A Prevalence-Weighted ICER Approach With Scenario Analyses for HTA Without RCT Evidence
Author(s)
Fawaz T. Chaudhry, MPhil.
Unit for Medical Devices, Norwegian Medical Products Agency, Oslo, Norway.
Unit for Medical Devices, Norwegian Medical Products Agency, Oslo, Norway.
OBJECTIVES: To develop and apply a prevalence-weighted method for estimating incremental cost-effectiveness ratios (ICERs) for a medical device in the absence of randomized controlled trial (RCT) data.
METHODS: A cost-utility analysis of a leadless pacemaker was conducted using subgroup-specific relative risks for mortality and infection, derived from published observational studies. Prevalence estimates from the Norwegian patient population were used to assign weights to each subgroup. These weights were applied to generate a population-representative, weighted ICER. Norwegian Diagnosis-Related Group (DRG)-based costs and EQ-5D utilities informed the model inputs. A series of scenarios were constructed using the weighted approach, each producing a new ICER reflecting changes in clinical effectiveness and disease severity. We introduced a novel “scenario frontier” to visualize the variation in ICERs across these plausible clinical scenarios using the weighted approach.
RESULTS: The prevalence-weighted ICER approach produced credible and population-aligned clinical profiles for modeling. ICERs across scenarios ranged from €18,832 to €59,920 per QALY, with the base case estimated at €28,248 per QALY. The scenario frontier provided a clear visual representation of how cost-effectiveness shifts as assumptions about subgroup composition, severity, and clinical effect vary across all subgroups.
CONCLUSIONS: This study introduces a generalizable method for integrating real-world clinical heterogeneity into cost-effectiveness modeling when RCT data are absent and further research may be impractical due to ethical considerations and high costs. By using prevalence-based weights to derive a weighted ICER per scenario, this approach enhances decision relevance. The scenario frontier method provides a clear, reproducible way to communicate uncertainty in HTA, helping decision-makers assess cost-effectiveness for high-risk subgroups when data are limited and pricing decisions must be made under fixed or constrained conditions.
METHODS: A cost-utility analysis of a leadless pacemaker was conducted using subgroup-specific relative risks for mortality and infection, derived from published observational studies. Prevalence estimates from the Norwegian patient population were used to assign weights to each subgroup. These weights were applied to generate a population-representative, weighted ICER. Norwegian Diagnosis-Related Group (DRG)-based costs and EQ-5D utilities informed the model inputs. A series of scenarios were constructed using the weighted approach, each producing a new ICER reflecting changes in clinical effectiveness and disease severity. We introduced a novel “scenario frontier” to visualize the variation in ICERs across these plausible clinical scenarios using the weighted approach.
RESULTS: The prevalence-weighted ICER approach produced credible and population-aligned clinical profiles for modeling. ICERs across scenarios ranged from €18,832 to €59,920 per QALY, with the base case estimated at €28,248 per QALY. The scenario frontier provided a clear visual representation of how cost-effectiveness shifts as assumptions about subgroup composition, severity, and clinical effect vary across all subgroups.
CONCLUSIONS: This study introduces a generalizable method for integrating real-world clinical heterogeneity into cost-effectiveness modeling when RCT data are absent and further research may be impractical due to ethical considerations and high costs. By using prevalence-based weights to derive a weighted ICER per scenario, this approach enhances decision relevance. The scenario frontier method provides a clear, reproducible way to communicate uncertainty in HTA, helping decision-makers assess cost-effectiveness for high-risk subgroups when data are limited and pricing decisions must be made under fixed or constrained conditions.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE27
Topic
Economic Evaluation, Medical Technologies, Methodological & Statistical Research
Topic Subcategory
Thresholds & Opportunity Cost
Disease
Cardiovascular Disorders (including MI, Stroke, Circulatory), No Additional Disease & Conditions/Specialized Treatment Areas