Developing drugs for rare diseases poses a range of clinical, regulatory, and commercial challenges. Small patient populations and limited research on orphan diseases make it difficult to set clinical endpoints, biomarkers, and outcomes measures. Sensitive patient subpopulations ranging from neonates and pediatrics to adults with co-morbidities make conducting clinical trials ethically problematic. The following case studies, webinars, blogs, and whitepapers will provide insights into the unique problems and solutions for this area of drug development.
Rare and Neglected Diseases Resource Center
