AMCP Partnership Forum Discusses State of Rare Disease Outcomes and Research, Orphan Drugs to Hit $242 Billion by 2024

April 16, 2021

The AMCP (Academy of Managed Care Pharmacy) held a virtual meeting for healthcare professionals, payers, researchers, and pharma companies in September 2020. A new article discusses key points communicated at the meeting. Notably, orphan drugs designed to treat rare diseases will lead to approximately $242 billion in sales by 2024, but treatment options are still lacking.

The authors remark, “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.” Learn more here.

(Source: JMCP, 2021)

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