Moving Towards Accountability for Reasonableness – A Systematic Exploration of the Features of Legitimate Healthcare Coverage Decision-Making Processes Using Rare Diseases and Regenerative Therapies as a Case Study

Document Type : Original Article


1 Analytica Laser, Montreal, QC, Canada

2 Analytica Laser, London, UK

3 Analytica Laser, New York City, NY, USA

4 National Health Care Institute (ZIN), Diemen, The Netherlands

5 Pfizer Inc, New York City, NY, USA (retired)

6 Liège University, Liège, Belgium

7 Center for Public Health Research, University of Milan-Bicocca, Milan, Italy

8 National School of Public Health IMIENS-UNED, Madrid, Spain

9 Department of Public Health, University of Alcalá, Alcalá de Henares, Spain

10 University of Leeds, Leeds, UK

11 Division of Health Economics, German Cancer Research Center (DKFZ), Heidelberg, Germany

12 University of Heidelberg, Heidelberg, Germany

13 ASST Niguarda and Regione Lombardia, Welfare Directorate, Milano, Italy


The accountability for reasonableness (A4R) framework defines 4 conditions for legitimate healthcare coverage decision processes: Relevance, Publicity, Appeals, and Enforcement. The aim of this study was to reflect on how the diverse features of decision-making processes can be aligned with A4R conditions to guide decision-making towards legitimacy. Rare disease and regenerative therapies (RDRTs) pose special decision-making challenges and offer therefore a useful case study.

Features operationalizing each A4R condition as well as three different approaches to address these features (cost-per-QALY-focused and multicriteria-based) were defined and organized into a matrix. Seven experts explored these features during a panel run under the Chatham House Rule and provided general and RDRT-specific recommendations. Responses were analyzed to identify converging and diverging recommendations.

Regarding Relevance, recommendations included supporting deliberation, stakeholder participation and grounding coverage decision criteria in normative and societal objectives. Thirteen of 17 proposed decision criteria were recommended by a majority of panelists. The usefulness of universal cost-effectiveness thresholds to inform allocative efficiency was challenged, particularly in the RDRT context. RDRTs raise specific issues that need to be considered; however, rarity should be viewed in relation to other aspects, such as disease severity and budget impact. Regarding Publicity, panelists recommended transparency about the values underlying a decision and value judgements used in selecting evidence. For Appeals, recommendations included a life-cycle approach with clear provisions for re-evaluations. For Enforcement, external quality reviews of decisions were recommended.

Moving coverage decision-making processes towards enhanced legitimacy in general and in the RDRT context involves designing and refining approaches to support participation and deliberation, enhancing transparency, and allowing explicit consideration of multiple decision criteria that reflect normative and societal objectives.


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