Regulatory Science Research in PMDA - kitasato-u.ac.jp

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



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



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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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


Pharmaceuticals & Medical Devices Agency 6

EMA FDA

PMDA



PMDA’s initiatives to advance Regulatory Science

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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;



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



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



Regulatory Science Research in PMDA

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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



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



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



Future workflow of review & consultation in PMDA

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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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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



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



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

Regulatory Science Research in PMDA - kitasato-u.ac.jp

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