Presentation (CTI)

OBJECTIVES: Aligning measures of clinical benefit with funding decisions by HTAs is essential to ensure patient access to high-value oncology therapies. The ESMO Magnitude of Clinical Benefit Scale (MCBS) grades treatments on trial design, endpoints, and patient-relevant outcomes, but its scores often diverge from HTA outcomes across jurisdictions. This study benchmarks ESMO-MCBS ratings against HTA decisions from three leading agencies, CDA, NICE, and SMC, analysing factors driving misalignment for solid tumors and highlighting implications for value-based access.
METHODS: Data were extracted from publicly available HTA reports and ESMO-MCBS scorecards. We identified 450 drug-indication pairs from HTA outcomes between 2010 and 2024, collecting molecule names, brand names, HTA outcomes, rationale for decision-making (including evidence uncertainties and social value judgements), dates, and ESMO-MCBS scores. Applying an analytical framework to benchmark ESMO grades against HTA outcomes, we defined misalignment as negative HTA outcome with high ESMO (MCBS=4 or 5) score or positive HTA outcome with low ESMO (MCBS=1 or 2) score. Misaligned cases were advanced to a second stage for detailed review.
RESULTS: Preliminary analysis identified 66 misaligned cases (14.7% of all HTA outcomes): 21 from CDA, 26 from NICE, and 19 from SMC. Seventeen cases were negative misalignments, in which high ESMO-MCBS scores coincided with negative HTA outcomes, primarily due to the lack of robust cost-effectiveness data, often a result of immature survival evidence. The remaining 49 were positive misalignments, where therapies with low clinical-benefit ratings were recommended for reimbursement, consistently justified by agencies through pricing negotiations, other criteria beyond costs and effects, and commercial access arrangements.
CONCLUSIONS: Our findings reveal systematic differences between clinical benefit frameworks and reimbursement decisions, particularly in cases where therapies with high clinical benefit are not recommended for reimbursement and highlight the importance and breadth of multiple criteria used in decision-making.