Docket Management
Docket: 02N-0152 - Timely Pediatric Studies and Adequate Pediatric Labeling for Human Drugs and Biologics
Comment Number: EC -37

Accepted - Volume 1

Comment Record
Commentor Dr. Edward Bartlett Date/Time 2002-07-01 15:12:32
Organization Johns Hopkins Community Physicians
Category Health Professional

Comments for FDA General
1. What changes to the pediatric rule, if any, would be necessary to integrate the BPCA and the pediatric rule more effectively? In determining the process of when pediatric studies are conducted, the FDA should rely on the detailed process for requesting pediatric studies of already marketed drugs and securing labeling that is outlined in the BPCA.
2. How would the criteria used by NIH and FDA under section 3 of the BPCA to request studies of already approved drugs relate to the standards promulgated in the pediatric rule and described in 21 CFR 201.23, 314.55, and 601.27 for requiring pediatric labeling for certain drugs and biological products? Which criteria are more appropriate for determining when studies are conducted? Pediatric Rule should mirror the scope of the BPCA and apply to all labeled and potential indications as well as new indications. If a company submits a supplemental indication to the FDA, it invokes the Pediatric Rule. It is important that appropriate pediatric studies be conducted for that new use; and if the current label lacks appropriate pediatric use information (e.g., for neonates) the FDA should also include in their requirement for pediatric studies of the new indication, any pediatric studies that may be needed for the currently labeled or potential indications.
3. What provisions, if any, of the BPCA could apply to biological products regulated under section 351 of the PHSA? The BPCA provides incentives to the pharmaceutical industry to study drugs but does not address biological products. Significant portions of therapeutics used in children are biological products. Without the Pediatric Rule there is no mechanism to ensure that pediatric studies are conducted on these important medications.
4. How does the provision in section 3 of the BPCA providing for a recommendation for a formulation change relate to the pediatric rule provision stating that in certain cases a sponsor may be required to develop a pediatric formulation? Should pediatric formulations be required in certain cases? The BPCA incentive of additional market exclusivity can only be applied once during the life cycle of a drug. When FDA requests pediatric studies under BPCA, all potential pediatric uses must be anticipated in the request. This request cannot be expanded later if additional studies are needed in very young children or newborns or if a new use is discovered for a drug. Once studies have been completed and the incentive has been granted, there is no obligation on the part of participating companies to generate additional pediatric data. The Pediatric Rule may be invoked in instances where pediatric information is essential but the BPCA is no longer available.
5. General Comments The BPCA sunsets in 2007. If it is not renewed, history suggests that the industry may not see it within their scope or financial best interest to continue to do pediatric drug studies. The Pediatric Rule will allow pediatric studies to continue. Because BPCA is voluntary, not all sponsors are interested in complying with the terms. The Pediatric Rule applies to all drugs and biologicals whose intended use in pediatrics is the same as adults, thus ensuring appropriate pediatric information.

EC -37