were found to need a smaller dose (approximately half the values seen in the male patients) (Roberts et al., 2003).1

Despite increases in the testing of drugs in pediatric populations, a pressing need for more study remains. Although incentives exist to study new, on-patent drugs, some argue that additional incentives are needed, especially to encourage testing of older drugs that are off-patent. The two existing laws that address the need to study drugs in pediatric populations—the Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA)—will sunset in October 2007 without congressional action.2

ORGANIZATION OF THIS SUMMARY

The following chapters summarize the presentations and discussions at the workshop. Chapter 2 reviews the regulatory framework for pediatric drug development and testing, summarizing BPCA and PREA and their impact. Chapter 3 addresses challenges to the development of drugs for children, including the barriers posed by ethical concerns, economic obstacles, and logistical and technical issues; the difficulty of devising appropriate formulations; and issues of dosing, bioavailability, and drug response. Chapter 4 considers the potential adaptation of existing models—such as vaccine development, European regulatory models, and the approach used by St. Jude Children’s Research Hospital to develop oncology drugs—to enhance pediatric drug development. The final chapter summarizes participants’ suggestions for solutions and next steps.

1

Fluvoxamine dosing was not based on body-weight. After the starting dose of 25 mg, the fluvoxamine dosing was titrated according to clinical response and tolerance, and the resulting fluvoxamine dose was in a range of 50–200 mg/day (on a bid schedule) in a 10-week study. In a pharmacokinetic study, consistent with clinical observations, fluvoxamine exposure (AUC and Cmax at steady state) was significantly higher in younger female patients compared to those in the corresponding age group of male patients. Hence, the label says that therapeutic effects in female children may be achieved with lower doses.

2

Subsequent to this workshop, both BPCA and PREA were reauthorized by Congress as part of the Food and Drug Administration Amendments Act of 2007, Public Law 110-85, which was signed by the President in September 2007.



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