Presentation (CTI)

OBJECTIVES: Negative decisions from Health Technology Assessment (HTA) agencies are often challenging for stakeholders to receive. However, negative decisions are invaluable in providing learning opportunities to better understand broader concerns of the HTA agencies.This study aimed to: 1) Develop a comprehensive dataset of negative reimbursement decisions from both the Irish National Centre for Pharmacoeconomics (NCPE) and England’s National Institute for Health and Care Excellence (NICE); and 2) Evaluate key drivers of negative recommendations across agencies, identifying similarities and differences between the UK and Ireland.
METHODS: Publicly available NCPE and NICE appraisal documentation from 2016 to 2026 was systematically reviewed using a retrospective content analysis approach to identify negative and conditional-negative reimbursement recommendations. The working dataset comprises 782 documents across 613 assessments, including 410 NCPE documents and 372 NICE documents. Decision drivers were extracted using a structured multi-label coding framework covering clinical, economic, comparator, affordability and robustness issues. Comparative analyses assessed the frequency and distribution of decision drivers by agency, therapeutic area, and recommendation outcome.
RESULTS: Across both agencies, several consistent themes underpinning negative decisions were identified, including uncertainty in comparative clinical effectiveness, reliance on immature or indirect evidence, concerns regarding extrapolation and model assumptions, and insufficient robustness of the submitted cost-effectiveness case. Negative recommendations were generally driven by the combined impact of unresolved clinical and economic uncertainty.
CONCLUSIONS: Negative recommendations from NICE and the NCPE were consistent with a core set of methodological and evidentiary challenges. The largest difference between the two agencies was regarding the budget impact analysis, a core focus of the NCPE, yet briefly mentioned in the final appraisal documents. The differences between NICE and NCPE highlight the importance of tailoring submissions to agency-specific expectations, particularly budget impact, value for money, and local relevance.