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Institute of Medicine (US) Roundtable on Research and Development of Drugs, Biologics, and Medical Devices; Yaffe S, editor. Rational Therapeutics for Infants and Children: Workshop Summary. Washington (DC): National Academies Press (US); 2000.

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Rational Therapeutics for Infants and Children: Workshop Summary.

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Preface

The Institute of Medicine's (IOM's) Roundtable on Research and Development of Drugs, Biologics, and Medical Devices evolved from the Forum on Drug Development, which was established in 1986. Sponsor representatives and IOM determined the importance of maintaining a neutral setting for discussions regarding long-term and politically sensitive issues justified the need to revise and enhance past efforts. The new Roundtable is intended to be a mechanism by which a broad group of experts from the public* and private sectors can be convened to conduct a dialogue and exchange information related to the development of drugs, biologics, and medical devices. Members have expertise in clinical medicine, pediatrics, clinical pharmacology, health policy, health insurance, industrial management, and product development; and they represent interests that address all facets of public policy issues.

From time to time, the Roundtable requests that a workshop be conducted for the purpose of exploring a specific topic in detail and obtaining the views of additional experts. Summaries of these workshops are prepared and disseminated to make the discussion readily accessible to the members of the Roundtable and other interested parties. Such workshop summaries are intended only to convey the presentations and discussion that took place at the workshop; they do not include any conclusions or recommendations on the part of the Roundtable or any further data or analysis of the issues.

The first workshop for the Roundtable was held on April 14 and 15, 1998, and was entitled Assuring Data Quality and Validity in Clinical Trials for Regulatory Decision Making. The summary on that workshop is available from IOM.

This workshop summary covers the second workshop, which was held on May 24 and 25, 1999, and which was aimed at facilitating the development and proper use of drugs, biologics, and medical devices for infants and children. It explores the scientific underpinnings and clinical needs, as well as the regulatory, legal, and ethical issues, raised by this area of research and development.

The environment for pediatric therapeutics has been altered by two important developments. First, the Food and Drug Administration Modernization Act, enacted in 1997, provides incentives to pharmaceutical manufacturers that conduct acceptable studies with children and drugs identified by the U.S. Food and Drug Administration, to be ones for which information on the effects on the pediatric population would be beneficial. Second, in December 1998 the U.S. Food and Drug Administration published a final role that established certain requirements for studies with the pediatric population and drugs that significantly affect infants and children.

This workshop addressed many important issues. For example, many questions about the biochemistry of the enzymes involved in drug metabolism remain unanswered, in particular, the variability in the responses of individuals to drugs. The differences in the pharmacodynamics and pharmacogenetics of drug metabolism between children and adults remain undetermined. In addition, the influence of diet on the process of induction of these enzymes during the various stages of development deserves extensive study. Three sets of concerns need to be addressed to advance the development of rational therapeutics for infants and children:

1.

What is the scientific basis for differences in the responses to drugs of humans during their life spans, from the time of development in fetuses, infants, and young children through the process of aging?

2.

How do the metabolic, immune, and other systems of infants and children function differently at various stages of growth, what are the factors that influence these differences, and do they alter responses to drugs?

3.

What data need to be known to determine pediatric drug dosages? Can dosages be extrapolated from data for adults? How can responses to drugs be evaluated ethically in children?

This summary on the workshop is organized as topic-by-topic descriptions of the presentations and discussions that occurred during the workshop. Although the proceedings were rich and wide-ranging, this workshop summary does not purport to be a comprehensive or exhaustive exploration of the issues discussed. Nor does it represent a consensus of views or opinions from IOM or the Roundtable. Rather, it summarizes a dialogue among representatives from different sectors and their thoughts on what research and what public policy issues may merit further attention. The editor's summary material at the beginning of each section provides some context and overview of the identified presentations.

On behalf of the members of the Roundtable, I express my warmest appreciation to the authors of this workshop summary for providing a timely and useful summary of the issues raised and the discussions that occurred. I also want to thank the individuals and organizations that gave valuable time to provide information and insight to the Roundtable through participation in the workshop. Each of the following contributed greatly: Charles H. Ballow, Kaleida Health Millard Fillmore Hospital; Mark Batshaw, George Washington University Medical Center, Children's Research Institute, Children's National Medical Center; Emmett Clemente, Ascent Pediatrics, Inc.; Thierry Cresteil, Centre National de la Recherche Scientifique, Institut Gustave Roussy, Villejuif, France; Sherin U. Devaskar, Magee Women's Hospital, University of Pittsburgh School of Medicine; Susan Ellenberg, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration; David Feigal, Jr., Center for Devices and Radiological Health, U.S. Food and Drug Administration; Jane Henney, U.S. Food and Drug Administration; Ralph Kauffman, Children's Mercy Hospital; Greg Kearns, Division of Pediatric Clinical Pharmacology, Children's Mercy Hospital; Michael Labson, Covington & Burling; Steve Leeder, Children's Mercy Hospital; Murray Lumpkin, Center for Drug Evaluation and Research, U.S. Food and Drug Administration; Michael MeGarvey, Blue Cross and Blue Shield of New Jersey, Inc.; Dianne Murphy, Center for Drug Evaluation and Research, U.S. Food and Drug Administration; Robert M. (Skip) Nelson, Medical College of Wisconsin; David Poplack, Baylor College of Medicine and Texas Children's Cancer Center; Stephen Spielberg, R.W. Johnson Pharmaceutical Research Institute; Stanley J. Szefler, National Jewish Medical and Research Center, University of Colorado Health Sciences Center; Robert Temple, Center for Drug Evaluation and Research, U.S. Food and Drug Administration; John Watchko, Department of Pediatrics, Magee Women's Hospital, University of Pittsburgh School of Medicine; Karen Weiss, Center for Biologics Evaluation and Research, U.S. Food and Drug Administration; and John T. Wilson, Department of Pediatrics, Louisiana State University Medical Center.

I also thank the following IOM staff for their valuable contributions in this activity: Vivian Nolan, Nicole Amado, Alden Chang, and Thelma Cox. Consultant and technical writer Kathi Hanna contributed significantly to the writing of many sections of the workshop summary. The extensive commentary and suggestions made by the copy editor, Michael Hayes, are gratefully acknowledged.

Finally, the Roundtable and IOM also wish to thank the sponsors that supported this activity. Financial support for this project was provided by the American Medical Association, Baxter International Inc., Eli Lilly, U.S. Food and Drug Administration (Center for Biologics Evaluation and Research, Center for Devices and Radiological Health, and Center for Ding Evaluation and Research), the Health Industry Manufacturers Association, Hoffmann-La Roche, Merck & Co., Inc., National Institutes of Health (Office of Rare Diseases and National Institute of Child Health and Human Development), Pfizer, and Wyeth-Ayerst.

SUMNER YAFFE, M.D.

EDITOR

Representatives of federal agencies serve in an ex officio capacity. An ex officio member of a group is one who is a member automatically by virtue of holding a particular office or membership in another body.

Footnotes

*

Representatives of federal agencies serve in an ex officio capacity. An ex officio member of a group is one who is a member automatically by virtue of holding a particular office or membership in another body.

Copyright 2000 by the National Academy of Sciences. All rights reserved.
Bookshelf ID: NBK225508
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