U.S. President Joe Biden has signed into law H.R. 9747, the “Continuing Appropriations and Extensions Act, 2025,” postponing the sunset date of the rare pediatric disease PRV program. The date by which a drug must be designated as a drug for a rare pediatric disease was changed from September 30, 2024, to December 20, 2024. The date by which such designated drug must be approved in order to receive a PRV remains September 30, 2026. This change is reflected on FDA’s webpage for the rare pediatric disease program, although the agency has noted that it may not be able to meet all review timelines if there is a surge in requests prior to the deadline. There are two primary legislative proposals that may renew the program either through 2028 or 2029.
H.R.3433, the “Give Kids a Chance Act of 2024,” was passed by the House of Representatives and is under consideration in the Senate’s Committee on Health, Education, Labor, and Pensions (HELP). It would reauthorize the rare pediatric disease PRV program such that FDA may award PRVs through September 30, 2029, give FDA new authority to enforce requirements that drug sponsors conduct pediatric studies, and incentivize drug makers to conduct pediatric clinical trials when studying combinations of multiple cancer drugs. The bill also adds a requirement for the Government Accountability Office (GAO) to conduct a report on the impact of PRVs, including examining whether the vouchers are successful in promoting development of new rare disease drugs and in expediting patient access.
The “Give Kids a Chance Act” also includes provisions from the “Retaining Access and Restoring Exclusivity” (RARE) Act, as it would specify that the seven-year market exclusivity period for drugs for rare diseases or conditions (i.e., orphan drug exclusivity) prohibits the approval of the same drug for the same approved use or indication with respect to the disease or condition, codifying in statute FDA’s long-standing interpretation of the Orphan Drug Act that was called into question in a 2021 case, Catalyst Pharms, Inc. v. Becerra.
Meanwhile, H.R. 7384, the “Creating Hope Reauthorization Act” would amend the date by which the drug must be designated as a rare pediatric disease drug to September 30, 2028 and the date by which the application must be approved to September 30, 2030. This bill was introduced in the House and referred to the Energy and Commerce Committee’s Subcommittee on Health.
If the rare pediatric disease PRV program is not reauthorized, eligibility to receive a voucher will be limited to products that receive rare pediatric disease designation by December 20, 2024, and are approved by September 30, 2026.
We will keep you updated as we hear of further developments on these efforts. In the meantime, if you have any questions on pediatric drug development in the U.S., please contact any of the authors of this alert or the Hogan Lovells attorney with whom you regularly work.
