This paper addresses the challenges of financing rare disease treatments, which are often high-cost due to small patient populations. It examines limitations of conventional reimbursement systems and proposes models such as annuity-based payments, milestone-based contracts, and pooled risk-sharing approaches. The report emphasizes aligning incentives among stakeholders—governments, payers, pharma, and patients—while ensuring equitable access. It underscores the importance of outcome-based models to ensure value for money and sustainability in supporting rare disease therapies.

Join for free to read