Friends of Cancer Research (Friends) convened a roundtable meeting on February 6, 2019, to identify key challenges and next steps to further characterize how real-world evidence (RWE) can fill evidence gaps about the performance of approved agents used in the real-world setting, including in populations that may not have been included in initial clinical trials. Roundtable participants included the FDA, leading data vendors, academia and research institutions, and other key stakeholders to better understand key challenges and develop objectives and next steps to be implemented in a 2019 RWE pilot project.

The overarching goal of the discussion was to establish a framework to help develop standardized definitions, measurements, and approaches to assess validity of key endpoints in a defined context.

Discussions at the roundtable help identified several key challenges that are listed below:

1. Data Quality and Standards. Currently, there are no uniform data standards or standardized real-world endpoint definitions in place to assess the fitness of the data for a specific RWE regulatory use-case and measure and report the quality of the underlying data.
2. Missing Data Elements in EHR/Medical Claims and Data Linkage Issues. No one data source may contain all necessary data elements. Additionally, challenges exist with missing data that may restrict analyses or compromise findings. The establishment of a common data model to integrate data or allow comparisons across health systems and data sources for specific use cases may be needed to facilitate more complete analyses.
3. Sensitivity of Real-World Endpoints and Ability to Detect Change. There is a need to demonstrate the ability of real-world endpoints to detect differences across groups. Studies are needed to establish how real-world endpoints relate to more traditional regulatory endpoints.
4. Requirements for Incorporating RWE into Regulatory Decisions. Transparency about study design and analysis is critical to ensure confidence in RWE results. Consistent and transparent mechanisms for pre-specifying analysis plans and an analytical framework to assess RWE (reliability, validity, etc.) are needed. These processes need to be broadly applicable to support the use of RWE for regulatory decisions across therapeutic areas

After discussing these key challenges in depth, roundtable participants identified several approaches and strategies to be employed in a pilot project. The project will help address several of these challenges and advance our understanding and inform uses of RWE.

The initial Friends’ RWE pilot project demonstrated that several different datasets could be used to extract several real-world endpoints in a relatively consistent manner.  In order to further characterize the role real-world endpoints may play in measuring treatment effect size, a subsequent pilot is in development to examine the ability of different real-world endpoints to detect treatment effectiveness in real-world patient populations. Additionally, data from this pilot project will help inform a framework containing key data elements, endpoint definitions, and algorithms that could be applied to other datasets representing different therapeutic areas and disease types.

Click here to learn more about our RWE framework and new pilot project.