[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
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
U.S. GOVERNMENT PUBLISHING OFFICE
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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
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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\
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\1\ The prepared statement of Senator Johnson appears in the
Appendix on page 35.
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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.
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\1\ The letters referenced by Senator Johnson appears in the
Appendix on page 193.
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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.
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\2\ The prepared statement of Ms. Olsen appears in the Appendix on
page 39.
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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.
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\1\ The prepared statement of Ms. McLinn appears in the Appendix on
page 88.
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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.
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\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.
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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.
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\1\ The fact sheet referenced by Senator Carper appears in the
Appendix on page 209.
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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
----------
[GRAPHIC(S) NOT AVAILABLE IN TIFF FORMAT]
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.
---------------------------------------------------------------------------
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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