OBJECTIVES: One-time treatments (e.g. CAR-T, cell and gene therapies) have the potential for unique health improvements. However, their high cost combined with uncertainties regarding their long-term effectiveness and safety create unique challenges. Innovative payment models could manage these challenges. This study examines the challenges and opportunities of outcomes-based and portfolio agreements for manufacturers and payers in Europe.

METHODS: A systematic literature review was conducted to identify the types of innovative models utilized across Europe for one-time curative treatments, followed by qualitative interviews (n=15) and a survey (n=50) of payer stakeholders to ratify findings from the secondary research and determine factors important in the selection of preferred innovative payment models.

RESULTS: Payers expressed interest in outcomes-based agreements to mitigate uncertainties of long-term clinical outcomes (effectiveness and safety) and demonstrate manageable budget impact and cost effectiveness.

Payment models that are preferred and recently implemented include 1) portfolio-level outcomes-based model to guarantee a given clinical outcome across a range of treatments 2) pay-for-performance that link reimbursement with the achievement of predefined clinical outcomes for an individual patient 3) annuity on reduced net price enabling confidential discounted payments spread over pre-determined periods.

Several payment models, such as portfolio-level agreements (e.g., cladribine agreement with GWQ in Germany) and outcomes-based agreements (e.g., tisagenlecleucel agreement with GWQ in Germany and MoH in Spain, axicabtagene ciloleucel agreement in Germany, and Autologous CD34+ stem cell therapy agreement with AIFA in Italy), were identified.

CONCLUSIONS: Outcomes-based and portfolio agreements can facilitate access of high cost one-time treatments in Europe. Multiple factors such as plausibility of effect, outcome measures selected, and type of patient population impact the selection of outcomes-based agreement. Moreover, the preferred payment model is linked with the specific objections raised by a payer based on the role (national, regional, local) within the overall healthcare system.