Khozin Sean, Dreyer Nancy A, Galante Dominic, Liu Raymond, Neumann Peter, Nussbaum Nathan, O'Shaughnessy Joyce, Patt Debra, Rimawi Mothaffar, Rugo Hope, Tolaney Sara M, Weiss Marisa, Brufsky Adam
Massachusetts Institute of Technology, Cambridge, MA, USA.
Phyusion LLC, 447 Broadway, 2 Floor #1104, New York, NY, 10013, USA.
Adv Ther. 2025 May 12. doi: 10.1007/s12325-025-03201-y.
Complementing randomized controlled trials, real-world evidence (RWE) from observational analyses can extend clinical insights in oncology. While healthcare stakeholders have published rigorous RWE frameworks and resources, a multidisciplinary think tank was established to further advance acceptance and use of RWE in treatment decision-making, with the focus on breast cancer (while recognizing relevance in oncology more broadly). Members discussed perceptions of RWE from a clinical perspective, across domains of data, methodology, and mindset, and "calls-to-action" for stakeholders. Agreement was reached on a primary "call-to-action," to develop clinically-relevant, patient-informed, real-world endpoints, and secondary "calls-to-action": establish a multidisciplinary consensus forum; publish examples of unique RWE value; build upon existing frameworks and resources; and tailor an approach for exhibiting utility to guideline bodies.
作为随机对照试验的补充，观察性分析得出的真实世界证据（RWE）能够拓展肿瘤学领域的临床见解。尽管医疗保健利益相关者已发布了严谨的RWE框架和资源，但仍成立了一个多学科智囊团，以进一步推动RWE在治疗决策中的接受度和应用，重点关注乳腺癌（同时认识到其在更广泛的肿瘤学领域的相关性）。成员们从临床角度，在数据、方法和思维方式等领域讨论了对RWE的看法，以及对利益相关者的“行动呼吁”。就主要的“行动呼吁”达成了共识，即制定与临床相关、以患者为导向的真实世界终点指标，以及次要的“行动呼吁”：建立一个多学科共识论坛；发布独特RWE价值的示例；在现有框架和资源的基础上进行拓展；并为向指南制定机构展示实用性量身定制一种方法。
