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

Abstract

Background: 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. Methods: 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. Results: 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. Conclusion: 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.

A systematic review of moral reasons on orphan drug reimbursement

“…Consequently, several authors specified that disease severity was not an argument specific to OMPs [ 44 , 75 , 86 , 88 , 104 , 112 , 117 , 142 , 150 , 205 ] and not all rare diseases were severe or life-threatening [ 52 , 55 , 63 , 130 ], thereby concluding that disease severity was not a good criterion to justify special status. Moreover, disease severity would need a unified method of measurement to serve as an objective reimbursement criterion [ 208 ]. In the considered body of literature, diseases were considered severe if, for instance, they were “chronic, deliberating and associated with reduced life expectancy” [ 53 ] or were associated with severe pain [ 212 ].…”

A multi-stakeholder multicriteria decision analysis for the reimbursement of orphan drugs (FinMHU-MCDA study)

“…Although health authorities make the decisions about reimbursement and access to the medications in the National Health System, it is of great importance to facilitate the participation of all sectors with decision-making capacity and opinion-leading positions on rare diseases so as to represent the different existing perspectives, including those of the management/payers, health professionals, and, especially in rare diseases, patients, who bring a unique perspective and are the best experts in their pathologies. Therefore, for the decision to be legitimate, a multi-stakeholder perspective must be incorporated in the development of tools that facilitate decision-making, with MCDA being one of the best approaches to facilitate this participation [ 27 ]. Another important element of this study, that strengthens these MCDA studies is the use of decompositional methods for eliciting preferences, such as the DCE, where the criteria are evaluated together [ 22 ].…”

HTA Agencies Need Evidence-Informed Deliberative Processes Comment on "Use of Evidence-Informed Deliberative Processes by Health Technology Assessment Agencies Around the Globe"

“…Arguably, multicriteria decision analysis frameworks may better support EDPs and stakeholder involvement than currently applied CE evaluations. 30,31 Health economists, in turn, might wish to spend more effort on closing the gap between societal values and the conventional logic of CE by focusing on the develop ment and operationalization of evaluation methods that better capture the full range of economic consequences, including relevant social norms and preferences from the perspective of reasonable, well-informed citizens. This might entail consideration of alternative evaluation paradigms.…”

“…Arguably, multi-criteria decision analysis frameworks may better support EDPs and stakeholder involvement than currently applied CE evaluations. 30 , 31 …”