WASHINGTON — Lawmakers advanced the reauthorization of an FDA program designed to stimulate the development of treatments for pediatric rare diseases, despite some opposition from Democrat lawmakers.
During the markup held on Thursday by the House Energy and Commerce Subcommittee on Health, lawmakers debated the reauthorization of the rare pediatric voucher program, which empowers the FDA to dole out vouchers to sponsors of pediatric rare disease drugs. Drugmakers can either use the voucher for a subsequent application or sell it to another drug developer, with many fetching about $100 million.
The program will sunset later this fall if it doesn’t get reauthorized.
Rare disease advocates support the program as a way to incentivize sponsors to create rare disease drugs. But some opponents are worried that the voucher program rewards bigger companies that would otherwise have the resources to develop the drugs.
E&C ranking member Frank Pallone (D-NJ) voiced some opposition to the bill, asserting that lawmakers haven’t scrutinized whether the bill is effective at improving access to rare disease treatments. He underscored that the program also needs to be evaluated to ensure that it doesn’t unduly tax the FDA’s resources.
“Unfortunately, the Republicans refuse to meet us halfway or even part of the way in my opinion,” he said.
Stakeholders were bracing for bumps in the reauthorization process earlier this year, saying they anticipated holdups from Senate HELP Committee Chair Bernie Sanders (I-VT) and others who were skeptical of rewarding drug makers with vouchers.
Karin HoelzerKarin Hoelzer, the director of policy and regulatory affairs at the National Organization for Rare Disorders and a proponent of the PRV program, told Endpoints News in an interview that she was pleased to see the panel come together to support rare disease despite some differences in opinion.
She pointed to a white paper authored this spring showing that the PRV program has resulted in new drugs in the past few years.
“From our perspective, the data’s really clear,” she said.
Lawmakers also advanced the Give Kids a Chance Act, sponsored by Reps. Anna Eshoo (D-CA) and Michael McCaul (R-TX), which would authorize the FDA to direct companies to study combinations of cancer drugs and therapies in pediatric populations.
The subcommittee also advanced the Retaining Access and Restoring Exclusivity Act, sponsored by Reps. Doris Matsui (D-CA) and Gus Bilirakis (R-FL), which would clarify that orphan drug exclusivity only applies for specific approved use of rare disease drugs.
In January 2023, the FDA refused to change its interpretation of when to award the seven years of orphan exclusivity granted to new rare disease drugs, following a controversial court loss that left many rare disease drugmakers in limbo.
“Having that clarity is really important to provide predictability to the drug development process,” Hoelzer said.
Next up, the bills will be taken up by the full Energy and Commerce Committee before they can move to the House floor.