December 14, 1998: Ralph E. Kauffman, Marion Merrell Dow Missouri
Response to AHRQ on CERTs
This is in response to the October 26, 1998 announcement from AHCPR inviting nominations of topics for study and ideas for implementation of a new program, Centers for Education and Research on Therapeutics, described in section 409 of the Food and Drug Administration Modernization Act of 1997 (FDAMA).
We believe a significant focus of this program must target children, women, and pregnancy as special and neglected populations. For example, critical areas of research affecting therapeutics in children include:
- Monitoring of adverse outcomes in children due to under- and over-dosing because of lack of age-appropriate labeling and age-appropriate formulations.
- Assessment of post-marketing experience with off-label use of drugs in children.
- Phase IV post-marketing surveillance of drugs in children to longitudinally evaluate post-marketing use and adverse events.
- A registry to identify significant adverse events in children related to extensive off-label use.
- Critical assessment of unmet therapeutic needs of children leading to development of a priority list of drugs to be labeled for children.
- Research on the pharmacoeconomic and pharmacoepidemiologic impact of lack of drug labeling for children.
Additional areas that need to be addressed include:
- Program projects to develop lay education efforts regarding appropriate use of medications which would focus on patient advocacy networks.
- Funding to educate physicians, both during residency/fellowship and subsequent practice, regarding efficacious and safe use of medications.
With respect to existing frameworks for responding to this mandate, there already is in existence the Pediatric Pharmacology Research Unit (PPRU) Network funded by the National Institute of Child Health and Human Development (NICHD). For the past 5 years this network has consisted of seven academic pediatric medical centers linked through NICHD to collaboratively perform therapeutics and pharmacology research in infants and children. This network has been inordinately successful in its mission and will be expanded January 1, 1999 to 13 pediatric centers. The PPRU network provides a funded ready-made platform within which the pediatric component of the CERT program would very efficiently be based. Within the PPRU network there already is a critical mass of pharmacology and therapeutics expertise, a large patient population, ethnic diversity, geographic diversity, a central operations center, biostatistics and data analysis capability, and extensive experience in conducting therapeutics research. Furthermore, the Food and Drug Administration (FDA) and pharmaceutical industry are very familiar with the PPRU. Other networks that also might provide existing platforms are the NICHD Neonatal Network, the NICHD Perinatal Network, and the National Institute of Mental Health (NIMH) Network of Research Units for Pediatric Psychopharmacology.
In summary, health care policy historically has excluded children from development of new therapeutic agents. This unfortunate de facto policy has had a major negative impact on health care for children for more than 30 years. We have just entered an era where this is rapidly changing due to recent legislative and regulatory initiatives, including FDAMA. These recent initiatives provide an extraordinary window of opportunity to address the therapeutic needs of children. The Centers for Education and Research on Therapeutics program offers an additional opportunity to help fund post-marketing research to complement the mechanisms currently in place for pre-clinical pediatric drug research and have a tremendous impact on health care of infants and children.
Ralph E. Kauffman, M.D.
Marion Merrell Dow/Missouri Chair of Medical Research
Professor, Pediatrics and Pharmacology
Director, Medical Research
Gregory Kearns, Pharm.D., FCP
Marion Merrell Dow/Missouri Professor of Pediatric Pharmacology
Professor of Pediatrics and Pharmacology, UMKC
Chief, Section of Pediatric Clinical Pharmacology & Experimental Therapeutics
William H. Caskey, Ph.D.
Director, Research and Grants Administration