Ge Chenghao, Guo Kaiyuan, Li Yi, Li Guanqiao, Zhang Hong, Yang Jiaxuan, Liu Yang, Yin Chen, Liu Sen, Xie Songmei, Chen Xiaoyuan
Tsinghua Clinical Research Institute, School of Medicine, Tsinghua University, Beijing, China.
School of Medicine, Tsinghua University, Beijing, China.
EClinicalMedicine. 2023 Apr 6;59:101953. doi: 10.1016/j.eclinm.2023.101953. eCollection 2023 May.
With the growing notion of patient-focused drug development, the quality of life and other patient-reported outcomes (PROs) of cancer patients are gaining considerable attention. Several drug regulatory agencies, including the U.S. Food and Drug Administration (FDA), are calling attention to PROs. This review aims to comprehensively characterise the application of PROs and regulatory considerations for PROs in the FDA-approved novel oncology drugs.
The FDA review documents and labels for novel oncology drugs approved from July 2017 to July 2022 were retrieved. We collected and analysed drug approval information, types of endpoints for PROs, PRO measures, designs of trials including PROs, and regulatory comments on PRO-related contents.
Results demonstrated that PROs were used more commonly for solid tumours than hematologic malignancies, which might be correlated with the disease characteristics. We further categorised and analysed existing PRO measures, providing insight for tool selection in future oncology trial design. Our findings also indicated that PROs currently do not play a significant role in oncology drug approvals. The major deficiencies related to PROs commented on by FDA reviewers were analysed, followed by recommendations for improvements.
This review demonstrates that PROs currently do not play a significant role in oncology drug marketing review, and how they can be used to support the approval of new oncology drugs is still in the exploratory stage. This current situation is not only related to the deficiencies in the design and implementation of PRO-related contents in oncology trials, but more importantly, it is a reminder that we should pay more attention to patient experience in the development of oncology drugs.
This study was not supported by any funding.
随着以患者为中心的药物研发理念日益深入人心，癌症患者的生活质量及其他患者报告结局（PROs）受到了广泛关注。包括美国食品药品监督管理局（FDA）在内的多个药品监管机构都在关注PROs。本综述旨在全面描述PROs在FDA批准的新型肿瘤药物中的应用情况以及相关监管考量。
检索了2017年7月至2022年7月期间FDA批准的新型肿瘤药物的审评文件和标签。我们收集并分析了药物批准信息、PROs的终点类型、PROs测量方法、包含PROs的试验设计以及对PROs相关内容的监管意见。
结果表明，PROs在实体瘤中的应用比血液系统恶性肿瘤更为普遍，这可能与疾病特征相关。我们进一步对现有的PROs测量方法进行了分类和分析，为未来肿瘤试验设计中的工具选择提供了参考。我们的研究结果还表明，PROs目前在肿瘤药物批准中并未发挥重要作用。分析了FDA审评人员指出的与PROs相关的主要缺陷，并提出了改进建议。
本综述表明，PROs目前在肿瘤药物上市审评中并未发挥重要作用，其如何用于支持新型肿瘤药物的批准仍处于探索阶段。这种现状不仅与肿瘤试验中PROs相关内容的设计和实施缺陷有关，更重要的是，它提醒我们在肿瘤药物研发中应更加关注患者体验。
本研究未获得任何资金支持。
