Department of Health Policy, London School of Economics and Political Science, Cowdray House, Portugal Street, London, UK.
Cancer Research UK and UCL Cancer Trials Centre, UCL Cancer Institute, University College London, London, UK.
Pharmacoeconomics. 2020 Dec;38(12):1297-1308. doi: 10.1007/s40273-020-00956-w.
Recently licensed cell and gene therapies have promising but highly uncertain clinical benefits. They are entering the market at very high prices, with the latest entrants costing hundreds of thousands of dollars. The significant long-term uncertainty posed by these therapies has already complicated the use of conventional economic evaluation approaches such as cost-effectiveness and cost-utility analyses, which are widely used for assessing the value of new health interventions. Cell and gene therapies also risk jeopardising healthcare systems' financial sustainability. As a result, there is a need to recalibrate the current health technology assessment methods used to measure and compensate their value. In this paper, we outline a set of technical adaptations and methodological refinements to address key challenges in the appraisal of cell and gene therapies' value, including the assessment of efficiency and affordability. We also discuss the potential role of alternative financing mechanisms. Ultimately, uncertainties associated with cell and gene therapies can only be meaningfully addressed by improving the evidence base supporting their approval and adoption in healthcare systems.
最近获得许可的细胞和基因疗法具有有前途但高度不确定的临床效益。它们以非常高的价格进入市场，最新进入者的成本高达数十万美元。这些疗法带来的重大长期不确定性已经使传统的经济评估方法（如成本效益和成本效用分析）的使用变得复杂，这些方法广泛用于评估新的卫生干预措施的价值。细胞和基因疗法也有可能危及医疗保健系统的财务可持续性。因此，有必要重新调整目前用于衡量和补偿其价值的卫生技术评估方法。在本文中，我们概述了一套技术调整和方法改进，以解决细胞和基因疗法价值评估中的关键挑战，包括效率和负担能力的评估。我们还讨论了替代融资机制的潜在作用。最终，只有通过改善支持其在医疗保健系统中获得批准和采用的证据基础，才能有意义地解决与细胞和基因疗法相关的不确定性。
