FDA Publishes Guidance on Rare Pediatric Disease Priority Review
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On July 30, 2019, FDA issued draft guidance that answers questions regarding priority review vouchers for certain rare pediatric disease treatments that meet criteria of the Food, Drug, & Cosmetic Act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 This draft guidance revises a previous draft guidance and clarifies the qualifications and process for requesting priority review vouchers.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.
Source: FDA
A Novel, Enhanced, and Sustainable Approach to Audit Trail Review
July 4th 2025Eli Lilly and Company developed an innovative and sustainable approach to audit trail review (ATR) aimed at reducing the ATR burden while adhering to regulatory expectations and data integrity (DI) principles. The process has transformed employees' understanding of ATR and complemented the DI by design approach, leading to better system designs that meet expected controls and reduce non-value-added data reviews.
