A Blueprint for Multi-use Disease Modeling in Health Economics: Results from Two Expert-Panel Consultations

Junfeng Wang, Xavier Pouwels, Bram Ramaekers, Geert Frederix, Chris Lieshout, Rudolf Hoogenveen, Xinyu Li, G. Ardine Wit, Manuela Joore, Hendrik Koffijberg, Anoukh Giessen, Saskia Knies and Talitha Feenstra ()
Additional contact information
Junfeng Wang: University Medical Center Utrecht
Xavier Pouwels: University of Twente
Bram Ramaekers: Maastricht University Medical Center+, CAPHRI Care and Public Health Research Institute
Geert Frederix: University Medical Center Utrecht
Chris Lieshout: University Medical Center Utrecht
Rudolf Hoogenveen: National Institute for Public Health and the Environment
Xinyu Li: Groningen Research Institute of Pharmacy
G. Ardine Wit: University Medical Center Utrecht
Manuela Joore: Maastricht University Medical Center+, CAPHRI Care and Public Health Research Institute
Hendrik Koffijberg: University of Twente
Anoukh Giessen: National Institute for Public Health and the Environment
Saskia Knies: National Health Care Institute
Talitha Feenstra: Groningen Research Institute of Pharmacy

PharmacoEconomics, 2024, vol. 42, issue 7, No 7, 797-810

Abstract: Abstract Background The current use of health economic decision models in HTA is mostly confined to single use cases, which may be inefficient and result in little consistency over different treatment comparisons, and consequently inconsistent health policy decisions, for the same disorder. Multi-use disease models (MUDMs) (other terms: generic models, whole disease models, disease models) may offer a solution. However, much is uncertain about their definition and application. The current research aimed to develop a blueprint for the application of MUDMs. Methods We elicited expert opinion using a two-round modified Delphi process. The panel consisted of experts and stakeholders in health economic modelling from various professional backgrounds. The first questionnaire concerned definition, terminology, potential applications, issues and recommendations for MUDMs and was based on an exploratory scoping review. In the second round, the panel members were asked to reconsider their input, based on feedback regarding first-round results, and to score issues and recommendations for priority. Finally, adding input from external advisors and policy makers in a structured way, an overview of issues and challenges was developed during two team consensus meetings. Results In total, 54 respondents contributed to the panel results. The term ‘multi-use disease models’ was proposed and agreed upon, and a definition was provided. The panel prioritized 10 potential applications (with comparing alternative policies and supporting resource allocation decisions as the top 2), while 20 issues (with model transparency and stakeholders’ roles as the top 2) were identified as challenges. Opinions on potential features concerning operationalization of multi-use models were given, with 11 of these subsequently receiving high priority scores (regular updates and revalidation after updates were the top 2). Conclusions MUDMs would improve on current decision support regarding cost-effectiveness information. Given feasibility challenges, this would be most relevant for diseases with multiple treatments, large burden of disease and requiring more complex models. The current overview offers policy makers a starting point to organize the development, use, and maintenance of MUDMs and to support choices concerning which diseases and policy decisions they will be helpful for.

Date: 2024
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DOI: 10.1007/s40273-024-01376-w

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