tatsuya kondo, m.d., ph.d.rs macroscopia balancing of multiple factor (risk/benefit cost/benefit,...

PMDA’s Regulatory Science and Innovation

Tatsuya Kondo, M.D., Ph.D.

Chief ExecutivePharmaceuticals and Medical Devices Agency (PMDA)

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Type of Financial Interest within last 12 months Name of Commercial Interest

Grants/Research Funding

Stock Shareholder

Consulting Fees

Employee

Other (Receipt of Intellectual Property Rights/Patent

Holder, Speaker’s Bureau)


Regulatory Science

“Rational Medicine” Initiative

International Activities by PMDA


Today’s topics

PMDA’s Three Major Services

Safety Triangle- Comprehensive risk management undertaken by three operations

-

SafetyContinuous

risk mitigation efforts

ReviewReduction

in risk

Relieffor health

Damage by ADRs

Japanese Citizens

World-class pharmaceutical

regulatory system for

protecting the safety of the

people

As the only regulatory authority in the world which plays three roles in an integrated manner,

PMDA contributes to improve the standard of medical care by delivering safer and higher

quality products faster to medical practice based on regulatory science


Innovative Treatment Candidatesarising from Japan

• Nobel prize awarded to many Japanese

researchers

• Strong research capacity especially in

regenerative medicine

Benefit Risk

??

Evaluate for

Regulation

New Technologies with Unknown Safety/Efficacy


RS EngineeringRS MicroscopiaScience of Evaluation

(Quality, Efficacy, Safety)

Benefit Risk

??

Ethical Science and Technology to Benefit People and Society

Science Technology

Achievements of RS Engineering

(e.g.)

Respond to translational research

Modeling & Simulation

Develop guidelines

Establish review standards

Draft legislation etc.

RS MacroscopiaBalancing of Multiple factor

(Risk/Benefit

Cost/Benefit, etc.)

Step 3) Comprehensive evaluation

Step 1) Mapping of elementsStep 2) Microscopic on each

elements

What is Regulatory Science?


Report on

Technology ARegulator

Regulator

Emerging Technologies

With Horizon Scanning…

・ Proactively scan the horizon

for emerging trends, technologies, etc.

・ Make necessary regulatory preparations.

Regulators： cannot keep pace

with accelerating innovation…

Without Horizon Scanning…

Stakeholders：unsure of

regulations…

Horizon Scanning


A patient-centric system• from the perinatal to the final stages of life

• based on the latest scientific knowledge

• takes a holistic approach to medicine

All concerned parties work hard to realize this idea. (healthcare professionals, medical companies,

government authorities, etc.)

What is the “Rational Medicine”?

“Rational Medicine“ Initiative

http://www.pmda.go.jp/english/about-pmda/0012.html




PMDA Published


on Feb. 2017

“Rational Medicine“ Initiative

http://www.pmda.go.jp/english/about-

pmda/0012.html



Name of product

(Applicant)Summary of product Indications

XOFLUZA Tablets 10mg/20mg

(baloxavir marboxil)

by Shionogi & Co., Ltd.

- an antiviral drug indicated for influenza

- novel mechanism

(suppresses influenza viral replication via inhibition of cap-

independent endonuclease enzymes required for viral mRNA

synthesis in host cells)

Influenza

Types A and B

図2 塩野義製薬株式会社プレスリリース資料から引用、一部改変

Novel mechanism of action developed in Japan (Shionogi & Co., Ltd)

11

Influenza Virus

Figure: Excerpted with partial revisions from Shionogi’s original press release

Attachment

Released

from cell

Particle formation

Invasion

Membrane fusion Duplication of genome RNA

mRNA elongation

Polymerase inhibitor(favipiravir)

Start of mRNA synthesis(Cap endonuclease)

Cap-dependent endonuclease inhibitor (baloxavir marboxil)

Viral

genome

RNA

< Timeline >

Oct. 2015: Designated for SAKIGAKE

Feb. 2018: Regulatory Approval

4 months

Oct. 2017: Submission for marketing approval

Particle formation

Neuraminidase inhibitor(e.g. oseltamivir, laninamivir,

peramivir)

Early Patient Access to Innovative Product (1)

Name of product

(Applicant)Summary of product Indications

TITANBRIDGETM

(device for thyroid cartilage

fixation)

by Nobelpharma Co., Ltd.

- A medical device to be used for the treatment of adductor

spasmodic dysphonia

- novel mechanism

(preventing excessively tight closure of the glottis and

maintaining the glottis open)

Type Ⅱ thyroplasty

Novel mechanism of action developed in Japan (Nobelpharma Co., Ltd)

Developed by Dr.

Nobuhiko Isshiki,

Prof. of Kyoto

Univ.

Kumamoto Univ. etc.,

cooperated to

conduct research

Manufactured by

Wakayoshi

Seisakusho Co.,

Ltd. (Fukui pref.)

Nobelpharma,

venture capital,

led them to

practical use

*With AMED research funding support of MHLW Improvement of dysphonia

Over-closing

prevention of glottis

< Timeline >

Feb. 2016: Designated for SAKIGAKE

Dec. 2017: Regulatory Approval

6 months

Jun. 2017: Submission for marketing approval

Early Patient Access to Innovative Product (2)


Functions:

1. Command center on regulatory science

2. Actively utilizes clinical trial data and EHR

3. Promotes innovative approaches to

advanced therapies and technologies

・Science Board

・Horizon Scanning

Office of

Research

Promotion

Office of

Medical

Informatics

and

Epidemiology

Office of

Advanced

Evaluation with

Electronic Data

Office of

New

Drugs

Office of

Safety

Regulatory Science Center (est. April 2018)


1. Innovation through product review with enhanced rigor and

rationality

2. Further promotion of regulatory science

3. More sophisticated safety measures through the use of real-

world data

4. Enhanced international partnerships

PMDA’ Initiatives on Rational Medicine

https://www.pmda.go.jp/files/000216304.pdf

Goals of Rational Medicine


RHSC

/GLP

PMDA’s Multilateral Cooperation


I am

here

As a meeting of the heads of agencies, I have actively participated in every International Coalition of Medicines Regulatory Authorities (ICMRA) meeting.

I strongly believe in the need for international cooperation in promoting regulatory harmonization, and have strengthened PMDA’s international activities

PMDA’s Participation in ICMRA


1

Confidentiality

Arrangement signed

PMDA staff

stationed at the

agency

Joint symposium

heldCooperative

Arrangement signed

Cooperative Arrangement on

cooperation of pharmacopoeia

signed

PMDA’s Bilateral Cooperation


Country Organization Type Since

DENMARK DKMA Confidentiality Arrangement May, 2018POLAND URPL WMiPB Confidentiality Arrangement Oct, 2017

SWEDEN MPA Confidentiality Arrangement Jun, 2017

WHO Confidentiality Arrangement Oct, 2016

EDQM of the Council of Europe Confidentiality Arrangement Sep, 2016

EDQM of the Council of Europe Memorandum of Cooperation Sep, 2016

EU ECConfidentiality Arrangement

(Medical Devices, extended)Jul, 2015

EU EC Confidentiality Arrangement (Cosmetics) Jul, 2015

THE

NETHERLANDSMEB Confidentiality Arrangement Nov, 2014

EU EC/EMA Confidentiality Arrangement (extended) Feb, 2013

FRANCE ANSM Confidentiality Arrangement Nov, 2012

ITALY AIFA Confidentiality Arrangement Nov, 2012

IRELAND IMB (HPRA) Confidentiality Arrangement Oct, 2011

SWITZERLAND Swissmedic Confidentiality Arrangement Nov, 2010

UK MHRA Confidentiality Arrangement Oct, 2010

Cooperation with European Organizations


19

Asian sites

Japan

APEC

1. Help raise the level of regulations in Asia as a whole

2. Training seminar at PMDA, local prefectures in Japan and Asian with manufacturing site

visits

3. APEC Center of Excellence (COE) for Training (Multi-Regional Clinical Trials and Pharmacovigilance)

PMDA Asia Training Center for Pharmaceuticals and Medical Devices Regulatory Affairs (ATC)


Thank you for your attention.

Work Together for patients!!

tatsuya kondo, m.d., ph.d.rs macroscopia balancing of multiple factor (risk/benefit cost/benefit,...

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