Promoting Access toBetter Innovative Drugs

FUJIWARA Yasuhiro, M.D., Ph.D.
Chief Executive,
Pharmaceuticals and Medical Devices Agency (PMDA)

23 rd April 20241

Today’s Topics
1. 5th Mid-term plan (FY2024–FY2028)

2. PMDA’s efforts to improve access to better
innovative products
3. PMDA’s international cooperation in Asia

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Today’s Topics
1. 5th Mid-term plan (FY2024–FY2028)

2. PMDA’s efforts to improve access to better
innovative products
3. PMDA’s international cooperation in Asia

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Basic structure for 5 th Mid-term planSafety Triangle

Review

For world-class regulatory system
Japanesecitizens

SafetyRelief⚫ For further “Quality” through Regulatory Science⚫ For Strategic international activities⚫ Governance and recruit professional personnel

4Direction for 5 th Mid-term plan
［FY2024-2028］⚫ For further “Quality” through Regulatory Science- Consultation/review for pharmaceuticals etc. for the innovative products- Proper follow-up of safety measures- Emergent response system e.g. Pandemic⚫ For strategic international activities‒ Regulatory support/Disseminate regulatory information to overseas
companies to develop innovative products in Japan⚫ Governance and professional personnel

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Today’s Topics
1. 5th Mid-term plan (FY2024–FY2028)

2. PMDA’s efforts to improve access tobetter innovative products
3. PMDA’s international cooperation in Asia

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Current Status in Japan
Current approval situation
U.S./EU/Japan

Undeveloped items
Japan

Unapproved products
Approved

Unapproved(Total)

Developing

Undeveloped

U.S.

136

734

EU

86

57

2631

Japan

0143

57

86

Ventures
Start-up

Orphans

Pediatrics

56%

47%

37%(48)

(40)

(32)

※ Of 86 items, 14 items (16%) were not categorized asventures, orphans or pediatrics※ Source: Prepared by the office of Pharmaceutical Industry Research based on published information from PMDA, FDA, and EMA, and Biotoday (Technomics, Inc.), andtabulated by the Ministry of Health, Labour and Welfare.
※ 1: Of the NMEs approved in Europe and the U.S. in 2016-2020, those not approved in Japan as of the end of 2022 are counted as unapproved.
※ 2: As of March 2023, items for which no development information was available are counted as undeveloped products in Japan.
※ 3: Figures are totaled for development companies with sales of less than US$500 million within 30 years of approval in Europe and the U.S.
※ 4: Compiled as orphans for items designated as orphan drugs by the time of approval in Europe and the U.S.
※ 5: As of the end of 2022, items approved for pediatric use in Europe and the U.S. are counted as pediatrics

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MHLW: Study group on pharmaceutical regulations
to strengthen drug discovery capabilities and ensure stable supplies in Japan
1st study group(10 Jul )Start from 10 July, 2023 and ongoing nowDevelopment• Designation of Orphan Drugs.
• Pharmaceutical reviews that contribute to the promotion ofdevelopment of pediatric drugs.

Clinical Trials• Organizing the necessity of Japanese data in the drug review• Introduction of more efficient clinical trials (ecosystem).

2 nd, 3 rd, 6 thstudy group(7 Aug , 13 Sep,13 Dec)

Post-marketing • How to conduct post-marketing drug use results surveys.
safety measures • Utilization of Real World Data in the pharmaceutical regulations.
4 th, 5 th study group(13 Oct, 15 Nov)

Quality• Approaches to drug reviews of drug manufacturing methods.

RegulatoryInformation• Dissemination of information on pharmaceutical regulations in Japanto Overseas.
5 th study group(15 Nov)

https://www.mhlw.go.jp/stf/shingi/other-iyaku_128701_00006.html (Japanese only)

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PMDA: Regulatory Advisory Centerfor Pediatric / Orphan drugs (Tentative)

Expected activities
1. Accelerate/expand orphan drug designation

2. To drug companies : A plan for pediatric drugs⇒ PMDA confirm the plan
3. Accelerate the evaluation
at “Study Group on Unapproved and Off-label Drugs of High Medical Need”

4. To companies: Subsidize the consultation fee etc.
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Regenerative Medical Products Interpreter
Accompanied support for appropriate consultation, etc.
~ from clinical research through clinical trials to product applications ~Implementing Body：PMDA

Adopt

AMEDP rog ram Su p e rv is or

AccompaniedSupport

Research
ThemesSupportConsult,Administrate
ProgressCore ClinicalResearch Centers

MHLW

PMDA

P roje c t Man ag e r
Consult,Administrate
Progress
Re g e n e rativ e Me d ic al
P rod u c ts In te rp re te r

SupportTranslational
ResearchCenters

MHLW: Ministry of Health, Labour and Welfare
MEXT: Ministry of Education, Culture, Sports, Science and TechnologyMEXT10For Overseas companies
Information on Japanese Pharmaceutical Regulations○MHLW/PMDA systematically disseminate, in English, on their website○The materials : Newly prepared/disseminated.

https://www.mhlw.go.jp/content/11120000/kousei_iyaku_shiryou_en_ol_230704.pdf

https://www.pmda.go.jp/files/000266927.pdf

11Today’s Topics
1. 5th Mid-term plan (FY2024–FY2028)

2. PMDA’s efforts to improve access to better
innovative products
3. PMDA’s international cooperation in Asia

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“Reliance”
Grand Design for Asian Pharmaceutical and Medical Device Regulatory Harmonization“When a regulatory authority of one country region conducts approval reviews or inspections, they
consider, attach importance to, and utilize in their regulatory activities, the outcomes of assessments
made by their counterparts in other countries/regions.”
Headquarters for Healthcare Policy of Japan (20 June 2019)

Draft Good regulatory practices for regulatory oversight of medical products“The act whereby the NRA (National Regulatory Authority) in one jurisdiction may take into accountand give significant weight to assessments performed by another NRA or trusted institution, or to any
other authoritative information in reaching its own decision. ”WHO (August 2020)

Statement from Global Medicines Regulators on the Value of Regulatory Reliance“Regulatory reliance, which is a mechanism to strengthen regulatory capacity, to improve healthsystems nationally and internationally, to increase the availability of medicines, to save financial
resources and to use human resources more strategically.“ICMRA (27 November 2020)

Momentum: “reliance” for worldwide!

13PMDA-ATCAsia Training Center for Pharmaceuticals andMedical Devices Regulatory Affairs

Capacity building/human resource development for Asian regulatorsAction Policy of PMDA-ATC
Universal health coverage through regulatory harmonization in Asia

Japan
Asian countries
Site visit/ Lectures /Case studies/Practical trainings

Tailored trainings to needs

Invites Asian regulators

Input Japanese knowledge/experiences
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The Commemorative Summit for the 50 th Yearof ASEAN-Japan Friendship and Cooperation

2. Partners for Co-creation of Economy andSociety of the Future
2.8 Health2.8.6 Promote cooperation on expansion of access toquality health services, pharmaceuticals and medicaldevices for non-communicable diseases including training
in regulating pharmaceuticals and medical devices
through platforms such as the Asia Training Center for
Pharmaceuticals and Medical Devices Regulatory
Affairs (PMDA-ATC);
Excerpt from the “Implementation Plan of the Joint Vision
Statement on ASEAN-Japan Friendship and Cooperation“(Photos: Cabinet Public Affairs Office)15

PMDA-ATC Seminars(Open to all regulators)
Trainings: FY 2023 (April 2023 to March 2024)
Period(days)

Location

1 Pediatric Review*1

4Tokyo (PMDA)

2 Quality Control (Herbal Medicine)

3Toyama

3 Pharmaceuticals Review

3Online4 Medical Devices*2

3Online5 Medical Devices

3Tokyo (PMDA)

6 Multi-Regional Clinical Trial (MRCT) *2, 3

4Tokyo (PMDA)

7 Good Manufacturing Practice (GMP)

3Online

8 Pharmacovigilance*24Online

Contents*1 Joint Seminar with U.S.FDA*2 APEC CoE Workshop*3 Collaboration with National Cancer Center Japan16

Updating the PMDA-ATC Training Seminars

More well-organized, thoughtful, and practicaltrainings- Questionnaire: From ATC Seminar Participants- Feedback: For refined seminar

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Cooperation between WHO and PMDA on
training for regulatory authorities

PMDAAsiaOffice

ASEANCountries• Systematizing:Collaboration on trainings/seminars
by PMDA/WHO through facilitators• Effective capacity building/training
in ASEAN countries.
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Long-term Training Program
Letter of Intent on a long-term
training program
signed by The Ministry of Health of theRepublic of Indonesia (MoH) & PMDA

Training for medical device
regulation
under the framework of the PMDA-ATC
Signing Ceremony of the Letter of Intent (LOI)on Long-term Training Program on 5 th July, 2023
PMDA Updates August 2023 https://www.pmda.go.jp/files/000263976.pdf

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PMDA Asia Office(Bangkok, Thailand)
Objective
Contribute to innovative products access with on-sitecommunicationthrough➢ Cooperation with ASEAN regulators➢ Regulatory harmonisation with Asian countries➢ Collaboration: Clinical research network

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International Collaboration and RelianceSignificantly important
than ever before• Globalisation of supply chainRegulatory
Convergency• Emergence of new technologies• Limited human resources• Response and Preparedness for pandemic(COVID-19 and the Next), etc...

International

Fast/Stable access

Collaboration

through reliance enhancement
ex. Contribute: ASEAN Joint Assessment scheme(by WHO/PMDA collaboration)

Information
Sharing

Capacity
Building

https://www.pmda.go.jp/english/int-activities/0010.html

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Thank you for your attention

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