Pediatric Drug Research: Food and Drug Administration Should More Efficiently Monitor Inclusion of Minority Children
Highlights
Drug effectiveness and adverse events can vary between children and adults and among racial and ethnic groups. The Food and Drug Administration (FDA) is authorized under the pediatric exclusivity provision to grant drug sponsors 6 months of additional exclusive marketing rights for conducting clinical drug studies in children. The Best Pharmaceuticals for Children Act of 2002 (BPCA) expanded this provision to require FDA to take into account the adequacy of minority representation in pediatric exclusivity studies. BPCA also directed GAO to evaluate the representation of minorities in such studies. GAO examined the extent to which minority children are represented, whether drugs that treat diseases disproportionately affecting minority groups are studied under the provision, and FDA's monitoring of the representation of minority children in the studies. GAO reviewed related FDA documents, FDA requests for pediatric studies and final study results, and interviewed FDA officials and other experts.
Recommendations
Recommendations for Executive Action
| Agency Affected | Recommendation | Status |
|---|---|---|
| Food and Drug Administration | To help the agency more efficiently monitor the participation of children of racial and ethnic groups in studies for additional exclusive marketing rights, the Commissioner of the Food and Drug Administration (FDA) should specify in written requests that study sponsors must use the racial and ethnic categories described in FDA's January 2003 draft guidance to identify study participants in their reports to the agency. FDA can refuse to grant 6 months of additional exclusive marketing rights under the pediatric exclusivity provision for sponsors that do not fairly respond to FDA's written requests. |
Closed – Implemented
Consistent with GAO's recommendation, FDA now includes in its written requests that reports are to include information on the representation of pediatric patients of ethnic and racial minorities, using the categories from the draft guidance. All pediatric patients enrolled in studies are to be categorized using one of the following designations for race: American Indian or Alaska Native, Asian, Black or African American, Native Hawaiian or other Pacific Islander, or White; and the following categories for ethnicity: Hispanic/Latino or Not Hispanic/Latino.
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