regulatory science research in pmda...epoch-making proposal leading the world advancing regulatory...
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12th Kitasato-Harvard Symposium, Tokyo May 14th 2013
Pharmaceuticals & Medical Devices Agency
Regulatory Science Research in PMDA
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Yoshiaki Uyama, Ph.D. Pharmaceuticals & Medical Devices Agency (PMDA)
Visiting Professor, Graduate School of Advanced Clinical Science, Chiba University Visiting Professor, Graduate School of Medicine, Nagoya University
GAP between
expectation and Reality
Medical Needs Traditional Science
Regulatory Science
SAE after approval, Lower success rate, Drug/Device lag, Insufficient risk communication, Uncertainty for deicion
Predictable model for efficacy/safety
New approach on risk communication and management
New study design and analytical tool
Objective evaluation tool for benefit/risk assessment
Concerns and Needs for medical services
Traditional Science
Advancing Regulatory Science
Current Issues
Ensure Social Balance
12th Kitasato-Harvard Symposium, Tokyo May 14th 2013
Pharmaceuticals & Medical Devices Agency
Regulatory Science Bridge
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Tominaga T et al, Clin Pharmacol Ther, 90: 29-31, 2011
Regulatory Science Bridge to a Valley of Death
12th Kitasato-Harvard Symposium, Tokyo May 14th 2013
Pharmaceuticals & Medical Devices Agency
Articles published by PMDA members
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2005 2006 2007 2008 2009 2010 2011 20120
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English
2005 2006 2007 2008 2009 2010 2011 20120
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Japanese
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12th Kitasato-Harvard Symposium, Tokyo May 14th 2013
Pharmaceuticals & Medical Devices Agency
PMDA’s Articles published in the journal (2013)
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Title Journal
Regulatory challenges in the review of data from Global Clinical Trials: PMDA perspective
Clin Pharmacol Ther. 2013, in press
Characteristics of pharmacogenomics/biomarker-guided clinical trials for regulatory approval of anti-cancer drugs in Japan
J Human Genet 2013, advance online publication, 9 May 2013; doi:10.1038/jhg.2013.36
Pharmacogenetics in the evaluation of new drugs: a multiregional regulatory perspective
Nat Rev Drug Discov. 12(2): 103-115, 2013.
Regulatory perspective on remaining challenges for utilization of pharmacogenomics-guided drug developments.
Pharmacogenomics. 14(2):195-203, 2013.
Improving clinical trial sampling for future research - an international approach: outcomes and next steps from the DIA future use sampling workshop 2011
Pharmacogenomics. 14(1):103-12, 2013.
The Roles of Regulatory Science Research in Drug Development at the Pharmaceuticals and Medical Devices Agency of Japan
Therapeutic Innovation & Regulatory Science, 47(1): 19-22, 2013.
Balancing Societal Needs and Regulatory Certainty: The Case Study of Peramivir in Japan
Clin Pharmacol Ther. 93: 342-34, 2013
12th Kitasato-Harvard Symposium, Tokyo May 14th 2013
Pharmaceuticals & Medical Devices Agency 6
EMA FDA
PMDA
12th Kitasato-Harvard Symposium, Tokyo May 14th 2013
Pharmaceuticals & Medical Devices Agency
PMDA’s initiatives to advance Regulatory Science
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12th Kitasato-Harvard Symposium, Tokyo May 14th 2013
Pharmaceuticals & Medical Devices Agency
PMDA
Pharmaceutical Affair Consultation
PMDA Science Board
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Basic Research
Seeds of medical
products discovered
in Japan
Non-Clinical Clinical Quality
Practical Use
Offices of Review; Drugs, Biologics, Medical Devices Offices of Safety
Scientific Consultation Review
NDA
Office of Review Innovation
Approval Post Market
Safety Measure
Science Board
Academia
Board Member
Innovative Medical
Products
e.g.; HAL
iPS iPS-derived products
Discovery in Basic research e.g;
12th Kitasato-Harvard Symposium, Tokyo May 14th 2013
Pharmaceuticals & Medical Devices Agency
Collaborative Graduate School Program
PMDA Staff Visiting Professor (Lecture in regulatory science) Graduate student (Ph.D. program); Research in University
University student
Graduate student (Ph.D. program); Research in PMDA
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PMDA Graduate School
Collaboration
Joint Graduate School agreement
12th Kitasato-Harvard Symposium, Tokyo May 14th 2013
Pharmaceuticals & Medical Devices Agency
Yamagata University
University of Tsukuba
Yokohama City University
Gifu Pharmaceutical University
Kobe University
Chiba University
Musashino University
Agreement with 17 University(As of end of March 2013)
Gifu University
Shujitu University
Teikyo University
University of Shizuoka
Collaborative Graduate School Program
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Osaka University
http://www.pmda.go.jp/regulatory/graduate_school.html
Kyoto Pharmaceutical University
Okayama University
Nagoya University
Nagoya City University
Hokkaido University
12th Kitasato-Harvard Symposium, Tokyo May 14th 2013
Pharmaceuticals & Medical Devices Agency
Regulatory Science Research in PMDA
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12th Kitasato-Harvard Symposium, Tokyo May 14th 2013
Pharmaceuticals & Medical Devices Agency
PMDA designated research (FY2013)
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1. Cross-product evaluation of placebo effects in neuropsychiatric disorders.
2. A study on the use of PK-PD M&S to determine pediatric dosage from relationship between PK-PD profiles of adults and children.
3. Current situation and challenges in the evaluation of drugs used in the elderly.
4. Evaluation of the effects of ethnic factors on the efficacy and safety of the drug based on global clinical trial data.
5. Cross-product evaluation of differences on approved doses between Japan and US/EU based on clinical trial data including PK/PD data
6. Effects on pharmacovigillance of post marketing surveillance targeting all cases
7. A study of risk-based approach on document-based GCP conformity inspection
http://www.pmda.go.jp/regulatory/research.html
12th Kitasato-Harvard Symposium, Tokyo May 14th 2013
Pharmaceuticals & Medical Devices Agency
Academia (University, Institute, Hospital)
○Proactive establishment of the guideline and standards
○Promoting development using innovative techniques
Outcome of research
Learning a state-of- the-art technology
↓ Improving a quality of review and other services in PMDA
Pharmaceuticals and Medical Devices Agency Reviewer
Researcher
Regulatory Science Research & Human Resource Exchange Program
(for developing innovative drug, device, cell & tissue products for practical use)
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Training in regulatory science
↓ Effective research & development for regulatory approval
Human Resource Exchange & Development
12th Kitasato-Harvard Symposium, Tokyo May 14th 2013
Pharmaceuticals & Medical Devices Agency
Research funds for developing innovative drug, device, cell & tissue products for practical use
Faculty of Pharmaceutical Sciences
Graduate School of Pharmaceutical Science
Hospital Nagoya City University, Graduate
School of Pharmaceutical Sciences
Graduate School of Pharmaceutical Sciences
Research Institute
Graduate School of Medicine
University of Tsukuba, Faculty of Medicine
Graduate School of Engineering
National Cerebral and Cardiovascular Center
National Cancer Center East Hospital
Kyusyu University, Graduate School of Medicine
Graduate School of Biomedical Engineering
Waseda University, Center for Advanced Biomedical Sciences
Graduate School of Medicine
Center for iPS Cell Research and Application (CiRA)
Foundation for Biomedical Research and Innovation
Medical Center (Hospital)
Chiba University, Graduate School of Medicine
Graduate school of Medicine
Drug
Device
Cell & Tissue
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Hokkaido University
Tohoku University
National Cancer Center Hospital
The University of Tokyo
National Center for Child Health and Development
Osaka University
Kyoto University
12th Kitasato-Harvard Symposium, Tokyo May 14th 2013
Pharmaceuticals & Medical Devices Agency
Future workflow of review & consultation in PMDA
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12th Kitasato-Harvard Symposium, Tokyo May 14th 2013
Pharmaceuticals & Medical Devices Agency
Future workflow of Review & Consultation in PMDA Dealing with state-of-the-art technology
iPS cell-based product Utilization of innovative methods
Pharmacometircs (Modeling & Simulation)
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12th Kitasato-Harvard Symposium, Tokyo May 14th 2013
Pharmaceuticals & Medical Devices Agency
Advanced workflow of review/consultation using innovative assessment techniques
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Giving additional
scientific value to submitted
data
e-Submission of study data
Database data Accumulation
Innovative Assessment Methods • Comprehensive analysis of
stratified data • Active utilization of Modeling &
Simulation Disease model Objective B/R assessment Identifying AE-related factors etc.
NDA etc.
Effective & High Quality Review & Consultation
• More evidence-based • More transparent
Sophisticated Review & Consultation Practical use of Innovative
Medical Products
Evaluation / Analysis by PMDA Effective and successful
development
More scientific regulatory decision
Epoch-making proposal leading the world
Advancing Regulatory Science
12th Kitasato-Harvard Symposium, Tokyo May 14th 2013
Pharmaceuticals & Medical Devices Agency
Regulatory Science Bridge
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Tominaga T et al, Clin Pharmacol Ther, 90: 29-31, 2011
Patients/ Society
Products of science
(Substance, Knowledge, Information)
Data assessment Data assessment
Stronger & More Complete Regulatory Science Bridge will help us in the future drug developments
Drug A
12th Kitasato-Harvard Symposium, Tokyo May 14th 2013
Pharmaceuticals & Medical Devices Agency
Information
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HOMEPAGE (English) http://www.pmda.go.jp/english/index.html
Regulatory Science Page http://www.pmda.go.jp/regulatory/index.html
E-mail: [email protected]
Thank you for your attention