Key Challenges Associated With Recent NICE Appraisals for Oncology Targeted Treatments
Author(s)
Ana Beatriz Fernandes, BSc, MSc1, Joel Russell, MSc2.
1Market Access and External Affairs, Bristol Myers Squibb, Uxbridge, United Kingdom, 2Health Economics and Outcomes Research, Bristol Myers Squibb, Uxbridge, United Kingdom.
1Market Access and External Affairs, Bristol Myers Squibb, Uxbridge, United Kingdom, 2Health Economics and Outcomes Research, Bristol Myers Squibb, Uxbridge, United Kingdom.
OBJECTIVES: To characterise the clinical and economic evidence submitted to the National Institute for Health and Care Excellence (NICE) for targeted oncology treatments.
METHODS: A data extraction sheet was designed to scrape key information from NICE appraisals for targeted oncology treatments. Data extraction categories included: disease area; targeted mutation; treatment type; clinical trial data (median follow-up time; clinical trial phase; primary and secondary endpoints); cost- effectiveness data (economic model structure; endpoints captured in the model; comparators included; modifier eligibility [end of life (EoL) /severity modifier]); external assessment “key issues” associated with the company submission; recommendation decision; and magnitude of cost-effectiveness. The review was pragmatically restricted between May 2021 - May 2025. Appraisals were selected for oncology treatments targeting genetic mutations only.
RESULTS: 40 appraisals were identified. 68% (n=27) of appraisals contained pivotal trial evidence from Phase 3 trials, 20% from Phase 2 trials, 8% Phase 1/ 2 trials and 5% other. 85% of appraisals from Phase 3 trials were NICE-recommended (81% baseline; 4% Cancer Drugs Fund [CDF]), and 100% of appraisals from Phase 2 trials were NICE-recommended. 73% (n=29) of appraisals utilised a partitioned survival model and 23% (n=9) utilised Markov models. More than half of the cancer histologies were classified as ‘severe’, with 40% of treatments satisfying the NICE severity modifier requirements (10% awarded a x1.7 modifier; 30% a x1.2 modifier), and 18% satisfying the EoL criteria. Identified key issues with appraisals were most commonly long-term clinical uncertainty (choice of extrapolation distribution, treatment waning, cure assumptions), but also included issues with constructing the counterfactual (choice of comparator and indirect treatment comparison issues).
CONCLUSIONS: 88% of oncology targeted treatments have been NICE recommended since 2021, despite variation in clinical trial maturity, severity of disease and key issues with appraisal evidence. Treatments without NICE recommendation were characterised by immature survival data and uncertain survival extrapolations.
METHODS: A data extraction sheet was designed to scrape key information from NICE appraisals for targeted oncology treatments. Data extraction categories included: disease area; targeted mutation; treatment type; clinical trial data (median follow-up time; clinical trial phase; primary and secondary endpoints); cost- effectiveness data (economic model structure; endpoints captured in the model; comparators included; modifier eligibility [end of life (EoL) /severity modifier]); external assessment “key issues” associated with the company submission; recommendation decision; and magnitude of cost-effectiveness. The review was pragmatically restricted between May 2021 - May 2025. Appraisals were selected for oncology treatments targeting genetic mutations only.
RESULTS: 40 appraisals were identified. 68% (n=27) of appraisals contained pivotal trial evidence from Phase 3 trials, 20% from Phase 2 trials, 8% Phase 1/ 2 trials and 5% other. 85% of appraisals from Phase 3 trials were NICE-recommended (81% baseline; 4% Cancer Drugs Fund [CDF]), and 100% of appraisals from Phase 2 trials were NICE-recommended. 73% (n=29) of appraisals utilised a partitioned survival model and 23% (n=9) utilised Markov models. More than half of the cancer histologies were classified as ‘severe’, with 40% of treatments satisfying the NICE severity modifier requirements (10% awarded a x1.7 modifier; 30% a x1.2 modifier), and 18% satisfying the EoL criteria. Identified key issues with appraisals were most commonly long-term clinical uncertainty (choice of extrapolation distribution, treatment waning, cure assumptions), but also included issues with constructing the counterfactual (choice of comparator and indirect treatment comparison issues).
CONCLUSIONS: 88% of oncology targeted treatments have been NICE recommended since 2021, despite variation in clinical trial maturity, severity of disease and key issues with appraisal evidence. Treatments without NICE recommendation were characterised by immature survival data and uncertain survival extrapolations.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA216
Topic
Economic Evaluation, Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Oncology, Personalized & Precision Medicine