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Seventy-seven percent of drugs for rare diseases with pivotal efficacy data have orphan designations, and most are approved with Phase III randomized controlled trials (Domike et al, 2024). This creates a regulatory paradox: FDA rules permit Phase II approval with post-marketing commitment data, yet sponsors conduct resource-intensive Phase III trials anyway. 

Seventy-seven percent of drugs for rare diseases with pivotal efficacy data have orphan designations, and most are approved with Phase III randomized controlled trials (Domike et al, 2024). This creates a regulatory paradox: FDA rules permit Phase II approval with post-marketing commitment data, yet sponsors conduct resource-intensive Phase III trials anyway.