ACCOUNTABLE AND REASONABLE PROCESSES FOR COVERAGE DECISION-MAKING FOR RARE DISEASE AND REGENERATIVE THERAPIES – WHICH CRITERIA SHOULD BE CONSIDERED AND WHY?

Author(s)

Wagner M1, Samaha D2, Casciano R3, Brougham M1, Petrie CD4, Abrishami P5, Avouac B6, Kind P7, Sarría-Santamera A8, Schlander M9, Mantovani LG10, Tringali M11
1Analytica Laser, Montreal, QC, Canada, 2Analytica Laser, London, UK, 3Analytica Laser, New York, NY, USA, 4Pfizer, Inc., Groton, CT, USA, 5Zorginstituut Nederland (National Health Care Institute), Diemen, The Netherlands, 6Medecin des Hopitaux de Paris, Paris, France, 7University of Leeds, Leeds, London, UK, 8National School of Public Health, Madrid, Spain, 9Institute for Innovation & Valuation in Health Care, Wiesbaden, Germany, 10University of Milano-Bicocca, Monza, Italy, 11Regione Lombardia, Milan, Italy

Introduction: According to the Accountability for Reasonableness framework, healthcare coverage decisions must be made based on reasons that ‘fair-minded’ stakeholders agree are relevant to resource allocation. We intended to explore this Relevance condition for legitimacy with respect to decision criteria and their selection, using rare disease and regenerative therapies (RDRTs) as a case study. Methods: Features of decision-making processes related to the relevance of decision criteria were defined, including 17 potential criteria, which were identified based on a review of seven published multicriteria frameworks proposed to be applicable for the appraisal of interventions targeting rare diseases. Seven experts from six countries explored and discussed these features during a panel (Chatham House Rule) and provided general and RDRT-specific recommendations for each feature. Responses were analyzed to identify converging and diverging recommendations. Results: Among 17 potential criteria, 13 were recommended by more than half of the panellists. Comparative efficacy/effectiveness, Comparative PROs, Comparative safety, Budget impact, affordability and opportunity costs, and Feasibility of implementation gained the greatest support (≥6/7 panelists). The use of the cost-effectiveness ratio as a principal decision criterion across disease areas was viewed as problematic, particularly when applied to RDRTs due to ethical and methodological challenges. Considering the Feasibility of implementation was deemed essential, especially for rare disease therapies, to ensure that potential benefits are realized in clinical practice. Disease severity was also recommended by most panelists (5/7), as it raised awareness of the goal of healthcare and reflected broadly shared social values. Conclusions: A wide range of decision criteria can be considered relevant for coverage decision-making in RDRTs. Panelists recommended that criteria selection should engage the wider public and rest on the goals of sustainable healthcare and the laws and values of society.

Conference/Value in Health Info

2018-05, ISPOR 2018, Baltimore, MD, USA

Value in Health, Vol. 21, S1 (May 2018)

Code

PCP18

Topic

Health Policy & Regulatory, Methodological & Statistical Research

Topic Subcategory

Confounding, Selection Bias Correction, Causal Inference

Disease

Rare and Orphan Diseases

Explore Related HEOR by Topic


Your browser is out-of-date

ISPOR recommends that you update your browser for more security, speed and the best experience on ispor.org. Update my browser now

×