Appendix A

Workshop Agenda

International Regulatory Harmonization Amid Globalization of Biomedical Research and Medical Product Development: An Institute of Medicine Workshop

February 13–14, 2013

National Academy of Sciences Building Lecture Room 2101 Constitution Avenue, NW Washington, DC 20418

Background and Meeting Objectives:
The past several decades have seen a rapid globalization of commerce, including within the medical product and technology sectors. Investigational studies are increasingly being conducted outside the countries that have a history as hubs for biomedical research, often in countries with limited regulatory capacity. Moreover, biopharmaceutical companies seeking global markets face requirements for regulatory submissions for the same product in numerous international jurisdictions that could introduce scientific requirements that are discordant with standards in their home markets. Discordant data requirements could result in additional clinical trials and animal studies, exposing more patients to experimental drugs and increasing the use of laboratory animals. There is a need for globally harmonized, science-based standards for the development and evaluation of safety, quality, and efficacy of medical products—both to enhance the efficiency and clarity of the drug development and evaluation process, and ultimately to promote and enhance product quality and the public’s health. There is also need for harmonization of standards for ongoing safety and quality surveillance of marketed biomedical products.

This public workshop will address needs for international harmonization of regulatory standards to support the development, evaluation, and surveillance of biomedical products. Specifically, the discussions at the workshop will help identify principles, potential approaches, and



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Appendix A Workshop Agenda International Regulatory Harmonization Amid Globalization of Biomedical Research and Medical Product Development: An Institute of Medicine Workshop February 13–14, 2013 National Academy of Sciences Building Lecture Room 2101 Constitution Avenue, NW Washington, DC 20418 Background and Meeting Objectives: The past several decades have seen a rapid globalization of commerce, including within the medical product and technology sectors. Investiga- tional studies are increasingly being conducted outside the countries that have a history as hubs for biomedical research, often in countries with limited regulatory capacity. Moreover, biopharmaceutical companies seek- ing global markets face requirements for regulatory submissions for the same product in numerous international jurisdictions that could introduce scientific requirements that are discordant with standards in their home markets. Discordant data requirements could result in additional clinical trials and animal studies, exposing more patients to experimental drugs and increasing the use of laboratory animals. There is a need for globally harmonized, science-based standards for the development and evaluation of safety, quality, and efficacy of medical products—both to enhance the efficiency and clarity of the drug development and evaluation process, and ultimately to promote and enhance product quality and the public’s health. There is also need for harmonization of standards for ongoing safety and quality surveillance of marketed biomedical products. This public workshop will address needs for international harmoniza- tion of regulatory standards to support the development, evaluation, and surveillance of biomedical products. Specifically, the discussions at the workshop will help identify principles, potential approaches, and 79

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80 INTERNATIONAL REGULATORY HARMONIZATION strategies to advance the development or evolution of more harmonized regulatory standards. The workshop objectives are to: • Provide an overview of the current global regulatory landscape. Identify n  Current organized efforts to promote and evolve harmonized standards, and examples of areas where standards are viewed as adequately harmonized. n  Areas of need for development or evolution of harmonized standards. • Identify the characteristics of a well-harmonized regulation. • Discuss principles to guide the establishment or evolution of harmonized regulations. • Discuss options and approaches that could facilitate or underlie systemic organizational efforts to develop and/or evolve harmonized standards. n  Discuss potential structures, methodologies, goals, and outcomes. Day One: February 13, 2013 8:30 a.m. Opening Remarks Steven Galson, Workshop Co-Chair Vice President for Global Regulatory Affairs Amgen Inc. Tom Bollyky, Workshop Co-Chair Senior Fellow for Global Health, Economics, and Development Council on Foreign Relations 8:50 a.m. Plenary Keynotes: Needs from the Perspective of Stakeholders Keynote Address: Industry Peter Honig VP and Head, Global Regulatory Affairs AstraZeneca

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APPENDIX A 81 Keynote Address: Regulator Hubert G. M. Leufkens Chair, Dutch Medicines Evaluation Board; Member, Committee on Human Medicinal Products, European Medicines Agency Session i: principles and definitional considerations Session Objectives: • Examine key definitions and terminology: “harmonization” vs. “convergence” vs. “cooperation” vs. “consensus standards.” • Discuss potential goals for initiatives, including differences arising from terminology. • Consider potential variations in approach depending on which terminology is adopted and how the desired outcome is defined. 9:35 a.m. Background and Session Objectives Andreas Seiter, Session Chair Senior Health Specialist Pharmaceuticals, Health, Nutrition, and Population World Bank 9:40 a.m. Series of Presentations History and Importance of Terminology, the Terminology Landscape, and Options for Regulators Mike Ward Manager, International Programs Health Canada Standards-Setting in the Context of Regulatory Harmonization Carolyn Compton President and CEO Critical Path Institute 10:20 a.m. Discussion with Speakers and Audience 10:40 a.m. BREAK

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82 INTERNATIONAL REGULATORY HARMONIZATION Session II: overview of current global regulatory landscape Session Objectives: • Provide an overview of the current global regulatory landscape. • Identify current organized efforts to promote and evolve harmo- nized standards. • Highlight examples of areas where standards are viewed as ade- quately harmonized and/or harmonization processes are viewed as well working. 11:00 a.m. Background and Session Objectives Hans Hogerzeil, Session Chair Professor of Global Health University of Groningen, the Netherlands 11:05 a.m. Series of Presentations  Conference on Harmonisation of Technical International Requirements for Registration of Pharmaceuticals for Human Use (ICH) Toshiyoshi Tominaga Professor Osaka City University Hospital Latin America James Fitzgerald Senior Advisor, Essential Medicines and Biologicals Pan American Health Organization APEC Regulatory Harmonization Steering Committee (RHSC) Mike Ward Manager, International Programs Health Canada

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APPENDIX A 83 East African Community (EAC) Medicines Registration Harmonization Project Margareth Ndomondo-Sigonda Pharmaceutical Coordinator New Partnership for Africa’s Development African Union 12:05 p.m. Discussion with Speakers and Audience Discussion Topics/Questions • Description and characterization of existing international standards-setting bodies • Description and examination of regional harmonization efforts • Identification and discussion of particular standards that have been developed 12:30 p.m. LUNCH Session iii: areas of need for harmonized standards and barriers to progress in addressing the gaps Session Objectives: • Discuss gaps in the current structures, approaches, and interna- tional standards leading to unnecessary discordance among regu- latory requirements. n  Identify top-priority areas where harmonized standards need to be developed or evolved. n  Consider regulatory requirements and harmonization needs across the full spectrum of medical product development, eval- uation, and monitoring/surveillance. • Discuss how gaps are identified and priorities are set within har- monization efforts. • Having considered the gaps and areas of need, identify the key barriers that stand in the way of addressing the identified needs. • Discuss approaches that have been tried and have failed to address the needs.

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84 INTERNATIONAL REGULATORY HARMONIZATION 1:15 p.m. Background and Session Objectives Steven Galson, Session Chair Vice President for Global Regulatory Affairs Amgen Inc. 1:20 p.m. Stakeholder Presentations: Gaps in Current Structures and High-Priority Areas of Need Overview: Gaps from the Regulator’s Perspective Douglas Throckmorton Deputy Director, Center for Drug Evaluation and Research U.S. Food and Drug Administration Overview: Gaps from the Perspective of NGOs/ Foundations/Product Development Partnerships Vincent Ahonkhai Deputy Director, Regulatory Affairs Bill & Melinda Gates Foundation 2:00 p.m. Overview of Charge to Breakout Groups Steven Galson, Session Chair Vice President for Global Regulatory Affairs Amgen Inc. 2:15 p.m. Breakout Groups Convene Concurrently General Discussion Topics/Objectives: • Identify the key barriers that stand in the way of addressing the identified needs. What are the issues that are the most pressing? • Discuss approaches that have been tried and have failed to address the needs. • Deliberate on potential options to address those high- priority needs for consideration at the workshop.

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APPENDIX A 85 1.  Qualification of Innovative Development Methods/Drug Development Tools [STAY IN LECTURE ROOM]  Richard Meibach, Novartis Pharmaceuticals (moderator) Martha Brumfield, Critical Path Institute (rapporteur) 2. Clinical Development [BOARD ROOM] Judith Kramer, Duke University (moderator)  Leslie Ball, U.S. Food and Drug Administration (rapporteur) 3.  valuation & Evidentiary Requirements [ROOM 125] E Tim Franson, FaegreBD Consulting (moderator)  Lawrence Liberti, Centre for Innovation in Regulatory Science (rapporteur) 4.  ostmarket Safety Surveillance [MEMBERS ROOM] P Amrit Ray, Johnson & Johnson (moderator)  Andy Stergachis, University of Washington (rapporteur) 5. Manufacturing Standards and Process [ROOM 114] Moheb Nasr, GlaxoSmithKline (moderator) Diane Zezza, Novartis Pharmaceuticals (rapporteur) 4:15 p.m. Breakout Groups Conclude 4:30 p.m. Reports from Breakout Groups 5:20 p.m. Day One Reflections 5:30 p.m. Adjourn Day One Day Two: February 14, 2013 8:30 a.m. Welcome and Reflections from Day One Steven Galson, Workshop Co-Chair Vice President for Global Regulatory Affairs Amgen Inc. Tom Bollyky, Workshop Co-Chair Senior Fellow for Global Health, Economics, and Development Council on Foreign Relations

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86 INTERNATIONAL REGULATORY HARMONIZATION Session iv: characteristics of harmonized regulations and regulatory structures Session Objectives: • Consider examples of standards-setting and regulatory harmoniza- tion from other sectors and their application to biomedical research and medical product regulation. • Identify the characteristics of a “well-harmonized regulation” or well-working process. • Discuss principles to guide the establishment or evolution of har- monized regulations or other desired process and outcomes. 8:35 a.m. Background and Session Objectives James Fitzgerald, Session Chair Senior Advisor, Essential Medicines and Biologicals Pan American Health Organization 8:40 a.m. Series of Presentations Radiation Safety Standards Cindy Flannery Senior Health Physicist U.S. Nuclear Regulatory Commission Reflections on the Experiences of the World Health Organization Lembit Rägo Coordinator for Quality and Safety of Medicines World Health Organization 9:10 a.m. Discussion with Speakers and Audience 9:40 a.m. BREAK Session v: finding solutions to the challenges of regulatory harmonization: options and systemic approaches Session Objectives: • Discuss options and approaches that could facilitate or underlie systemic organizational efforts to develop and/or evolve harmo- nized standards.

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APPENDIX A 87 • Discuss potential structures, methodologies, goals, and outcomes. • Examine these issues with respect both to development and imple- mentation of desired standards and/or processes. 10:00 a.m. Background and Session Objectives Michael J. Brennan, Session Chair Senior Advisor, Global Affairs Aeras 10:05 a.m. Reaction Panel: Potential Solutions from Stakeholder Perspectives Vincent Ahonkhai Deputy Director, Regulatory Affairs Bill & Melinda Gates Foundation Raymond Chua Group Director, Health Products Regulation Group Singapore Health Sciences Authority Mary Lou Valdez Associate Commissioner for International Programs U.S. Food and Drug Administration David Wood Coordinator of Quality, Safety and Standards: Immunization, Vaccines and Biologicals World Health Organization 10:55 a.m. Discussion with Speakers and Audience Discussion Topics/Questions: • What process or systemic approach holds most promise for supporting development of harmonized standards or processes? • What are the needed structures to support implementa- tion of harmonized standards or processes within various systems (e.g., training, capacity building, networks, other needs)? • How can novel harmonization/convergence strategies, policies, and processes be implemented to facilitate the efficient global introduction of quality products?

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88 INTERNATIONAL REGULATORY HARMONIZATION • How can we promote and expand on current harmo- nization/convergence strategies to alleviate regulatory roadblocks? 11:30 a.m. BREAK FOR LUNCH Session vi: concluding stakeholder discussion: tactics and strategies for a way forward Session Objectives: • Discuss key themes from the workshop. • Based on workshop presentations and discussions, identify tactics and strategies (both short and long term) for addressing the needs for developing and evolving more harmonized regulations and regulatory structures. 12:30 p.m. Closing Discussion with Panelists and Audience: Led by Workshop Co-Chairs Steven Galson, Workshop Co-Chair Vice President for Global Regulatory Affairs Amgen Inc. Tom Bollyky, Workshop Co-Chair Senior Fellow for Global Health, Economics, and Development Council on Foreign Relations 12:35 p.m. Panel 1: Presentation of Key Themes/Suggested Paths Hans Hogerzeil, Chair of Session I Director for Essential Medicines (former) World Health Organization Andreas Seiter, Chair of Session II Senior Health Specialist Pharmaceuticals, Health, Nutrition, and Population World Bank Steven Galson, Chair of Session III Vice President for Global Regulatory Affairs Amgen Inc.

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APPENDIX A 89 James Fitzgerald, Chair of Session IV Senior Advisor, Essential Medicines and Biologicals Pan American Health Organization Michael J. Brennan, Chair of Session V Senior Advisor, Global Affairs Aeras 1:00 p.m. Discussion with Speakers and Audience 1:30 p.m. Panel 2: Reflecting on Tactics and Strategies for a Way Forward Deborah Autor Deputy Commissioner for Global Regulatory Operations and Policy U.S. Food and Drug Administration Hans-Georg Eichler Senior Medical Officer European Medicines Agency Alan Morrison Vice President for International Regulatory Affairs and Safety Amgen Inc. 2:00 p.m. Discussion with Speakers and Audience 2:45 p.m. ADJOURN

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