Provision of Medical Information for Drugs used in Orphan and Pediatric Disorders

Abstract

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 for interventions in pediatrics. Challenges with products in these patient populations persist after 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 for and managing this effort, medical information practitioners need to consider the unique circumstances that make this effort less efficient, including dossiers based upon limited numbers of patients, few placebo-controlled trial reports, product novelty, and infrequent, specialized requests.