FDA Updates Guidance on Rare Pediatric Disease Priority Review Vouchers

Rare Daily Staff

The U.S. Food and Drug Administration issued updated draft guidance on Rare Pediatric Disease Priority Review Vouchers for industry, to clarify the process and reflect changes as a result of the Advancing Hope Act of 2016.

The guidance clarifies what the agency considers as a rare pediatric disease, defining it as a serious and life-threatening disease that primarily affects an individual from birth to age 18. In the context of the vouchers, it considers the pediatric population extending through 18 years of age to conform with the Advancing Hope Act. This is different than what the FDA considers a pediatric population in other contexts, which is generally 16 years of age and younger.

The draft reflects the agency’s current thinking on the process and requirements for the vouchers, which have been a lucrative incentive for developers of rare disease therapies who have secured them.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Companies have sold them for as much as $350 million and as little as $80.6 million.  

Under the 21^st Century Cures Act, the FDA will not be able to designate products for the rare pediatric disease priority review voucher program after September 2020, and the agency cannot award any rare pediatric review vouchers after September 30, 2022.

The FDA is accepting electronic or written comments on the draft guidance through September 30, 2019. Details for submitting comments can be found in the Federal Register.