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Yale Cancer Center Study Shows Liposomal Cytarabine/Daunorubicin Not Cost Effective to Treat Older Patients with AML

December 10, 2021

New research led by Yale Cancer Center shows it is not cost-effective to use the liposomal formulation of cytarabine and daunorubicin (previously known as CPX-351) compared to conventional cytarabine and daunorubicin to treat older patients with acute myeloid leukemia (AML). Liposomal cytarabine/daunorubicin has been shown to improve overall survival in a subset of older patients with AML, leading to its recent approval by the U.S. Food and Drug Administration (FDA). The findings of the cost-effectiveness analysis are being reported today at the 2021 American Society of Hematology (ASH) annual meeting in Atlanta, Georgia.

“Our results are in line with other studies evaluating the cost-effectiveness of novel drugs in oncology suggesting that many of these are not cost-effective,” said lead study author Jan Bewersdorf, MD, a former resident physician at Yale New Haven Hospital and Yale Cancer Center and now at Memorial Sloan Kettering Cancer Center. “This study highlights that the medication costs itself are the major contributor for this finding and that only a reduction in drug costs is able to make liposomal cytarabine/daunorubicin cost-effective.”

In their study, researchers conducted a partitioned survival analysis based on data from the original phase III clinical trial of liposomal cytarabine/daunorubicin. Model inputs such as patient characteristics, treatment patterns, health care resource utilization, and outcomes were based on the original trials or previously published estimates from the literature. The findings show treatment with liposomal cytarabine/daunorubicin led to additional costs of $319,660 per quality-adjusted life-year (QALY). In general, a medical intervention is considered to be cost-effective if it costs less than $150,000 per QALY. Therefore, we conclude that CPX-351 is not cost-effective at the current price. We also found that only a reduction in the price of liposomal cytarabine/daunorubicin by about 80% would make it cost-effective.

“Many novel therapies have now been approved for management of patients with AML, however all of these drugs are very expensive. Our work highlights the need to reduce the prices for novel drugs not only for AML, but across all oncology indications,” said Amer M. Zeidan, MD, senior author of the study. “Highlighting the increasing drug costs in the US and the health economic implications can support ongoing efforts and initiatives to curb novel drug costs” he added.

Yale’s George Goshua, MD, Kishan Patel, MD, Rory Shallis, MD, Nikolai Podoltsev, MD, PhD, and Scott F. Huntington, MD, MPH, also contributed to the research.

The Frederick A. DeLuca Foundation, an American Society of Hematology Physician-Scientist Career Development Award, the Yale Forget Scholarship and the Leukemia and Lymphoma Society Scholar in Clinical Research provided funding for this research.