Modernizing clinical trials is the U.S. FDA priority. As more diseases are being redefined based on genomic subtype, researchers have more novel targets and more opportunities to precisely modulate or even repair the basic biological drivers of illness. Precision guided medicines can demonstrate strong efficacy signals in early clinical trials, including in trials where small groups of patients are selected based on biomarkers or other criteria suggesting they’re likely to benefit. These trials can potentially allow earlier regulatory assessment of benefit and risk. When the FDA can make a positive approval decision, patients can gain earlier access to important new therapeutic options. To take advantage of these innovations, the FDA is also seeking new ways to modernize its approaches to accommodate these novel opportunities.
Sean Khozin is a board-certified oncologist and physician-scientist combining translational and clinical research with data science and health technology to address today’s challenges in therapeutic development. Dr. Khozin currently serves as Associate Director at FDA’s Oncology Center of Excellence, managing key aspects of the approval and regulation of oncology drugs, biologics, devices, and diagnostics. Areas of focus include novel clinical trial designs, big data analytics, real world evidence, complex phenotyping, and digital biomarker development. Dr. Khozin is also the founding Director of Information Exchange and Data Transformation (INFORMED; www.fda.gov/INFORMED), FDA’s first data science and technology incubator focused on de-risking emerging science and technology solutions by structuring strategic partnerships and supporting collaborative translational and clinical research studies to advance therapeutic development goals consistent with the agency’s public health priorities. Previously, Dr. Khozin was founder and Chief Medical Officer of SKMD, a multidisciplinary healthcare delivery network, and cofounder of HelloHealth, a health technology company specializing in building interoperable systems with telemedicine, point-of-care data visualization, and advanced analytics capabilities to optimize patient care and clinical research.
Dr. Eydelman is the Director of the Office of Ophthalmic, Anesthesia, Respiratory, ENT and Dental Devices at the FDA. She leads a large, multidisciplinary staff in the development, implementation, execution and management of the premarket, postmarket, compliance and quality programs. Dr. Eydelman received her M.D. degree from Harvard Medical School and a Doctorate in Health Sciences and Technology from Massachusetts Institute of Technology. Dr. Eydelman originated numerous symposia and workshops to facilitate medical device innovation and has been instrumental in expediting development of novel endpoints for clinical trials of pioneering technologies. She organized multi-stakeholder public-private partnerships; oversaw development of regulations, standards and guidance for industry. Dr. Eydelman has spearheaded numerous clinical and laboratory studies designed to improve the safety and effectiveness of medical devices. Dr. Eydelman has been granted a U.S. patent, published nearly 100 peer-reviewed articles, book chapters, and monographs, and presented over 250 lectures worldwide.
James Tcheng is an interventional cardiologist and informatician, and serves as Director of the Duke Cardiovascular Databank, Director of Performance Improvement for the Duke Heart Center, and CMIO of the Duke Heart Network. His work focuses on harmonizing the clinical definitions, data constructs, and associated informatics of cardiovascular clinical terminologies across academia, regulatory agencies, the life sciences industry, professional societies, and standards organizations, to improve the capture, communication, interoperability, and analysis of healthcare information. He is a key investigator of the FDA Medical Device Epidemiology Network (MDEpiNet) and principal investigator of the Pew Charitable Trusts-sponsored project, Improving Healthcare Data Interoperability. He previously chaired the American College of Cardiology Informatics and Health IT Committee.
MDEpiNet: The Journey to Real World Evidence
Despite the federal electronic health record program, real-world clinical data largely remains locked in analog documents. This talk will describe the Medical Device Epidemiology Network (MDEpiNet), a public-private partnership that is leveraging the Coordinated Registry Network paradigm for the capture of high-quality clinical data for evidence generation.
Dr. Waters is the team lead for the multi-agency/stakeholder Systemic Harmonization and Interoperability Enhancement for Laboratory Data (SHIELD) effort and the Real-World Evidence representative for the Office of In Vitro Diagnostics and Radiological Health (OIR) at FDA. He is an interdisciplinary scientific reviewer for a wide range of medical devices, with a focus on diagnostics, instrumentation and software. He leads efforts to build laboratory data infrastructure for a National Evaluation System for healthcare Technology (NEST) under the Medical Device Innovation Consortium (MDIC). Dr. Waters also participates in several data interoperability working groups, to implement infrastructure and improve lab data interoperability. Dr. Waters received his PhD from the USC Department of Molecular and Computational Biology, conducted his postdoctoral research at the National Institute of Standards and Technology (NIST) and was a Research Fellow for the National Research Council (NRC). He has over 20 years of experience in microbiology and molecular biology.