EVALUATING THE ROLE AND APPLICATION OF REAL-WORLD EVIDENCE IN NICE ONCOLOGY HEALTH TECHNOLOGY APPRAISALS AND ITS INFLUENCE ON HTA OUTCOMES (2016-2025)
Author(s)
Mark Chalmers, PhD1, PRATEEK KANADE, MPH2, Ravina Mathur, MPH2, Vidhyasagari Sundaram, MPH2.
1Partner, EY, Dublin, Ireland, 2Ernst & Young, BENGALURU, India.
1Partner, EY, Dublin, Ireland, 2Ernst & Young, BENGALURU, India.
OBJECTIVES: To evaluate the role and impact of real‑world evidence (RWE) on health technology assessment (HTA) outcomes for oncology therapies through a retrospective review of National Institute for Health and Care Excellence (NICE) technology appraisals conducted between 2016 and 2025.
METHODS: A retrospective review of NICE oncology technology appraisals for therapies approved between 2016 and 2025 was conducted using publicly available documents. RWE was defined as non‑randomized evidence from sources such as registries, observational studies, electronic health records, or real‑world comparators. Subsequently, the role of RWE in HTA decision-making was evaluated by analysing its role in addressing clinical and/or economic uncertainties and its association with HTA outcomes
RESULTS: A total of 224 NICE oncology technology appraisals conducted between 2016 and 2025 were included in the analysis. Of these, 8% (N=17) incorporated RWE. Among appraisals that included RWE, 88% (N=15) received a positive HTA recommendation, including 12% (N=2) recommended through managed access arrangements such as the Cancer Drugs Fund, while 12% (N=2) were not recommended. RWE was predominantly used to support comparator analyses (53%; N=9), followed by its application in informing long‑term effectiveness (29%; N=5) and addressing other evidence gaps (18%; N=3).
CONCLUSIONS: RWE plays an important role in supplementing clinical evidence within NICE oncology appraisals. However, only a small proportion of submissions incorporate RWE. Where used, RWE primarily supports comparator analyses and long‑term effectiveness and is particularly valuable in settings with limited clinical trial data or absence of direct comparisons. However, variability in its application and the broader evidence package makes its influence on HTA outcomes difficult to assess. There is a need for clearer guidance from HTA bodies, such as NICE, on the inclusion and role of RWE in HTA assessments to support consistent and transparent use in the future.
METHODS: A retrospective review of NICE oncology technology appraisals for therapies approved between 2016 and 2025 was conducted using publicly available documents. RWE was defined as non‑randomized evidence from sources such as registries, observational studies, electronic health records, or real‑world comparators. Subsequently, the role of RWE in HTA decision-making was evaluated by analysing its role in addressing clinical and/or economic uncertainties and its association with HTA outcomes
RESULTS: A total of 224 NICE oncology technology appraisals conducted between 2016 and 2025 were included in the analysis. Of these, 8% (N=17) incorporated RWE. Among appraisals that included RWE, 88% (N=15) received a positive HTA recommendation, including 12% (N=2) recommended through managed access arrangements such as the Cancer Drugs Fund, while 12% (N=2) were not recommended. RWE was predominantly used to support comparator analyses (53%; N=9), followed by its application in informing long‑term effectiveness (29%; N=5) and addressing other evidence gaps (18%; N=3).
CONCLUSIONS: RWE plays an important role in supplementing clinical evidence within NICE oncology appraisals. However, only a small proportion of submissions incorporate RWE. Where used, RWE primarily supports comparator analyses and long‑term effectiveness and is particularly valuable in settings with limited clinical trial data or absence of direct comparisons. However, variability in its application and the broader evidence package makes its influence on HTA outcomes difficult to assess. There is a need for clearer guidance from HTA bodies, such as NICE, on the inclusion and role of RWE in HTA assessments to support consistent and transparent use in the future.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HTA147
Topic
Health Technology Assessment, Real World Data & Information Systems
Topic Subcategory
Decision & Deliberative Processes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas