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Real-World Evidence

Pilot Project 2.0: Establishing the Utility of Real-World Endpoints

Recent Updates: Friends of Cancer Research (Friends) convened a roundtable meeting 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 who helped establish key objectives, challenges, and next steps to be implemented in a 2019 RWE pilot project.

 

The Problem: There is a need for better characterization of the role real-world endpoints may play in evaluating product effectiveness. FDA recently released their RWE Program framework describing how FDA plan to advance the use of RWE to support product evaluation. Improved understanding of appropriate methodology is necessary to evaluate and assess the validity of RWE. With appropriate implementation, RWE can fill evidence gaps about the performance of medical products used in a real-world setting, including populations that may not have been represented in clinical trials. 

 

The Solution: Friends is continuing its work to solve this problem through a new RWE pilot project that uses our existing framework to assess several frontline treatment regimens in real-world patients with advanced non-small cell lung cancer (aNSCLC). This pilot project includes three proposed analytic objectives:

 

  • Description of demographic and clinical characteristics of patients with aNSCLC. This will provide a baseline understanding of the similarity/differences among the datasets to better understand what confounding factors may need to be considered when interpreting the data.
  • Evaluate select real-world endpoints among PD-(L)1-containing frontline therapy regimens. This will assess the ability of real-world endpoints to measure differences in treatment effect size in frontline treatment regimens in aNSCLC while also evaluating the ability of these real-world endpoints to detect differences in subgroups and treatment.
  • Develop a framework proposal to assess performance of real-world endpoints in a given dataset. This will evaluate the robustness and validity of real-world data (RWD) to support the integration and utilization of RWE in regulatory decisions.

 

Establishing the Utility of Real-World Endpoints Project Overview

 

Pilot Project 1.0: Operationalizing and Validating Real-World Evidence

Recent Updates: Friends of Cancer Research hosted a public meeting titled The Future Use of Real-World Evidence on this topic on July 10, 2018. The meeting whitepaper, Establishing a Framework to Evaluate Real-World Endpoints, is now officially released.

 

The Definition: Real-world evidence (RWE) is the clinical evidence derived from data on the uses and potential benefits and/or risks of a medical product outside of a traditional clinical trial. This type of data and evidence are being studied to gain an improved understanding as to how they can relate to more traditional clinical endpoints and the long-term benefits of a product.

 

The Problem: Clinical trials do not necessarily reflect the breadth of data that can be collected in a more diverse real-world setting. There has been a lot of interest in RWE; however, parameters of use and conditions of use have not been well defined. Traditional clinical endpoints may be difficult to obtain and evaluate from real-world data. New metrics may be necessary.

 

The Solution: There needs to be a standard dataset curation process and validated framework so data collection can be operationalized. Friends of Cancer Research (Friends) will convene stakeholders to curate data to determine how endpoints generated from real world data correlate with overall survival and other key indicators of disease burden from prior clinical trials. The group will use this data to help inform the development of a validation framework. This project will help inform ongoing regulatory discussions on the use of RWE by proposing a standard approach for data collection, as well as provide vital information about the long-term value of a product including its safety and efficacy.

 

Operationalizing and Validating Real-World Evidence Project Overview

 

The Background:

Friends of Cancer Research hosted a series of meetings on RWE, which resulted in the following conclusions: