Institut Curie, Department of Drug Development and Innovation (D3i), Paris-Saclay University, Paris & Saint-Cloud, France.
F. Hoffmann-La Roche Ltd, Basel, Switzerland.
JCO Precis Oncol. 2022 Jul;6:e2200019. doi: 10.1200/PO.22.00019.
Precision oncology, where patients are given therapies based on their genomic profile and disease trajectory, is rapidly evolving to become a pivotal part of cancer management, supported by regulatory approvals of biomarker-matched targeted therapies and cancer immunotherapies. However, next-generation sequencing (NGS)-based technologies have revealed an increasing number of molecular-based cancer subtypes with rare patient populations, leading to difficulties in executing/recruiting for traditional clinical trials. Therefore, approval of novel therapeutics based on traditional interventional studies may be difficult and time consuming, with delayed access to innovative therapies. Real-world data (RWD) that describe the patient journey in routine clinical practice can help elucidate the clinical utility of NGS-based genomic profiling, multidisciplinary case discussions, and targeted therapies. We describe key learnings from the setup of WAYFIND-R (NCT04529122), a first-of-its-kind global cancer registry collecting RWD from patients with solid tumors who have undergone NGS-based genomic profiling. The meaning of 'generalizability' and 'high quality' for RWD across different geographic areas was revisited, together with patient recruitment processes, and data sharing and privacy. Inspired by these learnings, WAYFIND-R's design will help physicians discuss patient treatment plans with their colleagues, improve understanding of the impact of treatment decisions/cancer care processes on patient outcomes, and provide a platform to support the design and conduct of further clinical/epidemiologic research. WAYFIND-R demonstrates user-friendly, electronic case report forms, standardized collection of molecular tumor board-based decisions, and a dashboard providing investigators with access to local cohort-level data and the ability to interact with colleagues or search the entire registry to find rare populations. Overall, WAYFIND-R will inform on best practice for NGS-based treatment decisions by clinicians, foster global collaborations between cancer centers and enable robust conclusions regarding outcome data to be drawn, improve understanding of disparities in patients' access to advanced diagnostics and therapies, and ultimately drive advances in precision oncology.
精准肿瘤学,即根据患者的基因组特征和疾病轨迹为其提供治疗,正在迅速发展成为癌症管理的关键部分,这得益于生物标志物匹配的靶向治疗和癌症免疫疗法的监管批准。然而,基于下一代测序(NGS)的技术已经揭示了越来越多的分子癌症亚型,这些亚型的患者人数较少,导致传统临床试验的执行和招募困难。因此,基于传统介入性研究批准新型疗法可能困难且耗时,创新性疗法的获取也会延迟。描述常规临床实践中患者治疗过程的真实世界数据(RWD)可以帮助阐明基于 NGS 的基因组分析、多学科病例讨论和靶向治疗的临床效用。我们描述了 WAYFIND-R(NCT04529122)建立过程中的关键经验,这是一个首例全球癌症登记处,收集接受 NGS 基于基因组分析的实体瘤患者的 RWD。重新审视了 RWD 在不同地理区域的“普遍性”和“高质量”的含义,以及患者招募流程、数据共享和隐私问题。受到这些经验的启发,WAYFIND-R 的设计将帮助医生与其同事讨论患者的治疗计划,增进对治疗决策/癌症护理流程对患者结局的影响的理解,并提供一个平台,支持进一步的临床/流行病学研究的设计和开展。WAYFIND-R 展示了用户友好的电子病例报告表、基于分子肿瘤委员会决策的标准化采集,以及一个提供调查员访问本地队列级数据的仪表板,并使他们能够与同事互动或搜索整个登记处以找到罕见人群。总的来说,WAYFIND-R 将为临床医生提供基于 NGS 的治疗决策的最佳实践信息,促进癌症中心之间的全球合作,得出关于结局数据的可靠结论,增进对患者获得先进诊断和治疗机会差异的理解,并最终推动精准肿瘤学的发展。