RE:Clinical Trial Size in Orphan and Rare Diseases Accelerated Approval Pathway
For rare disease patients, time is always of the essence. Many rare diseases progress quickly and, moreover, delayed treatment only increases the economic burden on families. However, many patients with rare conditions have obtained faster access to treatments that provide meaningful advantages through FDA’s Accelerated Approval pathway. Congress established the pathway in 1992 to expedite the approval and availability of drugs and biologics intended to treat serious and lifethreatening diseases and conditions for which there are unmet medical needs. Products seeking Accelerated Approval rely on surrogate endpoints, or markers, “such as a ORR, and PFS.” These endpoints can be measured effectively over a shorter period of time in smaller studies.
Following Accelerated Approvals based on surrogate endpoints, drug developers also are required to conduct studies — phase 4 confirmatory trials — to confirm the clinical benefit of the medicine. Once confirmed, the FDA will grant traditional approval for the treatment.