Global Pediatric Development of Drugs, Biologics, and Medical Devices
1
Introduction
Regulations and initiatives continue to be
established to foster research and development of
drugs, biological products, and medical devices
for the pediatric population. However, there
continues to be a lag from the time of approval in
adults to the time of approval for pediatrics. To
facilitate the development of medicinal products
for pediatrics, global initiatives have been created
and are described in this chapter.
World Health Organization (WHO)
WHO plays a significant role in improving child
health from many different angles, including
nurturing care, promoting healthy growth and
development, and strengthening health services.
In 2007, WHO published Promoting Safety of
Medicines for Children1 following a recommenda-
tion of the WHO Advisory Committee for the
Safety of Medicinal Products. The Committee
expressed concerns on the lack of data on
medicines used in children. To further facilitate
the market access of pediatric drugs, WHO
published Model List of Essential Medicines for
Children in 2013 as the core drug list for a basic
healthcare system. This resource list considers the
most efficacious, safe, and cost-effective drugs
for priority pathologies in pediatric patients up
to 12 years old. In addition, WHO launched the
International Clinical Trials Registry Platform
(ICRTP) to ensure a complete view of research is
accessible to all those involved in healthcare deci-
sion making. The ICRTP also has been leveraged
to improve awareness and provide easier access
for clinical trials in children, contributing to
the Millennium Development Goal of reducing
child mortality.
The International Council for
Harmonization of Technical
Requirements for Pharmaceuticals
for Human Use (ICH)
ICH has published two guidelines to recom-
mend international standards and promote
harmonization in support of development of
pharmaceuticals intended for pediatric use. These
published guidelines are: Clinical Investigation
of Medicinal Products in the Paediatric Population
E11, adopted in 2000 with an addendum in
2017, and Nonclinical Safety Testing in Support of
Development of Paediatric Medicines S11, adopted
in 2020. A third guideline is under development,
with the objective of aligning terminology on
pediatric extrapolation, providing a system-
atic approach for utilizing extrapolation, and
providing guidance for both study designs and
statistical analysis methods for incorporating
extrapolation into a pediatric drug development
plan.2
The purpose of the E11 guideline3 is
to encourage and assist sponsors with global
medicinal product development. The guideline
provides considerations for initiating pediat-
ric development along with recommendations
for timing of studies and the types of studies
to be conducted, specifically pharmacokinetic,
Global Regulatory Initiatives
By Linda McBride, RPh, RAC
1
1
Introduction
Regulations and initiatives continue to be
established to foster research and development of
drugs, biological products, and medical devices
for the pediatric population. However, there
continues to be a lag from the time of approval in
adults to the time of approval for pediatrics. To
facilitate the development of medicinal products
for pediatrics, global initiatives have been created
and are described in this chapter.
World Health Organization (WHO)
WHO plays a significant role in improving child
health from many different angles, including
nurturing care, promoting healthy growth and
development, and strengthening health services.
In 2007, WHO published Promoting Safety of
Medicines for Children1 following a recommenda-
tion of the WHO Advisory Committee for the
Safety of Medicinal Products. The Committee
expressed concerns on the lack of data on
medicines used in children. To further facilitate
the market access of pediatric drugs, WHO
published Model List of Essential Medicines for
Children in 2013 as the core drug list for a basic
healthcare system. This resource list considers the
most efficacious, safe, and cost-effective drugs
for priority pathologies in pediatric patients up
to 12 years old. In addition, WHO launched the
International Clinical Trials Registry Platform
(ICRTP) to ensure a complete view of research is
accessible to all those involved in healthcare deci-
sion making. The ICRTP also has been leveraged
to improve awareness and provide easier access
for clinical trials in children, contributing to
the Millennium Development Goal of reducing
child mortality.
The International Council for
Harmonization of Technical
Requirements for Pharmaceuticals
for Human Use (ICH)
ICH has published two guidelines to recom-
mend international standards and promote
harmonization in support of development of
pharmaceuticals intended for pediatric use. These
published guidelines are: Clinical Investigation
of Medicinal Products in the Paediatric Population
E11, adopted in 2000 with an addendum in
2017, and Nonclinical Safety Testing in Support of
Development of Paediatric Medicines S11, adopted
in 2020. A third guideline is under development,
with the objective of aligning terminology on
pediatric extrapolation, providing a system-
atic approach for utilizing extrapolation, and
providing guidance for both study designs and
statistical analysis methods for incorporating
extrapolation into a pediatric drug development
plan.2
The purpose of the E11 guideline3 is
to encourage and assist sponsors with global
medicinal product development. The guideline
provides considerations for initiating pediat-
ric development along with recommendations
for timing of studies and the types of studies
to be conducted, specifically pharmacokinetic,
Global Regulatory Initiatives
By Linda McBride, RPh, RAC
1