DEFINING CURATIVE POTENTIAL IN EARLY-STAGE MELANOMA: WHERE PHYSICIANS AND PAYERS ALIGN AND WHERE THEY DON’T
Author(s)
Christopher Black, MPH, PhD1, Ian Daniel, MSPH, MSIS2, Jennifer Sander, MPH3, Sophie Boukouvalas, MSc4, Oriana Ciani, BSc, MSc, PhD5, Ron Akehurst, PhD6, Heather Shaw, MB ChB, MRCP7;
1Merck & Co. Inc, Rahway, NJ, USA, 2Avalere Health, Philadelphia, PA, USA, 3Avalere Health, Washington, DC, USA, 4Avalere Health, Athens, Greece, 5SDA Bocconi, Milan, Italy, 6Lumanity, Sheffield, United Kingdom, 7University College London Hospitals NHS Foundation Trust, London, United Kingdom
1Merck & Co. Inc, Rahway, NJ, USA, 2Avalere Health, Philadelphia, PA, USA, 3Avalere Health, Washington, DC, USA, 4Avalere Health, Athens, Greece, 5SDA Bocconi, Milan, Italy, 6Lumanity, Sheffield, United Kingdom, 7University College London Hospitals NHS Foundation Trust, London, United Kingdom
Presentation Documents
OBJECTIVES: To compare physician and payer perspectives on what constitutes “cure” in early-stage melanoma and how curative potential should be demonstrated.
METHODS: A SLR and HTA informed a conceptual framework that guided development of a 40-50 question online survey. Forty participants from France, Germany, Italy, and the UK were recruited (24 physicians and 16 payers). Physicians were melanoma specialists with ≥5 years’ experience; payers had national/regional oncology access and cost-effectiveness experience. Mixed-methods analysis summarized preferred terminology/definitions, evidentiary priorities (RFS, OS, conditional survival, RWE), acceptable follow-up maturity, and modeling acceptability (time-to-cure [TTC], cure fraction [CF]).
RESULTS: Both groups anchored “cure” in complete excision with negative margins plus sustained absence of local/regional/distant recurrence. Five-year relapse-free survival (RFS) marked an inflection for higher confidence, with confidence rising further at 6-10 years. Terminology diverged by stakeholder and audience: physicians more often used “cured” or “disease-free,” while payers favored “long-term survivor” or “recurrence-free”; most physicians (~80%+) and about half of payers tailored language to audience. Evidence priorities overlapped but differed in emphasis. Physicians prioritized long-term (≥5 years) trial RFS/OS and clinical plausibility of cure; payers emphasized conditional survival, QoL improvement while recurrence-free, statistical cure constructs (TTC/CF), visible plateau/stabilizing hazards, and data maturity (≥3-6 years) before accepting extrapolations. Both groups viewed long-term RWE as critical to corroborate trials and inform cure timing. Psychosocial factors (e.g., anxiety, survivorship needs) were considered relevant to communication and follow-up policy. Country-level variability appeared in confidence thresholds and modeling acceptance, but the 5-year benchmark was a consistent minimum across settings.
CONCLUSIONS: Physicians and payers share core clinical definitions of cure but differ in terminology, confidence thresholds, and evidence needs. Adopting a standardized lexicon distinguishing clinical remission from statistical cure, explicitly reporting evidentiary maturity, and using transparent modeling can add confidence to cure findings, enhance HTA credibility, and guide pragmatic policies for resource planning.
METHODS: A SLR and HTA informed a conceptual framework that guided development of a 40-50 question online survey. Forty participants from France, Germany, Italy, and the UK were recruited (24 physicians and 16 payers). Physicians were melanoma specialists with ≥5 years’ experience; payers had national/regional oncology access and cost-effectiveness experience. Mixed-methods analysis summarized preferred terminology/definitions, evidentiary priorities (RFS, OS, conditional survival, RWE), acceptable follow-up maturity, and modeling acceptability (time-to-cure [TTC], cure fraction [CF]).
RESULTS: Both groups anchored “cure” in complete excision with negative margins plus sustained absence of local/regional/distant recurrence. Five-year relapse-free survival (RFS) marked an inflection for higher confidence, with confidence rising further at 6-10 years. Terminology diverged by stakeholder and audience: physicians more often used “cured” or “disease-free,” while payers favored “long-term survivor” or “recurrence-free”; most physicians (~80%+) and about half of payers tailored language to audience. Evidence priorities overlapped but differed in emphasis. Physicians prioritized long-term (≥5 years) trial RFS/OS and clinical plausibility of cure; payers emphasized conditional survival, QoL improvement while recurrence-free, statistical cure constructs (TTC/CF), visible plateau/stabilizing hazards, and data maturity (≥3-6 years) before accepting extrapolations. Both groups viewed long-term RWE as critical to corroborate trials and inform cure timing. Psychosocial factors (e.g., anxiety, survivorship needs) were considered relevant to communication and follow-up policy. Country-level variability appeared in confidence thresholds and modeling acceptance, but the 5-year benchmark was a consistent minimum across settings.
CONCLUSIONS: Physicians and payers share core clinical definitions of cure but differ in terminology, confidence thresholds, and evidence needs. Adopting a standardized lexicon distinguishing clinical remission from statistical cure, explicitly reporting evidentiary maturity, and using transparent modeling can add confidence to cure findings, enhance HTA credibility, and guide pragmatic policies for resource planning.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
HTA26
Topic
Health Technology Assessment
Disease
SDC: Oncology, STA: Personalized & Precision Medicine