FRIENDS OF CANCER RESEARCH (Friends) is launching the next phase of its Real-World Evidence pilot project after a broad stakeholder meeting in February 2019. At the meeting, the U.S. Food and Drug Administration (FDA) and various data partners expressed interest in continuing to address several challenges to advance understanding and inform future uses of the Real-World Evidence project. This next phase includes 10 health-care research organizations that will be presenting their findings, establishing recommendations for real-world endpoint analysis, and evaluating whether real-world endpoints are indicators of clinical benefit at this year’s 8th Annual Blueprint for Breakthrough Forum.
Friends is continuing its work to solve issues related to the use and understanding of real-world evidence through this project by using its existing framework to assess several front-line treatment regimens in real-world patients with advanced non– small cell lung cancer (NSCLC).
Analytic Objectives
THIS PILOT PROJECT includes three proposed analytic objectives:
- Description of demographic and clinical characteristics of patients with advanced NSCLC: This will provide a baseline understanding of the similarity/differences among the data sets to better understand what confounding factors may need to be considered when interpreting the data.
- Evaluation of select real-world endpoints among immunotherapy-containing front-line therapy regimens: This will assess the ability of real-world endpoints to measure differences in treatment effect size in front-line treatment regimens in advanced NSCLC, while also evaluating the ability of these real-world endpoints to detect differences in subgroups and treatment.
- Development of a framework proposal to assess performance of real-world endpoints in a given data set: This will evaluate the robustness and validity of real-world data to support the integration and utilization of real-world evidence in regulatory decisions.
Earlier Findings
THE INITIAL Friends’ Real-World Evidence pilot project demonstrated that different data sets 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, pilot project 2.0 will 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 data sets representing different therapeutic areas and disease types.
Ten health-care research organizations and additional stakeholders participating in this pilot project include Aetion, ASCO CancerLinQ®, Cancer Research Network, Cota, FDA, Flatiron Health, IQVIA, Mayo Clinic, McKesson, National Cancer Institute Surveillance, Epidemiology, and End Results–Medicare linked database, OptumLabs, Syapse, and Tempus.
Read more information on the project, including information on last year’s phase of the pilot project, at focr.org/rwe. ■
