Latest 21st Century Cures Draft Scales Back Priority Review Voucher Program

The House Energy & Commerce Committee released a new version of the 21st Century Cures Act that scales back the rare pediatric disease priority review voucher program.

While the previous measure, released May 13, would have allowed the FDA to award these vouchers through June 30, 2022, the latest version cuts them off on Dec. 31, 2018.

The updated measure — now with an official bill number, H.R. 6 — was released ahead of full committee markup today where it will be introduced as a substitute for the measure adopted last week by the health subcommittee.

A rare pediatric disease priority review voucher allows a drugmaker to secure a six-month review of a subsequent drug, following approval of a drug that treats a rare childhood disease. The program, authorized under the 2012 FDA Safety and Innovation Act, is set to expire in March 2016.

The measure also would require the Government Accountability Office to look at the indications approved for each drug awarded a priority review voucher and whether the voucher affected the sponsor’s decision to invest in developing drugs to treat or prevent rare pediatric diseases. The comptroller general would also have to conduct an analysis of drugs approved using the voucher, including whether they address unmet needs or represent an advance over available therapies.

Shortening the voucher reauthorization period by four years may have the unintended effect of discouraging drugmakers from developing rare disease treatments, since companies need certainty when developing drugs for small patient populations, says Kurt Karst, a partner at D.C. law firm Hyman, Phelps & McNamara. If the program is cut, as the current measure proposes, firms in the early stages of drug development might not have time to qualify for a voucher and recoup their investment.

Aside from the voucher program, the FDA’s overhaul measure is largely unchanged from the previous one.

The package includes an extra $15 million for the FDA to run pilot programs aimed at extending the Sentinel surveillance system in fiscal years 2016 to 2020. While the system currently collects drug safety information from electronic health records and insurance claims data, the committee would like it to also include evidence from observational studies, therapeutic use and registries. This new information could be used to support approvals of new indications and inform postmarket study requirements.

Read H.R. 6 at www.fdanews.com/05-20-15-21CCbill.pdf. — Lena Freund