Monika Wagner
1* 
, Dima Samaha
2, Roman Casciano
3, Matthew Brougham
1, Payam Abrishami
4 , Charles Petrie
5, Bernard Avouac
6, Lorenzo Mantovani
7, Antonio Sarría-Santamera
8,9 , Paul Kind
10, Michael Schlander
11,12, Michele Tringali
131 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.
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.