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Challenges for Bringing Orphan Drugs to Market
Author(s):
Edward Ahn, CEO of MEDIPOST, Inc., reacts to the challenge of launching orphan drugs without complete evidence, particularly given their high cost and limited patient populations, and suggests strategies that can be used to address evidence gaps, enable market access, and support long-term patient benefit.
Not every product has a perfect data set at launch, and that’s where risk-sharing and value-based contracts come into play. This episode explores examples of how companies structure these agreements to address uncertainty, while still ensuring access. With large populations, like with osteoarthritis patients, cost-effectiveness becomes a primary lever.
Key discussion topics include:
- The tradeoff between pricing and access in large populations.
- Examples of successful risk-sharing and subscription models.
- How to position therapies for payer engagement despite imperfect data.
- Why targeting measurable, cost-driven outcomes is critical.
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