cultivate human resources &cultivate human …pmda’s efforts in medicinal area - cultivate...
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PMDA’s Efforts in Medicinal Area- Cultivate Human Resources &Cultivate Human Resources & Science Board -
Hideo Utsumi, Ph.D.Executive Director, andDirector General of the Office of Review Innovation
25th AnnualDirector General of the Office of Review Innovation Pharmaceuticals and Medical Devices Agency (PMDA)
EuroMeeting4-6 March 2013RAI, Amsterdam
Netherlands
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Today’s Presentation
1. Council for Science and Technology Policy, Cabinet Officey,
2. Establishment of the Science Board3. Current Status of Pharmaceutical Affairs
Consultation on R&D StrategyConsultation on R&D Strategy4. Advancing Regulatory Science and
CCollaboration with Academia
3
Innovation 25 (2006) ← Liberal Democratic PartySeptember 2006 Abe CabinetOctober 2006 Establishment of Committee for the Investigation of Innovation Promotion, Science
Council of JapanIt is said that the word "Innovation" is derived from the Latin "Innovare" (renew) (= "in" (within) + "novare" (change) ) In Japanese the word is rephrased to mean technological renovation andnovare (change) ). In Japanese, the word is rephrased to mean technological renovation and management reorganization or simply renovation or renewal, but innovation also means using new technology and ways of thinking in existing materials and structures to create new value and to make significant changes in society. “Innovation 25” is a long-term strategy initiative for the creation g g y g gyof innovation contributing to the growth with an eye on the year 2025.
(Preventive medicine for individual, Cell & Tissue Products-related technology, advanced nurse-robot, magic bullet for dementia)nurse robot, magic bullet for dementia)
Medical Innovation(November 2010) ← Democratic PartyJapanese Government established a multi-disciplinary group tasked with setting the agendas for medical innovation and research in the country for a ten to twenty-year period, and follow up them up to 50 years.Offi f M di l I ti (Fi t G l Y k N k P t Y i hi M t t )
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Office of Medical Innovation (First General;Yusuke Nakamura, Present;Yoichiro Matumoto)
Science & Technology Basic Plan- Aiming at a Nation that is CreativeAiming at a Nation that is Creative
in Science & Technology -
Basic Plan 4th Stage (FY2011 - 2015)
Basic Plan 2nd Stage
Basic Plan 3rd Stage(FY2006 - 2010)
• 25 Trillion Yen
・ 25 Trillion Yen・Promotion of the
Two Major Inno ations as aBasic Plan 2nd Stage
(FY2001 - 2005)• 24 Trillion Yen・Policy Strategically
• Making Promotion Strategy by the filed, Screening Strategic Science Technologies
Innovations as a Pillar of Growth:
Green Innovation & Life Innovation
Basic Plan 1st Stage(FY1996 - 2000)
・17 Trillion Yen
・Policy Strategically focusing on Science Technology
・ Science T h l S t
Science Technologies &National Key Technologies
• Human ResourcesI i R
・Building New R&D System
Technology System Innovation
• Increasing Resources for Competent Researches
Establishment of PMDA
Innovative Medicinal seeds from Academia in Japan
ACTEMRA® Injection (Tocilizumab (r-INN))
p
●Target Identification / Target Validation
f (O ) f
Approved in JAPAN; April 2005(First marketing authorization)
Professor Tadamitsu Kishimoto (Osaka University, Japan) identified IL-6 related to Castleman’s disease (Blood 1989; 74:1360-1367)
●Extensive research & Development
ACTEMRA® (Tocilizumab) is a humanized monoclonal antibody targeting IL-6 receptor developed by Osaka University and Chugai Pharmaceutical Co., Ltd.
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Innovative Medicinal seeds from Academia in Japan
XALKORI® capsules (Crizotinib(INN))
p
Approved in JAPAN; May 2012
●Target Identification / Target Validation
Approved in JAPAN; May 2012(International Birth Date: Aug. 2011)
Professor Hiroyuki Mano (Jichi Medical University, Japan) identified EML4-ALK fusion gene in non-small-cell-lung cancer (Nature 2007; 448:561-6 etc.,)
●Extensive research & Development●Extensive research & Development
XALKORI ® (Crizotinib) is the ATP competitive inhibitor of tyrosine kinase of the Hepatocyte growth factor receptor developed by Pfizer Inc. NH2
H ClN
CH3H
Cl
ClFN
N
7
Cl
HNCrizotinib
Innovative Medicinal seeds from Academia in Japanp
1950 201X
"HAL" (Hybrid Assistive Limb®)
University of TsukubaUniversity of Tokyo
OLYMPUS GASTROCAMERA GT-IHAL (Hybrid Assistive Limb®)
Copyright: The Japan Society of Mechanical Engineers.
8Copyright: CYBERDYNE INC
Today’s Presentation
1. Council for Science and Technology Policy, Cabinet Officey,
2. Establishment of the Science Board3. Current Status of Pharmaceutical Affairs
Consultation on R&D StrategyConsultation on R&D Strategy4. Advancing Regulatory Science and
CCollaboration with Academia
9
Environments surrounding PMDAWhat should PMDA do next in the course of achievement of acceleration?What should PMDA do next in the course of achievement of acceleration?
<Fiscal Year 2011 Plan>
品目Total Review Period
Regulatory Review Period
Applicants' time
New Drug
<Fiscal Year 2011 Plan>
New Drug(Priority) 9month 6month 3month
New Drug(Standard) 12month 9month 3month
Apr 1st Apr 1st
Increasing Number of Executives and Regular Employees
FY 2007 FY 2011
【Total Review Period New Drug(Priority)】(median)
<Record>Apr 12012
Apr 12012
Total 256 678Total 12.3mths 6.5mths(9.2mths)
Regulatory 4.9mths 4.2mths (4.1mths)Applicant 6.5mhs 2.0mths(5.0mths)Number 20 50(18)
Review Department
Safety
154
29
438
136Number 20 50(18)
FY 2007 FY 2011Total 20.7mths 11.5mths
【Total Review Period New Drug(Standard)】(median)
Safety Department 29 136
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Regulatory 12.9mths 6.3mthsApplicant 7.9mths 5.1mthsNumber 53件 80件
Issues of PMDA
① Being required to conduct review and consultation understanding of the research activities in stat-of-the-artunderstanding of the research activities in stat-of-the-art technologies such as antibody drug, Companion diagnostics, artificial heart, cellular & tissue-based products, medicine, , p , ,cancer vaccine etc.,.
② Being required to adequately conduct review and consultation ② e g equ ed to adequate y co duct e e a d co su tat oin the state of the art technologies from early stage of development for prompt supplying of products among the medical work front,.
③ Requiring a cooperation with academia, to continuously train for reviewers to catch up accelerating innovative technologies and contribute practical use of state of the art technologies.
11Science Board
For PMDA To Be More Science-BasedBasic
Research
Seeds of
Non‐clinical tests
CTQuality Tests
Practical use
Innovative Review Approve
Post Marketing
Pharmaceutical consultation on R&D Strategy
Seeds of new
drug / medical
medical productsClinical Trial
Consultation Review Post Marketing Safety MeasureR&D Strategymedical
devicesOffices of Review (Drugs & Medical Devices), Office of Safety
y
E t bli h t f th S i B d
Office of Review Innovation
Establishment of the Science BoardThe Science Board was established in May 2012 to discuss how PMDA can better cope with products with advanced science & technology, in
h d l l h b i h d l
B d b
each developmental stage such as basic research, development support, product review, and post market safety measures.
AcademiaBoard members
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Science Board and Office of Review InnovationOffice of Review Innovation
Office of Review Innovation
Director G l
Science Board
・Committee members: External experts from AcademiaGeneral
Associate Secretariat
p
・Declare Conflicts of Interest
・Not involved in the Review Process of individual products
Committee
Recommendation on PMDA tasks
Director General
Secretariat Director
MissionImprovements in the scientific aspects of review
Subcommittee
Reform PMDA reviews and related services based on science with consideration for actual medical practices Subcommittee
Deliberation on problems in each fieldCollaboration with PMDA working team (RS research, guideline development, etc.)
ac ua ed ca p ac ces
PMDA Office g p )
PharmaceuticalsMedical Devices
Bio-based Products
Cell & Tissues-Based products
Projects Across Multi-Offices in PMDAReview/Audit/
Inspection
RS
PMDA Office
Safety
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Inspection SGD
RS: Office of Regulatory ScienceSGD: Office of Standards and Guidelines Development
Rotating Science Board memberOutstanding researchers who have knowledge and experience concerning scientific evaluation on Pharmaceuticalsscientific evaluation on Pharmaceuticals and Medical Devices
Science B dBoard2years/person
Discuss how PMDA can better cope with products with advanced science & technologyLeading development for
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advanced science & technologyLeading development for innovative technology
Strengthen Review System in PMDA
Set up Office of Review Innovation (April 2012) and the Science Board (May 2012)
‐ Enhance partnership with academia ‐
Science Board
Subcommittee(Drug)
Subcommittee(Medical Device)
Concerned with
Associate director GeneralDirector General, ffi f i
Subcommittee(biologics)
Subcommittee(cell-and tissue-based products)
Concerned with Academia
Executive Director Center for product evaluation
Secretariat Director
Deputy Associate director GeneralOffice of Review
Innovation
Chief
Executive Director
(Review・Research)
Director Center for product evaluation
Deputy Center Director for medical Devices
S i E i
Review Section
Deputy Center Director for Cellular-and Tissue-based products
Executive
Relief Section
Safety Section
Senior Executive Director
(Technology Management)
Executive
Special Assistant
Tissue based products
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Administration Section
Executive Director
(General Coordination)
Main Roles of Science BoardE t ti I
Committee Thrashing out Science & Technology potentially applied to innovative
Extracting Issue
products(Drug/Medical Device) in the near future among the Cutting-edge of Exploratory Research,
On the advanced scientific technology, Requesting the subcommittee to discuss further for providing review and consultation services appropriatelydiscuss further for providing review and consultation services appropriately in PMDA.
Subcommittee On the subject of advanced scientific technology profound
Request
Review Offices On the subject of advanced scientific technology, profound researchers and the PMDA reviewers deliberate assessment tools in order to provide review and consultation services appropriately.
Review OfficesStudy meetings with profound researchers
Exchange Opinions
Committee Review OfficesProblem consciousnesses in PMDADiscussion on the proposed subjects
Coach for issues
Problem consciousnesses in PMDAAnxious to exchange views on
subjects with the Science Committee.
Discussion on the proposed subjectsRequesting adequate subcommittee
to discuss further for the issues.Propose
Request
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SubcommitteeProfound researchers and the PMDA reviewers deliberate the referred issues from the Science Committee.
Exchange Opinions
Request
1st Fiscal Year Activities
Jun NovOctSepAugJuly Dec Jan Feb Mar
2012 2013
Committee
Subcommittees
Pharmaceuticals
Medical DevicesMedical Devices
Bio-based Products
Cell & Tissues-Based products
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Possible Issues on Science Board
Under Discussion
Pharmaceuticals & Bio products• Discussion about personalized medicine
N li i l Ph l t di ti h ti l• Nonclinical Pharmacology studies on anticancer pharmaceuticals
Medical Devices• Discussion about
• Registry for Medical Device• Category of application for Generic Medical Device• Category of application for Generic Medical Device• Development for combination product
Cell- & tissue- Based productsCell & tissue Based products• Discussion about
• Tumorigenicity
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• Requirement for CPC
Today’s Presentation
1. Council for Science and Technology Policy, Cabinet Officey,
2. Establishment of the Science Board3. Current Status of Pharmaceutical Affairs
Consultation on R&D StrategyConsultation on R&D Strategy4. Advancing Regulatory Science and
CCollaboration with Academia
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Pharmaceutical Affairs Consultation on R&D Strategygy
Valley of Death -Short of funds, Knowledge on Regulation and development strategy, g g p gy
Strategic ConsultationPractical
Use
Basic Research
Pharmaceutical and
Strategic Consultation
Innovative Products
Pharmaceutical and Medical Devices
candidatesNon-
Clinical Study
Quality Study
Clinical Trial
Up to the levelf POC diStudy of POC studies
* Further studies are handled by the Regular
Consultation on quality or toxicity study of biologics,
Consultation on endpoints or sample size
Consultation
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y y gcell-and tissue-based products
p pof early clinical trial
Flow of R&D Strategy Consultation
Regulation Data Package Discussion
I t d tPre- Face-to-Face
Introductory Consultation(No Charge)
Consultation(No Charge)
Consultation (charged)
Binding(No Charge) - Not binding- 30 minutes
- Binding- Written record to Applicant- 2 hours
331 54370
331Consultations
54Consultations
Consultations
(7/1/2011 – 12/28/2012)21
Today’s Presentation
1. Council for Science and Technology Policy, Cabinet Officey,
2. Establishment of the Science Board3. Current Status of Pharmaceutical Affairs
Consultation on R&D StrategyConsultation on R&D Strategy4. Advancing Regulatory Science and
CCollaboration with Academia
22
Program of Collaborative Graduate Schools(MEXT)Graduate Schools(MEXT)
1.SummaryIt i f th d ti l d h th d f thIt is one of the educational and research method for the education in graduate schools to take advantage of utilities and/or human resources of extramural National Institutes and/orand/or human resources of extramural National Institutes and/or private laboratories which conduct higher level research.
2.Position of the systemA graduate school may, when deeming it to be effective from an d ti l t d i t d t h l t d t ll t t keducational standpoint, graduate school students allow to take
the necessary research guidance in extramural laboratory etc., (Article 13 in the requirements for establishing graduate schools)(Article 13 in the requirements for establishing graduate schools). The Program of collaborative graduate schools is systematically implement in this system.
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Program of Collaborative Graduate SchoolsGraduate Schools
PMDAGraduate
Collaboration
PMDA schoolConclusion of
•• PMDA StaffsPMDA Staffs–– Engaging on education/research in the university asEngaging on education/research in the university as
Conclusion of agreement
Engaging on education/research in the university as Engaging on education/research in the university as visiting professor etc.visiting professor etc.
–– Conducting the research and pursuing Ph.D. as graduate Conducting the research and pursuing Ph.D. as graduate g p g gg p g gstudentstudent
•• Graduate school studentsGraduate school students–– Accepted graduate students from university are provided Accepted graduate students from university are provided
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p g y pp g y pfor research guidance learning about PMDA’s operation for research guidance learning about PMDA’s operation and pursing Ph.D.and pursing Ph.D.
Program of Collaborative Graduate SchoolsGraduate SchoolsAgreement with 15 Universities (as of November, 2012)
Yamagata University
M hi U i it University of Tsukuba
C
Musashino University
Kyoto Pharmaceutical University Chiba University
Teikyo University
UniversityOkayama University
Yokohama City University
Shujitsu University
K b U i it Shi k P f t lGifu Pharmaceutical
University
Kobe University Shizuoka Prefectural University
Osaka University Nagoya University
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UniversityGifu University
Osaka University Nagoya University
医療イノベーションプロジェクト厚生労働省Enhancement on approval review / safety measures in response to the progress of technology (MHLW New Budget FY 2012:2.1 billion yen)
Promoting practical use of innovative drug, medical device, Cell- & Tissue-based product
(1)Enhancement on Approval Review/ Safety Measure in Response to the Progress of Technology 【1.2 billion yen】
○ Develop draft guideline/guidance based on Regulatory Science ○ Promote human resource exchange between PMDA & research institutes
(2)Developing guideline/guidance for innovative drug/medical device/biologics to streamline regulatory review based on Regulatory Science 【366 million yen】
Research I tit ti P l
Develop Guidelines
streamline regulatory review based on Regulatory Science 【366 million yen】
NIH
Establishment on approval review/safety measures
Institutions Personal Exchange
SupportCultivate Human Resource
MHLW
(3)Strengthen measure for the safety of unknown risk for innovative technology 【0.35 billion yen】
Resource
(4)Dealing with globalization of production and distribution of Drug, Medical Device, Cell- &Tissue-based product 【0.18 billion yen】
Promoting of personal exchangeDrug
・Cancer / Alzheimer Disease / Pediatric
北海道大学大学院薬学研究院
京都大学大学院医学研究科
北海道大学大学院医学研究科
・Biomarker・Gene therapy/Nucleic acid medicine / nanotechnology
東北大学大学院薬学研究科
大阪大学大学院薬学研究科
東北大学大学院医工学研究科
大阪大学大学院
京都大学iPS細胞研究所
Medical Devices・Innovative Therapeutic apparatus・Combination productQuantitative non clinical Evaluation
筑波大学医学医療系
国立循環器病研究センター 国立がん研究セ
大阪大学大学院医学系研究科
国立がん研究センター中央病院
Cell & Tissues
・Quantitative non-clinical Evaluation・Endoscope
東京大学医学部附属病院
研究センタンター東病院
千葉大学大学院医学研究院
早稲田大学(先端生
九州大学大学院医学研究院
・iPS / ES cells・Stroke / Spinal Cord injury・Clinical Path Initiative・Establish on Reflection Paper for
附属病院
名古屋市立大学大
東京大学大学院工学系研究科
先端医療振興財団
命医科学センター)
国立成育医療研究センター・病院
国立成育医療研究
Exchanging program in FY2012 Planning employment for 18 researchers from university, Research
Establish on Reflection Paper for evaluation
名古屋市立大学大学院薬学研究科 国立成育医療研究
センター
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g p y y,Institute etc., as accepted graduate students
Planning temporary transfer from 28 PMDA staffs (including non-regular staff)to University, Research Institute etc..
Summaryy
• Science Board was established to be more science based review / consultation in PMDA.
• Pharmaceutical Affairs Consultation on R&D Strategyffis offering consultation to innovative products
developed by academia/venture businesses.
• Exchanging program between Academia and PMDA ill lti t h d l d l d ftwill cultivate human resources, and also develop draft
guideline/guidance.
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Thank you for your y yattention !
http://www.pmda.go.jp/29