ISSUE: Orphan drugs present a particular challenge to health plans. Though they target small patient populations, their high costs, rising numbers, and strong backing by patient groups force plans to confront ever more delicate decisions about how to provide access. Further, because the FDA often approves orphan drugs based on clinical studies with less rigorous designs (for example, small sample sizes and lack of control arms), health plans are faced with heightened uncertainly regarding the evidence supporting these products. The panel will provide a forum to discuss how health plans are addressing these challenges and to debate approaches for accommodating the growing number of orphan drugs entering the marketplace.

OVERVIEW: The circumstances under which plans apply restrictions (patient subgroup restrictions, step therapy protocols, etc.) in orphan drug coverage decisions are unclear. James Chambers will moderate and will frame the issue by summarizing new research (based on a database of more than 2,000 orphan drug coverage policies) indicating that health plans restrict orphan drug coverage roughly 30% of the time and identifying factors associated with restricted coverage. Paul Melmeyer will represent the patient’s perspective, offering insights on patient experiences and the human consequences of restrictive policies. Susan Cantrell will provide a health plan perspective, describing the various approaches health plans are taking to rise to the challenge presented by orphan drugs. Chris Pashos will provide an industry perspective, discussing difficulties in evidence generation for orphan drugs and challenges in gaining market access. Panelists’ presentations will be limited to three PowerPoint slides (a maximum of 5 minutes), after which the moderator will encourage debate through Q&A and audience participation.