Effectiveness of FDA’s Pediatric Voucher Program Unclear, GAO Says

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By Bronwyn Mixter

March 3 — It's too early to determine whether the FDA's pediatric disease priority review voucher program stimulates development of drugs for rare pediatric diseases, a March 2 GAO report (GAO-16-319) said.

Under the Food and Drug Administration's rare pediatric disease priority review voucher program, a sponsor with approval for a new drug application (NDA) or biologics license application (BLA) for a rare pediatric disease may be eligible for a voucher that can be redeemed to get priority review for any subsequent marketing application.

These vouchers can be sold or transferred to another sponsor any number of times before the voucher is used, as long as the sponsor making the transfer hasn't yet submitted the application. The program was authorized by the Food and Drug Administration Safety and Innovation Act (FDASIA) of 2012, which included a provision requiring the GAO to study the program.

The GAO report said drugs usually take over 10 years to develop and there hasn't been enough time to determine whether the pediatric voucher program has been effective.

In addition to the report, the Congressional Rare Disease Caucus held a briefing on March 3 to discuss rare disease drug development. The caucus is a forum for members of Congress to collaborate on ideas and build support for legislation to improve the lives of people with rare diseases.

GAO Report's Findings

The report said that as of Dec. 31, 2015, there have been 11 requests for a pediatric voucher and of these, six have been awarded, two denied and three remain under review. The six drugs for which vouchers were awarded include one drug that treats a rare pediatric cancer and five drugs that treat rare metabolic diseases, the GAO said.

The GAO said four of the six awarded pediatric vouchers have been sold to other drug sponsors for prices ranging from $67.5 million to $350 million.

The report said that the pediatric voucher program is set to terminate on Oct. 1, and FDA officials told the GAO that they don't support the program's continuation because it “adversely affects the agency's ability to set its public health priorities by requiring FDA to provide priority review of new drug applications that would not otherwise qualify if they do not treat a serious condition or provide a significant improvement in safety or effectiveness.”

The FDA officials also said the program “strains the agency's resources.”

Repurposing Drugs

Rep. Gus Bilirakis (R-Fla.) said at the caucus briefing that he hopes senators will “quickly advance” a bill that would provide incentives for companies that repurpose previously-approved drugs for the treatment of rare diseases. The House passed the bill as part of the 21st Century Cures legislation in July 2015 .

The Senate bill, the Orphan Product Extensions Now Accelerating Cures (OPEN) Act, would provide an additional six months of market exclusivity to drugs that are repurposed and approved by the FDA for a new indication for a rare disease or condition. This would apply to currently approved drugs that are still under patent. The six-month extension would be in addition to other types of exclusivity, such as pediatric or qualified infectious disease product exclusivity.

“We must continue to encourage more research and innovation to find cures and treatments for rare diseases,” Bilirakis said. He also said he hopes the 21st Century Cures legislation will get to the president's desk by the end of 2016.

Sens. Orrin G. Hatch (R-Utah) and Amy Klobuchar (D-Minn.), two of the co-chairs of the caucus, expressed support for the OPEN Act. They introduced the senate version of the bill in May 2015 .

“Repurposing drugs is faster and less expensive” than developing a new drug, Klobuchar said.

FDA's Actions

Janet Woodcock, director of the FDA's Center for Drug Evaluation and Research (CDER), also spoke at the briefing, saying developers of rare disease drugs “need clarity” and “have to understand what the pathway to market is.”

Woodcock said the FDA recently announced the availability of $2 million in research grants to fund natural history studies in rare diseases. The aim of the grants “is to collect data on how specific rare diseases progress in individuals over time so that knowledge can inform and support product development and approval,” according to a Feb. 29 press release.

Additionally, Woodcock said patients groups have helped with research on rare diseases by doing things like pre-identifying patients with certain diseases who can participate in clinical trials.

Woodcock also said the FDA has been holding patient-focused drug development meetings. She said these meetings allow the FDA to hear about what it's like for a patient to live with a disease and what risks they're willing to tolerate in a potential treatment.

To contact the reporter on this story: Bronwyn Mixter in Washington at bmixter@bna.com

To contact the editor responsible for this story: Allison Gatrone at agatrone@bna.com