Chan Vivien Kin Yi, Yang Runqing, Wong Ian Chi Kei, Li Xue
Department of Pharmacology and Pharmacy, Li Ka Shing Faculty of Medicine, University of Hong Kong, Hong Kong SAR, China.
Department of Medicine, School of Clinical Medicine, Li Ka Shing Faculty of Medicine, University of Hong Kong, Hong Kong SAR, China.
Front Pharmacol. 2022 Jul 11;13:891149. doi: 10.3389/fphar.2022.891149. eCollection 2022.
PARP inhibitors have shown significant improvement in progression-free survival, but their costs cast a considerable financial burden. In line with value-based oncology, it is important to evaluate whether drug prices justify the outcomes. The aim of the study was to systematically evaluate PARP inhibitors on 1) cost-effectiveness against the standard care, 2) impact on cost-effectiveness upon stratification for genetic characteristics, and 3) identify factors determining their cost-effectiveness, in four cancer types. We systematically searched PubMed, EMBASE, Web of Science, and Cochrane Library using designated search terms, updated to 31 August 2021. Trial-based or modeling cost-effectiveness analyses of four FDA-approved PARP inhibitors were eligible. Other studies known to authors were included. Reference lists of selected articles were screened. Eligible studies were assessed for methodological and reporting quality before review. : A total of 20 original articles proceeded to final review. PARP inhibitors were not cost-effective as recurrence maintenance in advanced ovarian cancer despite improved performance upon genetic stratification. Cost-effectiveness was achieved when moved to upfront maintenance in a new diagnosis setting. Limited evidence indicated non-cost-effectiveness in metastatic breast cancer, mixed conclusions in metastatic pancreatic cancer, and cost-effectiveness in metastatic prostate cancer. Stratification by genetic testing displayed an effect on cost-effectiveness, given the plummeting ICER values when compared to the "treat-all" strategy. Drug cost was a strong determinant for cost-effectiveness in most models. : In advanced ovarian cancer, drug use should be prioritized for upfront maintenance and for patients with BRCA mutation or BRCAness at recurrence. Additional economic evaluations are anticipated for novel indications.
聚（ADP-核糖）聚合酶（PARP）抑制剂已显示出无进展生存期有显著改善，但其成本带来了相当大的经济负担。根据基于价值的肿瘤学，评估药物价格是否与疗效相符很重要。本研究的目的是系统评估PARP抑制剂在四种癌症类型中的情况：1）与标准治疗相比的成本效益；2）基因特征分层对成本效益的影响；3）确定决定其成本效益的因素。我们使用指定的检索词系统检索了PubMed、EMBASE、科学网和考科蓝图书馆，检索截至2021年8月31日。符合条件的是对四种美国食品药品监督管理局（FDA）批准的PARP抑制剂进行的基于试验或模型的成本效益分析。纳入了作者已知的其他研究。对所选文章的参考文献列表进行了筛选。在评审前，对符合条件的研究进行了方法学和报告质量评估。共有20篇原创文章进入最终评审。尽管在基因分层后性能有所改善，但PARP抑制剂作为晚期卵巢癌复发维持治疗并不具有成本效益。在新诊断的情况下改为一线维持治疗时可实现成本效益。有限的证据表明在转移性乳腺癌中不具有成本效益，在转移性胰腺癌中结论不一，而在转移性前列腺癌中具有成本效益。与“全治疗”策略相比，基因检测分层显示出对成本效益的影响，因为增量成本效果比（ICER）值大幅下降。在大多数模型中，药物成本是成本效益的一个重要决定因素。在晚期卵巢癌中，应优先考虑将药物用于一线维持治疗以及复发时具有BRCA突变或BRCA样特征的患者。预计对新适应症还需进行额外的经济学评估。
