Scientific analyzes results of cerebrospinal fluid in patients with transverse myelitis. [Adobe stock]
FDA’s RDEP in Context
Developing treatments for ultra-rare diseases presents fundamental challenges for traditional evidence generation. Patient populations for these conditions range from fewer than 10 to up to 1,000, making traditional randomized clinical trial (RCT) designs impractical and possibly unethical. These constraints require sponsors to adopt flexible evidence approaches…
