However, for hypertension  drugs where differences in racial response have been documented in adult  drug studies, FDA required, and drug sponsors included, larger numbers of  children from specific racial and ethnic groups. Most of FDA’s written  requests for studies that have been issued since BPCA took effect required  drug sponsors to report the number of racial and ethnic minorities in their  final study results. The Proportions of Children in Racial and Ethnic Minority Groups in Clinical Studies for Exclusive Marketing Rights Were Lower Than Their Proportions in the U.S. Population

Compared with their proportions in the U.S. population, smaller  proportions of African American, Hispanic, and Asian children were  included in clinical studies for the drugs that were granted 6 months of  additional exclusive marketing rights by FDA from January 4, 2002,  through March 6, 2003. Pediatric Studies for Hypertension Drugs Included More Children of Racial and Ethnic Minority Groups

FDA required that sponsors increase representation of children of ethnic  and racial minority groups in clinical studies for drugs used to treat  diseases that disproportionately affect children in such groups or where  evidence from studies on adults suggests that for certain classes of drugs  differences in metabolism or response for racial or ethnic groups exist. Drugs of Importance to Minority Children Are Being Studied in Response to Pediatric Exclusivity Provision Requests

Some drugs that may be used to treat diseases or conditions that  disproportionately affect children of racial and ethnic minority groups are  being studied under the pediatric exclusivity provision. In addition, 6 of the 22 written requests for new  studies that FDA issued to drug manufacturers during this period also  included treatments for diseases or conditions disproportionately affecting  minorities, such as type II diabetes, hypertension, sickle cell anemia, HIV,  and hepatitis B.

	FDA Monitoring of Data on Minority Representation Needs Improvement

FDA does not have a system in place to serve as a single source of data to  allow the agency to efficiently determine the extent of minority enrollment  in drug studies under the pediatric exclusivity provision. Further, we  found that some study reports submitted to FDA from drug sponsors did  not specify the race and ethnicity of study participants. For example, in  the completed studies for the 23 drugs granted additional exclusive  marketing rights that we examined, the race or ethnicity of 86 percent of  study participants was identified, but study sponsors did not specify the  race or ethnicity of 960 children, or 14 percent of the studies’ populations. Recently, FDA issued draft guidance to improve drug sponsors’ reporting  of racial and ethnic minority representation data, and FDA is planning to  develop a database to monitor demographic variables in drug trials across  the agency. Eighty-six percent of  study participants were identified by race or ethnicity. FDA officials told  us that it would be several years before the system is operational. FDA also agreed with our  recommendation and reported that it has already begun to implement it. First, FDA  was critical of our comparison of the proportions of minority children  study participants to the proportions of minority children in the  population. Further, FDA  has previously used the methodology we employed in its analyses of adult  study participants. To determine whether drugs used to treat conditions or diseases  disproportionately affecting minorities are being studied under the  pediatric exclusivity provision, we obtained data on the prevalence of  selected diseases or conditions that disproportionately affect minorities  and examined the list of drugs for which FDA has either granted exclusive  marketing rights or issued study requests from January 4, 2002, through  March 6, 2003, to determine if any of these drugs may be used to treat  these diseases or conditions.
Why GAO Did This Study

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.

What GAO Found

Compared with the proportions of children from racial and ethnic minority groups in the U.S. population, smaller proportions of children from minority groups were included in the pediatric clinical drug studies requested by FDA before the enactment of BPCA that GAO reviewed. However, FDA required, and drug sponsors included, larger proportions of African American children in clinical studies for hypertension drugs because there is evidence that hypertension is more prevalent and more severe among African Americans. Furthermore, FDA has requested that forthcoming studies for certain drugs include larger proportions of minority children. Studies of some drugs that may be used to treat diseases or conditions that disproportionately affect minorities have been completed and additional such studies have been requested by FDA. From January 4, 2002, through March 6, 2003, FDA granted additional exclusive marketing rights to four drugs that may be used to treat conditions such as hypertension, type II diabetes, and sickle cell anemia--conditions or diseases that disproportionately affect minority children. During that time, FDA also issued written requests for studies of six drugs for these conditions. FDA does not have a system in place to serve as a single source of data to allow the agency to efficiently determine the extent of participation of children by racial and ethnic group under the pediatric exclusivity provision. GAO found that some study reports submitted to FDA from drug sponsors did not specify the race and ethnicity of all study participants. Across all the studies for drugs granted additional exclusive marketing rights that GAO reviewed, 86 percent of study participants were identifiable by race or ethnicity, but the race or ethnicity of 14 percent of study participants was unknown. In January 2003, FDA issued draft guidance recommending that drug sponsors use standard definitions for race and ethnicity in drug studies. However, drug sponsors are not required to use these definitions. FDA has also begun to develop an agency-wide system to monitor demographic characteristics of study participants, such as age, sex, and race. FDA agreed with the GAO recommendation to specify the categories that sponsors should use to report minority representation as well as GAO's findings regarding the efficiency of its data collection systems. FDA expressed concerns about the GAO comparison of the proportion of minorities in drug studies to their proportion in the U.S. population. However, FDA had previously used the methodology GAO employed in its analyses of adult study participants.