Extending the QALY framework for HTA: Exploring the linkages among benefit-risk assessment, stratified medicines, performance-based risk-sharing, and comparative effectiveness research

Abstract :Theoretical and practical challenges and objections to the use of the QALY for pharmacoeconomic evaluations are well known and longstanding, but the recent ECHOUTCOME critique of the QALY has received international visibility and may revive the debate.  Still, the economic evaluation framework based on cost-utility analysis—estimating QALY impacts and cost-offsets in comparison to drug price—remains the scientific gold standard for the evaluation of new, patented medicines.  This talk aims to support the fundamental
soundness of this approach by discussing its usefulness in range of related HTA applications:  benefit-risk assessment, stratified medicines, performance-based risk-sharing arrangements, and comparative effectiveness research.  It is argued that adjustments are needed in some applications, such as considering the value-of-knowing in stratified medicine, or considering population-level health impact in benefit-risk assessment.  But QALY-based modeling remains the best tool we have to support decision-making about benefit, risk, and cost trade-offs for many important public health policy issues.