Abstract
Despite the opportunity for large health gains, there are many challenges associated with rare disease therapies. Among these are striking the appropriate balance between the urgency to respond to patient needs with the uncertainty that is often inherent in rare disease therapy datasets leading to concerns with developing and interpreting clinical data; uncertainty around financial impact, value determination, and affordability; and variation in approach to coverage and potential effects on access. To discuss these challenges and opportunities to address them, AMCP held a virtual multidisciplinary stakeholder forum on September 8-10, 2020. Forum participants represented diverse sectors of the health care industry, including integrated delivery systems, health plans, pharmacy benefit managers, employer groups, biopharmaceutical companies, patient advocacy organizations, health policy researchers, and consulting firms; they evaluated strategies to plan for and manage rare disease therapies and recommended best practices and next steps. DISCLOSURES: This forum was sponsored by the following: AstraZeneca, Dicerna, Genentech, National Pharmaceutical Council, Novo Nordisk, Pfizer, Precision Value, Sanofi, Sarepta Therapeutics, Seattle Genetics, Spark Therapeutics, and Takeda. These proceedings were prepared as a summary of the forum to represent common themes; they are not necessarily endorsed by all attendees nor should they be construed as reflecting group consensus.