Department of Pharmaceutical Outcomes & Policy, College of Pharmacy, University of Florida, Gainesville.
Department of Pharmacy Practice, College of Clinical Pharmacy, Imam Abdulrahman Bin Faisal University, Dammam, Kingdom of Saudi Arabia.
J Manag Care Spec Pharm. 2024 Oct;30(10):1178-1188. doi: 10.18553/jmcp.2024.30.10.1178.
In 2022-2023, the US Food and Drug Administration approved 2 novel gene therapies, valoctocogene roxaparvovec and etranacogene dezaparavovec, for hemophilia A and B, respectively. These one-time-administered gene therapies have been marketed at prices that create financial challenges for payers and patients. Understanding the magnitude and uncertainties around the long-term value of these therapies and how they can potentially relate to managed care practices is of high interest to the payer and patient community.
To conduct a systematic review of cost-effectiveness analysis (CEA) studies to assess (1) the long-term value of valoctocogene roxaparvovec and etranacogene dezaparavovec and (2) the relevance and validity of the underlying data and assumptions used in the CEA models and discuss how they relate to the challenges identified for CEAs of gene therapies.
A systematic review of cost-effectiveness studies of novel hemophilia A and B gene therapy was conducted. PubMed and Embase were searched for published studies from inception to January 12, 2024. Original research articles published in English that conducted a CEA on gene therapy treatments for hemophilia A and B, with a comparison of incremental costs and health effects, were considered. Critical appraisal of the quality of reporting and the underlying modeling assumptions were conducted to assess the relevance and validity of the results.
Two hundred thirty-eight studies were identified, of which 4 met the inclusion criteria. Three studies were conducted from a US health care perspective and 1 from a Dutch societal perspective. Despite the high upfront costs of the gene therapies, all included studies' (3 hemophilia A and 1 hemophilia B) modeled results showed that gene therapies had lower overall costs and better health outcomes compared with factor concentrate replacement therapies and emicizumab. The results were driven by the assumption that gene therapies will have a durable effect of at least 10 years and offset the high cost of the current standard of care. The modeled health improvements varied substantially across studies, showing that the long-term value is sensitive to varying clinical and economic assumptions.
The novel hemophilia gene therapy treatments can potentially be a cost-effective use of treatment resources if the treatment effects are durable over time. To reduce the risk for payers while still facilitating patient access, outcomes-based agreements similar to what has recently been proposed by the Centers for Medicare & Medicaid Services for sickle-cell therapies are well supported.
2022-2023 年，美国食品和药物管理局分别批准了两种新型基因疗法，即 valoctocogene roxaparvovec 和 etranacogene dezaparavovec，用于治疗血友病 A 和 B。这些一次性给药的基因疗法的定价给支付方和患者带来了财务挑战。了解这些疗法的长期价值及其不确定性，以及它们如何与管理式医疗实践相关，是支付方和患者群体非常关注的问题。
进行系统评价以评估成本效益分析（CEA）研究，以评估（1）valoctocogene roxaparvovec 和 etranacogene dezaparavovec 的长期价值，（2）CEA 模型中使用的基础数据和假设的相关性和有效性，并讨论它们与基因疗法 CEA 中确定的挑战的关系。
对新型血友病 A 和 B 基因治疗的成本效益研究进行了系统评价。检索了 PubMed 和 Embase 自成立至 2024 年 1 月 12 日发表的研究。纳入了评估基因治疗治疗血友病 A 和 B 的增量成本和健康效果的比较，并发表了英语原始研究文章。对报告的质量和基础模型假设进行了严格评估，以评估结果的相关性和有效性。
共确定了 238 项研究，其中 4 项符合纳入标准。3 项研究从美国医疗保健角度进行，1 项从荷兰社会角度进行。尽管基因疗法的前期成本很高，但所有纳入研究（3 项血友病 A 和 1 项血友病 B）的模型结果表明，与因子浓缩物替代疗法和emicizumab 相比，基因疗法具有更低的总体成本和更好的健康结果。结果的驱动因素是基因疗法将具有至少 10 年的持久效果，并抵消当前标准护理的高成本。模型中健康改善的幅度在研究之间差异很大，表明长期价值对不同的临床和经济假设敏感。
如果治疗效果随着时间的推移持久，新型血友病基因治疗可能是一种具有成本效益的治疗资源利用方式。为了降低支付方的风险，同时仍然为患者提供便利，最近医疗保险和医疗补助服务中心为镰状细胞疗法提出的基于结果的协议得到了很好的支持。
