Children are therapeutic orphans. The majority of drugs used in children are “off label”; that is, the data regarding dose, safety, or efficacy (and often all 3) are not deemed sufficient by the US Food and Drug Administration (FDA) for inclusion on the product label. Not surprisingly, off-label drug use increases the risk of adverse events. To address the knowledge gap leading to such use, the US government has enacted multiple laws to stimulate increased drug research and labeling for children. One such law is known as “pediatric exclusivity,” first passed in 1997 and most recently made permanent in 2012 under the FDA Safety and Innovation Act. Pediatric exclusivity grants pharmaceutical companies a 6-month patent extension if they study a product under a written request from the FDA. For so-called blockbuster drugs, those with ≥$1 billion in sales per year, the incentive is substantial.

In this issue of Pediatrics,...

You do not currently have access to this content.