CBSA and BIO urge you to reach out to Senator Hickenlooper to encourage his support of S.4583, a bill that would reauthorize the rare pediatric disease priority review voucher (PPRV) program before it expires on September 30, 2024. As NORD explains, the PPRV “has helped spur rare disease drug development in pediatric populations and brought almost four dozen therapies to market with critical safety and dosing data specific to children.” In a recent opinion piece in STAT News, rare disease parents Joe and Courtney Dion stress that the PPRV program is “one of the great victories” among “important reforms aimed at encouraging more research into rare diseases.” 

CBSA and BIO thank the sponsors of the House bill to reauthorize the PPRV for six years advanced by the House Energy & Commerce Committee’s Health Subcommittee by a bipartisan vote on May 16 as well as the sponsors of the new, equivalent bill introduced in the Senate on June 18. 

The PPRV Program: The rare pediatric disease priority review voucher (PPRV) program aims to incentivize drug development for rare pediatric diseases. Under this FDA voucher program, a sponsor who receives approval for a drug or biological product for a rare pediatric disease may qualify, if it meets certain criteria, for a voucher that can be redeemed to receive priority review for a different product. The sponsor may also transfer or sell the voucher to another sponsor. 

Why it Matters: As the National Organization for Rare Disorders (NORD) explains, the PPRV “has helped spur rare disease drug development in pediatric populations and brought almost four dozen therapies to market with critical safety and dosing data specific to children.” NORD emphasizes that the “significant uptake in pediatric rare disease approvals in recent years demonstrates the value of the program to the rare disease patient community, where more than 95% of rare diseases lack an FDA approved treatment.” In a recent opinion piece in STAT News, rare disease parents Joe and Courtney Dion stress that the PPRV program is “one of the great victories” among “important reforms aimed at encouraging more research into rare diseases.” PPRVs “have attracted an impressive amount of attention and private investment to this underserved area of medical research.” 

Expiration and Needed Reauthorization: The PPRV program’s authorization is set to expire on September 30, 2024. Without a timely reauthorization by Congress, the FDA will no longer be allowed to initiate the process necessary to issue new PPRVs and, as the Dions put it, “the difficult task of curing and treating rare childhood diseases will get even harder.” 

House Subcommittee Advances Bill: H.R.7384 – Creating Hope Reauthorization Act of 2024 was introduced by Reps. McCaul, Eshoo, Bilirakis, Barragan, Burgess, and Trahan on February 15, 2024.  On May 16, 2024, the House Energy & Commerce Committee’s Health Subcommittee approved H.R. 7384 to extend the PPRV program for six years with a bipartisan vote. House Energy & Commerce Committee Chair Cathy McMorris Rodgers pointed to the Dions’ story of their two children fighting muscular dystrophy as an example of why the PPRV program must be reauthorized. Subcommittee members on both sides of the aisle agreed that the PPRV program is a critical tool to incentivize the development of treatments for rare pediatric diseases. 

Senate Introduces Bill: The Senate recently introduced S.4583, an equivalent bipartisan bill, on June 18 and it is expected to go to the Senate Health, Education, Labor and Pensions (HELP) Committee in the coming weeks. You can read press releases from sponsors Senator Bob Casey (D-PA) and Senator Markwayne Mullin (R-OK). 

Call to Action: CBSA and BIO urge you to reach out to Senator Hickenlooper to encourage his support of S.4583, as he is an important voice on the Senate HELP Committee. You can email Hannah Berner, Sen. Hickenlooper’s Senior Policy Advisor, to express how important reauthorization of the PPRV program is to the development of drugs for rare pediatric diseases, since the costs of drug development paired with the risk involved with bringing a successful drug to market often discourage investment in the rare disease space—and especially for rare diseases unique to children.