Congressional Record, Volume 164 Issue 49 (Wednesday, March 21, 2018)

[Congressional Record Volume 164, Number 49 (Wednesday, March 21, 2018)]
[House]
[Pages H1738-H1748]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]

TRICKETT WENDLER, FRANK MONGIELLO, JORDAN McLINN, AND MATTHEW BELLINA
RIGHT TO TRY ACT OF 2018

Mr. BURGESS. Mr. Speaker, pursuant to House Resolution 787, I call up
the bill (H.R. 5247) to authorize the use of eligible investigational
drugs by eligible patients who have been diagnosed with a stage of a
disease or condition in which there is reasonable likelihood that death
will occur within a matter of months, or with another eligible illness,
and for other purposes, and ask for its immediate consideration in the
House.
The Clerk read the title of the bill.
The SPEAKER pro tempore. Pursuant to House Resolution 787, the bill
is considered read.
The text of the bill is as follows:

H.R. 5247

Be it enacted by the Senate and House of Representatives of
the United States of America in Congress assembled,

SECTION 1. SHORT TITLE.

This Act may be cited as the ``Trickett Wendler, Frank
Mongiello, Jordan McLinn, and Matthew Bellina Right to Try
Act of 2018''.

SEC. 2. USE OF UNAPPROVED INVESTIGATIONAL DRUGS BY PATIENTS
DIAGNOSED WITH A TERMINAL ILLNESS.

(a) In General.--Subchapter E of chapter V of the Federal
Food, Drug, and Cosmetic Act (21 U.S.C. 360bbb et seq.) is
amended by inserting after section 561A (21 U.S.C. 360bbb-0)
the following:

``SEC. 561B. INVESTIGATIONAL DRUGS FOR USE BY ELIGIBLE
PATIENTS.

``(a) Definitions.--For purposes of this section:
``(1) The term `eligible patient' means a patient--
``(A) who has been diagnosed with an eligible illness;
``(B) who has exhausted approved treatment options and is
not eligible to participate in (for a reason such as the
patient not meeting inclusion criteria) a clinical trial
designed to evaluate an investigational drug for the
treatment of such eligible illness with which the patient has
been diagnosed, including one involving the eligible
investigational drug, or for whom participation in such a
clinical trial is not feasible (for a reason such as a lack
of geographic proximity to the clinical trial), as certified
by a physician, who--
``(i) is in good standing with the physician's licensing
organization or board; and
``(ii) will not be compensated for so certifying; and
``(C) who has provided to the treating physician written
informed consent, as described in part 50 of title 21, Code
of Federal Regulations (or any successor regulations),
regarding the eligible investigational drug, or, as
applicable, on whose behalf a legally authorized
representative of the patient has provided such consent.
``(2) The term `eligible investigational drug' means an
investigational drug (as such term is used in section 561)--
``(A) for which a phase 1 clinical trial has been
completed;
``(B) that has not been approved or licensed for any use
under section 505 of this Act or section 351 of the Public
Health Service Act;
``(C)(i) for which an application has been filed under
section 505(b) of this Act or section 351(a) of the Public
Health Service Act, as applicable, that is active; or
``(ii) that is under investigation in a clinical trial
that--
``(I) is intended to form the primary basis of a claim of
effectiveness in support of approval or licensure under
section 505 of this Act or section 351 of the Public Health
Service Act; and
``(II) is the subject of an active investigational new drug
application under section 505(i) of this Act or section
351(a)(3) of the Public Health Service Act, as applicable;
and
``(D) the active development or production of which--
``(i) is ongoing;
``(ii) has not been discontinued by the manufacturer; and
``(iii) is not the subject of a clinical hold under the
regulations implementing section 505(i) or section 351(a)(3)
of the Public Health Service Act, as applicable.
``(3) The term `phase 1 trial' means a phase 1 clinical
investigation of a drug as described in section 312.21 of
title 21, Code of Federal Regulations (or any successor
regulations).
``(4) The term `eligible illness' means--
``(A) a stage of a disease or condition in which there is
reasonable likelihood that death will occur within a matter
of months; or
``(B) a disease or condition that would result in
significant irreversible morbidity that is likely to lead to
severely premature death.
``(b) Alternative Pathway for Eligible Patients With a
Terminal Illness.--
``(1) In general.--Eligible investigational drugs provided
to eligible patients in compliance with this section are
exempt from sections 502(f), 503(b)(4), and subsections (a)
and (i) of section 505 of this Act, and section 351(a) of the
Public Health Service Act so long as the conditions specified
in paragraphs (2), (3), and (4) are met with respect to the
provision of such investigational drugs.
``(2) Compliance with certain regulations.--The conditions
specified in this paragraph, with respect to an eligible
investigational drug referred to in paragraph (1), are that--
``(A) the eligible investigational drug is labeled in
accordance with section 312.6 of title 21, Code of Federal
Regulations (or any successor regulations); and
``(B) the provision of such eligible investigational drug
occurs in compliance with the applicable requirements set
forth in sections 312.7 and 312.8(d)(1) of title 21, Code of
Federal Regulations (or any successor regulations) that apply
to investigational drugs, subject to paragraph (5).
``(3) Notification.--The condition specified in this
paragraph, with respect to an eligible investigational drug
referred to in paragraph (1), is that the sponsor of such
eligible investigational drug notifies the Secretary of the
provision of such eligible investigational drug for use by an
eligible patient pursuant to this section. Such notification
shall be submitted within 7 business days of the provision of
such eligible investigational drug

[[Page H1739]]

as correspondence to the investigational new drug application
described in subsection (a)(2).
``(4) Adverse event reporting.--The condition specified in
this paragraph, with respect to an eligible investigational
drug referred to in paragraph (1), is that the sponsor or
manufacturer of such eligible investigational drug has
required, as a condition of providing the drug to a physician
for use by an eligible patient pursuant to this section, that
such physician will immediately report to such sponsor or
manufacturer any serious adverse events, as such term is
defined in section 312.32 of title 21, Code of Federal
Regulations (or any successor regulations), associated with
the use of the eligible investigational drug by the eligible
patient.
``(5) Application.--For purposes of this section, the
requirements set forth in sections 312.7 and 312.8(d)(1) of
title 21 of the Code of Federal Regulations (or any successor
regulations) are deemed to apply to any person who
manufactures, distributes, prescribes, dispenses, introduces
or delivers for introduction into interstate commerce, or
provides to an eligible patient an eligible investigational
drug pursuant to this section.
``(c) Use of Clinical Outcomes.--
``(1) In general.--Notwithstanding any other provision of
this Act, the Public Health Service Act, or any other
provision of Federal law, the Secretary may not use a
clinical outcome associated with the use of an eligible
investigational drug pursuant to this section to delay or
adversely affect the review or approval of such drug under
section 505 of this Act or section 351 of the Public Health
Service Act unless--
``(A) the Secretary makes a determination, in accordance
with paragraph (2), that use of such clinical outcome is
critical to determining the safety of the eligible
investigational drug; or
``(B) the sponsor requests use of such outcomes.
``(2) Limitation.--If the Secretary makes a determination
under paragraph (1)(A), the Secretary shall provide written
notice of such determination to the sponsor, including a
public health justification for such determination, and such
notice shall be made part of the administrative record. Such
determination shall not be delegated below the director of
the agency center that is charged with the premarket review
of the eligible investigational drug.
``(d) Reporting.--The manufacturer or sponsor of an
eligible investigational drug that provides an eligible
investigational drug pursuant to this section shall post on
the same publicly available internet website used by the
manufacturer for purposes of section 561A(b) an annual
summary of any provision by the manufacturer or sponsor of an
eligible investigational drug under this section. The summary
shall include the number of requests received, the number of
requests granted, the number of patients treated, the
therapeutic area of the drug made available, and any known or
suspected serious adverse events, as such term is defined in
section 312.32 of title 21, Code of Federal Regulations (or
any successor regulations), associated with the use of the
eligible investigational drug.
``(e) Rule of Construction.--Nothing in this section shall
be construed as limiting the authority of the Secretary to
require manufacturers or sponsors of investigational drugs to
review and report information relevant to the safety of such
investigational drug obtained or otherwise received by the
sponsor pursuant to part 312 of title 21, Code of Federal
Regulations (or successor regulations).''.
(b) No Liability.--Section 561B of the Federal Food, Drug,
and Cosmetic Act, as added by subsection (a), is amended by
adding at the end the following:
``(f) Liability.--
``(1) Alleged acts or omissions.--
``(A) Manufacturer or sponsor.--No manufacturer or sponsor
(or their agent or representative) of an investigational drug
shall be liable for any alleged act or omission related to
the provision of such drug to a single patient or small group
of patients for treatment use in accordance with subsection
(b) or (c) of section 561 or the provision of an eligible
investigational drug to an eligible patient in accordance
with this section, including, with respect to the provision
of an investigational drug under section 561 or an eligible
investigational drug under this section, the reporting of
safety information, from clinical trials or any other source,
as required by section 312.32 of title 21, Code of Federal
Regulations (or any successor regulations).
``(B) Physician, clinical investigator, or hospital.--
``(i) No licensed physician, clinical investigator, or
hospital shall be liable for any alleged act or omission
related to the provision of an investigational drug to a
single patient or small group of patients for treatment use
in accordance with subsection (b) or (c) of section 561, as
described in clause (ii), or the provision of an eligible
investigational drug to an eligible patient in accordance
with this section, unless such act or omission constitutes on
the part of such physician, clinical investigator, or
hospital with respect to such investigational drug or
eligible investigational drug--

``(I) willful or criminal misconduct;
``(II) reckless misconduct;
``(III) gross negligence relative to the applicable
standard of care and practice with respect to the
administration or dispensing of such investigational drug; or
``(IV) an intentional tort under applicable State law.

``(ii) The requirements described in this clause are the
requirements under subsection (b) or (c) of section 561,
including--

``(I) the reporting of safety information, from clinical
trials or any other source, as required by section 312.32 of
title 21, Code of Federal Regulations (or any successor
regulations);
``(II) ensuring that the informed consent requirements of
part 50 of title 21, Code of the Federal Regulations (or any
successor regulations) are met; and
``(III) ensuring that review by an institutional review
board is obtained in a manner consistent with the
requirements of part 56 of title 21, Code of the Federal
Regulations (or any successor regulations).

``(2) Determination not to provide drug.--No manufacturer,
sponsor, licensed physician, clinical investigator, or
hospital shall be liable for determining not to provide
access to an investigational drug under this section or for
discontinuing any such access that it initially determined to
provide.
``(3) Limitation.--
``(A) In general.--Except as set forth in paragraphs (1)
and (2), nothing in this section shall be construed to modify
or otherwise affect the right of any person to bring a
private action against a manufacturer or sponsor (or their
agent or representative), physician, clinical investigator,
hospital, prescriber, dispenser, or other entity under any
State or Federal product liability, tort, consumer
protection, or warranty law.
``(B) Federal government.--Nothing in this section shall be
construed to modify or otherwise affect the authority of the
Federal Government to bring suit under any Federal law.''.

The SPEAKER pro tempore. The gentleman from Texas (Mr. Burgess) and
the gentleman from New Jersey (Mr. Pallone) each will control 30
minutes.
The Chair recognizes the gentleman from Texas.

General Leave

Mr. BURGESS. Mr. Speaker, I ask unanimous consent that all Members
may have 5 legislative days in which to revise and extend their remarks
and to insert extraneous material on H.R. 5247.
The SPEAKER pro tempore. Is there objection to the request of the
gentleman from Texas?
There was no objection.
Mr. BURGESS. Mr. Speaker, I yield myself such time as I may consume.
Mr. Speaker, earlier this year, Members of Congress heard the
President during his State of the Union Address make a specific promise
to the American people that the passage of right-to-try legislation
would occur. This afternoon, I am proud to stand with the President and
the thousands of Americans with terminal illnesses, their families, and
their friends, in passing this important bill in the House.
Since 2014, nearly three out of four States, including my home State
of Texas, have passed a version of right-to-try laws. I am pleased that
the House is again considering H.R. 5247, the Trickett Wendler, Frank
Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2018,
so that terminally ill patients have a chance, or maybe a second
chance, at life. These patients are our constituents. They could be
someone we know. Let us take this opportunity to improve access to
experimental treatments for them.
Over the course of the past decade, our Nation has achieved an
unprecedented number of innovations and scientific breakthroughs.
Through the contributions of researchers in academia and the private
sector, Americans have more innovative treatments at their fingertips.
Despite these achievements, I still hear from patients with serious,
life-threatening conditions, including my own constituents in north
Texas, who are frustrated with what they see as regulatory barriers
from trying new therapies when everything else has failed.
Mr. Speaker, as a physician, I understand that access to
investigational drugs and therapies is a deeply personal priority for
those seeking treatment for their loved ones with serious, life-
threatening conditions.
To my friends on the other side of the aisle, I have a simple
question: Why do you not want to allow these patients to exercise their
right to fight for their future?
It is worth mentioning that the bill before us today is a revised,
more narrowly crafted version of the one that passed the Senate last
August. Since that time, the Energy and Commerce Subcommittee on Health
held a hearing in early October to consider the Senate bill, where
Members heard from

[[Page H1740]]

the Commissioner of the Food and Drug Administration, Dr. Scott
Gottlieb, about the agency's concerns. We also heard testimony from
patients and groups that support and oppose right to try.
From then to just recently, our committee engaged in multistakeholder
efforts to improve the original right-to-try bill, as passed by the
Senate. It entailed numerous conversations with patients, advocates,
the Administration, authors of the bill, and stakeholders on all sides
of this complex topic.
The Food and Drug Administration was never left out of the
discussion. In fact, the agency provided valuable input throughout the
process and up until the introduction of H.R. 5247. The aim was to open
the door to innovative, experimental drugs for terminally ill patients
without necessarily compromising the vital work and the mission of the
Food and Drug Administration.
The current compassionate use program at the Food and Drug
Administration does make a good faith effort to help patients who do
not qualify for clinical trials. But right to try would actually offer
patients an alternative pathway to access eligible investigational
drugs, so long as they are certified by a physician who is in good
standing and abides by the rules laid out in the bill.
Again, we have worked closely with the Food and Drug Administration
to ensure that this new, alternative pathway does not hinder or
conflict with the critically important oversight that the agency
conducts.
Additionally, this bill protects patients from manufacturers
mislabeling or misbranding drugs, requires sponsors and manufacturers
to report adverse events to the Food and Drug Administration, and
provides certain liability protections for parties participating in the
new pathway.
Mr. Speaker, this alternative pathway would also be limited to
individuals who are suffering from a disease or a condition where there
is a reasonable likelihood of death within a matter of months or
significant, reversible morbidity, and who have exhausted all FDA-
approved treatment options.
Lastly, it is essential that we do not create additional hurdles in
this process so that manufacturers in the drug approval process have
the certainty that they need.
The revised right-to-try bill clearly states that the Secretary of
the Department of Health and Human Services ``may not use a clinical
outcome associated with the use of an eligible investigational drug . .
. to delay or adversely affect the review or approval of such drug. . .
. `'
After months of work and thoughtful discussions, this legislation is
a positive step forward in our shared goal of improving care for
America's patients. It strikes the proper balance between ensuring
patient safety and granting access to new treatments.
The President outlined in his State of the Union Address that this
was an important priority for the administration. In the words of our
Vice President and former colleague, Mike Pence: ``It's about restoring
hope and giving patients with life-threatening diseases a fighting
chance.''
Mr. Speaker, for these reasons, I urge my colleagues in the House to
vote in support of H.R. 5247, and I reserve the balance of my time.
Mr. PALLONE. Mr. Speaker, I yield myself such time as I may consume.

Mr. Speaker, I rise today to voice my strong opposition to H.R. 5247,
the Right to Try Act of 2018.
This legislation, introduced only last week, is an egregious attempt,
in my opinion, by the Goldwater Institute to undermine the gold
standard drug approval process at the Food and Drug Administration.
The supporters of this bill claim to be helping desperate patients
who are looking for hope.
If this is such a patient-centered bill, then why does every major
patient organization overwhelmingly oppose it?
More than 100 patient organizations, including the National
Organization for Rare Disorders, the Friends of Cancer Research, and
the American Cancer Society have all written in opposition to this
legislation.
In a letter to congressional leadership, these 103 patient
organizations noted ``that the alternative pathway in the latest
version of this legislation is still less safe for our patients than
the current expanded access process under the FDA.''
It is not only the patient organizations that are voicing concerns.
Four former FDA Commissioners--Drs. Hamburg and Califf, who served
under the Obama administration; and Drs. McClellan and Andrew von
Eschenbach, who served under the Bush administration--also oppose this
legislation. That is two former Republican Commissioners and two former
Democratic Commissioners who are opposed to both the House bill and the
Senate bill on this same issue.
These four Commissioners explained their opposition by saying:
``There is no evidence that either bill would meaningfully improve
access for patients, but both would remove the FDA from the process and
create a dangerous precedent that would erode protections for
vulnerable patients.''
Mr. Speaker, I think most importantly, I would stress that this
legislation is simply not needed. There is already a successful program
in place today at the FDA in which seriously ill patients and their
doctors can request access to an experimental treatment from a
manufacturer. This application process, which takes as little as 45
minutes for a physician to complete, has been overwhelmingly
successful.
Last summer, a review by the Government Accountability Office found
that the FDA approves 99 percent of the requests submitted to the
agency. In fact, of the nearly 1,700 requests the FDA received last
year, only 9 were not approved.
Physicians and patients also receive approval quickly. Emergency
requests are often granted immediately over the phone and, on average,
receive a response within 4 days.
While the FDA approves 99 percent of the treatments it reviews
through this expanded access process, as it is called, it also adjusts
applications for 11 percent of the patients to improve patient safety
protections.
In order to protect patients, this review, in my opinion, should
continue. We must protect patients from bad actors or from dangerous
treatments that might make their lives worse. Just imagine the health
consequences to patients if these 11 percent of applicants had not been
adjusted.
This is the very reason that the FDA must be involved in the process.
If you eliminate FDA review, as this bill does, you are putting
patients at risk.
I want to talk a little bit about the fact that many States now have
right-to-try statutes. I fear that some Members--and I heard this last
week when the bill was on the suspension list--might support this
legislation under the false belief that the State right-to-try laws in
their States have provided help to patients. But nothing could be
further from the truth.
One example supporters of this legislation like to bring up is Dr.
Delpassand from Texas, who claims to have treated patients under the
State right to try.
Mr. Speaker, I include in the Record a letter from Mr. Andrew
McFadyen of The Isaac Foundation, who dispels this myth.

The Isaac Foundation,

March 20, 2018.
Rep. Greg Walden, Chair,
Rep. Frank Pallone, Ranking Member,
Energy & Commerce Committee.
Dear Mr. Pallone and Mr. Walden: I am writing to you
regarding your upcoming debate on HR 5247, the Right to Try
initiative fronted by the Goldwater Institute. I am the
Executive Director of The Isaac Foundation, an organization
that is dedicated to providing advocacy and support to
patients dealing with a wide range of disorders and needing
access to rare disease treatments. Our work pushes
international boundaries, with the bulk of our efforts taking
place in Canada and the United States. I am also a member of
the NYU Working Group on Compassionate Use and Pre-Approval
Access where we are making a concerted effort to improve and
address the issues around access to experimental medications,
and I'm involved with a non-profit called GE2P2.
I'm proud to say at The Isaac Foundation that we've never
been unsuccessful gaining access to life-saving medications
and treatments for patients in Canada, and our work directly
with pharmaceutical companies is helping countless patients
see similar results in the United States. We have had success
by being collaborative partners with industry, regulatory
authorities, and patients in need.
I watched the discussion last week with growing
consternation that many of our elected officials have not
taken the opportunity to fact-check claims being made by RTT
proponents. Most notably, continued mention of Right to Try
being used by Dr.

[[Page H1741]]

Delpassand out of Texas is both egregiously wrong and,
indeed, is the perfect example of why RTT should not be
passed by lawmakers.
In October 2016, I testified during Senator Ron Johnson's
hearing on Right to Try, at which Johnson introduced and
played a video created by the Goldwater Institute of Dr.
Delpassand. During that 3-minute video, Dr. Delpassand
explained that he was using the state RTT law to treat his
patients because the FDA would not allow him to do it through
an Expanded Access Program. Senator Johnson asked me what I
thought about this video--which included few facts, no
context, and was edited by the people fronting the RTT push
themselves. I explained that there must be a reason why Dr.
Delpassand was in the 1% of cases not allowed by the FDA and
vowed I would investigate.
In March of 2017, I received a set of documents from the
FDA under a FOIA request. They show that Dr. Delpassand's
clinic failed inspections during the clinical trial of
Lutathera (lutetium Lu 177 dotatate). Specifically, he failed
inspection due to 3 key and very important reasons:
1. Enrolling subjects into the study during a partial
clinical hold, issued by the Agency.
2. Underreporting of Adverse Events.
3. 1572-protocol noncompliance.
The failed inspections were discovered after complaint from
the CDER Good Clinical Practice Compliance Oversight Branch,
Division of Good Clinical Practice Compliance Evaluation,
Office of Scientific Investigations (OSI). A ``Clinical
Hold'' was placed on the lab and Dr. Delpassand. During a
clinical hold, subjects may not be given an investigational
drug. Dr. Delpassand and his clinic disregarded this clinical
hold and enrolled 6 patients.
Additionally, and just as concerning in terms of patient
safety, Dr. Delpassand's clinic failed to promptly report
significant new adverse events or risks to the FDA. This
failure to report was noted numerous times during the
inspection. The inspection also found numerous other areas of
concern. I have attached the full report for your
consideration.
After these inspections, the FDA would not allow Dr.
Delpassand to open an EAP at his clinic for patients in need,
and rightly so. They FDA did, however, allow 42 different
locations the ability to provide this drug for patients
requiring access, including two sites in Texas. A quick
search on ClinicialTrials.gov shows this information, further
proving that the FDA has been able to provide patients the
required access they need, ensuring the environment that they
are receiving the drug they need is safe.
My understanding of the situation is that the company
running the clinical trial distanced themselves from Dr.
Delpassand after these failed inspections. Without company
support, and without the FDA's permission to open an EAP, Dr.
Delpassand had to use the state legislation to provide drug
to his patients. Questions remain, however, such as how Dr.
Delpassand paid for the product he was giving his patients,
did patients themselves have to pay for that drug supply
(which isn't allowed under the Texas RTT law) and who, if
anyone, was overseeing the program to ensure safety of the
patients, especially after multiple infractions were seen
during the failed FDA inspection.
Most important, it should be noted that the FDA process
here worked exactly how it is supposed to. A lab was
inspected for safety to ensure patients are looked after in
the appropriate fashion. That inspection placed a hold on
further treating of patients due to numerous infractions. The
FDA worked with the company to ensure access for patients
across the USA in 42 different sites, helping to monitor
adverse events while also allowing the product to advance to
approval. That product was approved by the FDA in January
2018.
Also importantly, RTT was used because it was the only way
for Dr. Delpassand to treat patients in his clinic after it
failed inspection. RTT is a loophole designed to allow people
who cannot otherwise follow safety rules set forth by the FDA
that are meant to protect vulnerable patients. It's not being
used--anywhere--to provide patients with hope or access to
life-saving drugs.
One final note, and one that I've not see mentioned
anywhere. HR 5247 includes the name of a young child--a brave
child battling Duchenne Muscular Dystrophy--named Jordan
McLinn. Jordan has been photographed numerous times with Vice
President Pence, and is often used as an example of why Right
to Try is needed. The problem with these optics is that
Jordan has never received any treatment under Right to Try,
even though Right to Try has been available in his state of
Indiana for 3 years. He already has access to the life-saving
treatment he needs--through an FDA approved clinical trial.
He's doing well on that trial drug, as I understand it, and
receives all the benefits of FDA oversite to ensure his
safety on that trial. In essence, the child used to promote
RTT is the perfect example of why the FDA process works and
is needed.
The true reality is that the landscape for access to
medications for dying patients does not change tomorrow if a
Federal Right to Try law is passed today. Very clearly, those
patients in dire need of help today will wake up tomorrow
needing access to the same life-saving treatments, and feel
the same despair because they will not be getting the access
they need through Right to Try.
The barrier to that access here isn't the FDA, and no Right
to Try law enacted by lawmakers in this country is going to
remove the true barrier--pharmaceutical companies. The
gatekeepers to these medications are the pharmaceutical
companies themselves, and we need to be working
collaboratively as a team--Industry, Government, physicians,
and Patients--to craft solutions that will work for everyone,
keeping in mind that we are all on the same side, that we all
want the same thing--broad and expeditious access to life-
saving medications for patients in need.
I understand how difficult this is for patients--I see it
every day, and I feel it every night as I check in on my son
(who is battling his own devastating and very rare disease)
to make sure he is still breathing, to make sure he is still
with us. But I also understand that the change we all need
will not come with Right to Try. It will come through
collaboration with all stakeholders and by providing
companies the safety and assurances they need to make their
medications available to our dying patients.
Lawmakers should be spending their time helping make that
collaboration happen because that is how we are going to save
our dying patients. They should not spin their wheels passing
legislation like Right to Try that looks good, and feels
good, but will do nothing for those in need. If they do, they
are doing a disservice to a large and very vulnerable group
of patients now and in the future, my own son, my own hero
Isaac, included.
Thank you for your time on this matter.
Sincerely,
Andrew McFadyen,
Executive Director, The Isaac Foundation.

Mr. PALLONE. Andrew McFadyen said:

Dr. Delpassand claims to have used right to try because FDA
would not allow him to do expanded access. And this was for a
very good reason. FDA placed a clinical hold on a study, due
to the fact that his clinic was not reporting serious,
adverse events, as required; and he continued to enroll
patients, despite the clinic hold.
The work of Dr. Delpassand's study was associated with 40
deaths and 2 hospitalizations. FDA's clinical hold on Dr.
Delpassand's work is a sign to me that FDA's expanded access
pathway was working to prevent bad actors from continuing to
expose vulnerable patients to experimental treatments.

Mr. Speaker, H.R. 5247 is dangerous for our patients. It is an
unprecedented attempt to roll back the FDA's oversight of
investigational treatments. I urge my colleagues to stand with more
than 100 organizations that have come forward to oppose this misguided
and, I believe, harmful legislation.
Mr. Speaker, I reserve the balance of my time.
Mr. BURGESS. Mr. Speaker, I yield 3 minutes to the gentleman from
Pennsylvania (Mr. Fitzpatrick), one of the authors of the bill.
Mr. FITZPATRICK. Mr. Speaker, I want to thank Chairman Walden, Mr.
Burgess, Mr. Griffith, and my friends Andy Biggs and Senator Johnson
for their unflinching commitment to see right to try debated, passed,
and signed into law.
Moreover, I thank the overwhelming bipartisan majority of the House,
who, just last week, supported the Trickett Wendler, Frank Mongiello,
Jordan McLinn, and Matthew Bellina Right to Try Act, and proved
emphatically that right to try is about more than politics. It is about
hope.
Each year, thousands of Americans receive a life-altering diagnosis
of a terminal illness. Even with the amazing work done in American
medical research and development, for too many families, access to
these potentially lifesaving treatments will come too late or not at
all.

{time} 1315

As their Representatives, we should each endeavor to support these
individuals in their time of need, as well as support new pathways to
potentially lifesaving treatment. That is what right to try is all
about.
For those patients caught between traditional drug approval delays, a
clinical trial process for which they do not qualify and limited time,
right to try 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.
Although the FDA has a program that allows terminal patients to apply
for early access to a promising treatment, right to try is needed
because the FDA compassionate use process doesn't help enough people.
Moreover, the application process is complicated, time consuming, and
expensive. Only about 1,200 people each year can make it through the
application process.
In 2014, more than 12,000 people in France were using investigational

[[Page H1742]]

treatments through that government's equivalent program. If a country
with one-fifth the population of the United States can help 900 percent
more people then the FDA's plan, clearly, is not working.
In Australia, doctors are allowed to work directly with drug and
device manufacturers to provide investigational treatments to terminal
patients without the government's approval. They simply must report to
the government at some point that the patient received the drug. No
permission slip is required.
This bill requires robust informed consent between the patient,
doctor, and manufacturer, while requiring notification be given to the
FDA after an unapproved drug becomes available to an eligible patient
and requires doctors and manufacturers to report adverse events to the
FDA.
Mr. Speaker, when a life hangs in the balance, the Federal Government
should not stand in the way of access to these potential treatments. I
have traveled our district in all corners and have met so many
different people--L.J. Kidon and Claire Concilio, most recently. Mr.
Speaker, these people are an inspiration for this bill. They want to
see this bill passed. Let's get this done for them.
Mr. PALLONE. Mr. Speaker, I yield such time as she may consume to the
gentlewoman from Illinois (Ms. Schakowsky), who is the ranking member
for the Digital Commerce and Consumer Protection Subcommittee.
Ms. SCHAKOWSKY. Mr. Speaker, I thank the gentleman for his generosity
and time.
I want to say, Mr. Speaker, that I rise in opposition to H.R. 5247
because it creates a dangerous back door for modern-day snake oil
salesmen, a back door around the FDA approval process for people who
may or may not be preying on desperate people, and it ignores that
there actually is a safe pathway for terminally ill patients to get
treatment.
This bill failed to pass last week and it should fail again. It is a
harmful policy that both Republican-and Democratic-appointed former FDA
Commissioners concluded there is ``no evidence'' that this bill ``would
meaningfully improve access for patients, but would just remove the FDA
from the approval process and create a dangerous precedent that would
erode protections for vulnerable patients,'' the most vulnerable
patients. People whose lives are in danger feel that they will try
anything, and there are people out there who will prey on that.
This bill denies patients what they really need, which is safe and
effective treatments. This bill strips away important safeguards in the
name of helping patients, but it does not, and that is why 78 patient
groups and doctors, groups like the American Cancer Society, the Cystic
Fibrosis Foundation, and the Leukemia & Lymphoma Society, oppose this
bill. In total, there are now 110 groups opposing this bill.
Mr. Speaker, I include in the Congressional Record a multipage list
of opponents to this bill.

Groups Opposed to Right To Try Legislation

ADNP Kids Research Foundation; AIDS Action Baltimore;
Alliance for Aging Research; Alliance for Regenerative
Medicine; American Academy of Neurology; American Association
of Justice; American Cancer Society Cancer Action Network;
American Lung Association; American Society of Clinical
Oncology; American Syringomyelia and Chiari Alliance Project;
Amyloidosis Support Groups; Association for Creatine
Deficiencies; Benign Essential Blepharospasm Research
Foundation; Biomarin; Bonnie J. Addario Lung Cancer
Foundation; Breast Cancer Action; Bridge the Gap--SYNGAP
Education and Research Foundation CancerCare; Cancer
Prevention and Treatment Fund; Charlotte and Gwenyth Gray
Foundation to Cure Batten Disease.
Children's Cause for Cancer Advocacy; Children's
Cardiomyopathy Foundation; Congenital Hyperinsulinism
International; CurePSP; Cutaneous Lymphoma Foundation; Cystic
Fibrosis Foundation; Defeat MSA; The Desmoid Tumor Research
Foundation; The Disability Rights Legal Center; Dupl5q
Alliance; Dysautonomia Foundation; Equal Access for Rare
Disorders; Fight Colorectal Cancer; FORCE: Facing Our Risk of
Cancer Empowered; Former FDA Commissioner Margaret Hamburg;
Former FDA Commissioner Robert Califf; Friedreich's Ataxia
Research Alliance (FARA); Friends of Cancer Research; Georgia
State University College of Law; The Global Foundation for
Peroxisomal Disorders.
Glutl Deficiency Foundation; The Guthy-Jackson Charitable
Foundation; Hemophilia Federation of America; Hematology/
Oncology Pharmacy Association; HLRCC Family Alliance; Hope
for Hypothalamic Hamartomas; Hyper IgM Foundation, Inc.;
International Fibrodysplasia Ossificans Progressiva (FOP)
Association; International Myeloma Foundation; International
Pemphigus and Pemphigoid Foundation; International Society
for Stem Cell Research; International Waldenstrom's
Macroglobulinemia Foundation (IWMF); The Isaac Foundation;
Jack McGovern Coats' Disease Foundation; The LAM
Foundation; The Leukemia & Lymphoma Society; Lymphoma
Research Foundation; Li-Fraumeni Syndrome Association (LFS
Association/LFSA); LUNGevity Foundation; Max Cure
Foundation.
M-CM Network; Mattie Miracle Cancer Foundation; MitoAction;
MLD Foundation; Moebius Syndrome Foundation; The MSA
Awareness Shoe; Mucolipidosis Type IV Foundation; The Myelin
Project; Myotonic Dystrophy Foundation; National Brain Tumor
Society; National Coalition for Cancer Survivorship; National
Comprehensive Cancer Network; National Consumers League;
National Health Council; National MPS Society; National
Niemann-Pick Disease Foundation; National Organization for
Rare Disorders (NORD); National Patient Advocate Foundation;
National Physicians Alliance; National PKU Alliance.
National PKU News; National Women's Health Network;
Neurofibromatosis Northeast; NYU Langone Health; Operation
ASHA; Our Bodies Ourselves; PRP Alliance, Inc.; Prevent
Cancer Foundation; Public Citizen; Rare and Undiagnosed
Network (RUN); Sarcoma Foundation of America; Scleroderma
Foundation; The Snyder-Robinson Foundation; Sofia Sees Hope;
SSADH Association.
Susan G. Komen; TargetCancer Foundation; Treatment Action
Group; The Turner Syndrome Society; TMJA (Temporomandibular
Joint Disorders patient organization); United Leukodystrophy
Foundation; United Mitochondrial Disease Foundation (UMDF);
University of Pennsylvania Perelman School of Medicine;
Veterans Health Council; Vietnam Veterans of America; VHL
Alliance; Washington Advocates for Patient Safety; Woody
Matters; Worldwide Syringomyelia & Chiari Task Force; Yale
School of Public Health.

Ms. SCHAKOWSKY. Mr. Speaker, it opens the door for bad actors to take
advantage of terminally ill patients. It is the FDA's job to ensure
that drugs are safe and effective, and we can't trust manufacturers to
act as this gatekeeper.
There is already a safe process for terminally ill patients to access
experimental treatments. Under what is called the expanded access
program, 99 percent of applications are approved. The expanded access
program plays a vital safety role.
I am very troubled by what can happen to patients in some States who
undergo treatment from right-to-try companies. In 19 States, patients
using an investigational drug could actually lose their hospice
coverage; in 6 States, they could be denied home care coverage. These
are the very people who are dependent on hospice and home healthcare,
and this bill would cause them to lose that coverage.
This is not a humane, patient-centered bill for people who are facing
death; it is just a dangerous pathway for bad actors to exploit those
very people.
Mr. Speaker, I urge my colleagues to oppose H.R. 5247, and, again, I
thank the gentleman for the opportunity to speak against this piece of
legislation.
Mr. BURGESS. Mr. Speaker, at this time, I am pleased to yield such
time as he may consume to the gentleman from Oregon (Mr. Walden), the
chairman of the full committee.
Mr. WALDEN. Mr. Speaker, I rise today on behalf of the patients, the
patients who face terminal diagnoses but have exhausted all available
known treatment options.
Before us today we have legislation that received 260 bipartisan
votes last week; that was nearly 260 votes to increase patient access
to investigational drugs through a new pathway.
I want to thank Dr. Burgess for his incredible work on our Energy and
Commerce Committee to do our due diligence, to take an issue that is
important to our citizens and our colleagues and make sure that it has
been properly vetted, reviewed, and improved upon from what we got from
the Senate.
Mr. Speaker, 38 States have right-to-try laws, including my own State
of Oregon. This is something that people want and deserve. Wisconsin
will make it number 39 once the bill they have passed gets across
Governor Scott Walker's desk.
While the State policies vary, they have a common goal, and that is
helping vulnerable patients. President Trump praised the movement
during

[[Page H1743]]

the State of the Union, saying: ``People who are terminally ill should
not have to go from country to country to seek a cure--I want them to
have a chance right here at home.''
I have spoken to the President directly about what we are doing here,
and he gave me a shout-out when he was up in New Hampshire the other
day about moving this bill forward. We worked closely with the Vice
President and his team and with Scott Gottlieb, who is the doctor who
heads the FDA, the Food and Drug Administration, to get a really good,
thoughtful product before this House, and they support what we are
doing here. President Trump also highlighted this bill, as I said, when
he was in New Hampshire.
It is important to note that this isn't the first time we have
considered this bill. As you may know, last week, we tried to move this
on the suspension calendar, never imaging that the Democrats would
actually whip against giving dying patients the right to try one of
these drugs. We had 32 Democrats support this legislation, and that is
why we brought it back under regular order in a rule today.
Now, today, there is an existing process, and you have heard about
it--and we looked at this in the committee--for patients to access
unapproved drugs. The FDA oversees expanded access, commonly known as
compassionate use. This program has been critical in helping patients
access experimental drugs. It does work.
Commissioner Gottlieb and the agency should be commended for their
continued work to improve the expanded access program for patients. As
you have heard from my colleagues on both sides of the aisle, this
program works, and works effectively, but it doesn't do it all, and
that is why this legislation is before us.
To improve upon this successful program, the bill before us today
provides liability protections for manufacturers, sponsors, physicians,
clinical investigators, and hospitals that participate in the existing
expanded access program and the new alternative pathway that we create
under this legislation.
This was a very big issue for those who needed to be brought into
participation who otherwise might have sat on the sidelines and never
made these drugs available. This provision removes one of the biggest
hurdles that patients face and that was identified by the Government
Accountability Office; it is the biggest hurdle they face in getting
access to experimental therapies: manufacturers' hesitancy to
participate. That is the hurdle we are trying to overcome today in a
safe way.
The bill also creates a new alternative pathway for patients who do
not qualify for a clinical trial. This legislation strengthens patient
protections with clearer informed consent and adverse event reporting.
The bill also ensures the FDA is notified when a patient receives an
unapproved drug through the new alternative pathway to ensure proper
oversight.
Mr. Speaker, I want to thank my colleagues in the House, and
especially Dr. Burgess on the Health Subcommittee, but also
Representative Brian Fitzpatrick; Andy Biggs, who is behind me; Morgan
Griffith; and our Vice President, Mike Pence. I am grateful for their
work and for their understanding that our job here in the House is to
do our work: to hear from people who are affected or might be affected,
to improve upon products, to go through regular order, and to bring
this bill to you today.
Mr. Speaker, I urge all of my colleagues in the House to support this
legislation.
Mr. PALLONE. Mr. Speaker, I yield such time as she may consume to the
gentlewoman from California (Ms. Matsui).
Ms. MATSUI. Mr. Speaker, I thank the gentleman for yielding to me.
Mr. Speaker, I rise in opposition to H.R. 5247, the so-called right-
to-try legislation. This bill does not give patients the right to try;
rather, it gives patients the right to request, which fails to address
real barriers to accessing experimental drugs such as drug costs or
company restrictions.
I will reiterate that patients already have the right to try through
an expedited process that approves 99 percent of requests it receives.
This legislation, however, fails to recognize that, if a patient is
denied access, it is usually because a drug manufacturer says no due to
manufacturer concerns about safety or side effects, not because the FDA
denied a request.
I know, like everyone else, I have heard from many constituents
suffering from terminal illnesses such as ALS who are desperate for
cures; and I believe that every single one of us in this Chamber has
confronted, in some way, a family member--mother, father, spouse--who
had, heartbreakingly, an illness that had no cure. We have gone through
the process many times, and I think we all have felt desperate from
time to time.
However, having said that, just because a person at the end has no
hope, to try something that might make things worse so you cannot go on
to a more peaceful resolution would be hurtful not only to the patient,
but to the family.
Opening up unregulated pathways to drugs after only a phase 1
clinical trial may expose patients to severe and unpredictable side
effects. This bill would prevent FDA from documenting these side
effects and, worse, would prevent FDA from protecting other patients
from a similar fate.
When a loved one is in pain, the last thing a family wants is to
cause further suffering. We need clinical trials to ensure drugs are
safe and effective and to find real cures for patients, and we need the
FDA to be a part of the process as a matter of patient protection for
all.
Rescinding any FDA oversight on unproven therapies that have not
undergone multiple clinical trials is a slippery slope. The expedited
process we have now is working, and I cannot support a bill that offers
a ``right to ask'' alongside proposals that could be dangerous for all.
Mr. Speaker, I urge my colleagues to oppose this bill.
Mr. BURGESS. Mr. Speaker, I am pleased to yield 3 minutes to the
gentleman from Virginia (Mr. Griffith), a valuable member of the Energy
and Commerce Committee, the vice chairman of the Oversight and
Investigations Subcommittee.
Mr. GRIFFITH. Mr. Speaker, I have heard people say that they don't
want to support a bill that makes things worse. We have people who are
terminal, whose life expectancy is measured in months, not in decades,
and how do you make things worse?
I said last week, and I repeat it today, that if I--if, and I am not,
thank God--but if I were faced with one of these heart-rendering
situations, I would take any risk, including injecting monkey urine, if
that meant I could spend a few more days, months, or years with my
children.

{time} 1330

I think many people are in that same boat, and the American people
deserve a right to try. When we were doing our hearings on this, we had
an Energy and Commerce Subcommittee on Health hearing where Lieutenant
Commander Matthew Bellina, who graduated from Virginia Tech in my
district and served in the United States Navy, testified before us. He
said, in the conclusion of his comments: ``I know that it is probably
too late for me, and I have 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 served will respect their
liberties and their right to make their own decisions about their
medical treatments.''
He suffered from ALS, as I said. I have had three friends during my
lifetime die of that: Ray Robrecht, my predecessor a couple terms back
in the Virginia House of Delegates; Julie Mullins, whose family I have
known for decades; and Mike Ahern, who was connected with the United
States Senate through his sister. All of these folks were people who
lived in Salem County or Roanoke County, and they all died from ALS.
They were all brave people. They should have had the right to try to
see if they could make an improvement for others.
Even more poignant are my family friends who lost both a grandparent
and their mother to Huntington's chorea. I was their family lawyer. I
did their will. I would like to believe, and I know they would have
liked the option, that their mother would have chosen the right to try,
knowing that, even if it failed, it might help another generation
because, as you know, Huntington's chorea is a genetically transmitted
disease.

[[Page H1744]]

So I do not understand why people are afraid of letting people try
who have no other hope, whose life is going to be cut short, without
taking that Hail Mary pass. And so I hope that everyone will support
this reasonable, measured effort to let people have a choice and a
right to try.
Mr. PALLONE. Mr. Speaker, I yield myself such time as I may consume.
Mr. Speaker, I want to explain some other reasons why I am very
opposed to this bill. I am concerned that H.R. 5247 essentially does
nothing to address what may be the true barrier to expanded access, and
that is the determination by the manufacturer as to whether or not they
will provide access to their product that is under development. And I
want to stress, there is nothing in this legislation before us today
that would compel a manufacturer to grant access upon request.
Further, I believe that trusted manufacturers like J&J, or Johnson &
Johnson, which is headquartered in my district, have already said that
any compassionate use request must be subject to FDA review. Now, I
have heard my colleagues refer to this as a Hail Mary pass for the
terminally ill. I think, in reality, it is offering false hope of a
cure to patients and their families when there is no guarantee that any
patient will receive access to treatment from a manufacturer.
In fact, H.R. 5247 sets an extremely low threshold for the types of
experimental treatments that may be available through this alternative
pathway by allowing patients access to investigational treatments that
have only completed a phase 1 clinical trial. Patients will be exposed
to treatments with no or relatively little data that they are actually
effective. These extremely small trials only examine the safety and
toxicity of a drug and do not determine the effectiveness or potential
side effects. Access at this phase 1 stage in the development could
expose patients to untested products and further harm and result in
delaying access to a treatment that may be more appropriate and more
beneficial for their underlying disease or condition.
Only 1 in 10 products move on from phase 1 clinical trials to FDA
approval. Mr. Speaker, the bill does not make any adverse-event
reporting to the FDA immediate. It also limits FDA's ability to use
clinical outcomes associated with the use of an investigational product
when reviewing a product for approval if it could adversely impact its
review. It also prevents any entity from being held liable for use of
the treatment.
Again, these are some of the many reasons that more than 100
organizations oppose this dangerous bill.
Mr. Speaker, I reserve the balance of my time.
Mr. BURGESS. Mr. Speaker, I yield 4 minutes to the gentlewoman from
Indiana (Mrs. Brooks), another valuable member of the Committee On
Energy and Commerce and the Subcommittee on Health.
Mrs. BROOKS of Indiana. Mr. Speaker, the right-to-try legislation
will be considered on the House floor today. This is about giving
people hope to try. It is about hope to try investigational drugs which
have passed the first of three phases of the FDA clinical trial
process, the safety testing phase. And these investigational drugs
could possibly prolong or save the lives of terminally ill patients.
I like to remind my colleagues that a little boy was in Washington,
D.C., on the House floor just last week when we first voted on this
legislation. Prior to that vote, I had met Jordan during an Energy and
Commerce Committee hearing focusing on the implementation of the 21st
Century Cures Act.
Jordan McLinn is a second grader from Indianapolis who may look like
any other healthy child, but he has Duchenne muscular dystrophy, or
DMD, a fatal, degenerative condition which causes muscle weakness. DMD
is caused by an absence of dystrophin, a protein that helps keep muscle
cells intact. Oftentimes, kids born with DMD are wheelchair-bound by
age 12, and they have a life expectancy of just 25 years old.
DMD is a genetic disease that is typically passed on to boys through
their mother's X chromosome. But sometimes the mother is not a carrier,
there is no family history of the disease, but a child is born with the
disease anyway. This is what happened to Jordan McLinn. His mother,
Laura, is not a carrier. This disease does not run in their family.
Jordan was born with DMD, but it was not diagnosed until he was 4
years old. So can you imagine what this family has been through? After
Jordan was diagnosed, his family hit the ground running, trying to find
the best possible treatment options and therapies for people with DMD.
His mother, Laura, was quoted in The Indianapolis Star today in an
article focused on the right-to-try bill saying: ``The reason we have
remained on this journey and fighting so hard for it is not necessarily
for Jordan immediately. It's for all the patients that we've met along
the way.''

Jordan and his family have been on this journey advocating this fair
and compassionate bill in Indiana and beyond for Jordan but also for so
many others. In 2015, then-Governor Mike Pence signed Indiana's right-
to-try law with Jordan McLinn by his side. Now a total of 38 States
have already passed laws that take a variety of approaches to helping
vulnerable patients. By passing this legislation in the House today, we
will increase access--nationwide--to unapproved, investigational drugs
for patients with a terminal illness.
In that same IndyStar article I mentioned earlier, Laura shared that
Jordan has always wanted to be a firefighter, but now, after coming to
the Nation's Capital many times, he has aspirations to be something
else. He now wants to be President of the United States. This bill
allows Jordan to have those big dreams, and it will be providing
patients across this country with hope.
Yes, it is hope, hope for patients that they may find the cure
someday that they have been searching and fighting for, hope for
patients and their families that there will be more time to make more
memories that can last a lifetime.
In closing, I would just like to emphasize how critically important
it is that Congress join together to support the bill for the millions
of Americans who fight for their lives because of a terminal illness. I
urge my colleagues to support this bill.
Mr. PALLONE. Mr. Speaker, may I inquire how much time I have
remaining?
The SPEAKER pro tempore. The gentleman from New Jersey has 15\1/2\
minutes remaining.
Mr. PALLONE. Mr. Speaker, I yield such time as he may consume to the
gentleman from Texas (Mr. Gene Green), the ranking member of the Health
Subcommittee.
Mr. GENE GREEN of Texas. Mr. Speaker, I thank my ranking member for
yielding to me. I rise in opposition to the right-to-try legislation
that would bypass the Food and Drug Administration's longstanding
review and oversight of drug treatments and endanger patients with
life-threatening diseases.
Many States have passed this right-to-try piece of legislation,
including my home State of Texas, but the States don't have the FDA.
The Federal Government has the right to be able to make sure we can
protect both constituents and consumers. My heart goes out to the loved
ones who are terminally ill and desperate for a breakthrough treatment.
I cannot support legislation that offers false hope to the terminally
ill and their families.
The FDA has a pathway whereby those in need of investigational
medications may seek to obtain them. This program is known as the
expanded access pathway, or compassionate use, and has been in the law
since 1987. Over the last decade, the FDA has a clinical hold on only
two commercial drug development programs due to adverse events
associated with compassionate use.
There are many patient advocacy groups that are opposing this
legislation. Groups such as the Alliance for Aging Research, the
American Cancer Society Cancer Action Network, American Lung
Association, the American Society of Clinical Oncology, the Cystic
Fibrosis Foundation, Defeat MSA, the Disability Rights Legal Center,
and dozens more that are committed to seeking effective treatment cures
to many diseases which are terminal, are against this bill. These
patients' rights groups seek to ensure that the medication that is
offered to individuals is safe, has been tested, and has gone

[[Page H1745]]

through the proper approval process before it is given to a patient.
The most vulnerable and terminally ill individuals deserve to have
access to safe therapies that have undergone the necessary approval
process before being given to those who can least afford to receive
unproven treatment that may do them more harm than good. In addition to
the physical harm which unproven treatments may cause, there is also
the risk of financial exploitation of terminally ill patients given
that such treatments are not covered by insurance. Manufacturers are
not required to cover the cost of investigational treatment.
The majority's decision to go around our committee's consideration
and effort to pass the bill on suspension last week exemplifies what
this legislation is trying to do, circumvent existing rules and
processes that have been created to protect Americans from hasty
decisions.
I ask my colleagues on both sides of the aisle to stand up for
Americans facing serious and life-threatening diseases by opposing this
unnecessary and potentially dangerous legislation.

Mr. BURGESS. Mr. Speaker, I yield 3 minutes to the gentleman from
Arizona (Mr. Biggs), one of the primary drivers on this legislation.
Mr. BIGGS. Mr. Speaker, I thank the gentleman from Texas for
yielding. I also pay my respects and give honor to Ron Johnson, the
Senator from Wisconsin who championed the bill in the Senate and gave
us a superb bill; also, Chairman Walden and his committee, who have
worked hard to give us this bill today; and my original cosponsor on
the bill that I introduced, Mr. Fitzpatrick from Pennsylvania.
Mr. Speaker, I want to address just a couple of things that I think
are really intriguing to me because it certainly seems a bit
condescending to me when I hear people say: I am not going to support
this because it gives false hope that people might be taken advantage
of by bad actors. They cannot identify the bad actors, but they might
be taking advantage of them. That is a fallacious and specious argument
to make when you are denying people who have a terminal illness, who
have been diagnosed with a terminal illness, who have gone through the
already approved FDA processes in order to get and petition a
pharmaceutical company for an experimental drug that might prolong
their life and might heal them.
False hope, that argument, is the argument that I am hearing. But the
reality is these people are individuals. They have a higher sense of
reality than virtually anybody else I know because their mortality is
there. They want the opportunity. It is not false hope. It is hope.
Support of this bill is compassionate. Support of this bill is fair.
I have also heard that there may be some liability issues on the part
of pharmaceutical companies which might impede them from providing
drugs. Yet, in order to satisfy them, the bill itself says that they
are exculpated unless their conduct is willful or criminal. That means
that they have protection.
What I am asking here today, and what everyone with whom I have met
over the years who want a right to try is asking, is simply a chance to
have some determination and control over their own lives.

{time} 1345

One of the intriguing arguments I hear today and I heard last week
is, well, you know what, the pharmaceutical companies aren't compelled
to provide these drugs. So my immediate question is: Oh, so you would
be more comfortable, then, if we would have included a compulsory means
in the bill? Did you want the pharmaceutical companies to be compelled
to provide these?
The answer would be no. It is simply they don't like this bill. They
don't want the bill.
When you have 38, soon to be 39, States that want to give their
citizens, Americans all, the right to try to preserve their lives and
to be healed and have a chance, they need to get that; they need that
opportunity. We need to give it to them today.
Mr. PALLONE. Mr. Speaker, I yield myself such time as I may consume.
Mr. Speaker, there has been a lot of misinformation spread by
supporters of this legislation that FDA is a barrier to patients
receiving access to these investigational treatments, and I want to be
very clear that that is simply not the case.
FDA's expanded access program approves nearly all requests for
investigational drugs or biologics it receives. For the past 5 years,
FDA's approval rate for expanded access requests has been over 99
percent. In fiscal year 2017, as I previously mentioned, only nine
individual requests were denied.
FDA also conducts its review quickly. FDA physicians are available 24
hours a day to approve any emergency expanded access requests the
agency receives, typically granting emergency requests immediately,
over the phone, and nonemergency requests in a median time of 4 days
and, generally, no longer than 30 days.
FDA has also taken actions to streamline the expanded access request
process for physicians to make it less burdensome. I think that was
mentioned by Mr. Walden, the chairman.
Pharmaceutical companies can choose to deny a patient access to an
experimental treatment because, for example, there is not enough of the
drug available or they are concerned about dangerous side effects. The
fact is, when a patient is denied access to an experimental treatment,
it is because the company has said no, not the FDA.
So let's be clear as to what this legislation is. It is an attempt to
undermine the authority of the expert public health agency charged with
reviewing drugs to ensure their safety and efficacy.
I would urge my colleagues to oppose this grab at FDA's authority.
That is really what this legislation is all about.
Mr. Speaker, I reserve the balance of my time.
Mr. BURGESS. Mr. Speaker, I yield 3 minutes to the gentleman from
Georgia (Mr. Allen).
Mr. ALLEN. Mr. Speaker, I rise today to encourage my colleagues to
join me in supporting H.R. 5247, the Right to Try Act, and I thank Dr.
Burgess and the Energy and Commerce Committee for bringing this
important legislation to the floor of this House.
In certain States across our Nation, patients who are diagnosed as
terminally ill are being told by doctors that all of the treatment
options have been exhausted because they do not have access to
experimental drugs. This type of overregulation by the Federal
Government is creating hopeless situations for thousands of Americans
whom we hold dearest to our hearts.
This right-to-try legislation allows terminal patients to have a
choice on whether or not an experimental approach is the path for them,
as sometimes, and many times, this is their only option.
Should this bill become Federal law, our terminally ill patients will
have increased access, nationwide, to unapproved drugs, leading to more
scientific breakthroughs that will benefit all Americans and, in lots
of cases, will save a life.
Now is the time for Congress to take action and give terminally ill
patients a fighting chance for their God-given right to life. How in
God's name can this Congress deny an American the right to life?
Mr. Speaker, I urge all my colleagues to join me today in supporting
this bill on this floor.
Mr. PALLONE. Mr. Speaker, I yield myself such time as I may consume.
Mr. Speaker, I said before that I have found that some Members were
looking to vote for this bill because they said: Well, we have the
right to try in our State by State statute, so what is the difference
if we do it on the Federal level?
I just want to stress again that the State right-to-try laws do not
give patients a right to try effectively and have done little to expand
access to investigational treatments.
There are 37 States and the District of Columbia that have enacted
right-to-try laws, and there is no evidence that anyone has obtained an
investigational treatment via these laws that couldn't have been
obtained through FDA's expanded access program.
Right-to-try laws do not compel companies to provide patients access
to investigational treatments; therefore, under these State laws,
patients still do not have a right to try, only the

[[Page H1746]]

right to request the treatment from the company.
State right-to-try laws do not address the fundamental barriers of
cost and company restrictions. Neither the FDA nor States require
insurers or pharmaceutical companies to cover the cost or reduce the
costs of these often expensive treatments. Instead, these laws put
patients at higher risk by prohibiting or weakening FDA's oversight of
investigational treatments.
Mr. Speaker, I reserve the balance of my time.
Mr. BURGESS. Mr. Speaker, I yield 3 minutes to the gentleman from
Tennessee (Mr. Roe), the chairman of the Committee on Veterans'
Affairs.
Mr. ROE of Tennessee. Mr. Speaker, I rise today in strong support of
H.R. 5247, the Right to Try Act.
I am a physician and scientist with over 40 years experience treating
patients, some of whom had the dreaded diagnosis of cancer.

Six months ago, I was operated on for cancer, and I, to this day, am
a cancer survivor. If needed, I would like to have the right to try.
A little over 3 years ago, my beloved wife, Pam Roe, a nurse and
friend, died of stage IV colon cancer. She would have liked to have had
the right to try.
Less than 2 months after that, one of the best friends I will ever
have in my life, Phil Street, a Vietnam veteran, Air Force veteran,
died of a cancer related to Agent Orange. Phil would have liked to have
had the right to try.
My senior partner in medical practice, a year later, good friend, was
diagnosed with brain cancer. Dr. Cone would have liked the right to
try.
Shortly after that, Linda Baines, a scrub nurse that I have operated
with hundreds of times in my medical practice, was diagnosed with
cancer. Linda would have liked the right to try.
I have two dear friends at this moment who are both being treated for
stage IV cancer. If those treatments don't work--and I have had to look
patients in the eye and say, Mr. Speaker: ``Your life is not in my
hands anymore. It is in God's hands''--they would like to have the
right to try.
I tell you this: all these patients want and deserve is a right to
try. Please, I am asking you to support this legislation.
Mr. PALLONE. Mr. Speaker, may I inquire how much time remains and
whether the gentleman has additional speakers on his side.
Mr. BURGESS. Mr. Speaker, I will be closing.
The SPEAKER pro tempore. The gentleman from New Jersey has 9 minutes
remaining. The gentleman from Texas has 3\1/2\ minutes remaining.
Mr. PALLONE. Mr. Speaker, I yield myself such time as I may consume.
Mr. Speaker, I just want to stress that, as I said before, we have
the four previous FDA Commissioners, two Democrats and two Republicans
appointed by President Bush, who have raised serious concerns about
this legislation because it excludes FDA review and they think could
pose serious risks to vulnerable patients.
I just wanted to read, once again, a statement that they made jointly
to The Washington Post, where they said: ``There is no evidence that
either bill would meaningfully improve access for patients, but both
would remove the FDA from the process and create a dangerous precedent
that would erode protections for vulnerable patients.''
Mr. Speaker, I just want to stress to my colleagues on both sides of
the aisle that my concern is that no one is actually going to be able
to get an experimental drug by this bill. In other words, if you are a
manufacturer that actually has done something and come up with an
experimental drug that you believe will make a difference to someone
who is terminally ill, you are likely going to want to go through the
FDA expanded access process because then there is a seal of approval
that the FDA has actually looked at this and said that it is relatively
safe to use.
So my real fear is that the only thing this is going to do is open up
to the possibility of some charlatan, fly-by-night snake oil drug
company or manufacturer who is going to make all kinds of claims that
have not been reviewed by the FDA for any kind of safety, and that then
people may say: Okay. Well, I will take that because I am terminally
ill and I might as well try something.
But that isn't really what we should be doing here. We should be
providing a process, as the FDA does right now, where, if someone is
terminally ill and they want to try something, they at least have some
certification of approval by the FDA that this is something that may
help them, that may make a difference, and that, in the case of about
11 percent of the cases where the application is made to the FDA, some
changes are made to make sure that even though there is a certain level
of risk, that that level of risk is reduced by the FDA putting on
additional safety precautions.
So my real concern here is I don't want people to vote for this
legislation thinking that somehow it is going to make a difference. I
really don't believe that is true. Otherwise, I wouldn't urge the
opposition that I am. But for all these reasons, I do urge strong
opposition to this bill and ask that my colleagues vote ``no.''
Mr. Speaker, I yield back the balance of my time.
Mr. BURGESS. Mr. Speaker, I yield myself the balance of my time.
Mr. Speaker, yesterday, during the rule debate on this bill, I
outlined a case where the previous Speaker, Nancy Pelosi, provided the
right to try for a patient, a Democratic donor, back in my home State
of Texas. So, really, all we are asking today is that we give regular
Americans, the forgotten men and women of this country, the same rights
that the Speaker of the House provided to a Democratic donor back in
October of 2008.

Yesterday I quoted from an article from the Dallas Morning News. I
have a different but similar article today talking about the same case,
talking about the individual who had a diagnosis of multiple myeloma.
There was a drug that perhaps would provide some hope. The individual
was clearly terminal. This monoclonal antibody that was primarily used
to treat multiple sclerosis might show some efficacy in treating the
advanced form of multiple myeloma that this patient had. The drug had
been through phase 1 clinical trials. The patient did not have time for
the drug to go through phase 2 and phase 3 clinical trials.
The article says:

Enter Nancy Pelosi. Through means to which we have never
been privy, Ms. Pelosi got the FDA to give the manufacturer
the all-clear to give the drug to the patient. The patient
got the drug, the patient took the drug, but, unfortunately,
the patient died anyway, but his family remains grateful to
the Speaker for interceding on his behalf.

I don't doubt that they are.
Yesterday, I quoted the Dallas Morning News article where the
patient's spouse said, somehow, Nancy Pelosi got it done.
Well, do you know what, Mr. Speaker? You shouldn't have to depend on
the Speaker of the House to intercede on your behalf to get the FDA to
get the manufacturer to make a drug available. If you are really up
against a bad situation, wouldn't it be better if we provided everyone
that same pathway?
That is what this bill does today. That is why the right-to-try
legislation was advocated by the President of the United States. In
fact, I think it was the only legislative priority that the President
laid out during his State of the Union Address where he wanted to see
Congress act.
So today, we are going to do that. Today, we are going to act. It is
an important bill. I encourage my colleagues to vote in favor of it.
Mr. Speaker, I yield back the balance of my time.
The SPEAKER pro tempore. All time for debate has expired.
Pursuant to House Resolution 787, the previous question is ordered on
the bill.
The question is on the engrossment and third reading of the bill.
The bill was ordered to be engrossed and read a third time, and was
read the third time.

{time} 1400

Motion to Recommit

Mr. PALLONE. Mr. Speaker, I have a motion to recommit at the desk.
The SPEAKER pro tempore. Is the gentleman opposed to the bill?
Mr. PALLONE. I am opposed to the bill in its current form.
The SPEAKER pro tempore. The Clerk will report the motion to
recommit.

[[Page H1747]]

The Clerk read as follows:

Mr. Pallone moves to recommit the bill H.R. 5247 to the
Committee on Energy and Commerce with instructions to report
the same back to the House forthwith, with the following
amendment:

Strike section 2 and insert the following:

SEC. 2. USE OF UNAPPROVED INVESTIGATIONAL DRUGS BY PATIENTS
DIAGNOSED WITH A TERMINAL ILLNESS.

(a) In General.--Chapter V of the Federal Food, Drug, and
Cosmetic Act is amended by inserting after section 561A (21
U.S.C. 360bbb-0) the following:

``SEC. 561B. INVESTIGATIONAL DRUGS FOR USE BY ELIGIBLE
PATIENTS.

``(a) Use of Clinical Outcomes.--
``(1) In general.--The Secretary shall issue guidance
describing the Secretary's consideration and evaluation, for
purposes of the review of, and decision on whether to
approve, a marketing application under section 505 of this
Act or section 351 of the Public Health Service Act for an
investigational drug, of clinical outcomes associated with
the provision by a sponsor or manufacturer of such drug under
subsection (b) or (c) of section 561. Such guidance shall
address--
``(A) specific instances in which the Secretary will
determine that the public health requires such consideration
and evaluation;
``(B) specific instances in which a sponsor may request
such consideration and evaluation; and
``(C) the context in which such consideration and
evaluation will occur, particularly with regard to
information and data relevant to the evaluation of a
marketing application under section 505 of this Act or
section 351 of the Public Health Service Act for the
investigational drug.
``(2) Guidance.--
``(A) Draft guidance.--Not later than 1 year after the date
of enactment of this section, the Secretary shall issue draft
guidance with a public comment period regarding the use of
clinical outcomes associated with the use of an
investigational drug that a sponsor or manufacturer has
provided under subsection (b) or (c) of section 561, as
described in paragraph (1).
``(B) Final guidance.--Not later than 1 year after the
public comment period on such draft guidance ends, the
Secretary shall issue final guidance.
``(b) Posting of Information.--Not later than 1 year after
the date of enactment of this section, the Secretary shall
post on the internet website of the Food and Drug
Administration and update annually, categorized by
therapeutic area--
``(1) the number of requests that were received by the Food
and Drug Administration for the provision by a sponsor or
manufacturer of an investigational drug under subsection (b)
or (c) of section 561; and
``(2) the number of such requests that were granted.''.
(b) Reporting.--Section 561A of the Federal Food, Drug, and
Cosmetic Act (21 U.S.C. 360bbb-0) is amended adding at the
end the following:
``(g) Reporting.--The manufacturer or sponsor of an
eligible investigational drug shall post on the same publicly
available internet website used by the manufacturer for
purposes of subsection (b) of this section an annual summary
of any provision by the manufacturer or sponsor of an
investigational drug under subsection (b) or (c) of section
561. The summary shall include the number of requests
received, the number of requests granted, the number of
patients treated, the therapeutic area of the drug made
available, and any known or suspected serious adverse events.
Such annual summary shall be provided to the Secretary upon
request.''.
(c) Liability.--Section 561 of the Federal Food, Drug, and
Cosmetic Act (21 U.S.C. 360bbb) is amended--
(1) by redesignating subsection (e) as subsection (f); and
(2) by inserting after subsection (d) the following:
``(e) Liability.--
``(1) Alleged acts or omissions.--
``(A) Manufacturer or sponsor.--No manufacturer or sponsor
(or their agent or representative) of an investigational drug
provided to a single patient or small group of patients for
treatment use shall be liable for any alleged act or omission
related to the provision of such drug, so long as such drug
was provided in accordance with subsection (b) or (c),
including the reporting of safety information, from clinical
trials or any other source, as required pursuant to section
312.32 of title 21, Code of Federal Regulations (or any
successor regulations).
``(B) Physician, clinical investigator, or hospital.--
``(i) No licensed physician, clinical investigator, or
hospital shall be liable for any alleged act or omission
related to the provision to a single patient or small group
of patients for treatment use of an investigational drug in
accordance with the requirements described in clause (ii),
unless such act or omission constitutes on the part of such
physician, clinical investigator, or hospital with respect to
such investigational drug--

``(ii) The requirements described in this clause are the
requirements under subsection (b) or (c), including--

``(I) the reporting of safety information, from clinical
trials or any other source, as required pursuant to under
section 312.32 of title 21, Code of Federal Regulations (or
any successor regulations);
``(II) ensuring that the informed consent requirements of
part 50 of title 21, Code of the Federal Regulations (or any
successor regulations) are met; and
``(III) ensuring that review by an institutional review
board is obtained in a manner consistent with the
requirements of part 56 of title 21, Code of the Federal
Regulations (or any successor regulations).

``(2) Determination not to provide drug.--No manufacturer,
sponsor, licensed physician, clinical investigator, or
hospital, nor the Secretary, shall be liable for determining
not to provide access to an investigational drug under this
section or for discontinuing any such access that it
initially determined to provide.
``(3) Limitation.--
``(A) In general.--Except as set forth in paragraphs (1)
and (2), nothing in this section or section 561B shall be
construed to modify or otherwise affect the right of any
person to bring a private action against a manufacturer or
sponsor (or their agent or representative), physician,
clinical investigator, hospital, prescriber, dispenser, or
other entity under any State or Federal product liability,
tort, consumer protection, or warranty law.
``(B) Federal government.--Nothing in this section or
section 561B shall be construed to modify or otherwise affect
the authority of the Federal Government to bring suit under
any Federal law.''.

Mr. PALLONE (during the reading). Mr. Speaker, I ask unanimous
consent to dispense with the reading.
The SPEAKER pro tempore. Is there objection to the request of the
gentleman from New Jersey?
There was no objection.
The SPEAKER pro tempore. Pursuant to the rule, the gentleman from New
Jersey is recognized for 5 minutes in support of his motion.
Mr. PALLONE. Mr. Speaker, this is an amendment to the bill, or the
final amendment to the bill, which will not kill the bill or send it
back to committee. If adopted, the bill will immediately proceed to
final passage, as amended. And this amendment would offer a more
targeted approach to improving the FDA's current expanded access
program.
In October, the Energy and Commerce Committee held a hearing on the
widely opposed Senate right-to-try legislation. At that hearing, we
heard concerns from FDA Commissioner Gottlieb and also from
manufacturers, academic experts, and patient groups that S. 204 was
legislation that would expose broad numbers of patients to harm, and
sought to hamstrung the FDA's ability to oversee or engage in any
meaningful way on the use of investigational treatments.
Since that time, my colleagues on the other side of the aisle have
drafted new legislation that maintains, in my opinion, the same harmful
approach prohibiting FDA review of experimental treatments. The FDA is
part of the process for a reason. It protects patients from potentially
bad actors or from experimental treatments that might do more harm than
good.
So my motion to recommit, Mr. Speaker, abandons this harmful attempt
to undermine the FDA's expanded access pathway and, instead, seeks to
make two improvements that have been identified as meaningful by both
manufacturers and patient groups.
This proposal will also not be any surprise to Chairman Walden or
Chairman Burgess because it was the bipartisan proposal our staffs were
negotiating prior to the introduction of the current Republican bill.
So I want to stress that, unlike the current bill, H.R. 5247, this
proposal is not based on the false premise that FDA approval is a
barrier to accessing investigational treatments. Rather, it addresses
the two key problems identified by expert witnesses at our hearing: how
the FDA will utilize clinical outcomes of investigational treatments
and liability protection.
To that end, under this motion to recommit, the FDA is directed to
issue guidance to manufacturers specifically on how and when the FDA
will consider clinical outcomes, and when a sponsor may request the
consideration of such outcomes when it comes time to submit an
application for approval for the investigational treatment.
This will provide manufacturers with the clarity they are seeking
regarding

[[Page H1748]]

how allowing patients access to drugs that are still under development
may impact their ability to gain full FDA approval. It will also ensure
that there is a public process for such guidance, ensuring that
stakeholders will have the opportunity to offer their views on this
issue.
Mr. Speaker, the motion to recommit also provides liability
protection to manufacturers, physicians, clinical investigators, and
hospitals, if they are in compliance with the current law and
regulations for expanded access. If you are a manufacturer, a
physician, or a hospital that is in compliance with current rules and
requirements related to expanded access, you will receive protection
for allowing access to the investigational treatment.
Finally, it also provides transparency around the number of expanded
access requests the FDA receives and grants, how many requests a
manufacturer receives and grants, and if there are any serious adverse
events. This transparency, I believe, will provide clear data as to how
many patients are making expanded access requests and how often these
requests are granted or denied by the FDA and manufacturers.
Mr. Speaker, I believe that these legislative fixes will go a long
way to bolstering the existing successful expanded access pathway,
while maintaining the critical review and oversight of the agency
charged with protecting our public health, that being the FDA.
I just want to say that, last fall, FDA Commissioner Gottlieb
testified on right-to-try efforts and told our committee: ``There is a
perception that certain products that aren't being offered under FDA
expanded access will be offered under right-to-try, and I don't see
that.''
That is our current Commissioner Gottlieb, who I respect a great
deal.
Rather than creating an unnecessary alternative pathway that
threatens our drug approval process and our clinical trial program, I
would urge my colleagues to join with Democrats and 103 patient
organizations in supporting the current expanded access program.
These targeted improvements under the motion to recommit to the
existing program are, I think, a way to achieve a better goal. So I
urge my colleagues to support my motion to recommit and oppose this,
what I consider, dangerous Republican proposal in the bill before us.
Mr. Speaker, I yield back the balance of my time.
Mr. BURGESS. Mr. Speaker, I claim the time in opposition to the
motion to recommit.
The SPEAKER pro tempore. The gentleman from Texas is recognized for 5
minutes.
Mr. BURGESS. Mr. Speaker, while well-intentioned, this motion to
recommit falls short of providing vulnerable patients full access to
experimental treatments.
Providing clarity on how negative side effects will be accounted for
during drug approvals is helpful. Giving manufacturers, sponsors,
physicians, hospitals, and clinical investigators certainty on
liability protections is meaningful. Taken together, these improvements
to the existing expanded access program could lead to enhanced
manufacturer and sponsor participation and increased patient access.
But this would not provide an alternative pathway for patients who
cannot get into a clinical trial and have been rejected from
participation in the existing compassionate use program.

This bill before us today does provide an alternative pathway, one
that strengthens patient protections with clearer informed consent and
real-time adverse event reporting. This bill--the underlying bill--also
makes certain that the FDA is notified when a patient receives an
unapproved drug through the new alternative pathway to ensure proper
oversight. These are significant patient protections.
With this motion to recommit, we have a choice. The underlying bill
is the only choice that gives those patients in the greatest need of
help access to investigational drugs, with their consent, even after
they were rejected from participating in a clinical trial or expanded
access.
Mr. Speaker, the choice is clear. We need to vote to expand patient
access. We need to vote down the motion to recommit. We need to vote
for the underlying bill.
Mr. Speaker, I yield back the balance of my time.
The SPEAKER pro tempore. Without objection, the previous question is
ordered on the motion to recommit.
There was no objection.
The SPEAKER pro tempore. The question is on the motion to recommit.
The question was taken; and the Speaker pro tempore announced that
the noes appeared to have it.
Mr. PALLONE. Mr. Speaker, on that I demand the yeas and nays.
The yeas and nays were ordered.
The SPEAKER pro tempore. Pursuant to clause 8 of rule XX, further
proceedings on this question will be postponed.

____________________