European Organisation for Research and Treatment of Cancer (EORTC), Brussels, Belgium; Clinical Pharmacology and Pharmacotherapy Research Unit, Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium.
European Organisation for Research and Treatment of Cancer (EORTC), Brussels, Belgium.
ESMO Open. 2023 Apr;8(2):100878. doi: 10.1016/j.esmoop.2023.100878. Epub 2023 Feb 21.
Real-world data (RWD) have quickly emerged as an important source of information to address uncertainties about new treatments, including novel anticancer therapies. Many stakeholders are using such data and the evidence derived therefrom to answer the questions that remain about the safety and effectiveness of antitumor medicines after their approval by regulators. Our objective was to investigate the academic RWD study landscape and explore to what extent RWD are being integrated into investigator-initiated clinical research.
We designed an online survey that was distributed between May and August 2022 to representatives of cancer cooperative groups active in Europe, North America, South America, Asia, and/or Oceania.
In total, 125 cooperative groups operating in 58 different countries and conducting research across 13 distinct cancer domains participated in the survey. While most of the responders (67.2%) did not have a formal policy in place to gather and utilize RWD, a majority (68.0%) had carried out studies involving the analysis of such data before, both for exploratory and confirmatory purposes. The groups that were experienced in capturing and interpreting RWD had mainly worked with observational RWD that were not predominantly prospective or retrospective in nature and which originated from disease registries, electronic health records, and patient questionnaires. They perceived the low costs and the large scale of RWD research to be its most significant benefits, and viewed the accompanying methodological and operational challenges as its biggest constraints. However, they did not have a common understanding of what RWD were. Despite their experience with analyzing RWD, their research portfolio still primarily comprised traditional clinical trials; 62.5% of the groups that had never undertaken any RWD studies were nonetheless planning to initiate them in the future.
Cancer cooperative groups are already incorporating RWD studies into their research agendas, but still lack knowledge and expertise in this regard, and do not agree on what RWD are. The conduct of conventional clinical trials continues to be their priority.
真实世界数据（RWD）迅速成为解决新疗法不确定性的重要信息来源，包括新型抗癌疗法。许多利益相关者正在使用这些数据及其从中得出的证据，来回答监管机构批准后关于抗肿瘤药物安全性和有效性的遗留问题。我们的目的是调查学术 RWD 研究现状，并探讨 RWD 在多大程度上被纳入研究者发起的临床研究中。
我们设计了一份在线调查，于 2022 年 5 月至 8 月分发给在欧洲、北美、南美、亚洲和/或大洋洲活跃的癌症合作组的代表。
共有 125 个合作组分布在 58 个不同的国家，在 13 个不同的癌症领域开展研究，参与了这项调查。虽然大多数（67.2%）的回应者没有制定正式的政策来收集和利用 RWD，但大多数（68.0%）之前已经进行过涉及此类数据分析的研究，包括探索性和验证性研究。有经验的 RWD 捕获和解释者主要使用观察性 RWD，这些数据主要不是前瞻性或回顾性的，来自疾病登记处、电子健康记录和患者问卷。他们认为 RWD 研究的最大优势是成本低、规模大，并认为随之而来的方法和操作方面的挑战是其最大的限制。然而，他们对 RWD 的含义并没有一个共同的理解。尽管他们有分析 RWD 的经验，但他们的研究组合仍然主要由传统的临床试验组成；62.5%的从未进行过任何 RWD 研究的小组仍计划在未来开展此类研究。
癌症合作组已经将 RWD 研究纳入其研究议程，但在这方面仍然缺乏知识和专业知识，并且对 RWD 是什么没有达成共识。进行传统的临床试验仍然是他们的首要任务。
