尽管科学界和监管机构对此很感兴趣，但关于健康相关生活质量 (QoL) 结果、疗效结果和药物批准之间关系的数据有限。我们更新了之前发表的阶段性荟萃研究接受全身治疗的实体瘤患者的 III 期临床试验，于 2012 年至 2021 年在 11 家选定期刊上发表。对于目前的分析，我们重点关注在高级环境中进行的研究。主要结局是全球生活质量结果与研究主要终点 (EP1)、总生存期 (OS) 和无进展生存期 (PFS) 的关联，而次要结局是监管机构批准的治疗中全球生活质量阳性结果的频率[欧洲药品管理局 (EMA)/食品和药物管理局 (FDA)]。进行了描述性分析，并测试了 QoL 结果与研究和出版物特征之间的关联。从 2012 年到 2021 年，确定了 592 篇符合条件的出版物。主要终点是 298 项临床试验中的 OS（50.3%） 304 项临床试验中的 PFS 和 PFS (51.4%)。 124 项试验（41.6%）报告了 EP1 分析的阳性结果，OS 为 EP1，182 项试验（59.5%）PFS 为 EP1。在 OS 和 PFS 为阳性的研究中，总体 QoL 结果分别为 39 项 (31.5%) 和 45 项研究 (24.7%)。 FDA 和 EMA 分别批准了 143 项 (24.2%) 和 142 项研究 (24%)。其中，全球生活质量结果分别有 55 项（38.5%）和 56 项（39.4%）研究呈阳性。监管机构批准的大多数药物都有生活质量结果，但从 2012-2016 年到 2017-2021 年，全球生活质量结果呈阳性的批准比例并没有显着增加。尽管监管机构最近批准的大多数癌症治疗药物都有生活质量数据，少数研究已证明生活质量有所改善，并在主要终点方面取得了积极成果。版权所有 © 2024 作者。由爱思唯尔有限公司出版。保留所有权利。

Despite the interest from the scientific community and regulatory agencies, limited data are available on the association between health-related quality-of-life (QoL) results, outcome of efficacy and drug approvals.We updated the previously published meta-research study of phase III clinical trials in patients with solid tumours treated with systemic treatments, published from 2012 to 2021 in 11 selected journals. For the present analysis, we focused on studies conducted in the advanced setting. The primary outcome was the association of global QoL results with study primary endpoints (EP1), overall survival (OS) and progression-free survival (PFS), while a secondary outcome was the frequency of positive global QoL results among treatments approved by regulatory agencies [European Medicines Agency (EMA)/Food and Drug Administration (FDA)]. A descriptive analysis was carried out and the association between QoL results and characteristics of studies and of publications was tested.Five hundred and ninety-two eligible publications were identified from 2012 to 2021. The primary endpoint was OS in 298 clinical trials (50.3%) and PFS in 304 clinical trials (51.4%). A positive result in EP1 analysis was reported in 124 trials (41.6%) with OS as EP1 and in 182 trials (59.5%) with PFS as EP1. Among studies with positive OS and PFS, global QoL results were positive in 39 (31.5%) and 45 studies (24.7%), respectively. FDA and EMA approvals were available for 143 (24.2%) and 142 studies (24%), respectively. Among these, global QoL results were positive in 55 (38.5%) and 56 studies (39.4%), respectively. QoL results were available for most drugs approved by regulatory agencies, but the proportion of approvals with positive global QoL results was not significantly increased from 2012-2016 to 2017-2021.Despite QoL data being available for most cancer treatments recently approved by regulatory agencies, QoL improvement has been demonstrated in a minority of studies with positive results in the primary endpoint.Copyright © 2024 The Author(s). Published by Elsevier Ltd.. All rights reserved.