Navigating the Challenges of Rare Disease Drug Development

Clinical trials for rare disease therapies face many challenges due to the small patient population on which they draw, the fact that often there may be many unanswered questions about a specific disease, and the potential variation in the way a rare disease manifests itself in patients. We spoke to John Boland, vice president of product development for the Atlantic Research Group, about the contract research organization’s recent white paper on “Critical Considerations for Rare and Orphan Disease Trial Planning” (http://bit.ly/2qE2qpb), the special challenges of conducting rare disease clinical trials, and how to best address these unique challenges.