Docket Management
Docket: 02N-0152 - Timely Pediatric Studies and Adequate Pediatric Labeling for Human Drugs and Biologics
Comment Number: EC -69

Accepted - Volume 3

Comment Record
Commentor Dr. Tracy Hunley Date/Time 2002-07-08 09:06:14
Organization Vanderbilt University Medical Center
Category Academic

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? Relaxing or retiring ANY of the authorities currently in the Pediatric Rule is inappropriate and would BE TO THE DETRIMENT OF CHILDREN. PBCA is time limited, voluntary, and subject to continuation by Congress. The Pediatric Rule MUST remain in place IN ITS ENTIRETY!!
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? The 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; if the current label lacks appropriate pediatric use infromation, 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 Pediatric Rule is the ONLY mechanism that ensures that biological products will be studied and avaailable for children. NO PROVISION of BPCA applies specifically to biological products since the legislation focuses on drugs covered by Food, Drug, and Cosmetic Act and the vast majority of biologics are covered under the Public Health Service Act. Furthermore the most innovative new therapies are biologic products. Loss of the Pediatric Rule would again relegate children to second class status with regard to the pharmaceutical industry.
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? Appropriate formulations are an essential component of providing medicine to children. IT IS REQUISITE for studies in infants and younger children to develop age appropriate formulations, if necessary. Failure to require needed formulations for specific age populations NEGATES the intent of both the BPCA and the Pediatric Rule. Further, the BPCA is further weakened by its recommendation for formulation changes only to studies completed under public contract--allowing an out for companies not wanting to scale up the formulation for distribution to the general public.
5. General Comments The Pediatric Rule should remain in place in its entirety because -----It ensures that children are NOT a therapeutic afterthought of the pharmaceutical industry! Children get serious, complicated diseases in need of the most advanced medications available. Loss of the Pediatric Rule will once again force pediatricians and pediatric subspecialists to use medications off label, without thorough testing in children, and increasingly, allow insurance companies to deny payment for experimental therapies--experimental because testing was not mandated of the pharmaceutical industry. -----Only the Pediatric Rule includes biological products, amonth the most innovative new medications. -----The Pediatric Rule captures drugs and age populations that the BPCA cannot. -----BPCA sunsets in 2007. The pharmaceutical industry will not keep up testing in children without the Pediatric Rule being kept in place IN ITS ENTIRETY. -----The Pediatric Rule is mandatory, i.e testing in children is mandated. This alone indicates that the health of children truly matters. The BPCA is voluntary relegating child health to the purview of those who may or may not want to do testing, but often intend the drug or biologic product for pediatric use. Only the Pediatric Rule ensures the necessary pediatric testing.

EC -69