BRIEF—US FDA publishes new rare disease therapy guidance

The US Food and Drug Administration has published a revised draft guidance - Rare Diseases: Common Issues in Drug Development.

This agency says that the guidance will assist sponsors of drugs and biologics for rare diseases, helping them to conduct more efficient and successful drug development programs.

The document covers a range of topics including natural history studies, the use of biomarkers and methods for proving safety and efficacy. The original draft guidance was published in 2015.