Cost-Effectiveness Analyses in AML: What Have We Learned, How Should This Impact Patient Care, and What Needs to Be Done in the Future?
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Cancer Outcomes, Public Policy, and Effectiveness Research (COPPER) Center, Yale University, New Haven, Connecticut
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Cancer Outcomes, Public Policy, and Effectiveness Research (COPPER) Center, Yale University, New Haven, Connecticut
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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.
Submitted September 23, 2022; final revision received December 20, 2022; accepted for publication January 5, 2023. Published online April 10, 2023.
Disclosures: Dr. Huntington has disclosed serving as a consultant for ADC Therapeutics, Arvinas, AstraZeneca, Celgene, Bayer, Genentech, Pharmacyclics, AbbVie, Epizyme, Novartis, Seagen, Servier, Tyme Inc., and TG Therapeutics; and receiving grant/research support from DTRM Biopharma, Celgene, and TG Therapeutics. Dr. Zeidan has disclosed receiving institutional grant/research support from Celgene/Bristol-Myers Squibb, AbbVie, Astex, Pfizer, MedImmune/AstraZeneca, Boehringer-Ingelheim, Trovagene/Cardiff Oncology, Incyte, Takeda, Novartis, Aprea, and ADC Therapeutics; serving as an advisor and/or consultant and receiving honoraria from AbbVie, Otsuka, Pfizer, Celgene/Bristol-Myers Squibb, Jazz, Incyte, Agios, Boehringer-Ingelheim, Novartis, Acceleron, Astellas, Daiichi Sankyo, Cardinal Health, Taiho, Seattle Genetics, BeyondSpring, Cardiff Oncology, Takeda, Ionis, Amgen, Janssen, Epizyme, Syndax, Gilead, Kura, Chiesi, ALX Oncology, BioCryst, and Tyme Inc.; serving as a scientific advisor for Novartis, AbbVie, Geron, Syros, BioCryst, ALX Oncology, and Celgene/Bristol-Myers Squibb; and receiving travel support from Pfizer, Novartis, and Cardiff Oncology. Dr. Bewersdorf has disclosed having no financial interests, arrangements, affiliations, or commercial interests with the manufacturers of any products discussed in this article or their competitors.
Funding: A.M. Zeidan is a Leukemia and Lymphoma Society Scholar in Clinical Research and was supported by a National Cancer Institute Cancer Clinical Investigator Team Leadership Award. Research reported in this publication was in part supported by the National Cancer Institute of the National Institutes of Health under award numbers P30 CA016359 and P30 CA008748.
Disclaimer: The content is solely the responsibility of the authors and does not necessarily represent the official views of the NIH.
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