Real-World Data to Generate Real-World Evidence in Regulatory Decision-Making

项目摘要 更详细地了解癌症新疗法的安全性和有效性的基本原理是 引人注目。虽然癌症治疗一直对健康和治疗质量都有风险， 在过去的十年里，治疗学发生了很大的变化。最重要的是， 癌症的生物学特征导致对可能受益的人群的定义更加微妙， 以前是可能的。随着基因组学的广泛应用和由此产生的精确分子生物学的规范化， 目标癌症的特征，并通过（虽然早期）敏感性和耐药性的标志物， 免疫治疗，无论是适应症和使用新的治疗方法是导致更高的反应 率和更有利的结果，至少从临床试验的进展来看。并行进展 医疗保健和电子健康记录（EHR）的普遍采用，加上质量的进步 生活质量（QOL）和其他患者报告的结果（PRO），使更深入的分析成为可能。 随着新疗法在整个医疗系统中的推广使用，新疗法的效果和风险也越来越大。继 鼓励和定义使用真实世界数据（RWD）以提供真实的- 世界证据（RWE），这一资助机制旨在促进捕获，组织和 分析RWD以产生RWE。我们在此提出了一个多管齐下的方法， 策略：1）重新设想如何获得稳健的、有代表性的和准确的RWD 从现有的临床试验过程; 2）有效的RWD方法如何在罕见病例中产生稳健的RWE 和不太常见的肿瘤一样。为了帮助实现这一目标，我们与Optum Life合作， 科学，既可以帮助招募具有特定分子特征的不同人群， 在各个领域拥有扩展数据库的人群。我们的目标首先是协调第三阶段， 对新疗法进行IV期评价，以获得III期标准疗法的真实证据，以及 第二，使用这种合作小组模式，建立一个新的合作小组， 真实世界数据IV期方法评估罕见肿瘤新疗法的可行性。我们 通过在短时间内开发这些严格的数据收集方法，我们将 补充平行开发的数据收集信息学方法，并为它们提供 用于比较分析的强大数据库。

PROJECT SUMMARY The rationale for a more detailed understanding of the safety and efficacy of new therapies for cancer is compelling. While cancer therapies have always had an element of risk to both well-being and to quality of life, much has changed in therapeutics in the past ten years. Most important, a greater understanding of the biological characteristics of cancer has led to more nuanced definitions of populations that may benefit than were previously possible. With greater use of genomics and resulting specification of precise molecular characteristics of target cancers, and through (though early) markers of sensitivity and resistance to immunotherapeutics, both the indications and the use of novel therapeutics is resulting in higher response rates and more favorable outcomes, at least as judged by progress in clinical trials. Parallel advances in medical care and the universal adoption of an electronic health record (EHR), coupled with advances in quality of life (QOL) and other patient-reported outcomes (PROs), have made possible a deeper analysis of the effects and the risks of new therapies as their use is diffused throughout the medical system. Following upon several guidances to the effect of encouraging and defining the use of real-world data (RWD) to provide real- world evidence (RWE), this funding mechanism is designed to foster approaches to capture, organize, and analyze RWD to produce RWE. We propose herein a many-pronged approach that brings together two strategies: 1) a reimagining of how acquisition of robust, representative, and accurate RWD could be obtained from existing clinical trials' processes; 2) how an effective RWD approach could generate robust RWE in rare and less common tumors alike. To assist in this goal, we have developed a collaboration with Optum Life Sciences, both to help recruit diverse populations with specific molecular profiles, and to characterize populations with extended databases in various domains. Our Aims are first, to harmonize Phase III and Phase IV evaluations of new therapies to obtain real-world evidence of the standard therapy in Phase III, and subsequently of the successful therapy in Phase IV; second, using this cooperative group model, to establish feasibility of a real-world data Phase IV approach to the evaluation of new therapies for rare tumors. We expect that by developing these approaches to rigorous data collection in a short timeframe, we will be complementary to informatics approaches to data collection developed in parallel and provide for them a robust database for comparative analyses.