March 29, 2011
Venable Conference Center
E11200 Capitol Room
575 7th Street, N.W.
Washington, DC 20004
Background:
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, the Department of Health and Human Services (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 an 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 will (1) examine ways to advance regulatory science for MCM development and regulatory evaluation; (2) identify scientific opportunities to improve, simplify, or speed MCM development; and (3) identify 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 the Department of Defense (DoD) to support the research, development, evaluation, and production of MCMs.
- Review novel scientific methodologies used by academia and indusry to facilitate 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.
| 8:00 a.m. | Welcome and Introductions |
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LESLIE BENET, Workshop Co-Chair |
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Professor, School of Pharmacy |
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University of California, San Francisco |
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JOHN REX, Workshop Co-Chair |
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Infection Clinical Vice President |
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AstraZeneca |
| 8:10 a.m. | Keynote Address: Importance and Promise of Regulatory Science and Charge to Workshop Participants |
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JESSE GOODMAN |
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Chief Scientist, Deputy Commissioner for Science and Public Health |
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Food and Drug Administration |
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| 8:25 a.m. | Enterprise Activities and Needs for MCM Regulatory Science |
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GEORGE KORCH |
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Acting Principal Deputy Assistant Secretary for Preparedness and Response |
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Office of the Assistant Secretary for Preparedness and Response |
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Department of Health and Human Services |
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| 8:35 a.m. | Overview of the MCM Initiative: Challenges and Opportunities |
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LUCIANA BORIO |
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Acting Director, Office of Counterterrorism & Emerging Threats |
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Senior Advisor for Medicine and Public Health, Office of the Chief Scientist |
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Office of the Commissioner, Food and Drug Administration |
SESSION I: ENTERPRISE AND STAKEHOLDER PERSPECTIVES ON NEEDS TO ADVANCE MEDICAL COUNTERMEASURE REGULATORY SCIENCE
Session Objectives:
- Provide a broad overview of current efforts under way at FDA and within HHS and DoD to support the research, development, evaluation, and production of MCMs.
- Provide a broad overview of MCM development challenges and where regulatory science can advance MCM development.
- Identify and discuss the highest-priority MCM regulatory science needs to advance MCM development.
Presentation/Panel 1: FDA
| 8:45 a.m. | Session Introduction and Panel Objectives |
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GEORGE KORCH |
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Acting Principal Deputy Assistant Secretary for |
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Preparedness and Response |
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Office of the Assistant Secretary for Preparedness and Response |
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Department of Health and Human Services |
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| 8:50 a.m. | Presentations: Overview of Enterprise MCM Regulatory Science Agendas: What does the agency need? |
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CAROLYN WILSON |
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Associate Director for Research |
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Center for Biologics Evaluation and Research |
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Food and Drug Administration |
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SUSAN McCUNE |
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Deputy Director, Office of Translational Sciences |
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Center for Drug Evaluation and Research |
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Food and Drug Administration |
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MURRAY MALIN |
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Center for Devices and Radiological Health |
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Food and Drug Administration |
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| 9:50 a.m. | Discussion/Question and Answer Session |
| 10:20 a.m. | BREAK |
| Presentation/Panel 2: Enterprise and Other Stakeholders | |
| 10:35 a.m. | Panel Introduction and Objectives |
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MARY PENDERGAST |
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President |
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Pendergast Consulting |
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| 10:40 a.m. | Presentations and Panel Discussion: What do the partners need? |
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GERALD PARKER |
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Deputy Assistant to the Secretary of Defense for Chemical and Biological Defense Programs |
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Department of Defense |
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MICHAEL KURILLA |
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Director, Office of BioDefense Research Affairs |
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Associate Director for BioDefense Product Development |
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National Institute of Allergy and Infectious Disease |
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National Institutes of Heath |
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RICHARD HATCHETT |
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Chief Medical Officer and Deputy Director |
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Biomedical Advanced Research and Development Authority |
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Office of the Assistant Secretary for Preparedness and Response |
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Department of Health and Human Services |
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MAY CHU |
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Director, Laboratory Science Policy & Practice Program Office |
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Centers for Disease Control |
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RICK LYONS |
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Director, Infectious Disease Research Center |
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Chief Scientific Officer, Infectious Disease SuperCluster Colorado State University |
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ERIC ROSE |
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CEO and Chair, Board of Directors |
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SIGA Technologies, Inc. |
| 11:45 a.m. | Discussion/Question and Answer Session |
| 12:15 p.m. | LUNCH |
SESSION II: CUTTING-EDGE EFFORTS TO ADVANCEMEDICAL COUNTERMEASURE REGULATORY SCIENCE
Session Objectives:
- Review novel scientific methodologies used by academia and industry to discover and develop next generation vaccines, biologics, drugs, and devices.
- Identify major gaps in currently available tools to predict and evaluate product safety, efficacy, and quality.
- Identify approaches to developing regulatory science tools and methodologies to address emerging technologies, targets, and novel products as well as innovative approaches for predicting safety and efficacy.
- Discuss a path forward for the MCM regulatory science agenda.
- Discuss metrics to be used for gauging success of a scientific agen1da for evaluating MCMs.
- Examine how partnerships and other collaborative approaches can facilitate the advancement and ongoing support of regulatory science for MCM development, evaluation, and utilization.
| 1:00 p.m. | Session Introduction and Objectives |
|
LESLIE BENET, Workshop Co-Chair |
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University of California, San Francisco |
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JOHN REX, Workshop Co-Chair |
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AstraZeneca |
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Panelists will |
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PANEL: EX VIVO APPROACHES TO MODELING EFFICACY
| 1:05 p.m. | Panel Objectives and Introduction |
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LAUREN BLACK, panel chair |
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Senior Scientific Advisor, Navigators |
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Charles River Laboratories |
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| 1:10 p.m. | Panel Discussion: Animal Models, In Silico Models, Biomarkers |
| In Silico Models | |
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RAMON FELCIANO |
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Founder & SVP of Research |
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Ingenuity Systems |
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| Biomarkers | |
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N. LEIGH ANDERSON |
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Founder & CEO |
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Plasma Proteome Institute |
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| Animal Models | |
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ELIZABETH LEFFEL |
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| 1:50 p.m. | Discussion with Invited Discussants and Workshop Attendees |
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DRUSILLA BURNS |
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Chief, Laboratory of Respiratory and Special Pathogens |
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Center for Biological Evaluation and Research |
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JUDY HEWITT |
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Chief, Biodefense Research Resources Section |
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Office of Biodefense Research Affairs |
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DMID/NIAID/NIH |
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VIKRAM PATEL |
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Office of Testing and Research |
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Office of Pharmaceutical Sciences, CDER |
PANEL: SAFETY AND REAL-TIME MONITORING
| 2:25 p.m. | Panel Objectives and Introduction |
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CARL PECK, panel chair |
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Professor, Pharmacology and Medicine |
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University of California, San Francisco |
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| 2:30 p.m. | Panel Discussion: Post Deployment Surveillance and Side Effects; Toxicology Markers |
| Toxicology Markers | |
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ROBERT HOUSE |
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President |
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DynPort Vaccine Company LLC |
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| Post Deployment Surveillance and Side Effects | |
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KENNETH MANDL |
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| 2:50 p.m. | Discussion with Invited Discussants and Workshop Attendees |
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MARIETTA ANTHONY |
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Predictive Safety Testing Consortium |
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Critical Path Institute |
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RICHARD FORSHEE |
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Associate Director for Research |
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Office of Biostatistics and Epidemiology |
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Center for Biologics Evaluation and Research |
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HENRY FRANCIS |
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Center for Drug Evaluation and Research |
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Food and Drug Administration |
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ROBERT C. NELSON |
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Product Safety Assurance Services, Inc. |
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DONNA MENDRICK |
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National Center for Toxicological Research |
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Food and Drug Administration |
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| 3:20 p.m. | BREAK |
PANEL: DIAGNOSTICS AND STATISTICAL TECHNIQUES
| 3:35 p.m. | Panel Objectives and Introduction |
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BRUCE BURLINGTON, panel chair |
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Independent Consultant |
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| 3:40 p.m. | Panel Discussion: Diagnostics |
| Diagnostics | |
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LT. COL. DANIEL WATTENDORF |
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U.S. Air Force Program Manager, Defense Sciences Office DARPA |
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| 3:50 p.m. | Discussion with Invited Discussants and Workshop Attendees |
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CHARLES DAITCH |
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Chief Executive Officer |
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Akonni Biosystems |
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DAVID ECKER |
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Founder, DVP and Carlsbad General Manager |
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Ibis Biosciences |
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SALLY HOJVAT |
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Center for Diagnostics and Radiological Health, FDA |
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| 4:25 p.m. | Panel Discussion: Statistical Techniques |
| Statistical Techniques | |
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STEPHEN RUBERG |
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Distinguished Research Fellow & Scientific Leader, Advanced Analytics Eli Lilly & Co. |
| 4:35 p.m. | Discussion with Invited Discussants and Workshop Attendees |
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ESTELLE RUSSEK-COHEN |
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Acting Division Director, Division of Biostatistics |
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Office of Biostatistics and Epidemiology, CBER/FDA |
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JEFFREY WETHERINGTON |
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Research Director, Statistics |
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GlaxoSmithKline |
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| 5:00 p.m. | ADJOURN |
March 30, 2011
Venable Conference Center
E11200 Capitol Room
575 7th Street, N.W.
Washington, DC 20004
SESSION II, CONT’D: CUTTING-EDGE EFFORTS TO ADVANCEMEDICAL COUNTERMEASURE REGULATORY SCIENCE
Session Objectives:
- Review novel scientific methodologies used by academia and industry to discover and develop next generation vaccines, biologics, drugs, and devices.
- Identify major gaps in currently available tools to predict and evaluate product safety, efficacy, and quality.
- Identify approaches to developing regulatory science tools and methodologies to address emerging technologies, targets, and novel products as well as innovative approaches for predicting safety and efficacy.
- Discuss a path forward for the MCM regulatory science agenda.
- Discuss metrics to be used for gauging success of a scientific agenda for evaluating MCMs.
- Examine how partnerships and other collaborative approaches can facilitate the advancement and ongoing support of regulatory science for MCM development, evaluation, and utilization.
| 8:00 a.m. | Welcome and Day 2 Overview |
|
LESLIE BENET, Workshop Co-Chair |
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University of California, San Francisco |
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JOHN REX, Workshop Co-Chair |
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AstraZeneca |
PANEL: ANTIMICROBIALS, VACCINES, AND VACCINE ADJUVANTS
| 8:15 a.m. | Panel Objectives and Introduction |
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LINDA A. MILLER, panel chair |
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Director, Clinical Microbiology |
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GlaxoSmithKline |
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| 8:20 a.m. | Panel Discussion: Antimicrobials, Vaccines, and Vaccine Adjuvants |
| Antimicrobials | |
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KEVIN JUDICE |
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CEO & Chief Scientific Officer |
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Achaogen |
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| Vaccines | |
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ALAN MAGILL |
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Director, Division of Experimental Therapeutics |
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Walter Reed Army Institute of Research |
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| Adjuvants | |
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DEBBIE DRANE Senior VP R&D CSL Biotherapies |
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| 9:00 a.m. | Discussion with Invited Discussants and Workshop Attendees |
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ED COX |
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Center for Drug Evaluation and Research |
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Food and Drug Administration |
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DAVID FRUCHT |
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Capt., U.S. Public Health Service |
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Chief, Laboratory of Cell Biology |
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Division of Monoclonal Antibodies |
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CDER/FDA |
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BASIL GOLDING |
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Center for Biologics Evaluation and Research |
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HANA GOLDING |
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Chief, Laboratory of Retrovirus Research, Division of Viral Products |
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Office of Vaccines Research and Review, CBER |
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ED NUZUM |
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Chief, Biodefense Vaccines & Other Biological Products Development Section |
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Office of Biodefense Research Affairs |
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DMID/NIAID/NIH |
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ALAN SHAW |
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Chief Scientific Officer |
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Vaxinnate |
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| PANEL: THE FUTURE | |
| 9:45 a.m. | Panel Objectives and Introduction |
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GIGI KWIK GRONVALL, panel chair |
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Senior Associate, Center for Biosecurity |
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UPMC |
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| 9:50 a.m. | Panel Discussion: Synthetic Biology, Computational Biology, and Platform Technologies |
| Synthetic Biology | |
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JOHN GLASS Senior Scientist J. Craig Venter Institute |
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| Computational Biology | |
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MICHAEL KATZE |
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Professor of Microbiology |
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University of Washington |
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| Platform Technology | |
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PAT IVERSEN |
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Senior Vice President of Research & Innovation AVI BioPharma |
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| 10:30 a.m. | Discussion with Invited Discussants and Workshop Attendees |
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WILLIAM FOGLER |
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Senior Director, Portfolio Analysis and Planning |
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Intrexon Corporation |
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DONNA MENDRICK |
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Director, Division of Systems Biology |
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National Center for Toxicological Research |
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Food and Drug Administration |
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HARVEY RUBIN |
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Executive Director, Institute for Strategic Threat Analysis and Response |
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University of Pennsylvania |
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| 11:00 a.m. | BREAK |
SESSION III: MEDICAL COUNTERMEASURE REGULATORYSCIENCE NEEDS FOR AT-RISK POPULATIONS
Session Objectives:
- Identify needs that are specific or unique to at-risk populations (e.g., pediatric populations) that should be considered in developing a regulatory science agenda for MCM development.
- Provide an overview of where regulatory science can advance MCM development for these populations.
- Discuss a MCM regulatory science agenda for at-risk populations.
| Panelists will | |
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| 11:15 a.m. | Introduction and Session Objectives |
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JOHN BRADLEY, panel chair |
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Director, Infectious Diseases |
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Rady Children’s Hospital San Diego |
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| 11:20 a.m. | Panel Discussion |
| Pediatric | |
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DAVID SIEGEL |
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NICHD |
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| Pregnancy/OB-Gyn | |
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CHRISTIAN R. MACEDONIA Joint Chiefs of Staff, the Pentagon |
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| 11:45 a.m. | Discussion with Attendees |
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PRAVIN JADHAV Center for Drug Evaluation and Research Food and Drug Administration |
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NANCY MESSONNIER Centers for Disease Control |
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LISA MATHIS |
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Center for Drug Evaluation and Research |
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Food and Drug Administration |
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ROBERT NELSON |
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Senior Pediatric Ethicist/Lead Medical Officer |
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Office of Pediatric Therapeutics |
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Office of the Commissioner, Food and Drug |
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Administration |
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| 12:30 p.m. | LUNCH |
SESSION IV: FUTURE DIRECTIONS—DISCUSSION WITHWORKSHOP PARTICIPANTS AND ATTENDEES
Session Objectives: Discuss what opportunities and challenges exist to implementing the models discussed at the workshop for advancing regulatory science. What should be on the agenda for regulatory science for development and evaluation of MCMs? Discuss strategies and needs to implement the MCM regulatory science agenda.
|
LESLIE BENET, Workshop Co-Chair |
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University of California, San Francisco |
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JOHN REX, Workshop Co-Chair |
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AstraZeneca |
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| 1:30 p.m. | Discussion with Panelists and Workshop Attendees led by workshop co-chairs |
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PHYLLIS ARTHUR |
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Director, Health & Regulatory Affairs |
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Biotechnology Industry Organization |
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GAIL CASSELL, Drug Forum Co-Chair |
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Visiting Professor, Department of Social Medicine, |
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Harvard Medical School Vice President, TB Drug Discovery, Infectious Disease |
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Research Institute, Seattle |
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RICHARD HATCHETT |
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Chief Medical Officer and Deputy Director |
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Biomedical Advanced Research and Development Authority |
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Office of the Assistant Secretary for Preparedness and Response |
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Department of Health and Human Services |
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CARL PECK |
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Professor, Pharmacology and Medicine |
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University of California, San Francisco |
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ED NUZUM |
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Chief, Biodefense Vaccines & Other Biological Products |
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Development Section |
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Office of Biodefense Research Affairs |
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DMID/NIAID/NIH |
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ERIC ROSE |
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CEO and Chair, Board of Directors |
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SIGA Technologies, Inc. |
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| 3:00 p.m. | Discussion with Workshop Attendees led by workshop co-chairs |
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LESLIE BENET, Workshop Co-Chair |
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University of California, San Francisco |
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JOHN REX, Workshop Co-Chair |
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| 4:30 p.m. | Closing Remarks |
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JEAN HU-PRIMMER |
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Senior Advisor for Regulatory Policy |
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Acting Director, MCMi Regulatory Science |
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Office of the Chief Scientist |
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Office of the Commissioner, Food and Drug |
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Administration |
