Pilot Project 2.0: Surveying the Use of Real-World Endpoints

Natalie Kim | August 28, 2020

In the first post of the Real-World Evidence (RWE) blog series, we discussed Pilot Project 1.0 , and the collaborative effort to build consensus around the role of RWE in oncology. In this post, we will explore Pilot Project 2.0: Establishing the Utility of Real-World Endpoints, a key element about RWE that requires further investigation for drug development.

In Pilot Project 1.0, using aligned definitions and methodology for extraction of demographic and clinical characteristics of advanced non-small lung cancer (aNSLC) patients, we investigated if we could assess real-world endpoints, such as time to treatment discontinuation, as proxy endpoints for overall survival (OS) in RWE. Using this framework, we identified similar patient populations and patient outcomes across different RWE datasets.

On September 18, 2019, Friends of Cancer Research (Friends) and Alexandria Real Estate Equities, Inc. hosted the 8th Annual Blueprint for Breakthrough Forum, where we discussed Pilot Project 2.0 of the Real-World Evidence project. This project included 10 healthcare research organizations who presented their findings and recommendations for the future use of real-world endpoints. Currently, there is a lack of characterization about the potential role real-world endpoints can play in evaluating product effectiveness. Based off findings from Pilot Project 1.0, the goal of the second pilot was to examine if we could show, with RWE endpoints, improved direction and magnitude of treatment effect using immuno-oncology (IO) therapy compared to chemotherapy.

Given the accumulating clinical experience with IO therapies, and mounting evidence from clinical trials, it is generally expected that patients have improved responses on IO therapy compared to chemotherapy. While additional analyses will be presented this upcoming September, results of Pilot Project 2.0 trended towards improved outcomes in patients receiving IO instead of chemotherapy, which demonstrated that we were able to replicate clinical experience in a real-world study.

The overarching goal of both Pilot 1.0 and Pilot 2.0 is to create a better understanding of RWE through robust examination in a specific clinical setting that can support expanded application of this type of data. Through the pilot projects we improved knowledge of the strengths and limitations of real-world data, more effective uses of the data, and targeted application of evidence. These findings can lead to a more thorough understanding of patient care and outcomes in routine clinical practice and efficacy of new drugs in the post-market setting.

As we improve our understanding and characterization of the underlying RWE data and its uses, we can use this data to inform and support drug development, including regulatory and payer decisions. In our next blog post, we will cover the expanded application of the RWE pilot framework to studies in the United Kingdom and France.

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