Docket Management
Docket: 02N-0152 - Timely Pediatric Studies and Adequate Pediatric Labeling for Human Drugs and Biologics
Comment Number: EC -51

Accepted - Volume 1

Comment Record
Commentor Dr. Harris Stutman Date/Time 2002-07-03 13:16:21
Organization Eclipsys Corporation
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? Retiring or relaxing any authorities currently in the Pediatric Rule is inappropriate and would be to the detriment of children. BPCA is time-limited, voluntary and subject to continuation by the Congress. Those facts speak directly to the need to ensure that the Pediatric Rule remains 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 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 Pediatric Rule is the only mechanism that ensures that biological products will be studied and available for children. No provision of BPCA applies specifically to biological products since the legislation focuses on drugs covered by the Food, Drug and Cosmetic Act (FDCA) and the vast majority of biologics are covered under the Public Health Service Act. Moreover, some of the most innovative new therapies now and in the future are biological products, which are not covered under BPCA.
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 medications for the pediatric population. It is a 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 the BPCA and the Pediatric Rule. BPCA limits its reference to “recommendation” for formulation changes only to studies completed under public contract. It may not always be possible to scale up the formulation for distribution to the general public.
5. General Comments As a pediatrician and infectious disease specialist with long experience in pharmaceutical studies in children, I think I can provide informed comment on the relationship between the 1998 Pediatric Rule and the BPCA. The American Academy of Pediatrics (AAP) of which I am a longtime member has advocated for appropriately tested and labeled medications for infants, children and adolescents for over 40 years and asked me to support this position. I am delighted to do so, since as a medical researcher, the effects of previous testing policy has always been obvious to me. Securing safe and appropriate drugs for use by children has had a long and laborious history. Significant progress toward pediatric drug studies and labeling has been made over the last five years. The current dual approach is the best one to follow: 1) incentives for voluntary studies of drug safety and dosing by industry (extended in January 2002 in the BPCA); and 2) a regulation requiring pediatric studies, the Pediatric Rule. Earlier this year a proposal was made to suspend the Pediatric Rule. The FDA showed much foresight in promulgating the rule and it would be most inappropriate to do this and take a step backward in guaranteeing the ability to have medications intended (or potentially intended) for children not to be so evaluated. The Pediatric Rule is an essential approach to ensure that children have the quality and quantity of drugs they need, and are not treated as a superficial irritant on the more important adult studies (I am biased as a pediatrician, and a pediatric researcher, of course). All new drugs should be studied for pediatric use unless the FDA grants a waiver, for drugs with no conceivable pediatric use. This puts children on the same level as adults. I agree with the AAP that all components of the 1998 Pediatric Rule must be preserved. It is a comprehensive approach to securing pediatric studies. FDA has not yet invoked all the provisions of the Pediatric Rule; however, together they weave a safety net for children to ensure that children have appropriate drugs available for their use. Thank you for your consideration of these comments.

EC -51