From Evidence Gaps to Launch Delays: A Review of Health Technology Assessment Critiques and Health Economics and Outcomes Research Strategy Readiness
Author(s)
Ting-Yen Chen, MSc1, Vaishali Sahabote, Bpharmacy, MBA2, João Leite, MSc3, McVin Cheen, PhD4, Weiwei Xu, PhD, MD4.
1IQVIA, London, United Kingdom, 2IQVIA, Hyderabad, India, 3IQVIA, Lisbon, Portugal, 4IQVIA, Amsterdam, Netherlands.
1IQVIA, London, United Kingdom, 2IQVIA, Hyderabad, India, 3IQVIA, Lisbon, Portugal, 4IQVIA, Amsterdam, Netherlands.
OBJECTIVES: To review health technology assessments (HTAs) with delayed decisions and synthesise key evidence gaps.
METHODS: Completed HTAs of health technologies indicated for endocrine and metabolic disorders by National Institute for Health and Care Excellence (NICE) and Canada’s Drug Agency (CDA-AMC) since April 2022 were identified. Appraisals with delayed decisions, defined as >1.5 times the published standard submission-to-recommendation duration, were analysed to identify key criticisms and synthesise evidence gaps.
RESULTS: Among the 24 HTA reports, six NICE and nine CDA-AMC appraisals were reviewed, with nine excluded due to ultra-rare indications. Population-related evidence gaps included generalisability (n=6 , 40%), under-representation of key subgroups (n=1, 7%), and baseline imbalances between groups (n=3, 20%). Gaps in clinical evidence included limited endpoint relevance (n=6, 40%), HRQoL outcomes uncertainty (n=3, 20%), and lack of robust indirect treatment comparisons (ITCs) (n=3, 20%). Economic evidence gaps involved utility uncertainty (n=5, 33%), extrapolation assumptions (n=5, 33%), and drug acquisition factors (n=2, 13%). Compared to NICE, CDA-AMC focused more on the robustness of pivotal trials and ITCs, with criticisms related to baseline imbalances and quality of the ITCs highlighted exclusively by CDA-AMC.
CONCLUSIONS: A holistic HEOR strategy with a clear evidence generation roadmap plays a critical role in supporting timely and optimal HTA outcome. Prior to pivotal trials, reviewing HTAs of comparable analogue products and seeking expert input (e.g., scientific advice, advisory boards) on trial population, stratification factors, and endpoints can mitigate issues of generalisability. A well-structured strategy and statistical analyses plan for patient-reported outcome is crucial for generating robust evidence to inform economic models. A thorough ITC feasibility assessment that considers various analytical methods, assumptions and scenarios in the absence of direct evidence can mitigate uncertainty. Finally, early planning for real-world evidence generation and post‐hoc subgroup and sensitivity analyses will strengthen the evidence package for HTA submissions.
METHODS: Completed HTAs of health technologies indicated for endocrine and metabolic disorders by National Institute for Health and Care Excellence (NICE) and Canada’s Drug Agency (CDA-AMC) since April 2022 were identified. Appraisals with delayed decisions, defined as >1.5 times the published standard submission-to-recommendation duration, were analysed to identify key criticisms and synthesise evidence gaps.
RESULTS: Among the 24 HTA reports, six NICE and nine CDA-AMC appraisals were reviewed, with nine excluded due to ultra-rare indications. Population-related evidence gaps included generalisability (n=6 , 40%), under-representation of key subgroups (n=1, 7%), and baseline imbalances between groups (n=3, 20%). Gaps in clinical evidence included limited endpoint relevance (n=6, 40%), HRQoL outcomes uncertainty (n=3, 20%), and lack of robust indirect treatment comparisons (ITCs) (n=3, 20%). Economic evidence gaps involved utility uncertainty (n=5, 33%), extrapolation assumptions (n=5, 33%), and drug acquisition factors (n=2, 13%). Compared to NICE, CDA-AMC focused more on the robustness of pivotal trials and ITCs, with criticisms related to baseline imbalances and quality of the ITCs highlighted exclusively by CDA-AMC.
CONCLUSIONS: A holistic HEOR strategy with a clear evidence generation roadmap plays a critical role in supporting timely and optimal HTA outcome. Prior to pivotal trials, reviewing HTAs of comparable analogue products and seeking expert input (e.g., scientific advice, advisory boards) on trial population, stratification factors, and endpoints can mitigate issues of generalisability. A well-structured strategy and statistical analyses plan for patient-reported outcome is crucial for generating robust evidence to inform economic models. A thorough ITC feasibility assessment that considers various analytical methods, assumptions and scenarios in the absence of direct evidence can mitigate uncertainty. Finally, early planning for real-world evidence generation and post‐hoc subgroup and sensitivity analyses will strengthen the evidence package for HTA submissions.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA150
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
Diabetes/Endocrine/Metabolic Disorders (including obesity)