同源重组修复缺陷指导下的他拉唑帕尼联合恩杂鲁胺治疗转移性去势抵抗性前列腺癌患者：成本效益分析

HRR deficiency-guided use of talazoparib plus enzalutamide in patients with metastatic castration-resistant prostate cancer: a cost-effectiveness analysis.

作者信息

Rui Mingjun, Wang Yingcheng, Wei Qiran, You Joyce H S

机构信息

School of Pharmacy, Faculty of Medicine, The Chinese University of Hong Kong, Shatin, Hong Kong SAR, China.

School of Pharmacy, Faculty of Medicine, The Chinese University of Hong Kong, 8/F, Lo Kwee-Seong Integrated Biomedical Sciences Building, Shatin, Hong Kong SAR, China.

出版信息

Ther Adv Med Oncol. 2025 Jul 13;17:17588359251356109. doi: 10.1177/17588359251356109. eCollection 2025.

DOI:10.1177/17588359251356109

PMID:40661990

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12256737/

Abstract

BACKGROUND

Talazoparib plus enzalutamide showed significant improvement of progression-free survival in metastatic castration-resistant prostate cancer (mCRPC) patients with alterations in homologous recombination repair (HRR) genes.

OBJECTIVES

This study aimed to evaluate the cost-effectiveness of HRR status-guided use of talazoparib plus enzalutamide in patients with mCRPC from the U.S. payer perspective.

DESIGN

A partitioned survival model was used to simulate outcomes of a hypothetical cohort of mCRPC patients.

METHODS

Three treatment strategies were evaluated: (1) talazoparib plus enzalutamide for HRR-deficient patients and enzalutamide for non-HRR-deficient patients (HRR-guided talazoparib plus enzalutamide), (2) talazoparib plus enzalutamide for patients with or without HRR deficiency (empirical talazoparib plus enzalutamide), and (3) enzalutamide for patients with or without HRR deficiency (empirical enzalutamide). Primary outcomes were direct medical costs, life-years (LYs), quality-adjusted life-years (QALYs), and incremental cost per QALY gained (incremental cost-effectiveness ratio, ICER). Sensitivity analyses were performed to examine the robustness of model results.

RESULTS

In base-case analysis, the empirical enzalutamide strategy had the lowest cost and QALYs gained (US$752,383; 2.4764 QALYs), followed by HRR-guided talazoparib plus enzalutamide (US$878,517; 2.6490 QALYs), and empirical talazoparib plus enzalutamide (US$1,201,221; 2.8418 QALYs). The ICER of the HRR-guided talazoparib plus enzalutamide group (vs empirical enzalutamide) was US$730,671 per QALY gained, and the ICER of empirical talazoparib plus enzalutamide (vs HRR-guided talazoparib plus enzalutamide) was US$1,673,457 per QALY gained. Both ICERs of the HRR-guided strategy and empirical combination therapy were higher than the willingness-to-pay threshold (US$100,000 per QALY) and were not accepted as cost-effective. Sensitivity analyses found drug costs of talazoparib and enzalutamide to be the key influential factors on the cost-effectiveness of HRR-guided therapy.

CONCLUSION

HRR-guided talazoparib plus enzalutamide does not appear to be cost-effective for mCRPC patients from the U.S. payer perspective.

摘要

背景

他拉唑帕利联合恩杂鲁胺在同源重组修复（HRR）基因发生改变的转移性去势抵抗性前列腺癌（mCRPC）患者中显著改善了无进展生存期。

目的

本研究旨在从美国支付方的角度评估HRR状态指导下他拉唑帕利联合恩杂鲁胺用于mCRPC患者的成本效益。

设计

采用分区生存模型模拟一组假设的mCRPC患者的结局。

方法

评估了三种治疗策略：（1）HRR缺陷患者使用他拉唑帕利联合恩杂鲁胺，非HRR缺陷患者使用恩杂鲁胺（HRR指导下的他拉唑帕利联合恩杂鲁胺），（2）无论有无HRR缺陷患者均使用他拉唑帕利联合恩杂鲁胺（经验性他拉唑帕利联合恩杂鲁胺），以及（3）无论有无HRR缺陷患者均使用恩杂鲁胺（经验性恩杂鲁胺）。主要结局为直接医疗成本、生命年（LYs）、质量调整生命年（QALYs）以及每获得一个QALY的增量成本（增量成本效益比，ICER）。进行敏感性分析以检验模型结果的稳健性。

结果

在基础病例分析中，经验性恩杂鲁胺策略成本最低且获得的QALYs最少（752,383美元；2.4764个QALYs），其次是HRR指导下的他拉唑帕利联合恩杂鲁胺（878,517美元；2.6490个QALYs），以及经验性他拉唑帕利联合恩杂鲁胺（1,201,221美元；2.8418个QALYs）。HRR指导下的他拉唑帕利联合恩杂鲁胺组（对比经验性恩杂鲁胺）每获得一个QALY的ICER为730,671美元，经验性他拉唑帕利联合恩杂鲁胺（对比HRR指导下的他拉唑帕利联合恩杂鲁胺）每获得一个QALY的ICER为1,673,457美元。HRR指导策略和经验性联合治疗的两个ICER均高于支付意愿阈值（每QALY 100,000美元），不被认为具有成本效益。敏感性分析发现他拉唑帕利和恩杂鲁胺的药物成本是HRR指导治疗成本效益的关键影响因素。