[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.

                          ____________________