Background and Objectives
During public health emergencies such as influenza pandemics or chemical, biological, radiological/nuclear (CBRN) attacks, safe and effective vaccines, drugs, diagnostics, and other medical countermeasures (MCMs) are essential to protecting national security and the well-being of the public. As highlighted in the March 2010 report of the National Biodefense Science Board, “FDA has not been able to fulfill its implicit national security mission, in large part because of a lack of resources…It is imperative for America’s health and progress for FDA to be provided adequate resources to bring its regulatory science into the 21st century…Doing so will greatly enhance the FDA’s ability to support MCM development and licensing.”
In August 2010, HHS released its Public Health Emergency Medical Countermeasures Enterprise Review, which made numerous recommendations to transform the public health emergency countermeasures enterprise to increase its speed, agility, capacity, and success rate, including the promotion of regulatory innovation and investment in regulatory science at FDA. To promote regulatory innovation and investment in regulatory science at FDA, FDA has established a MCM initiative. This initiative seeks to accelerate MCM development towards approval and consists of a multifaceted action plan that includes: (1) enhancing the regulatory review process for the highest priority MCMs and related technologies; (2) advancing regulatory science to support MCM development and evaluation; and (3) modernizing the legal and regulatory framework to support public health preparedness and response. Regulatory science for MCM development and evaluation is essential for FDA to establish clear regulatory pathways for product approval based on the most advanced scientific foundations available and realize the promise of new technologies for flexible, rapidly scalable development and manufacturing of vaccines and other MCMs.
This workshop: (1) examined ways to advance regulatory science for MCM development and regulatory evaluation; (2) identified scientific opportunities to improve, simplify, or speed MCM development; and (3) identified tools and methods to improve the predictability and success rate of candidate MCMs.
Meeting Objectives:
- Provide a broad overview of current efforts underway at FDA and other agencies within HHS and DoD to support the research, development, evaluation, and production of MCMs.
- Review novel scientific methodologies used by academia and industry to facilitat development of next generation vaccines, biologics, drugs, and devices.
- Identify major gaps in currently available tools to predict and evaluate product safety, efficacy, and quality.
- Identify opportunities for collaboration and coordination with FDA and among relevant federal as well as industry programs to support the MCM initiative’s regulatory science program and to develop better defined pathways for product approval.
- Identify regulatory science tools and methodologies to address emerging technologies, targets, and novel products as well as innovative approaches for predicting safety and efficacy; for example, biomarkers and in silico modeling.
Planning Committee
Leslie Z. Benet (Co-Chair), University of California, San Francisco
John Rex (Co-Chair), AstraZeneca
Margaret Anderson, FasterCures
Phyllis Arthur, Biotechnology Industry Organization
Anthony Bahinksi, Wyss Institute for Biologically Inspired Engineering, Harvard University
Luciana Borio, U.S. Food and Drug Administration
Gail H. Cassel, Harvard Medical School
Steven K. Galson, Amgen, Inc.
John Grabenstein, Merck & Co., Inc.
Robert Kadlec, PRTM Management Consultants
Michael Katze, University of Washington
George Korche, U.S. Department of Health and Human Services
Michael Kurilla, National Institute of ALlergy and Infectious Diseases
Bernard Munos, InnoThink
Gerald Parker, Department of Defense
Mary Pendergast, Pendergast Conulsting
Nicki Pesik, Center for Disease Control and Prevention
Eric Toner, Center for Biosecurity of the UPMC
Below are materials for the workshop, including an agenda and presentations.
For additional information on the workshop, including the planning committee and sponsors, please visit our project page.