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 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



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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

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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