Project:

Real World Evidence

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Real World Data and Evidence in the Evaluation of Medical Products

When incorporated into randomized clinical trials, “real world evidence” (RWE)—the result of combining and analyzing “real world data” (RWD) from electronic health records, payment and insurance claims databases, quality and disease registries, patient-generated data and even mobile devices—may provide insights into important scientific or clinical questions that would otherwise be difficult or impossible to answer. It may also help create a more accurate picture of patient experiences while reducing clinical trial costs and burdens.  

However, despite growing interest in leveraging the potential benefits of RWD and RWE for clinical research, there are relatively few examples to inform such efforts. A lack of consensus among stakeholders about appropriate approaches and methods for using RWD and RWE to support clinical trials has slowed progress in this field.

CTTI’s work will increase the appropriate use of randomized clinical trials leveraging RWD sources and RWE for regulatory submissions.

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The use of RWD and RWE in randomized studies offers great potential for improving the quality and efficiency of clinical trials, but lack of awareness and consensus among researchers about valid approaches to leverage these data sources has slowed adoption. The recent inclusion of RWE provisions in the 21st Century Cures Act and Prescription Drug User Fee Act VI Commitment Letter highlights the need to better examine these potential benefits. Actionable recommendations that clarify the best approaches to incorporate RWD and RWE into clinical trials will help accelerate the appropriate use of these resources and benefit all stakeholders in the clinical trials enterprise. Initial project emphasis will focus on opportunities for supporting the conduct of trials intended to support the evaluation of the safety and efficacy of new medical products, including new indications and labeling changes.

Real World Data and Evidence in the Evaluation of Medical Products (“Real World Evidence”) (2017–ongoing).

  • Describe how RWD sources such as electronic health records, payment claims, and registries can be used to support planning and execution of randomized controlled trials
  • Identify barriers and potential solutions to adoption of RWE generation
  • Identify concerns with RWD/RWE, describe how they can be addressed, and clarify when using RWD/RWE is impractical or unwise
  • Describe practical models and operational guidance for the use of RWD in randomized clinical trials to generate RWE in specific clinical trial operations activities

CTTI’s Real World Evidence Project will increase the appropriate use of randomized clinical trials leveraging RWD sources and RWE for regulatory submissions.

  • Landscape review
  • Stakeholder interviews
  • Expert meeting

Project Team Members

Rolesort descending Name Affiliation
EC Champion Joe Selby Patient-Centered Outcomes Research Institute
EC Champion (emeritus) Rich Platt Harvard University
Project Manager Gerrit Hamre Clinical Trials Transformation Initiative
Social Scientist Brian Perry CTTI
Team Lead Dianne Paraoan Food and Drug Administration
Team Leader Lesley Curtis Duke University
Team Leader Scott Evans Society for Clinical Trials
Team Leader Jane Perlmutter Individual Patient/Caregiver
Team Leader Jack Sheehan Janssen
Team Member Naomi Aronson Blue Cross Blue Shield
Team Member Ani John Genentech
Team Member Ken Carson Flatiron
Team Member Cathy Critchlow Amgen
Team Member Ruthie Davi Medidata
Team Member Sarah Leatherman Department of Veterans Affairs
Team Member Jerry Heatley Abbott
Team Member Jessie Juusola Evidation Health
Team Member John Laschinger Food and Drug Administration
Team Member Amanda Niskar Individual Patient/Caregiver
Team Member Eric Peterson Duke University
Team Member Sudha Raman Duke University
Team Member David Thompson Syneos Health
Team Member M. Khair ElZarrad FDA CDER
Team Member Ryan Ferguson Department of Veterans Affairs
Team Member Barbara Bierer Harvard University