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Ethical Conduct of Clinical Research Involving Children
fied the dozen highest-priority drugs needing pediatric testing (DHHS, 2003a). Most of the drugs are no longer under patent, and therefore, the “pediatric exclusivity” incentives are largely irrelevant. The secretary of DHHS announced that $25 million in federal funds would be allocated to support research on these drugs in 2003.
Recently, Congress passed the Pediatric Research Equity Act of 2003, (P.L. 108-155). It gives FDA the authority to require pediatric studies of certain drugs and biological products. In the future, companies submitting requests for approval to market a new drug or biologic (or a new indication, formulation, dosing regimen, or route of administration for an already approved product) will be required to submit information about the safety and effectiveness of the product in relevant pediatric populations. Testing may not be required if the agency determines that it is appropriate to extrapolate data from studies with adults (usually with some supplementary pediatric pharmacokinetic or other study data). Submission of pediatric data may be deferred under certain conditions (e.g., the adult but not the pediatric studies are completed). The requirements may also be fully or partially waived under several conditions (e.g., pediatric studies are impossible or highly impractical or existing evidence suggests that the drug would be unsafe or ineffective for children). If a waiver were granted on the basis of evidence that a drug would be unsafe or ineffective, the drug label would be required to include information to that effect.
Other agencies that are not part of FDA or NIH may also have been influenced by these initiatives. For example, in its program of Centers for Education and Research on Therapeutics (CERTs), which supports research and education on effective therapeutics (i.e., drugs, medical devices, and biologics), the Agency for Health Research and Quality has designated one center to focus specifically on medical therapies for children (UNC, 2003). The CERTs program was authorized in P.L. 105-115.
Food and Drug Administration Rules and Policies
Even though the best and brightest pediatric minds have helped us establish dosages for children, we’re finding out that the dose is different than we thought in some cases. And that probably came as a surprise to most of us.
Richard Gorman, quoted by Meadows, 2003, unpaged
In 1979, FDA issued regulations on the content and format of labels for human prescription drugs (FDA, 1979a). The regulations stated that if drug companies made statements about the pediatric use of a drug for an approved indication, the statement had to be based on substantial evidence from adequate and well-controlled studies, unless FDA waived that require-