Fostering Further Collaboration between PMDA and Applicants with Efficient Project Management in Drug Development

Fostering Further Collaboration between PMDA and Applicants with Efficient Project Management in Drug Development Kazuishi Sekino Review Director Office of New Drug I Pharmaceuticals & Medical Devices Agency Pharmaceuticals and Medical Devices Agency, Japan

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Outline of Presentation 1. Current Situation of Approval Review 2. The Elements of Project and Priority 3. Progress Management of Approval Review Process 4. Progress Management of Scientific Advice process 2015 DIA, Inc. All rights reserved. 12th Annual Meeting DIA JAPAN 2015 November 15-17 Tokyo Big Sight Ariake 3

Review Time for New Drugs (median) (Months) (Total review time = Regulatory review time + Applicant s time) The PMDA s Second Mid-term Plan The PMDA s Third Mid-term Plan (Numbers of approved products) FY2014 PMDA: Pharmaceuticals and Medical Devices Agency <https://www.pmda.go.jp/files/000206389.pdf> 2015 DIA, Inc. All rights reserved. 12th Annual Meeting DIA JAPAN 2015 November 15-17 Tokyo Big Sight Ariake 4

Six regulatory Authorities Approval Time of New Drugs in 2004-2013 EU US Japan Canada Switzerland Australia Regulatory Affairs Professional Society 2015/1/14 2015 DIA, Inc. All rights reserved. 12th Annual Meeting DIA JAPAN 2015 November 15-17 Tokyo Big Sight Ariake 5

The PMDA s Third Mid-term Plan(FY2014 to FY2018) The plan for prompt realization of zero review lag for new drugs Review Time for New Drugs (Priority Review) Total review time (Regulatory review time + Applicant s time);9 months(6 months + 3 months) Review Time for New Drugs (Standard Review) Total review time (Regulatory review time + Applicant s time) ;12 months(9 months + 3 months) Target percentile FY2014 FY2015 FY2016 FY2017 FY2018 60% 60% 70% 70% 80% PMDA: Pharmaceuticals and Medical Devices Agency <http://www.pmda.go.jp/guide/jyohokokai/kohyo/file/1-7keikaku3.pdf> 2015 DIA, Inc. All rights reserved. 12th Annual Meeting DIA JAPAN 2015 November 15-17 Tokyo Big Sight Ariake 6

Outline of Presentation 1. Current Situation of Approval Review 2. The Elements of Project and Priority 3. Project Management for Process of Approval Review 4. Project Management for Process of Scientific Advice 2015 DIA, Inc. All rights reserved. 12th Annual Meeting DIA JAPAN 2015 November 15-17 Tokyo Big Sight Ariake 7

The Three Elements of Project and Priority Time (start and end time, the necessary time) Resource (reviewers) Project Scope, Quality (different each time) Top Priority (Mandatory Goal) Second Priority (Goal to Strive) Allowance Time Resource Scope, Quality プロジェクトマネジメント理論編 ( 第 2 版 ) 総合法令出版一部改変 2015 DIA, Inc. All rights reserved. 12th Annual Meeting DIA JAPAN 2015 November 15-17 Tokyo Big Sight Ariake 8

Flow of Review for New Drugs (in the past) Submission PMDA MHLW Pre- Submission Meeting NDA Filling PMDA s Evaluation List of Questions to Applicant PMDA s Evaluation External Expert Discussion PMDA s Opinion PAFSC Approval by MHLW Standard Review 1.9-2.5 months Initial Meeting Assessment Report 0.2-0.7 months 4.7-8.3 months 1.2-1.9 months 0.7-1.9 months Standard Review: Total 12 months Priority Review: Total 9 months MHLW: Ministry of Health, Labour and Welfare NDA: New Drug Application PMDA: Pharmaceuticals and Medical Devices Agency PAFSC: Pharmaceutical Affairs and Food Safety Council 2015 DIA, Inc. All rights reserved. 12th Annual Meeting DIA JAPAN 2015 November 15-17 Tokyo Big Sight Ariake <Administrative Notice of MHLW, March 30, 2012> 10

Flow of Review for New Drugs (at present) Submission PMDA MHLW Pre- Submission Meeting NDA Filling PMDA s Evaluation List of Questions to Applicant PMDA s Evaluation External Expert Discussion PMDA s Opinion PAFSC Approval by MHLW Standard Review Priority Review 2.4 months 1.9 months Initial Meeting 0.6 months 0.3 months Assessment Report 5.7 months 1.5 months 0.9 months 4.4 months 1.3 months 0.8 months Standard Review: 12 months Priority Review: 9 months MHLW: Ministry of Health, Labour and Welfare NDA: New Drug Application PMDA: Pharmaceuticals and Medical Devices Agency PAFSC: Pharmaceutical Affairs and Food Safety Council 2015 DIA, Inc. All rights reserved. 12th Annual Meeting DIA JAPAN 2015 November 15-17 Tokyo Big Sight Ariake <Administrative Notice of MHLW, January 30, 2015> 12

Flow of Scientific Advice for New Drug Development Applicant s Action Intention to Apply Submission of Document Response to List of Questions Response to PMDA s Opinion Pre- Submission Meeting Scheduling Preparing Document PMDA s Evaluation List of Questions to Applicant PMDA s Evaluation PMDA s Opinion to Applicant Face to Face Meeting Minutes 1 week (2-5 weeks) (1 week) 1 week (1 week) 1 week 1 month 2 months (8-11 weeks) 5 weeks PMDA: Pharmaceuticals and Medical Devices Agency 2015 DIA, Inc. All rights reserved. 12th Annual Meeting DIA JAPAN 2015 November 15-17 Tokyo Big Sight Ariake 14

Number of Scientific Advice for New Drugs (Review Category 1; Gastrointestinal Drugs etc.) (N) <https://www.pmda.go.jp/0017.html> 2015 DIA, Inc. All rights reserved. 12th Annual Meeting DIA JAPAN 2015 November 15-17 Tokyo Big Sight Ariake 15

US & EU Japan Example of the GANNT chart (I) 2013 2014 2015 2016 2017 2018 2019 1Q 2Q 3Q 4Q 1Q 2Q 3Q 4Q 1Q 2Q 3Q 4Q 1Q 2Q 3Q 4Q 1Q 2Q 3Q 4Q 1Q 2Q 3Q 4Q 1Q 2Q 3Q 4Q Hypothetical situation Best timing? Reflection of PMDA s opinion to protocol? Top-Line Results Scientific Advice PMDA PMDA Applicant s Decision Making Phase I trial Phase II trial Delay of enrollment of Japanese patients? FDA, EMA FDA, EMA Global Phase III trial Phase I trial Phase II trial EMA: European Medicines Agency FDA: US Food and Drug Administrationl PMDA: Pharmaceuticals and Medical Devices Agency 2015 DIA, Inc. All rights reserved. 12th Annual Meeting DIA JAPAN 2015 November 15-17 Tokyo Big Sight Ariake 16

US & EU Japan Example of the GANNT chart (II) 2014 2015 2016 2017 2018 2019 2020 1Q 2Q 3Q 4Q 1Q 2Q 3Q 4Q 1Q 2Q 3Q 4Q 1Q 2Q 3Q 4Q 1Q 2Q 3Q 4Q 1Q 2Q 3Q 4Q 1Q 2Q 3Q 4Q Hypothetical situation Best timing? Phase I trial PMDA Reflection of PMDA s opinion to protocol? Best road map? FDA, EMA, PMDA Top-Line Results Scientific Advice Applicant s Decision Making FDA, EMA Global Phase II trial Global Phase III trial Phase I trial Phase IIa trial EMA: European Medicines Agency FDA: US Food and Drug Administrationl PMDA: Pharmaceuticals and Medical Devices Agency 2015 DIA, Inc. All rights reserved. 12th Annual Meeting DIA JAPAN 2015 November 15-17 Tokyo Big Sight Ariake 17

Items for the Project Management Time (starting and ending time, the required time) Project Resource (reviewers) Examples of items for the project management The target product profile The strategic development plan (and current status) The GANNT chart (development history and project scheduling, including discussion to regulatory agencies) etc. Scope, Quality (different each time) Effective utilization of pre-submission meeting (sharing the information between applicant and PMDA) プロジェクトマネジメント理論編 ( 第 2 版 ) 総合法令出版一部改変 PMDA: Pharmaceuticals and Medical Devices Agency 2015 DIA, Inc. All rights reserved. 12th Annual Meeting DIA JAPAN 2015 November 15-17 Tokyo Big Sight Ariake 18