SECTION 3

Study design and execution for rare diseases

Designing and executing rare disease clinical trials poses unique challenges. There are fewer patients, many are children, knowledge of the condition may be incomplete, and fewer sites can administer complex treatments such as cell and gene therapies—to name just a few. This section includes advice and lessons learned on selecting optimal endpoints, implementing complex, innovative trial designs, and engaging with patient advocacy groups to make it easier for patients to participate in clinical research.

Download the executive summary of the full report