availability 
of other successful treatments for that condition. So, when we 
come up with something that is invariably fatal, we treat that 
differently. If we, instead, are presented with a drug that is 
to treat allergic rhinitis--right?--that is a totally different 
matter. And, this whole program is about serious and life-
threatening illnesses for which there are no acceptable 
therapies as an alternative, right?
    So, we are already in that box from the get-go, here, and 
so, it is not a very high bar--and that is expressed, 
mathematically, as the 99-percent approval rate.
    Senator Lankford. Right. But, you were discussing, earlier, 
while we are in this box, the difference between the efficacy 
and then the safety issues as well--and talking about the first 
stage of it, then getting on to the second stage, we are 
getting more and more on efficacy and its own effectiveness and 
trying to evaluate that. At what point do you see through this 
process that someone is terminally ill and begins to see some 
effect that the risk outweighs that? That is pretty much the 
crux of this conversation.
    Dr. Lurie. Right.
    Senator Lankford. When the first level is done and patients 
hear the stories--or people that are not patients hear the 
stories--this seems to be effective, but then there is the 
pause to say, ``We are going to go through multiple other 
areas.'' How can we quickly get people in there that want to be 
able to take a higher risk knowing that stages two and three 
have not been done?
    Dr. Lurie. Yes, well, again, the answer is almost always. I 
mean--99 percent--we almost always, in this bucket, are going 
to say that the potential for benefit outweighs the risk. It is 
in the very rare circumstance that we say no.
    I would caution, though, that there is a lot of information 
that is out there about the claimed effectiveness of drugs--
some of which gets a lot of press attention--and some of that 
simply is not so. Some of it is just not accurate information. 
And, it ends up creating a lot of noise, which is very 
difficult for us to refute.
    Senator Lankford. What about the double blind studies? Once 
you start going through the process on a terminal illness and 
you deal with those folks that are getting the placebos and you 
see the individuals that are getting the drugs and see a rapid 
response.
    Dr. Lurie. Right.
    Senator Lankford. How do you all deal with the moral issues 
and the ethical issues of leaving those that are getting the 
placebo--knowing that they are in a terminal status, as opposed 
to trying to shift them over?
    Dr. Lurie. OK. So there----
    Senator Lankford. This is a science versus ethics 
conversation.
    Dr. Lurie. I understand, absolutely, and so, there are kind 
of two elements to that. First is outside of the FDA, and the 
other is inside of the FDA. The outside of the FDA is that, in 
any significant randomized controlled trial, with or without a 
placebo, there is a committee that meets on a periodic basis 
called the Data Safety Monitoring Board (DSMB). And, they take 
a peek at the data, maybe every 6 months, to see what is going 
on, because what you would not want--as I think you are 
concerned about--is that people were in some long-term trial 
going on for years and years and years, and it turns out that 
back at 6 months you knew that people were getting seriously 
damaged by the drug and it continued for 2\1/2\ years more. Or, 
that after 6 months, there is such incredible evidence of 
effectiveness that you might as well have stopped right there 
and then, and you did not, and years went by, and people were 
still in the placebo group and the drug was not approved, 
right? Nobody wants that.
    So, these Data Safety Monitoring Boards look in 
approximately every 6 months. They have rules about on which 
grounds they will stop the trial. And, if you meet that 
threshold, the trial is stopped, the blind is broken, and the 
data is made available. Right? So, that is how that works.
    Now, with respect to the FDA, it does not happen especially 
often, but it does happen. There are some drugs that are just 
gangbusters, right? Many of the drugs we have, they help, but 
then, occasionally, you come along with something that just is 
hugely beneficial. And, we have a category for them. We have 
four expedited programs, and one of them is called 
breakthrough. And, if you turn out to have a drug that does 
that--something that is really so remarkable--then you get the 
breakthrough therapy, and with that comes a series of 
advantages that should get you to market sooner, because the 
last thing the FDA wants is to have in its files some drug that 
could make a huge difference to patients and people not getting 
it. That would be terrible.
    Senator Lankford. OK, so the 6-month time period, 
obviously, for someone who has 1 year or 2 years--or they are 
feeling profound effects of the drug that is diminishing, 
whether it is the ability to walk or the ability to be able to 
speak, whatever it may 
be--the 6-month time period on the double blind to be able to 
come back and evaluate it seems like a long time in those 
situations.
    Dr. Lurie. No. That is a different matter, Senator. So, the 
6 months is the amount of time--now the trial is done where we 
start talking 6 months. The trial is done. Perhaps, it has been 
stopped by the DSMB, perhaps not. But, the 6 months is from the 
time that the application is presented to the FDA until the 
time at which we make a decision. OK? That is a different 
matter and that does take a certain amount of time. It used to 
be, when these things were not done electronically, we would 
get literally a roomful of boxes of information, right? Huge 
amounts of stuff that we had to make our way through. And, I 
should say, parenthetically, that the FDA is the only agency in 
the world who gets that raw data, right? There is nobody else 
in the world who does.
    Senator Lankford. But what I am trying to figure out is 
while the study is ongoing----
    Dr. Lurie. Right.
    Senator Lankford. Maybe I misspoke on this earlier. While 
the study is ongoing and you are seeing an effect for those 
that are receiving the drug and they are receiving benefit--
their speech is improving, their muscular function is 
improving, their organs, or whatever it may be--and those that 
are in the placebo are not.
    Dr. Lurie. Right.
    Senator Lankford. And, you see a significant number there--
I am talking about during the study--the ethical conversation 
to say, ``I have people in this study that are terminal,'' and 
that you are allowing the study to be able to go through, to 
the end, when you see an obvious effect.
    Dr. Lurie. Right. But, what I am trying to explain is that 
the FDA is not involved, in general, at that point, right? This 
is the company who has sponsored the trial----
    Senator Lankford. But, does the FDA require a double blind 
study to be able to go through the process?
    Dr. Lurie. Well, it depends on the nature of the drug, the 
condition, et cetera, et cetera. But, the point is that there 
is a control, and that control is the DSMB. And, the people who 
hire the DSMB, in effect, are the companies whose drug would 
come to market. So, the conversation that you are worried 
about--and it is a completely reasonable one, and it is what 
the DSMB is there to address--is something that is taking place 
before the product is presented to the FDA. Right?
    Senator Lankford. Right.
    Dr. Lurie. It is with the company at that point.
    Senator Lankford. Right.
    Dr. Lurie. And, the very concern that you are worried about 
is what the DSMB is for.
    Senator Lankford. Well, I am concerned that the FDA has a 
requirement and that they are trying to fulfill that 
requirement to be able to get the drug to market, but that it 
may inadvertently doom some child or some adult to be stuck in 
a situation where they are receiving the placebo when there is 
a benefit----
    Dr. Lurie. No, the FDA wants----
    Senator Lankford. I understand that it is a rare thing to 
be able to see a rapid benefit like----
    Dr. Lurie. Right. No. The FDA, wants there to be DSMBs. The 
FDA does not want people to go out and collect data for longer 
than is necessary to establish safety and effectiveness. And, 
if we can figure it out even earlier than expected, that is 
wonderful. We say, ``stop the trial, please present the data, 
and come to the FDA.'' If it is that great, there is a fair 
chance it will wind up in priority review. But, we do not want 
patients stuck in clinical trials that are just adding new 
patients--right?--and not really any meaningful new information 
about safety and effectiveness. If that is what is happening, 
we want the trial stopped. We want to see the data as soon as 
possible.
    Senator Lankford. Mr. Chairman, may I ask one more question 
on this?
    Chairman Johnson. Sure.
    Senator Lankford. My question is about the toolkit 
licensing that has been proposed out there when you are dealing 
with a drug that has been permitted for a certain organ, but, 
instead, opening it up for a certain type of treatment, for 
instance, a cancer treatment. So, it is actually a certain 
cancer. It has been approved for, maybe, the stomach, but this 
is trying to move to another 
area--and I have heard this ongoing conversation. I will tell 
you, I am not a physician, but I wanted to ask about where that 
is moving in the conversation.
    Dr. Lurie. Senator, I am not prepared to discuss that 
today, but I am happy to look into it further and bring you 
back some answers.
    Senator Lankford. OK. I would be glad to be able to get 
that, because that may broaden out and accelerate some of the 
process as well.
    Dr. Lurie. OK.
    Senator Lankford. Thank you, Mr. Chairman.
    Chairman Johnson. Thank you, Senator Lankford.
    I think we have spent too much time on this 100 hours, but 
I will point out, on January 4, 2015, you wrote an article in a 
blog, and here is a quote: ``We estimate that physicians will 
be able to complete the finalized version of the form in just 
45 minutes as compared to the 100 hours listed on the previous 
form.'' Again, this was in February 2015.
    Dr. Lurie. Right.
    Chairman Johnson. It took you 16 months to get that new 
form out there. And, both of those are estimates, right? I 
mean, was it really 100 hours before?
    Dr. Lurie. No. Again----
    Chairman Johnson. And, now it is only 45 minutes?
    Dr. Lurie. The 100 hours was estimating something 
different, right? It was estimating the amount of time to fill 
out a commercial Investigational New Drug (IND), as we call 
them, the application to administer the drug to patients. So, 
it was estimating something different.
    When we went out and developed the estimate for this, I 
went out and I actually found people--physicians, within the 
Federal Government, partly in the FDA--we went to the Centers 
for Disease Control and Prevention (CDC) and we went to the 
National Institute of Health (NIH). We said, ``Tell us how long 
it takes.''
    Chairman Johnson. The bottom line is that for the previous 
expanded access form, the FDA estimated it took 100 hours to 
fill it out----
    Dr. Lurie. No, I would not say that----
    Chairman Johnson. Then, it took you 16 months to come up 
with a shorter form. And, now you are saying it takes 45 
minutes. I am just kind of pointing out the FDA takes a little 
while to do these things.
    Dr. Lurie. Well, Senator, look, I am the guy who put the 
group together, so I am happy to take the criticism home. But, 
I will say this: Under the way our processes work--and I think 
it is a process with which you are familiar--we have to put out 
a draft guidance, which we did in February 2015. We have to 
wait a certain number of days for comments to come in. We have 
to review those comments.
    Now, what we decided to do this time was not only to just 
finalize that draft guidance, which is related to this form, we 
also decided to do more while we were at it. So we did not do 
one. We did three guidances.
    Chairman Johnson. That is fine. Again, we are spending too 
much time on this one form--100 hours. I want to get to the 
real crux of the problem here, because I think we are confusing 
the approval rate versus the ability of a patient like Matt to 
actually get a drug and have the ``Right to Try.'' So, a 99-
percent approval rate is one thing, but what is holding up, for 
example, Matt trying to contact a manufacturer 50 times to have 
access? And, from my standpoint, there are three things 
standing in the way of manufacturers actually taking his phone 
call and making their drugs available under some kind of 
expanded access or ``Right to Try.''
    First of all it is just the cost of providing it. These are 
companies. They are responsible to shareholders, and as Senator 
Paul was talking about, there is a profit motive that drives 
innovation and drives some of these discoveries. That cannot be 
minimized. Next, it is just the adverse effect and the effect 
of that--and you spoke to that earlier. The other thing is 
liability protection. So, there is an enormous impediment for 
manufacturers to agree to make these drugs available to 
somebody like Matt.
    I guess I would just like you to speak to all three of 
those elements again. The cost. Companies develop drugs 
because, in the end, they are reporting to shareholders and 
they have to make a profit. So, it is difficult for companies 
to just give things away. A lot of them do. And, again, it is a 
$2.6 billion cost, on average, for a successful drugs--and that 
entails the cost of trying to develop all of the other drugs 
that fail.
    So, first of all, talk about the cost. Under the current 
system, expanded access, what is the allowable rate of 
reimbursement versus what is it actually cost to manufacturers? 
And, just give us a generalization of that.
    Dr. Lurie. So, one of the three guidances which I was 
referring to is exactly on that point, and what we say in it is 
that you can recover, under expanded access, the direct cost of 
providing the drug. And, the reason for that--and this is the 
place that we reached after taking comment from the public--is 
that you do not want to set it so high that some unscrupulous 
person might charge an arm and a leg to some vulnerable 
patient, right? So, that was the balance that we struck--and it 
seemed to be acceptable to most people.
    Chairman Johnson. So, again, companies can get that direct 
cost.
    Dr. Lurie. Yes.
    Chairman Johnson. And that would include surgeons, as we 
are hearing from----
    Dr. Lurie. Yes.
    Chairman Johnson. OK.
    Dr. Lurie. They can get the direct cost, correct.
    Chairman Johnson. Now talk about just the liability. One of 
the things our bill offers is liability protection, which I do 
believe people, like Matt, Frank, and people in the audience, 
would sign a stack of liability waivers if they could have 
access.
    Dr. Lurie. Right. I am afraid you are straying beyond my 
area of expertise at this point.
    Chairman Johnson. So, under expanded access, do you know 
one way or the other whether there is liability protection?
    Dr. Lurie. I cannot speak to that, Senator.
    Chairman Johnson. And, then speak again to the adverse 
impact--or the adverse effect--and how that really is going to 
weigh into a manufacturer's decision of whether or not they 
want to take that call from Matt and make a drug available.
    Dr. Lurie. Right. Well, we are hoping that they pay 
attention to the study that we published, because I think it is 
immensely reassuring. I mean, 2 out of 1,000 drugs were put on 
clinical hold--and only temporarily. I think from that, if I 
were a manufacturer, I would say, ``Well, that is really pretty 
reassuring.'' And, quite honestly, if I were a manufacturer, I 
would want to know about the adverse effects that my drugs 
might cause sooner rather than later. I would not want to have 
my drug on the market because some adverse effect was ignored 
during expanded access--only to have it recur, where you might 
face liability when the drug is actually on the market.
    But, Senator, we see some other reasons why the companies 
hold back--and I think you have heard a lot of testimony about 
how frequently that happens. And, I pointed out how one company 
has turned down, in 6 months, more than the FDA turned down in 
5 years for all drugs.
    Chairman Johnson. But, tell me why. Do you have any idea 
why that one company turned--was it because of lack of 
liability protection? Was it because of the concern about 
adverse effect? Was it because the direct cost really did not 
reimburse them properly? Do you have any idea why they turned 
them down?
    Dr. Lurie. Sorry. Why the company----
    Chairman Johnson. Correct. Do you have any idea what the 
rationale was?
    Dr. Lurie. Why do companies turn them down?
    Chairman Johnson. Yes. I mentioned three reasons. Do you 
have additional----
    Dr. Lurie. I do. And, one is--and this sound totally banal, 
but it is true--there may not be enough drug around, OK? And, 
the companies are not in the business of making massive 
quantities of drugs for products that may never be approved. 
And, remember that lots of these expanded access products will 
never be approved, right?
    Chairman Johnson. Some of these drugs are extremely 
expensive to manufacture, correct?
    Dr. Lurie. Some of them are. And, you would not want to be 
making excess amounts of those if your product was never going 
to be approved. So, what they tend to do is to make an amount, 
maybe a little bit of excess, that will support the clinical 
trial. And, that is the second part of my answer.
    The companies agree with us that the best way to get safe 
and effective drugs to people is through the clinical trial 
process--and they do not want to see that undermined in any 
way. And, we agree with them because we think, in the end, that 
is how you do it. Then, you are sure. And, it is not one 
patient at a time, as compelling as one patient is. There are 
thousands of patients behind them who we also need to think 
about. And, for them, it is the clinical trial process that 
will be lifesaving in some cases.
    Chairman Johnson. So, let me finish out, just kind of going 
back to the question that Senator Lankford was talking about in 
regard to breakthrough drugs. Once again, here is the FDA 
making decisions for people--and, every drug is different and 
every situation is different. But, again, I just put myself in 
the position of a parent with a child, where you see over the 
years, through a clinical trial, that a drug, like the drug 
that was just approved for DMD is having a positive impact. 
And, your only access is to do a clinical trial, thinking that 
your child is maybe getting just saline. There has to be some 
way to give those parents the right to just, actually, make 
sure they get the drug. Do you understand that?
    Dr. Lurie. I do, Senator.
    Chairman Johnson. When it all comes right down to it, going 
back to the assemblyman from California, talking about, you 
have the right to die, why not the ``Right to Try.'' There is 
just something, in terms of the FDA making these decisions for 
parents and patients, that we have to come to grips with to let 
individuals make that decision--rather than have the FDA make 
it for them. At a certain point--and, again, from my 
standpoint, we are talking about once you have gone through 
Phase I, because there is a certain level of safety that has 
been agreed to. Again, you always are assessing a drug for 
safety even way past approval. You are always looking for that. 
I guess just respond to that.
    Dr. Lurie. Well, it is hard to say that the FDA is making 
those decisions for people when we approve 99 percent of our 
applications. I mean, to me, if I am looking at a process----
    Chairman Johnson. But, again, that is not talking about all 
of the impediments for people even in applying.
    Dr. Lurie. Right, but that is not us, sir.
    Chairman Johnson. Well, it is the adverse impact. It is the 
approval process. It is all of those things. You are a part of 
that whole process.
    Dr. Lurie. I think we do need to keep the approval process 
and the expanded access process, separate in this conversation. 
And, there are efforts that Congress is discussing about the 
approval process, but that seems, to me, a different matter 
here. And, we should not really mix them together.
    With regard to expanded access, we do understand how 
patients feel. And, it is for that reason--and, taking into 
account risk and benefit in their particular context--how 
desperate they can be--that is the reason they practically all 
get approved.
    Chairman Johnson. So, we have 31 States-- maybe with 
California passing it, 32 States--with these ``Right-to-Try'' 
laws on the books. And, all we are trying to do is get the 
Federal Government to kind of stay out of the way so that those 
``Right-to-Try'' laws will actually work in the States. But, 
you cannot tell me one way or the other whether the FDA is 
going to allow those States' ``Right-to-Try'' laws to work.
    Dr. Lurie. Well, again, the Agency does not have a position 
on any of those bills or, for that matter, the Federal one. 
But, I will say this: If I were looking at a process--a multi-
step process with multiple collaborators and partners--
industry, the doctor, the FDA, and so on--and I looked at a 
part of that process that approved 99 percent of applications--
that improved many of those that we got and did so quickly, I 
probably would not be looking at that part as the part to 
reform.
    Chairman Johnson. Thank you, Dr. Lurie.
    Dr. Lurie. Thank you.
    Chairman Johnson. The hearing record will remain open for 
15 days until October 7, at 5 p.m., for the submission of 
statements and questions for the record. This hearing is 
adjourned.
    [Whereupon, at 12:22 p.m., the Committee was adjourned.]

                            A P P E N D I X

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