Docket Management
Docket: 02N-0152 - Timely Pediatric Studies and Adequate Pediatric Labeling for Human Drugs and Biologics
Comment Number: EC -64

Accepted - Volume 3

Comment Record
Commentor Dr. Jeffrey Leiser Date/Time 2002-07-07 09:49:07
Organization Riley Hospital for Children
Category Health Professional

Comments for FDA General
5. General Comments July 7, 2002 To Whom it May Concern: I am writing to express my dismay with and opposition to suggestions that portions of the 1998 Pediatric Rule be relaxed or abandoned. I am a pediatric nephrologist, and care for children from infancy through young adulthood with kidney-related diseases. Over the past decade new medications have allowed my colleagues and me to more effectively treat a wide variety of disorders. But the picture is a mixed one. Almost none of these medications have been evaluated for safety and efficacy in children in a manner equivalent to what was required prior to their approval for use in adults. The consequences of this are substantial. These medications are important, but there is neither infrastructure nor financial resources to study them in a properly organized manner. Introduction into clinical use in children is haphazard, and sometimes hazardous. For example, angiotensin converting enzyme inhibitors are presently the most generally useful group of medications for treating elevated blood pressure and for slowing the progression of kidney failure. However, when the first angiotensin converting enzyme inhibitor, captopril, was beginning to be tried in neonates, there resulted several case reports of strokes due to excessive lowering of blood pressure. We now know that neonates are more sensitive to these medications. I suspect that, if captopril had been studied in neonates as part of the more rigorously structured pre-marketing approval process, such disasters might have been avoided. In addition, most of these medications are not manufactured in formulations appropriate for young children. My patients are dependent on “extemporaneous” preparations, i.e. preparations not provided by the manufacturer but instead compounded by individual pharmacists. Here again, though, the process of demonstrating drug stability is haphazard, and there is the increased risk of lack of standardization and compounding errors. The voluntary incentives for studies of medication safety and dosing in children by pharmaceutical companies (the Best Pharmaceuticals for Children Act) are helpful. However, without the Pediatric Rule, children will continue to be denied full access to the range of important new medications and new biological products becoming available to treat human disease. Children should not remain second-class citizens in the pharmaceutical arena. The availability to them of the full range of new products being developed for the treatment of human diseases should be a certainty, not an option. The Pediatric Rule should remain in place in its entirety. It should apply to all new medications and biological products. For established medications, in the context of application for a supplemental indication, the Pediatric Rule should apply not only to that new indication, but also to current indications, and to other potential indications that would be of specific importance to children. And, for medications and biological products falling under the scope of the Pediatric Rule, pharmaceutical companies need to make available age-appropriate formulations, so that children have safe and reliable access in reality and not just in theory. I hope these comments will be helpful in your deliberations. Thank you for your consideration. Jeffrey Leiser, MD-PhD Department of Pediatrics Section of Pediatric Nephrology Riley Hospital for Children 699 West Drive, Room 213 Indianapolis, IN 46202

EC -64