ArticleInternational journal of health policy and management2019
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.
Article in International journal of health policy and management, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers, 1 of them a synthesis that pooled it.
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Who cites it
12 citing papers in PubMed, 1 synthesis or guideline pooled it.
- A systematic review of moral reasons on orphan drug reimbursement.Orphanet journal of rare diseases · 2021Pooled it
- Integrating Equity in Topic Selection for Orphan Medicinal Products: Policy Implications for Health Technology Assessment in Rare Diseases.Applied health economics and health policy · 2026Review
- Implicit ethical choices in ultra-rare therapy development: researchers' perspectives on patient selection and communication, therapy value, and accessibility.BMC medical ethics · 2026Article
- Assessing the national essential medicines list selection processes: Instrument development and testing.PloS one · 2026Article
- Letter to the Editor - Qualitative evidence submitted by patients to NICE: need for more quality or unrealistic and unfair requirements?Journal of comparative effectiveness research · 2025Article
- Setting priorities for healthcare interventions in Indonesia: a comprehensive conceptual framework.International journal for equity in health · 2025Review
- Procedural fairness and the resilience of health financing reforms in Ukraine.Health policy and planning · 2023Article
- Criteria for the procedural fairness of health financing decisions: a scoping review.Health policy and planning · 2023Article
- The reimbursement for expensive medicines: stakeholder perspectives on the SMA medicine nusinersen and the Dutch Coverage Lock policy.BMC health services research · 2022Article
- Public legitimacy of healthcare resource allocation committees: lessons learned from assessing an Israeli case study.BMC health services research · 2022Article
- How Can We Optimize the Value Assessment and Appraisal of Orphan Drugs for Reimbursement Purposes? A Qualitative Interview Study Across European Countries.Frontiers in pharmacology · 2022Article
- A multi-stakeholder multicriteria decision analysis for the reimbursement of orphan drugs (FinMHU-MCDA study).Orphanet journal of rare diseases · 2021Article
Corrections and comments
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Authors and funding
12 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundThe 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.
methodsFeatures 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.
resultsRegarding 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.
conclusionMoving 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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Registered trials
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