2020 DIA药物信息年会暨展览会

Orphan drug development is filled with challenges globally, in legislative polices, research, and clinical trials. In contrast to most therapeutic areas, in which randomized, double-blind, placebo-controlled studies of adequate sample size and follow-up are typical, in rare diseases, patients are scarce and trial designs are more varied. In this setting, how does one maintain scientific validity, clinical relevance, and statistical integrity, and meet statutory standards for marketing approval? This session is to underscore the opportunities, successes in orphan drug development and challenges using relevant case studies, globally and domestically.