The overview of PMDA activities

The overview of PMDA activities Atsushi TAMURA, Ph.D. International Coordination Officer for Medical Devices Pharmaceuticals & Medical Devices Agency

Who are we? PMDA (Pharmaceuticals and Medical Devices Agency) is Japanese regulatory agency, working together with Ministry of Health, Labour and Welfare. Our obligation is to protect the public health by assuring safety, efficacy and quality of pharmaceuticals and medical devices. We conduct scientific reviews of marketing authorization applications for pharmaceuticals and medical devices, monitoring of their post-marketing safety. We are also responsible for providing relief compensation for sufferers from adverse drug reaction and infections by pharmaceuticals or biological products.

PMDA s Mission To provide safer and more effective pharmaceuticals and medical devices as fast as we can 3

Philosophy of PMDA PMDA continues to improve the public health and safety of our nation by reviewing applications for marketing approval of pharmaceuticals and medical devices, conducting safety measures, and providing relief to people who have suffered from adverse drug reactions. We conduct our mission in accordance with the following principles: We pursue the development of medical science while performing our duty with greater transparency based on our mission to protect public health and the lives of our citizens. We will be the bridge between the patients and their wishes for faster access to safer and more effective drugs and medical devices. We make science-based judgments on quality, safety, and efficacy of medical products by training personnel to have the latest technical knowledge and wisdom in their field of expertise. We play an active role within the international community by promoting international harmonization. We conduct services in a way that is trusted by the public based on our experiences from the past.

Our Responsibilities [MHLW] Ultimate Responsibilities in policies & administrative measures [PMDA] Science base review, examination, data analysis, etc. to assist MHLW S measures

PMDA three major functions/tasks PMDA (Phermaceuticals and Medical Devices Agency) was established in April, 2004 to ensure the following 3 tasks to protect public health. Review and Audit for Drugs/ Medical Devices Efficacy and Safety Post- marketing Safety Operations for Drugs / Medical Devices Relief Service for ADR and Other Infectious Disease Clinical Trial Consultation Review of Efficacy and Safety Conformity Audit for Application Materials of GLP,GCP and GMP/QMS Reinforced Safety Information (Database) Scientific Review and Research for Safety Information Information Provision (via the Internet), Pharmaceutical Consultation for Consumers Provision of Medical Expenses, Disability Pensions etc. Relief Service for SMON, HIV-positive and AIDS patients 6

Auditor Auditor Key Organization Chart of PMDA Chief Management Officer Offices of General Affairs/ Financial Management Office of Planning and Coordination Office of International Programs Office of Reg. Sci. Operations Senior Executive Director Chief Relief Officer Office of Relief Funds Office of Review Administration Office of Review Management Chief Executive Director (Center for Product Evaluation) Office of New Drug I - V Office of Medical Devices I - III Review Department Office of OTC/Generic Drugs Office of Biologics I - II Executive Director Office of Conformity Audit (Inspections such as GLP/GCP and GPSP) Executive Director Chief Safety Officer Office of Compliance and Standards Office of Safety I - II (GMP/ QMS Inspection) Post-marketing Department http://www.pmda.go.jp/english/about/organization.html 7

Contents 1. Organizational Updates 2. Safety Measures 3. PMDA International Vision 4. Regulatory Science 5. Science Board 8

1. Organizational Updates Staff Size Strengthen Review System 9

PMDA Staff Size 800 700 600 500 400 300 Administrative part Safety Department Review Department Planned 341 319 291 256 426 521 605 648 678 751 200 100 0 2004 2005 2006 2007 2008 2009 2010 2011 2012 2013 10

Strengthen Review System in PMDA- Special Assistant for Chief Executive (Feb. 2012) Office of Review Innovation (Apr. 2012) Science Board (May 2012) 2 Deputy Directors for Center for Product Evaluation (June 2012) 11

New Organization (as of June 2012) - Enhance partnership with academia - Subcommittee(Drug) Science Board Subcommittee(Medical Device) Subcommittee(biologics) Subcommittee(cell-and tissue-based products) Office of Review Innovation, Director General Associate director General Deputy Associate director General Executive Director (Review Research) Secretariat Director Director, Center for product evaluation Deputy Center Director for medical Devices Review Section Chief Executive Special Assistant Senior Executive Director (Technology Management) Deputy Center Director for Cellular-and Tissue-based products Safety Section Executive Director (General Coordination) Relief Section Administration Section 12

To reduce Drug Submission Lag 1. Promoting Global Clinical Trial Developing Guidelines Holding MRCT Workshop 2. Consultation Pharmaceutical Affairs Consultation on R&D Strategy Prior Assessment Consultation on Drugs 3. Efforts in Regulatory Science Collaboration with Academia Establishment of the Science Board MHLW/PMDA/Academia Collaborative Study

2. Safety Measures New Risk Management System Electronic Medical Record Network Project for Drug Safety 14

Improving Safety Measures Collection of Information Analysis Assessment of Safety measure effects New Risk management system Planning and Implementation of Safety measures Hypothesis Evaluation of hypothesis Crisis management Goal Prevention of serious drug safety-related crisis from Japan Effective encouragement of proper drug use. Ensuring credibility to post-market safety management system.

Electronic Medical Record Network Project for Drug Safety Budget approved FY2011 Governmental to initiate Safety 10Mil. Data project The network construction will be completed by FY2013. Researchers Joint Study Collaboration Data request Collaborative Hospital Database Ethical Committee Network of the Collaborative Hospitals Database Database Quick actions Collaborative study Claim Data Ordering Data Health Records Laboratory Data Database Database Desired Outcomes: Risk & benefit review of medical technologies to provide safer healthcare. Quick and appropriate measures to ensure drug safety.

3. PMDA International Vision 17

PMDA International vision PMDA EPOCH TOWARD 2020 Concrete goals for PMDA to attain by 2020 as one of world s premier medical products regulatory agencies (Published in November 2011) 18

- PMDA EPOCH toward 2020-1. Excellence in Performance 2. Partnership with the Orient 3. Contribution to Harmonization 19

4. Regulatory Science MHLW/PMDA/Academia Collaborative Study Regulatory Science Cycle Promotion of Regulatory Science Program of Collaborative Graduate Schools 20

MHLW /PMDA /Academia Collaborative Study (MHLW FY 2012 Budget) 1.2B yen (about $15M US) Establishment of evaluation methods for safety and efficacy based on Regulatory Science Enhancement of personnel exchange among PMDA, Research Institutes, NIHS MHLW Support Research Intuitions Personnel exchange 366M yen (about $4.6M US) for; Developing guidance for innovative drug/medical device/biologics to streamline review process NIHS Develop Guidelines Cultivate Human Resource

Regulatory Science Cycle Advanced RS Data Acquisition Scientific Reflection Evaluation & Estimation Regulatory Action Balancing Factors 22

Promotion of Regulatory Science Policy recommendations released in 2011 Committee on Pharmaceutical of Science, Science Council of Japan (August 19, 2011) Regulatory Science is seen as Science that adjusts science technology outcome to it s most favorable shape in harmonization between human and society that is an essential concept to prove risk/benefit and to ensure safety of pharmaceuticals and medical devices. Target for Drug discovery Drug discovery by Academia Seeds Discovery Lead compounds Identification Optimization Valley of Death Non-clinical Trial Clinical Trial License out to Industry R&D by Industry For Developments Eying their Exits(Practical Use), for Rapid Processes Leading Collaboration to Practical Use, and for Ensuring the Efficacy and Safety with Academia Promotion of Regulatory Science Research is Essential 23

Program of Collaborative Graduate Schools Agreement with 12 Universities (as of June, 2012) Yamagata University Musashino University University of Tsukuba Kobe University Shujitsu University Osaka University Gifu Pharmaceutical University Gifu University Chiba University Teikyo University Yokohama City University Shizuoka Prefectural University 24

5 Establishment of the Science Board 25

Why Science Board now? With Review Goal (Time) achieved and Staffers increasing, We at PMDA are required to: 1 Provide review and consultation services with understanding of advanced technologies (antibody-based drugs, companion diagnostic drugs, ventricular assist devices, regenerative medicine, cancer vaccines, etc.). 2 Provide consultation/advice on the advanced scientific technology from the early stage of development to deliver the medicinal products utilizing advanced technologies sooner to the medicinal scene. 3 Keep close relationship with the academia to have the PMDA reviewers updated to the accelerating innovation. Science Board 26

Science Board and Office of Review Innovation Office of Review Innovation Director General Science Board Committee members: External experts from Academia Not involved in the Review Process of individual products Secretariat Director Secretariat Director s office Mission Reform PMDA reviews and related services based on science with consideration for actual medical practices Associate Director General Committee 1 Review policy for innovative medical products 2 Development of guidelines 3 Regulatory Science Research 4 Personnel exchanges 5 Election of External review experts 6 Improvements in the scientific aspects of review Subcommittee PMDA Offices Review Safety Deliberation on problems in each field Collaboration with PMDA working team (RS research, guideline development, etc.) Pharmaceuticals Medical Devices Bio-based products Cell- & tissue- Based products RS SGD RS: Office of Regulatory Science SGD: Office of Standards and Guidelines Development 27

Basic Research Seeds of new drug / medical devices originated in Japan For PMDA To Be More Science-Based Quality Tests Non-clinical tests Pharmaceutical consultation on R&D Strategy Office of Review Innovation Establishment of the Science Board The Science Board was established in May 2012 to discuss how PMDA can better cope with products with advanced scientific technology, in each developmental stage such as basic research, development support, product review, and post market safety measures. CT Clinical Trial Consultation Review Review Approve Post Marketing Post Marketing Safety Measure Offices of Review (Drugs & Medical Devices), Office of Safety Practical use Innovative medical products Board members Academia 28

To Improve Public Health Review Safety Relief 29

Thank you for your attention! E-Mail: tamura-atsushi@pmda.go.jp URL: http://www.pmda.go.jp/ Atsushi TAMURA, Ph.D. International Coordination Officer for Medical Devices Pharmaceuticals & Medical Devices Agency JAPAN ( 独 ) 医薬品医療機器総合機構国際業務調整役 ( 医療機器担当 ) 田村敦史 100-0013 東京都千代田区霞ヶ関 3-3-2 TEL 03(3506)9456 FAX 03(3506)9572 30