Pediatric Drug Research: Substantial Increase in Studies of Drugs for Children, But Some Challenges Remain Page: 3 of 11
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exclusivity provision.2 We also examined supporting documents provided
by FDA, as well as those provided by the other groups we contacted. We
conducted our work from March through April 2001 in accordance with
generally accepted government auditing standards.
In brief, since enactment of the pediatric exclusivity provision, both the
numbers of drugs studied in children and the therapeutic classes they
represent have substantially increased. Hundreds of studies are being
done on drugs that are important to pediatric patients. Some are tests on
relatively small numbers of pediatric patients to determine the correct
dose for a specified age group. Others are more complex and costly
evaluations of a drug's safety and effectiveness in children of various ages.
While there has been some concern that exclusivity may be sought and
granted primarily for drugs that generate substantial revenue, most of the
drugs studied are not top sellers, and less than 1 in 10 generates revenues
of more than $1 billion a year. As of April 1, 2001, 28 drugs had been
granted marketing exclusivity extensions, and research results have
provided new and useful information about how drugs work in children,
which have been incorporated into labels for 18 drugs. However,
challenges remain to ensure that the results of pediatric research are
expeditiously incorporated into drug labels, and that incentives are
provided to encourage pediatric studies of off-patent drugs widely used in
According to the American Academy of Pediatrics, only about a quarter of
all approved drugs marketed in the United States have had clinical trials
performed involving pediatric patients. FDA's January 2001 report to
Congress on the pediatric exclusivity provision noted that evidence from
several studies conducted since 1973 showed that between 71 and 81
percent of drugs were inadequately labeled for use in pediatric patients.
According to the legislative history of FDAMA, several factors appear to
have contributed to the lack of pediatric studies. Drug companies
indicated that they had little incentive to perform pediatric studies on
drugs they intended to market primarily to adults and that these drugs
would provide little additional revenue from use in children. Companies
also said they were concerned about liability and malpractice issues and
2 The Pediatric ExclusivityProvision, January2001 Status Report to Congress, Department
of Health and Human Services, U.S. Food and Drug Administration.
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/m1/3/: accessed September 25, 2018), University of North Texas Libraries, Digital Library, digital.library.unt.edu; crediting UNT Libraries Government Documents Department.