在美国食品和药物管理局 (FDA) 批准的适应症不断变化的情况下，估计上皮性卵巢癌 (EOC) 患者每年有资格接受 PARP 抑制剂治疗并从中受益的百分比。这是一项使用公开数据的模拟回顾性观察研究晚期 EOC 患者。 PARPi 资格基于 2014 年至 2023 年 FDA 的批准和撤销，以及已发布的人口和基因组数据。临床试验数据用于估计治疗效果。 PARPi 包括 olaparib、niraparib 和 rucaparib 进行汇总分析，并根据分子分类和治疗时机进行子分析。结果报告为适合任何癌症定向治疗的 EOC 患者的百分比。2014 年至 2021 年间，PARPi 被批准用于 EOC 的 9 种不同适应症；到2023年减少到6个适应症。合格率从2014年的2.0%（95% CI，1.3%-1.6%）增加到2021年的最高93.4%（95% CI，90.1%-94.6%）。患者的最大百分比2021 年，2 年 PFS 获益率为 22.0%（95% CI，17.2%-26.8%），预计到 2024 年将下降至 13.0%（95% CI，9.9%-15.9%）。这一下降大部分发生在同源重组缺陷的 BRCA 野生型人群（8.4% 至 4.0%）。PARPi 资格的增加速度高于受益率，导致 2021 年之前人群水平的受益与资格比较低。最近 FDA 的撤回提高了这一比率受益患者绝对数量随之减少。为了进一步优化卵巢癌靶向治疗的人群水平获益与资格比，我们需要确定更好的生物标志物、治疗组合和新的治疗靶点。版权所有 © 2024 作者。由爱思唯尔公司出版。保留所有权利。

To estimate the annual percentage of patients with epithelial ovarian cancer (EOC) who could be eligible for and benefit from PARP inhibitor therapy amidst changing US Food and Drug Administration (FDA)-approved indications.This is a simulated retrospective observational study using publicly available data on patients with advanced-stage EOC. PARPi eligibility is based on FDA approvals and withdrawals from 2014 through 2023, along with published demographic and genomic data. Clinical trial data is used to estimate treatment benefit. PARPi including olaparib, niraparib, and rucaparib are analyzed in aggregate with sub-analyses by molecular classification and treatment timing. Results are reported as the percentage of EOC patients appropriate for any cancer-directed therapy.PARPi were approved for 9 different indications in EOC between 2014 and 2021; reduced to 6 indications by 2023. Eligibility increased from 2.0% (95% CI,1.3%-1.6%) in 2014 to a maximum of 93.4% (95% CI,90.1%-94.6%) in 2021. The maximum percentage of patients with 2-year PFS benefit was 22.0% (95% CI, 17.2%-26.8%) in 2021, projected to decrease to 13.0% (95% CI, 9.9%-15.9%) in 2024. Most of this decrease was seen in the homologous recombination deficient, BRCA wild-type population (8.4% to 4.0%).PARPi eligibility increased at a greater rate than benefit resulting in a low population-level benefit-to-eligibility ratio until 2021. Recent FDA withdrawals improved this ratio with an accompanied decrease in the absolute number of patients benefiting. To further optimize population-level benefit-to-eligibility ratio of targeted therapies in ovarian cancer, we need to identify better biomarkers, treatment combinations, and novel therapeutic targets.Copyright © 2024 The Authors. Published by Elsevier Inc. All rights reserved.