Mechanisms to provide safe and effective drugs for children.

Abstract

* Abbreviation: FDA — : Food and Drug Administration 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 , Wharton et al1 review the labeling changes related to pediatric exclusivity. These 3 items are worth additional consideration. First, although the FDA issued 401 written requests, pharmaceutical companies chose to respond to fewer than … Address correspondence to Daniel K. Benjamin Jr, MD, PhD, MPH, Duke University, Duke Clinical Research Institute, PO Box 17969, Durham, NC 27715. E-mail: danny.benjamin{at}duke.edu