Global Pediatric Development of Drugs, Biologics, and Medical Devices
113
Introduction
The Ministry of Health, Labour and Welfare
(MHLW), commonly known as Kōrō-shō
in Japanese, is the cabinet-level governmen-
tal branch that oversees pharmaceutical drug
development policy and makes the final decision
on product marketing in Japan.1 In conjunc-
tion with the MHLW, the Pharmaceutical and
Medical Device Agency (PMDA) conducts
scientific reviews of pharmaceutical marketing
authorization applications, including benefit-risk
assessments of the quality, efficacy, and safety
of products across the development continuum,
from preclinical to risk mitigation in the post-
marketing setting. PMDA also is responsible for
good clinical practice (GCP) inspections, good
manufacturing practice (GMP) inspections,
clinical trial consultation, and relief services for
health damage caused by risk factors.2
The MHLW and PMDA requirements for
approval based on the quality, efficacy, and safety
of pharmaceuticals, quasi-pharmaceutical prod-
ucts, cosmetics, medical devices, and regenerative
medicine products is regulated under Article 1 of
the Pharmaceutical and Medical Devices (PMD)
Act.3 This is the region-specific legislation that
mandates plans for the development of adult
pharmaceuticals in Japan. Conversely, there are
no laws or regulations established for pediatric
drug development in Japan.4 Table 10-1 provides
a comparison of Japan, US, and EU pediatric
development regulations.
History
There is currently no planned date for issuance of
Japan-specific pediatric legislation. Per PMDA,
the lack of pediatric study feasibility is a priority
that must be considered prior to implementation
of any regulation.5 Appropriately sized pediatric
domestic studies leading to the needed level of
evidence is often difficult to perform in Japan
due to the number of children in the region
ranging from ages 0–14 years.6 However, there
are a number of ongoing key initiatives support-
ing pediatric development in Japan. Table 10-2
provides a timeline of key initiatives supporting
pediatric development in Japan. As a found-
ing member of the International Council for
Harmonisation of Technical Requirements for
Pharmaceuticals for Human Use (ICH), since
December 2000, Japan has recognized ICH E11
Guideline on Clinical Investigation of Medicinal
Products in the Pediatric Population.7 The original
guidance was published and issued in Japan as
Notification No. 1334 of the Evaluation and
Licensing Division. This guidance provides
an outline of critical issues in pediatric drug
development and approaches to the safe, efficient,
and ethical study of medicinal products in the
pediatric population.8 It was later amended (ICH
E11 (R1)) in 2017 to complement and provide
clarification and current regulatory perspective
on scientific advancements relevant to pediatric
populations and drug development.9
In February 2010, the Unapproved Drugs/
Indications Scheme and study group were
established. The scheme objective is to improve
Drug Development—Japan
By Monique Carter, MS, RAC
10
113
Introduction
The Ministry of Health, Labour and Welfare
(MHLW), commonly known as Kōrō-shō
in Japanese, is the cabinet-level governmen-
tal branch that oversees pharmaceutical drug
development policy and makes the final decision
on product marketing in Japan.1 In conjunc-
tion with the MHLW, the Pharmaceutical and
Medical Device Agency (PMDA) conducts
scientific reviews of pharmaceutical marketing
authorization applications, including benefit-risk
assessments of the quality, efficacy, and safety
of products across the development continuum,
from preclinical to risk mitigation in the post-
marketing setting. PMDA also is responsible for
good clinical practice (GCP) inspections, good
manufacturing practice (GMP) inspections,
clinical trial consultation, and relief services for
health damage caused by risk factors.2
The MHLW and PMDA requirements for
approval based on the quality, efficacy, and safety
of pharmaceuticals, quasi-pharmaceutical prod-
ucts, cosmetics, medical devices, and regenerative
medicine products is regulated under Article 1 of
the Pharmaceutical and Medical Devices (PMD)
Act.3 This is the region-specific legislation that
mandates plans for the development of adult
pharmaceuticals in Japan. Conversely, there are
no laws or regulations established for pediatric
drug development in Japan.4 Table 10-1 provides
a comparison of Japan, US, and EU pediatric
development regulations.
History
There is currently no planned date for issuance of
Japan-specific pediatric legislation. Per PMDA,
the lack of pediatric study feasibility is a priority
that must be considered prior to implementation
of any regulation.5 Appropriately sized pediatric
domestic studies leading to the needed level of
evidence is often difficult to perform in Japan
due to the number of children in the region
ranging from ages 0–14 years.6 However, there
are a number of ongoing key initiatives support-
ing pediatric development in Japan. Table 10-2
provides a timeline of key initiatives supporting
pediatric development in Japan. As a found-
ing member of the International Council for
Harmonisation of Technical Requirements for
Pharmaceuticals for Human Use (ICH), since
December 2000, Japan has recognized ICH E11
Guideline on Clinical Investigation of Medicinal
Products in the Pediatric Population.7 The original
guidance was published and issued in Japan as
Notification No. 1334 of the Evaluation and
Licensing Division. This guidance provides
an outline of critical issues in pediatric drug
development and approaches to the safe, efficient,
and ethical study of medicinal products in the
pediatric population.8 It was later amended (ICH
E11 (R1)) in 2017 to complement and provide
clarification and current regulatory perspective
on scientific advancements relevant to pediatric
populations and drug development.9
In February 2010, the Unapproved Drugs/
Indications Scheme and study group were
established. The scheme objective is to improve
Drug Development—Japan
By Monique Carter, MS, RAC
10