Bewersdorf Jan Philipp, Huntington Scott F, Zeidan Amer M
Leukemia Service, Department of Medicine, Memorial Sloan Kettering Cancer Center, New York, New York.
Hematology Section, Department of Internal Medicine, Yale School of Medicine, New Haven, Connecticut.
J Natl Compr Canc Netw. 2023 Apr 10;21(5):522-528. doi: 10.6004/jnccn.2023.70012.
Acute myeloid leukemia (AML) is the most common acute leukemia in adults in the United States and has seen the approval of several novel agents over the past decade. Similar to treatments for other hematologic and solid malignancies, these novel agents are costly. In the setting of finite financial resources in the healthcare system, the concept of cost-effectiveness analyses has been developed to compare the estimated costs and associated benefits expected with different interventions (eg, drugs, diagnostic tests, procedures). Although drug approvals in the United States are not based on budgetary considerations, cost-effectiveness analyses can inform health policy decisions, resource allocation, and societal debates. However, such analyses are only capturing parts of the costs and benefits to the healthcare system, payers, and consumers, and are based on modeling assumptions with inherent limitations. In addition, cost-effectiveness analyses for several of the novel agents approved for treatment of AML are limited and have reported conflicting results. This review uses cost-effectiveness analyses of azacitidine/venetoclax and liposomal cytarabine/daunorubicin as examples to review considerations and best practices when conducting and interpreting such studies. To ensure adequate interpretability of cost-effectiveness studies, transparency in the model inputs/assumptions, data sources, and funding is of great importance, as evidenced by the discrepant conclusions across studies. Furthermore, the perspective and the healthcare system from which a cost-effectiveness analysis is conducted are important to consider because practice patterns and drug prices between countries can be variable. However, with advances in health economic modeling techniques, adherence to best practices, and increasing public interest in these types of studies, cost-effectiveness analyses can become an important tool to inform various stakeholders in the healthcare system to allocate limited resources most efficiently.
急性髓系白血病（AML）是美国成年人中最常见的急性白血病，在过去十年中已有多种新型药物获批。与其他血液系统和实体恶性肿瘤的治疗方法类似，这些新型药物价格昂贵。在医疗保健系统资金有限的情况下，成本效益分析的概念应运而生，用于比较不同干预措施（如药物、诊断测试、手术）预期的估计成本和相关效益。尽管美国的药物批准并非基于预算考虑，但成本效益分析可为卫生政策决策、资源分配和社会辩论提供参考。然而，此类分析仅涵盖了医疗保健系统、支付方和消费者成本与效益的一部分，且基于存在固有局限性的建模假设。此外，针对几种获批用于治疗AML的新型药物的成本效益分析有限，且结果相互矛盾。本综述以阿扎胞苷/维奈克拉和脂质体阿糖胞苷/柔红霉素的成本效益分析为例，回顾进行和解释此类研究时的注意事项和最佳实践。为确保成本效益研究具有足够的可解释性，模型输入/假设、数据来源和资金的透明度至关重要，各研究得出的不同结论就证明了这一点。此外，进行成本效益分析所采用的视角和医疗保健系统也很重要，因为不同国家的实践模式和药品价格可能存在差异。然而，随着卫生经济建模技术的进步、对最佳实践的遵循以及公众对这类研究兴趣的增加，成本效益分析可成为一种重要工具，为医疗保健系统中的各利益相关方提供信息，以便最有效地分配有限资源。
