ISSUE: Several limitations and challenges have been identified when conducting economic evaluations of gene replacement therapies (GRT) using standard approaches, such as recommended by HTA organisations using cost-effectiveness analysis as a key driver of decisions. These include in particular: 1) assessment of clinical effectiveness and safety based on small clinical trials, often single-arm; 2) valuation of health outcomes for children, who are often targeted by GRT; 3) time horizon and extrapolation, as there is substantial uncertainty long-term effects, positive or negative; 4) possible development of new payment models specific to GRT. The panel will provide a forum to discuss potential solutions for key methodological issues in health economic evaluation of gene replacement therapies.

OVERVIEW: The moderator will initiate the debate by providing an overview of the gene replacement therapies pipeline and the evidence in development. He will introduce four questions on which the panellists will debate. 1) Under which conditions are single-arm studies acceptable and sufficient for decision-making? 2) Should generic utility instruments like EQ-5D be used for the valuation of health outcomes in severe paediatric diseases? 3) Are analyses over a lifetime horizon feasible and appropriate despite substantial uncertainty around the long-term efficacy and safety? Extrapolation approaches will be discussed. 4) Are the difficulties of economic evaluations for GRT alleviated with the use of new payment models? The panellists, Mondher Toumi, Ataru Igarashi, and Omar Dabbous, will succinctly present their position on these questions, providing academic and industry perspectives. Time will be reserved for debate between panellists, focusing about points of divergence in opinion, with participation of the audience.