The Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) were designed to encourage more pediatric studies of drugs used for children. The FDA asked the IOM to review aspects of pediatric studies and changes in product labeling that resulted from BPCA and PREA and their predecessor policies, as well as assess the incentives for pediatric studies of biologics and the extent to which biologics have been studied in children. The IOM committee concludes that these policies have helped provide clinicians who care for children with better information about the efficacy, safety, and appropriate prescribing of drugs. The IOM suggests that more can be done to increase knowledge about drugs used by children and thereby improve the clinical care, health, and well-being of the nation's children.
1 Front Matter; 2 Summary; 3 1 Introduction; 4 2 Children's Growth and Development and Pediatric Drug Studies; 5 3 Policy Framework for BPCA and PREA; 6 4 Ethical Issues in Pediatric Drug Studies; 7 5 Safety and Efficacy Assessments in Studies Conducted Under BPCA and PREA; 8 6 BPCA, PREA, and Drug Studies with Neonates; 9 7 Outcomes of Written Requests, Requirements, Studies, and Labeling Changes; 10 8 Pediatric Studies of Biologics; 11 References; 12 Appendix A: Study Activities, Methods, and Public Meetings; 13 Appendix B: Dissemination of Information from Pediatric Studies Conducted Under BPCA and PREA; 14 Appendix C: Biologics in Pediatrics; 15 Appendix D: Biologics Studied and Not Studied in Children; 16 Appendix E: Written Requests for Studies of Pediatric Hypertension: Longitudinal Changes in FDA Specifications; 17 Appendix F: Committee and Staff Biographies; 18 Index
National Research Council, Division of Behavioral and Social Sciences and Education, Institute of Medicine, and Families Board on Children, Youth, Steve Olson
Institute of Medicine, Board on the Health of Select Populations, and Transgender Health Issues and Research Gaps and Opportunities Committee on Lesbian, Gay, Bisexual
Institute of Medicine, Board on Population Health and Public Health Practice, Division of Health Promotion and Disease Prevention, Division of International Health
Institute of Medicine, Committee to Develop Methods Useful to the Department of Veteran Affairs in Estimating Its Physician Requirements, Joseph Lipscomb
Institute of Medicine, Board on Health Sciences Policy, Committee on Accelerating Rare Diseases Research and Orphan Product Development, Thomas F. Boat, Marilyn J. Field
and Medicine National Academies of Sciences, Engineering, Institute of Medicine, and Families Board on Children, Youth, Board on the Health of Select Populations, Committee to Evaluate the Supplemental Security Income Disability Program for Children with Mental Disorders, Joel T. Wu, Thomas F. Boat
Institute of Medicine, Board on Health Sciences Policy, and Practice Committee on Conflict of Interest in Medical Research, Education, Marilyn J. Field, Bernard Lo
Institute of Medicine, Board on Health Sciences Policy, Committee on Postmarket Surveillance of Pediatric Medical Devices, Hugh Tilson, Marilyn J. Field, Marilyn J Field
Institute of Medicine, Board on Neuroscience and Behavioral Health, Committee on Incorporating Research into Psychiatry Residency Training, Thomas F. Boat, Kathleen M. Patchan, Michael T. Abrams
Institute of Medicine, Board on Health Sciences Policy, Committee on Palliative and End-of-Life Care for Children and Their Families, Richard E. Behrman, Marilyn J. Field
Institute of Medicine, Board on Health Sciences Policy, Committee on Clinical Research Involving Children, Richard E. Behrman, Marilyn J. Field, Richard E Behrman, Marilyn J Field