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.
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.
|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 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 Leader||Juli Tomaino||FDA CDER|
|Team Member||Kathrena Aljallad||The Life Raft Group|
|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||Alyson Karesh||Food and Drug Administration|
|Team Member||John Laschinger||Food and Drug Administration|
|Team Member||Amanda Niskar||Individual Patient/Caregiver|
|Team Member||Dianne Paraoan||Food and Drug Administration|
|Team Member||Eric Peterson||Duke University|
|Team Member||Sudha Raman||Duke University|
|Team Member||David Thompson||inVentiv Health|