OBJECTIVES: Cell and gene therapy products offer potential curative effects to chronic but are imbedded with data uncertainty and high-costs. New reimbursement models are emerging, that serve as a catalyst for adoption of cell and gene therapies in the future. This research aims to review the cost-effectiveness evidence for ex vivo gene therapy Strimvelis, and CAR T-cell therapies Kymriah (Tisagenlecleucel), Yescarta (Axicabtagene Ciloleucel) and explore the emerging reimbursement models.

METHODS: Pubmed, MEDLINE and Cochrane Library database were searched to December 2019 for (i) ex vivo gene therapy for adenosine deaminase deficiency severe combined immunodeficiency (Strimvelis) and CAR T-cell therapies (ii) tisagenlecleucel (Kymriah) and (iii) axicabtagene ciloleucel (Yescarta) and (iv) reimbursement models for cell and gene therapies.

RESULTS: A total of 13 articles were identified as relevant for full-text screening: 8 (66%) analysed cost-effectiveness of Strimvelis, Kymriah and Yescarta in a NHS healthcare setting. The cost per quality adjusted life year (QALY) estimates were highly variable due to uncertainty in the data.

CONCLUSIONS: New reimbursement models are set to accommodate future high-cost of cell and gene therapy with data uncertainty. Proposed managed access agreements (MMA) encourage collaboration between NHS England and the manufacturer to manage the risks.