The FDA's pediatric rule

Athough the proposed FDA rule on pediatric clinical trials raises a host of political and regulatory questions, biotech companies already have begun to wrestle with the practical considerations of childhood studies.

The new rule proposed last week would require companies to assess the safety and effectiveness of drugs and biologics in pediatric patients where they were previously focused on adults. The regulation is intended to ensure that new pharmaceuticals that are therapeutically important and/or are likely to be used in children have adequate pediatric labeling at the time of, or soon after, approval (see BioCentury Extra Aug. 14).

The questions facing industry are what compounds will be subject to the requirements, what will happen to compounds in the clinic and nearing FDA evaluation or already on the market, and what it will mean for development budgets and timelines to conduct unexpectedly necessary clinical trials in pediatric patients.

The answers may have to wait for the final rule, and for subsequent discussions between individual companies and the FDA. The industry has 90 days to comment on the proposed rule, and a public hearing also is planned.

In the meantime, however, some biotech companies already have a wealth of experience in pediatric trials, which help to identify the practical issues involved in conducting studies in children.

Clinical issues

The American Academy of Pediatrics guideline for the ethical conduct of studies to evaluate drugs in pediatric populations states "studies to be carried out in children must be scrutinized for all potential risks, including those that are not usually of concern when considering studies in adults. These include discomfort, inconvenience, pain, fright, separation from parents or familiar surroundings, effects on growth or development of organs, and size or volume of biologic samples."