[House Hearing, 115 Congress]
[From the U.S. Government Publishing Office]


           EXAMINING PATIENT ACCESS TO INVESTIGATIONAL DRUGS

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

                                HEARING

                               BEFORE THE

                         SUBCOMMITTEE ON HEALTH

                                 OF THE

                    COMMITTEE ON ENERGY AND COMMERCE
                        HOUSE OF REPRESENTATIVES

                     ONE HUNDRED FIFTEENTH CONGRESS

                             FIRST SESSION

                               __________

                            OCTOBER 3, 2017

                               __________

                           Serial No. 115-60
                           
                           
  
[GRAPHIC NOT AVAILABLE IN TIFF FORMAT]                         


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                    COMMITTEE ON ENERGY AND COMMERCE

                          GREG WALDEN, Oregon
                                 Chairman

JOE BARTON, Texas                    FRANK PALLONE, Jr., New Jersey
  Vice Chairman                        Ranking Member
FRED UPTON, Michigan                 BOBBY L. RUSH, Illinois
JOHN SHIMKUS, Illinois               ANNA G. ESHOO, California
TIM MURPHY, Pennsylvania             ELIOT L. ENGEL, New York
MICHAEL C. BURGESS, Texas            GENE GREEN, Texas
MARSHA BLACKBURN, Tennessee          DIANA DeGETTE, Colorado
STEVE SCALISE, Louisiana             MICHAEL F. DOYLE, Pennsylvania
ROBERT E. LATTA, Ohio                JANICE D. SCHAKOWSKY, Illinois
CATHY McMORRIS RODGERS, Washington   G.K. BUTTERFIELD, North Carolina
GREGG HARPER, Mississippi            DORIS O. MATSUI, California
LEONARD LANCE, New Jersey            KATHY CASTOR, Florida
BRETT GUTHRIE, Kentucky              JOHN P. SARBANES, Maryland
PETE OLSON, Texas                    JERRY McNERNEY, California
DAVID B. McKINLEY, West Virginia     PETER WELCH, Vermont
ADAM KINZINGER, Illinois             BEN RAY LUJAN, New Mexico
H. MORGAN GRIFFITH, Virginia         PAUL TONKO, New York
GUS M. BILIRAKIS, Florida            YVETTE D. CLARKE, New York
BILL JOHNSON, Ohio                   DAVID LOEBSACK, Iowa
BILLY LONG, Missouri                 KURT SCHRADER, Oregon
LARRY BUCSHON, Indiana               JOSEPH P. KENNEDY, III, 
BILL FLORES, Texas                   Massachusetts
SUSAN W. BROOKS, Indiana             TONY CARDENAS, California
MARKWAYNE MULLIN, Oklahoma           RAUL RUIZ, California
RICHARD HUDSON, North Carolina       SCOTT H. PETERS, California
CHRIS COLLINS, New York              DEBBIE DINGELL, Michigan
KEVIN CRAMER, North Dakota
TIM WALBERG, Michigan
MIMI WALTERS, California
RYAN A. COSTELLO, Pennsylvania
EARL L. ``BUDDY'' CARTER, Georgia

                         Subcommittee on Health

                       MICHAEL C. BURGESS, Texas
                                 Chairman
BRETT GUTHRIE, Kentucky              GENE GREEN, Texas
  Vice Chairman                        Ranking Member
JOE BARTON, Texas                    ELIOT L. ENGEL, New York
FRED UPTON, Michigan                 JANICE D. SCHAKOWSKY, Illinois
JOHN SHIMKUS, Illinois               G.K. BUTTERFIELD, North Carolina
TIM MURPHY, Pennsylvania             DORIS O. MATSUI, California
MARSHA BLACKBURN, Tennessee          KATHY CASTOR, Florida
CATHY McMORRIS RODGERS, Washington   JOHN P. SARBANES, Maryland
LEONARD LANCE, New Jersey            BEN RAY LUJAN, New Mexico
H. MORGAN GRIFFITH, Virginia         KURT SCHRADER, Oregon
GUS M. BILIRAKIS, Florida            JOSEPH P. KENNEDY, III, 
BILLY LONG, Missouri                     Massachusetts
LARRY BUCSHON, Indiana               TONY CARDENAS, California
SUSAN W. BROOKS, Indiana             ANNA G. ESHOO, California
MARKWAYNE MULLIN, Oklahoma           DIANA DeGETTE, Colorado
RICHARD HUDSON, North Carolina       FRANK PALLONE, Jr., New Jersey (ex 
CHRIS COLLINS, New York                  officio)
EARL L. ``BUDDY'' CARTER, Georgia
GREG WALDEN, Oregon (ex officio)

                                  (ii)
                             
                             
                             C O N T E N T S

                              ----------                              
                                                                   Page
Hon. Michael C. Burgess, a Representative in Congress from the 
  State of Texas, opening statement..............................     1
    Prepared statement...........................................     2
Hon. Gene Green, a Representative in Congress from the State of 
  Texas, opening statement.......................................     3
Hon. Frank Pallone, Jr., a Representative in Congress from the 
  State of New Jersey, opening statement.........................     5
    Prepared statement...........................................     7
Hon. Marsha Blackburn, a Representative in Congress from the 
  State of Tennessee, opening statement..........................     8
Hon. Greg Walden, a Representative in Congress from the State of 
  Oregon, prepared statement.....................................   168

                               Witnesses

Hon. Brian K. Fitzpatrick, a Representative in Congress from the 
  Commonwealth of Pennsylvania...................................     9
    Prepared statement...........................................    11
Hon. Andy Biggs, a Representative in Congress from the State of 
  Arizona........................................................    13
    Prepared statement...........................................    15
Scott Gottlieb, Ph.D., Commissioner, Food and Drug Administration    19
    Prepared statement...........................................    22
    Answers to submitted questions \1\...........................   216
John E. Dicken, Director, Health Care, Government Accountability 
  Office.........................................................    59
    Prepared statement...........................................    62
    Answers to submitted questions \1\...........................   219
Naomi Lopez Bauman, Director of Healthcare Policy, Goldwater 
  Institute......................................................    75
    Prepared statement...........................................    78
    Answers to submitted questions...............................   222
LCDR Matthew Bellina, U.S. Navy (Retired)........................    83
    Prepared statement...........................................    85
    Answers to submitted questions \1\...........................   225
Kenneth I. Moch, President and Chief Executive Officer, Cognition 
  Therapeutics, Inc..............................................    88
    Prepared statement...........................................    91
    Answers to submitted questions...............................   227
Alison Bateman-House, Ph.D., Assistant Professor, Division of 
  Medical Ethics, Department of Population Health, New York 
  University Langone Health......................................   119
    Prepared statement...........................................   121
    Answers to submitted questions \1\...........................   230
Ellen V. Sigal, Ph.D., Chair and Founder, Friends of Cancer 
  Research.......................................................   150
    Prepared statement...........................................   152
    Answers to submitted questions \1\...........................   232

                           Submitted Material

S. 204, the Trickett Wendler, Frank Mongiello, Jordan McLinn, and 
  Matthew Bellina Right to Try Act of 2017, submitted by Mr. 
  Burgess........................................................   170
H.R. 1020, the Compassionate Freedom of Choice Act of 2017 , 
  submitted by Mr. Burgess.......................................   179
Letter of September 19, 2017, from Alliance for Aging Research, 
  et al., to Mr. Walden and Mr. Pallone, submitted by Mr. Green..   184

----------
\1\ Dr. Gottlieb, Mr. Dicken, LCDR Bellina, Dr. Bateman-House, and Dr. 
Sigal did not answer submitted questions for the record by the time of 
printing.
Letter of September 5, 2017, from Public Citizen, et al., to 
  Representatives in Congress, submitted by Mr. Green, submitted 
  by Mr. Green...................................................   187
Letter of March 16, 2017, from Michael A. Carome, Director, and 
  Sarah Sorscher, Researcher, Public Citizen's Health Research 
  Group, to Representatives and Senators in Congress, submitted 
  by Mr. Green...................................................   190
Article, ``How Often Are Drugs Made Available Under the Food and 
  Drug Administration's Expanded Access Process Approved?'' by 
  Amy E. McKee, et al., The Journal of Clinical Pharmacology, 
  Volume 57, Number S10, 2017, submitted by Mr. Green............   192
Statement of the Association of Clinical Research Organizations, 
  May 22, 2017, submitted by Mr. Green...........................   199
Fact Sheet of June 2016, ``Patient Access to Investigational 
  Therapies,'' Food and Drug Administration, Department of Health 
  and Human Services, submitted by Mr. Green.....................   201
Form FDA 3926 (2/16), ``Individual Patient Expanded Access 
  Investigational New Drug Application (IND),'' Food and Drug 
  Administration, Department of Health and Human Services, 
  submitted by Mr. Green.........................................   202
Statement of Diana Zuckerman, President, National Center for 
  Health Research, October 3, 2017, submitted by Mr. Green.......   204
Letter of September 12, 2017, from Abigail Alliance for Better 
  Access to Developmental Drugs, et al., to Representatives in 
  Congress, submitted by Mr. Burgess.............................   207
Letter of September 28, 2017, from Goldwater Institute, et al., 
  to Representatives in Congress, submitted by Mr. Burgess.......   210
Letter of October 2, 2017, from Hon. Ron Johnson, a U.S. Senator 
  from the State of Wisconsin, and Hon. Joe Donnelly, a U.S. 
  Senator from the State of Indiana, to Mr. Walden, et al., 
  submitted by Mr. Burgess.......................................   213

 
           EXAMINING PATIENT ACCESS TO INVESTIGATIONAL DRUGS

                              ----------                              


                        TUESDAY, OCTOBER 3, 2017

                  House of Representatives,
                            Subcommittee on Health,
                          Committee on Energy and Commerce,
                                                    Washington, DC.
    The subcommittee met, pursuant to call, at 10:30 a.m., in 
Room 2322, Rayburn House Office Building, Hon. Michael C. 
Burgess (chairman of the subcommittee) presiding.
    Members present: Representatives Burgess, Guthrie, Barton, 
Shimkus, Murphy, Blackburn, Lance, Griffith, Bilirakis, 
Bucshon, Brooks, Mullin, Hudson, Collins, Carter, Walden (ex 
officio), Green, Engel, Sarbanes, Schrader, Eshoo, DeGette, and 
Pallone (ex officio).
    Staff present: Ray Baum, Staff Director; Adam Buckalew, 
Professional Staff Member, Health; Kelly Collins, Staff 
Assistant; Zack Dareshori, Staff Assistant; Paul Edattel, Chief 
Counsel, Health; Adam Fromm, Director of Outreach and 
Coalitions; Jay Gulshen, Legislative Clerk, Health; Jennifer 
Sherman, Press Secretary; Hamlin Wade, Special Advisor for 
External Affairs; Jeff Carroll, Minority Staff Director; 
Tiffany Guarascio, Minority Deputy Staff Director and Chief 
Health Advisor; Samantha Satchell, Minority Policy Analyst; 
Andrew Souvall, Minority Director of Communications, Member 
Services, and Outreach; Kimberlee Trzeciak, Minority Senior 
Health Policy Advisor; and C.J. Young, Minority Press 
Secretary.
    Mr. Burgess. The Subcommittee on Health will come to order, 
and I recognize myself for 5 minutes for the purpose of an 
opening statement.

OPENING STATEMENT OF HON. MICHAEL C. BURGESS, A REPRESENTATIVE 
              IN CONGRESS FROM THE STATE OF TEXAS

    I want to thank everyone for joining us this morning. We 
are here to explore an issue that is very personal to many 
patients, to their families across this country who are 
suffering from serious life-threatening conditions or terminal 
illnesses, and that is the availability to access 
investigational drugs and devices.
    Currently, the United States Food and Drug Administration 
conducts an expanded access program aimed at helping these 
patients who do not qualify for clinical trials to help them 
gain access to therapies that are unapproved by the FDA.
    I understand the feelings and the passions of individuals 
who believe these therapies have the potential to save their 
life or offer them a chance to alter the course of their 
illness.
    I also recognize that the Food and Drug Administration must 
strike the right balance between ensuring public safety and 
granting access to new treatments.
    Today, we will convene four panels of witnesses. I first 
want to welcome Representatives Brian Fitzpatrick and Andy 
Biggs to our subcommittee. We look forward to hearing your 
statements this morning on the actions that you both have 
taken.
    Of course, we are pleased to welcome Dr. Scott Gottlieb. 
Dr. Gottlieb, no stranger to this subcommittee, but I believe 
this is your first opportunity to come before us as the 
Commissioner of the Food and Drug Administration. So we, 
certainly, welcome your appointment to that post and welcome 
you to the committee. It's nice to have you here.
    Afterwards, we welcome Mr. John Dicken, the director of 
healthcare at the United States Government Accountability 
Office, and then, finally, we will hear from other stakeholders 
who are deeply engaged on this issue.
    Our Nation has experienced an unprecedented amount of 
innovation and scientific breakthrough over the last decade 
from researchers in our finest academic institutions and from 
those working in the pharmaceutical and medical device 
companies.
    However, I hear from patients with serious life-threatening 
conditions, constituents in north Texas, being frustrated with 
what they see as a regulatory barrier from trying and 
experimenting with new therapies when all others have failed 
them.
    It seems we are at a crossroads when lifesaving treatments, 
while not yet approved, exist but patient cannot have access.
    Since 2014, 37 States, including Texas, have passed a 
version of Right to Try laws through strong grassroots 
movements.
    With that in mind, it is my hope that this hearing will 
start a constructive discussion on this important issue. The 
subcommittee will also examine several pieces of Federal 
legislation--S. 204, the Trickett Wendler Right to Try Act of 
2017 authored by Senator Ron Johnson of Wisconsin; 
Representatives Biggs' and Fitzpatrick's House companion bills; 
and H.R. 1020, the Compassionate Freedom of Choice Act of 2017, 
introduced by our fellow Health Subcommittee member, Morgan 
Griffith of Virginia.
    Members of this subcommittee have many questions and are 
looking forward to hearing from all of the witnesses. We want 
to learn the Food and Drug Administration's steps to streamline 
and communicate the expanded access program.
    We want to dive in to what the Government Accountability 
Office found recently regarding this expanded access program, 
and we want to hear from our patient advocates and thought 
leaders on this topic.
    There are strong view and I am confident that what comes 
out of this hearing will lead to a productive discussion and 
all of us getting closer to meeting the needs of our 
constituents and solving problems tomorrow that seem insoluble 
today.
    [The prepared statement of Mr. Burgess follows:]

             Prepared statement of Hon. Michael C. Burgess

    The subcommittee will come to order.
    The Chair will recognize himself for an opening statement.
    We are here today to explore an issue that is very personal 
to many patients, and their families, across the United States 
who are suffering from serious, life-threatening conditions or 
terminal illnesses--the availability to access investigational 
drugs and devices. Currently, the U.S. Food and Drug 
Administration conducts an expanded access program aimed at 
helping these patients who do not qualify for clinical trials 
gain access to therapies unapproved by the agency. I understand 
the feelings and passions of individuals who believe these 
therapies have the potential to save their life or to offer 
them a chance to alter the course of their illness. I also 
recognize that the Food and Drug Administration must strike the 
right balance between ensuring public safety and granting 
access to new treatments.
    Today we will convene four panels of witnesses. First, I 
want to welcome Representatives Brian Fitzpatrick and Andy 
Biggs to our subcommittee. We look forward to hearing your 
statement this morning on actions you both have taken. Next, I 
want to welcome Dr. Scott Gottlieb. Dr. Gottlieb, I believe 
today's hearing is your first opportunity to come before our 
subcommittee--it is nice to have you here. Afterwards, we 
welcome Mr. John Dicken, Director of Health Care at the U.S. 
Government Accountability Office. Later, we will hear from 
other stakeholders who are deeply engaged on this issue.
    Our Nation has experienced an unprecedented amount of 
innovation and scientific breakthroughs over the last decade 
from researchers in our finest academic institutions and those 
working in the pharmaceutical and medical device companies. 
However, I hear from patients with serious, life-threatening 
conditions--my constituents from North Texas--being frustrated 
with what they see as regulatory barrier from trying and 
experimenting with new therapies when all others have failed. 
It seems we are at a crossroad when life-saving treatments, 
while not yet approved, exist but patients cannot access. Since 
2014, 37 States, including my State of Texas, have passed a 
version of Right to Try laws, through a strong grassroots 
movement. With that in mind, it is my hope this hearing will 
start a constructive discussion on this important issue.
    The subcommittee will also examine several pieces of 
Federal legislation: S. 204, the Trickett Wendler Right to Try 
Act of 2017, authored by Senator Ron Johnson of Wisconsin, 
Representatives Biggs and Fitzpatrick's House companion bills, 
and H.R. 1020, the Compassionate Freedom of Choice Act of 2017, 
introduced by our fellow Health Subcommittee member, 
Representative Griffith.
    Members of this subcommittee have many questions and are 
looking forward to hearing from all of our witnesses. We want 
to learn the Food and Drug Administration's steps to streamline 
the expanded access program. We want to dive into what the 
Government Accountability Office found recently regarding the 
expanded access program. We will hear from patient advocates 
and thought leaders on this topic. There are strong views but I 
am confident what comes out of this hearing will lead to a 
productive discussions and all of us getting closer to meeting 
the needs of all of our citizens.
    I again want to welcome all of our witnesses and thank you 
for being here today. I look forward to your testimony.
    I would like to yield the balance of my time to Ms. 
Blackburn of Tennessee for a statement.

    Mr. Burgess. I want to thank all our witnesses for being 
here today, and I will not yield the balance of my time, Mrs. 
Blackburn, but will be happy to recognize any Member on the 
Republican side who would like a minute and 12 seconds.
    Seeing none, I will yield back my time and recognize the 
gentleman from Texas, Mr. Green, 5 minutes for an opening 
statement, please.

   OPENING STATEMENT OF HON. GENE GREEN, A REPRESENTATIVE IN 
                CONGRESS FROM THE STATE OF TEXAS

    Mr. Green. Thank you, Mr. Chairman, and I would also like 
to thank our administrator, Dr. Gottlieb, for being here and 
our two colleagues.
    Mr. Chairman, expanding access, also known as compassionate 
use, allows patients to gain access to unapproved treatments 
that are on some stage of investigation outside a clinical 
trial.
    The FDA has a long history of facilitating access to 
investigational therapies for terminally ill patients who are 
out of approved options and are ineligible for a clinical 
trial.
    The 1997 FDA Modernization Act made amendments to allow 
patients to access investigational products under certain 
safety conditions.
    In 2009, the agency revised its regulations to establish 
new categories of expanded access and streamlined the 
regulatory process for its program.
    Last year, the FDA released guidance for industry about 
expanded access so that companies would better understand the 
rules of the road and avoid denying requests based on 
uncertainty.
    It also streamlined the application, significantly reducing 
the time it takes to complete to, roughly, 45 minutes.
    The FDA responds to individual patient access requests 
quickly and emergency requests are often granted immediately 
over the phone, something I know firsthand.
    Today, we are examining two legislative proposals that are 
commonly referred to Right to Try bills. I am confident that we 
all strongly support helping patients with serious and life-
threatening illnesses get the care they need and exercise their 
right to make their own decisions about the risk they are 
willing to take.
    Families with a loved one face terminal illness out of the 
FDA-approved options can and do have the right to seek out 
treatments that are in the early stages of investigation.
    Unfortunately, the bills they are considering today are 
well meaning but based on inaccurate premise. These proposals 
would simply take the FDA out of the equation when the FDA 
authorizes more than 99 percent of all expanded access 
requests.
    There are very legitimate frustrations with the current 
system and this committee, through the 21 Century Cures Act and 
the FDA Reauthorization Act, has worked to address some of 
them, but more can be done.
    There is a widespread lack of knowledge about the FDA's 
expanded access program. We need to fill this education gap by 
partnership with doctors and nurses and patients organizations 
and local advocates so patients in need know what their options 
are.
    The FDA has made its Web site more user friendly, 
streamlined the application process, and has a turnaround time 
of days, not weeks or months, and less than 24 hours in certain 
emergency situations--again, something I have witnessed 
firsthand.
    But the agency is correctly working to do more to clarify 
some myths and uncertainty that lead a manufacturer to deny a 
request.
    There is a rampant misunderstanding about compassionate use 
that also must be addressed. The FDA does not have the 
authority to force a company to make investigational products 
available.
    From October 2015 to September 2016, the FDA received 1,554 
requests for expanded access, investigational new drugs and 
protocols and ultimately allowed 1,545 of those requests to 
proceed.
    This is an approval rating of 99.4 percent. Of course, 
there is some requests for investigational products that 
companies deny and we can do a better job of ensuring that 
doesn't happen in inappropriate reasons including a lack of 
clarity about how adverse events would be treated.
    Ultimately, the best way to speed access to drugs and 
development is through a clinical trial process. We have worked 
to do a better job on making clinical trials available on an 
equitable basis for all patients.
    But expanding clinical trial access we can reduce the 
number of patients seeking access to investigational drugs 
outside of the trials and ultimately help even more patients by 
getting drugs approved and widely available.
    I believe we can and should do more to advance policies 
that genuinely increase access to promising investigational 
therapies for patients in need.
    However, removing the FDA from the process of assessing a 
therapy outside a clinical trial is not likely to facilitate 
any increased access to drugs in early trial stages.
    Instead, we should be looking to examine principal reasons 
by patients interested in experimental therapies are unable to 
attain them through clinical trials or the existing expanded 
access and provide solutions to these real barriers.
    We also must continue strong oversight of the 
implementation of requirements within the 21 Century Cures and 
have greater clarity from FDA on the use of adverse event data.
    I appreciate our witnesses and, Mr. Chairman, I would like 
to ask unanimous consent to place a number of items into the 
record: the patient organization letter opposing Right to Try, 
American Cancer Society, Cancer Action Network, Friends of 
Cancer Research, the Leukemia Lymphoma Society and 18 other 
organizations, a letter to Congress regarding S. 204 submitted 
by Public Citizen and 17 other organizations. Do you want me to 
read this whole list, or can I just submit it?
    Mr. Burgess. Without objection, so ordered.
    [The information appears at the conclusion of the hearing.]
    Mr. Green. Thank you.
    Mr. Burgess. Does the gentleman yield back his time?
    Mr. Green. I yield back my time.
    Mr. Burgess. Gentleman yields back his time.
    Not seeing the chairman of the full committee having 
arrived yet, the Chair is prepared to yield to the ranking 
member of the full committee, Mr. Pallone of New Jersey, 5 
minutes for an opening statement, please.

OPENING STATEMENT OF HON. FRANK PALLONE, JR., A REPRESENTATIVE 
            IN CONGRESS FROM THE STATE OF NEW JERSEY

    Mr. Pallone. Thank you, Mr. Chairman.
    Today's discussion is of great importance for so many 
patients and families who are facing diseases with no other 
treatment options and when someone has exhausted all of the 
available treatment options they will sometimes explore the 
possibility of trying unproven experimental therapies.
    It is this desire that has led to calls for Federal 
legislation that would grant patients the right to try 
investigational products.
    It is understandable that someone suffering from a disease 
that has no more options would want to try anything that could 
help them fight their disease.
    Fortunately, both the FDA and Congress have taken some 
actions that provide some hope. Through the FDA's expanded 
access program, patients are able to get access to 
investigational products.
    This FDA program approves 99 percent of all requests for 
investigational drugs or biologics that it receives. Last year, 
FDA received more than 1,500 requests and only nine were not 
approved.
    And despite this high approval rate, supporters of Right to 
Try laws have argued that the process is too slow and 
burdensome. But I have not seen evidence that this is the case.
    In fact, FDA often grants emergency requests for expanded 
access immediately over the phone and nonemergency requests are 
processed in an average of four days.
    Despite these quick turnarounds, FDA responded to these 
criticisms. Last year, the agency streamlined their current 
process even further so that filling out an application now 
takes less than an hour.
    FDA also released additional guidance to industry outlining 
the expanded access program's requirements and addressing 
common questions related to the different programs and 
submissions process, and all this was done to alleviate any 
confusion that may have existed in the past and I want to 
commend the agency for its commitment to improving expanded 
access and for its responsiveness to the concerns it heard from 
doctors and patients.
    Now, this committee has also led efforts to facilitate 
greater access to investigational products for patients who are 
looking for additional options.
    Last year, we passed the 21st Century Cures Act, which 
provides greater transparency to expanded access programs by 
requiring manufacturers or distributors of investigational 
drugs to make publicly available their expanded access policies 
for the first time.
    And then this summer we passed the FDA Reauthorization Act, 
which works to improve access to clinical trials for patients. 
The law does this by requiring FDA to conduct a public meeting 
on clinical trial criteria, report on barriers to patients 
participating in clinical trials, and offer potential solutions 
to include additional populations of patients.
    The FDA Reauthorization Act also requires FDA to issue 
additional guidance to manufacturers regarding how clinical 
trials can be expanded to include broader populations and 
improve access to treatment for patients who may not qualify 
for these trials.
    These are all meaningful steps that I believe will help to 
address some of the criticisms we will hear today. Now, our 
discussion today is important because I am concerned that the 
legislation being considered could expose seriously ill 
patients to greater harm instead of the greater access that 
they are looking for.
    The Senate legislation would lower the bar for safety and 
effectiveness by allowing access to investigational drugs that 
have only completed a Phase 1 clinical trial, and that's an 
extremely small trial that does not determine the effectiveness 
of potential side effects of the drug.
    There is also no assurance in the Senate bill that a 
manufacturer will provide patients with an investigational 
treatment under this pathway.
    Today, pharmaceutical companies can choose to deny patient 
access to an experimental treatment because there is not enough 
of the drug available or because they are concerned about 
dangerous side effects.
    The Senate legislation also erodes important patient 
safeguards. It limits FDA's ability to use clinical outcomes 
associated with the use of investigation product when reviewing 
a product for approval.
    It also prevents any entity from being held liable for use 
of the treatment. Now, while I appreciate the intent of the 
Senate legislation, I have a hard time supporting it in its 
current form and I guess what I am hoping is that we will hear 
today about alternative solutions that may provide more 
meaningful access to investigational products without 
undermining FDA's ability to protect patients from this harm 
because the last thing I want to do is give patients false hope 
and to have Congress pass legislation that will not in fact 
help someone access investigational treatments.
    So, hopefully, we will hear more about, you know, ways that 
we could make some changes that don't sacrifice safety, and I 
look forward to what I hope will be a thoughtful discussion 
about a path forward.
    I yield back, Mr. Chairman.
    [The prepared statement of Mr. Pallone follows:]

             Prepared statement of Hon. Frank Pallone, Jr.

    Thank you, Mr. Chairman. Today's discussion is of great 
importance to so many patients and families who are facing 
diseases with no other treatment options. And when someone has 
exhausted all of the available treatment options, they will 
sometimes explore the possibility of trying unproven 
experimental therapies. It is this desire that has led to calls 
for Federal legislation that would grant patients the right to 
try investigational products.
    It is understandable that someone suffering from a disease 
that has no more options would want to try anything that could 
help them fight their disease. Fortunately, both the Food and 
Drug Administration (FDA) and Congress have taken action that 
provides some hope.
    Through the FDA's expanded access program, patients are 
able to get access to investigational products. This FDA 
program approves 99 percent of all requests for investigational 
drugs or biologics that it receives. Last year, FDA received 
more than fifteen hundred requests, and only nine were not 
approved.
    Despite this high approval rate, supporters of Right to Try 
laws have argued that the process is too slow and burdensome. 
But I have not seen evidence that this is the case. In fact, 
FDA often grants emergency requests for expanded access 
immediately over the phone, and nonemergency requests are 
processed in an average of four days.
    Despite these quick turnarounds, FDA responded to these 
criticisms. Last year, the agency streamlined the current 
process even further so that filling out an application now 
takes less than an hour. FDA also released additional guidance 
to industry outlining the expanded access program's 
requirements, and addressing common questions related to the 
different programs and submission process. All of this was done 
to alleviate any confusion that may have existed in the past. I 
commend the agency for its commitment to improving expanded 
access and for its responsiveness to the concerns it heard from 
doctors and patients.
    This committee has also led efforts to facilitate greater 
access to investigational products for patients who are looking 
for additional options. Last year, we passed the 21st Century 
Cures Act which provides greater transparency to expanded 
access programs by requiring manufacturers or distributors of 
investigational drugs to make publicly available their expanded 
access policies for the first time.
    And then this summer, we passed the FDA Reauthorization 
Act, which works to improve access to clinical trials for 
patients. The law does this by requiring FDA to conduct a 
public meeting on clinical trial criteria, report on barriers 
to patients participating in clinical trials, and offer 
potential solutions to include additional populations of 
patients. The FDA Reauthorization Act also requires FDA to 
issue additional guidance to manufacturers regarding how 
clinical trials can be expanded to include broader populations 
and improve access to treatments for patients who may not 
qualify for these trials. These are meaningful steps that I 
believe will help to address some of the criticisms we will 
hear today.
    Our discussion today is also important because I am 
concerned that the legislation being considered could expose 
seriously ill patients to greater harm instead of the greater 
access that they are looking for. The Senate legislation would 
lower the bar for safety and effectiveness by allowing access 
to investigational drugs that have only completed a Phase 1 
clinical trial. That is an extremely small trial that does not 
determine the effectiveness or potential side effects of a 
drug.
    There is also no assurance in the Senate bill that a 
manufacturer will provide patients with an investigational 
treatment under this pathway. Today, pharmaceutical companies 
can choose to deny a patient access to an experimental 
treatment because there is not enough of the drug available or 
because they are concerned about dangerous side effects.
    The Senate legislation also erodes important patient 
safeguards. It limits FDA's ability to use clinical outcomes 
associated with the use of an investigational product when 
reviewing a product for approval. And it also prevents any 
entity from being held liable for use of the treatment.
    While I appreciate the intent of the legislation, I cannot 
support it in its current form. I hope that today's discussion 
will offer alternative solutions that may provide more 
meaningful access to investigational products without 
undermining FDA's ability to protect patients from harm. The 
last thing I want to do is give patients false hope, and to 
have Congress pass legislation that will not in fact help 
someone access investigational treatments.
    I want to thank the witnesses for being here today, and 
look forward to what I hope will be a thoughtful discussion 
about a path forward.

    Mr. Burgess. Chair thanks the gentleman. The gentleman 
yields back.
    The Chair now recognizes the gentlelady from Tennessee, 
Mrs. Blackburn, 5 minutes for an opening statement.

OPENING STATEMENT OF HON. MARSHA BLACKBURN, A REPRESENTATIVE IN 
              CONGRESS FROM THE STATE OF TENNESSEE

    Mrs. Blackburn. Thank you, Mr. Chairman, and I am not going 
to take that full 5 minutes and will submit my full opening 
statement for the record.
    I do want to welcome our colleagues here to the committee. 
I want to welcome Dr. Gottlieb. We are just so pleased that we 
are going to be able to take up what is, I think, a really 
important issue for us to address when we look at healthcare, 
and that is the right to try.
    And we want to commend the FDA for going through the 
process and taking some efforts to simplify and expedite 
request. We do think that it is important for Congress to do 
something legislatively to ensure patient access to promising 
treatments but do it with the appropriate disclosure 
requirements and the liability protections.
    So we welcome all of you that are here today. We appreciate 
the time and effort that has gone into this and the fact that 
we are going to have the multiple panels so we can kind of 
drill down and do a good solid look at this from the patient 
perspective, from the legislative perspective, from the 
regulatory perspective.
    So to each of you, welcome, and thank you and I yield back.
    Mr. Burgess. Chair thanks the gentlelady. The gentlelady 
yields back. This concludes member opening statements. The 
Chair would remind Members, pursuant to committee rules all 
Members' opening statements will be made part of the record.
    The gentlelady from Tennessee is quite correct. We have a 
total of four panels of witnesses testifying before the 
subcommittee today.
    To start us off, we are going to hear from two of our House 
colleagues--Congressman Brian Fitzpatrick of Pennsylvania and 
Congressman Andy Biggs of Arizona.
    We appreciate both of you being here with us this morning. 
Congressman Fitzpatrick, you're recognized for 5 minutes for 
your statement.

 STATEMENTS OF HON. BRIAN K. FITZPATRICK, A REPRESENTATIVE IN 
 CONGRESS FROM THE COMMONWEALTH OF PENNSYLVANIA, AND HON. ANDY 
 BIGGS, A REPRESENTATIVE IN CONGRESS FROM THE STATE OF ARIZONA

             STATEMENT OF HON. BRIAN K. FITZPATRICK

    Mr. Fitzpatrick. Thank you, Mr. Chairman.
    Good morning. I want to start by thanking Chairman Burgess, 
Ranking Member Green, Vice Chairman Guthrie, and all members of 
this subcommittee for holding this hearing. It's a very 
important hearing on the right to try, and I also want to thank 
my colleague and friend, Andy Biggs, for your partnership on 
this issue.
    Fellow colleagues, each year thousands of Americans receive 
the devastating news of a terminal diagnosis. Even with the 
amazing work done in American medical research and development, 
for far too many families access to these potentially 
lifesaving treatments will come too late or not at all.
    Thousands of terminally ill patients suffer needlessly 
while waiting final approval for drugs, therapies, and other 
medical technologies, and while the Food and Drug 
Administration carries out its three-phase approval process, 
which can take years and cost billions of dollars, many 
patients simply want a chance to try treatments that are 
already demonstrated to be safe.
    Mr. Chairman, it is my hope that we can come together with 
Federal regulators and industry leaders to clear the path 
forward to care for those who are fighting just for a shot at 
living.
    A bill that was unanimously passed by the Senate--
unanimously passed by the Senate--will offer them a chance to 
extend their lives.
    The Right to Try Act would ensure that terminally ill 
patients, together with their physicians and pharmaceutical 
manufacturers, can administer investigational treatments where 
no alternative exists.
    In fact, this bipartisan idea is already the law in 37 
States in our Nation. A Federal Right to Try law would prevent 
the Government from blocking access to potentially lifesaving 
medications.
    It would require patients who are unable to participate in 
clinical trials to first try all other available treatments.
    Mr. Chairman, I want to note that these provisions only 
apply to terminally ill patients. It does not undo the FDA 
approval process but, rather, provides a potential lifeline to 
those who simply cannot wait.
    It requires a physician to certify that all other options 
were exhausted or unavailable. This maintains the incentives 
for patients to seek out and join clinical trials.
    This bill requires that a product meet demonstrated levels 
of safety by obtaining FDA Phase 1 approval. We have worked 
with the drug companies to ensure that adverse outcomes are not 
used against any ongoing application for approval.
    Additionally, patients, doctors, and manufacturers do not 
assume any additional liability under this act. For those 
patients caught in between traditional drug approval delays, 
clinical trial process for which they do not qualify and 
limited time, this Right to Try legislation simply establishes 
the freedom for patients and their doctors to try therapies 
where the benefits far outweigh the risks.
    It gives them the option of trying to save their life. 
Whether it is a father courageously battling ALS or a brave 
child living with Duchene muscular dystrophy, my colleagues, 
they deserve the right to try.
    I want to sincerely thank the committee for your time and 
consideration and as your colleague I ask that you work with us 
to get this done on behalf of all terminally ill patients 
across America.
    All that we ask--all that we ask is that this bill be put--
be put on the floor of the House and allow each one of us to 
cast our vote and go home and answer for that vote.
    Mr. Chairman, I yield back.
    [The prepared statement of Mr. Fitzpatrick follows:]
    [GRAPHICS NOT AVAILABLE IN TIFF FORMAT]
    
    Mr. Burgess. Chair thanks the gentleman. The gentleman 
yields back.
    Chair recognizes the gentleman from Arizona, Mr. Biggs, 5 
minutes, please, for your statement.

                  STATEMENT OF HON. ANDY BIGGS

    Mr. Biggs. Thank you. Thank you, Mr. Chairman.
    I, first, also thank Chairman Burgess, Ranking Member 
Green, and all the members of the committee for allowing me to 
address you today.
    I am here with my friend and colleague, Representative 
Brian Fitzpatrick, to fight for passage for the Right to Try 
Act.
    This bill that I introduced with Mr. Fitzpatrick in 
February now has dozens of bipartisan co-sponsors including 
members in this very room today.
    I am particularly pleased that Senator Ron Johnson's bill 
will be considered today as well. As the committee may know, 
Senator Johnson's bill passed the Senate by unanimous consent.
    Anyone who understands the arcane procedures of that 
chamber can attest that this is no mean feat. I am strongly 
supportive of Mr. Johnson's efforts. He has been a tireless 
advocate of Right to Try for years.
    I won't take up a great deal of this committee's time 
elaborating on the virtues of the bill Representative 
Fitzpatrick and I introduced because, frankly, very little 
explanation is necessary and Mr. Fitzpatrick has done a great 
job explaining it.
    Fundamentally, our legislation allows terminally ill 
patients who have no further options--I repeat, no further 
options--the opportunity to try experimental drugs that could 
save their own lives.
    Yes, there are also provisions in our bill to protect both 
the patients themselves and the pharmaceutical companies who 
want to participate.
    But those provisions are secondary to its primary purpose. 
The primary purpose of our Right to Try Act is to give brave 
patients across this country some choice over their own 
destinies when all other avenues are closed.
    We should all share the same goal of doing everything we 
can for patients fighting to save their lives and I have no 
doubt that the intentions of everyone in this room are good.
    So what are we waiting for? Why isn't this committee doing 
everything possible to get Right to Try passed out of Congress 
and onto President Trump's desk? That's really the next step. 
We need to get this out of the House.
    The status quo is not the answer. We will hear claims today 
from the FDA and other agency officials that their own expanded 
access program is working and continues to improve.
    There may be some truth to that and I am sure that 
Commissioner Gottlieb works tirelessly to help as many terminal 
patients as he can.
    But that program is simply not enough. Frankly, that 
program was not put into high gear without Federal legislation 
looming.
    I know that the program is simply not enough because I have 
talked to dozens and dozens of patients, family members and 
advocates, who tell me it is not enough.
    They come to my office. They call me on the phone. They 
write me impassioned letters. These same advocates have ensured 
that Right to Try has become law in 37 States.
    Think about that for a moment. In half of those 37 States, 
Right to Try laws passed with unanimous support--bipartisan 
support--and in my home State of Arizona, voters approved this 
initiative with nearly 80 percent of the popular vote and I am 
convinced that the other 20 percent were just the folks that 
always vote no.
    At a time when pundits are claiming that our politics are 
broken--Republicans and Democrats can't come together on 
anything--here is a cause--here is the cause that Americans of 
all political stripes believe in.
    I was first introduced to Right to Try while serving in the 
Arizona State Legislature with fellow legislator and friend, 
Laura Knaperek. By 2014, she was no longer a legislator but she 
was an advocate, suffering in the fight of her life against 
ovarian cancer.
    Her mission became to see Right to Try passed into law. In 
the end, her efforts for this cause succeeded beyond everyone's 
wildest expectations. Unfortunately, Laura is no longer with 
us. She lost her brave battle with cancer but her legacy as a 
tireless patient advocate lives on.
    I will continue to carry on Laura's fight, not just for her 
but for all those brave patients across this country who are 
battling against the odds every day.
    I fight for Bertrand Might, for Jordan McLinn, for Matt 
Bellina, who is testifying today, and I fight for the countless 
other patients who deserve a right to try. I urge you to join 
in that fight. We must act further without delay.
    Thank you again, Mr. Chairman, and Ranking Member Green and 
members of the committee. I yield back.
    [The prepared statement of Mr. Biggs follows:]
    [GRAPHICS NOT AVAILABLE IN TIFF FORMAT]
    
    Mr. Burgess. And the gentleman yields back, and the Chair 
thanks the gentlemen.
    The Chair thanks both gentlemen for being here, taking time 
to share with us your stories and your passion and taking the 
time to testify before the subcommittee. It is helpful to us in 
our deliberations.
    Again, I want to stress that there are four panels today so 
we are going to move smartly to the next panel. As is 
customary, there will not be questions for the Members from the 
Members, but following each of the other panels there will be 
opportunities for questions from Members.
    Our second panel we are very, very pleased to have Dr. 
Scott Gottlieb, Commissioner of the United States Food and Drug 
Administration.
    Doctor, we certainly sincerely appreciate you being here 
today, and you are now recognized for 5 minutes for your 
opening statement, please.

   STATEMENT OF SCOTT GOTTLIEB, COMMISSIONER, FOOD AND DRUG 
                         ADMINISTRATION

    Dr. Gottlieb. Good morning, Mr. Chairman, Mr. Ranking 
Member, and members of the subcommittee.
    I want to thank you for the opportunity to testify this 
morning. This is my first time testifying before the Energy and 
Commerce Committee, and I'd like to take a moment to thank you 
for your strong support of FDA and its public health mission.
    I know this committee and this subcommittee in particular 
worked hard to enact the 21st Century Cures legislation and 
FDARA, and I also want to acknowledge your continued efforts to 
modernize the review and approval of OTC products through user 
fee legislation and I look forward to working with you closely 
on all of our shared goals.
    Throughout my career I've worked to advance policies to 
enable terminally ill patients to obtain earlier access to 
promising new drugs and this includes preapproval access to 
experimental medicines.
    As a cancer survivor who used an approved drug in an off-
label fashion in the treatment of my own cancer, I've grappled 
with some of these issues first-hand.
    While my cancer was very curable, I know that many patients 
with serious illness face long odds, and their best chance at 
gaining an advantage in those odds is with something unproven 
and experimental, and we need to make sure that we serve these 
patients.
    Before I discuss these goals and the issues related to 
Right to Try legislation, I'd like to take a moment first to 
acknowledge the tragedy in Las Vegas and then to expand on 
another tragedy unfolding in the south, the crisis facing 
Puerto Rico.
    I want to just brief the committee on some steps that are 
going on right now with respect to that crisis at FDA.
    I was grateful for the opportunity to accompany the 
Secretary of the Department of Homeland Security on her trip to 
Puerto Rico on Friday.
    I visited with my FDA team stationed in San Juan where we 
have about a hundred full time staff. Our large staff is a 
reflection of the significant medical product manufacturing 
capacity on that island.
    We are now engaged in a sweeping effort to cross the entire 
agency to provide direct assistance to our staff and fellow 
citizens on the island and this includes efforts to get food 
and medical products onto the island and get hospitals back 
into full operation.
    But the devastation in Puerto Rico presents a broader 
challenge to the FDA because it is home to a very large medical 
product manufacturing base for both drugs and devices.
    Some of these facilities make products that could be in 
shortage if production is sharply diminished or pushed offline. 
This is particularly concerning because some of these products 
are critical to Americans.
    A loss of access to these drugs and devices could have 
significant public health consequences. This includes products 
for the treatment of cancer and a lot of other unmet medical 
needs.
    Getting these facilities back online is a public health 
priority. It's also a priority of Puerto Rico's recovery. I've 
discussed this matter directly with the Governor of Puerto Rico 
and his staff.
    These sites directly employ about 90,000 residents of 
Puerto Rico and represent 30 percent of the island's GDP. 
Puerto Rico is home to an excellent high-quality manufacturing 
for sophisticated medical products including many injectable 
drugs and complex devices.
    A highly skilled, highly dedicated, highly productive 
Puerto Rican workforce enables the success of this industry.
    If we don't get these facilities back online in a timely 
way and they decide to relocate after this disaster, it would 
jeopardize the island's economic future. For many reasons, not 
least our concern for the people of Puerto Rico, we need to 
work to help to restore this manufacturing base.
    I can tell you the leadership of FDA is committed to all 
these efforts. We stand with the people of Puerto Rico. I have 
been personally engaged in troubleshooting these issues, 
working directly with my colleagues at HHS and DHS and the 
staff of the Governor of Puerto Rico, and I am available to 
brief this committee directly on these efforts.
    On the topic we are here to discuss today, I want to share 
some insight into some of the recent steps FDA took to improve 
our expanded access program and continue to facilitate access 
to promising drugs targeted to unmet needs prior to approval 
for patients with serious or immediately life-threatening 
illnesses who don't have other alternatives.
    I have announced some new policy actions that we are taking 
today and we intend to take additional steps in the near 
future.
    Critics of these efforts may look at our actions 
individually and say that none of these measures will 
materially change the current balance.
    But this effort cannot be solved in one step. We need to 
look across the totality of what we are doing to measure the 
impact of our endeavors.
    My goal is to establish a framework that preserves our 
current approval process while making sure that there are 
efficient achievable avenues for patients to access promising 
drugs targeted to unmet needs.
     We need to serve all the interests of patients facing 
serious illness who lack good options. This includes their 
interest in trying unproven drugs.
    I am committed to this goal. I believe in this right. I 
support this idea. I look forward to answering questions.
    Thanks a lot.
    [The prepared statement of Dr. Gottlieb follows:]
    [GRAPHICS NOT AVAILABLE IN TIFF FORMAT]
    
    Mr. Burgess. The Chair thanks the gentleman for his 
statement and I am in agreement with the Commissioner about the 
need for our intention upon helping the citizens--United States 
citizens in Puerto Rico and their recovery and I think you'll 
see some of our efforts in the children's health insurance bill 
that we mark up tomorrow.
    Already beginning some effort to--with some help that is--
that is going to be available there. But it by no means 
completes that task and this committee--this Congress will have 
a significant ahead of it in recovering from the--from the 
storms of September.
    I am going to recognize myself for 5 minutes for questions, 
and we will alternate between Republicans and Democrats.
    Commissioner, I guess my first question is, you know, when 
I arrived in the United States Congress, I don't think I had 
prior knowledge, as a practising physician for 25 years--I 
don't think I knew about clinicaltrials.gov and so as a follow-
on to that, how are we communicating, yes, clinicaltrials.gov 
and making sure people are aware that there are clinical trials 
that are available but then, moreover, the availability of 
these expanded use programs? So what do you see as a 
communication strategy coming from the agency in that regard?
    Dr. Gottlieb. Well, Mr. Chairman, in answer to your 
question, I think the short answer is until recent years we 
probably didn't communicate very well and that's why patients 
face more obstacles getting access to experimental drugs than 
perhaps they should have.
    With the help of this committee we have taken new steps to 
try to make information about the availability of drugs through 
expanded access programs more available, easier to find.
    There's provisions in 21 Century Cures Act that requires 
sponsors to post notification of the availability of drugs 
through expanded access programs on their Web site.
    We are starting to work with sponsors to gain compliance 
with that. There's also provisions that they need to post 
information about clinical trials to clinicaltrials.gov and we 
are working with sponsors to broaden the compliance with that 
as well.
    But we are not just relying on those measures, as potent 
and as important as they are. We are also working with the 
private sector and patient group interests to create some new 
tools and one of those tools is something I am talking about--I 
talked about today in my written testimony for the record, 
something called the Navigator, which we developed with the 
Reagan-Udall Foundation, which is going to create a one-stop 
portal for access to information about expanded use programs.
    Right now that tool is targeted to drugs for oncology, for 
cancer. We announced today that we are going to broaden it for 
drugs targeted to rare diseases and we look to broaden this 
even further.
    I think that this could become a consolidated web portal, 
if you will, for access to this kind of information so patients 
have one place to go and we've been working closely with 
sponsors to get them to report to this--to this new tool.
    Mr. Burgess. I thank you for that answer. In our next panel 
we are going to hear from the Government Accountability Office 
and their recommendation for action that they give at the 
conclusion of the GAO report.
    So their recommendation--let me just read it here--``the 
Commissioner of the Food and Drug Administration should clearly 
communicate how the agency will use adverse event data from 
expanded access use when reviewing drugs and biologics for 
approval and marketing in the United States.''
    So we are going to hear testimony in the next panel that 
this recommendation has been given to the FDA and can you kind 
of brief us as to the status of that recommendation?
    Is this something of which you were aware? Is this 
accurate?
    Dr. Gottlieb. Well, I hate to short circuit the testimony 
of my colleague at the GAO, but we've taken their advice and 
we've announced that today.
    And so we've doubled down on a proposition that we have 
long held that this information typically isn't used in the 
consideration of a product and a product's approval and we've 
clarified in a new guidance document that we are posting today 
that the circumstances under which this information would and 
wouldn't be used and the bottom line is that information 
gleaned from an expanded access program is exceedingly unlikely 
to be incorporated into a consideration of the approvability of 
a product.
    We are saying today in the guidance document that we must 
consider the circumstances in which the product is being used 
as a component of whether we'll consider whether or not an 
adverse event recognized in the use of that product is 
attributable to the drug.
    And in the setting of an expanded access program when you--
when you have a patient with a terminal illness who is 
oftentimes on a lot of other therapy, it is very hard to make a 
determination that any one drug was responsible for any one 
observation in that setting and so we are exceedingly unlikely 
to use that information.
    And just to reinforce that, we looked across a decade of 
experience with expanded access, 322 products that were 
approved over that period of time, 28 percent of which had 
expanded access opportunities associated with the products, and 
we could find no instance where information gleaned from an 
expanded access program was used to deny approval of the drug.
    We found one instance where information gleaned from the 
expanded access program was incorporated into drug labeling, 
and we actually found one instance where the data gleaned from 
the expanded access program actually informed our consideration 
of the effectiveness of that product and helped lead to its 
approval.
    Mr. Burgess. I thank you for the answer.
    I will yield back and recognize the gentleman from Texas, 5 
minutes for questions, please.
    Mr. Green. Thank you, Mr. Chairman, and again, Dr. 
Gottlieb, thank you for being here.
    The legislation we are considering today offered by Senator 
Johnson proposes to offer terminally ill patients a new pathway 
to investigational products without FDA review or approval.
    One of my concerns with this legislation how broadly it 
would apply. For example, under the Senate legislation, an 
eligible patient is defined as a patient diagnosed with a life-
threatening disease or condition.
    My first question, Commissioner, as I understood, S. 204 
would provide eligibility to a much broader range of patients 
than those with terminal illness and even under State Right to 
Try laws.
    Would you discuss further when a patient population is 
eligible for FDA's expanded access program currently and what 
patient population would be eligible under S. 204?
    Dr. Gottlieb. I appreciate the question, Congressman.
    I think your statement embedded in the question is correct. 
Right now, our expanded access program is generally available 
for patients facing life-threatening conditions and terminal 
illness.
    We provide for both emergency and nonemergency situations. 
As part of the technical assistance that we provided to 
Congress in their consideration of this bill, one of the 
comments that we made is with respect to the definition of a 
terminally ill patient.
    If you look across the State laws and States that have 
passed Right to Try laws, the language typically speaks about a 
patient being terminally ill to qualify for consideration under 
the Right to Try provisions.
    Congress, in consideration of some of this legislation--and 
there's various bills that have been considered by this body--
but in some of these legislative measures have broadened that 
to include life-threatening diseases or diseases that could be 
life threatening--excuse me, diseases that are--that are either 
terminal or life threatening, and this, in our estimation, 
could also potentially include chronic illnesses like diabetes 
or other diseases that while not--don't set a patient on a 
terminal course in an immediate way, certainly are life-
threatening diseases.
    And so one of the suggestions that we've had in our 
technical assistance, and it is also a component in my written 
testimony, is to consider more carefully the definition and 
maybe map it more closely to what some of the States have done 
in their consideration of this measure.
    Mr. Green. OK. So the two issues would be terminal or life 
threatening?
    Dr. Gottlieb. That's right, Congressman. As part of our 
technical assistance, we urge Congress to consider that 
language and consider whether or not it should be defined as a 
patient who is terminally ill, similar to what the State laws 
have done.
    The component of a life-threatening disease is a broader 
definition and, as Dr. Burgess would probably agree, there's a 
lot of chronic illnesses that are certainly life threatening 
but not immediately terminal.
    Mr. Green. My understanding from supporters of the Senate 
legislation and from those supporting the State Right to Try 
laws is that the intent is to help support increased access to 
investigational products for terminally ill patients.
    If we are to consider legislation moving forward regarding 
this goal, it is my hope that we would all agree that we should 
align any legislation with that targeting population we are 
discussing in terminally ill patients and that way to make most 
terminally ill patients access to the drug is to have drug 
approval by conducting clinical trials.
    I am not convinced that the FDA is a barrier to 
investigational treatments and I continue to have concern about 
this legislation. But I appreciate your testimony on this 
today. Yes?
    Dr. Gottlieb. I just want to--if I may follow up, with 
your--with your indulgence, Congressman.
    The reason--the reason why I think it might be important to 
consider how we define terminal illness here and not--and make 
sure we are not too expansive if we do move forward with this 
legislation is so as not to broaden it in a way that it might 
undermine its intended purpose.
    I think the more we broaden this measure and the more it is 
opened up to a broader set of conditions, the more we risk 
undermining the central purpose of the legislation and that 
would be--that would be the policy reason for considering how 
we define that.
    Mr. Green. Thank you, Mr. Chairman. I am almost out of time 
and I don't think you have time to answer the question.
    One of my concerns it requires doctors and distributors to 
report adverse events. We need to make sure that's--if the 
legislation moves forward we need to make sure that's defined 
correctly, Mr. Chairman, and I yield back my time.
    Mr. Burgess. Gentleman yields back. The Chair thanks the 
gentleman.
    Chair recognizes the gentleman from Oregon, Mr. Walden, 
chairman of the full committee, 5 minutes for questions, 
please.
    Mr. Walden. I thank the gentleman and I appreciate the 
indulgence of the committee. We have been down with another 
hearing on Equifax and the little data breach issue that only 
affected 145.5 million Americans. So I have been done at that 
hearing.
    Dr. Gottlieb, first of all, delighted to have you before 
the committee. We are delighted you're at the FDA. We 
appreciate the reforms that you're bringing to that agency and 
we look forward to a long continued interaction with you and 
this committee in the work that you're doing.
    The FDA recently took action to simplify the expanded 
access process, specifically the new form for physicians as 11 
elements compared to the previous 26 elements, and there is now 
a partnership with the Reagan-Udall Foundation to help patients 
and physicians navigate the process.
    I know it may be a bit premature, but are you able to share 
any statistics on the impact of these two modernizations to the 
expanded access program?
    Dr. Gottlieb. Congressman, it is too early for us to really 
draw any conclusions about, you know, the direct impact that it 
has had. We hope it will be very impactful.
     Mr. Walden. And I appreciate your testimony and that of 
our colleagues, certainly, Mr. Fitzpatrick and Mr. Biggs, and I 
know your own personal experience.
    And, you know, having lost loved ones to really tough 
diseases, especially cancer, and I think we all sort of grasp 
isn't there something else out there I can try, and it is that 
balance in public policy of patient safety versus trying to 
help people with terminal illnesses get access to something 
that could help them.
    And so when you look at this legislation--I know you talk 
about better definition on the terminal illness piece--could 
you speak more to that and what--why that clarification might 
be necessary?
    Because I have been told by at least one of the Senate 
sponsors of this bill that they are not looking for the House 
to make any changes out of fear it may fail if it goes back 
with changes. And so I am concerned about that.
    Dr. Gottlieb. Well, I mean, the bottom line is that the 
definition, if it were to incorporate life-threatening diseases 
is broad and as a clinician I can certainly contemplate a lot 
of diseases that are life threatening but not immediately life 
threatening, and the way it is written I think the agency would 
have to, as a matter of legal policy, have to interpret that 
potentially expansively.
    So it could--it could sweep in a whole range of conditions 
for which we didn't intend. And I would just be mindful that if 
the goal is to make sure that we are serving the interests of 
patients who are facing terminal illnesses, the more we broaden 
this provision and the more we potentially sweep in conditions 
for which we might be exposing people to unwanted side effects 
from experimental therapies, the more we risk undermining the 
whole venture that we are trying to engage in here, which is to 
narrowly tailor something to people who really don't have good 
options from available therapy.
    Mr. Walden. And do you think the way this is currently 
written could hurt people then?
    Dr. Gottlieb. Well, I think the way this----
    Mr. Walden. The process?
    Dr. Gottlieb. I think the way this is currently written it 
could undermine some of the goals of the policy and we've been 
consistent in providing that technical assistance all the way 
through.
    And so I am representing the agency's point of view. In 
terms of hurting people, to the--to the extent that we are 
trying to strike a balance between taking potentially some 
significant risk in a setting of a terminal illness and 
allowing patients to take that risk and make that informed 
judgment and then opening up that same risk to patients who 
don't necessarily face the same circumstances, we are certainly 
going to be exposing patients with potentially less severe 
conditions to a risk that we might think as a matter of public 
policy is only appropriate if we are being good stewards of the 
public health is only appropriate in a setting of a terminal 
illness.
    And so I think we need to be just cognizant of that. We are 
willing to allow patients to take certain risks in one setting. 
We think it is their right.
    The question is do we think it is appropriate for patients 
who are in a much different setting to contemplate those same 
risks outside of a regulatory process that we've carefully 
constructed. That makes careful balances.
    Mr. Walden. So help me understand this, if you can, the 
term life-threatening disease or condition. What--in real 
people speak, what does that mean? Who would be--what sort of 
conditions?
    Dr. Gottlieb. Well, as a physician who used to treat until 
recently hospitalized patients, I would consider advanced 
diabetes a life-threatening disease. I would consider class two 
heart failure a life-threatening disease.
    There's a lot of Americans with those conditions. They're 
not immediately life threatening. A lot of those patients will 
go on to live many years, but they face a chronic illness that 
is life-threatening, certainly. They might eventually succumb 
to their illness. That is a broad category of patients.
    So, with that language, we potentially open it up to a very 
broad category of patients, and I can tell you through 
discussions that I've had with attorneys at FDA I think we'd 
have to interpret that broadly.
    I don't think that we'd be able to, as a matter of our own 
interpretation of the law, further narrow that. I think, if 
anything, we would have to interpret that fairly expansively.
    Mr. Walden. All right. My time has expired. Thank you very 
much.
    Mr. Burgess. Chair thanks the gentleman. The gentleman 
yields back. The Chair recognizes the gentleman from Oregon, 
Dr. Schrader, 5 minutes for questions, please.
    Mr. Schrader. Thank you, Mr. Chairman. Appreciate that.
    Somewhat along, I guess, the same lines of the question 
that's been going on here, I guess, Mr. Gottlieb, seems like 
there are a lot of provider groups that are not enthusiastic 
about the need for this legislative change. Would you comment 
on that?
    Dr. Gottlieb. Well, we've heard from a lot of provider 
groups, certainly, and some groups that represent patients 
about concerns related to this legislation and I think the 
general concern is about the risk of undermining a regulatory 
process that has been carefully crafted over many years to 
strike a very careful balance.
    I think people do worry about upsetting that balance, given 
all the thought that has gone into how we've created that 
framework.
    Mr. Schrader. Was the--you have indicated that you already 
made some changes based on the GAO report. Was the report 
overall favorable or unfavorable to the current program?
    Dr. Gottlieb. I felt that--I can only speak for my own 
interpretation of the report--I felt the report was overall 
favorable--a favorable view of what FDA was doing with some 
targeted recommendations about improvements that we can make.
    Mr. Schrader. And, you know, again, it has been mentioned 
that 99 percent of the expanded use or compassionate use 
applications are approved.
    How does it get much better than that with this new 
legislation? How would this new legislation affect that 
approval rate?
    Dr. Gottlieb. Well, the legislation is certainly not going 
to affect--you know, affect an approval rate that, to your 
point, is 99 percent and actually getting better than 99 
percent is a sweep of over a decade, and, when you look in the 
more recent years, I think the agency has gotten even more 
vigilant at trying to move these things through the agency in 
an efficient fashion and approve these.
    I think there is a perception, and I can't speak to the 
perception, that there are certain companies and products that 
aren't necessarily being offered under the current construct 
and the Right to Try legislation might provide more of an 
incentive and an opportunity.
    Probably an opportunity incentive would be the wrong word--
an opportunity for companies to offer products in a different 
setting.
    I don't necessarily see that same opportunity, because I 
think that the biggest obstacle to offering drugs through 
expanded access is the supply constraints.
    I think we ought to think about how we address that 
separately. It think there might be ways to address that 
through incentives.
    But from my perception where I sit--and I've been on the 
other side of this--I've worked in--with small biotech 
companies before I came to the agency, as the committee knows--
the biggest obstacle I see is the availability of supply for 
patients who want to get access to unproven therapies.
    Mr. Schrader. Given the fact that there are all these 
States that are passing or have passed Right to Try 
legislation, why would they be doing that if the program seems 
to be working so well by, you know, your testimony--would 
indicate working so well at this time?
    Why are States doing that? Are we seeing a big upsurge or 
uptick in new drugs, new medical devices being approved in 
these Right to Try States we wouldn't through your process?
    Dr. Gottlieb. It is hard to tell. We don't have data yet, 
Congressman.
    I mentioned that I used a drug experimentally in my--in the 
treatment of my own cancer at the outset. I had a very curable 
cancer. I was told that I had an over 90 percent chance of 
curing my cancer.
    What I was looking for was how do I get 90 percent to 91 
and 92 percent, and the way I was going to do that was to look 
for pristine clinical data that could help inform me how to use 
available therapy in a better fashion to slightly improve my 
odds.
    That's very different from a patient who's told that they 
have a 10 or 20 percent chance of surviving their illness and 
they are looking for something very different.
    They're not looking necessarily for a study that's going to 
tell them how to get 20 percent to 25 percent. They are looking 
for something unproven--a silver bullet--something that could 
dramatically change their odds, and invariably that's going to 
be something experimental, and if it wasn't then they wouldn't 
be told that they only have a 20 percent chance of surviving.
    I think we need to make sure we serve both patients. I am 
not sure that we always do. I am committed to doing that. 
That's why we are working on the reforms that we are doing.
    I think that there's a broad perception out there that we 
don't always serve both patient communities well and that's 
been the impetus for these Right to Try laws.
    I think that there are things we can do. We'll certainly 
work with Congress on this legislation. If Congress passes it, 
we will certainly implement it in a robust fashion.
    I still think that there is a lot that I can do as the FDA 
Commissioner to try to improve programs for patients who are 
told that your chances of surviving your illness are 20 
percent.
    Mr. Schrader. Very good. Thank you. I yield back.
    Mr. Burgess. Chair thanks the gentleman. The gentleman 
yields back.
    The Chair recognizes the gentleman from Texas, Mr. Barton, 
the vice chairman of the full committee, 5 minutes for 
questions, please.
    Mr. Barton. Well, thank you, Mr. Chairman. Thank you for 
holding this hearing.
    Doctor, we appreciate you being here. I really just have 
one basic question and that is if we--if we believe in the 
doctor-patient relationship, which I do, if your doctor comes 
to the decision that all reasonable conventional therapeutic 
efforts have been exhausted in trying to protect your life and 
is willing to state that, and if the patient is willing to 
forego any legal lawsuit claims against some of these new 
therapies, why wouldn't the FDA approve that?
    And I am told at the staff level that the FDA has been 
extremely responsive the last 3 or 4 years in approving 
requests for new treatments when the patient has exhausted all 
of their options.
    But, you know, I listened to your answer to Chairman Walden 
and it sure does seem to me that even with the best of 
intentions the FDA still thinks they know better than the--than 
the doctor who's treating the patient.
    Dr. Gottlieb. Well, Congressman, I appreciate the 
questions. I am not sure that I agree with the conclusion.
    We do approve it. The bottom line is we do approve it and, 
you know, data has been quoted here that in more than 99 
percent of cases that we have a request even on an emergency 
basis or a nonemergency basis we do approve it, and in 10,000 
encounters, requests for expanded access in a nonemergency 
setting where we--where we denied about 25 of them, in about 
half of those denials it was because the drug just wasn't 
available and in other cases it is because we know that the 
drug is on a clinical hold for a significant safety reason but 
the public doesn't know that because the existence of the 
clinical hold is confidential information.
    You know, we are committed to continuing to push on this 
and to make it easier for patients to access it. I think the 
issue isn't do we approve it do we not approve it.
    The bottom line is in the vast, vast majority of cases we 
do approve it. The issue is, is it always available and do 
patients always know about it. And I think on the question of 
do patients always know that they can pursue these options, we 
can make that easier.
    We can make that information more readily available with 
the help of Congress and the provisions in the 21 Century 
Cures.
    On the question of whether or not it is always available, 
the answer is, unfortunately, it is not. Unfortunately, these 
products are supply constrained because of manufacturing 
constraints.
    I think there, too, there are things that we can do through 
how we design clinical trials that potentially could make more 
product available in the setting--in the preapproval setting.
    Mr. Barton. Then why not just empower the FDA to say that 
we approve it but you may not be able to get the drug--you may 
not be able to get the therapy because it is not available.
    Or, if you tell them no, say because this stuff is most of 
the time not working--we put a hold on it because it is not 
helping anybody, I mean, I am with you on that.
    But my brother died of liver cancer and they tried all the 
conventional therapies in the world on him and it just wasn't 
working, and we got him into a clinical trial that was helping 
90 percent of the liver cancer patients but the 10 percent it 
didn't help it expedited the disease and he was, unfortunately, 
one of the--in the 10 percent group that it accelerated his 
cancer as opposed to terminated it.
    But we knew what we were doing. We took that chance. He and 
his wife and his--myself and my mother, we all--and his pastor, 
we--we said we are going to give this a shot because if it 
works it will really help, and it didn't.
    But we didn't--we didn't then go back and say, oh, jump on 
the FDA for doing it. We knew up front what the risk was and I 
don't--I just don't see--I mean, Mr. Griffith has a bill before 
this committee right now, and there are others, let's err on 
the side of the doctor/patient knows more.
    And I am not being negative on the FDA but you're trying to 
protect the broad public health, which is commendable. But I 
would--I would say in this case let's pass some law that makes 
it easier to get this stuff--agreeing that in most cases you 
folks have been very positive about giving them the chance.
    With that, Mr. Chairman, I yield back.
    Mr. Guthrie [presiding]. Thank you. The gentleman yields 
back.
    I recognize Ms. Eshoo for 5 minutes for questions.
    Ms. Eshoo. Thank you, Mr. Chairman, and welcome, Dr. 
Gottlieb. It's wonderful to have you here, and congratulations 
on heading up the FDA.
    In listening to everyone, I am reminded that we are all a 
diagnosis away from something and I admire how you not only 
handled your own challenge.
    But it is a source of comfort to me that--not to you 
probably but that you had this challenge and that you can view 
so many of these issues through that lens and I think that that 
is very important and it has really added a lot to, I think, to 
your testimony today.
    What I am struggling to figure out what is broken here. The 
FDA has very good figures. I have read the GAO report and, 
overall, I agree with your description of it and they do add 
some things that the FDA can do.
    But what do you think is broken here? It is my 
understanding that if a patient--it starts with the patient. 
Patient goes to the doctor and says, I have either read out or 
I have heard about or whatever such and such a experimental 
drug and I want it.
    The doctor then has to request that of the manufacturer? Is 
there something broken down that breaks down in that process? 
Because we have bills before us that suggest that it is larger 
than what the numbers--what the data suggests.
    So can you identify what you think is broken?
    Dr. Gottlieb. Well, I would like to just start, 
Congresswoman--I appreciate the opportunity to answer your 
question.
    I am--in response to the last question, I am in favor of 
giving patients--sick patients options and in the setting of a 
patient who's suffering--in the setting of most patients that's 
the safe and approved option that's been reviewed by the FDA.
    Ms. Eshoo. Right.
    Dr. Gottlieb. But sometimes that's an unproven option and 
sometimes the risk of nothing is worse than the risk of 
something experimental and we need to consider that and we do 
consider that through our expanded access program.
    But this is a complicated issue, and to your point, there 
are things that aren't working that are frustrating the ability 
of patients even who have a physician who's willing to work 
with them, even who I have identified a drug that they think 
can help their illness, even with an FDA that is devoting a lot 
of new resources to trying to facilitate access to these 
products.
    Even with all of that, patients still have trouble getting 
access to products that they think can help save their life.
    Ms. Eshoo. But why are they having trouble getting access 
to it?
    Dr. Gottlieb. The supply--the biggest reason is supply.
    Ms. Eshoo. It is the supply?
    Dr. Gottlieb. The biggest reason is that when we do 
clinical trials--when companies do clinical trials, they don't 
have continuous manufacturing.
    They don't have large facilities online pumping out endless 
supplies of a drug. They will do what they--what they call 
discontinuous batches.
    They'll do--they'll do runs just to create batches of drug 
supply and API--active pharmaceutical ingredients--sufficient 
for the clinical trial and that supply doesn't go through the 
good manufacturing standards that a supply of drug goes through 
that's commercially available.
    Ms. Eshoo. On this supply issue, do either one of the bills 
address any of this?
    Dr. Gottlieb. No. We would have to think of different ways 
to provide incentives or perhaps a different clinical trial 
framework to try to get at that issue.
    Ms. Eshoo. Uh-huh. Now, one of the bills before us today 
would allow patients to access the investigational drugs while 
the other would allow patients to access investigational drugs 
and devices. That's a--that's a whole another very important 
area.
    Now, if patients are granted access to unapproved medical 
devices that a physician isn't trained to use, there could be, 
I think, some bad outcomes.
    Now, I understand that medical device companies already 
face many challenges to enrolling patients in clinical trials. 
The Right to Try proposals that include devices could divert 
patients from otherwise--I think from participating in a 
clinical trial.
    So give us your thoughts on Right to Try legislation 
including medical devices in addition to drugs.
    Dr. Gottlieb. Well, I think your statement is correct. I 
agree with it. Medical devices are tools in the hands of 
physicians. Physicians often have to undergo very rigorous 
training on devices, even after they are newly approved.
    And so there's a different set of considerations and 
potential risks associated with making devices available in a 
setting where you don't have the normal structure--regulatory 
structure in place.
    But setting that aside, we believe that the compassionate 
use framework on the medical device side of our house is 
working quite well, has a very quick turnaround time.
    We don't necessarily see the same considerations in that 
setting that we see in the setting of new drugs nor are we 
likely to see the availability of the devices to be used in a 
preapproval way like we might have.
    At least in certain circumstances, you have drug supply 
preapproval that could be lotteried out in many cases to 
patients who want to get access to it on an expanded access 
basis. In the medical device setting, you typically would not 
have excess medical device supply.
    Ms. Eshoo. I don't understand your answer. [Laughter.]
    Do you--are you saying that you don't think it is necessary 
to include devices to drugs?
    Dr. Gottlieb. We don't--we don't see the same--I don't see 
the same concerns in part because the compassionate use program 
on the medical device side house--of the house is working well 
and I also would say I don't see the same opportunity for 
patients because to the extent that I've said that the supply 
of the drugs is constrained preapproval the supply of devices 
preapproval is even more constrained.
    Ms. Eshoo. Thank you very much.
    Thank you, Mr. Chairman.
    Mr. Burgess [presiding]. Gentlelady yields back.
    The Chair recognizes the gentleman from Kentucky, Mr. 
Guthrie, 5 minutes for questions, please.
    Mr. Guthrie. Thank you very much, and I will ask a 
question, Dr. Gottlieb. Thanks for coming. I appreciate you 
being here.
    Kind of the scenario that my friend from Texas, Mr. Barton, 
gave when he said that his brother or people get to the point 
where the doctor said everything conventionally has been done 
for you--there's nothing else we can do for you and then you 
have the right to try, and that's a traumatic time. I know it 
is a traumatic thing and people are looking at opportunities 
and that's what's available for them.
    So they choose to go to the experimental side--the unproven 
side--and agree to pay for that treatment. So but what's 
unclear in the legislation is what if--and I quote, you said 
that you could get unwanted side effects.
    So what if the unwanted side effects creates a whole series 
of health--puts them back in the hospital? And so instead of 
agreeing to pay what--an X amount for some kind of treatment 
all of a sudden there's new hospital bills that could be 
astronomical that's not looked at in the legislation.
    So my question, do you have any insight or opinion on how 
to best examine or solve this type of issue?
    Dr. Gottlieb. Well, I think it is one of the unknowns 
associated with using, you know, any product that hasn't gone 
through a full evaluation where we don't know the scope of the 
effectiveness of the product, you know, and we don't know--we 
certainly don't know the full scope of the side effects.
    A product that has gone through a Phase 1 clinical trial--
we call a Phase 1 trial a safety trial, but it is a trial for 
determining safety--to answer the question on whether or not 
the drug can proceed into the next phases of clinical trials 
doesn't fully establish the safety profile of the product.
    We are continuously learning about the safety of a product 
all through the three phases of a clinical study, and, in fact, 
a lot of what we learn about the safety of products is in the 
post-market setting.
    So there are a lot of unknowns in this setting and we need 
to be cognizant of that and, you know, patients who use these 
products through an expanded access program we make sure that 
they are cognizant of it.
    Mr. Guthrie. But my question gets into if they agree to pay 
for this and then it leads into further medical costs outside 
of just the experiment that puts them back in the hospital and 
so forth, which I guess would be part of it, do you have any 
opinion how that should be addressed?
    Dr. Gottlieb. The system--I would--I would put that 
question to my colleague, Seema Verma, at CMS. I mean, this is 
going to be an issue for the broader health system and for the 
payers to have to contemplate because the cost would be--would 
be born back on the--on the payer system.
    Mr. Guthrie. OK. Thanks for that.
    And I do want to mention just a comment to you while you're 
here. I do want to mention one more issue regarding the 
potential threat of glass fragment contamination.
    As you may know, the FDA issued an advisory regarding glass 
fragment contamination for injectable drugs in 2011. I ask that 
you look into and fully consider updating the advisory to 
reflect recent discoveries. So no reason for that.
    And for my final couple of minutes, I understand--and 
there's an Equifax hearing going on downstairs, so I was there 
earlier and I understand you talked about your trip to Puerto 
Rico.
    I just ran into my colleague on the way up here from the 
Virgin Islands and, you know, it is very dramatic or very--very 
drastic situation and dramatic as well and that's going on 
there.
    And would you just kind of update us on what you're doing 
to combat potential drug shortages and access to issues that 
may come as a result of damages. I know your trip to Puerto 
Rico and also the Virgin Islands.
    Dr. Gottlieb. As I mentioned, we have a list of about 40 
drugs that we are very concerned about. It reflects maybe about 
10 different first. These are drugs--13 of them are sole-
sourced drugs.
    They're only manufactured in Puerto Rico to supply the 
entire U.S. market and these are--these are important 
medicines. These are drugs--these are HIV medications and 
chemotherapeutics and injectable drugs that are hard to 
manufacture.
    There's biologics. There's very sophisticated medical 
devices manufactured down there. The biggest issue right now--
well, there's a lot of issues.
    One is getting gasoline and basic sustenance to employees 
so they can return to work. People are living in very difficult 
circumstances there and I met a lot of--a lot of local resident 
who work for FDA. But the longer-term issue that we are 
grappling with and worried about is power supply.
    The grid is probably going to be stood back up. They'll 
create some micro grids. They won't stand up the whole 
electrical grid. They'll create micro grids.
    But the challenge for the manufacturers is that they need 
stable power and typically they need dual feeds coming in 
because of the equipment that they use. And we know that the 
grid is going to be unstable for a long period of time.
    In fact the power company would like to reconnect the 
manufacturing facilities because as they bring up the power 
system they need load balance and the manufacturers are regular 
users of power. But manufacturers want to stay off the grid 
right now.
    And so they are going to be operating for long periods of 
time, potentially, on their generators--generators that were 
never meant to operate for months and months on end.
    So they don't have necessarily the fuel Thanks to do it and 
they might not have generators that are up to that challenge.
    And so we are trying to trouble shoot that with them on an 
individual basis now and trying to put in place contingencies 
if things do go wrong and backups if we need them.
    We've been doing that manufacturer by manufacturer, working 
very closely with DHS and the staff of the Governor of Puerto 
Rico, who we are now in personal contact with who understands 
the implications and the importance of this manufacturing base 
not just for all of the United States but for the island of 
Puerto Rico as well.
    Mr. Guthrie. Thank you very much. I appreciate your 
efforts.
    Mr. Burgess. Chair thanks the gentleman. The gentleman 
yields back.
    The Chair recognizes the gentleman from Maryland, Mr. 
Sarbanes, 5 minutes for questions, please.
    Mr. Sarbanes. Thank you, Mr. Chairman.
    Thank you, Dr. Gottlieb, for your testimony here today. I 
am sort of picking up on the line of questioning from 
Congresswoman Eshoo in terms of trying to understand what the 
piece of this process that you would view--that one would view 
as broken when you've got over a 99 percent rate of responding 
to these requests for approval.
    And, obviously, there is a constituency out there that 
feels that notwithstanding what the FDA is doing in its efforts 
to respond to these inquiries and requests that there's still 
something more than can be done in terms of accessing expanded 
treatment options.
    So maybe--could you give me the 30-second caution that 
FDA--sort of a digest of a lot of what you've been saying--the 
caution you would give us as we are examining and reviewing and 
debating the pieces of legislation that kind of stimulated the 
hearing today and that we got some testimony about at the 
outset?
    From your perspective, what would you just say to us--
here's what I would look out for, be cautious about as you're 
examining the kind of Right to Try legislation that's being 
proposed?
    Dr. Gottlieb. Well, I would just say, you know, you asked 
about the--and I know I am limited to 30 seconds. You asked 
about the obstacles.
    You know, one of the obstacles is are patients informed and 
we are trying to do all we can to make sure they are informed 
of these opportunities.
    The other obstacle I talked about was--which is just the 
supply. That's harder to fix. I think there's a perception that 
this legislation will create more pressure on companies to 
offer the drug so that might create more pressure on the 
companies to have a supply available.
    I think that's an open question. I think that's something 
Congress should contemplate.
    But in terms of the question of the caution, you know, in 
addition to the technical assistance we've provided that is 
more detailed about legislative language, I just would be 
mindful that we don't create a process and a policy framework 
where the only people who take advantage of the avenue are 
people who have the least promising products.
    I think what we want to--what we want to do is create a 
framework where the most promising products are being made 
available to patients and this doesn't become sort of an 
opportunity for those sponsors or maybe even individual 
clinicians who want to do some advanced marketing of a product 
to use this vehicle.
    And I am not saying that this legislation will do that. I 
am just saying if I was providing feedback to Congress of what 
to be mindful of, that's something that I would caution 
Congress around.
    Mr. Sarbanes. Well, actually I appreciate that because 
you've led--in your answer you've gone right to the place that 
I have some anxiety about, which is the potential to create 
something that may start small but would grow as a kind of 
unregulated space and that once established as a kind of 
alternative route, not just for patients that are genuinely 
seeking whatever option is available to them but for 
manufacturers as well, it becomes a kind of alternative space 
in which to operate and then it could be vulnerable to some 
unscrupulous activity, in a sense creating a place where the 
opportunity to experiment with experimental drugs is expanded 
and that's what makes me a little bit nervous.
    So in the 1 minute that's left maybe you could speak to 
that.
    Dr. Gottlieb. Well, I would build on it by saying--you used 
the word ``manufacturer.'' I would build on it by saying it is 
not just the manufacturer.
    We recently took regulatory action against two clinics that 
were marketing unapproved products as regenerative medicine. In 
one case, we had U.S. marshals seize a product that we felt was 
creating certain public health concerns, and I won't get into 
the details of it today since it is an ongoing activity.
    But there's also going to be individual providers who 
potentially could promulgate products under this--under this 
framework and one of the--one of the elements of feedback that 
we've given to Congress through our technical assistance is to 
make sure that the patient protections that Congress intended 
to be available under this legislation are also available to 
patients who are getting products directly from physicians or 
physician-operated clinics and not just manufacturer because 
the way Congress crafted the draft legislation it could be 
interpreted in certain settings as those patient protections 
only applying to products promulgated by manufacturers, by 
sponsors, and in fact under this legislation it will also be 
providers who are promulgating products.
    Mr. Sarbanes. OK. That's very helpful. Thank you.
    Mr. Burgess. Gentleman yields back. The Chair thanks the 
gentleman.
    The Chair recognizes the gentleman from New Jersey, Mr. 
Lance, 5 minutes for questions, please.
    Mr. Lance. Thank you, Mr. Chairman.
    Good morning to you, Dr. Gottlieb. The legislation passed 
unanimously in the Senate. Were you involved in that or was it 
a situation with your--with a predecessor?
    Dr. Gottlieb. That happened on my predecessor's watch.
    Mr. Lance. And it is unusual--not unique but it is unusual 
when legislation passes unanimously in the Senate I think that 
would be fair to say and from your perspective, reviewing it, 
knowing that you were not then in charge, why do you think that 
this legislation passed unanimously in the Senate?
    Dr. Gottlieb. Actually, I'll reopen the record to say I am 
not sure the date that it passed. It might have been on my 
watch but----
    Mr. Lance. Fair enough.
    Dr. Gottlieb [continuing]. So we'll just--we'll leave it 
open.
    Mr. Lance. But you were new to your responsibilities. I 
understand that.
    Dr. Gottlieb. But, look, I think as I've stated in my 
comments here today, this touches on a very important issue and 
it touches on an issue that I think is very visceral for most 
Americans.
    We have all--most of us have seen loved ones, 
unfortunately, or friends succumb to serious illness and in 
certain situations we've seen them do that in a setting of 
feeling like they didn't have good options to try to beat 
back--beat back a serious illness.
    And so, you know, the idea of being able to get access, we 
are seeing all this new technology, all these extremely 
promising drugs and development.
    We are seeing the potential to fundamentally cure pediatric 
inherited disorders through things like gene therapy and 
regenerative medicine.
    With all this technology coming online, I understand the 
desire of people who are--who are stricken with the disease now 
to want access to that. I think that this--I think this 
phenomenon is being driven in part by the opportunities we have 
available to us now.
    Mr. Lance. Thank you. As a matter of full disclosure, since 
you have kindly indicated you might technically have been in 
charge but certainly the bulk of the work in the Senate was 
before you were in charge, I have worked with Congressman 
Fitzpatrick.
    The district that I am honored to represent borders his 
district although we are in different States, and I've worked 
with Mr. Worthington, who is in this room, on this very 
important issue.
    The FDA may place a clinical hold on a drug. If, for 
example, human volunteers are being subject to unreasonable and 
significant risks of illness or injury, has the FDA placed a 
clinical hold on a drug as a result of an adverse event during 
an expanded access protocol?
    Dr. Gottlieb. I don't know the answer to the question. I 
would tend to think not, just given the numbers of situations 
where we've recognized adverse events in the setting of an 
expanded access program that have led to any kind of regulatory 
decision.
    You know, we've done some systematic looks back and found 
very few instances where something we observed in the setting 
of an expanded access program has prompted us to take certain 
regulatory actions.
    Certainly, the inverse case where we have--you know, I 
think I mentioned previously a large percentage of the very 
small number of cases where we might deny a patient a request 
for--to use a drug in an expanded access setting is predicated 
on the existence of a clinical hold that might not be known to 
the public because it is commercially confidential information.
    Mr. Lance. Thank you. If you would, at your convenience 
could you get back to us, to the subcommittee, on whether or 
not that has occurred? I would appreciate that.
    Dr. Gottlieb. Sure.
    Mr. Lance. Thank you.
    This is a very difficult issue and I certainly understand 
your point of view. I think there are many of us in Congress 
who are sympathetic to what occurred in the Senate and, 
certainly, sympathetic to the legislation of our colleagues who 
testified, to my immediate right, and we want to continue to 
work with you.
    But, certainly, I believe there is merit to the legislation 
that's being considered.
    Thank you for your testimony, Dr. Gottlieb.
    Dr. Gottlieb. Thanks a lot, Congressman.
    Mr. Burgess. Chair thanks the gentleman. Gentleman yields 
back.
    The Chair recognizes the gentleman from Virginia, Mr. 
Griffith, 5 minutes for questions, please.
    Mr. Griffith. Thank you very much, Mr. Chairman. Thank you, 
Dr. Gottlieb for being here with us today.
    In testimony earlier today you were talking about the 
definition of terminal. I would note that neither House bill, 
neither Mr. Fitzpatrick and Mr. Biggs' or mine goes the step 
beyond terminal--that the Senate went and I can appreciate 
that. Then we got into, you know, what the definition of 
terminal ought to be.
    I am happy to work with you all on that. I think, if I 
remember correctly, and you correct me if I am wrong, that you 
indicated somewhere around 20 percent survival odds was where 
you would probably put it. I'd probably push it a little 
higher.
    Dr. Gottlieb. I wouldn't. I didn't----
    Mr. Griffith. I misunderstood----
    Dr. Gottlieb [continuing]. Mean to suggest that there is an 
objective figure. I was just using the example of a patient 
who's given a very grim prognosis. I would certainly consider 
20 percent odds of survival grim.
    Mr. Griffith. And I would, too, and I think that's where 
this is coming from. I might push that a little higher. 
Anything less than 50/50--you know, if it were me, I'd want to 
be able to find out what was out there.
    Dr. Gottlieb. It was grim when I was told it was 90 percent 
odds of living 5 years. That felt pretty grim at the time, too, 
Congressman.
    Mr. Griffith. Yes, sir. I can appreciate that.
    So I want to work with you on that, but I do think that we 
need to pass something, and we'll try to figure it out.
    But I can see where you're concerned about chronic--you 
know, FDA was created in 1906 to protect Americans, not to get 
in the way of them taking treatment and I will--if it were to 
be me and I--right now, I am fine.
    But as Ms. Eshoo said, we are all one diagnosis away from 
facing something. I would take the chance with the silver 
bullet or the Hail Mary, and we are going to hear testimony 
later today that the--some wealthy Americans are going to other 
countries to get treatments or to get drugs.
    And so my question would be if it has already been approved 
somewhere else and you have a terminal diagnosis, why shouldn't 
you be able to get that in the United States?
    Dr. Gottlieb. Well, I think you're touching on the issue of 
reciprocity, which is--which is some legislation that has been 
introduced in other--other settings, whether or not FDA should 
predicate approvals here in the U.S. on the basis of foreign 
approvals.
    And we are certainly happy to work with Congress on those 
legislative ideas. The framework that we operate in right now 
is a requirement that we determine safety and efficacy based on 
our statute and clinical trials that we work with sponsors to 
conduct and evaluate.
    You know, another element of this consideration is also 
relying more on foreign data, which we are doing as a matter of 
regulatory policy and that is something we can do without new 
legislative authority.
    We can do that within the constructs of our current 
regulatory considerations and we are looking for ways to do 
that.
    Mr. Griffith. And I appreciate that and I appreciated your 
comments earlier about some of the new things that you're doing 
and that you're announcing today and I appreciate that as well.
    You know, the GAO report found that when the FDA did not 
allow a request for expanded access to proceed one of the 
reasons listed was due to the requested drugs demonstrated a 
lack of efficacy for its intended use.
    But what if data showed that the intended use--it may not 
have been the intended use but that it actually had benefits 
that were unexpected in another area and you're facing that 
terminal illness that it does have the benefit for. I am just 
curious how the FDA would deal with that in its current 
process.
    Dr. Gottlieb. Well, most of the cases where we are 
authorizing or, you know, allowing drugs to be used, in 99 
percent of the cases where we--where we allow patients to use 
drugs in the setting of expanded access it's in a setting that 
is not the intended use of the product--that they'd be used for 
which the drug itself is being studied.
    So it's in an unstudied indication or an indication that 
might be being evaluated in very small clinical trials.
    When we--when you--when the GAO says that it was something 
when we didn't allow it to be used and something unproven 
because of something we knew, typically it was something we 
knew because we get a lot of clinical data from a lot of 
different sponsors and we might know that a drug in a certain 
class doesn't work in that class because of other data that we 
are seeing.
    And so then we might make a judgment that we shouldn't 
allow that drug to be made available in a setting of expanded 
access when we have objective proof that it's not going to 
provide any benefit.
    I mean, bear in mind, we know that 70 percent of all drugs 
that are offered in an expanded access are never approved by 
FDA. So the vast majority of people who will use a drug through 
expanded access are using a drug that doesn't work.
    Mr. Griffith. And I appreciate that. I am running out of 
time so I am just going to make one last comment. I do think 
that the two House versions both have device in there.
    I think we should keep that because, as you said, science 
is moving fairly quickly. That's one of the reasons that people 
want to try these things before you all have had a chance.
    There's some wonderful things out there with science. 
Again, if I had a diagnosis with inoperable cancer and they had 
a new nanobot technology, I'd be finding out where I could get 
that, and that is considered a medical device and it may be 
very, very helpful.
    Not ready yet, but if it were when I was ready or needed 
it, I'd want to be able to use it.
    Thank you so much for your time, and I yield back.
    Mr. Burgess. Gentleman yields back. The Chair thanks the 
gentleman.
    The Chair recognizes the gentleman from North Carolina, Mr. 
Hudson, 5 minutes for questions, please.
    Mr. Hudson. Thank you. Dr. Gottlieb, thank you for being 
here today with your testimony.
    FDA categorizes expanded access and the four different 
types of requests, as you're aware, are single patient, single 
patient emergency, intermediate size, and treatment for 
widespread populations.
    While the standard process seems to get a lot of attention, 
I'd like to ask more about the intermediate size and treatment 
for widespread populations.
    How are these two types of requests separate and unique 
from the larger clinical trial?
    Dr. Gottlieb. Congressman, we could get you more detailed 
information because there's a spectrum of opportunities.
    It is the case that, for example, and I think you mentioned 
this--when a drug is--in the period of time when it's completed 
its clinical trials but is awaiting approval decisions, 
companies will open up large expanded access programs typically 
like simple large protocols and offer drugs on a protocol 
basis.
    I think that these are--these are important opportunities 
because what we are talking about today, a one-off request for 
a drug--an individual patient and their doctor working with the 
agency to ask for a drug in a single situation.
    I think what we'd like to see is more opportunities to 
offer products in things like simple large safety trials and 
certain simple protocols where patients aren't being randomized 
but some basic information is being collected that can help 
inform--inform what we know about that product but also provide 
for more wide scale access.
    And this gets into a broader question around how do we 
embrace different clinical trial designs and if we can go down 
these routes we can come up with constructs I think can enable 
much broader access preapproval.
    Mr. Hudson. Makes sense. Are these patients incorporated 
into the broader clinical trial population for the purposes of 
data collection and efficacy?
    Dr. Gottlieb. Sometimes. Sometimes we are collecting data 
from these kinds of protocols. Sometimes we are not.
    I think to the extent that we can get into collecting more 
data and being able to make efficient use of that data it can 
help accelerate the development process.
    So this is something, you know, that we are looking at when 
we talk about seamless clinical trials. You know, we talk about 
allowing the study of different indications within the confines 
of a single clinical trial.
    These are all some of the new scientific frameworks that we 
are looking at to try to--try to evolve how we do clinical 
trials and I think can both allow us to get better information 
and make the development process itself more efficient but also 
enable larger, more access to drugs preapproval and in some 
kind of clinical trial where there is--where there is good 
protections being afforded to patients as well.
    Mr. Hudson. Have there been any cases where patients have 
been denied access to a clinical trial but received access 
through an intermediate size or treatments for widespread 
populations as a result of the expanded access program?
    Dr. Gottlieb. Oh, I am sure there has, Congressman.
    Mr. Hudson. Is the expanded access program alone adequate 
to address the needs of patients and physicians who are seeking 
to obtain investigational drugs?
    Dr. Gottlieb. Well, I don't think we'd be here today if 
there was a perception by Congress and the broader community 
that the existing system was adequate.
    And I am not going to tell you that the existing system is 
perfect. That's why we announced a set of changes today and 
that is why, as part of that announcement, I committed to do 
additional things down the road that--some of which we are 
working on right now to help continue to improve that process.
    Mr. Hudson. Great. We look forward to working with you on 
that.
    Dr. Gottlieb. Thanks a lot.
    Mr. Hudson. With that, Mr. Chairman, I will yield back.
    Mr. Burgess. Chair thanks the gentleman.
    The Chair recognizes the gentleman from Georgia, Mr. 
Carter, 5 minutes for questions, please.
    Mr. Carter. Thank you, Dr. Gottlieb, for being here.
    Help me understand, basically. We are talking about two 
different scenarios here. We are talking about drugs that have 
been approved already by the FDA for something but what they 
are wanting to be used for is not an indication so physicians 
are trying to use it off label, if you will.
    And we are also talking about investigational drugs that 
have not been approved yet but are in the pipeline and is--am I 
right in that?
    Dr. Gottlieb. Well, I think that you're right that those 
are two constructs that exist for patients to get access to 
unproven therapy.
    I was a patient who used an approved product in an off-
label fashion and that is actually typically what you see in 
these settings.
    You'll see products used--especially oncology you'll see 
products used off-label. I think what we are focused on with 
respect to the legislation here, respectfully, is the second 
scenario that you offered, which is a product that hasn't yet 
been approved by the FDA but patients want to use it in an 
experimental or investigational way.
    Mr. Carter. OK. It's my understand the FDA--your 
responsibility is to protect the public from any side effects, 
any bad effects that a medication may have but also to make 
sure that it's available if it could benefit the public as 
well. Is that correct?
    Dr. Gottlieb. Well, I think the scope of the FDA's mission 
is broader. I think the scope of our mission and our 
responsibility to patients is much broader in this context.
    I would--I would tweak it by saying I think our 
responsibility is to make sure that patients and providers are 
fully informed of both the risks and the benefits in these 
settings.
    Mr. Carter. OK. Having said that, can you explain to me why 
the FDA keeps putting their head in the sand when it comes to 
medical marijuana?
    I am not--and I don't want to hear marijuana is a Schedule 
One drug for investigational use only. But here we have--I 
don't know how many States we are up to now--that have approved 
it.
    Here we have all these States, and most of them with a 
different strength of what they've approved, and yet the FDA 
just continues to ignore that.
    Isn't it your responsibility to address that?
    Dr. Gottlieb. Well, I see people who are developing 
products based on marijuana, making all kinds of clinical 
claims on the market.
    I see people who are developing products making claims that 
marijuana has antitumor effects in the setting of cancer, and I 
think reasonable people can ask reasonable questions about 
whether marijuana is a chemotherapeutic agent.
    So, you know, it's a much broader question, Congressman, 
about where our responsibility is to step into this and start 
to ask questions about the claims that are being made.
    Mr. Carter. And that is my question. Where does your 
responsibility come in? It would appear to me, when you've got 
all these States that are approving it, it would appear that 
the FDA should be stepping in to give some kind of consistency 
here.
    Dr. Gottlieb. Well, I think that we'll have some answers to 
this question very soon because I think we do bear a 
responsibility to start to address these questions.
    Mr. Carter. Let me ask you, the bills that we are 
considering today how will that change your approach? Will it 
change your approach at all? Will it change your role in the 
process at all?
    Dr. Gottlieb. If these bills are passed, we look forward to 
working with Congress to make sure that they are faithfully 
implemented.
    It will--it will open up a new vehicle for patients to 
potentially get access to certain therapies. I think the 
question that I outlined throughout my testimony today still 
remains about whether or not sponsors will offer these 
opportunities on any--on any greater basis and whether or not 
this legislation alone is enough to compel sponsors to have 
supply available to offer products more generously on an 
expanded access basis.
    I think that those questions remain unanswered. I don't 
have an answer to those questions.
    Mr. Carter. OK. Two more things, real quick.
    First of all, you've read over the legislation, I assume, 
that is being proposed?
    Dr. Gottlieb. Certainly.
    Mr. Carter. Is there any part of it that you think that the 
FDA potentially could have trouble because what--understanding 
or implementing because what I don't want to happen is to have 
legislative intent interpreted by the agency when that is not 
what we were intending to do?
    Dr. Gottlieb. Well, I mean, I've outlined some of the--some 
of the places----
    Mr. Carter. We've had that experience before in other 
areas.
    Dr. Gottlieb. Right. Well, look, and legislation can be 
interpreted differently by different FDA Commissioners as well, 
as you're well aware.
    I have outlined some of the areas where we think that there 
might be ambiguity in the current language right now where 
Congress might take closer consideration of how certain things 
are crafted and how certain things are worded to potentially 
tighten this up.
    And we have tried to be constructive. We will continue to 
try to be constructive and work with Congress if this 
legislation does advance.
    Mr. Carter. OK. Once last question--I just--you know, and I 
go back to my question at the beginning--I am to understand 
your answer about medical marijuana is that FDA is going to be 
addressing that situation very soon?
    Dr. Gottlieb. Congressman, you know, the question is should 
we be taking enforcement action against people who are making 
certain claims in the market? Because I don't necessarily 
understand your question. We don't have--we have 20----
    Mr. Carter. My question is simple: Why does the FDA 
continue to ignore medical marijuana when we have States who 
are approving it?
    Dr. Gottlieb. Well, this----
    Mr. Carter. We have States who are actually taking on the 
responsibility of approving medications.
    Dr. Gottlieb. We have two frameworks that we operate in.
    One is sponsors who bring us applications requesting that 
we approve a product for a certain intended use. We have 20 
INDs and active INDs in house right now that are for marijuana 
products.
    They are typically for marijuana extracts because 
delivering an active pharmaceutical ingredient through 
inhalation isn't always the most efficient route.
    The other question that gets to your question is whether or 
not there are certain claims being made in the market by people 
who are marketing marijuana in interstate commerce that are 
unapproved new drug claims and could potentially put people at 
risk. That's a separate question.
    I think that we are addressing--we will address the sweep 
of these questions in time, including the questions put before 
us from sponsors that have 20 INDs.
    Mr. Carter. If you can -- can I get some kind of idea of 
when you're going to address this?
    Dr. Gottlieb. Well, we have 20 INDs in house and so we are 
addressing those as part of our review process.
    Mr. Carter. Do those 20 INDs have all these States 
approving them already?
    Dr. Gottlieb. These are--these typically are sponsors who 
are putting products through--trying to put products through a 
scientific process and not just marketing it on a Web site.
    Mr. Burgess. And the Chair would advise there's likely to 
be a multiagency approach to this. It is not going to be 
exclusively through the Food and Drug Administration.
    Mr. Carter. And that is well understood. But, certainly, 
they have a role in it that I feel like they are ignoring.
    Mr. Burgess. And the gentleman's time is expired and the 
Chair will recognize the gentlelady from Colorado, Ms. DeGette, 
5 minutes for questions, please.
    Ms. DeGette. Thank you very much, Mr. Chairman. Tempting 
though it may be to follow up on this medical marijuana, being 
from Colorado, I want to talk to you----
    [Laughter.]
    Ms. DeGette [continuing]. Broadly about the current 
safeguards that are in place under the FDA's expanded access 
program to protect patients.
    Can you please describe those for me?
    Dr. Gottlieb. The safeguards that we have in place with 
respect to patients who get products through our current----
    Ms. DeGette. Right.
    Dr. Gottlieb. Well, the requests come into to FDA and we 
are asked to evaluate them, and we do go through the protocols 
and make certain assessments and in certain cases we provide 
feedback to the providers.
    As has been stated here, we grant over 99 percent of the 
requests. But there are about 10 percent where we make certain 
modifications to protect patient safety and the most common 
modification that we'll make is to give feedback to adjust the 
dose and that will be on information that we might have about 
what the--what the most potentially beneficial dose of the 
product might be.
    Another modification that we'll oftentimes make is on the 
informed consent. Sometimes the consent that is being provided 
to the patient might not be comprehensive. And so we'll ask for 
modifications to be made to the informed consent.
    So that gives you a flavor of the kinds of protections that 
we think we are providing by being part of this process and 
part of the evaluation.
    Ms. DeGette. You know, as you say in your testimony, the 
Senate Right to Try legislation tries to apply some of the 
protections to investigational product use under Right to Try, 
but it doesn't make clear that the requirements apply to all 
individuals who might provide a drug under Right to Try.
    Can you explain how this loophole might be exploited?
    Dr. Gottlieb. Well, I think you're referring to the--how 
the legislation currently tries to map to existing regulations 
in terms of importing some of the existing patient protections 
that exist in regulation to apply to patients in the setting in 
one version of the bill.
    The way we interpret it there is the potential that as a 
matter of law you could interpret the regulations that exist as 
applying to sponsors, companies, and I think what we are likely 
to experience in the setting of Right to Try, if we look at 
some of the anecdotal experience in the States--and right now 
we only have anecdotal experience, because we don't have any 
data about the availability of drugs that have been provided 
through these Right to Try laws--but it is possible that it 
will be the case that some of the products that will be offered 
under the framework contemplated by this legislation will be 
offered by individual sponsors or small clinics that might not 
qualify as a sponsor for purposes of the way the regulation is 
currently crafted.
    Ms. DeGette. Yes. Thanks.
    Now, as I understand it, of the 99 percent of requests for 
expanded access that FDA has approved, the agency proposed 
changes in 10 percent of the applications to ensure patient 
safety either through dosing changes, informed consent, or 
safety monitoring.
    Under the Senate-passed legislation, the FDA review of INDs 
would no longer be required. Can you talk to us a little bit 
further about how--about what you see the FDA's role in 
reviewing these INDs and whether it protects patients--whether 
under this new legislative paradigm some things could 
potentially be missed because the FDA is not reviewing it?
    Dr. Gottlieb. Well, we certainly believe that we are 
helping to provide additional safeguards and protections to 
patients. I think we would state very strongly that we also 
think we are providing additional opportunities to patients 
because, you know, in terms of--you mentioned the issue of the 
dose adjustments.
    Sometimes we will request dose adjustments because we might 
have information to suggest, based on other trials ongoing that 
we are looking at, that if there is a benefit to be derived it 
would have to be a higher dose or it might have to be a lower 
dose.
    And so we are making adjustments to help maximize the 
opportunity for the patient to derive a benefit and not 
experience a side effect.
    Ms. DeGette. So what I am hearing you say is, is the agency 
is really concerned about making sure these--that the dosages 
are correct and all of that.
    You're not really trying to use this as a barrier to people 
getting much-needed medication for some of these diseases.
    Dr. Gottlieb. Well, I think statistics speak louder than 
anecdote and if we are granting well over 99 percent of these 
requests, both the emergency and nonemergency requests, the 
agency--the agency's process once a patient walks up to the 
door and is able to walk through that process, that process 
where we are applying a level of review is not in and of itself 
a barrier.
    I mean, the numbers demonstrate that. I mean, the question 
is are patients able to walk up to that door and that's where 
we are making reforms and trying to put in place new tools like 
the Navigator to get more patients into that door.
    Ms. DeGette. And you're open to more requests like that?
    Dr. Gottlieb. We absolutely are.
    Ms. DeGette. Thank you. I yield back.
    Mr. Burgess. Chair thanks the gentlelady. The gentlelady 
yields back.
    And recognizes the gentlelady from Indiana, Mrs. Brooks, 5 
minutes for questions, please.
    Mrs. Brooks. Thank you, Mr. Chairman, and good to see you, 
Dr. Gottlieb.
    I want to continue to discuss briefly about the expanded 
access program but then also I want to make sure we spend a 
little bit of time just talking about your recent trip to 
Puerto Rico.
    But with respect to the expanded access and the FDA's 
desire to increase the requests and so forth, of the 99 percent 
of the requests made for expanded access and which are 
approved, I understand that only about 30 percent of those 
therapies actually make it through the full clinical trial 
process.
    And so what are the steps a manufacturer has to then go 
through to proceed on with the clinical trial when they are 
including the expanded access?
    Dr. Gottlieb. Well----
    Mrs. Brooks. How does it impact their clinical trials?
    Dr. Gottlieb. We would say it doesn't impact the clinical 
trials and, you know, one of the questions has been does--could 
something observed in the setting of an expanded access program 
where you have drugs being provided in a more unstructured way, 
typically by physicians who might not be as familiar with the 
product itself--could something--could an observation made in 
that setting go on to help delay the development process and 
that's always been argued to be something that causes 
manufacturers' reluctance to offer these.
    We would say no, and what I would say simply in response to 
your question is these two things can exist in parallel and 
they do exist in parallel.
    Companies will offer drugs on an expanded access basis and 
they'll have an ongoing clinical development program. The 
question for the sponsor--and I mentioned earlier I have been 
on the other side of this working with small biotech companies 
before coming into this position--the question for the sponsor 
is just the ability to both service the expanded access 
program--these oftentimes are small companies--but also have 
the product available--also have the supply.
    Mrs. Brooks. And then does the process--how do the results 
from the expanded access--are they included in the data and the 
findings in the clinical trials or are they in a little 
separate set of findings?
    Dr. Gottlieb. Well, we don't sequester the information but 
what we've said today in the guidance that we promulgated and 
what we've observed when we've gone back and looked at this 
systematically is typically the information, if there is any 
information to be gleaned from the expanded access program, 
doesn't have an impact on the development program one way or 
the other.
    We found very few situations--we looked at 321 regulatory 
approvals over a 10-year period. Twenty-eight percent of the 
drugs had expanded access.
    We could find only two instances where something observed 
in the expanded access setting informed the drug approval.
    In one case, it led to labeling around a certain safety 
issue and in one case it actually helped us approve the drug by 
helping to augment the information we had about the 
effectiveness of the product.
    So it is atypical, very atypical, that information gleaned 
in this setting would impact the drug approval and the guidance 
we put out today is sort of doubling down on our assertion that 
it is atypical.
    We are saying it is very, very atypical that we would 
consider something in that setting in part because these 
settings are very unstructured and the patients are very sick.
    Mrs. Brooks. Thank you. I would like to turn to Puerto Rico 
and thank you for making the trip to Puerto Rico and, 
obviously, manufacturing over 50 pharmaceutical facilities--as 
you said, thousands of employees producing treatments for 
cancer, HIV, immunosuppressants, and so forth.
    Has the agency ever faced this kind of challenge before 
after a natural disaster and has FDA ever dealt with something 
with this much impact, with this many companies ever being 
impacted?
    Dr. Gottlieb. I, certainly, have no recent memory. I've 
been around the agency for 15 years either as an observer on 
the outside doing policy work or working for three separate 
commissions.
    I've never seen something on this scale where we've had a 
region that had so much important, concentrated manufacturing 
impacted in such a profound way.
    I mean, our priority first and foremost is to the people of 
Puerto Rico, and we are doing a lot to provide them direct 
assistance. But this is an existential risk that we face as a 
nation if these facilities are permanently impacted.
    And I will just state that the facilities themselves are 
intact.
    Mrs. Brooks. Right.
    Dr. Gottlieb. The challenge is going to be the logistics of 
maintaining their operations and moving--getting their workers 
to work, maintaining their operations on what are right now 
generators and in moving product off the island.
    The issue of moving the product off the island is 
improving. The getting the workers to work is starting to get 
better.
    The companies themselves have done a lot to provide direct 
assistance to their employees. They are opening the cafeterias. 
They are offering three meals a day, providing gasoline to 
their employees. I've been on the phone with many of these 
CEOs.
    My biggest long-term concern right now is the power and 
also the secondary supply chain--are they going to be able to 
get supplies from their local suppliers who we are not 
necessarily monitoring as closely and they might not be FDA-
regulated facilities.
    Mrs. Brooks. My time has expired.
    However, I did wonder, since we haven't dealt with this, 
might there be protocols to be put in place in the future in 
case anything like this were to happen, unless there are 
already protocols in place to work with these manufacturers to 
mitigate the shortfalls?
    Dr. Gottlieb. There--there are, and these are hardened 
facilities that have substantial generators--I mean, 800,000 
kilowatt generators on some of these facilities, bigger than 
that.
    I don't think anyone anticipated something on this scale 
where a Category 4 hurricane went through the longitudinal 
access of the island and decimated the entire island.
    Mrs. Brooks. Thank you. I yield back.
    Mr. Burgess. Chair thanks the gentlelady. Gentlelady yields 
back.
    Dr. Gottlieb, if I could, let me just ask you, on the 
guidance that you're going to be providing does it address the 
issue for someone who has been on--someone who's been on a 
critical trial, the drug is not approved, and yet the 
perception of the patient is this is the only thing that helped 
me, and so now that product is not going to be available? Would 
that be available under an expanded use?
    Dr. Gottlieb. Well, I don't--it depends on is it a 
circumstance where the company made a decision not to go 
forward with the further development of product or the company 
is continuing to develop the product and then they are going to 
provide it subsequent to the clinical trial in sort of an open 
label fashion.
    In the latter circumstance, we see a lot of companies doing 
that. In the former circumstance, when companies do curtail 
development of products in clinical trials it's because they've 
deemed them not to work but certain patients felt they are 
deriving a benefit, this doesn't address it.
    I think it would take something that Congress would have to 
do to address that kind of a circumstance.
    Mr. Burgess. Very well. Good to know.
    I do want to thank you for being here and your indulgence 
through the testimony and the questions.
    We are going to transition without a break to our third 
panel, and we are ready to hear from Mr. John Dicken, director 
of health care in the United States Government Accountability 
Office.
    Dr. Dicken, we'll give you a moment to get situated and 
then you'll be recognized for 5 minutes whenever you're ready.

STATEMENT OF JOHN E. DICKEN, DIRECTOR, HEALTH CARE, GOVERNMENT 
                     ACCOUNTABILITY OFFICE

    Mr. Dicken. Great. Thank you, Chairman Burgess, and members 
of the subcommittee.
    I am pleased to be here today to discuss GAO's recent 
report on FDA's expanded access program. As you have been 
hearing this morning, this program allows patients with serious 
or life-threatening ailments and no other comparable medical 
options to obtain access to investigational drugs and 
biologics--that is, those that are not yet approved for FDA 
marketing.
    FDA receives and reviews these expanded access requests and 
determines whether to allow them to proceed. It's also 
important to note that other entities also have roles.
    For example, manufacturers decide whether to give patients 
access to their investigational drugs, Institutional Review 
Boards must approve their investigational access treatment 
plans, and physicians treat the patients with the 
investigational drugs and monitor their progress.
    My testimony today briefly highlights three key findings 
from our July report. First, I will speak about what is known 
about the number, type, and time frames of expanded access 
requests received by FDA; second, what actions FDA and other 
stakeholders have taken to improve expanded access; and third, 
how FDA uses data from expanded access in the drug approval 
process. In addition, I will highlight a recommendation we have 
made to FDA to improve the program.
    First, we found that FDA allowed to proceed nearly all, 99 
percent, of the nearly 5,800 expanded access requests that were 
submitted from fiscal years 2012 through 2015.
    Almost 96 percent of these requests were for single 
patients with more than 2,400 requested on an emergency basis. 
FDA typically responded to these emergency requests within 
hours and responded to all other requests within 30 or fewer 
days.
    In the rare cases when FDA did not allow a request to 
proceed, the most common reasons were incomplete applications, 
unsafe dosing, the treatments demonstrated lack of efficacy, or 
the availability of adequate alternative therapies.
    We also found that FDA and others have taken steps to 
improve patient access through this program. For example, in 
response to concerns that the process to request expanded 
access was cumbersome, FDA simplified its Web site, guidance, 
and forms.
    Efforts by other stakeholders include a project to educate 
and streamline the process by which Institutional Review Boards 
approve treatment plans for expanded access use and the 
creation of an advisory group to help drug manufacturers manage 
expanded access requests.
    Finally, we examined FDA's use of safety reports based on 
the use of drugs allowed through expanded access. Manufacturers 
sponsoring clinical trials included--including any expanded 
access use must submit safety reports to FDA that include 
adverse events data.
    FDA reported using adverse events data from expand use in a 
few cases during the drug approval process but not more widely 
because expand access use does not have the same controls as 
clinical trials.
    For example, FDA data show that there were only two 
instances from 2005 through 2014 in which adverse events from 
expanded access use contributed to FDA delaying a drug's 
development by imposing a clinical hold on the drug's use.
    However, several manufacturers and other stakeholders we 
interviewed raised concerns that FDA is not consistently clear 
about how it uses expanded access adverse events data during 
the drug approval process.
    Our review of documents that FDA uses to communicate with 
drug manufacturers about expand access found that only one 
included a reference to FDA's use of these data.
    Manufacturers know that this lack of clear information can 
influence their decision whether or not to give patients access 
to their drugs.
    Based on this finding, we recommended that FDA should 
clearly communicate how the agency will use adverse events data 
from expand access use when reviewing drugs and biologics for 
approval.
    FDA agreed with our recommendation and I was pleased to 
hear FDA Commissioner Gottlieb announce this morning new 
guidance in response to GAO's recommendation.
    We believe that this additional clarity could help allay 
manufacturers' concerns and help meet the goal of FDA 
facilitating expanded access to drugs for patients with serious 
or life-threatening conditions when appropriate.
    Chairman Burgess and members of the subcommittee, this 
concludes my statement. I will be pleased to respond to any 
questions you may have.
    [The prepared statement of Mr. Dicken follows:]
    [GRAPHICS NOT AVAILABLE IN TIFF FORMAT]
    
    Mr. Burgess. I thank you for your testimony.
    We will move into the question and answer portion of the 
hearing. I would like to recognize Mr. Guthrie from Kentucky, 5 
minutes for his questions, please.
    Mr. Guthrie. Thank you very much. Thank you, Mr. Dicken, 
for being here. Appreciate you being here today.
    And what are some of the ways, like the 14 other 
stakeholders you spoke with--what are the ways they are working 
to improve the expanded access process?
    Mr. Dicken. Right. We heard of several efforts that were 
ongoing. Certainly, some deal with the transparency and 
education about the program.
    There's an effort by the Reagan-Udall Foundation that's 
working with FDA to create a Navigator that will allow more 
information.
    Certainly, the effort by this committee and Congress and 
the 21 Century Act make legislation that requires manufacturers 
to include information about their policy for expanded access 
also adds two kinds of transparency in the information that's 
available.
    There are other efforts that we heard about that deal with 
streamlining the Institutional Review Board process or having 
assistance from manufacturers' pilot program.
    I think a witness in the last panel will speak more about 
efforts to help manufacturers consider and manage these types 
of requests.
    Mr. Guthrie. OK. Thank you. And you got sort of to the 
answer of this next question but I am going to ask it again and 
give you a chance to elaborate at the very end of your--of your 
comments there.
    It says your report found the FDA does not consistently and 
clearly communicate how it uses adverse effects data from 
expanded access used in the drug approval process.
    Can you please summarize how FDA communicates currently, 
which you sort of did, and how GAO recommends they change? If 
you can elaborate again.
    Mr. Dicken. Sure. We--during the course of our work, we 
reviewed a range of materials that FDA provides to communicate 
with manufacturers and others about the expanded access 
program. That includes various guidance documents as well as 
acknowledgments when they are expand access requests.
    Across those multiple documents we found that FDA had 
updated one that provides some general information. They had a 
question and answer that provides some information at a general 
level about how they would use adverse events data.
    But we continued to hear from drug manufacturers' and 
others' concerns that was not as consistent or as clear as 
possible.
    So we were pleased that FDA did agree with the 
recommendation and, certainly, Commissioner Gottlieb's 
testimony in the case that they intend to clarify more 
guidance, going forward.
    Mr. Guthrie. OK. Thank you.
    And your report indicates that FDA allowed 99 percent of 
expanded access requests they received to proceed. Did you look 
at the reasons for why FDA would not allow a request to proceed 
and if so, what did you find?
    Mr. Dicken. Yes. We looked at FDA's data on why. They 
indicated that they did not allow the exceptions--the 1 percent 
that did not proceed.
    The types of issues that FDA indicated were either the FDA 
had identified that there was evidence that was ineffective for 
the incident treatments--either that there might have been 
availability of other treatments including clinical trials that 
individuals may have been able to participate in or also 
incomplete information or safety concerns.
    Mr. Guthrie. Well, I think Dr. Gottlieb mentioned earlier 
when he was here that part of the reason was that there was a 
confidential hold because there were some adverse effects that 
was identified but wasn't public knowledge.
    Did you see that and I guess what I am getting at it 
appeared that unless there was a specific reason that people 
weren't getting approved to go into the right--were getting the 
right to try unless there was some specific adverse effect.
    I think you said--I think you said two things--one, there 
was nonavailability. Two was that there was some confidential--
I forget the term that he used but a hold--that they knew there 
was an adverse effect but they couldn't put that out publicly.
    And so is that what you found and, you know, it seems like 
everybody, unless there's a specific reason not to, are getting 
the chance to try.
    Mr. Dicken. And so I think that's a fair characterization--
that it's only in very isolated incidences that they had 
additional information that may have raised concerns about the 
safety.
    I will note that this is not the only player, that there 
are other decisions including that they need to have approval 
from the manufacturer before proceeding with an expanded access 
request.
    Mr. Guthrie. OK. Thank you.
    That concludes my questions and I yield back.
    Mr. Burgess. Gentleman yields back. Chair thanks the 
gentleman.
    Chair recognizes the gentleman from Texas, Mr. Green, 5 
minutes for questions, please.
    Mr. Green. Thank you, Mr. Chairman, and thank you for being 
here, and looking at the GAO helps us in looking at what we may 
do with this legislation.
    The report found that some manufacturers expressed concerns 
regarding how adverse events associated with expanded access 
would impact the drugs' development or ultimate approval.
    GAO recommended the FDA should clearly communicate how it 
uses adverse events data from expanded use in the drug approval 
process, a recommendation that FDA just a few minutes ago 
agreed with.
    Can you elaborate on this recommendation and how will 
greater clarity from the FDA on the use of adverse event data 
from expanded use improve upon patient access to these 
investigational therapies?
    Mr. Dicken. Yes. This was a concern that as we interviewed 
manufacturers and other participants in the process that 
several raised that this was affecting their decision making 
about agreeing to expanded access requests.
    We also heard, and you have heard testimony today, that 
there have been very rare instances--only two instances--when 
there has been a delay or a clinical hold.
    And so that led to the if there were more clarity as to 
what the circumstances where FDA would consider that 
information that in many cases, because this information does 
not have the same controls that a clinical trial has, I think 
Commissioner said it's often not useful in the drug development 
and approval process.
    But when it is, there was concern and so more clarity that 
it's only in isolated circumstances and that that would be a 
concern the appropriate context seemed important to help allay 
those manufacturers' concerns and hopefully help improve access 
for patients that could get investigational therapies when 
appropriate.
    Mr. Green. OK. Thank you.
    A central component of the 2017 report focused on what's 
known as number type and the time frames of expanded access 
requests received by the FDA.
    The bills we are considering today would take the FDA out 
of the process altogether. Would it be possible to even know 
the universe of expanded access or Right to Try requests made 
absent any FDA involvement and do you think this lack of 
accountability by a company potentially illegitimate claiming 
to have an IND expose patients to bad actors.
    If the FDA is out of the picture, how do we know the 
adverse actors?
    Mr. Dicken. So on the first part of that about the total 
universe, you know, we know the data on how many are reaching 
FDA. We reported on that.
    We did also talk to manufacturers--a subset of nine 
manufacturers--with experience in the process and their 
experiences really varied. There was no consistent data on how 
much requests they are getting.
    But, certainly, they had requests, from dozens to hundreds 
of requests in some cases, for expand access. But there is not 
consistent information across all manufacturers of how often 
they would be getting these requests.
    Certainly, under current authority FDA's key part of 
developing--the drug development approval requires a clinical 
trial and approvals in that process and looking at information 
from other sources including, where appropriate, expanded 
access use.
    Mr. Green. OK. And you mentioned in your testimony the use 
of this data by FDA, while limited, is still a source of 
concern for manufacturers looking to get their products 
approved.
    Could you elaborate on the concerns expressed in your 
interviews with manufacturers regarding FDA's guidance on this 
issue?
    Mr. Dicken. Yes. I think the concerns were that if there is 
uncertainty as to whether or not and how FDA would consider a 
situation where the therapy doesn't work.
    These are terminally ill individuals. There will be 
outcomes that no one wants but that are negative. And so in the 
uncertainty of how FDA would consider that information, that 
led them to have concerns about making some of the approvals.
    That's where we think more clarity on the limited 
circumstances in which FDA does consider this is very important 
and recognition of the context that these are individuals that 
are not in clinical trial settings, that are terminally ill, 
and how--whether or not that is relevant information that FDA 
could--would find useful.
    Mr. Green. Thank you. Thank you, Mr. Chairman.
    Mr. Burgess. Chair thanks the gentleman. Gentleman yields 
back.
    The Chair recognizes the gentleman from Virginia, Mr. 
Griffith, 5 minutes for questions, please.
    Mr. Griffith. Thank you, Mr. Chairman.
    Let me just say I appreciate your report. I appreciate the 
fact that the FDA--Dr. Gottlieb, earlier today, said he's going 
to take a number of those recommendations and they are 
announcing some steps that may improve the process.
    We have already heard from other witnesses that--or from 
other members of Congress who have asked you about the concerns 
of manufacturers and I think you covered that, which is where 
some of my questions were going to go.
    Let me ask a little bit of a follow-up in a slightly 
different direction. Does the FDA also require that safety data 
include the reporting and use of data on patients that 
benefited from the expanded access treatment?
    So previously we've talked about all the concerns about 
manufacturers about the adverse. Does the FDA use the things 
that turned out well and how did manufacturers--if so, and how 
did manufacturers respond to that?
    Mr. Dicken. Right. There are requirements and then this, as 
you've acknowledged, on safety reporting. I think we heard from 
FDA that there were circumstances when they saw other 
information such as dosage or other information that might be 
useful and they can prove it and that there were limited 
instances that's also limited here where the information could 
be used by the manufacturer in supporting its application for 
approval.
    And so in those cases, if the manufacturer is providing 
information to FDA in some limited cases this also helps 
support FDA's decision for approval or labeling or dosing.
    Mr. Griffith. Well, and I do think that it's important that 
FDA consider both because, you know, it may not be the best 
evidence. We might want to have the full clinical trial to get 
the best evidence.
    But when you have somebody who is using this process it is 
at least some evidence of whether it's good or bad or helpful 
or not helpful, and I do appreciate it.
    With that, Mr. Chairman, I yield back.
    Mr. Burgess. Chair thanks the gentleman. The gentleman 
yields back.
    And Mr. Dicken, let me just ask you. You heard Commissioner 
Gottlieb--Dr. Gottlieb and I discussed a little bit and he 
didn't want to steal the testimony that you were--you were 
going to provide.
    It didn't bother me at all. I was perfectly willing to pre-
empt any impact that you might make. But do you feel that the 
answer that I got was that satisfactory? Was that fulsome in 
that response as far as the adverse reporting issue?
    Mr. Dicken. Yes. Certainly, it did not--I was pleased to 
have that discussion happen earlier as well and agree that, you 
know, certainly, the adverse event reporting I think was a fair 
characterization. So yes, thank you.
    Mr. Burgess. And I am going to--again, your preface or your 
premise, as you started out with your report, was that there 
was the perception that the program has been criticized by 
physician and patient advocacy groups for being too burdensome 
and confusing.
    But now as we've worked through this process with the 
guidance that the FDA is going to be providing with perhaps 
some of the legislative products that are out there, do you 
feel like we are generally moving in the correct direction to 
get--to get therapies to patients in a timely fashion that will 
actually impact their clinical course?
    Mr. Dicken. Yes. I think we heard from patients and groups 
and providers and manufacturers that they thought progress was 
being made in improving the expanded access program and, 
certainly, continued to streamline and educate providers, 
individuals, and manufacturers about that.
    We still are hearing, still, during the course of our work, 
that even though FDA has streamlined their application that 
some others, such as Institutional Review Boards occasionally 
may still ask for the more complex information, and there have 
been efforts to kind of educate so that more streamlined 
information can be used not only by FDA but other entities that 
need to approve this expanded access use.
    Mr. Burgess. So, I mean, the Institutional Review Board, 
that's a--that's a good thing. We want that independent look at 
a request for expanded use.
    At the same time, I mean, if someone is not--if someone is 
just out practising in the community and they have a patient 
who has this request, it can be difficult for them, that--the 
IRB itself becomes a barrier, does it not?
    Mr. Dicken. And that is where I think there were some 
efforts to help educate IRBs who may only in some cases 
experience these requests occasionally and so some efforts to 
both educate IRBs to perhaps have some specialized IRBs that 
would have more experience with this process and help minimize 
and streamline that as an obstacle.
    Mr. Burgess. And to even provide some flexibility within 
the IRB structure itself where something needs to happen in a 
more--where time becomes a critical factor. Do I understand 
that correctly?
    Mr. Dicken. That is correct.
    Mr. Burgess. Now, you did not--at least--well, let me just 
see if I can ask this in the right way. It really wasn't your 
function to assess the liability concerns that some 
manufacturers might have. Is that correct?
    Mr. Dicken. That's correct.
    Mr. Burgess. Is that a fair statement? That's why it's not 
really addressed in your report?
    Mr. Dicken. Yes. We did not independently assess that. We 
did ask manufacturers and others about what their concerns were 
and I think you've heard about some of those concerns.
    Others are outlined in our report and those dealt more with 
supply, with concern about any public backlash if they should 
deny it, about risks and potential benefits.
    Mr. Burgess. Yes. I think we are going to hear a little bit 
more about that.
    Well, seeing no other members wishing to ask questions, I 
do want to thank you for your testimony today. Thank you for 
your participation in the--in the hearing.
    We are going to transition to our final panel, again doing 
so without a break in the action. It will take a few minutes 
more because we do have a little bit larger panel now for our 
final panel.
    But I ask our witnesses to take their seats and each 
witness--after you get a chance to get situated each witness 
will have an opportunity to give a statement followed by 
questions from Members.
    And there is no pressure on the technical challenge to get 
the name--and, again, each witness is going to be recognized 
for 5 minutes to give a general statement, and then we'll 
follow that with questions from the Members.
    On our fourth and final panel, we are going to hear from 
Ms. Naomi Lopez Bauman, director of Healthcare Policy at the 
Goldwater Institute; Lieutenant Commander Matthew Bellina, 
United States Navy, patient advocate; Mr. Kenneth Moch, 
president and CEO of Cognition Therapeutics; Dr. Alison 
Bateman-House, assistant professor, Department of Population 
Health, New York University, Langone Health; and Dr. Ellen 
Sigal, chairperson and founder, Friends of Cancer Research.
    We appreciate each of you being here with us today and you 
will each be recognized 5 minutes for an opening statement.
    Ms. Lopez Bauman, we will recognize you for 5 minutes.

   STATEMENTS OF NAOMI LOPEZ BAUMAN, DIRECTOR OF HEALTHCARE 
 POLICY, GOLDWATER INSTITUTE; LCDR MATTHEW BELLINA, U.S. NAVY 
   (RETIRED); KENNETH I. MOCH, PRESIDENT AND CHIEF EXECUTIVE 
 OFFICER, COGNITION THERAPEUTICS, INC.; ALISON BATEMAN-HOUSE, 
    PH.D., ASSISTANT PROFESSOR, DIVISION OF MEDICAL ETHICS, 
 DEPARTMENT OF POPULATION HEALTH, NEW YORK UNIVERSITY LANGONE 
 HEALTH; ELLEN V. SIGAL, PH.D., CHAIR AND FOUNDER, FRIENDS OF 
                        CANCER RESEARCH

                STATEMENT OF NAOMI LOPEZ BAUMAN

    Ms. Lopez Bauman. Chairman Burgess, Ranking Member Green, 
and other members of the committee, thank you for the 
opportunity to address you today.
    My name is Naomi Lopez Bauman and I am the director of 
healthcare policy at the Goldwater Institute. We began our work 
on Right to Try about 5 years ago.
    Doctors and patients approached the institute because dying 
patients were not getting access to the innovative treatments. 
Meanwhile, the wealthy and well-connected could seek innovative 
treatment overseas, leaving most others behind with few 
options.
    Diego Morris, who was diagnosed with osteosarcoma at age 
10, is one of those lucky few. His family relocated to England 
for an entire year so that he could obtain a leading treatment 
that 7 years later has yet to receive U.S. approval.
    It's also considered the standard of care in many countries 
around the world. Diego is now a healthy 17-year-old who is now 
helping to ensure that other patients like him are not left 
behind.
    Something is desperately wrong when terminal patients who 
are out of options are required to stand in line for permission 
to seek an investigational treatment that their doctor is 
recommending and that a manufacturer is willing to make 
available.
    Right to Try is about the terminal patients who don't fit 
into a control group, who can't afford to travel overseas or 
move to another country, and who simply want permission to seek 
the same treatments that other patients, sometimes in the same 
medical facility, are already receiving.
    This inequity occurs despite the fact that one of the 
bedrock principles of medical ethics is patient autonomy. When 
a life hangs in the balance, decisions about healthcare are 
ultimately for the patient to make.
    That is the basis of the State Right to Try laws, and I am 
very happy to report that yesterday the Senate in Pennsylvania 
unanimously passed Right to Try, so now in Pennsylvania it has 
passed both chambers unanimously and we hope will be the 38th 
State that will be a Right to Try State, and we are still 
proceeding in the additional States as well.
    But under these State laws, if you have a terminal 
diagnosis and you have exhausted all other options, you may 
seek, under your doctor's care and direction, investigational 
treatments that have passed Phase 1 of the FDA clinical trials 
and are continuing to undergo FDA evaluation.
    Simply put, this law extends to all terminal patients who 
are dying and out of options the same right to try to save 
one's own life that is already enjoyed by the wealthy and well-
connected and the lucky few that are in the clinical trials.
    At the worst time of his life, Mark Hayutin of California 
was facing terminal cancer and insurmountable odds when he 
became a patient of Dr. Ebrahim Delpassand, a nuclear medicine 
physician who was testing a promising treatment.
    Then the FDA terminated the study that Mark was 
participating in because there was no longer a need for more 
patient data. Mark was left without the ability to complete his 
treatment.
    It is because of the Texas Right to Try law that Mark was 
eventually able to complete the treatments. Today, Mark credits 
Dr. Delpassand and the Texas Right to Try law for saving his 
life.
    The Federal Right to Try legislation under consideration 
today is not a call to ignore research or undermine science or 
for doctors to abandon their obligations to their patients or 
for drug companies to disregard the complex ethical questions 
such as how to distribute limited supplies of drugs.
    And, obviously, Right to Try is not a guarantee that an 
investigational medicine will work or that patients and doctors 
will have perfect information to make these informed decisions. 
And as the FDA admits, no system can ensure against all risks.
    But that isn't the question for us today. The question is 
who should ultimately decide what level of risk is acceptable 
to a dying patient: Federal officials or the patients 
themselves, in consultation with their doctors?
    Thank you for your consideration of Senate Bill 204, the 
Right to Try Act. I yield back to the Chair.
    [The prepared statement of Ms. Lopez Bauman follows:]
    [GRAPHICS NOT AVAILABLE IN TIFF FORMAT]
    
    Mr. Burgess. Chair thanks the gentlelady for yielding back.
    Lieutenant Commander Bellina, you are recognized for 5 
minutes for a statement, please.

               STATEMENT OF LCDR MATTHEW BELLINA

    LCDR Bellina. Ladies and gentlemen, thank you for inviting 
me here to speak today in my--I don't think I am on.
    Is that better? Yes, way better. All right.
    And my diaphragm is failing a bit here so if I get hard to 
hear, Mr. Moch, if you'd give me a poke I will speak up.
    Ladies and gentlemen, thank you for inviting me to speak 
here today. I also want to extend a heartfelt thank you to my 
representative, Congressman Brian Fitzpatrick. He has been a 
tireless advocate not only for the ALS community but for all 
terminally ill Americans.
    In my advocacy work, I have met literally thousands now 
terminally ill people and their families and the vast majority 
ask me how can anybody oppose the Right to Try bill.
    I appreciate that sentiment, but I also do respect the fact 
that they are well-meaning people with ideological differences. 
I would like to illustrate the arguments I've heard and why I 
believe they are based on faulty logic.
    The one that I hear all the time--and it's been thrown 
around a lot today--is, you know, we had this expanded access 
program--we already approved 99 percent, you know, why do we 
need this bill?
    On average, there are less than 2,000 applications per 
year, by conservative estimates. There are nearly 30 million 
Americans living with incurable conditions. I would like to 
draw an analogy.
    Imagine there were 30 million Americans eligible or food 
stamps. Two thousand applied and were approved. The other 
29,998,000 never completed an application, and they starved to 
death.
    Would we be congratulating ourself on that kind of stat? 
That never offends me. The major difference I see is that food 
stamp reform would involve a fiscal note, and this bill 
doesn't. So, in my mind, it's better.
    The FDA's involvement--and, really, Dr. Gottlieb, I think, 
did a great job. Their involvement--they've tried so hard but 
their involvement has a chilling effect on the manufacturers, 
and that is the supply issue that he was talking about.
    The other argument I hear pretty often is that the State 
Right to Try bills have had little impact, so why should we 
pursue a Federal bill? The hundred or so case patients in Texas 
that you mentioned would have a very different opinion.
    But let's assume for argument's sake that that hundred 
people is not enough for us to make an effort here today. I 
think the big issue is the courts and their broad 
interpretation of the interstate commerce clause.
    Most pharmaceutical companies are trying to sell a drug in 
more than one State. So, you know, we need a Federal law to 
protect them in that case.
    I am sympathetic. You're going to hear from Mr. Moch here 
in a minute and other pharmaceutical executives. I know this 
makes their job harder when, you know, you have patient 
communities and social media calling them out--you know, why 
aren't you giving the drug to this person or that?
    I would say the issue is that, you know, they have to have 
the courage and tell the community what they think is right and 
wrong, and sometimes the answer is no. And I do appreciate 
that, but we can't let the FDA be the bad guy.
    And I will sum up by saying I know it's probably too late 
for me. I made my peace with that.
    I need to know before I die that if my children find 
themselves in this unenviable position, this Nation that I 
proudly serve will respect their liberties and the right to 
make their own decision about their medical treatments.
    Thank you for having me. God bless.
    [The prepared statement of LCDR Bellina follows:]
    [GRAPHICS NOT AVAILABLE IN TIFF FORMAT]
    
    Mr. Burgess. We thank the gentleman for his service and 
thank him for his testimony.
    Mr. Moch, you are recognized for 5 minutes, please.

                  STATEMENT OF KENNETH I. MOCH

    Mr. Moch. That's a tough start for me.
    Good afternoon, Chairman Burgess, Ranking Member Green, and 
members of the subcommittee.
    My name is Kenneth Moch and I am the president and CEO of 
Cognition Therapeutics, a company developing what we hope is a 
new medicine for Alzheimer's disease.
    Over the course of my career, I have been the CEO or co-
founder of five biotechnology companies focussed on developing 
new medicines for terminal or life-threatening diseases 
including serving as CEO of an antiviral therapeutics company 
called Chimerix.
    Starting in late 2009, Chimerix provided its experimental 
antiviral called brincidofovir under expanded access to 430 
critically ill individuals.
    This was one of the largest expanded access programs 
undertaken by a biotech company, at its peak accounting for an 
estimated 6 percent of the expanded access requests to the 
entire FDA and an estimated 30 percent of the requests to the 
antiviral drug division.
    The FDA was never a hindrance to granting these requests 
and the FDA staff we dealt with including the division director 
were extraordinary in their help and their compassion and their 
clear understanding of the critical needs of the patients.
    Right to Try legislation would not have changed anything 
that we did during this multiyear program. At the end of 2012, 
we made the difficult decision to cease the expanded access 
program and focus on the pathway to FDA approval.
    Fifteen months later in March of 2014, the family of a 
critically ill 7-year-old boy named Josh Hardy started a social 
media campaign access to brincidofovir.
    The high profile #SaveJosh campaign catalysed international 
debate on issues of ethics and equity in expanded access and 
raised questions regarding the role of patient advocacy and 
social media and, in many ways, led to the ongoing discussion 
today about Right to Try.
    Let me state clearly that I am an advocate of expanded 
access--what I prefer to call preapproval access--when it is 
appropriate for the medicine under development.
    The testimony today has been heartfelt, truly heartfelt, 
and I believe that everybody in this room, if we had a family 
member who was critically ill--a child, a parent, a sibling--or 
if we were critically ill ourselves would do everything in our 
power to gain access to an experimental medicine that might 
increase the chance of survival.
    That being said, expanded access programs raise social, 
ethical, and moral conflicts and dilemmas regarding access to 
experimental medicines.
    How does society or a company balance the immediate needs 
of a critically ill individual, in many cases a child, versus 
the potential needs of many future patients?
    Who is advocating for those future patients who might not 
receive a needed medicine because FDA approval is delayed by 
even a week or a month?
    And I am not talking about the FDA delaying the approval 
process--the review process--but rather, what might happen if 
because of an unexpected finding our outcome some percentage of 
potential participants choose not to enroll in a clinical 
trial, slowing down the development time line?
    Being very granular, what would have happened to the 
brincidofovir clinical development program and even to Chimerix 
if, after a global social media campaign, Josh Hardy had 
received brincidofovir and shortly thereafter died?
    We live in a world of social media, and while the FDA might 
not react to patient--to the--might not react, the patient 
community likely would have.
    In other words, you can't look at Right to Try legislation 
without looking at all of the implications and applications of 
this law.
    At the time of the #SaveJosh campaign, I characterized this 
ethical dilemma as not being about Josh but about the many 
future Joshes. This question is the challenge that faces each 
of you as you discuss and think about Right to Try legislation.
    Let me also say that I am not a supporter of Right to Try 
legislation. In my opinion, this legislation does nothing to 
help patients in need.
    I believe there are things that need to be done but Right 
to Try is not in any way addressing the complexities of drug 
development.
    And given that the FDA only considers expanded access 
requests when it is received by the drug sponsor and approves 
over 99 percent of these requests, the decision to grant 
expanded access requests fall to the leadership of the company 
developing the new medicine, not the FDA.
    It is crucial to understand the extraordinary complexity of 
developing new medicines as well as the fragility of the 
biotechnology companies that are the predominant sources of 
these innovations.
    No ethical company that I know of would ever release an 
experimental medicine outside the FDA's regulatory process. A 
basic mantra is that all drugs have side effects and cutting 
scientific corners creates unbounded risks.
    There is simply no monolithic answer to the question of 
when circumstances and timing are right to undertake an 
expanded access program because each experimental medicine is 
different, the safety and efficacy parameters are different, 
the clinical development processes and regulatory pathways are 
different, and the patient populations in need are different.
    Expanded access is not drug development, and Right to Try 
is not drug development, and given this fact, it is not 
unreasonable for a company to decide not to initiate an 
expanded access program until there is sufficient data 
demonstrating the efficacy and safety of an experimental 
medicine.
    In closing, I believe that Right to Try legislation as 
currently crafted is not the answer to any of the questions 
that have been raised about providing experimental medicines to 
critical or terminally ill patients.
    Bypassing the FDA is not in anyone's interest and no 
ethical company I know would do so. At the best, Right to Try 
will not help people and at the worst, I believe, it could do 
harm.
    I thank you for your time.
    [The prepared statement of Mr. Moch follows:]
    [GRAPHICS NOT AVAILABLE IN TIFF FORMAT]
    
    Mr. Burgess. And we thank you for your testimony.
    Dr. Bateman-House, you are recognized for 5 minutes, 
please.

               STATEMENT OF ALISON BATEMAN-HOUSE

    Dr. Bateman-House. Mr. Chairman, Ranking Member Green, and 
the members of the Health Subcommittee, I am Dr. Alison 
Bateman-House, an assistant professor of medical ethics at NYU 
Langone Health.
    Thank you, first, for having this hearing and also for the 
opportunity to be here with you today. It's wonderful to see so 
many people engaged in trying to help patients who find 
themselves in exceedingly dire straits.
    I co-chair a working group on compassionate use and 
preapproval access. This group is composed of patient 
advocates, members of the pharmaceutical industry, individuals 
with clinical trial and compassionate use experience, 
bioethicists, lawyers, venture capitalists, and individuals 
with both experience at the FDA or the Reagan-Udall Foundation 
for the FDA.
    This working group was formed before the Right to Try 
movement began and there was no litmus test of any sort on 
Right to Try or any other topic for members to pass to be 
invited to the group. And yet, every member of our group 
opposes Right to Try on ethical, legal, and pragmatic grounds.
    The working group was founded in the aftermath of Josh 
Hardy's quest to gain access to brincidofovir that Mr. Moch 
just spoke of. That case and others made public headlines and 
indicated that there was dissatisfaction with the existing 
system for accessing investigational medicines outside of 
clinical trials.
    So our task was a specific mission--to study access to 
investigational drugs outside of clinical trials from the 
vantage point of all stakeholders to identify what problems 
existed and to propose solutions.
    We have identified many concerns with the current system 
and we have proposed several ways to address these concerns. I 
will review some of these briefly. But before I go any further, 
I want to make two points very clear.
    First, after more than 3 years of studying all facets of 
compassionate use or preapproval access, including the right to 
try, the working group has found that the FDA's expanded access 
program has been doing an excellent job in helping patients 
obtain access to experimental drugs.
    Earlier today, we heard Representative Fitzpatrick say that 
Right to Try would ``prevent the Government from blocking 
access to potentially lifesaving treatments.''
    This is a solution for a problem that does not exist. We 
have heard repeatedly today that the Government is not the 
barrier to people getting access.
    The second point I want to drive home is that no piece of 
Right to Try legislation either on the State or Federal level 
addresses the myriad issues the working group has identified in 
this space.
    So what issues have we found? First, as we've heard today, 
there's a widespread lack of knowledge about the expanded 
access program.
    My working group has tried to address this dearth of 
knowledge by hosting webinars, publishing and speaking 
extensively and partnering with patient organizations for 
events like Ask an Expert sessions.
    But, obviously, our small volunteer group is unable to fill 
a national educational gap.
    So we have told the FDA that it needs to step up and to 
make sure that there's more understanding in this process. But 
this responsibility for increased education cannot rest solely 
on the FDA.
    Doctors and nurses organizations, pharmaceutical trade 
associations, and all sorts need to step up and be involved.
    Another especially troubling issue is that of rampant, 
inaccurate, even mythological beliefs. Some patients believe 
the FDA can force companies to give access to drugs. This is 
not true.
     Another widespread myth is that the FDA is slow in 
handling requests. This is not true. Another myth is that the 
United States somehow has an incredibly small number of 
patients being served.
    We don't know if this is true or not. When people say that 
less than 2,000 requests have been approved, those are 
protocols. We don't know how many patients are in those 
protocols.
    We know about half the protocols are single patients, so 
just one, but the others could be anywhere from hundreds to 
thousands of patients. We don't know.
    And the last myth that I have heard is that, you know, 
focussing on legal liability prosecution is necessary--that 
somehow we need to protect companies from legal risk. This is 
also a myth.
    And because these myths are persistent, widespread, and may 
well be leading companies, doctors, or hospitals to turn down 
patient requests, they have to be dealt with.
    So these among others are some of the problems that the 
working group has identified and you will note that I have not 
identified Right to Try much in what I've said because none of 
these issues are dealt with in any of the Right to Try laws.
    I will quote a recent letter from 22 patient organizations 
that say, quote, ``Our organizations support patient access to 
unapproved therapies.''
    But S. 204 and H.R. 878 do not effectuate policy changes 
that would afford our patients greater access to promising 
investigational therapies. Instead, these bills would likely do 
more harm than good.
    In closing, I want to point out one way that Right to Try 
laws have already caused harm, and that is by taking what was 
already a confusing situation and making it even more 
confusing.
    We now have 37 State laws--it's not one coherent law, they 
are each individually different--plus a potential for a Federal 
law.
    You know, especially when patients cross State lines to 
seek health care or when you have hospital or insurance 
organizations that span State lines, such complexity is the 
enemy of patients.
    I thank you for your time, and I look forward to your 
questions and I yield back to the Chair.
    [The prepared statement of Dr. Bateman-House follows:]
    [GRAPHICS NOT AVAILABLE IN TIFF FORMAT]
    
    Mr. Burgess. Thank you for testimony.
    Dr. Sigal, you're recognized for 5 minutes for a statement, 
please.

                  STATEMENT OF ELLEN V. SIGAL

    Dr. Sigal. Chairman Burgess, Ranking Member Green, I am 
honored to be here today, and members of the committee.
    I am Ellen Sigal, chair of Friends of Cancer Research, a 
group--a nonprofit that is committed to innovation, 
accelerating better treatments for patients that are safe and 
effective.
    I founded Friends over 20 years ago, driven by the profound 
loss of my dear sister, Gale. After many years battling cancer, 
Gale had exhausted every option.
    As metastatic breast cancer raged through her body, 
defeating all conventional treatments she found, she faced a 
final decision--succumb to the disease or wage one last battle 
with an experimental bone marrow transplant known to kill 20 
percent of patients.
    Gale chose to fight. In Gale's case, the side effects of 
the treatment were swift and violent. Within two days, at the 
age of 40 she was dead, leaving her 4-year-old daughter and 
husband behind.
    All of us here today agree on the basic premise--more must 
be done to save patients' lives. We must continue to ensure our 
regulatory system is expediting therapies as safely and quickly 
as possible.
    Friends of Cancer Research took huge steps towards this 
beginning 5 years ago, when we worked with many on this 
committee to create the breakthrough therapies designation. It 
has been incredibly successful.
    This is progress, but I will acknowledge much more needs to 
be done. In addition, a predominant reason why patients seek 
expanded access to experimental therapies in the first place is 
that they are unable to attain them by enrolling in clinical 
trials.
    By expanding eligibility criteria and taking down barriers 
that oftentimes disqualify a patient from participating in a 
trial to begin with, we can make additional progress.
    Legislation before Congress seeks to grant all terminally 
ill patients the right to try experimental therapies once 
approved alternatives have failed, even though the FDA 
authorizes 99 percent of compassionate use requests.
    Serious changes to today's legislative proposal are needed 
before this law is safe for patients. First, provisions for 
informed consent are essential.
    A significant majority of early-phase drugs are dangerous 
and ultimately prove ineffective with upwards of 90 percent 
never being brought to the market.
    Any legislation that goes forward cannot circumvent the FDA 
and must be carefully crafted to assure that we do not create a 
loophole for those seeking to profit off the sick by offering 
false hope. This is reprehensible.
    Second, the limits of Right to Try must be clear. Even if 
patients receive the right to request an experimental therapy, 
the drug company developing the therapy is under no obligation 
to provide it.
    Patients petitioning for expanded access deserve accurate 
information about whether the potential benefits outweigh the 
risks. This is highly personal calculus. It's impossible if 
drug companies do not monitor and report side effects.
    A key component is transparency. Patients have long been 
frustrated that they could not find information about expanded 
access on sponsor Web site and didn't know how to make a 
request for the sponsor.
    The Reagan-Udall Foundation, which I am honored to chair, 
recently launched an expanded access Navigator for 
compassionate use of experimental therapies.
    The Navigator is currently being piloted in oncology with 
the goal of increasing accessibility to information for 
patients and providers.
    We have already--we already have three dozen companies that 
contribute their information and had 10,000 visitors to the 
site. In the very near future, this program will expand to 
include rare diseases.
    While I fully believe that dying patients should have 
access to promising treatments, we must not subject patients to 
false hope or unacceptable side effects. With significant 
adjustments, Federal Right to Try legislation could help very 
sick patients.
    One of these adjustments is that patients must have more 
and immediate access to information about significant adverse 
events or death of patients that have previously been given the 
therapy.
    Another adjustment would be the establishment of the 
designated central Institutional Review Board with the 
predominant focus of coordinating and dealing with expanded 
access requests.
    The current legislative proposals would likely do more harm 
than good. I encourage the committee to consider other policy 
options that would truly improve the ability for patients to 
safely access unapproved therapies.
    Thank you very much.
    [The prepared statement of Dr. Sigal follows:]
    [GRAPHICS NOT AVAILABLE IN TIFF FORMAT]
    
    Mr. Burgess. I want to thank each of you for your 
testimony. It's been a very powerful panel. We will begin the 
Member question portion by recognizing the gentleman from 
Kentucky for 5 minutes for his questions.
    Mr. Guthrie. Thank you, Mr. Chairman.
    Lieutenant Commander, thank you for your service.
    LCDR Bellina. Thank you.
    Mr. Guthrie. We appreciate what you do and appreciate your 
service. I am one that's--we need to figure out how to handle 
expanded access.
    I think, Dr. Sigal, you summed it up. We need to do it 
right, do it correct, and give people opportunities to make 
informed decisions within what we have to sort out and try to 
figure out so we don't do more harm than good.
    I think, as you mentioned, so we need to do it right, and 
that's why I think this hearing has been important and your 
willingness to testify has really been helpful and we 
appreciate that.
    One thing that--that when you start getting into this that 
some of the unknown, not just in the FDA or all the other 
issues is just other things and, one, I understand that hospice 
services are provided once you've exhausted all options, and 
then after you've exhausted all options you get hospice 
services for care and comfort until the end of life--kind of 
end-of-life care.
    And my understanding is it could jeopardize hospice 
services if you go into an experimental treatment. And so the 
question I get to, I think--in Dr. Bateman-House's testimony, 
you said that 19 State patients receiving expanded access drugs 
lose their hospice coverage and six States say these patients 
may be denied coverage for home health assistance.
    So my question--I think you talked about it too, Ms. Lopez 
Bauman--if both of you would talk about this, and two 
questions.
    How would a Federal Right to Try Act impact access to 
hospice services, and the second, how do States who have passed 
this legislation balance access to hospice services with a 
right to try? Do you want to start first?
    Ms. Lopez Bauman. Thank you for your question--and the 
Right to Try Act, Senate Bill 204, was amended prior to the 
Senate vote in order to address and accommodate a lot of these 
concerns and such as specifying how adverse event data can be 
used, requiring reporting to the FDA, capping allowable charges 
to direct costs only, and limiting manufacturer reliability.
    I can tell you that that is--that in the insurance area, 
end-of-terminal and end-of-life and hospice benefits and those 
things, those are terms under the insurance--under the State 
insurance laws and regulations.
    And so, even if you might technically not be eligible for 
hospice because you are continuing to seek treatment, it 
doesn't mean that that patient will not get that treatment.
    I would also like to point out that these laws have 
undergone 4 years of addressing these kinds of stakeholder 
concerns and in the 37 States and counting where it is now law, 
and the message from across the country is really loud and 
clear that terminal patients shouldn't have to beg the Federal 
Government for permission to pursue these options.
    And I would also like to point out something. We've heard a 
lot about opposition to Right to Try because of--based on false 
hopes.
    But I would like to point out that we've heard a lot today 
about how the FDA is addressing the adverse event issue.
    I very quickly took a look at the guidance that they issued 
today, and it's really important to point out--and I direct you 
to questions 25 and 26 in the guidance--where they are 
actually--they are changing basically the standard for 
reporting adverse events. But they are--they are not addressing 
how they are going to actually deal with those adverse events.
    So after a GAO report, after years of patients talking to 
Congress and the FDA about the need for actually clarifying how 
the FDA will address adverse events, it's still not done, not 
even today, even though we've heard about it all morning.
    Mr. Guthrie. OK. I only have just a little bit amount of 
time. So I want to give Dr. Bateman-House a chance to answer 
the question about access to hospice services.
    Dr. Bateman-House. Right. So in addition to potentially 
losing access to hospice services under some of these State 
bills, you could lose, as you mentioned, access to home health 
care.
     You also, under a few of the bills, could actually lose 
access to health insurance for six months post, you know, this 
treatment that you get through Right to Try.
    Mr. Guthrie. Are States trying to address--then fix, as Dr. 
Lopez Bauman kind of----
    Dr. Bateman-House. Well, so as----
    Mr. Guthrie. Once that's been passed, they look at it and 
they say, oh, unintended consequence and we try to fix----
    Dr. Bateman-House. I always say, unfortunately, we are 
working in a data-free environment. Despite our best efforts as 
a group, we have only found two doctors who admit to giving 
treatments to patients under Right to Try. They're both in the 
State of Texas. Texas does not have a reporting requirement.
    We have no idea what happened with those individual 
patients. One of the States that does have a reporting 
requirement, Oregon, we contacted them to try to find out, you 
know, the experience of the patients, and did they lose access 
to anything, et cetera.
    They had no record of anyone being treated under Right to 
Try. California also has a reporting requirement, but the law 
has only been in effect for about a year or so.
    Mr. Guthrie. And we've got about 30 seconds. I know your 
opposition to the bills before us. But is there a protocol or, 
as Dr. Sigal kind of suggested--is it ``Seegal'' or ``Segall''? 
``Seegal''?
    Dr. Sigal. ``Seegal.'' Whichever.
    Mr. Guthrie. ``Seegal''--Dr. Sigal. I had a professor named 
Dr. ``Segall,'' spelled the same way. That can be put in place 
so this could be--this could work? Or is this just something 
you don't think can work at all?
    Dr. Bateman-House. Well, the thing that I don't think has 
been said today is that it's not supplanting the FDA. What it's 
doing is it's doing an alternative pathway.
    So if you want to go through expanded access you still can. 
If you want to go through Right to Try, you can't--I mean, you 
can also do that if the Federal bill were to pass.
    Honestly, as Ken Moch said, I have said all along I don't 
think any reputable company will give access to drugs this way. 
So I really think it's a moot issue.
    Mr. Guthrie. Well, thank you. My time has expired so I 
yield back.
    Mr. Burgess. Chair thanks the gentleman. Gentleman yields 
back.
    The Chair recognizes the gentleman from Texas, Mr. Green, 5 
minutes for questions.
    Mr. Green. Thank you, Mr. Chairman, and having served 20 
years in the Texas legislature and had a number of issues when 
I was serving there, whether it be cancer treatment with peach 
pits--laetrile--DMSO, who had--and States have an ability to do 
that, whereas on the Federal level we have an FDA since 1906 
and so it may be easier for States to say, well, you have the 
right to try.
    And, basically, I agree with that. If I was terminally ill 
or needed--I would want that. But I also know we have this 
agency that has tried to protect us for over a hundred years, 
and to do it.
    Dr. Sigal, I know that you've spent considerable time and 
effort on working with researchers and sponsors to help enroll 
patients in clinical trials.
    I represent a district in Houston and we have some great 
clinical trials whether it be at MD Anderson, Methodist 
Hospital, any of ours. In fact, our chairman actually went to 
medical school in Houston.
    But I am greatly concerned that the Right to Try 
legislation would confuse families and patients on what role 
the FDA plays and how they can access the FDA, and let me give 
you an example.
    A couple of years ago when we had the Ebola scare, I was 
concerned that there was something on a lab table that would 
treat these patients, and I checked with them and I was told 
that they did that and the FDA gave 24 hours' notice that they 
could give that.
    These patients were U.S. citizens. They were doctors. They 
were cognizant of what they were doing, and the sad part is we 
don't know whether that medication helped them or not because, 
you know, it wasn't a trial. It didn't have a comparison.
    So but what would the impact on increasing access to 
investigational drugs through Right to Try legislation outside 
the clinical trials have on clinical trial enrollment, and do 
you believe it would endanger or delay clinical trial 
enrollment?
    Dr. Sigal. The answer is yes. The clinical trial system is 
not perfect but it is the gold standard and we do need to work 
on exclusionary criteria on it.
    However, if patients think they can circumvent it and get 
this drug off a clinical trial through Right to Try, clearly, 
they are going to try to do it.
    Unfortunately, there will be probably no company--reputable 
company that will allow their drug to be used that way. But I 
do think we can do a lot about clinical trials and we can do a 
lot more in informed consent.
    But in fact the clinical trial system is the best we have. 
We need to have more patients enrolled in it. We know that. We 
need to look carefully at exclusionary criteria, and also we 
are doing a lot about innovation.
    FDA now is--we are working on lung cancer master protocols. 
There are single arm trials. There's seamless drug development. 
There's a lot going on in this field to expedite drug 
development so patients can have the benefit of these 
treatments earlier because that's what we want.
    Mr. Green. OK. And another concern I have is that 
pediatrics--we also have the great hospital, Texas Children's, 
and those facilities all over the country, and just because, 
you know, children are different than adults and we need to 
have trials with children, and I know Congress over the years 
has encouraged that. Would that also impact pediatric clinical 
trials?
    Dr. Sigal. The answer is yes. We need more of them. We know 
we need more. Twenty-first Century Cures just have really 
important provisions to expedite that and to really handle with 
drug development on it.
    But, again, the same issue--if people think that they can 
access a drug through Right to Try, they are going to 
circumvent the clinical trial process and then we won't know 
the data.
    We won't know exactly what happened on it and, again, the 
ability for the patients to access these trials is--or this 
Right to Try is going to be highly limited and really very 
worrisome.
    Mr. Green. OK. Thank you, Mr. Chairman. I yield back.
    Mr. Burgess. Chair thanks the gentleman. Gentleman yields 
back.
    Chair recognizes the gentleman from Virginia, Mr. Griffith, 
5 minutes for questions, please.
    Mr. Griffith. Thank you. Thank you very much. I have to say 
I don't think that patients who are dying are going to be 
confused, particularly if we say this has not yet been approved 
by the FDA. Would you agree with that, Ms. Lopez Bauman?
    Ms. Lopez Bauman. Thank you for your question. Yes. The 
Right to Try Act, Senate Bill 204, actually works in tandem 
with the current updated process and that is why Right to Try 
is only available to patients who have exhausted approved 
treatments, who are unable to participate in the clinical 
trial, and why Right to Try only applies to medicines that are 
already being considered by the FDA and are continuing to be 
evaluated by the FDA, and I would like----
    Mr. Griffith. So by the time a patient has gotten to that 
point, they are fairly well educated on the issues, at least 
related to their condition and disease?
    Ms. Lopez Bauman. I think that's true, but I think that the 
words of Dr. Razelle Kurzrock--who at one point ran one of the 
Nation's largest clinical trials, actually, at MD Anderson--
explained that the process was so burdensome that they only 
submitted one application per year.
    This was a clinical trial of more than 1,000 patients, and 
to quote Dr. Kurzrock, that there were so many barriers that 
even at one of the best places in the world and one of the 
largest apartments that this, as their day-in and day-out job, 
it was still very challenging.
    Mr. Griffith. And I appreciate that.
    I also think that it's important that we do have informed 
consent. Both House bills have that, and so any language that 
you might want to provide to make that stronger for us I would 
greatly appreciate that, Dr. Sigal.
    But I would appreciation any language that you could 
provide. Unfortunately, time is of the essence so I can't get 
that language right now later if you could provide us with some 
opportunities.
    Lieutenant Commander, again, thank you for your service. 
You mentioned that you hoped that it would be different when 
your children were grown up. How old are your kids?
    LCDR Bellina. All right. I have a 6-year-old, a 4-year-old, 
and a 7-month-old at home--three boys.
    Mr. Griffith. Well, I know that's got to be a great joy for 
you.
    LCDR Bellina. It is, and I do want to also throw out there, 
and I hope everybody hears this: There's this notion floating 
around that expanded access isn't getting used because people 
don't know or can't figure it out.
    I find that deeply offensive. I would say the ALS patients 
I know are bright, well-informed. A lot of them know more than 
the researchers and the doctors they work with, and the idea 
that they wouldn't apply because they can't figure it out, I 
don't even know what to say.
    Mr. Griffith. Yes, I appreciate that.
    Back to you, Ms. Lopez Bauman. What legal protections do 
patients in the 37 States that have passed Right to Try laws 
have that patients in other States do not? And we are running 
out of time, so if you could keep it as quick as possible.
    Ms. Lopez Bauman. So, really, what it comes down to is that 
in the States where Right to Try is now law, it's about 
allowing terminal patients more freedom to access the right 
treatment at the right time.
    And it's not a guarantee but it is an assertion that 
patients have a right to medical autonomy and that bureaucratic 
and administrative barriers shouldn't be standing in the way.
    And I'd like to point out that earlier this year, this very 
own legislative body implicitly endorsed the right to try for 
terminal patients to seek investigational treatments to save 
their own lives.
    Remember little Charlie Gard, who was granted residency to 
seek an investigational treatment here in the United States. He 
was granted residency after the U.K. blocked his parents' right 
to seek an investigational treatment.
    And so this has already been implicitly endorsed by your 
legislative body. We have the vehicle and it has been vetted 
and stakeholder concerns have been addressed. It's time to act.
    Mr. Griffith. I am going to open this up for anybody to 
send me a response afterwards. But I do want the lieutenant 
commander to respond to this.
    In your article--I believe it's your article--in the 
Washington Post you indicated that in 2014 nearly 25,000 people 
in France were using investigative treatments through the 
French government's equivalent program, and yet we had less 
than 2,000--I think that was your reference earlier--to the 
food stamp program.
    LCDR Bellina. That is correct.
    Mr. Griffith. And what are the differences in the French 
program that allow them to get access to them, even though they 
have a much smaller population than we do, that we don't have?
    LCDR Bellina. Well, I think Dr. Gottlieb was 100 percent 
correct that it's a supply issue. It's not a demand issue, and 
I think their legislation allows for the demand--the market to 
drive this supply is what we see there and we don't have that 
here. I think this bill is a big step in addressing that.
    Mr. Griffith. I appreciate that.
    My time is up, but if anybody else would like to give me a 
written response to that of what they see as either pros or 
cons with the French law versus the American law I would 
appreciate it.
    And with that, Mr. Chairman, I yield back.
    Mr. Burgess. Chair thanks the gentleman. Gentleman yields 
back.
    Dr. Sigal, let me ask you--and thank you for bringing the 
case of your sister to us. It was very powerful.
    I was actually in practice in the 1990s, so I remember that 
controversy very well, not with your sister but with the high-
dose chemotherapy and rescue with stem cell transfer for 
metastatic breast cancer.
    And it was quite controversial, and there was a sense--
perhaps relating to what Mr. Moch encountered--there was a 
sense that, hey, here's something that will work when nothing 
else will, but it's expensive and so therefore it's denied.
    Can you tell us what has now happened with the therapy that 
your sister received? Is that still a viable clinical pathway 
for patients to follow?
    Dr. Sigal. Well, the answer is no. I mean, at the time she 
had metastatic disease. There were no options. But she did go 
into that knowing that there was a 20 percent fatality. This 
was the decision that she made and, of course, she died from 
it.
    Later on when we did do clinical trials we realized that 
that therapy was not effective. But because patients refusing 
to go into clinical trials at the time it took us a much longer 
time.
    I mean, today our system is swifter. We are, at the FDA, 
approving drugs in single arms, some with 10 and 15 patients. 
When we have the breakthrough mechanism and when we see really 
good evidence early, it's all hands on deck and they are 
getting to market earlier.
    We are the fastest in the world. We published at Friends 5 
years ago EMA versus FDA, because we were told that we were 
slower than Europe, and we were shocked.
    We went back and did the study ourselves and were shocked 
that we were faster, and when we told the FDA they said nobody 
will believe you--you have to publish this in a peer review 
journal, and we did. And, in fact, the importance is not 
faster, but the issue is better and gold standard.
    So we all understand the burden of disease and particularly 
for dying patients who have no risks. So the ability to get 
them on trials, to look at exclusionary criteria, and to look 
at treatments that work.
    But most importantly, patients really need to have 
information--informed consent. They may decide they want to 
take the risk.
    But they can't make that decision with their doctor unless 
they have the data, and if they don't have the data in Phase 1, 
where it is really only safety and no efficacy, and if that's 
not available to them, what decision that's informed will be 
made by that patient and the physician? You need data, and then 
it is up to the patient if they want to participate.
    Mr. Burgess. Well, let me ask a question then, Ms. Lopez 
Bauman. The case that you reference--Diego, with the 
osteosarcoma who's now 17 and was diagnosed when he was age 10, 
and you said that medicine is still not available in the United 
States, is that correct?
    Ms. Lopez Bauman. That's correct. If I recall correctly, it 
has passed Phase 3 and this summer there was an FDA advisory 
council that voted against approval. Of course, the ultimate 
approval has not been made by the FDA.
    But I think it is really important to point out that the 
current process only serves less than one half of 1 percent of 
terminal patients in this country and it is only the well-
connected and the affluent that are able to go to other 
countries to get this kind of treatment.
    And Right to Try is about making this available to everyone 
to at least pursue. I mean, obviously, there are no guarantees.
    I would also like to point out something about safety. 
We've been talking about safety quite a bit, and Dr. Gottlieb 
explained how he was treated off-label for his own cancer, and 
I'd like to just explain to the committee that doctors can 
prescribe FDA-approved treatments for off-label uses, where 
medicines that are used to treat conditions other than what the 
FDA says it is approved for.
    So what that means is that these are--these are prescribed 
and this is completely legal and lawful and it is actually very 
common, particularly in areas where there's a very serious 
disease, where there's--without proof of efficacy.
    And this--and this is done very frequently. About one-fifth 
of all off-label prescriptions are--about one-fifth of all 
prescriptions are written off label and in cases where--in 
cases where there aren't a lot of options, particularly the 
more serious types of cancer, it is the majority of the time.
    And so this idea that we can't allow doctors with their 
patients to make decisions about what might be an appropriate 
treatment and, really, just run roughshod over patient autonomy 
is, I think, is really the wrong----
    Mr. Burgess. I am just going to interrupt you for a second 
because, again, I am getting such a completely different story, 
Dr. Sigal, and your story on Diego's osteosarcoma medication. 
Is there a question of the efficacy of the medication and 
that's why it hasn't been approved?
    Ms. Lopez Bauman. Well, I mean, there certainly isn't in 
other countries. It's available in Mexico, Israel, all over 
Europe. He went to the U.K. and in, I believe, 2014 it actually 
won the----
    Mr. Burgess. But let me--I am going to stop you for a 
second. There really wouldn't be an off-label option for Diego.
    Ms. Lopez Bauman. Not in his case. But what I am saying is 
that this idea of talking about risk or that you can't 
prescribe something without knowing the efficacy is actually 
not true.
    In our current system, it is perfectly legal for a 
physician to prescribe off-label and it is actually very 
common.
    Mr. Burgess. Sure. Very common.
    Ms. Lopez Bauman. And so Right to Try, I don't believe, 
poses, you know, additional risks and burdens on the doctors 
and the patients in terms of a lack of information or not 
having perfect information, because we are already using off-
label treatments in a lot of different areas of health care, 
and, in fact, one-fifth of all prescriptions are off-label.
    So this idea that you have to have efficacy or you have to 
have more data before you can give a patient permission to use 
it is, I think, absolutely unacceptable and that the default 
should be that patients should have the right to try to save 
their own lives.
    Mr. Burgess. And I don't disagree. But, again, as Dr. Sigal 
so eloquently pointed out in her sister's case, that perhaps 
was premature to be utilizing that type of therapy.
    And I agree, we are much better now. Put the United States 
breast cancer statistics up against anyone in the world. That 
is truly one of the bright spots, as far as developmental 
therapeutics is concerned.
    Mr. Moch, I just have to ask you, sort of the last--the 
last tier about the issue of the legal liability, and what is--
you're the one who served as--I guess on the board or the CEO 
of an actual company that had to deal with this.
    So that's a real concern for a company, is it not, that 
someone will come back after the fact and say, ``I was harmed 
by your product''?
    Mr. Moch. So I've actually been CEO of five companies 
involved in this space.
    The answer is no. I think that's not the argument, and it 
is not one of the reasons that I've looked at for not making an 
experimental medicine available, and I've done it in multiple 
companies.
    You have informed consent. There are going to be side 
effects. I think everybody knows in these cases these are 
terminally ill patients.
    Again, I think--and I just--I have no other way to say 
this. As being right between this debate, I cautioned everybody 
that you're looking at a specific issue in a vacuum.
    I see--the plural of anecdote is not data. I can pull out 
lots of examples of people who survived or died or have been 
problems, and you're looking at a particular case that's a 
statement that's made to make a point.
    You have to look at the totality. That's not being done in 
this discussion in a way that really is--for me, as a drug 
developer in five companies--is frustrating, I will be clear.
    There are lots of reasons that people will make or will not 
make a drug available under expanded access. The Right to Try 
laws address none of those reasons, and I think that is--in my 
original statement, I said this is feel-good legislation for 
legislators, and I am sorry to say it so bluntly.
    The percentage of legislators who voted for Right to Try 
legislation is about equal to the percentage of FDA approvals 
of expanded access applications. It's not a relevant 
comparison, but it is a very relevant comparison.
    The problem here is that drug development is very complex. 
You do now know, in most cases--in fact, you do not know the 
safety of a drug after Phase 1. I have taken drugs through 
Phase 3 and had them fail for safety issues in Phase 3.
    Hundreds of millions of dollars are spent. You think you 
know the answer, and 40 or 50 percent of drugs still fail in 
Phase 3.
    The drug development process where you're trying to alter a 
biological system that's evolved over how many hundreds of 
millions of years, and you're trying to alter one system in a 
human being.
    Doesn't happen that way. You get side effects. You get 
issues. You don't know what the number is or percentage is.
    So the argument that Right to Try legislation is going to 
make more people have access to experimental medicines does not 
exist in my mind as a drug developer nor in anybody I know, and 
I can't say it more bluntly than that.
    I know it is a very emotional thing. I know we all want--
look, I've done more expanded access than most drug developers 
with a biotechnology company.
    I want to see it happen. This doesn't do anything. If you 
want to talk about at some point how to do things that are 
helpful, then you've got to get a group of people in a room and 
have a meaningful discussion.
    This discussion really doesn't address those issues.
    Mr. Burgess. Well, I actually look forward to having that 
discussion. So you have set the stage for perhaps our second 
hearing in this regard.
    But this has been fascinating today and, clearly, we 
haven't heard the end--this is not the end of the story. But 
very powerful panel, and I thank you all for spending time with 
us today.
    I don't see any other Members who have not yet asked 
questions. So, again, I will thank you for being here today. We 
have received outside feedback from a number of organizations 
on these bills.
    So I'd like to submit statements from the following, for 
the record: Right to Try, the National Conference of State 
Legislatures as well as a letter from our Senate colleagues--
Senator Johnson and Senator Donnelly.
    Without objection, so ordered.
    [The information appears at the conclusion of the hearing.]
    Mr. Burgess. Pursuant to committee rules, I remind Members 
they have 10 business days to submit additional questions for 
the record, and I ask the witnesses to submit their response 
within 10 business days upon receipt of the questions.
    And without objection, the subcommittee is adjourned.
    [Whereupon, at 1:34 p.m., the subcommittee was adjourned.]
    [Material submitted for inclusion in the record follows:]

                 Prepared statement of Hon. Greg Walden

    Today, our Health Subcommittee is reviewing an important 
and often emotional topic--patient access to experimental drugs 
for our family members, friends, and other loved ones battling 
serious or immediately life-threatening illnesses.
    Having lost close family members in such circumstances, I 
understand the passion people have for finding a life-saving 
cure.
    Previous barriers to investigational drugs led to a 
nationwide movement, resulting in the establishment of the Food 
and Drug Administration's expanded access program. Commonly 
referred to as compassionate use, this 1980s-era process has 
helped patients who do not meet the guidelines of clinical 
trial participation receive experimental access to unapproved 
drugs. The individual's physician and the drug developer must 
agree the potential benefits outweigh any patient safety 
concerns with the FDA facilitating the process. According to 
the nonpartisan Government Accountability Office, 99 percent of 
the nearly 5,800 applications were approved from 2012 through 
2015.
    However, there is ongoing concern that some patients may 
not be allowed to access investigational drugs even after 
exhausting all other treatment options. We must examine whether 
there are regulatory, legal, or commercial barriers to patient 
access for experimental drugs.
    This has led some advocates to promote a nationwide 
grassroots movement for State Right to Try laws. To date, 37 
States have enacted such laws, according to the National 
Conference of State Legislatures, including my home State of 
Oregon. Many patients, like Navy veteran Matt Bellina, here 
with us today, have moving stories to tell. And they deserve a 
voice in this complex discussion.
    Ultimately, this issue is about fairness. As a 
representative body, our responsibility is to strike the 
delicate balance of individual liberty, and patient safety in 
public policy.
    For many on this committee, today marks your first formal 
exposure to patient access to investigational drugs. Today is 
an opportunity for all of us to learn more.
    Thank you, Mr. Chairman, for holding this hearing. And 
thank you to our four panels of witnesses, I appreciate you 
taking the time to be with us today. I yield the balance of my 
time.

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