Messages for the 10th Memorial Round Table Future Drug ... · Future Drug Development and...

Future Drug Development and Regulatory Science

Messages for the 10th Memorial Round Table

Dr Tatsuya KondoChief ExecutivePharmaceuticals & Medical Devices Agency(PMDA)

10th Annual Meeting DIA Japan 2013November 6-8 | Tokyo

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PMDA’s Role・Three Major Services～ Safety Triangle ～

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SafetyContinuous risk mitigation efforts

ReviewReduction of risk

ReliefRelief measures foradverse health

effects

Japanese citizens

Three‐pillar System to assure public safety

Proud system unique to Japan


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Strategies and Measures for PMDA Innovation

Shorten review time・Reduce drug lag・Reduce device lag

Strengthen and enhance safety measures

Philosophy（Mission Statement）

Regulatory science

Global partnership(Win-Win Relationship)

Increase staffs Enhance training program Academic cooperation Science BoardJoint Graduate School ProgramHuman resource exchange program

Industry‐Government‐Academia collaboration

Pharmaceutical affairs consultation Cross‐sectional project within PMDA IT‐based safety measuresMIHARI ProjectProject for developing medical information database infrastructure

Risk Manager（RM）

Risk Management Plan（RMP） GLP, GCP, GMP ,QMS inspection programs Adverse health effect relief system International strategic plan International liaison officers to US and EU Global partnership with US, EU and Asian

countries（ICH, IMDRF, PIC/S, etc.）

Pharmaceutical affairs are the ultimate medical ethics, and regulatory science is

the underlying science.

Issues with PMDA（past 5 years）

Basic policies to address the issues Efforts made so far


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PMDA continues to improve the public health and safety of our nation by reviewing applications for marketing approval of pharmaceuticals and medical devices, conducting safety measures, and providing relief to people who have suffered from adverse drug reactions.

We conduct our mission in accordance with the following principles:

• We pursue the development of medical science while performing our dutywith greater transparency based on our mission to protect public health and the lives of our citizens.

• We will be the bridge between the patients and their wishes for fasteraccess to safer and more effective drugs and medical devices.

• We make science‐based judgments on quality, safety, and efficacy of medicalproducts by training personnel to have the latest technical knowledge andwisdom in their field of expertise.

• We play an active role within the international community by promotinginternational harmonization.

• We conduct services in a way that is trusted by the public based on our experiencesfrom the past.

Our Philosophy （September, 2008）

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GAP between

expectation and Reality

Medical Needs Traditional Science

Regulatory Science

SAE after approval, Lower success rate, Insufficient risk communication, Uncertainty for decision

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


Roles of Regulatory Sciencein drug development

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Regulatory Science Bridge

8Tominaga T et al, Clin Pharmacol Ther, 90: 29-31, 2011


Details of 3 Factors in Regulatory Science

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Data Assessment

Higher efficiency

BiomarkerM&SAdaptive Designand more

Higher quality

Objective and Quantitative assessment etc. What is Benefit? What is safety?

Better balance

Better methods for benefit/risk assessment

Medical and social needs

Patients voiceand more


• For example,– Efficacy evaluation based on “primary

endpoint”– Safety evaluation based on dose-adverse

event relationship

Micro-Perspective in Regulatory Science

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Assessing data of a study

Microscopic observation


RegulatoryScience

Macro-Perspective in Regulatory Science

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News item×

Active-controlled RCT×

CohortStudy○

ExpertOpinion○

Cohortstudy×

CaseReport

× CaseReport ×

○

Placebo-controlled RCT○

Article

News item

×

○

Rumors

×ExpertOpinion

○ Article

Truth

Evaluation based on Regulatory Science

Scientific, Non-bias,Objective,

Value to the Societyin promoting the Health


Engineering Drug Development

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Pharmaceutical Science

MedicalScience

Biostatistics

AgriculturalScience

Jurisprudence

Economics

Sociology

Veterinary Medicine

Technological Science

Business Administration

Education

Seeds for a drug

Multidisciplinary Expertise in Regulatory Science

Regulatory Science Tree

Practical Drug Use

and More

Ethics


PMDA initiatives to Advance Regulatory Science

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PMDAPharmaceutical Affair

Consultation

PMDA Science Board

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Basic Research

Seeds of medical products

discovered in Japan

Non-Clinical ClinicalQuality

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

AcademiaBoard Member

Innovative Medical

Products

e.g.;HAL

iPSiPS-derived products

Discovery in Basic researche.g;


• 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

Collaborative Graduate School Program

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ＰＭＤＡGraduate School

Collaboration

Joint Graduate School agreement

The agreement with 18 Universities (as of October 2013)


Academia (University, Institute, Hospital)

Proactive establishment of the guideline and standards

Promoting development using innovative techniques

Outcome of research 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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Effective research & development for

regulatory approval

Human ResourceExchange & Development

Training in regulatory science Learning a state‐of‐ the‐art technology

Advanced workflow of review/consultation using innovative assessment techniques

A rational & effective evaluation process for regulatory decision

Giving additional scientific value to submitted data Regulatory Science

e‐Submission of study data

Innovative assessment techniques Comprehensive analysis of stratified data

Sophisticated NDA review

Database

data

Accumulation

Innovative evaluation methods Active utilization of Modeling &

Simulation Disease model Objective B/R assessment Identifying AE‐related factors etc.

NDA etc.• More predictable

efficacy/safety after approval• Reduction of applicant’s work

load• More scientific regulatory

decision

• Epoch‐making proposal leading the world

• Proactive publication of guideline

More evidence‐based consultation

Sophisticated Consultation

Cross‐Products Analysis

Cooperation with Academia

Practical use of Innovative Medical Products

Analysis by PMDA

Effective and High Quality Review

Effective and Successful Development


Data assessment

Regulatory Science Bridge

18Tominaga T et al, Clin Pharmacol Ther, 90: 29-31, 2011

Products of science

(Substance, Knowledge, Information)

Data assessment

More comprehensive and stronger “Regulatory Science Bridge” will help us develop a drug in the future

Patients/Society

Drug A


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Toward Global PMDA

Safety

Review

Relief

Japanesecitizens

Offer an innovative medicine based on Japanese technology to the WorldAdvancing Regulatory Science through International Collaborations

Contribute to Global Health

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Thank you for your attentionご清聴ありがとうございました

谢谢감사합니다

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