Clinical Therapeutics/Volume 30, Number 11, 2008
Eunice Kennedy Shriver National Institute of Child Health
and Human Development Pediatric Formulation Initiative:
Selected Reports from Working Groups
George P. Giacoia, MD; Perdita Taylor-Zapata, MD; and Donald Mattison, MD
Obstetric and Pediatric Pharmacology Branch, Center for Research for Mothers and Children, Eunice
Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of
Health, Department of Health and Human Services, Bethesda, Maryland
ABSTRACT
Background: The Pediatric Formulation Initiative
(PFI) is a project of the Eunice Kennedy Shriuer
National Institute of Child Health and Human
Development (NICHD). The PFI was established to
address the issue of the lack of appropriate formula-
tions in children and to use this activity as a means to
improve pediatric formulations, as mandated by the
Best Pharmaceuticals for Children Act of 2002 and
2007. The PFI began in 2005 with the formation of
3 working groups-Scientific, Economics, and Taste
and Flavor. These groups began the process of identi-
fying issues, gathering needed information, and con-
sidering possible ways to overcome barriers to the
development of pediatric drug formulations.
Objective: The purpose of this supplement was to
provide details of the working groups' activities
through presentation of full-length articles. Also pre-
sented is an article that discusses the 2007 European
Union (EU) regulation on medicinal products for pe-
diatric use.
Methods: Information for this article was gathered
from the proceedings of a PFI workshop, sponsored
by the NICHD, that was held in Bethesda, Maryland,
on December 6 and 7,2005, as well as postworkshop
discussions of the different working groups.
Results: The increased awareness that the majority
of medications used today have not been labeled for
use in children, and have not been tested to define
safety, efficacy, and appropriate dosing, has led to the
passage of legislation in the United States and in the
EU to create incentives to stimulate the testing of
drugs in this special population. It is imperative that
the problems associated with the compounding and
use of extemporaneous formulations as described in
this supplement be addressed. Regulatory barriers to
November 2008
the availability of commercially developed pediatric
formulations in different countries will need to be
minimized or removed. New drug delivery systems
will need to be tested and made available to pediatric
patients. Further research in the mediators of bitter
taste and study of taste blockers, as well as newer
methods for taste testing in pediatrics, should be en-
couraged. An overarching goal for the future is ad-
dressing the economic barriers to develop appropriate
pediatric dosage forms for drugs with limited market
penetration. The lack of appropriate formulations is
part of a larger problem that includes limited develop-
ment and manufacture of medicines tailored for pedi-
atric patients (particularly those affected by neglected
diseases), insufficient investment in drug trials, and
limited research on drug disposition in various pediat-
ric populations worldwide.
Conclusion: The solution to these issues will re-
quire alignment of vision and commitment as a global
priority of policy makers, regulators, scientists, phar-
maceutical sponsors, academic institutions, govern-
ments, and research foundations. (Clin Ther. 2008;
30:2097-2101) © 2008 Excerpta Medica Inc.
Key words: National Institute of Child Health and
Human Development, Pediatric Formulation Initia-
tive, pediatrics, extemporaneous, compounding.
INTRODUCTION
The Pediatric Formulation Initiative (PFI) is a project
of the Eunice Kennedy Shriuer National Institute of
Accepted for publicatIOn August 28,2008
dOl:1 0.1 016/J.cIinthera.2008.11.017
0149-2918/$32.00
© 2008 Excerpta Medica Inc. All nghts reserved.
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