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evidence to support regulatory decision making is discussed in the FDA final guidance for medical devices (2017) and draft guidance for drugs and biologics (2019).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
President & CEO, PROMETRIKA, LLC
Dr. Miganush Stepanians is the President and CEO of PROMETRIKA, which she founded in 2003. Dr. Stepanians has more than 30 years of experience in drug development, with a specific focus on biostatistics and data management. In addition to overall responsibility for the company’s scientific and operational activities, Dr. Stepanians is a practicing biostatistician, directly involved in study design and analysis, strategic product development planning, and commercialization support. Dr. Stepanians has designed the analyses for the Integrated Summaries of Efficacy and Safety for more than 15 successful marketing applications (NDAs; MAAs) and has presented on behalf of sponsors in meetings with FDA. She has particular expertise in challenging study design and analysis problems, including adaptive design trials, and has participated as a voting or non-voting member of a number of Independent Data Monitoring Committees. Prior to founding PROMETRIKA, Dr. Stepanians served as Director of Biostatistics and Data Management at Muro Pharmaceutical/Viatris, Inc., which she joined in 1993. In this capacity, Dr. Stepanians headed a department of 20 biostatisticians, programmers and data managers. As a member of several global project teams at Muro, Dr. Stepanians was a key contributor to the design of drug development programs and had primary oversight of statistical and data management activities. She had statistical oversight for the development and submission of three successful NDAs and interacted extensively with the FDA. Prior to joining Muro, Dr. Stepanians worked in the biostatistical arena for seven years, first at the New England Medical Center and then at Boston University’s Statistics and Consulting Unit, providing statistical services for a number of pharmaceutical companies in a wide range of indications. She has authored a number of journal articles.