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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., (© 2024. The Author(s).)

Aim: Real-world data and real-world evidence (RWE) are becoming more important for healthcare decision making and health technology assessment. We aimed to propose solutions to overcome barriers preventing Central and Eastern European (CEE) countries from using RWE generated in Western Europe. Materials & methods: To achieve this, following a scoping review and a webinar, the most important barriers were selected through a survey. A workshop was held with CEE experts to discuss proposed solutions. Results: Based on survey results, we selected the nine most important barriers. Multiple solutions were proposed, for example, the need for a European consensus, and building trust in using RWE. Conclusion: Through collaboration with regional stakeholders, we proposed a list of solutions to overcome barriers on transferring RWE from Western Europe to CEE countries.

Objectives: This study aimed to investigate whether the profit margins of pharmaceuticals would influence the outcome of reimbursement decisions within the Dutch policy context.Methods: We conducted a discrete choice experiment among 58 Dutch decision makers. In 20 choice sets, we asked respondents to indicate which of 2 pharmaceutical treatment options they would select for reimbursement. Options were described using 5 attributes (disease severity, incremental costs per quality-adjusted life-year, health gain, budget impact, and profit margin) with 3 levels each. Additionally, cognitive debriefing questions were presented, and for validation debriefing, interviews were conducted. Choice data were analyzed using mixed logit models, also to calculate marginal effects and choice probabilities.Results: Results indicated that the specified levels of profit margins significantly influenced choices made. Decision makers were less likely to reimburse a product with a higher profit margin. The relative importance of profit margins was lower than that of the included traditional health technology assessment criteria, but not negligible. When asked directly, 61% of respondents indicated that profit margin should play a role in reimbursement decision making, although concerns about feasibility and the connection to price negotiations were voiced.Conclusions: Our results suggest that if available to decision makers the profit margin of pharmaceutical products would influence reimbursement decisions within the Dutch policy context. Higher profit margins would reduce the likelihood of reimbursement. Whether adding profit margin as an additional, explicit criterion to the health technology assessment decision framework would be feasible and desirable is open to further exploration. [ABSTRACT FROM AUTHOR]