BHM and BHM-Based Extrapolations in an HTA Framework
Author(s)
Katrin Haeussler, PhD1, Kyle Dunton, BSc, MSc2, Lirong Zhang, MSc3, Georgie Weston, MSc4, Charlotte Sophie Wilhelm-Benartzi, PhD5, Anne Correges, MSc6, Lucas Hynes, MSc1, Arthur Allignol, PhD7.
1Daiichi Sankyo Europe GmbH, Munich, Germany, 2Daiichi Sankyo, Uxbridge, United Kingdom, 3Daiichi-Sankyo, Munich, Germany, 4AstraZeneca, Cambridge, United Kingdom, 5Daiichi Sankyo Italia Sp.A., Monte Porzio Catone, Italy, 6Daiichi Sankyo Oncology France, Paris, France, 7Daiichi Sankyo Europe, Munich, Germany.
1Daiichi Sankyo Europe GmbH, Munich, Germany, 2Daiichi Sankyo, Uxbridge, United Kingdom, 3Daiichi-Sankyo, Munich, Germany, 4AstraZeneca, Cambridge, United Kingdom, 5Daiichi Sankyo Italia Sp.A., Monte Porzio Catone, Italy, 6Daiichi Sankyo Oncology France, Paris, France, 7Daiichi Sankyo Europe, Munich, Germany.
OBJECTIVES: Accounting for heterogeneity is critical when combining different sources of information; this is important especially in the accurate estimation of cost-effectiveness and subsequent health technology assessment (HTA) decision-making (Murphy et al. 2021). The UK’s National Institute for Health and Care Excellence (NICE) also now recommends that “when heterogeneity between groups within a population is a concern, any assumptions about homogeneity or heterogeneity⋯ must be clearly presented, tested and fully explored” (NICE 2020).
METHODS: The traditional use of Bayesian Hierarchical Models (BHM) is in the context of basket trials. BHMs can also be used to extrapolate time-to-event outcomes to inform cost-effectiveness analysis for HTA payer dossiers as in McCarthy et al. (McCarty et al. 2024). This work will examine a method to use BHMs in simulated data to extrapolate time-to-event outcomes in parametric distributions including the Weibull, Exponential, Lognormal, and Gamma accelerated failure time (AFT) parametric distributions. Survival functions will be estimated via the cumulative distribution function extrapolated for a specific follow-up time; variance-covariance matrices will be estimated, and survival and hazard functions plotted.
RESULTS: BHMs allow the treatment effect in any specific basket to be informed by the effects in other incorporated baskets, thereby: a) maximising the information available, b) increasing the precision of estimates and c) reducing the probability of obtaining unreliable estimates in baskets with only a few patients (Murphy et al. 2021)
CONCLUSIONS: BHMs are a valuable tool especially for sparse data in oncology, informing cost-effectiveness modelling.
METHODS: The traditional use of Bayesian Hierarchical Models (BHM) is in the context of basket trials. BHMs can also be used to extrapolate time-to-event outcomes to inform cost-effectiveness analysis for HTA payer dossiers as in McCarthy et al. (McCarty et al. 2024). This work will examine a method to use BHMs in simulated data to extrapolate time-to-event outcomes in parametric distributions including the Weibull, Exponential, Lognormal, and Gamma accelerated failure time (AFT) parametric distributions. Survival functions will be estimated via the cumulative distribution function extrapolated for a specific follow-up time; variance-covariance matrices will be estimated, and survival and hazard functions plotted.
RESULTS: BHMs allow the treatment effect in any specific basket to be informed by the effects in other incorporated baskets, thereby: a) maximising the information available, b) increasing the precision of estimates and c) reducing the probability of obtaining unreliable estimates in baskets with only a few patients (Murphy et al. 2021)
CONCLUSIONS: BHMs are a valuable tool especially for sparse data in oncology, informing cost-effectiveness modelling.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
MSR47
Topic
Economic Evaluation, Health Technology Assessment, Methodological & Statistical Research
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Oncology