Drug development process for a product with a primary pediatric indication.

This article reviews the drug development process in the United States, focusing on practical issues and new U.S. Food and Drug Administration (FDA) regulations and guidance for developing a drug with a primary pediatric indication. Atomoxetine, a novel treatment for attention-deficit/ hyperactivity disorder (ADHD), is used to illustrate how the modern drug development process works and to highlight changes in the development of ADHD treatments since the introduction of the stimulants over 50 years ago. In addition to dealing with unique regulatory requirements and guidance, developing a drug for use in a pediatric population poses novel challenges in diverse areas including biomedical ethics, developmental pharmacology, and clinical trial design and implementation.