ISSUE: Conventional cost-effectiveness analysis (CEA; i.e., assessing pharmaceuticals through a cost per quality-adjusted life year [QALY] framework) originated from a societal commitment to maximize population health despite limited resources: a utilitarian approach that has produced pricing and reimbursement systems that generally favor health technologies for common diseases. This framework has been slow to evolve with our understanding of the impact of rare diseases, which in turn has complicated the assessment of orphan medicinal products (OMPs) meant to treat rare diseases.

OVERVIEW: Elisabeth Fenwick will moderate and will begin the panel discussion with a brief overview of conventional CEA and the context of rare diseases / OMPs. The primary limitations of conventional CEA with respect to OMPs are (1) the rigidity of the cost per QALY threshold, (2) the difficulty of fitting the patient experience of a rare disease into the QALY construct, and (3) the restricted scope of how costs and benefits are measured. Proposed approaches for addressing these shortcomings can be broadly grouped into three non-mutually exclusive categories: (1) augmenting the valuation context, (2) adjusting the valuation context, and (3) increasing the willingness-to-pay threshold for OMPs. The panel will discuss and debate the shortcomings of conventional CEA when applied to the evaluation of orphan drugs in a rare disease setting. Additionally, the panel will discuss and debate the merits and limitations of augmented and alternative approaches to conventional CEA within the aforementioned context. Lastly, the panelists will debate the future of conventional CEA in a context where pharmaceuticals are becoming more specialized and targeting increasingly less prevalent diseases and smaller patient subgroups.