Preparing for and Managing Rare Diseases - Proceedings from the AMCP Partnership Forum
Preparing for and Managing Rare Diseases
Proceedings from the AMCP Partnership Forum
Thursday, Mar. 11, 2021, 2-3pm ET
Despite the opportunity for large health gains, there are many challenges associated with therapies for rare diseases. To discuss these challenges as well as opportunities to address them, AMCP held a virtual multidisciplinary stakeholder forum September 8-10, 2020. Forum participants represented diverse sectors of the healthcare industry, including integrated delivery systems, health plans, pharmacy benefit managers (PBMs), employer groups, biopharmaceutical companies, patient advocacy organizations, health policy researchers, and consulting firms; they evaluated concepts related to planning for and managing rare disease therapies and recommended best practices and next steps. Priority challenge areas identified by this forum’s participants pertained to designing clinical trials with often small populations and heterogeneous disease course, and interpretation of the data for determining appropriate patients, efficacy, and duration of effect; uncertainty around financial impact and value as well as overall affordability; and variation in approach to coverage including the timing of coverage, the evidence used in evaluation, utilization management strategies, and access considerations. Strategies to meet these challenges will be discussed.
- Erin Lopata, PharmD, MPH
- Kat Wolf Khachatourian, PharmD, MBA
Chief Quality Integration Officer
Physicians of Southwest Washington/Multicare Connected Care
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