FDA: FIRMS SHOULD SUPPLEMENT PEDIATRIC CLINICAL TRIALS WITH JUVENILE ANIMAL STUDIES

The FDA recommends that drug companies supplement their pediatric clinical trials with juvenile animal studies to better determine a drug's effect on children, according to a new guidance that notes such studies may influence post-marketing, labeling and use restriction decisions.

The FDA is worried about serious, irreversible adverse effects that "cannot be adequately, ethically, and safely assessed" in pediatric clinical trials, said the guidance, which was published in the Federal Register.

Pediatric clinical trials often are insufficient to determine a drug's effect on children, the guidance says. Delayed or irreversible adverse effects may be better identified in animal studies than in clinical trials, which may have been of insufficient duration to demonstrate those effects, the agency explains.

Differences between mature and immature systems make assessments of drug toxicity difficult, the agency adds. Those differences include developmental changes in metabolism, body composition, receptor expression and function, growth rate, and organ functional capacity.

To address such issues, drug sponsors should communicate with the agency to determine whether juvenile animal studies are needed, the guidance says. The agency may then use these studies to make product approval decisions. For instance, sponsors may be required to conduct long-term, follow-up human safety studies as a postmarket commitment, the guidance says.

The guidance, "Nonclinical Safety Evaluation of Pediatric Drug Products," is available at http://www.fda.gov/OHRMS/DOCKETS/98fr/03d-0001-gdl002.pdf (http://www.fda.gov/OHRMS/DOCKETS/98fr/03d-0001-gdl002.pdf).