FDA Issues Draft Guidance on Rare Diseases
The agency revised draft guidance to enable more efficient and successful drug development programs for the treatment or prevention of rare diseases.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 guidanceto 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The draft guidance, Rare Diseases: Common Issues in Drug Development Guidance for Industry, was originally issued in 2015.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
Guidance on Quality Culture Standards
June 3rd 2025Matt Cushing, VP of Quality and Science, Nelson Labs, and Susan J. Schniepp, distinguished fellow at Regulatory Compliance Associates, a Nelson Labs Company, discuss PDA/ANSI Standard 06-2025: Assessment of Quality Culture Guidance Documents, Models, and Tools, which was published in February 2025.
