good submission practice (gsubp) guideline for applicantsedu.tcfst.org.tw/edm/gsp/speaker/session...

September 17, 2015Isao Sasaki

Astellas Pharma Inc. / JPMA

Good Submission Practice (GSubP)Guideline for Applicants

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Topics

1. About APAC (Asia Partnership Conference of Pharmaceutical Associations)

2. Concept of Good Registration Management (GRM)

3. Contents of GSubP Guideline

4. Implementation plan of GSubP

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What is APAC?

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Established by 12 R&D based pharmaceutical associations in Asia in 2012

Fourth annual convention meeting was held in Tokyo in April 2015

APAC Members

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DomesticR&D typeAssociation

NationalResearchInstitute

Academia

Japan

JPMA

(OCCI)

China

SIMM

RDPAC

PhIRDA

South Korea

KDDF

KPMA

KRPIA

Malaysia

PhAMA

Thailand

TCELS

India

NCBS

Hong‐Kong

HKU pharmacy programm

Singapore

EDB

SAPI

Taiwan

Academia

Sinica

DCB

TRPMA

IRPMA

Indonesia

IPMG

Philippines

PHAP OPPIHKAPI PReMA

BIOTECH‐CORP

(Pink = RA-EWG, Green = DA-EWG)

InternationalR&D typeAssociation

Early Access to New

Medicine

R&D

Intellectual property/ Compulsory licensing

Regulations&

Approval system

Medical insurance/ Reimburse‐ment/HTA

Product quality/

Distribution/ Counterfeit medicines

Medical infrastructure

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“To expedite the launch of innovative medicines for the peoples in Asia”

Mission

APAC Organization

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APAC

RA‐EWGRegulations & Approvals

DA‐EWGDrug Discovery Alliances Future WG for ATM

DA Expert Team RA Expert Team Future Expert Team

Steering Committee

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APAC RA‐EWG Direction

To expedite the launch

of innovative medicines

for the peoples in

Asia

Road MapIdentified Issues

IND

NDA

ClinicalTrials

GMP

AnalysisReport

Deliverables at 2nd APAC

Ass

essm

ent o

f rea

dine

ss

Con

sulta

tion

with

regu

lato

rs

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Ass

essm

ent &

gap

ana

lysi

s

Step１

Collaboration with stakeholders

(e.g. regulators)

Goal

Regulatory acceptancefor an early initiation

of multi-national clinical trials in Asia

Regulatory acceptance and Co-review

system for NDA

SimultaneousNDA in

all Asian countries

Acceleration of mutual recognition of GMP

inspection

APAC Mission

Concept Paper

Promoting regulatory convergence in Asia would

lead to achieve the APAC Mission

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Goal and Approach of RA‐EWG

Promotion of Regulatory ConvergenceNDA Co-review system

Acceptance of NDA review

Promote each task in close collaboration with the Asian regulatory authorities.

Task A: Good Registration Management Introduce Good Submission Practice Support promotion of Good Review Practice by making proposals (Position Paper)

Task B: Convergence of NDA Requirements Make proposals to facilitate regulatory

convergence of NDA requirements

Topics





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GRevP & GSubP

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Promoted by regulatory authorities under APEC RHSC topic

To enhance quality, transparency, timeliness, predictability of review

Good Review Practice (GRevP)

Industry initiative in response to GRevP

To enhance quality of application submission and its management aiming to obtain early approval

Good Submission Practice (GSubP)

What we expect with GSubP

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For review authorities1. No need to make

unnecessary inquiry2. Possible to focus on key

points/issues during review

3. Possible to enhance efficiency and timeliness of review

For applicants1. Risk of rejection and

number of critical/major deficiencies can be reduced

2. Possible to gain reputation and trust in high quality submission

3. Possible to obtain early approval

Enhance efficiency and quality of product registration process

Concept of Good Registration Management (GRM)

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1. Synergic effects by parallel promotion of GRevP and GSubP2. Close communication between review authorities and applicants

ApplicantsReview Authorities Applicants



Good Registration Management

Deliverables of RA‐EWG in 4th APAC (Apr. 2015)

APAC GSubP Guideline

http://www.jpma.or.jp/english/apac/4th_apac.html

APAC Position Paper

Improve quality of submissionand its management

Make proposals to support facilitation of GRevP

Objective:

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Topics





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Scope of APAC GSubP Guideline

Setting out general and high level guidance for applicants to make good submission

Sufficiently expandable to accommodate additional annexes or ancillary documents in the future

Focusing on NDA of human drugs including line extensions (e.g., new indication, dosage)

Covering the whole product registration process from development, submission and review stages up to approval

Aiming to balance what applicants should do with what reviewers could also keep in mind

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Possible documentation structure of GSubP

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High‐level Practical & Specific

Country specific GSubP guideline or annex Based on regulatory

system/process and requirements in the country

SOPs/manuals for submission To be prepared in

applicant’s organization

GSubP Guideline Provides general &

high level guidance for applicants in all regions

History of GSubP Guideline

Apr. 2014 Provided concept of GSubP in the APAC Good Registration Management (GRM) Policy Document

Aug. 2014 Agreed in APEC RHSC that APAC would work on draft of GSubP Guideline

Dec. 2014 First draft of APAC GSubP GuidelineMar. 2015 Final draft released for APAC endorsementApr. 2015 Obtained endorsement at the 4th APAC convention

meeting. Start revision* of the GuidelineAug. 2015 Submitted revised GSubP Guideline to APEC RHSC for

review

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* Minor revisions were made to expand scope of the guideline1. beyond APAC region2. other medical products, e.g. generic drugs, medical devices

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GSubP Guideline: Table of Contents

1. INTRODUCTION2. PRINCIPLES OF GOOD

SUBMISSION3. MANAGEMENT OF

SUBMISSION 4. COMMUNICATIONS5. COMPETENCY AND

TRAINING 6. GLOSSARY7. REFERENCES

GSubP Guideline for Applicants

1. Introduction2. Glossary3. Principles of a good

review4. Managing the review5. Communications6. Review personnels7. Conducting the review

Bibliography

Good review practices: guidelines for national and

regional regulatory authorities

WHO Technical Report Series, No. 992, 2015, Annex 9

1. Strong Scientific Rationale and Robust Data with Clarification of Benefit‐Risk Profile

2. Compliance to Up‐to‐date Regulatory Requirements

3. Well‐Structured Submission Dossier with Appropriate Cross‐references

4. Reliability, Quality, Integrity and Traceability of Submission Documents and Source Data

5. Effective and Efficient Communications

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2. PRINCIPLES OF GOOD SUBMISSION

3. MANAGEMENT OF SUBMISSION

Planning for submission Start discussion on submission strategy from early stage of

product development Use support tools effectively e.g. check‐list, template, glossary

Quality Check Provide instructions on QC for

Study report and summary document writing Compiling of dossier Electronic dossier

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Preparation and Submission of Application Dossier Provide general instructions on report/summary writing,

compiling and submission Encourage creating SOPs

Support tools

Check-list:....... may include name of each document with information such as responsible person/party, target date and status. Such list will be useful not only to check if there is any missing component but also to manage the whole process of submission preparation efficiently

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Support tools

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Glossary:It is important to keep consistency of terminology used throughout a submission dossier. .... recommended to create a list of general glossary before initiating preparation of study reports and summaries.

Template:..... help authors to prepare each component document in structured and consistent manner complying with the required format and contents.... It will also enhance efficiency of preparation. Submission with a unified format of study reports and summaries also enables reviewers to perform review smoothly.

4. COMMUNICATIONS

Communications with review authorities Make effective use of pre‐/post‐ submission meetings Manage inquiry and response appropriatelye.g. clarifications, timeline management

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Manage inquiry and response appropriately

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An example of clear inquiryRegarding the treatment for <proposed indication>, there is no effectiveness and safety data to support such use on <specific population> patients. Please provide data (clinical and PK data) to justify the dosage and administration for such treatment on <specific population> patients as claimed in the draft package insert. Please put special effort into elucidating the reasons and basis for the differences in doses between <specific population> patients over and under <body weight> Kg.

Inquiry and response....., it is important for applicants to clarify and understand the background as well as intention of the reviewer with that inquiry. To make it possible, the review authorities often allow applicants to ask for clarification. Applicants should make the best use of such opportunity.

4. COMMUNICATIONS

Communications with review authorities Make effective use of pre‐/post‐ submission meetings Manage inquiry and response appropriatelye.g. clarifications, timeline management

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Communications amongst applicants Confirm operation model, role and responsibility of the

submission team & members Establish standard working procedure and

communication platform

Communications amongst applicants

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Among submission team members

Among regionse.g. head quarters – affiliates

Among license partnerse.g. originator - licensees

5. COMPETENCY AND TRAINING

Core Competency of Applicants Scientific knowledge and expertise Good understanding of up‐to‐date regulations Other hard and soft skills,

e.g. Planning & PJ management, medical writing, IT skills for e‐submission, problem solving, communication

Integrity, reliability and ethical standards

Training and Capacity Building Participate in external training programs Gain experiences through day‐to‐day operations,

e.g. in‐house training, self‐training, OJT Create and use archive of experiences and knowhow

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Core competency of applicants

5.1 Core Competency of ApplicantsGood understanding of up-to-date regulationsApplicants should always keep abreast with the latest regulatory environment. This can be done by following the regulatory authorities’ website and check updated news, notices or highlights.If available, applicants can also subscribe to a mail delivery service provided by the review authorities to allow applicants to receive updated regulatory information from the authorities’ website periodically.

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Applicants try to follow new information on the authorities’ website

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PMDA (Eng website)

http://www.pmda.go.jp/english/index.htmlCDE (Eng website) http://www.cde.org.tw/eng/

TFDA (Eng website) http://www.fda.gov.tw/EN/index.aspx

Topics



3. History and contents of GSubP Guideline


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Current and Next steps for GSubP Guideline2015• Review request on the updated GSubP at the APEC

RHSC (Aug. 2015) Expanded the scope of GSubP : Beyond Asia (reviewed thru

IFPMA) & added other medical products• Dissemination of the concept of GSubP through

Conference, Symposium, Workshop and Seminar2016 ‐• Finalization of GSubP in align with GRevP• Initiation of GSubP training for applicants

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The RA‐EWG intends widespread implementation of the GSubP guideline through the training.

1. Basic Concept of GRM

2.Outline of GRevP Guideline 3. Outline of GSubP Guideline

Common Training

Applicant Specific GSubP Training

Reviewer Specific GRevP Training To be developed in each

review authority To be developed in each country/

area by industry Based on the requirements of

application submission in that country/area

Draft structure of GRM* training

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* Good Registration ManagementTraining for reviewers

4. Internationally harmonized guidelines/standards

1. Basic Concept of GRM

2.Outline of GRevP Guideline 3. Outline of GSubP Guideline

Common Training

Reviewer Specific GRevP Training To be developed in each

review authority

Draft structure of GRM* training

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* Good Registration Management

4. Internationally harmonized guidelines/standards

Training for applicants

Applicant Specific GSubP Training

To be developed in each country/ area by industry

Based on the requirements of application submission in that country/area

Further improvement in transparency, predictability and timeliness of review by facilitating communication

APACGSubP

Guideline

APACPosition Paper

RA‐EWG Activities and Future Goal‐ Promotion of Good Registration Management

Enhance efficiency of NDA review

Realize early access to new medicines for peoples in Asia

Improve quality of submissionand its management

Make proposals to support facilitation of GRevP

Reduced number of critical deficiencies

Decrease of rejections

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Good Registration Management

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