Abstract
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. 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. 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. 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.
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