Back to: FDA Regulatory Education for Industry (REdI) 2024 Conference – Biologics Track
Presenter
Hector S. Izurieta, MD, MPH, PhD
Associate Director
Novel Clinical Investigations Office of Vaccines Research and Review (OVRR)
Center for Biologics Evaluation and Research (CBER)
US Food and Drug Administration (FDA)
Hector S. Izurieta, MD, MPH, PhD, is Associate Director for Novel Clinical Investigations at the Office of Vaccine Research and Review (OVRR), CBER, FDA. He has extensive U.S. and international vaccine research and public health experience, having previously worked at CDC (including in the EIS program), PAHO, WHO, Médecins Sans Frontières and other international organizations. He has authored around 100 manuscripts on real-world evidence studies of vaccine safety and effectiveness, methods for observational study research, and on the natural history of pandemics and epidemics including COVID-19, Influenza, Measles, Tetanus, Diphtheria, Pertussis, and others. His investigations have been cited extensively and were used for regulatory- decision making and public health recommendations by FDA, CDC, PAHO/WHO, and multiple national authorities.
Abstract
Hector Izurieta presents on the use of real world evidence within the FDA’s vaccine pathways, focusing on Accelerated Approval and Emergency Use Authorization (EUA). FDA employs multiple regulatory pathways for vaccines, such as traditional approval, Accelerated Approval, and animal rule approval, each demanding distinct levels of evidence for effectiveness. The EUA pathway provides a rapid route for investigational products during a public health emergency, requiring FDA to determine that known and potential benefits outweigh known and potential risks, based on the totality of scientific evidence. Real world evidence was utilized in decisions for COVID-19 vaccines under EUA, for instance, in authorizing the third dose. Accelerated Approval facilitates earlier approval for vaccines targeting serious conditions with significant advantages over existing treatments. This pathway is based on demonstrating effect on a surrogate endpoint reasonably likely to predict clinical benefit. A key requirement for Accelerated Approval is conducting adequate and well-controlled post-marketing confirmatory studies to confirm the clinical benefit, typically expected within approximately five years. An example discussed is the chikungunya vaccine approved via Accelerated Approval, which relied on seroresponse as a surrogate and requires post-licensure observational studies using real world evidence. While not always the primary route, these pathways can incorporate real world evidence when appropriate and available, though often real world evidence is used to aid in decision-making or in post-market studies rather than being the sole basis for initial approval.