Presentation (CTI)

OBJECTIVES: To characterize how payers and health economists define and assess “cure” in early-stage melanoma, including preferred terminology, evidence requirements, timing thresholds, cross‑country differences, and implications for modeling, reimbursement, and policy.
METHODS: A literature/HTA review informed a piloted, online mixed‑methods questionnaire for stakeholders in France, Germany, Italy, and the UK, including 16 payers/health economists. Quantitative and qualitative responses were analyzed to evaluate terminology choices, evidentiary priorities (recurrence‑free survival [RFS], overall survival [OS], conditional survival), acceptable follow‑up for cure assessment, role of real‑world evidence (RWE), and country patterns.
RESULTS: Payers preferred patient‑facing terms such as “long‑term survivor,” “recurrence‑free,” and “disease‑free,” reserving “cured/statistically cured” for technical contexts. Terminology selection was influenced by audience, perceived recurrence risk, treatment stage, and guidelines. Long‑term RFS milestones and conditional survival were viewed as most informative indicators of curative potential; melanoma‑specific OS prioritized when available. About half identified 5‑year RFS as a benchmark for higher confidence in cure; many favored follow‑up beyond 5 years to address late recurrence and patient heterogeneity. Acceptance of cure modeling (e.g., mixture/non‑mixture cure models), conditional survival estimates, parametric extrapolations depended on adequate data maturity (often 3-6 years observed), visible hazard stabilization/plateau, sufficient at‑risk numbers, and biological plausibility. RWE-particularly long‑term follow‑up from registries and EHRs-was rated critical to corroborate trial findings and inform cure timing outside trials. Even with substantial evidence, defining cure remains challenging due to late relapse risk and response variability. Country differences emerged: UK respondents showed greater consistency and clearer thresholds; Germany/Italy exhibited varied views; France emphasized data quality and clinical plausibility.
CONCLUSIONS: A pragmatic, multi‑source approach is needed for cure determination in early‑stage melanoma. While 5‑year RFS is commonly cited for confidence, stronger claims require longer follow‑up, corroborative RWE, and transparent modeling. Clear communication of “statistical” versus individual cure, plus visual survival/hazard evidence, can support payer confidence, and guide HTA decision/policy.