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  1. Oncology Center of Excellence

Calibrating real-world evidence studies in oncology against randomized trials: ENCORE

External Institution: Brigham and Women’s Hospital, Harvard Medical School

External Collaborators: Shirley Wang, PhD, ScM; Janick Weberpals, RPh, PhD; Sebastian Schneeweiss, MD, ScD; Sushama Kattinakere Sreedhara, MBBS, MSPH; Elyse Dicesare, BA; Massimiliano Russo, PhD; Robert Glynn, PhD, Sc.D.; Heba Edrees, PharmD; Alex Cervone, BA; Ken Kehl, MD, MPH

FDA Collaborators: Donna Rivera, PharmD, MSc; Pallavi Mishra-Kalyani, PhD; Paul Kluetz, MD; Catherine Lerro, PhD, MPH; Sonia Singh, MD

Project Start Date: October 2022

Regulatory Science Challenge

Randomized controlled trials (RCTs) are generally regarded as the gold standard for establishing the efficacy of drugs used to treat cancer. Non-interventional studies using real world data can provide complementary evidence of effectiveness; however, there are questions about what kind of data sources and which methods can be used to generate valid results.

Project Description & Goals

Building on a process co-developed with the FDA through RCT DUPLICATE, this expansion to oncology is going to emulate the approach to 12 randomized clinical trials using multiple EHR data sources. The process includes an emphasis on transparency with documented assessment of data fitness of the RWD source for each trial and conducting extensive sensitivity analyses to assess robustness of findings and trial eligibility criteria (e.g., age, race, comorbidity).

This project aims to develop methodological and data insights using pharmacoepidemiology and statistics to design observational and analysis approaches to trial emulation that may provide guidance on  potential use of real-world evidence (RWE) for regulatory science in oncology.

The objectives of this project are to: 

  1. Develop and expand a process to evaluate the ability of RWE to support evidence of effectiveness in oncology. This includes systematic evaluation of the suitability of data in relation to the study design.
  2. Demonstrate and examine the systematic process by emulating 12 oncology trials across 4 cancers and assessing the results of RCT-RWE study pairs.

Further Information


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