Creating Hope Reauthorization Act

Floor Speech

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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''.

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Mr. PALLONE. 4439.

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.

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Mr. PALLONE. I have no additional speakers, and I reserve the balance of my time, Mr. Speaker.
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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, 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.

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