[论文解读] A Causal Roadmap for Generating High-Quality Real-World Evidence

Lauren Eyler Dang, Susan Gruber+24 more|arXiv (Cornell University)|May 11, 2023

Health Systems, Economic Evaluations, Quality of Life被引用 10

一句话总结

该论文提出一种显式、迭代的因果路线图，用于预先指定分析研究设计，透明评估因果假设，并比较设计/分析选项，以生成高质量的真实世界证据（RWE），用于监管和临床用途。

ABSTRACT

Increasing emphasis on the use of real-world evidence (RWE) to support clinical policy and regulatory decision-making has led to a proliferation of guidance, advice, and frameworks from regulatory agencies, academia, professional societies, and industry. A broad spectrum of studies use real-world data (RWD) to produce RWE, ranging from randomized controlled trials with outcomes assessed using RWD to fully observational studies. Yet many RWE study proposals lack sufficient detail to evaluate adequacy, and many analyses of RWD suffer from implausible assumptions, other methodological flaws, or inappropriate interpretations. The Causal Roadmap is an explicit, itemized, iterative process that guides investigators to pre-specify analytic study designs; it addresses a wide range of guidance within a single framework. By requiring transparent evaluation of causal assumptions and facilitating objective comparisons of design and analysis choices based on pre-specified criteria, the Roadmap can help investigators to evaluate the quality of evidence that a given study is likely to produce, specify a study to generate high-quality RWE, and communicate effectively with regulatory agencies and other stakeholders. This paper aims to disseminate and extend the Causal Roadmap framework for use by clinical and translational researchers, with companion papers demonstrating application of the Causal Roadmap for specific use cases.

研究动机与目标

• 在临床政策和监管决策中，推动对高质量真实世界证据（RWE）的需求。
• 提供一个统一、明确的框架（因果路线图），指导RWE研究设计的预设和评估。
• 实现因果假设的透明评估，以及在设计/分析选项之间进行客观比较。
• 通过标准化RWE生成过程，促进与监管者和相关方的沟通。

提出的方法

• 提出一个明确、逐项、迭代的过程，称为因果路线图。
• 要求在一个统一框架内对分析性研究设计进行事前规范。
• 推动对因果假设的透明评估，以及对设计/分析选项的标准。
• 基于预先指定的标准，支持对竞争设计/分析的客观比较。
• 旨在传播并扩展该框架，以用于临床与转化研究，并附有配套的用例论文。

实验结果

研究问题

• RQ1 结构化、迭代的路线图如何提高RWE研究的事前规范和透明度？
• RQ2 为确保高质量RWE，必须评估哪些因果假设，以及如何透明地评估？
• RQ3 因果路线图如何促成对RWE研究设计与分析的客观比较？
• RQ4 该框架在哪些方面能够改善与监管机构及相关方的沟通？

主要发现

• 因果路线图提供一个明确、逐项的流程，用于指导RWE研究设计和分析。
• 该框架强调对因果假设的透明评估。
• 它促进对设计和分析选项的客观、事前比较。
• 路线图旨在改善与监管机构及其他相关方的沟通。
• 本文将该路线图定位为可扩展的，附有示范实际用例的配套用例论文。