Cost-Effectiveness Analyses in AML: What Have We Learned, How Should This Impact Patient Care, and What Needs to Be Done in the Future?

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 he-matologic and solid malignancies, these novel agents are costly. In the setting of finite financial resources in the healthcare system, the con-cept 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 considera-tions, 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 in-herent limitations. In addition, cost-effectiveness analyses for several of the novel agents approved for treatment of AML are limited and have re-ported conflicting results. This review uses cost-effectiveness analyses of azacitidine/venetoclax and liposomal cytarabine/daunorubicin as exam-ples 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 con-ducted 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.J Natl Compr Canc Netw 2023;21(5):522-528 doi: 10.6004/jnccn.2023.70012