4. using a representative sample of studies conducted since 1997 under PREA or precursor regulations, review and assess the number and type of pediatric adverse events;

5. review and assess the number and importance of biological products for children that are being tested as a result of the amendments made by the Biologics Price Competition and Innovation Act of 2009 [passed in 2010] and the importance for children, health care providers, parents, and others of labeling changes made as a result of such testing;

6. review and assess the number, importance, and prioritization of any biological products that are not being tested for pediatric use; and

7. offer recommendations for ensuring pediatric testing of biological products, including consideration of any incentives, such as those provided under section 505A of the Federal Food, Drug, and Cosmetic Act or section 351(m) of the Public Health Service Act.

The 13-member study committee appointed by the IOM met five times between December 2010 and October 2011. Three of these meetings included public sessions during which the committee heard from a range of interested parties, including government officials from FDA and the National Institutes of Health and individuals from organizations representing pharmaceutical and biotechnology companies, pediatricians, researchers, and advocates. The agendas for the public sessions follow this overview of study activities and methods.

The committee also sought assistance from consultants for the preparation of background papers and other analyses to supplement those undertaken by the committee. The consultants are listed after the committee members in the front of this report. The background papers prepared by consultants appear as Appendixes B and C, and Appendix D presents information on biologics studied in children, much of which was checked or compiled by a consultant.

The committee’s statement of task refers to written requests, studies, and labeling changes that have been occurred since 1997. However, the provisions of the FDA Modernization Act creating the written request mechanism and the pediatric exclusivity incentive did not go into effect until July 1, 1998, and the effective date of the 1998 Pediatric Rule was April 1, 1999. Therefore, the committee used these dates as the start dates for its sampling of FDA documents. Because FDA may not post relevant documents for some period after the approval of a product or labeling change, the committee chose December 31, 2010, to be the cutoff point for its sample.

FDA supplied the committee with its master list of labeling changes for the specified 1998 to 2010 time period. It also supplied a list of 14 products



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