[Congressional Record Volume 164, Number 44 (Tuesday, March 13, 2018)]
[House]
[Pages H1521-H1528]
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. WALDEN. Mr. Speaker, I move to suspend the rules and pass 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.
The Clerk read the title of the bill.
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 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
[[Page H1522]]
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 (Mr. Curtis). Pursuant to the rule, the
gentleman from Oregon (Mr. Walden) and the gentleman from New Jersey
(Mr. Pallone) each will control 20 minutes.
The Chair recognizes the gentleman from Oregon.
General Leave
Mr. WALDEN. Mr. Speaker, I ask unanimous consent that all Members may
have 5 legislative days in which to revise and extend their remarks and
insert extraneous material into the Record on the bill.
The SPEAKER pro tempore. Is there objection to the request of the
gentleman from Oregon?
There was no objection.
Mr. WALDEN. Mr. Speaker, I yield myself such time as I may consume.
Mr. Speaker, I rise today on behalf of the patients who face terminal
diagnoses but have exhausted all available treatment options. These are
patients like Jordan McLinn, who is with us today.
Jordan is a tireless fighter who self-advocates for others living
with Duchenne muscular dystrophy. He is a namesake of this bill we are
considering, like Matt Bellina, who testified before the Health
Subcommittee last year. Because of folks like Jordan and Matt, we have
a chance to increase patient access to experimental therapies.
Thirty-eight States across our great land have right-to-try laws,
including my home State of Oregon. Wisconsin, with a bill on its way to
Governor Scott Walker's desk, will soon make it 39. While the State
policies vary, they have a common goal: helping vulnerable patients.
President Trump praised the movement during 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 to give them a chance here at
home.''
Now, today, there is an existing process 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.
Commissioner Scott Gottlieb and the Agency, the FDA, should be
commended for their continued work to improve the expanded access
program for patients.
To improve this successful program, the bill before us today also
provides liability protections for manufacturers, sponsors, physicians,
clinical investigators, and hospitals that participate in the existing
expanded access program and the new alternative pathway created under
this legislation.
This provision removes one of the biggest hurdles that patients have
faced in getting access to these medicines, in gaining access to
experimental therapies, as identified by the Government Accountability
Office: manufacturer hesitancy to participate. That is the big hurdle.
We seek to overcome it with this legislation.
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, the Food and Drug Administration, is
notified when a patient receives an unapproved drug through the new
alternative pathway to ensure there is proper oversight.
Mr. Speaker, I want to thank the House sponsors of this legislation
who have worked tirelessly to bring this to a good place today: Brian
Fitzpatrick, our colleague from Pennsylvania; Andy Biggs from Arizona;
and Morgan Griffith from Virginia. I also thank the Vice President,
with whom Jordan and I met today. I am grateful for their work on
behalf of these courageous patients, and I urge all my colleagues in
the House to support this legislation.
Mr. Speaker, I reserve the balance of my time.
{time} 1745
Mr. PALLONE. Mr. Speaker, I yield myself such time as I may consume.
I rise today in strong opposition to H.R. 5247, or the Right to Try
Act of
[[Page H1523]]
2018. Supporters of this legislation, Mr. Speaker, have claimed that it
will provide seriously ill patients, who have exhausted all of their
available treatment options, access to experimental therapies free from
the barriers of FDA oversight.
While it is understandable that someone suffering from a disease who
has no more options would want to try anything that could help them
fight their disease, this legislation delivers the false hope to
patients and their families that they will receive a cure to their
underlying disease or condition.
In fact, this legislation provides patients and their families
nothing more than the right to ask a manufacturer for access to early
stage, unproven treatments. Like other so-called right-to-try
proposals, H.R. 5247 is based on the false premise that patients are
not receiving access to the investigational treatments as a result of
the Food and Drug Administration, and this simply not the case.
Through the FDA's existing expanded access program, seriously ill
patients are able to request access to investigational products. The
FDA approves 99 percent of all requests for investigational drugs or
biologics that it receives through this program.
Last year, FDA received more than 1,500 requests, and only 9 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, Mr. Speaker. In fact, FDA
often grants emergency requests for expanded access immediately over
the phone, and nonemergency requests are processed in an average of 4
days.
FDA has even made improvements to streamline the process. For
example, FDA has revised the application for physicians to ensure that
it now takes less than an hour to complete. FDA has 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, and how outcomes will be
considered as part of the review process.
Last fall, FDA Commissioner Gottlieb testified on right-to-try
efforts and told our committee that: ``There is a perception that
certain products that aren't being offered under FDA expanded access .
. . will be offered under right-to-try. I don't see that,'' the
commissioner said. As I have said, the review process is working well,
but this legislation would completely take FDA out of the review
process. This is dangerous and could put patients at serious risk.
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. While FDA approves 99 percent of the treatments it
reviews, it also revises applications for 11 percent of patients to
improve patient safety protections.
In order to protect patients, this review should continue. We must
protect patients from bad actors or from dangerous treatments that
would make their lives worse. I am extremely concerned that the
legislation we are considering today is advancing a solution to address
barriers to investigational treatments that do not exist and could
expose seriously ill patients to greater harm instead of the greater
access that they are looking for.
The true barrier to any expanded access is the determination by the
manufacturer as to whether or not they will provide access to their
products that are under development. But nothing in the legislation
before us today would compel a manufacturer to grant access upon
request.
Further, H.R. 5247 would allow patients access to investigational
treatments that have only completed a phase I clinical trial. That is
an extremely small trial. It does not determine the effectiveness, or
the potential side effects of a drug. Access at this stage in the
development could expose patients to untested products, further harm,
and result in delaying access to a treatment that may be more
appropriate and more beneficial for their underlying disease or
condition.
H.R. 5247 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 if it
could adversely impact its review. It also prevents any entity from
being held liable for use of the treatment.
And while I appreciate, Mr. Speaker, the intent of this bill, I can't
support it. The last thing I want to do is give patients false hope and
to potentially put them at risk by completely removing FDA from the
review and approval process.
Finally, Mr. Speaker, it is outrageous, in my opinion, that a bill of
this magnitude is being considered under a suspension of the rule. As
my Republican colleagues well know, bills considered under suspension
are traditionally bipartisan bills that have worked their way through
the appropriate committees with overwhelming bipartisan support.
This bill was never considered by the Energy and Commerce Committee.
In fact, it was only introduced today. A bill with such critical
patient safety implications should not be considered in this fashion.
So I urge my colleagues to oppose this misguided legislation and stand
with the more than 100 organizations that have come forward expressing
their concern for patients and the unnecessary risk this legislation
could expose our Nation's most vulnerable to.
Mr. Speaker, I reserve the balance of my time.
Mr. WALDEN. Mr. Speaker, I now have the honor of yielding 3 minutes
to the gentleman from Pennsylvania (Mr. Fitzpatrick), who has been,
even before he got to the Congress, an extraordinary advocate for this
cause and for the patients with terminally ill conditions.
Mr. FITZPATRICK. Mr. Speaker, I want to thank Chairman Walden; Mr.
Burgess; Mr. Griffith; my friend, Andy Biggs; and Senator Ron Johnson
for their resolute commitment to see the Right to Try Act brought to a
vot today.
Mr. Speaker, each year, thousands of Americans receive the
devastating diagnosis of a terminal illness. And 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. 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 the right to try is all about. As the chairman
indicated, 38 States have passed this bill with near unanimous,
bipartisan support. A version of this bill unanimously passed the
United States Senate.
However, we know Congress cannot legislate miracles. That is why,
when talking about the right to try, we are careful not to represent it
as a cure itself. The reality is that, while passing this measure is a
step, the families and advocates we have worked closely with for years
know that the right to try isn't a guarantee. It is about protecting
hope and protecting opportunity--hope and opportunity for those like my
constituent, Lieutenant Commander Matthew Bellina, a retired naval
aviator and father of three, who was diagnosed with ALS in 2014.
Following the onset of his symptoms, Matt was grounded from flying.
He eventually moved back home to Bucks County with his wife, Caitlin,
and his three children to be surrounded by family and friends.
Although this disease stopped Matt's military service, he quickly
picked up the fight with his new battle, involving himself in the ALS
community and becoming a strong advocate for right-to-try legislation.
Together with Jim Worthington and the Have a Heart Foundation, Matt
advocated for the right to try across the Nation.
While the FDA has a program that allows terminally ill patients to
apply for early access to promising treatment, the right to try is
needed because the FDA's compassionate use process doesn't help enough
people. Only about 1,200 people a year can make it through the current
time-consuming and expensive application process. Comparatively, Mr.
Speaker, in 2014, more than 12,000 people in France were using
investigational treatments through that government's equivalent
program.
If a country with one-fifth of the population of the United States
can help 900 percent more people, the FDA program clearly is not
working. This bill
[[Page H1524]]
does not gut the FDA or fundamentally change the relationship between
doctor and patient. What it does is give Americans facing a terminal
diagnosis a new pathway for treatments undergoing clinical trials.
I want to read something in closing, Mr. Speaker, that I received
from Matt Bellina, who is with us today. ``Please let them know that I
have had ALS too long to meet the exclusion criteria for any promising
trials. No drug company will offer me treatments under the current EAP
guidelines. Two reputable companies have already indicated that they
would try to treat me under the rules of this bill. A vote against this
is essentially a vote to kill me. It is a vote to make my wife a widow
and leave my boys fatherless. I can't stop anyone from voting that way,
but please ask them to have the respect to look my family in the eye
when they cast'' that vote.
Mr. Speaker, when a life hangs in the balance, the Federal Government
should not stand in the way of access to potentially lifesaving
treatment.
Mr. PALLONE. Mr. Speaker, I yield such time as he may consume to the
gentleman from Texas (Mr. Gene Green), who is the ranking member of our
Subcommittee on Health.
Mr. GENE GREEN of Texas. Mr. Speaker, I thank my ranking member for
allowing me to speak tonight.
Mr. Speaker, I rise in opposition to the Right to Try Act,
legislation that would bypass the Food and Drug Administration's
longstanding review and oversight of drug treatments and endanger
patients with life-threatening diseases.
My heart goes out to the families of loved ones who are terminally
ill and desperate for a breakthrough treatment. I, too, have lost loved
ones and wished there was an experimental therapy available to save
them.
FDA has decades of experience dealing with experimental therapies
that have not received final approval. In 1987, the FDA created
expanded access, better known as compassionate use, and gives
terminally ill patients access to therapies still under clinical
trials. FDA approves nearly all requests for investigational drugs. For
the last 5 years, the FDA approval rate for this expanded access is
over 99 percent. In fact, FDA physicians are available 24 hours a day
to approve emergency requests.
My daughter is an infectious disease physician at the University of
Nebraska Medical Center. They used the FDA's compassionate pathway to
provide experimental therapy for an American doctor, a U.S. citizen,
who had contracted Ebola while in Africa in 2014. FDA approved that
request for that experimental treatment over the telephone in less than
24 hours. There is a solution other than this bill.
The new path created in this legislation is not necessary, and, in
fact, may endanger the health and safety of terminally ill patients by
bypassing FDA's oversight and expertise.
Mr. Speaker, I also want to speak on the importance of following
regular order. The House Energy and Commerce Committee has been working
with stakeholders and Federal agencies for years on creating incentives
and pathways for the new generation of breakthrough therapies.
Two years ago, these efforts culminated with the passage of the 21st
Century Cures Act, which I am proud to be a champion of. The 21st
Century Cures Act went through regular order, including hearings;
Member discussions; and compromises between regulators, stakeholders,
and regulators.
It is not easy or quick, but regular order works because it gives the
committees of jurisdiction the opportunity to debate and refine the
legislation. This legislation we are currently considering did not go
through regular order. In fact, it was just introduced earlier today,
purposely avoiding consideration before our Energy and Commerce
Committee due to its shortcomings.
I hope we can agree on the importance of following regular order and
observe our Chamber's rules and traditions. The American people deserve
nothing less. I ask my colleagues on both sides of the aisle to stand
up for Americans facing these serious and life-threatening diseases by
opposing this unnecessary and potentially dangerous legislation.
Mr. WALDEN. Mr. Speaker, I yield 2 minutes to the gentleman from
Texas (Mr. Barton), the former chairman of the full committee and the
current vice chairman.
(Mr. BARTON asked and was given permission to revise and extend his
remarks.)
Mr. BARTON. Mr. Speaker, I have listened to my friends on the
minority talk about the reasons they are opposing this bill, and a
normal piece of legislation that would have some merit didn't go
through regular order, things of this sort. But, Mr. Speaker, when the
house is burning down and you need the fire department, you don't ask
if they followed proper procedure to get somebody out there to put out
the fire.
My brother had liver cancer at the age of 44. He had tried every
conventional therapy known to modern medicine, and it wasn't working.
Now, he had a brother, myself, who was a subcommittee chairman of the
committee of jurisdiction over the FDA. I contacted the FDA, and we got
him in a special protocol for an investigational drug that was under
approval. It wasn't approved. And the doctors and the people at the FDA
told my brother and his family: If it works, it is going to really help
you. But if it doesn't, you are going to die sooner.
Well, he was going to die anyway, Mr. Speaker. So he signed the
informed consent and he took the drug and it didn't work, but he had
that last shot. Now, I don't know what this debate about false hope is.
When you have no hope, perhaps false hope is better than none at all.
All this bill does is let people who have no other hope for
conventional therapy, if a drug has at least passed stage one at the
FDA approval process, and their doctor thinks it will help them, if
they give an informed consent, they can try it.
Now, my friends on the Democratic side are correct that, most of the
time under the existing protocol, the FDA approves it without a
problem. But why should the FDA approve it if you are about to die
anyway? That is what this bill does. By the way, it passed the Senate
with unanimous consent. Now, that is a miracle in itself.
Let's pass it here in the House and give hope a chance for these
patients who are terminally ill and have no hope at all today.
Mr. PALLONE. Mr. Speaker, I yield 2 minutes to the gentlewoman from
California (Ms. Matsui).
Ms. MATSUI. Mr. Speaker, I thank the ranking member for yielding to
me.
Mr. Speaker, I rise in opposition to this proposed right-to-try
legislation. This bill offers patients false hope. It proposes a
pathway to experimental drugs that offers absolutely no guarantee of
access, while stripping patients of any legal or financial recourse,
and places clinical trials at risk.
{time} 1800
Last week, I am sure like everyone else, I heard from many
constituents on behalf of their families and communities with
devastating diseases, like multiple sclerosis.
When a family member is faced with a devastating diagnosis, you would
do anything and try anything to improve their quality of life. I know.
I have been there with family members in such heartbreaking situations.
But this bill would not necessarily make it easier to get experimental
treatments and it would definitely make it harder for patients in the
future to get treatments. We need clinical trials to ensure drugs are
safe and effective and to find real cures and treatments for these
patients.
Because this bill would be dangerous for patients both today and in
the future, many disease groups oppose the bill, including the National
Organization for Rare Disorders, the American Cancer Society, the
Cystic Fibrosis Foundation, and more.
Rushing this bill without proper bipartisan oversight places the
American people in the way of real harm. Rescinding FDA oversight on
unproven therapies is a perilous proposition.
Mr. Speaker, I urge my colleagues to oppose this bill.
Mr. WALDEN. Mr. Speaker, I yield myself 5 seconds.
Mr. Speaker, the last two speakers from California and Texas, two of
our biggest States, a grand total of two legislators voted ``no.''
Otherwise, it was unanimous in both those States to do what we are
doing here today.
[[Page H1525]]
Mr. Speaker, I yield 2 minutes to the gentleman from Arizona (Mr.
Biggs), an incredible advocate of this legislation.
Mr. BIGGS. Mr. Speaker, I thank Chairman Walden for yielding. I am
grateful for the work he has done on this. I am also grateful to my
friends, Representatives Fitzpatrick and Griffith, as well as Senator
Johnson, for their advocacy here.
I don't want to get this crucial point lost: it is not us; it is the
courageous patients and their friends and their families who deserve
the most recognition about how far we have come to get this bill
passed. Today is for them, not for us.
Thirty-eight States, soon to be 39 States, have passed this bill.
That is enough to amend the U.S. Constitution, but here we stand
because some have come and said we shouldn't give people false hope.
There is no such thing as false hope. You either have hope or you
have no hope. In this instance, this bill gives tens of thousands,
perhaps hundreds of thousands, or millions even, the hope that they can
avail themselves of medication that might prolong their life or maybe
even be a cure. These people who have advocated are fighters.
I hear about patient groups who oppose this, yet the States, our
employers, they approve this. Every day, Laura McLinn, the mother of
Jordan McLinn, receives countless emails from people similarly
situated, saying: We need to pass the Right to Try Act. I need that
right to try.
I am told: Oh, well, we take care of 1,500 a year.
Mr. Speaker, 1,500 a year, when there are literally tens of thousands
of people who need their opportunity.
We are not mandating even. We are providing an opportunity. We are
providing an option both for the patient and even the pharmaceutical
company.
Now, I heard in the opening statement from my friend across the aisle
that we are not compelling them to do it.
Would he feel more comfortable if we compelled pharmaceutical
companies to provide those potential lifesaving medications?
We need to recognize that this bill is not for us in this Chamber. It
is for Matt Bellina, Jordan McLinn, and Laura McLinn. It is for those
who are similarly situated.
We have waited long enough. Let's get this done.
Mr. PALLONE. Mr. Speaker, I yield 3 minutes to the gentlewoman from
Illinois (Ms. Schakowsky).
Ms. SCHAKOWSKY. Mr. Speaker, I rise in opposition to H.R. 5247
because it actually creates a dangerous back door around the Food and
Drug Administration approval process and it ignores that there is a
safe pathway for terminally ill patients to get the treatment that they
need.
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. It is not patient friendly. That is why 78 patients and
doctor groups are all opposed to this legislation, like the American
Cancer Society, the National Brain Tumor Society, the Leukemia and
Lymphoma Society, and the Vietnam Veterans of America.
Mr. Speaker, I include in the Record this 5-page list of the opposing
groups.
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. We can't trust manufacturers to act
as a gatekeeper.
The important thing to know is there is already a safe process for
terminally ill patients to access experimental treatments. Under the
Expanded Access Program, 99 percent of applications are approved, and
they are done in a speedy way.
This process is not merely a rubber stamp. The FDA plays a vital role
in ensuring these experimental treatments are safe.
Even more important, in 19 States that have passed right-to-try laws,
patients using an investigational drug can lose their hospice care; and
in 6 States, they can be denied home healthcare. These are the very
people who depend on hospice and home care, and they could lose those
services.
This is not a humane, patient-centered bill for people who are facing
death. It is just a dangerous pathway for bad actors to exist.
Let's go with the positive ability right now that we have. Ninety-
nine percent of those desperate people looking for hope will get it
from the Food and Drug Administration. So I urge my colleagues to
oppose H.R. 5247.
Mr. WALDEN. Mr. Speaker, when Illinois took this up, they approved it
169-1 in their assembly.
Mr. Speaker, I yield as much time as he may consume to the gentleman
from Virginia (Mr. Griffith).
Mr. GRIFFITH. Mr. Speaker, I thank Chairman Walden for yielding.
Mr. Speaker, I have heard people say that this bill gives folks a
false hope. There is no false hope.
They know it is a Hail Mary pass. They know it is unlikely to
succeed, but they are willing to make the decision and the choice to
take that chance.
I have heard that patients will be at risk, that they lose their
safeguards. They have received a terminal diagnosis. They know they are
at risk. They don't care about safeguards. They want to fight for life.
They know they have that terminal disease or diagnosis and they may
lose a few weeks, as we heard from my colleague, but they may gain
years, and they are willing to take that risk.
[[Page H1526]]
Mr. Speaker, I have to tell you, if I had a terminal diagnosis, I
would even consider injecting monkey urine if I thought it would give
me a few more months or a few more years with my children, who are
currently 18, 12, and 10. Others may choose not to try something. They
may not want the right to try. They may not want to try the Hail Mary
pass, but they should have the choice. They should have the right to
try.
Mr. PALLONE. Mr. Speaker, I yield myself such time as I may consume.
Mr. Speaker, as I have said, I have great concerns that H.R. 5247
would expose our most vulnerable and desperate patients to unnecessary
risk.
Supporters of this legislation have argued that those patients who
are suffering from a terminal illness deserve the right to take their
health and treatment into their own hands, as they are faced without
any other treatment options. Some have even asked: What risk could be
worse than the risk of death?
As Arthur Caplan, a bioethicist from NYU, has pointed out: ``There
are things worse than death; being made to die faster, being made to
die more miserably.''
These are all very real scenarios that patients could be exposed to
under the misleading and ill-conceived right-to-try pathway.
As I stated before, while the FDA approved 99 percent of the requests
it received, of those, they revised 11 percent in order to protect
patients. If this bill becomes law, the FDA no longer will have the
opportunity to make those revisions and to protect vulnerable patients.
We must protect patients from bad actors or from dangerous treatments
that might make their lives worse. That is why more than 100
organizations have written in opposition to this legislation, including
83 patient organizations like the National Organization for Rare
Disorders, the Friends of Cancer Research, the American Cancer Society,
Cancer Action Network.
In a letter to the Speaker and the Democratic leader, the patient
organizations noted that ``the alternative pathway in the latest
version of the legislation is still less safe for our patients than the
current expanded access process'' that the FDA uses.
Dr. Ellen Sigal, the chair and founder of Friends of Cancer Research,
said: ``In its current form, the proposed legislation does nothing for
patients other than provide false hope by allowing them to request a
drug with no evidence of efficacy they may never receive and, should
they receive it, may do more harm than good.''
So I think we should all be concerned about protecting patients.
Rather than rushing this bill through today, I would urge my colleagues
to oppose this legislation and to come back to the table to find a
solution that will streamline Expanded Access Programs while protecting
patients from unnecessary harm.
Mr. Speaker, I reserve the balance of my time.
Mr. WALDEN. Mr. Speaker, I yield 2 minutes to the gentleman from
Texas (Mr. Burgess), the chair of our Health Subcommittee. Texas voted
unanimously for the Right to Try Act.
Mr. BURGESS. Mr. Speaker, I thank the chairman for yielding.
Mr. Speaker, just a little over a month ago, President Trump stood
here at this podium behind me and told us: ``People who are terminally
ill should not have to go from country to country to seek a cure.''
Along with President Trump, I want to give patients a chance right
here at home.
A little over a year ago, this House passed the 21st Century Cures
Act, made unprecedented acceleration of discoveries. Thanks to our
researchers and our academic institutions, and those working in the
pharmaceutical and medical device companies, Americans have access to
more and more innovative treatments. However, I continue to hear from
patients with serious life-threatening conditions, including my
constituents in north Texas, who are frustrated with what they see as
regulatory barriers from trying and experimenting with new therapies
when everything else has failed.
When potentially lifesaving treatments exist but remain unavailable
to patients, we have an opportunity to move past what has long been a
dilemma towards delivering a hopeful message.
Since 2014, 38 States, including Texas, have passed a version of
right-to-try laws.
I am pleased that the House of Representatives is considering right-
to-try legislation that gives patients a chance at life by improving
access to experimental treatments.
Mr. Speaker, a lot of people deserve thanks for getting this bill to
us today, but, in particular, I want to thank the President of the
United States, President Trump, and Vice President Pence for their
leadership in this effort.
Mr. Speaker, I urge my fellow Members to support this bill.
Mr. PALLONE. Mr. Speaker, I yield myself such time as I may consume.
Mr. Speaker, there are a lot of reasons to oppose this bill today,
and I have given a number of them, but the primary reason being the
need to continue protecting patients by ensuring that the FDA remains a
part of the process.
While we are speaking about process, I have to also oppose this
legislation based on the inappropriate way the Republican majority is
bringing this bill to the floor. Bills considered under suspension have
traditionally gone through the committee process with overwhelming
bipartisan support, and neither of those things is the case with this
bill.
It was introduced today.
Does the majority really believe they are giving Members the
appropriate time to view this bill when it was introduced at 2 p.m.?
Patient access and patient safety should be shared goals among
Democrats and Republicans, goals that could be achieved if this
legislation was not being rushed to the floor under an arbitrary
deadline.
Legislation such as this, that carries such great risk of patient
harm, should be considered carefully, with attention paid to the
unintended consequences that could follow.
Mr. Speaker, I would urge my colleagues to oppose this unnecessary
and risky legislation, and to return to the regular order of the
committee to consider legislation that would protect both patients from
harm and the FDA from the weakening of the agency's role in our drug
approval process.
We should not be voting on a bill of this consequence that was
introduced this afternoon.
Mr. Speaker, I reserve the balance of my time.
{time} 1815
Mr. WALDEN. I yield 2 minutes to the gentleman from Georgia (Mr.
Carter), a distinguished member of our committee and a pharmacist by
training and trade.
Mr. CARTER of Georgia. Mr. Speaker, I rise today in support of the
Right to Try Act because this legislation will improve access to
potentially lifesaving treatments for patients with terminal diseases
or conditions.
Currently, patients can only receive drugs that are undergoing FDA
review through clinical trials, through compassionate use, or expanding
access. They access these unapproved treatments exclusively through the
FDA but not through the drug sponsor. This critical legislation would
establish informed consent for patients to access unapproved drugs that
could save their lives.
This bill still guards patients from manufacturers misbranding or
mislabeling drugs and specifies that any unapproved drug used in the
alternative pathway must have an active application and is not the
subject of a clinical hold.
I thank my good friend Chairman Burgess and the rest of my colleagues
on the committee for moving this legislation forward and working with
the administration and stakeholders on all sides of these issues. This
is a great step forward towards ensuring our patients get to take
advantage of the incredible pharmaceutical therapies that our
manufacturers are known for.
I applaud the Energy and Commerce Committee for their work in moving
this legislation forward, and I urge my colleagues to support this
legislation.
Mr. PALLONE. Mr. Speaker, may I ask the gentleman how many speakers
he has left.
Mr. WALDEN. Mr. Speaker, we have two, I believe, left.
Mr. PALLONE. Mr. Speaker, I yield myself such time as I may consume.
[[Page H1527]]
Mr. Speaker, I just want to talk about two other aspects of this bill
that I haven't so far. One is the fact that States have actually
implemented right-to-try laws that have done little to expand access to
investigational treatment. Although 17 States and the District of
Columbia have enacted right-to-try laws, 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 right to request
the treatment from the company. State right-to-try laws do not address
the fundamental barriers of cost and accompanying restrictions.
Neither the FDA nor States require insurers or pharmaceutical
companies to cover the cost or reduce the cost of these expensive
treatments. Instead, these laws put patients at a higher risk by
prohibiting or weakening FDA oversight of investigational treatments.
With regard to clinical trials, the legislation could also expose
patients to unnecessary risk by allowing access to investigational
drugs that have only completed a phase I clinical trial. Phase I trials
are extremely small trials, in the range of 20 to 80 patients, and are
used primarily to determine toxicity. They do not determine
effectiveness or potential side effects. Patients could suffer from
harmful side effects or delay enrolling in a clinical trial program for
a treatment that actually has evidence of efficacy for their disease or
condition.
Finally, the bill would weaken the FDA's ability to oversee the
adverse events or other clinical outcomes from the use of
investigational drugs and provide broad liability protections for
manufacturers, leaving patients with no recourse in the case of an
adverse effect.
I just wanted to mention those.
Mr. Speaker, I reserve the balance of my time.
Mr. WALDEN. Mr. Speaker, I yield 2 minutes to the gentleman from
Georgia (Mr. Allen).
Mr. ALLEN. Mr. Speaker, I rise today to urge my colleagues to join me
in supporting the Right to Try Act.
When those we hold dearest are diagnosed as terminally ill, the last
thing we want to hear is that all treatment options have been
exhausted. This is why I have been a longtime supporter of the Right to
Try Act. Currently, 38 States have already passed right-to-try
legislation to assist vulnerable patients, including my home State of
Georgia.
By allowing terminally ill patients the access to unapproved drugs
and therapies, we are giving them a fighting chance for their God-given
right to life. Although these drugs cannot guarantee a road to
recovery, they can provide a better alternative in many hopeless
situations and pave the way for more scientific breakthroughs.
Congress should keep breaking down regulatory barriers. Like the
bill's name says, patients have a right to try. All Americans should
have the right to choose.
Mr. Speaker, I thank the Energy and Commerce Committee for passing
this important legislation out of committee, and I urge my colleagues
to join me in supporting this bill on the House floor.
How in the world could anyone oppose the right to choose life?
Mr. WALDEN. Mr. Speaker, may I inquire as to how much time remains
for each side.
The SPEAKER pro tempore (Mr. Weber of Texas). The gentleman from
Oregon has 3 minutes remaining. The gentleman from New Jersey has 1
minute remaining.
Mr. PALLONE. Mr. Speaker, I yield myself such time as I may consume.
Mr. Speaker, I just want to conclude, if I could, in opposition to
this bill by quoting some of the former FDA Commissioners.
This is from Dr. Margaret Hamburg, who said:
I am deeply concerned by the draft legislation
being considered to remove the FDA from the proposals
around right to try. Excluding the FDA will not benefit
those patients and would be a mistake. There is no need to
create a new potentially dangerous paradigm by passing
this legislation which does not address the real issues at
hand and could have unintended negative consequences,
leading to a possible impediment of the development and
approval of safe and effective therapies.
And then, finally, is the former FDA Principal Deputy Commissioner,
Joshua Sharfstein, who said:
FDA review allows doctors and patients to tell the
difference between a medication that works and one that does
not. Evidence also orients the pharmaceutical market towards
developing products that produce meaningful benefits for
patients instead of just hope. Undermining FDA review by
giving a right to patients to try anything at any time will
leave more patients in desperate situations with fewer
options and less understanding of what could really make a
difference.
Again, Mr. Speaker, I would urge opposition to this legislation.
Mr. Speaker, I yield back the balance of my time.
Mr. WALDEN. Mr. Speaker, I yield myself such time as I may consume.
Mr. Speaker, when the Energy and Commerce Committee took up this
issue in its broadest form, we heard from the FDA Commissioner, we
heard from patients, we heard from family members, and what we heard
was that there are barriers in States that preclude these State laws
from working.
That is what the Government Accountability Office told us. They
identified two issues--liability and use of outcomes--as the two
barriers as to why these laws passed in 38, soon to be 39, States. And
in many cases--most cases, I would say--these laws have passed
unanimously, with Republicans and Democrats back home supporting them,
including in my own State. I think it was unanimous in both the house
and the senate, all controlled by Democrats, in Oregon.
We have listened to our constituents; we have observed what has
happened in our States--great laboratories--and we are acting here
today to allow those who have been given this wretched, wretched
prescription that their life is about to end to have a chance and a
choice. That is what we are doing today. We are overcoming the barriers
that exist at the State level. We are doing it in a reasonable and
thoughtful way that protects patient safety and creates this new
alternative pathway for them.
This is important legislation. It is not often in this body we get
this opportunity to make this kind of a change and provide chance and
hope for those who see their loved ones dying before their eyes.
I met with Jordan McLinn and his mother, Laura, earlier today. They
have been incredible advocates for this cause. And they had just come
from a meeting with Vice President Pence, who, with the President, has
been an extraordinary supporter of this effort.
From his Bible, Jordan showed me the Parable of the Lost Sheep, which
is one of his favorites. It is a parable he had shared with the Vice
President.
That Parable of the Lost Sheep tells us that not a single sheep
should be lost, that the shepherd cares about them all. That same
sentiment is what brings us here to right to try today.
Every opportunity to save a life matters, and every patient deserves
that right to try. That is the legislation before us today, Mr.
Speaker. It is well conceived, it is well thought out, and it will make
a difference in saving lives.
I encourage my colleagues to vote ``yes'' and pass this legislation
and give people a right to try.
Mr. Speaker, I yield back the balance of my time.
The SPEAKER pro tempore. The question is on the motion offered by the
gentleman from Oregon (Mr. Walden) that the House suspend the rules and
pass the bill, H.R. 5247.
The question was taken.
The SPEAKER pro tempore. In the opinion of the Chair, two-thirds
being in the affirmative, the ayes 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 motion will be postponed.
[[Page H1528]]
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