OBJECTIVES: Outcomes-based reimbursement (OBR) is an attractive risk-sharing approach to the managed entry of Advanced Therapy Medicinal Products (ATMPs). However, its use is limited in practice. The aim of this study was to review the literature discussing OBR for ATMPs in European markets to identify the barriers to, and opportunities for its use.

METHODS: A comprehensive scoping review of the literature published between Jan 2009 – May 2022 was conducted. Publications were included in the review if they discussed OBR agreements for ATMPs (or related factors) in European markets. Verbatim extracts from relevant discussions of OBR were captured using a standardised charting form and subsequently free-form coded by a single reviewer. Coded data were analysed for descriptive and overarching analytic themes representative of concepts describing the European OBR landscape.

RESULTS: Searches returned 1,671 records. After removing duplicates, 1,593 publications remained for title and abstract screening. Of these publications, 262 were passed for full-text assessment, of which 84 were included in the review. Content analysis of relevant text from each publication included identified 15 descriptive themes within the literature, capturing a range of diverse factors, such as models of OBR application, structural features of health systems and pharmaceutical market dynamics. Descriptive themes were abstracted into six analytical themes representing barriers to, and opportunities for, the increased use of outcomes-based agreements in the routine reimbursement of ATMPs: Suitability and Sustainability of Innovative Payment Models, Outcomes-based Contract Development, Health System Readiness, Burden of Administration, Accelerated Access to Innovative Medicines and Conflicts of Interest.

CONCLUSIONS: OBR may be a valuable tool in the development of a sustainable market for ATMPs. Payers and manufacturers should maintain awareness of market factors influencing OBR and anticipate the challenges and opportunities posed by each to facilitate early and effective dialogue between stakeholders in the cooperative implementation of OBR agreements.