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OCR for page 83
A
Workshop Agenda
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 chemi -
cal, 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 pub -
lic. As highlighted in the March 2010 report of the National Biodefense
Science Board, “FDA has not been able to fulfill its implicit national secu-
rity mission, in large part because of a lack of resources. . . . It is impera -
tive 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 pub-
lic 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 reg-
ulatory 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
83
OCR for page 84
84 ADVANCING REGULATORY SCIENCE FOR MCM DEVELOPMENT
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:
• rovide a broad overview of current efforts underway at FDA and
P
other agencies within HHS and the Department of Defense (DoD)
to support the research, development, evaluation, and production of
MCMs.
• eview novel scientific methodologies used by academia and indus-
R
try to facilitate development of next generation vaccines, biologics,
drugs, and devices.
• dentify major gaps in currently available tools to predict and evalu-
I
ate product safety, efficacy, and quality.
• dentify opportunities for collaboration and coordination with FDA
I
and among relevant federal as well as industry programs to support
the MCM initiative’s regulatory science program and to develop bet-
ter defined pathways for product approval.
• dentify regulatory science tools and methodologies to address emerg-
I
ing technologies, targets, and novel products as well as innovative
approaches for predicting safety and efficacy; for example, biomark-
ers and in silico modeling.
8:00 a.m. Welcome and Introductions
LesLie Benet, Workshop Co-Chair
Professor, School of Pharmacy
University of California, San Francisco
John Rex, Workshop Co-Chair
Infection Clinical Vice President
AstraZeneca
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85
APPENDIX A
8:10 a.m. Keynote Address: Importance and Promise of Regulatory
Science and Charge to Workshop Participants
Jesse Goodman
Chief Scientist, Deputy Commissioner for Science and
Public Health
Food and Drug Administration
8:25 a.m. Enterprise Activities and Needs for MCM Regulatory
Science
GeoRGe KoRch
Acting Principal Deputy Assistant Secretary for
Preparedness and Response
Office of the Assistant Secretary for Preparedness and
Response
Department of Health and Human Services
8:35 a.m. Overview of the MCM Initiative: Challenges and
Opportunities
Luciana BoRio
Acting Director, Office of Counterterrorism & Emerging
Threats
Senior Advisor for Medicine and Public Health, Office
of the Chief Scientist
Office of the Commissioner, Food and Drug
Administration
SESSION I: ENTERPRISE AND STAKEHOLDER
PERSPECTIVES ON NEEDS TO ADVANCE MEDICAL
COUNTERMEASURE REGULATORY SCIENCE
Session Objectives:
• rovide a broad overview of current efforts under way at FDA and
P
within HHS and DoD to support the research, development, evalua-
tion, and production of MCMs.
• rovide a broad overview of MCM development challenges and
P
where regulatory science can advance MCM development.
• dentify and discuss the highest-priority MCM regulatory science
I
needs to advance MCM development.
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86 ADVANCING REGULATORY SCIENCE FOR MCM DEVELOPMENT
Presentation/Panel 1: FDA
8:45 a.m. Session Introduction and Panel Objectives
GeoRGe KoRch
Acting Principal Deputy Assistant Secretary for
Preparedness and Response
Office of the Assistant Secretary for Preparedness and
Response
Department of Health and Human Services
8:50 a.m. Presentations: Overview of Enterprise MCM Regulatory
Science Agendas: What does the agency need?
• dentify the goals and objectives of MCM regulatory
I
science agenda from the FDA Centers.
• dentify challenges to and information needs for MCM
I
regulatory science programs.
caRoLyn WiLson
Associate Director for Research
Center for Biologics Evaluation and Research
Food and Drug Administration
susan mccune
Deputy Director, Office of Translational Sciences
Center for Drug Evaluation and Research
Food and Drug Administration
muRRay maLin
Center for Devices and Radiological Health
Food and Drug Administration
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
maRy PendeRGast
President
Pendergast Consulting
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87
APPENDIX A
10:40 a.m. Presentations and Panel Discussion: What do the partners
need?
• dentify MCM product development challenges that can
I
be addressed with MCM regulatory science.
• dentify recurrent and overarching themes for stalled
I
MCM product development and utilization that can be
addressed through MCM regulatory science.
• iscuss highest-priority regulatory science needs to
D
advance MCM development.
GeRaLd PaRKeR
Deputy Assistant to the Secretary of Defense
for Chemical and Biological Defense Programs
(DATSD(CBD))
Department of Defense
michaeL KuRiLLa
Director, Office of BioDefense Research Affairs
Associate Director for BioDefense Product Development
National Institute of Allergy and Infectious Disease
National Institutes of Heath
RichaRd hatchett
Chief Medical Officer and Deputy Director
Biomedical Advanced Research and Development
Authority
Office of the Assistant Secretary for Preparedness and
Response
Department of Health and Human Services
may chu
Director, Laboratory Science Policy & Practice Program
Office
Centers for Disease Control
RicK Lyons
Director, Infectious Disease Research Center
Chief Scientific Officer, Infectious Disease SuperCluster
Colorado State University
eRic Rose
CEO and Chair, Board of Directors
SIGA Technologies, Inc.
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88 ADVANCING REGULATORY SCIENCE FOR MCM DEVELOPMENT
11:45 a.m. Discussion/Question and Answer Session
12:15 p.m. LUNCH
SESSION II: CUTTING-EDGE EFFORTS TO ADVANCE
MEDICAL COUNTERMEASURE REGULATORY SCIENCE
Session Objectives:
• eview novel scientific methodologies used by academia and indus-
R
try to discover and develop next generation vaccines, biologics, drugs,
and devices.
• dentify major gaps in currently available tools to predict and evalu-
I
ate product safety, efficacy, and quality.
• dentify approaches to developing regulatory science tools and
I
methodologies to address emerging technologies, targets, and novel
products as well as innovative approaches for predicting safety and
efficacy.
• iscuss a path forward for the MCM regulatory science agenda.
D
• iscuss metrics to be used for gauging success of a scientific agenda
D
for evaluating MCMs.
• xamine how partnerships and other collaborative approaches can
E
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
University of California, San Francisco
John Rex, Workshop Co-Chair
AstraZeneca
Panelists will
• rovide high-level description of MCM regulatory
P
science tools and state of the science.
• iscuss regulatory science opportunities and
D
challenges. What is needed to advance regulatory
science to undergird regulatory decisions?
• iscuss highest-priority regulatory science needs to
D
advance MCM development and utilization.
• dentify opportunities for partnerships and
I
collaboration.
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89
APPENDIX A
Panel: ex ViVo aPProaches to Modeling efficacy
1:05 p.m. Panel Objectives and Introduction
LauRen BLacK, panel chair
Senior Scientific Advisor, Navigators
Charles River Laboratories
1:10 p.m. Panel Discussion: Animal Models, In Silico Models,
Biomarkers
In Silico Models
Ramon FeLciano
Founder & SVP of Research
Ingenuity Systems
Biomarkers
n. LeiGh andeRson
Founder & CEO
Plasma Proteome Institute
Animal Models
eLizaBeth LeFFeL
Director of Nonclinical Sciences
PharmAthene
1:50 p.m. Discussion with Invited Discussants and Workshop
Attendees
dRusiLLa BuRns
Chief, Laboratory of Respiratory and Special Pathogens
Center for Biological Evaluation and Research
Judy heWitt
Chief, Biodefense Research Resources Section
Office of Biodefense Research Affairs
DMID/NIAID/NIH
ViKRam PateL
Office of Testing and Research
Office of Pharmaceutical Sciences, CDER
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90 ADVANCING REGULATORY SCIENCE FOR MCM DEVELOPMENT
Panel: safety and real-tiMe Monitoring
2:25 p.m. Panel Objectives and Introduction
caRL PecK, panel chair
Professor, Pharmacology and Medicine
University of California, San Francisco
2:30 p.m. Panel Discussion: Post Deployment Surveillance and Side
Effects; Toxicology Markers
Toxicology Markers
RoBeRt house
President
DynPort Vaccine Company LLC
Post Deployment Surveillance and Side Effects
Kenneth mandL
Faculty, Children’s Hospital Informatics Program
Harvard-MIT Division of Health Sciences and
Technology
2:50 p.m. Discussion with Invited Discussants and Workshop
Attendees
maRietta anthony
Predictive Safety Testing Consortium
Critical Path Institute
RichaRd FoRshee
Associate Director for Research
Office of Biostatistics and Epidemiology
Center for Biologics Evaluation and Research
henRy FRancis
Center for Drug Evaluation and Research
Food and Drug Administration
RoBeRt c. neLson
Product Safety Assurance Services, Inc.
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91
APPENDIX A
donna mendRicK
National Center for Toxicological Research
Food and Drug Administration
3:20 p.m. BREAK
Panel: diagnostics and statistical techniques
3:35 p.m. Panel Objectives and Introduction
BRuce BuRLinGton, panel chair
Independent Consultant
3:40 p.m. Panel Discussion: Diagnostics
Diagnostics
Lt. coL. danieL WattendoRF
U.S. Air Force
Program Manager, Defense Sciences Office
DARPA
3:50 p.m. Discussion with Invited Discussants and Workshop
Attendees
chaRLes daitch
Chief Executive Officer
Akonni Biosystems
daVid ecKeR
Founder, DVP and Carlsbad General Manager
Ibis Biosciences
saLLy hoJVat
Center for Diagnostics and Radiological Health, FDA
4:25 p.m. Panel Discussion: Statistical Techniques
Statistical Techniques
stePhen RuBeRG
Distinguished Research Fellow & Scientific Leader,
Advanced Analytics
Eli Lilly & Co.
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92 ADVANCING REGULATORY SCIENCE FOR MCM DEVELOPMENT
4:35 p.m. Discussion with Invited Discussants and Workshop
Attendees
esteLLe RusseK-cohen
Acting Division Director, Division of Biostatistics
Office of Biostatistics and Epidemiology, CBER/FDA
JeFFRey WetheRinGton
Research Director, Statistics
GlaxoSmithKline
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 ADVANCE
MEDICAL COUNTERMEASURE REGULATORY SCIENCE
Session Objectives:
• eview novel scientific methodologies used by academia and indus-
R
try to discover and develop next generation vaccines, biologics, drugs,
and devices.
• dentify major gaps in currently available tools to predict and evalu-
I
ate product safety, efficacy, and quality.
• dentify approaches to developing regulatory science tools and
I
methodologies to address emerging technologies, targets, and novel
products as well as innovative approaches for predicting safety and
efficacy.
• iscuss a path forward for the MCM regulatory science agenda.
D
• iscuss metrics to be used for gauging success of a scientific agenda
D
for evaluating MCMs.
• xamine how partnerships and other collaborative approaches can
E
facilitate the advancement and ongoing support of regulatory science
for MCM development, evaluation, and utilization.
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93
APPENDIX A
8:00 a.m. Welcome and Day 2 Overview
LesLie Benet, Workshop Co-Chair
University of California, San Francisco
John Rex, Workshop Co-Chair
AstraZeneca
Panel: antiMicrobials, Vaccines, and Vaccine adjuVants
8:15 a.m. Panel Objectives and Introduction
Linda a. miLLeR, panel chair
Director, Clinical Microbiology
GlaxoSmithKline
8:20 a.m. Panel Discussion: Antimicrobials, Vaccines, and Vaccine
Adjuvants
Antimicrobials
KeVin Judice
CEO & Chief Scientific Officer
Achaogen
Vaccines
aLan maGiLL
Director, Division of Experimental Therapeutics
Walter Reed Army Institute of Research
Adjuvants
deBBie dRane
Senior VP R&D
CSL Biotherapies
9:00 a.m. Discussion with Invited Discussants and Workshop
Attendees
ed cox
Center for Drug Evaluation and Research
Food and Drug Administration
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94 ADVANCING REGULATORY SCIENCE FOR MCM DEVELOPMENT
daVid FRucht
Capt., U.S. Public Health Service
Chief, Laboratory of Cell Biology
Division of Monoclonal Antibodies
CDER/FDA
BasiL GoLdinG
Center for Biologics Evaluation and Research
hana GoLdinG
Chief, Laboratory of Retrovirus Research, Division of
Viral Products
Office of Vaccines Research and Review, CBER
ed nuzum
Chief, Biodefense Vaccines & Other Biological Products
Development Section
Office of Biodefense Research Affairs
DMID/NIAID/NIH
aLan shaW
Chief Scientific Officer
Vaxinnate
Panel: the future
9:45 a.m. Panel Objectives and Introduction
GiGi KWiK GRonVaLL, panel chair
Senior Associate, Center for Biosecurity
UPMC
9:50 a.m. Panel Discussion: Synthetic Biology, Computational
Biology, and Platform Technologies
Synthetic Biology
John GLass
Senior Scientist
J. Craig Venter Institute
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95
APPENDIX A
Computational Biology
michaeL Katze
Professor of Microbiology
University of Washington
Platform Technology
Pat iVeRsen
Senior Vice President of Research & Innovation
AVI BioPharma
10:30 a.m. Discussion with Invited Discussants and Workshop
Attendees
WiLLiam FoGLeR
Senior Director, Portfolio Analysis and Planning
Intrexon Corporation
donna mendRicK
Director, Division of Systems Biology
National Center for Toxicological Research
Food and Drug Administration
haRVey RuBin
Executive Director, Institute for Strategic Threat
Analysis and Response
University of Pennsylvania
11:00 a.m. BREAK
SESSION III: MEDICAL COUNTERMEASURE REGULATORY
SCIENCE NEEDS FOR AT-RISK POPULATIONS
Session Objectives:
• dentify needs that are specific or unique to at-risk populations (e.g.,
I
pediatric populations) that should be considered in developing a regu-
latory science agenda for MCM development.
• rovide an overview of where regulatory science can advance MCM
P
development for these populations.
• iscuss a MCM regulatory science agenda for at-risk populations.
D
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96 ADVANCING REGULATORY SCIENCE FOR MCM DEVELOPMENT
Panelists will
• dentify recurrent and overarching challenges for MCM
I
product development, implementation, and use specific
to at-risk populations that can be addressed with MCM
regulatory science.
• iscuss highest-priority regulatory science needs to
D
advance MCM development and utilization for at-risk
populations. What tools could be used to support
regulatory review and determinations of use in these
populations in an emergency?
Discuss issues for at-risk populations such as: lack of
■
dosing information, and needs for safety and efficacy
models.
Specific scientific gaps in treating or vaccinating
■
pregnant women.
11:15 a.m. Introduction and Session Objectives
John BRadLey, panel chair
Director, Infectious Diseases
Rady Children’s Hospital San Diego
11:20 a.m. Panel Discussion
Pediatric
daVid sieGeL
NICHD
Pregnancy/OB-Gyn
chRistian R. macedonia
Joint Chiefs of Staff, the Pentagon
11:45 a.m. Discussion with Attendees
PRaVin JadhaV
Center for Drug Evaluation and Research
Food and Drug Administration
nancy messonnieR
Centers for Disease Control
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97
APPENDIX A
Lisa mathis
Center for Drug Evaluation and Research
Food and Drug Administration
RoBeRt neLson
Senior Pediatric Ethicist/Lead Medical Officer
Office of Pediatric Therapeutics
Office of the Commissioner, Food and Drug
Administration
12:30 p.m. LUNCH
SESSION IV: FUTURE DIRECTIONS—DISCUSSION WITH
WORKSHOP 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
University of California, San Francisco
John Rex, Workshop Co-Chair
AstraZeneca
1:30 p.m. Discussion with Panelists and Workshop Attendees led by
workshop co-chairs
• Synthesize workshop discussions.
• ropose key opportunities to develop a research agenda
P
and roadmap for the MCM regulatory science initiative.
PhyLLis aRthuR
Director, Health & Regulatory Affairs
Biotechnology Industry Organization
GaiL casseLL, Drug Forum Co-Chair
Visiting Professor, Department of Social Medicine,
Harvard Medical School
Vice President, TB Drug Discovery, Infectious Disease
Research Institute, Seattle
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98 ADVANCING REGULATORY SCIENCE FOR MCM DEVELOPMENT
RichaRd hatchett
Chief Medical Officer and Deputy Director
Biomedical Advanced Research and Development
Authority
Office of the Assistant Secretary for Preparedness and
Response
Department of Health and Human Services
caRL PecK
Professor, Pharmacology and Medicine
University of California, San Francisco
ed nuzum
Chief, Biodefense Vaccines & Other Biological Products
Development Section
Office of Biodefense Research Affairs
DMID/NIAID/NIH
eRic Rose
CEO and Chair, Board of Directors
SIGA Technologies, Inc.
3:00 p.m. Discussion with Workshop Attendees led by workshop
co-chairs
LesLie Benet, Workshop Co-Chair
University of California, San Francisco
John Rex, Workshop Co-Chair
AstraZeneca
4:30 p.m. Closing Remarks
Jean hu-PRimmeR
Senior Advisor for Regulatory Policy
Acting Director, MCMi Regulatory Science
Office of the Chief Scientist
Office of the Commissioner, Food and Drug
Administration
