Drug Sponsors Agreed to Study the Majority of On-Patent Drugs with Written Requests under BPCA, but No Studies Were Conducted When Drug Sponsors Declined the Written Requests

Most of the on-patent drugs for which FDA requested pediatric drug  studies under BPCA were being studied, but no studies resulted when the  requests were declined by drug sponsors. Drug Sponsors Agreed to Conduct Pediatric Drug Studies for Most On-Patent Drugs with Written Requests Issued under BPCA

From January 2002 through December 2005, FDA issued 214 written  requests for on-patent drugs to be studied under BPCA, and drug sponsors  agreed to conduct pediatric drug studies for 173 (81 percent) of those. FNIH had not funded the study of any  of these drugs. Most Drugs Granted Pediatric Exclusivity under BPCA Had Labeling Changes, but the Process for Making Changes Was Sometimes Lengthy

Most drugs—about 87 percent—that have been granted pediatric  exclusivity under BPCA have had labeling changes as a result of the  pediatric drug studies conducted under BPCA. Pediatric drug studies  conducted under BPCA showed that children may have been exposed to  ineffective drugs, ineffective dosing, overdosing, or side effects that were  previously unknown. For the  remaining 18 drugs (about 40 percent), it took from 238 to 1,055 days for  FDA to complete the scientific review process and approve labeling  changes. For 7 of those drugs, it took more than a year to complete the  scientific review process and approve labeling changes. We found that the drugs studied  under BPCA represented more than 17 broad categories of disease. Table 4  provides information on some of the drugs studied for pediatric use and  what is known about the diseases that are relevant to children. HHS stated that the draft report provided a  significant amount of data and analysis and generally explains the BPCA  process. First, HHS commented  that the report does not sufficiently acknowledge the success of BPCA. Nevertheless, the draft  report extensively discussed HHS accomplishments such as the number of  studies conducted, the number and importance of labeling changes that  FDA approved, and the wide range of diseases, including some that are  common, serious, or life threatening to children, for which drugs were  studied. We are sending copies of this report to the Secretary of Health and Human  Services, appropriate congressional committees, and other interested  parties. Appendix I: Scope and Methodology

In this report, we (1) assessed the extent to which pediatric drug studies  were being conducted for on-patent drugs under the Best Pharmaceuticals  for Children Act (BPCA), including when drug sponsors declined to  conduct the studies; (2) evaluated the impact of BPCA on labeling of drugs  for pediatric use and the process by which the labeling was changed; and  (3) illustrated the range of diseases treated by the drugs studied under  BPCA. We reviewed data provided to  us by the Foundation for the National Institutes of Health (FNIH)—a  nonprofit corporation independent of NIH—about funding for pediatric  drug studies of on-patent drugs.
Why GAO Did This Study

About two-thirds of drugs that are prescribed for children have not been studied and labeled for pediatric use, which places children at risk of being exposed to ineffective treatment or incorrect dosing. The Best Pharmaceuticals for Children Act (BPCA), enacted in 2002, encourages the manufacturers, or sponsors, of drugs that still have marketing exclusivity--that is, are on-patent--to conduct pediatric drug studies, as requested by the Food and Drug Administration (FDA). If they do so, FDA may extend for 6 months the period during which no equivalent generic drugs can be marketed. This is referred to as pediatric exclusivity. BPCA required that GAO assess the effect of BPCA on pediatric drug studies and labeling. As discussed with the committees of jurisdiction, GAO (1) assessed the extent to which pediatric drug studies were being conducted under BPCA for on-patent drugs, including when drug sponsors declined to conduct the studies; (2) evaluated the impact of BPCA on labeling drugs for pediatric use and the process by which the labeling was changed; and (3) illustrated the range of diseases treated by the drugs studied under BPCA. GAO examined data about the drugs for which FDA requested studies under BPCA from 2002 through 2005. GAO also interviewed officials from relevant federal agencies, pharmaceutical industry representatives, and health advocates.

What GAO Found

Drug sponsors have initiated pediatric drug studies for most of the on-patent drugs for which FDA has requested studies, but no drugs were being studied when drug sponsors declined these requests. Sponsors agreed to 173 of the 214 written requests for pediatric studies of on-patent drugs. In cases where drug sponsors decline to study the drugs, BPCA provides for FDA to refer the study of these drugs to the Foundation for the National Institutes of Health (FNIH), a nonprofit corporation. FNIH had not funded studies for any of the nine drugs that FDA referred as of December 2005. Most drugs (about 87 percent) granted pediatric exclusivity under BPCA had labeling changes--often because the pediatric drug studies found that children may have been exposed to ineffective drugs, ineffective dosing, overdosing, or previously unknown side effects. However the process for approving labeling changes was often lengthy. It took from 238 to 1,055 days for information to be reviewed and labeling changes to be approved for 18 drugs (about 40 percent), and 7 of those took more than 1 year. Drugs were studied under BPCA for the treatment of a wide range of diseases, including those that are common, serious, or life threatening to children. These drugs represented more than 17 broad categories of disease, such as cancer. The Department of Health and Human Services stated that the report provides a significant amount of data and analysis and generally explains the BPCA process, but expressed concern that it did not sufficiently acknowledge the success of BPCA or clearly describe some elements of FDA's process. GAO incorporated comments as appropriate.