Cost-effectiveness Analysis of Prophylaxis Versus On-demand Treatment for Children With Moderate or Severe Hemophilia A in China.

IF 3.2 4区医学 Q2 PHARMACOLOGY & PHARMACY

Clinical therapeutics Pub Date : 2024-12-01 Epub Date: 2024-10-16 DOI:10.1016/j.clinthera.2024.09.003

Yaohan Zhou, Zhengping Li, Guoqing Liu, Zhenping Chen, Wanru Yao, Gang Li, Yingzi Zhen, Xiaoling Cheng, Di Ai, Kun Huang, Wang Cao, Runhui Wu

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引用次数: 0

Abstract

Background: It is still being determined if prophylaxis (PR) has superior cost effectiveness compared with on-demand (OD) treatment for moderate or severe hemophilia A (HA) children in China.

Objective/purpose: To evaluate the cost-effectiveness of PR and OD treatment for children with moderate or severe HA without inhibitors in China.

Methods: A retrospective cost-effectiveness study was conducted on 640 HA children (373 and 267 children were on the PR and OD treatment, respectively) from January 2021 to November 2022. The Markov model was used to estimate the economic and clinical outcomes and would run for 17 yearly cycles with the initial age at 2 years. The transfer probabilities were extracted from the data of "Hemophilia Home Care Center" and the literature published. All patients' drug costs were collected from the data of "Hemophilia Home Care Center". One-way and probabilistic sensitivity analyses were conducted on the data to evaluate the robustness of the results.

Results/findings: PR was consistently associated with higher overall quality-adjusted life years (QALYs) compared with OD treatment (9.59 QALYs vs. 6.85 QALYs). The incremental cost-effectiveness ratio (ICER) of PR compared with the OD treatment was calculated to be approximately US$12,151.35 (RMB¥81,778.55) per QALY gained. This amount was lower than the willingness-to-pay (WTP) threshold of US$38,212.74 (RMB¥257,171.71). One-way sensitivity analysis found that the results were sensitive to the cost of OD and PR treatments.

Conclusions/implications: This study indicated that PR is cost-effective compared with OD treatment for children with moderate or severe HA without inhibitors in China.

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中国中度或重度 A 型血友病患儿预防性治疗与按需治疗的成本效益分析。

背景：目标/目的：评估中国中度或重度甲型血友病（HA）患儿接受预防性治疗（PR）和按需治疗（OD）的成本效益：2021年1月至2022年11月，对640名HA患儿（分别有373名和267名患儿接受PR和OD治疗）进行了成本效益回顾性研究。该研究采用马尔可夫模型来估算经济和临床结果，该模型将运行17个年周期，初始年龄为2岁。转院概率来自 "血友病家庭护理中心 "的数据和发表的文献。所有患者的药物费用均来自 "血友病家庭护理中心 "的数据。对数据进行了单向和概率敏感性分析，以评估结果的稳健性：与 OD 治疗相比，PR 的总体质量调整生命年（QALYs）更高（9.59 QALYs vs. 6.85 QALYs）。经计算，PR 与 OD 治疗相比，每获得一个 QALY 的增量成本效益比 (ICER) 约为 12,151.35 美元（81,778.55 元人民币）。这一金额低于支付意愿（WTP）阈值 38,212.74 美元（257,171.71 元人民币）。单向敏感性分析发现，研究结果对 OD 和 PR 治疗的成本很敏感：本研究表明，在中国，对于无抑制剂的中度或重度HA患儿，PR治疗与OD治疗相比具有成本效益。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Clinical therapeutics 医学-药学

CiteScore

6.00

自引率

3.10%

发文量

154

审稿时长

9 weeks

期刊介绍： Clinical Therapeutics provides peer-reviewed, rapid publication of recent developments in drug and other therapies as well as in diagnostics, pharmacoeconomics, health policy, treatment outcomes, and innovations in drug and biologics research. In addition Clinical Therapeutics features updates on specific topics collated by expert Topic Editors. Clinical Therapeutics is read by a large international audience of scientists and clinicians in a variety of research, academic, and clinical practice settings. Articles are indexed by all major biomedical abstracting databases.