Pilot Project 2.0: Establishing the Utility of Real-World Endpoints
Recent Updates: On September 18th, Friends of Cancer Research (Friends) and Alexandria Real Estate Equities hosted this year's 8th Annual Blueprint for Breakthrough Forum, showcasing Pilot Project 2.0 of Real-World Evidence project. This phase included 10 healthcare research organizations that presented their findings, establishing recommendations for real-world endpoint analysis, and evaluating whether real-world endpoints are indicators of clinical benefit. Friends is beginning a new phase of the project to expand the analyses into specific patient populations while comparing treatment options and will be presenting more recommendations based on these data sets in 2020. To read a recap of the meeting on September 18th, click here, and read below for a full project description and timeline.
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
10 Healthcare research organizations and additional stakeholders participating in this pilot project include: Aetion, ASCO CancerLinQ/Concerto HealthAI, Cancer Research Network, COTA, FDA, Flatiron Health, IQVIA™, Mayo Clinic, McKesson, NCI SEER-Medicare Linked Database, OptumLabs®, Syapse, Tempus
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
Friends of Cancer Research hosted a series of meetings on RWE, which resulted in the following conclusions:
- Numerous challenges limit the full implementation of RWE, such as ensuring consistent data extraction; data merging processes; and defining real-world endpoints
- Proposed approaches for designing prospective pilot studies were identified to help assess the contribution of RWE to regulatory decisions
- Testing the ability of real world data to address important questions in drug development will require the development of agreed upon data standards, a validation framework, and a pilot project to assess the full capabilities of data