Presented by Dianne Murphy, M.D.

Associate Director for Pediatrics, Center for Drug Evaluation and Research, U.S. Food and Drug Administration

In 1994, FDA provided a new regulatory approach to encourage studies with pediatric populations to develop information on the proper use of a product being prescribed for the pediatric population. With that regulatory approach, where appropriate, FDA could conclude that the course of the disease and the effects of a drug, both beneficial and adverse, were sufficiently similar in the pediatric and adult populations to permit extrapolation of efficacy in adults to efficacy in children. In such a situation, there would be no need to again prove efficacy in the pediatric population. Unfortunately, the rule did not achieve its intended objectives. Seventy-seven percent of the labeling changes proposed by drug companies in response to this rule did not result in improved labeling for children. To improve on that record, two additional legislative and regulatory tools are now available: (1) Section III of the 1997 Food and Drug Administration Modernization Act (FDAMA; PL 105–115), which provides 6 months of additional marketing exclusivity, and (2) the 1998 Pediatric Rule which requires studies in the pediatric population unless such studies are waived or deferred.

Under FDAMA, which is voluntary, the studies to be submitted by the sponsor must be responsive to a written request issued by FDA. The way in which this actually works is that the sponsor first submits a proposal of what studies it thinks will be necessary. FDA's written request in response to that proposal is based on its assessment of what studies are needed to produce a health benefit in the pediatric population. Having a sponsor submit a proposal first allows FDA to determine the level of interest in and commitment to pursuing studies for a particular moiety. Because the additional exclusivity will attach to the ''active moiety," the sponsor may need to provide the results of studies for all products with that moiety if those products are being used in pediatrics but are not appropriately labeled for such use.

As of May 1999, of the 15 technical divisions in the FDA's Center for Drug Evaluation and Research, all but two had received proposals. As of May 23, 1999, there were 133 proposals and FDA had issued 83 written requests asking for 155 studies. Fifty-five of these studies were efficacy and safety studies, 55 were pharmacokinetic studies, and the remaining were pharmacokinetic or pharmacodynamic, safety only, or "other" types of studies. "Other" includes prophylaxis studies and studies of the actual use of over-the-counter products. During this period 14 studies were proposed for the neonatal population and 24 studies were proposed for the population less than 2 years old. Physiologic and functional childhood changes and development often occur across the arbitrary

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