FDA Issues Draft Guidance on Rare Diseases
The agency revised draft guidance to enable more efficient and successful drug development programs for the treatment or prevention of rare diseases.
On Jan. 17, 2019, FDA announced that it issued a revised draft guidance to enable more efficient and successful drug development programs for the treatment or prevention of rare diseases.
The draft guidance, Rare Diseases: Common Issues in Drug Development Guidance for Industry, was originally issued in 2015. The revised guidance covers a range of topics such as natural history studies, use of biomarkers, and methods for demonstrating effectiveness and safety.
Comments and suggestions should be submitted within 60 days of publication.
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.
