After many years of encouragement and soft incentives, it is now a legal requirement for
drug companies to develop medicines for children. Bolstering this regulatory initiative is an
extra six months of patent protection provided to companies that develop and provide drugs
for pediatric populations. In the US and EU, pediatric study (PSP) or investigation plans (PIP)
need to be submitted to the regulators in early drug development, to indicate studies that
will be conducted in this population. For drugs not likely to be used by children, deferrals or
waivers can be requested.
As part of the PSP and PIP, a suitable pediatric dose needs to be established. Dose selection
and optimization is one of the major reasons for failures in pediatric drug development
trials. While allometric scaling may be suitable in some cases for dose prediction, more
mechanistic models are often needed to account for the complex interaction between
the developmental physiological, biochemical and drug related factors in children. Modelinformed drug development (MIDD), specifically physiologically based pharmacokinetics
(PBPK) is an invaluable and proven tool that can help avoid unnecessary pediatric studies,
ensure clinical trial dose selection is based on science and minimize clinical trial enrollment,
while delivering relevant treatment data.
By Dr. Karen Rowland Yeo, Sr VP of Client and Regulatory Strategy & Trevor Johnson, PhD, Principal Scientist