PMDA’s Perspectives in Global Clinical Data Evaluation for ... · 7th Annual Conference in Japan...
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PMDA’s Perspectives in Global Clinical Data Evaluation for Drug Approval
April 15th, 2013
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
7th Annual Conference in Japan for Asian New Drug Development April 15-16, 2013 | Nakano Sunplaza, Tokyo, Japan
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7th Annual Conference in Japan for Asian New Drug Development April 15-16, 2013 | Nakano Sunplaza, Tokyo, Japan
Recent trend of Dug-Lag in Japan
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FY2006 FY2007 FY2008 FY2009 FY20100
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1.2
2.4
1.5 1.5
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1.21
0.70.5
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2.4
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2.22
1.1
Lag in Development Time
Lag in Review Time
Drug
-Lag
(Yea
r)
Total Lag
7th Annual Conference in Japan for Asian New Drug Development April 15-16, 2013 | Nakano Sunplaza, Tokyo, Japan
Japanese:http://www.pmda.go.jp/operations/notice/2007/file/0928010.pdf
English:http://www.pmda.go.jp/operations/notice/2007/file/0928010-e.pdf
Japanese version English version
Basic principles on Global Clinical Trials
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7th Annual Conference in Japan for Asian New Drug Development April 15-16, 2013 | Nakano Sunplaza, Tokyo, Japan
Trends of Global Clinical Trials including Japan
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% o
f MR
CTs
Num
bers
of C
TN
FY2007(4-9)
FY2008(10-3)
FY2009 FY2010 FY2011(4-9) (10-3) (4-9) (10-3) (4-9) (10-3) (4-9) (10-3) (4-9) (10-12)
FY2012
7th Annual Conference in Japan for Asian New Drug Development April 15-16, 2013 | Nakano Sunplaza, Tokyo, Japan
Operational Area of GCTs
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0
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EastAsia only
Asia only
Asia+欧米
World wide
No Other Asia
Uncertain
7th Annual Conference in Japan for Asian New Drug Development April 15-16, 2013 | Nakano Sunplaza, Tokyo, Japan
Target Disease of GCTs
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7th Annual Conference in Japan for Asian New Drug Development April 15-16, 2013 | Nakano Sunplaza, Tokyo, Japan
Development Strategy for drug approval in Japan
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FY2007 FY2008 FY2009 FY2010 FY2011 FY20120
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Total Bridging GCT
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of A
ppro
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% of Bridging % of GCT % of Asian GCT
%
7th Annual Conference in Japan for Asian New Drug Development April 15-16, 2013 | Nakano Sunplaza, Tokyo, Japan
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Name of Drug Indication Approval Tolterodine Overactive bladder with symptoms of urge urinary
incontinence, urgency, and frequency Apr. 2006
Insulin glulisine Diabetes mellitus Apr. 2009 Peramivir * Type A and Type B Influenza virus infection Jan. 2010 Temsirolimus Advanced renal cell carcinoma Jul. 2010 Laninamivir * Type A and Type B Influenza virus infection Sep. 2010
Edoxaban* Prevention of venous thromboembolism after major orthopedic surgery Apr. 2011
Indacaterol Chronic obstructive pulmonary disease (COPD), including chronic bronchitis and/or emphysema. Jul. 2011
Gefitinib EGFR-Positive unresectable or metastatic non-small cell lung cancer (NSCLC) Nov. 2011
Aripiprazole Manic episodes associated with bipolar disorder Jan. 2012
Exenatide Type II diabetes mellitus (adjunctive to diet, exercise and treatment with SU) Mar. 2012
Esomeprazol Risk reduction of low-dose asprin-induced gastric or duodenal ulcer Jun.2013
Stratera Attention-Deficit/Hyperactivity Disorder in adulthood Aug.2013 Insulin degludec* Diabetes mellitus Sep.2013 Insulin degludec/ Insulin aspart* Diabetes mellitus Dec.2013
Fesoterodine Overactive bladder with symptoms of urge urinary incontinence, urgency, and frequency Dec.2013
Asian GCT-based Drug approval in Japan
7th Annual Conference in Japan for Asian New Drug Development April 15-16, 2013 | Nakano Sunplaza, Tokyo, Japan
New Guidance (Sep 5th 2012): Basic principles on Global Clinical Trials
(Reference Cases)
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日本語:http://www.pmda.go.jp/regulatory/file/guideline/new_drug/GCT_jirei.pdf 英語:http://www.pmda.go.jp/regulatory/file/english_guideline/new_drug/GCT-jirei_en.pdf
7th Annual Conference in Japan for Asian New Drug Development April 15-16, 2013 | Nakano Sunplaza, Tokyo, Japan
• Accumulating new scientific knowledge and regulatory experiences after the publication of the 2007 guideline
• Diversifying a cooperation style between Japan and other countries; e.g. Asian GCT, Large-scale GCT
• Reinforcing the regulatory cooperation among Japan, China and South Korea as well as that among Japan, U.S. and Europe.
Background of the New Guidance
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Further promotion of GCTs, especially in East-Asian region, is the key to successful drug development
7th Annual Conference in Japan for Asian New Drug Development April 15-16, 2013 | Nakano Sunplaza, Tokyo, Japan
• Data from well-designed and conducted global clinical trials in East Asia is acceptable for new drug application in Japan.
• To conduct East Asia GCT appropriately and successfully, it is necessary to consider impacts on drug efficacy/safety of ethnic factor among East Asian populations before starting the trial
• Further accumulation and review of scientific data and information on East Asian populations will deepen our understanding of ethnic differences and ensure a smooth and appropriate conduct of global clinical trials in this region.
• It is encouraged to consider to include global clinical trials to be conducted in East Asia as part of drug development plan and accumulate information.
Major points in the new guidance (Special points to consider in East-Asian GCTs: 1/2)
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7th Annual Conference in Japan for Asian New Drug Development April 15-16, 2013 | Nakano Sunplaza, Tokyo, Japan
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Major points in the new guidance (Special points to consider in East-Asian GCTs: 2/2)
• East-Asian GCT can be performed in any disease • Proactive planning of East-Asian GCT is encouraged
especially for diseases with high morbidity in East Asia (e.g., gastric cancer and hepatitis)
• Positioning of East-Asian GCT in entire global development plan should be clarified in advance
• All activities in East Asia should be carried out in cooperation with those in the U.S. and Europe.
7th Annual Conference in Japan for Asian New Drug Development April 15-16, 2013 | Nakano Sunplaza, Tokyo, Japan
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Major points in the new guidance (Other points to consider)
• Daily cooperation with foreign partners (Affiliate, HQ, other regulatory agency etc.)
• Optimize a strategy based on collected data • Acceptability of foreign population data on PK/PD • Sample size calculation in case of large-scale GCT • Safety assessment based on GCT, especially describing
an importance of Japanese data on long-term safety evaluation
7th Annual Conference in Japan for Asian New Drug Development April 15-16, 2013 | Nakano Sunplaza, Tokyo, Japan
Classification of development strategies based on PK-profile
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Major difference
No major difference
No major difference
Major difference
PK comparison among East-Asian population
Regional study in East Asia
World wide collaboration
Collaboration study in
Japan/US/EU Local study in Japan
Comparison of PK between Japanese and Caucasian
Major Difference No Major difference
PK comparison among East-Asian population
7th Annual Conference in Japan for Asian New Drug Development April 15-16, 2013 | Nakano Sunplaza, Tokyo, Japan
Future GCTs
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7th Annual Conference in Japan for Asian New Drug Development April 15-16, 2013 | Nakano Sunplaza, Tokyo, Japan
• Effects of ethnic factors on drug efficacy/safety should be more characterized
• Methods for planning/evaluation should be established – Sample size calculation, consistency
evaluation etc. • Regulatory harmonization
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Challenges for better GCTs
Advancing regulatory science
7th Annual Conference in Japan for Asian New Drug Development April 15-16, 2013 | Nakano Sunplaza, Tokyo, Japan
• China/Korea/Japan Tripartite Working Group • MHLW Research group (Kawai study group, Saito study group)
– Examining impacts of ethnic factor on drug efficacy/safety
– Clarifying points to consider in better planning and data evaluation of GCTs
Regulatory Science Research
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7th Annual Conference in Japan for Asian New Drug Development April 15-16, 2013 | Nakano Sunplaza, Tokyo, Japan
Development Strategy
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Japan
Other Asia
US/EU
PK, Safety
Exploratory, Dose-Finding
Confirmatory POC
Japan only
West only
7th Annual Conference in Japan for Asian New Drug Development April 15-16, 2013 | Nakano Sunplaza, Tokyo, Japan
Development Strategy
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Japan
Other Asia
US/EU
PK, Safety
Exploratory, Dose-Finding
Confirmatory POC
Japan only
West only
J/W GCT
J/W GCT
Japan/West
7th Annual Conference in Japan for Asian New Drug Development April 15-16, 2013 | Nakano Sunplaza, Tokyo, Japan
Development Strategy
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Japan
Other Asia
US/EU
PK, Safety
Exploratory, Dose-Finding
Confirmatory POC
Japan only
West only
J/W GCT
J/W GCT
Early Japan/West
J/W GCT
7th Annual Conference in Japan for Asian New Drug Development April 15-16, 2013 | Nakano Sunplaza, Tokyo, Japan
Development Strategy
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Japan
Other Asia
US/EU
PK, Safety
Exploratory, Dose-Finding
Confirmatory POC
Japan only
West only
W/J/EGCT
W/J/EGCT
West/Japan/ Asia
7th Annual Conference in Japan for Asian New Drug Development April 15-16, 2013 | Nakano Sunplaza, Tokyo, Japan
Development Strategy
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Japan
Other Asia
US/EU
PK, Safety
Exploratory, Dose-Finding
Confirmatory POC
Japan only
West only
Asian GCT
Asian GCT Asia
7th Annual Conference in Japan for Asian New Drug Development April 15-16, 2013 | Nakano Sunplaza, Tokyo, Japan
Development Strategy
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Japan
Other Asia
US/EU
PK, Safety
Exploratory, Dose-Finding
Confirmatory POC
Japan only
West only
Asian GCT
Asian GCT Asia
Asian GCT
Asian GCT Early
Asia
Asian GCT
7th Annual Conference in Japan for Asian New Drug Development April 15-16, 2013 | Nakano Sunplaza, Tokyo, Japan
Development Strategy
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Japan
Other Asia
US/EU
PK, Safety
Exploratory, Dose-Finding
Confirmatory POC
Japan only
West only
W/J/EGCT
W/J/EGCT
W/J/E GCT
W/J/E GCT
Asia Leading East
Asian GCT
7th Annual Conference in Japan for Asian New Drug Development April 15-16, 2013 | Nakano Sunplaza, Tokyo, Japan
Development Strategy
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Japan
Other Asia
US/EU
PK, Safety
Exploratory, Dose-Finding
Confirmatory POC
Japan only
West only
Asian GCT
Asian GCT
Real Asia
Asian GCT
Asian GCT
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EU North America USA
Canada Asia
Drugs from Asia to the world
Cooperation for better drug developments
Pharmaceuticals & Medical Devices Agency
Information
• 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
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