Cost-Effectiveness Modeling of Gene Therapies: Challenges and Solutions

This session focuses on advancing economic modelling approaches for high-cost, complex therapies, particularly in rare diseases and advanced treatments. Presentations include a systematic review of modelling challenges and solutions for rare disease technologies, early economic modelling to estimate the cost-effectiveness and value-based price of gene therapy for urofacial syndrome, and insights from NICE and CADTH-AMC appraisals of CAR-T cell therapies. The session concludes with an evaluation of the cost-effectiveness and budget impact of gene therapy for sickle cell disease in the Netherlands.