Real World Data and Evidence in the Evaluation of Medical Products
Real world data (RWD) and real world evidence (RWE) may provide insights into important scientific or clinical questions that would otherwise be difficult or impossible to answer—however, despite this great potential, a lack of consensus among stakeholders about appropriate approaches and methods for using RWD and RWE to support trials has slowed adoption.
To address this issue, CTTI conducted in-depth qualitative interviews with representatives from academia, biopharmaceutical companies, health systems, and other organizations to identify the challenges of and opportunities for incorporating RWE in clinical research. In June 2018, CTTI convened an expert meeting with key stakeholders to review the research findings and begin to develop actionable solutions. Forthcoming recommendations and resources are currently in progress, which will clarify best approaches for using RWE from electronic health records (EHRs) and claims data in randomized clinical trials for regulatory submission.
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).
CTTI’s Real World Evidence Project will increase the appropriate use of randomized clinical trials leveraging RWD sources and RWE for regulatory submissions.
|Team Member||Ani John||Genentech - a member of the Roche Group|
|Team Member||Barbara Bierer||Harvard University|
|Social Scientist||Brian Perry||Clinical Trials Transformation Initiative|
|Team Member||Cathy Critchlow||Amgen|
|Team Member||David Kronfeld||Medidata|
|Team Member||David Thompson||Syneos Health|
|Team Leader||Dianne Paraoan||Food and Drug Administration|
|Team Member||Eric Peterson||Duke University|
|Team Member||Farhan (CJ) Hameed||Pfizer Inc.|
|Project Manager||Gerrit Hamre||Clinical Trials Transformation Initiative|
|Team Leader||Jack Sheehan||Janssen|
|Team Leader||Jane Perlmutter||Individual Patient/Caregiver|
|Team Member||Jerry Heatley||Abbott|
|Team Member||Jessie Juusola||Evidation Health|
|EC Champion||Joe Selby||Patient-Centered Outcomes Research Institute|
|Team Member||John Laschinger||Food and Drug Administration|
|Team Member||Ken Carson||Flatiron|
|Team Member||Lesley Curtis*||Duke University|
|Team Member||M. Khair ElZarrad||FDA/CDER|
|Team Member||Naomi Aronson||Blue Cross Blue Shield|
|EC Champion (emeritus)||Rich Platt||Harvard University|
|Team Member||Ruthie Davi||Medidata|
|Team Member||Ryan Ferguson||Department of Veterans Affairs|
|Team Member||Sarah Leatherman||Department of Veterans Affairs|
|Team Leader||Scott Evans||Society for Clinical Trials|
|Team Leader||Sudha Raman||Duke University|