OBJECTIVES: Tisagenlecleucel is a chimeric antigen receptor T-cell therapy for B-cell acute lymphoblastic leukaemia and diffuse large B-cell lymphoma, which has achieved reimbursement using OBR schemes in Europe and the US. OBR schemes are becoming popular to manage uncertainty and alleviate the financial burden on healthcare systems when adopting high-cost medicines. This research will compare reimbursement models that have been developed for tisagenlecleucel.

METHODS: We performed secondary research to compare the reimbursement models across France, Germany, Italy, Spain, UK and US, using qualitative analysis of HTA appraisals and published papers.

RESULTS: In the UK and France, tisagenlecleucel is reimbursed based on coverage with evidence development (CED) schemes. Reassessment of reimbursement and cost is reviewed annually based on long-term data from clinical trials, and real-world data in clinical practice. In addition to CED, Germany also adopted outcome-based-rebates to reimburse tisagenlecleucel based on individual patient outcomes and survival; a new approach to reimbursement in this market. In contrast, Italy and Spain have adopted a distinctive payment model, whereby payments are made in two or three instalments respectively, depending on individual patient outcomes. In the US, payment for tisagenlecleucel is only made when patients respond to treatment within 30 days, demonstrated by a complete blood count.

CONCLUSIONS: The differences in OBR schemes across these markets are a result of the need to mitigate uncertainty in different healthcare structures, varying degrees of willingness to pay and priorities. Payers are showing openness to different reimbursement mechanisms to enhance patient access, demonstrated by the novel OBR approaches for tisagenlecleucel in Germany, Italy and Spain. This shows promise for the future reimbursement of high-cost therapies and demonstrates payer willingness to engage in payment models if; a) the clinical need for a product is significant, and b) there are no data, legal or administrative hurdles which cannot be overcome.