Cost-effectiveness analysis of azacitidine maintenance therapy in patients with acute myeloid leukemia

ABSTRACT

Background

The QUAZAR AML-001 trial (NCT01757535) showed survival benefits with the maintenance treatment of oral azacitidine(CC-486) for acute myeloid leukemia(AML) in first complete remission. We conducted a cost-effectiveness analysis to explore the costs and benefits of oral azacitidine in AML.

Methods

We constructed a Markov model to evaluate the economic value of oral azacitidine. The time horizon was 10-years. The health utility scores and until prices of medical costs were acquired from previous studies and GoodRX. The transition probabilities were derived from the survival curves of the QUAZAR AML-001 study. Outcomes were measured in quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratio (ICER).

Results

Compared with placebo, oral azacitidine improved 0.39 QALY, with an increasing cost of $458,928.66. The ICER of oral azacitidine is $1,176,740.15(P < 0.05). Deterministic sensitivity analysis showed that the price of oral azacitidine has a significant impact on ICERs (P < 0.05). Probability sensitivity analysis showed that the probability of cost-effectiveness for oral azacitidine is 0.

Conclusion

In the United States, oral azacitidine is unlikely to be cost-effective for AML patients at current prices.

Clinical trial registration

The trial is registered at ClinicalTrials.gov (CT.gov identifier: NCT01757535)

KEYWORDS:

• Acute myeloid leukemia
• oral azacitidine
• maintenance treatment
• cost-effectiveness analysis
• CC-486

Disclosure statement

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Author contributions

T Niu put forward the idea of this study. J Zhu analyzed and interpreted the data generated in the clinic trail. J Wang helped to search the cost data. J Zhu and Q Wu performed the analysis and contributed equally in writing the manuscript. All authors read and approved the final manuscript.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was supported by the 1.3.5 Project for Disciplines of Excellence, West China Hospital, Sichuan University (No. ZYJC21007), the Incubation Program for Clinical Trials (No. 19HXFH030), the Achievement Transformation Project (No. CGZH21001), and the Translational Research Grant of NCRCH (No. 2021WWB03).