RARE Daily

Legislation to Reauthorize Rare Pediatric Disease Designation Introduced

February 20, 2024

Rare Daily Staff

A bi-partisan group of Congressional representatives has introduced legislation to reauthorize the Rare Pediatric Disease Designation, a tool that has provided incentives and critical funding to rare disease drug developers.

Last week Representatives Gus Bilirakis (R-Florida), Anna Eshoo (D-California), Michael McCaul (R-TX), Lori Trahan (D-Massachusetts), Michael Burgess (R-Texas), and Nanette Barragán (D-California) introduced the Creating Hope Reauthorization Act of 2024.  The legislation would reauthorize the Creating Hope Act of 2011, which expanded the FDA priority review voucher program to incentivize pharmaceutical companies to develop new drugs for children with rare cancers and other rare pediatric diseases.

“This bill provides incentives for research and development by providing pediatric cancer drug developers with vouchers to speed up reviews by the Food and Drug Administration of new drug products,” said Rep. Eshoo. “The Creating Hope Reauthorization Act will save children’s lives.”

Children make up as much as half of those living with rare diseases, yet their treatment options are extremely limited. Treatments intended for adults are often too harsh for children, leaving them with life-altering complications even after their disease is cured. The Creating Hope Act works to solve this problem by expanding the cost-neutral Food and Drug Administration priority review voucher program, which allows pharmaceutical companies to expedite FDA review of more profitable drugs in return for developing treatments for rare pediatric diseases.

Since the bill’s passage in 2011, 46 PRVs have been awarded for 35 different rare pediatric diseases.  The Creating Hope Reauthorization Act of 2024 would reauthorize the Creating Hope Act—which expires this September—for another four years.

Though the Congressional reps stressed the power of the program as an incentive to drug developers to pursue therapies for rare pediatric disease populations, the legislation has been a source of funding to drive the development of new therapies. That’s because the priority review vouchers are transferable, and many of the companies that have gained one have sold them for prices that are usually around $100 million each. At a time when rare disease drug developers have faced challenges raising money, they have proved to be a welcome source of non-dilutive funding.

Groups like the National Organization for Rare Disorders and the Everylife Foundation for Rare Diseases are supporting the legislation and called for its passage.

“The creation of the Pediatric Priority Review Voucher injected hope and innovation incentives into the pediatric drug development pipeline. We must continue to advance solutions for the estimated 30 million Americans living with rare diseases,” said Jamie Sullivan, vice president of policy for EveryLife Foundation. “This legislation ensures the sustainability of a critical incentive driving progress in drug development for devastating rare diseases that predominantly affect children, all without burdening taxpayers.”

Photo: Rep. Anna Eshoo (D-California)

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