Since its inception in 1992, the FDA has greenlit nearly 300 new drugs under its accelerated approval pathway, with notable nods in oncology, Alzheimer’s disease, and amyotrophic lateral sclerosis. It has not, however, been leveraged extensively for rare diseases, where it could be a particularly good fit. The majority of gene therapies target rare diseases. According to a study published in BMC, better access to the accelerated approval pathway could reduce development costs by approximately 60%, increase investment value and enable development of three times as many rare disease drugs even if investment stayed flat. Approval through this pathway is largely reliant on biomarkers as surrogate endpoints - which clinical trials for gene therapies are often designed to measure. But accelerated approvals of gene therapies and other rare disease drugs have been few and far between. This panel discussion will propose changes intended to optimize the program for therapies targeting rare and genetic diseases and discuss the appropriate instances in which to seek accelerated approval.