Moderators: Patrick Gee (Chesterfield, USA); Helina Kassahun (Amgen, USA); Stefan James (Stockholm, SWE); Matthew Roe (Durham, USA)
Cardiovascular disease has historically been the subject of a wide variety of study designs falling under the RWE umbrella, including long-term endpoint trials, pragmatic clinical trials, and health-economic evaluations.
Given the increasing interest in real-world evidence (RWE) generation to inform the development of new therapeutics, the underlying data sources, technology approaches, and study solutions for pragmatic trials with regulatory implications are evolving rapidly.
Real-world data (RWD) obtained from electronic health records (EHRs), administrative claims databases, or directly from patients via digital health applications and/or biosensors/wearables are increasingly being utilized to design, plan, and execute clinical trials and observational studies.
However, recent experiences with pragmatic trials and observational studies using RWD sources and technology advances have highlighted several important considerations including how to best access, utilize, and protect RWD during all phases of a clinical trial, opportunities for patients to directly acquire and share their health data from RWD sources to increase opportunities for clinical trial participation, and methodologies for assessing data quality with RWD sources.
The 21st Century Cures Act in the US has increased FDA acceptance of RWE and given rise to the term “regulatory-grade RWE.” But how is the regulatory-grade criterion for regulators and payers defined and what does it take for RWE to meet it?
Issues of data validity, data provenance, and data protection related to RWE continue to evolve.
This issue is complicated by variations in real world data RWD quality, rigor of RWD validation, analysis, and evidentiary standards for different regulatory and reimbursement decisions (post-marketing safety assessments, changes to product labeling, comparative effectiveness).
Strong collaborations among all stakeholders will be needed to conduct the clinical trials and observational studies of the future.