[Congressional Record Volume 166, Number 169 (Tuesday, September 29, 2020)]
[House]
[Pages H5023-H5025]
From the Congressional Record Online through the Government Publishing Office [www.gpo.gov]
{time} 1630
CREATING HOPE REAUTHORIZATION ACT
Mr. PALLONE. Mr. Speaker, I move to suspend the rules and pass the
bill (H.R. 4439) to amend the Federal Food, Drug, and Cosmetic Act to
make permanent the authority of the Secretary of Health and Human
Services to issue priority review vouchers to encourage treatments for
rare pediatric diseases, as amended.
The Clerk read the title of the bill.
The text of the bill is as follows:
H.R. 4439
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 ``Creating Hope
Reauthorization Act''.
SEC. 2. EXTENSION OF AUTHORITY TO ISSUE PRIORITY REVIEW
VOUCHERS TO ENCOURAGE TREATMENTS FOR RARE
PEDIATRIC DISEASES.
Section 529(b)(5) of the Federal Food, Drug, and Cosmetic
Act (21 U.S.C. 360ff(b)(5)) is amended--
(1) by striking ``December 11, 2020'' each place it appears
and inserting ``September 30, 2024''; and
(2) in subparagraph (B), by striking ``December 11, 2022''
and inserting ``September 30, 2026''.
The SPEAKER pro tempore. Pursuant to the rule, the gentleman from New
Jersey (Mr. Pallone) and the gentleman from Oregon (Mr. Walden) each
will control 20 minutes.
The Chair recognizes the gentleman from New Jersey.
General Leave
Mr. PALLONE. Mr. Speaker, I ask unanimous consent that all Members
may have 5 legislative days in which to revise and extend their remarks
and include extraneous material on H.R. 4439.
The SPEAKER pro tempore. Is there objection to the request of the
gentleman from New Jersey?
There was no objection.
Mr. PALLONE. Mr. Speaker, I yield myself such time as I may consume.
Mr. Speaker, H.R. 4439 would extend the pediatric rare disease
priority review voucher program at the Food and Drug Administration.
This priority review voucher, or PRV, was originally created in 2012
with the intent to create an incentive for drug manufacturers to
develop therapies for rare pediatric diseases that affect neonates,
infants, children, and adolescents.
The program requires FDA to award a PRV to the sponsor of an
application that receives approval as a drug or biologic to treat a
rare pediatric disease. Since the program's creation, 22 of these PRVs
have been awarded, with five awards in 2019.
Now, I have long been a supporter of increased research and
development of treatments for rare diseases, and I am proud that our
committee was able to come to consensus on a reasonable extension of
this program. Nevertheless, some observers of this program have shown
our committee evidence that the PRV program has not provided the
incentive value intended by Congress when it was first enacted.
This program was supposed to incentivize new development of pediatric
products that would not otherwise have occurred. However, the
Government Accountability Office reviewed the program and concluded
that the agency could not find definitive evidence that the program is
incentivizing pediatric drug development. Additionally, FDA has said
that PRVs drain agency resources away from the agency's public health
mission and have a negative impact on the morale of agency staff.
For these reasons, I could not support a permanent reauthorization of
the program. But I recognize that many pharmaceutical developers have
argued that the PRV provides an incentive for drug development, with
one going so far as to say that the PRV was a pivotal consideration for
making investments. I worked with Representatives Butterfield, the
bill's sponsor, and Ranking Member Walden, as well, to support a
reauthorization of the program for 4 years.
Mr. Speaker, the Energy and Commerce Committee will continue to
provide oversight to examine the effectiveness of this program and its
effect on FDA resources. The committee will also carefully scrutinize
it with hopes that it serves its intended purpose and leads to new
treatments and cures for rare pediatric diseases.
With this hope, Mr. Speaker, I ask Members to support this bipartisan
bill. I urge the Senate to swiftly take action on H.R. 4439, and I
reserve the balance of my time.
Mr. WALDEN. Mr. Speaker, I yield myself such time as I may consume.
Mr. Speaker, I rise today in support of H.R. 4439, the Creating Hope
Reauthorization Act, introduced by our colleagues, Representative
Butterfield and Representative McCaul. This bill extends the pediatric
priority review voucher program for an additional 4 years.
This program, which has had bipartisan support since it was first
created in 2012 with the passage of the Food and Drug Administration
Safety and Innovation Act, aims to incentivize the development of
therapies to treat rare pediatric diseases.
The pediatric PRV program has already proven successful in
encouraging innovation. In fact, 22 therapies have been approved for
the treatment of 18 rare pediatric diseases since its inception.
However, we still have a long way to go. Nearly 95 percent of all rare
diseases do not have an FDA-approved treatment, leaving many patients
with no options.
This long-term reauthorization of the program will provide certainty
to those currently developing or considering investment in innovative
therapies to treat rare pediatric diseases.
Bipartisan bills that encourage biomedical innovation like the one we
are considering today mean continued hope for children and their
families that, one day, there will be a treatment and that there will
be a cure.
So, Mr. Speaker, I urge my colleagues to support this important
legislation, and I reserve the balance of my time.
Mr. PALLONE. I have no additional speakers, and I reserve the balance
of my time, Mr. Speaker.
Mr. WALDEN. Mr. Speaker, I yield 3 minutes to the gentleman from
Florida (Mr. Bilirakis).
Mr. BILIRAKIS. Mr. Speaker, I rise today in support of H.R. 4439, the
Creating Hope Reauthorization Act.
Tragically, pediatric cancer remains the number one disease that
leads to the death of American children. While survival rates have
improved for some types of pediatric cancers, thousands of children are
lost to cancer each year, and many more encounter life-threatening
complications relating to harsh chemotherapies.
Children have significantly fewer treatment options than adults, Mr.
Speaker, and oftentimes must rely on treatment regimens developed for
adults because pediatric-specific treatments simply do not exist.
Unfortunately, as the popular healthcare adage goes, children are not
little adults.
Despite their significant need, pharmaceutical companies have had
trouble developing treatments for pediatric cancer and rare diseases
because of the small population and high cost of bringing these
specific treatments to market.
FDA's priority review voucher program has proven to be a boon to
incentivizing the development of therapies to treat rare pediatric
diseases. While progress has been made in the development of pediatric
therapies--in fact, 22 therapies have been approved
[[Page H5024]]
for the treatment of 18 rare pediatric diseases since 2012, which is
really great--there is still more work to be done however. Nearly 95
percent of all rare diseases do not have an FDA-approved treatment,
leaving many patients without options.
As co-chair of the Rare Disease Caucus, I sincerely appreciate the
good work from Ranking Member McCaul, of course, the ranking member of
the Committee on Energy and Commerce, and the chairman of the Committee
on Energy and Commerce for bringing this bill forward, and also
Congressman Butterfield.
So, Mr. Speaker, I urge my colleagues to join us in passing the
Creating Hope Reauthorization Act, which will extend this vital
innovation lifeline for rare pediatric treatments and cures at FDA.
Mr. PALLONE. Mr. Speaker, I reserve the balance of my time.
Mr. WALDEN. Mr. Speaker, I yield such time as he may consume to the
gentleman from Texas (Mr. McCaul), who is a serious leader on this
issue and has been for a long time.
Mr. McCAUL. Mr. Speaker, let me thank the ranking member, Mr. Walden,
from Oregon for his steadfast support on this issue. I want to thank
the chairman for getting to this point where we are back on the floor,
and the Energy and Commerce Committee, in general, for its support of
this bill 4 years ago and, now, a 4-year reauthorization.
As the founder and co-chair of the Childhood Cancer Caucus, I have
met with countless cancer patients and advocates who have asked me for
one thing: hope. They want meaningful hope, something realistic and
something tangible, better cures, safer treatment, and more research
dollars.
So, alongside my colleagues, Mr. Butterfield, Mr. Kelly, and Ms.
Speier, we have used the Childhood Cancer Caucus to produce that hope.
This caucus has yielded tangible results for the hundreds of thousands
of patients, advocates, and parents who were just hoping for better
results.
When we first started this caucus over a decade ago, there were only
two new drugs developed to specifically treat pediatric cancer in the
prior 20 years. I would talk to these children and their parents, and
they would tell me that the cancer just had no treatment, or if there
was a treatment, it was of a drug developed in the 1960s or older.
Many of these drugs were meant to be used by adults, and their intent
was to kill the cancer before it killed the person. That is why Mr.
Butterfield and I worked together to pass the original Creating Hope
Act in 2011, to try to create a market to get more pediatric cancer and
rare disease drugs approved in this country.
Specifically, our bill created a voucher award within the FDA where
companies that developed rare pediatric disease drugs have priority on
future products that could save kids' lives.
I am happy to report that this program has been very successful.
Since 2014 when the first voucher was issued, 22 new drugs for rare
diseases in kids have been approved by the FDA, including two drugs for
childhood cancers, with more expected on the way.
I have seen, firsthand, the wonders of this program.
In September of 2013, a constituent of mine named Rex Ryan was
diagnosed with stage 4 neuroblastoma at just 18 months old. After
exhausting all options, Rex was blessed to have received a novel new
drug that was developed and approved in part because of the incentive
of the Creating Hope Act. Rex is now cancer free and a healthy third
grader saved by the drug developed because of this program.
It is for Rex and all the other children who have been saved because
of this program and will be saved because of this program that I stand
here today in support of the bill.
I think, most remarkably, of a dear friend of mine, Dr. Allison from
MD Anderson Cancer Center, who had a childhood cancer clinical trial
going and was able to use the voucher program to obtain FDA approval
for a treatment now called immunotherapy. Immunotherapy is a
breakthrough in cancer treatment not just for children but for all
adults. He got the Nobel Prize in medicine for it.
While this bill before us today reauthorizes the program for another
4 years, I would hope to see, when we come back again, that this
program will be made permanent. So I look forward to working with my
colleagues in the Congress to achieve that goal.
I appreciate the support from the advocates and members of my team,
especially my outgoing legislative director, Thomas Rice, who spent
countless hours meeting with the advocates, the brains behind the
operation, and Nancy Goodman, who gave us all these great ideas to
develop four major pieces of legislation. I can't thank Thomas enough
for all his great work.
Together, we can and will defeat childhood cancer. It is vital that
we keep this program alive so that we keep hope alive for the hundreds
of thousands of children and their families who are waiting for the big
break of a treatment.
Mr. WALDEN. Mr. Speaker, I want to thank my colleague from Texas for
his great leadership on this issue. I encourage our colleagues to vote
for this bill, and I yield back the balance of my time.
Mr. PALLONE. Mr. Speaker, I also urge support for the legislation,
and I yield back the balance of my time.
Ms. JACKSON LEE. Madam Speaker, I rise in support of H.R. 4439, the
``Creating Hope Reauthorization Act,'' which amends the Federal Food,
Drug, and Cosmetic Act to make permanent the authority of the Secretary
of Health and Human Services to issue priority review vouchers to
encourage treatments for rare pediatric diseases.
Under Section 529 to the Federal Food, Drug, and Cosmetic Act (FD&C
Act), FDA will award priority review vouchers to sponsors of rare
pediatric disease product applications that meet certain criteria.
By definition, rare diseases do not affect much of the population, so
there is little financial gain for pharmaceutical companies.
The U.S. government has created the Orphan Drug Act and the rare
pediatric disease priority review voucher programs to add financial
incentives for development of medicines, which is making the
development of drugs for rare diseases more common.
However, many of the drugs for rare diseases are approved for adults.
Physicians sometimes have no choice but to prescribe offlabel use of
such drugs for children.
Drugs for rare diseases, whether approved for pediatric patients or
being used off label, often come in formats that are inappropriate for
children.
Most young children have trouble swallowing tablets or capsules, and
many children are sensitive to bitter-tasting drugs.
Sometimes, however, such formats are all that is available, which can
lead to difficulty in effectively treating young patients.
This is why H.R. 4439, the ``Creating Hope Reauthorization Act'' is
needed.
Under this program reinstated by this bill, a sponsor who receives an
approval for a drug or biologic for a ``rare pediatric disease'' may
qualify for a voucher that can be redeemed to receive a priority review
of a subsequent marketing application for a different product.
Without the passage of this bill, according to the current statutory
sunset provisions for the Rare Pediatric Disease Priority Review
Voucher Program, after September 30, 2020, which means the FDA may only
award a voucher for an approved rare pediatric disease product
application if the sponsor has rare pediatric disease designation for
the drug, and that designation was granted by September 30, 2020.
Further, without this bill after September 30, 2022, FDA may not
award any rare pediatric disease priority review vouchers.
Enactment of this bill will mean that requests for rare pediatric
disease designation submitted within two weeks of a request for fast
track designation or orphan drug designation are entitled to a 60-day
review.
Requests for rare pediatric disease designation not submitted with a
request for fast track designation or orphan drug designation are
reviewed in a timely manner, however, the 60-day response time does not
apply.
It is important for us to act today, because Friday, July 31, 2020,
is the last business day that is not less than 60 days prior to
September 30, 2020.
The Offices of Orphan Products Development and Pediatric.
Therapeutics will continue to review all rare pediatric disease
designation requests, but they cannot commit to providing a response to
requests received after July 31, 2020.
Pediatric rare diseases have received increased attention in recent
years due to greater public awareness, significantly improved
understanding and treatment.
Rare diseases can be difficult to diagnose in children, and many
physicians do not have the proper training and knowledge to diagnose
such diseases.
According to a 2014 Medscape Multispecialty article, the average
patient with a rare disease sees 7.3 physicians before diagnosis,
[[Page H5025]]
and 70 percent of physicians believe additional training in rare
diseases would be helpful.
A rare disease can spread and worsen during the lengthy time before
diagnosis and the start of the proper treatment.
I ask my colleagues to join me in supporting this bill.
Ms. ESHOO. Madam Speaker, I rise in support of H.R. 4439, the
Creating Hope Reauthorization Act. I'm proud to have advanced this
bipartisan bill through my Health Subcommittee and I'm pleased to
support it on the Floor today.
The Creating Hope Reauthorization Act sponsored by Representative
G.K. Butterfield helps children access pediatric cancer drugs.
Pediatric cancer is the number one disease killer of American
children, but pharmaceutical companies often avoid developing pediatric
cancer drugs because of the small market and the high risks associated
with studying and testing drugs for children.
The Creating Hope Reauthorization Act provides incentives for the
research and development of pediatric cancer drugs by providing the
developers with the valuable Priority Review Vouchers which allow the
recipient to speed up the FDA review of any one of its new drug
products.
Since its passage the GAO studied the pediatric priority review
vouchers and found that pharmaceutical developers said Priority Review
Vouchers were a factor in drug development decisions.
Dr. Crystal Mackall of the Stanford Center for Cancer Cell Therapy
said that, ``The voucher program has been remarkedly impactful for
childhood cancers. Before the program, I used to go with my hat in hand
to beg investors to consider a potential drug. Now people take a second
look and are interested in developing drugs. We're just getting started
on this new way of thinking about children's drugs. The voucher program
required a culture change around how to think of the pediatric drug
business model, which in the drug development world could take a
while.''
As Dr. Mackall said, this program seeks to shift decision-making
early in the lengthy drug development cycle. A lengthy reauthorization
of 4 years as offered in the AINS will be beneficial for this decision-
making and I urge my colleagues to support this bill.
The SPEAKER pro tempore. The question is on the motion offered by the
gentleman from New Jersey (Mr. Pallone) that the House suspend the
rules and pass the bill, H.R. 4439, as amended.
The question was taken; and (two-thirds being in the affirmative) the
rules were suspended and the bill, as amended, was passed.
The title of the bill was amended so as to read: ``A bill to amend
the Federal Food, Drug, and Cosmetic Act to extend the authority of the
Secretary of Health and Human Services to issue priority review
vouchers to encourage treatments for rare pediatric diseases.''.
A motion to reconsider was laid on the table.
____________________