The efficacy and economic evaluation of roxadustat treatment for anemia in patients with kidney disease not receiving dialysis

ABSTRACT

Background

This study aimed to assess the efficacy, tolerance, and cost-effectiveness of roxadustat treatment for anemia in patients with chronic kidney disease not receiving dialysis (CKD ND).

Methods

A meta-analysis was conducted to evaluate the clinical efficacy and tolerance of roxadustat for the correction of anemia associated with CKD ND, and a Markov model was developed to evaluate the cost-effectiveness of roxadustat compared with a placebo.

Results

The meta-analysis results showed that compared with a placebo, roxadustat treatment was associated with a remarkably higher rate of clinical response and the differences in the rate of adverse events between these two regimens were not significant. Moreover, roxadustat treatment (70 mg, three times per week) provided an additional 0.49 QALYs at a cost of $12,526 in the time horizon of 5 years, resulting in an ICER of $25,563 per QALY, with approximately 60% probability to be cost-effective at a $29,295 per QALY willingness-to-pay (WTP) threshold from the perspective of Chinese medical system.

Conclusion

For the treatment of anemia in Chinese patients with CKD ND, roxadustat is much more effective than a placebo; moreover, it is cost-effective at conventional WTP thresholds.

KEYWORDS:

• Cost-effectiveness
• anemia
• chronic kidney disease
• roxadustat
• meta-analysis

Article Highlights

• Roxadustat is a novel therapy for the treatment of anemia in patients with chronic kidney disease (CKD) through inhibition of the hypoxia-inducible factor (HIF) prolyl hydroxylase.

• It is of great importance to know whether roxadustat should be administrated to patients with CKD not receiving dialysis (CKD ND) for Chinese decision makers since roxadustat is expensive and the medial resources are limited.

• Compared to a placebo, roxadustat exhibits a powerful effect in the correction of CKD ND-associated anemia without enhancing any side effects.

• At conventional WTP thresholds, roxadustat might be a cost-effective strategy in the correction of CKD ND-associated anemia compared to a placebo, with the probability of approximately 60% at the current negotiated price of roxadustat.

• The likelihood of roxadustat to be a cost-effective treatment for the CKD ND-associated anemia would be close to 100% if the price of roxadustat was further reduced to one-half of the negotiated price.

Declaration of interest

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.

Supplemental material

Supplemental data for this article can be accessed here.

Reviewer disclosures

A reviewer on this manuscript has disclosed being a member on the advisory board at meetings supported by Amgen, Astellas, GSK, Roche and Vifor Pharma. Peer reviewers on this manuscript have no other relevant financial relationships or otherwise to disclose.

Author contributions

Zhanhong Hu and Hong Tao were responsible for study design, data collection and the manuscript writing; Aiming Shi and Jie Pan were served as advisors. All the authors have approved the final version of the manuscript to be published.

Additional information

Funding

This paper was not funded.