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Liposomal Cytarabine/Daunorubicin May Not Be Cost-Effective in Newly Diagnosed AML

April 27, 2022

May 2022

The liposomal formulation of cytarabine/daunorubicin does not appear to be cost-effective against a conventional willingness-to-pay threshold of $150,000 per quality-adjusted life-year (QALY) in the treatment of newly diagnosed acute myeloid leukemia (AML), according to study findings published in Blood.

Liposomal cytarabine/daunorubicin is approved in the U.S. for the treatment of newly diagnosed secondary AML. Approval was based on a randomized phase III trial that showed an overall survival benefit with the therapy compared with conventional cytarabine/daunorubicin (7+3) in a subset of patients with newly diagnosed secondary AML, explained lead author Jan Philipp Bewersdorf, MD, of Yale University.

“However, the health economic implications of this new approval are not well-characterized,” he said. “In light of increasing health care expenses in general and in oncology in particular, a health economic evaluation of novel drugs can potentially inform health-policy decisions.”

In contrast to other health care systems, the U.S. does not base approvals on cost-effectiveness considerations, said corresponding author Amer Zeidan, MD, of Yale University. “However, as often is the case for hospitalized patients with AML who have Medicare or Medicaid as a primary insurance, the reimbursement hospitals receive for the hospitalization costs by the Centers for Medicare and Medicaid Services (CMS) are based on diagnosis-related groups.”

The diagnosis-related groups, Dr. Zeidan explained, are a fixed sum based on a diagnosis and not the actual expenses incurred. “Thus, the higher medication costs of liposomal cytarabine/daunorubicin compared with 7+3 may not be reflected in the diagnosis-related groups and therefore this warrants consideration of the economic implications of using such therapies for hospitalized patients,” he said.

For the study, researchers performed a partitioned survival analysis of data from an original phase III trial and post-hoc analyses resulting in the ultimate U.S. Food and Drug Administration approval of liposomal cytarabine/daunorubicin. The analysis included patients with newly diagnosed AML (median age = 68 years) who received either liposomal cytarabine/daunorubicin or 7+3 induction and consolidation therapy.

The mean sales price for liposomal cytarabine/daunorubicin was added to the costs of induction and consolidation therapy with 7+3. After collecting all costs related to therapy, the investigators adjusted these costs for inflation to 2020 U.S. dollars.

The lifetime costs of liposomal cytarabine/daunorubicin and 7+3 were $415,258 and $256,415, respectively, which represented an incremental cost of $157,424 with liposomal therapy. The incremental cost-effectiveness ratio (ICER) of the base-case model was $319,660/QALY, as based on an incremental gain of 0.49 QALYs for liposomal treatment compared with 7+3.

In a probabilistic sensitivity analysis, the investigators observed a median ICER of $311,974 (95% CI $231,660-$402,707) with 7+3 conventional treatment favored in 99.98% of 10,000 iterations at a $150,000/QALY willingness-to-pay threshold.

“On a health system level, the study adds to the growing body of evidence highlighting the substantial costs of new drug approvals in oncology on the health care budget,” Dr. Bewersdorf said. “Thus, our results could lend support to ongoing efforts aimed to curb increasing medication costs.”

Since the study included patients between ages 60 and 75, the results may not be generalizable to other age groups, given that liposomal cytarabine/daunorubicin is indicated for all adults with newly diagnosed secondary AML. Dr. Zeidan added that changes in health care utilization, such as usage of allogeneic hematopoietic cell transplantation and inpatient versus outpatient treatment, with liposomal cytarabine/daunorubicin in real-world practice warrants continued study.

Additional research may also be needed to further assess the willingness-to-pay threshold in the modern era, according to Dr. Bewersdorf.

“Whether the willingness-to-pay threshold of $150,000 per QALY [in this study] is still justified today or may need to be revisited is an important question on a more general health economics level,” he stated.

Any conflicts of interest declared by the authors can be found in the original article.


Bewersdorf J, Patel K, Goshua G, et al. Cost-effectiveness of liposomal cytarabine/daunorubicin in patients with newly diagnosed acute myeloid leukemia [published online ahead of print, 2022 Mar 17]. Blood. doi: 10.1182/blood.2021014401.




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