Presentation (CTI)

OBJECTIVES: As more therapies with a curative intent are developed, it is important that cure assumptions within health economic models are sufficiently justified. With limited guidelines currently in place, this study provides a framework that can be used by health technology assessment bodies and manufacturers when implementing cure within economic evaluations.
METHODS: A systematic literature review of NICE technology appraisals incorporating cure assumptions was conducted (2015 to 2025). Extraction items included: technology appraisal identifier, publication date, intervention, disease category, model structure, how cure was modelled, decision outcome, and impact of cure assumption on the reimbursement decision. A framework informed by the findings of the review was developed containing the following domains: biological/clinical plausibility, empirical plausibility and statistical/structural plausibility. Application of the framework to all technology appraisals included in the initial systematic literature review was subsequently conducted.
RESULTS: Of the 2,191 technology appraisals identified, 677 were included for screening and 57 were included for data extraction. The technology appraisals comprised of submissions in 16 diseases areas, with over half being in either leukaemia (n=15) or lymphoma (n=13). Modelling of cure was primarily implemented using either a cure point (60%, n=34) or a mixture cure model (28%, n=16). Upon application of the criteria, validation of a cure assumption by clinical experts was the most common source of evidence (95%; n=54), whilst 61% (n=35) validated cure using literature, and 53% (n=30) relied on past technology appraisal precedents.
CONCLUSIONS: There is a currently no standardised approach for the implementation and assessment of cure assumptions within health economic models. This study provides a framework to support the evaluation, validation and implementation of cure assumptions within health technology assessment submissions.