Docket Management
Docket: 02N-0152 - Timely Pediatric Studies and Adequate Pediatric Labeling for Human Drugs and Biologics
Comment Number: EC -70

Accepted - Volume 3

Comment Record
Commentor Mrs. Ellen Vasser Date/Time 2002-07-08 11:37:23
Organization Children's Hospital of The King's Daughters
Category Health Professional

Comments for FDA General
Questions
1. What changes to the pediatric rule, if any, would be necessary to integrate the BPCA and the pediatric rule more effectively? The Pediatric Rule ensures that children and adolescents are no longer a therapeutic afterthought by the pharmaceutical industry. It is an essential and successful tool in ensuring that children and adolescents have the quality and quantity of drugs they need. All new drugs must be studied for pediatric use at the time a drug comes to market unless the FDA grants a waiver. This makes medications for children and adolescents a certainty, not an option and puts children and adolescents on a level playing field with adults for the first time. I believe 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 and adolescents to ensure that appropriate drugs are available for their use. -Retiring or relaxing any authorities currently in the Pediatric Rule is inappropriate and would be to the detriment of children and adolescents. It must always be kept in mind that 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? Noting again that the BPCA is subject to continuation by Congress and that future reauthorization is uncertain, 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; and if the current label lacks appropriate pediatric use information, 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. 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.
3. What provisions, if any, of the BPCA could apply to biological products regulated under section 351 of the PHSA? It is essential that the Pediatric Rule remain in place because it is the only mechanism that ensures that biological products will be studied and available for children and adolescents. 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. 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. This provision was included to acknowledge that once a formulation is developed in the study phase, while it may be necessary to manufacture that formulation, it may not always be possible to scale up the formulation for distribution to the general public.
5. General Comments Thank you for your consideration of these comments.




EC -70