FDA announces availability of revised draft guidance on Rare Diseases: Common Issues in Drug Development Guidance for Industry

Today the U.S. Food and Drug Administration (FDA) is making available a revised draft guidance titled Rare Diseases: Common Issues in Drug Development Guidance for Industry. This guidance is intended to assist sponsors of drug and biological products for the treatment or prevention of rare diseases in conducting more efficient and successful drug development programs. By covering a range of topics such as natural history studies, use of biomarkers and methods for demonstrating effectiveness and safety the FDA is seeking to share with industry useful information gleaned since the original draft guidance published in 2015. 

More information on FDA’s efforts pertaining to rare diseases is available on FDA’s Office of Orphan Products Development and Center for Drug Evaluation and Research’s Rare Diseases Program websites. 

To read more Press Release articles, click here.