Purpose: The objective of this session is to examine the state of U.S. health plan coverage for orphan drugs and to discuss barriers to access and opportunities for alignment across stakeholders.

Description: Evaluating treatments for rare diseases is challenging given small populations, limited evidence on long-term benefits, and the high cost of clinical trials. As a result, access to orphan drugs can vary substantially across payers, suggesting that economic, clinical, and societal factors are not always weighed consistently. Access to FDA-approved treatment options may be restricted by payers in light of limited efficacy data or by financial barriers including high out of pocket costs. Many patients with rare conditions may also rely on the off-label use of treatments approved for more common conditions, though insurance coverage for this strategy is uncommon. Understanding how these challenges impact patient access is key for developing a ‘fairer’ assessment framework that is more sustainable and comprehensive.

The session will begin with an introduction to the current landscape of treatment coverage for rare and orphan diseases among U.S. health plans, including an overview of the types of restrictions commonly placed on orphan drugs as well as the role various factors play in influencing policy decisions, such as disease prevalence and cost-effectiveness (20 minutes, Chambers). The session will continue with the presentation of an established framework for ‘fair’ access, which incorporates policy and ethical research to inform the appropriateness of insurance cost sharing policies. How this framework may differ for rare disease will be explored, given unique challenges such as limited standard of care and uncertainties in trial outcomes (20 minutes, Emond). The session will conclude with a summary of the challenges to access from the patient perspective and recommendations on how to promote a more patient-centered evaluation strategy (20 minutes, Dave).