OBJECTIVES: Advanced therapy medicinal products (ATMPs) are breakthrough therapies with great therapeutic potential. However, they are often one-shot treatments that are associated with a high-cost and value uncertainty. This creates challenges with traditional upfront payment models and ultimately patient access. The aim of this study is to identify novel payment models for ATMPs and assess their potential impact on patient access and cost-containment.

METHODS: A scoping review of published articles and grey literature pertaining to reimbursement models for ATMPs was conducted, following the Arskey & O’Malley framework. Records were searched using PubMed, Google Scholar and the Google search-engine (grey literature). Record exclusion was based on pre-determined inclusion criteria and conducted in two steps: 1) title- and 2) abstract- screening. Statements on novel models and their impact on patient access and cost-containment were retrieved and mapped. Records published between 2009 and 2022 were included.

RESULTS: A total of 130 records were retrieved. Stages one and two of record exclusion resulted in the inclusion of 46 and 21 records, respectively. Four novel reimbursement models were identified: the Outcomes-based risk-sharing schemes (OB-RSS); the Annuity-based model (ABm); the Hybrid model (OB-RSS with ABm), and the Pooled-funding model (PFm). The OB-RSS was discussed in all 21 records. The ABm, Hybrid model and PFm were discussed in 15, two and three records, respectively. All models could expand patient access through risk-sharing among stakeholders. All models could promote cost-containment through rebates for failed treatment outcomes (OB-RSS and Hybrid models), spreading of payments over time (ABm) and the introduction of multiple payers (PFm).

CONCLUSIONS: Novel payment models could aid in addressing the challenges of high costs and patient access associated with ATMPs. However, many barriers to their implementation exist. Infrastructural changes are required for their optimization. Further study and development of payment models for ATMPs should be conducted.