PORTAL Congressional Testimony on Rare Pediatric Disease Priority Review Voucher Program

Neeraj Patel

Today, PORTAL Director Aaron Kesselheim testified as a witness before the Subcommittee on Health of the House Committee on Energy and Commerce during a hearing on "Improving Access to Care: Legislation to Reauthorize Key Public Health Programs." Dr. Kesselheim urged Congress against passing H.R. 4439, which would permanently reauthorize the rare pediatric disease priority review voucher program. He outlined shortfalls of the program and pointed to alternative approaches that Congress can take to improve availability and access to drugs for rare pediatric diseases.

In describing the shortfalls of the program, Dr. Kesselheim's testimony references a February 2019 Health Affairs study by PORTAL researchers and colleagues. He writes, "My colleagues and I at Harvard Medical School showed no increases after 2012 in the rate of new drugs entering clinical trials for rare pediatric diseases when compared to drugs for rare adult diseases, which did not earn a voucher."

Dr. Kesselheim also discussed the overstated and diminishing economic value of the voucher incentive, the potentially dangerous public health risks associated with accelerated review of these types of products, and strains that the program has put on the FDA.

Moreover, Dr. Kesselheim outlined alternative approaches that can be taken to increase development of and access to drugs for rare pediatric disease: "For example, Congress could provide greater up-front public funding or tax credits for research into rare pediatric diseases. Another approach would be to provide greater support for late-stage development through public-private partnerships with non-profit organizations. Naturally, such partnerships should include guarantees about affordable access to the rare pediatric disease drugs that emerge because of the public’s involvement in reducing the risks and costs of research and development."

Dr. Kesselheim's full written testimony can be found here.  Full video of the hearing is available below.