Drug Information Journal - November 2010 - (Page 757)
Julia Dunne, MA, MD Office of Pediatric Therapeutics, Food and Drug Administration, Silver Spring, Maryland Lala Margaryants, PharmD Scientific Centre of Drug and Medical Technology Expertise, Yerevan, Republic of Armenia M. Dianne Murphy, MD Office of Pediatric Therapeutics, Food and Drug Administration, Silver Spring, Maryland Ann M. Myers, RPh, MPH Office of Pediatric Therapeutics, Food and Drug Administration, Silver Spring, Maryland Debbie Avant, RPh Office of Pediatric Therapeutics, Food and Drug Administration, Silver Spring, Maryland William J. Rodriguez, MD, PhD Office of Pediatric Therapeutics, Food and Drug Administration, Silver Spring, Maryland
Globalization Facilitates Pediatric Drug Development in the 21st Century
Introduction: US legislation, supported by strengthened ethical frameworks and improved trial design, has produced significant increases in the number of pediatric clinical trials. This has global implications. Method: We reviewed all submissions of pediatric data received by the US FDA from 2002 to 2007 in response to new FDA pediatric initiatives. Results: Although 54% of the trials were multinational, the US dominated as a trial location. The European Union and Latin America followed. Few trials specifically studied neonates, infants, and toddlers. Conclusion: Although most pediatric drug programs are global, the United States remains the dominant location for pediatric trials. This distribution differs for adult trials. The balance may change in the future. EU and FDA regulators should continue to discuss coordinated approaches to minimize unnecessary pediatric trials and to optimize trial design, safety, and conduct so that the limited pediatric populations available are enrolled only in ethically implemented, scientifically important trials.
A historical reluctance to study medicines in children has necessitated decades of off-label use for most pediatric prescribing (1–3). Ethical concerns and the difficulties of conducting trials in children fueled the reluctance, which was exacerbated by the lack of sufficient commercial returns for the pharmaceutical industry (4). This unacceptable situation, however, is changing. More robust ethical, regulatory, and legal frameworks exist to ensure the protection of children, who can neither volunteer nor give informed consent to take part in trials (5–7). Furthermore, it is agreed that children are not small adults (8) and pediatric trials are needed to establish the correct dose, efficacy, and safety of a medicine in that population (9,10). Increasing involvement of pediatric expertise has improved the design and conduct of these trials. Finally, important changes in US legislation both provided financial incentives and imposed requirements on the pharmaceutical industry to study medicines in children (1 1,12). This has produced a significant increase in the number of pediatric trials con-
Key Words Globalization; Pediatric clinical trial; FDA Correspondence Address Julia Dunne, Food and Drug Administration, 10903 New Hampshire Ave, Building 32, Room 5154, Silver Spring, MD 20993 (email: firstname.lastname@example.org). The views presented in this article do not necessarily reflect those of the Food and Drug Administration.
ducted since 1997 (10). The European Union pediatric medicines regulations, which were adopted in 2007, are also based on a framework of incentives and requirements and will lead to a further stimulation of pediatric drug development (13). We wished to explore the location and other characteristics of recently conducted pediatric trials in view of the relatively small and geographically scattered pediatric population that is available to participate in trials and in view of the increasing globalization of drug development. In particular we wished to explore whether, as with adult clinical trials, sponsors are shifting the location of pediatric trials away from the United States (14,15). We undertook a descriptive study of dossiers submitted by the pharmaceutical industry in response to FDA’s issuance of written requests for pediatric data on products that were already authorized for use in adults. If performed as requested, responses to FDA written requests can provide additional market exclusivity for the sponsor. We analyzed the information from a 5-year period according to trial location and, where available, patient numbers. We also examined the age ranges studied, loSubmitted for publication: March 11, 2010 Accepted for publication: July 15, 2010
Drug Information Journal, Vol. 44, pp. 757–765, 2010 • 0092-8615/2010 Printed in the USA. All rights reserved. Copyright © 2010 Drug Information Association, Inc.
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