OBJECTIVES: As oncologic therapies claiming curative potential are launched, the demonstration of economic value depends on economic evaluations (EEs) based on assumptions of cure. We analyzed the health technology assessment (HTA) reports on EEs submitted to support the reimbursement of curative oncologic therapies to identify critiques that could potentially pose challenges to economic discussions for reimbursement.

METHODS: First, we selected CAR-T drugs as the therapies of research given their curative claims, novel technology, and early data evaluations. We selected HTA agencies in Canada, Australia, England, and Sweden to analyze the global HTA perspective and reviewed HTA submissions for CAR-T drugs for three oncologic conditions, and extracted the critiques raised by HTA agencies on the submitted EEs.

RESULTS: HTA agencies preferred 3-health state (progression-free; progressed; death) models for EEs, with a time horizon reflective of the natural history of the disease. Due to clinical uncertainties (short follow-up time, patient size) to support curative claims, HTA agencies mostly accepted conservative cure points (5 years) and mortality risks higher for the treated population than for the overall population for a lifetime period. Moreover, in the absence of mature survival data, HTA agencies preferred survival values modelled through spline/standard-parametric models rather than mixture-cure models, requesting survival assumptions to be in line with data collected through clinical trials. Finally, as with most therapies, HTA agencies preferred utility values derived from clinical trials for all states and challenged drug cost inputs different from the costs of reimbursed therapies.

CONCLUSIONS: The economic value of curative drugs is constrained by clinical trial limitations that pose uncertainties on curative claims and the validity of EEs. HTA agencies prefer conservative EEs that fit the clinical data and local context rather than optimistic models that assume an uncertain cure. Early local health economics and payer consultation is critical to ensure the development of robust EEs.