PBI34 IMPACTS OF LONG-TERM UNCERTAINTY AND FAMILY SPILLOVER EFFECTS ON THE COST-UTILITY OF APPROVED TREATMENTS FOR SMA TYPE I

Abstract

In April 2019 the Institute for Clinical and Economic Review (ICER) released a report on nusinersen and onasemnogene abeparvovec-xioi (OA-xioi) compared to best supportive care (BSC) in infantile-onset spinal muscular atrophy (SMA I). ICER discussed family spillover effects for these treatments but did not implement them in the economic model. The objective of this analysis was to explore the impact of family spillover and curve fitting strategies for long-term survival prediction on the incremental cost per QALY gained from nusinersen and OA-xioi compared to BSC.