Pediatric Drug Research: Substantial Increase in Studies of Drugs for Children, But Some Challenges Remain Page: 4 of 11
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the difficulty of attracting enough pediatric patients for studies because of
the small number of children with a particular disease.
Previous FDA efforts to address the problem of inadequate pediatric
testing and drug labeling information had been unsuccessful. For example,
in 1994 FDA tried to encourage sponsors to provide more pediatric
information and conduct new studies. However, it did not require
sponsors to conduct new pediatric studies, and pediatric use information
did not substantially increase.
In 1997, the Congress recognized the importance of learning more about
how drugs work in children by including in FDAMA a financial incentive
for pharmaceutical manufacturers and drug sponsors to conduct pediatric
studies and submit the results to FDA. The pediatric exclusivity provision
offered 6 months of additional marketing exclusivity for drugs tested by
manufacturers and other sponsors for use in children. This provision also
required FDA to develop, prioritize, and publish an annual list of approved
drugs for which new pediatric information may produce health benefits in
the pediatric population. FDA's initial priority list, issued in May 1998, was
developed based on recommendations from experts in pediatric research
from the American Academy of Pediatrics, PhRMA, GPhA, the National
Institutes of Health, the Pediatric Pharmacology Research Units Network,3
the U.S. Pharmacopoeia, and several others. To be included on FDA's
priority list, a drug had to meet one of the following criteria:
* The drug would be a significant improvement compared to marketed
products labeled for use in the treatment, diagnosis, or prevention of a
disease in the relevant pediatric population.
* The drug is widely used in the pediatric population, with at least 50,000
projected uses per year.
* The drug is in a class or for an indication for which additional therapeutic
or diagnostic options are needed for pediatric patients.
The process for obtaining the pediatric exclusivity extension usually
begins when a sponsor submits a proposal to conduct pediatric studies to
FDA.4 If FDA officials believe the studies will provide useful information,
3In 1994, NICHD established a network of Pediatric Pharmacology Research Units (PPRUs)
to facilitate and conduct pediatric drug trials that can improve pediatric labeling of new
and existing drugs.
4Drugs do not have to be on FDA's priority list in order for FDA to consider a sponsor's
GAO-01-705T Pediatric Drug Research
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United States. General Accounting Office. Pediatric Drug Research: Substantial Increase in Studies of Drugs for Children, But Some Challenges Remain, text, May 8, 2001; Washington D.C.. (digital.library.unt.edu/ark:/67531/metadc290169/m1/4/: accessed December 10, 2018), University of North Texas Libraries, Digital Library, digital.library.unt.edu; crediting UNT Libraries Government Documents Department.