FDA releases updated draft guidance on drug development for rare diseases

By Dr Nicola Davies

In January of this year, the US Food and Drug Administration released updated draft guidance for industry entitled “Rare Diseases: Common Issues in Drug Development.” This replaces the previous version issued in 2015.1

The 2019 draft guidance was developed to help sponsors improve the research and drug development process for rare diseases given the unique challenges of this sector. The revised guidance contains updates on investigations into the natural history of rare diseases, surrogate biomarkers, clinical trial endpoints, study design, and patient safety. An added recommendation for sponsors to involve patients, caregivers, and advocates in the rare disease drug development process is also included.