This report explores how payer approaches to access and reimbursement for rare disease therapies are changing in response to rising costs, increasing therapeutic options, and ongoing evidence limitations.
Drawing on payer survey findings and market experience, it highlights five key trends shaping rare disease management:
1. Expanded use case management programs
2. Greater reliance on external clinical experts
3. Growing adoption of value‑based and alternative payment models
4. Restriction of prescribing and administration to Centers of Excellence
5. More complex management when multiple therapies are available within the same indication
You'll also get practical recommendations that emphasize early payer engagement, strong evidence generation, use of real‑world data and registries, alignment with clinical experts and care centers, and sustained collaboration with payers.
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