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

Add to Calendar 12/4/2019 8:00:00 AM 12/4/2019 10:00:00 AM Closing the Real-World Evidence Gap: Pragmatic Clinical Trials & Observational Studies

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evidence to support regulatory decision making is discussed in the FDA final guidance for medical devices (2017) and draft guidance for drugs and biologics (2019).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.
MassBio, 300 Technology Square 8th Fl, Cambridge, MA 02139
Senior Medical Director, Flatiron Health
Rebecca Miksad is a medical oncologist and health outcomes researcher who serves as senior medical director at Flatiron Health. In this role, Rebecca focuses on generating real-world evidence, establishing regulatory-grade quality standards and contributing to the development of clinically-relevant methods to real-world data evaluation and analyses. Rebecca maintains a clinical practice at Boston Medical Center. A nationally recognized clinician and researcher, Rebecca regularly publishes in leading medical journals, serves on national committees and lectures internationally. Prior to joining Flatiron Health, Rebecca was an assistant professor at Harvard Medical School, senior scientist at the Institute for Technology Assessment at Massachusetts General Hospital, and director of gastrointestinal oncology and gastrointestinal oncology clinical trials at Beth Israel Deaconess Medical Center (BIDMC). Rebecca’s academic research focused on improving cancer treatment decision-making through better characterization of patient outcomes and increasing relevance of clinical trial endpoints. Rebecca earned a BA cum laude in economics from Harvard University and an MD with honors in research from Cornell University. She was a resident in internal medicine at Cornell University/New York-Presbyterian Hospital, and completed hematology and medical oncology fellowships at Harvard Medical School/BIDMC. Rebecca earned a MMS from Harvard Medical School and an MPH in Clinical Effectiveness from the Harvard School of Public Health in clinical effectiveness (with an emphasis on decision science). She also completed a post-doctoral fellowship in the Dana-Farber/Harvard Cancer Center Program in Cancer Outcomes Research Training (PCORT).