The guidance answers questions about how the agency awards priority review vouchers to sponsors of treatments for rare pediatric diseases.
On July 30, 2019, FDA issued draft guidance that answers questions regarding priority review vouchers for certain rare pediatric disease treatments that meet criteria of the Food, Drug, & Cosmetic Act (FD&C Act). As part of the FD&C Act, FDA may give special incentives to companies for the development of treatments for rare pediatric diseases. This draft guidance revises a previous draft guidance and clarifies the qualifications and process for requesting priority review vouchers.
Specifically, the guidance gives detailed answers to questions regarding the definition of a rare pediatric disease, eligibility requirements, a sponsor’s responsibilities after approval of an application, designation information requests, the submission process, marketing applications, and use and transfer of a rare pediatric disease priority review voucher. The guidance also discusses drug-drug combinations, previously approved drugs, and orphan drug designation questions.
