The Webinar

Pediatric Extrapolation presentation slides

Lynne Yao, MD is Director, Division of Pediatric and Maternal Health in the Office of New Drugs, Center for Drug Evaluation and Research, US Food and Drug Administration (FDA). Dr. Yao started at FDA as a Medical Officer and primary reviewer on the Inborn Errors of Metabolism team in the Division of Gastroenterology and Inborn Errors Products (DGIEP) in 2008 and was a team leader in DGIEP from 2009-2012. Dr. Yao graduated from the George Washington University School of Medicine, completed residency in Pediatrics at Walter Reed Army Medical Center, and fellowship in Pediatric Nephrology at the Georgetown University Children’s Medical Center. Dr. Yao is board certified in both Pediatrics and Pediatric Nephrology.

Vaccine Development presentation slides

The development of novel vaccines designed to prevent disease caused by infectious agents is a highly complex process. It is characterized by a large set of parallel development risks ranging from uncertainty over the nature of the desired immune response to uncertainty over the ability to fully control at-scale manufacturing. In “normal” times, the investment risks (financial and opportunity) are mitigated by addressing the challenges in a roughly linear fashion to de-risk the uncertainties and increase the relative probability of success over time. As such, it is not unusual for a novel vaccine development program to take up to ten years and occasionally more. The development process requires a high level of control, continuous quantitative characterization, and rigorous regulatory supervision. The presentation will discuss the “nature” of prophylactic vaccines and how this nature underlies specific development challenges and uncertainties. The presentation will also discuss the unique circumstances surrounding the development of COVID vaccines, and specifically the extraordinary challenges associated with the goal of developing and scaling-up an effective vaccine in less than one year(!). The discussion will conclude with lessons learned that will (hopefully) help in the vaccine response to the next pandemic.

Emilio A. Emini, PhD is the Director of the Tuberculosis and HIV Program at the Bill and Melinda Gates Foundation. In this role, he leads the foundation’s efforts to control the global TB and HIV epidemics. Emilio served as Senior Vice President of Vaccine Research and Development at Pfizer Inc., a position into which he transitioned after Pfizer's acquisition of Wyeth Pharmaceuticals in late 2009. At Wyeth, he led the R&D program that resulted in licensure of Prevenar 13, Pfizer’s vaccine for the prevention of pneumococcal disease. Emilio joined the pharmaceutical industry in 1983 at the Merck Research Laboratories where, as Executive Director of the Department of Antiviral Research, he led the biological research that resulted in the development of one of the first highly active antiretroviral therapies for the treatment of HIV infection. As head of vaccine research at Merck beginning in 1996, he led a research team that was involved in the successful development of multiple pediatric and adolescent vaccines. In 2006, Emilio was awarded the Distinguished Alumnus Award from the Cornell University Graduate School of Medical Sciences. He is a Fellow of the American Academy of Microbiology, a Fellow of the International Society for Vaccines, a Fellow of the College of Physicians of Philadelphia, and a former Trustee of the National Foundation for Infectious Diseases.

RWD presentation slides - Mark Levenson

RWE presentation slides - Nirosha M. Lederer

Levenson

Emerging sources of digital health data, including electronic medical records, app-based data, and wearable device data, may be leveraged to address regulatory questions concerning prescription drugs. FDA has extensive experience using digital health data to address questions on the safety of drugs. More recently, FDA has initiated efforts to explore the use of these data sources, known as real-world data, to address questions on the efficacy of drugs. This talk will review these efforts and apply the regulatory standard for the demonstration of efficacy to the evaluation of studies based on real-world data.

Mahendraratnam Lederer

COVID-19 has disrupted drug development, evidence generation, and care-delivery. This presentation will provide an overview of how COVID-19 has accelerated the generation and adoption of real-world data (RWD)/real-world evidence (RWE) for drug development and its potential for the future.

Mark Levenson is the Director of the Division of Biometrics 7 in the Office of Biostatistics/Office of Translational Sciences/Center for Drug Evaluation and Research of the U.S. Food and Drug Administration (FDA). At FDA, he has been the primary reviewer or secondary reviewer on many major pre-market and post-market drug safety problems. He contributes to statistical policy and guidance development in the areas of drug safety and real-world evidence. He is a member of the CDER Medical Policy Program Review Committee and the FDA Real-World Evidence Committee. Dr. Levenson received a PhD in Statistics from the University of Chicago and BA from Cornell University in Mathematics.

Nirosha Mahendraratnam Lederer, PhD, MSPH is the Director of Real-World Evidence RWE Strategy at Aetion. Immediately prior, she was a Managing Associate at the Duke-Margolis Center for Health Policy, where she helped to lead the Center’s RWE portfolio. Previously she served as a Subject Matter Expert in the Oncology Center of Excellence at FDA. While there, she helped implement patient-focused drug development in cancer products including clinical trial study design and product review, as well as foster consensus across U.S. and ex-U.S. healthcare stakeholders on best practices for patient-reported outcome capture, analysis, and communication. Dr. Lederer has over a decade of pharmaceutical policy and health economics and outcomes research experiences including providing evidence-generation advisory services at Avalere Health, working in commercial and medical roles at Genentech and Bristol-Myers Squibb, respectively, and serving on Capitol Hill during the passage of the Affordable Care Act. She received her PhD in Health Outcomes and Policy from the UNC Chapel Hill with a focus on large database analyses and decision-sciences. She received her MSPH in Health Policy & Management from the Johns Hopkins Bloomberg School of Public Health and BA in Public Health from the Johns Hopkins University.