Provision of Medical Information for Drugs Used in Orphan and Pediatric Disorders
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Drug Information Journal. Vol. 33. pp. 87-90. 1999 Printed in the USA. All rights reserved.
PROVISION OF MEDICAL INFORMATION FOR DRUGS USED IN ORPHAN AND PEDIATRIC DISORDERS DAVIDA. GRAVES,PHARMD Associate Director, Medical Information. Genentech, Inc., South San Francisco, California
Since 1983, pharmaceutical companies have studied and marketed products for orphan disease, taking advantage of the market exclusivity provided by the Orphan Drug Act. More recently, similar incentives are also being provided f o r interventions in pediatrics. Challenges with products in these patient populations persist afier the product is on the market, including how the medical department supports practitioners who have questions that go beyond the limits of approved prescribing information. When planning f o r and managing this effort, medical information practitioners need to consider the unique circumstances that make this effort less eficient, including dossiers based upon limited numbers of patients, few placebo-controlled trial reports, product novelty, and infrequent, specialized requests. Key Words: Orphan drugs; Pediatrics; Unsolicited requests; Drug information
INTRODUCTION THE ORPHAN DRUG ACT was passed in 1983. This law provides an innovator company with seven years of market exclusivity, independent of patent status, as an incentive to investigate therapeutic interventions in rare disorders. As a consequence, a number of treatment innovations have been pursued in the clinic in recent years. Indeed, drug development in orphan diseases is apparent in the United States but not in other countries, where this market exclusivity does not exist (1). In contrast to adults, the pediatric population is much smaller, and pediatric disorders have been “orphaned” over the years. “Pediatric” disorders, for the purposes of this discussion, are those limited to patients below the age of majority, making their routine inclusion in trials more difficult. Conduct of
Reprint address: David A. Graves, ph@, Associate Director, Medical Information. Genentech, Inc., 1 DNA Way, South San Francisco, CA 94080.
adequate studies is difficult due to parental hesiiancy regarding enrollment of their child in a placebo-controlled trial. The Food and Drug Administration (FDA) Modernization Act of 1997 provides incentives to the industry to attempt studies in pediatrics (six months of additional market exclusivity), but it does not reduce the problems associated with studying this group of patients. Efficacy in adults will often be supportive of pediatric experience, but this is not always the case. Minimal discussion has emerged about how drugs in these unusual circumstances are supported by industry once they are on the market. (While some of these challenges might be more common to biologics, most would be evident for any new therapeutic modality. Therefore, for this discussion, the use of the term “drug” includes biologics.) Difficulties associated with addressing questions about the appropriate use of drugs in these disorders persist after a
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