Closing the Real-World Evidence Gap: Pragmatic Clinical Trials & Observational Studies

December 4, 2019 8:00 AM - 10:00 AM

MassBio, 300 Technology Square 8th Fl, Cambridge, MA 02139

Clinical trials are performed at specialized sites, with entry criteria that selects subjects likely to show efficacy but who are not necessarily representative of the broad population of patients. There is increasing concern on the part of regulators that explanatory clinical trials, which confirm a clinical hypothesis, tend to overestimate the effectiveness and underestimate the safety of medicinal products and devices. The use of real-world evidence to support regulatory decision making is discussed in the FDA final guidance for medical devices (2017) and draft guidance for drugs and biologics (2019). The guidances outline prospective pragmatic clinical trials (PCTs) and retrospective observational studies as the means to bridge the gap between explanatory trials and adoption evaluation. In this forum, our panel of experts will discuss the evolution and future of PCTs and observational studies as important tools for integration of real-world evidence in medical product development.

Sponsored by the Biostatistics, Data Management & Clinical Trials (BSDMCT) Forum Working Group.