The Newborn Drug Development Initiative

Abstract

The Best Pharmaceuticals for Children Act (BPCA; Pub L 107-109) was enacted in January 2002 and will sunset in October 2007. The BPCA established processes for studying off-patent and on-patent drugs that are used in pediatric population. Although some drugs have been successfully developed for the neonate (eg, surfactant, nitric oxide), drug development for the youngest, least mature, and most vulnerable pediatric patients is generally lacking. Most drugs are empirically administered to newborns once efficacy has been demonstrated in adults and usefulness is suspected or demonstrated in the older pediatric population. Unfortunately, this process undermines the ability to perform the appropriate studies necessary to demonstrate a drug's short-and long-term safety and efficacy and establish appropriate dosing in neonates. The Newborn Drug Development Initiative Workshop I (held March 29 -30, 2004) specifically addressed scientific, clinical, and ethical concerns in the development of trials of pediatric therapeutic agents for neonates. Implementation of the BPCA for all pediatric populations will foster collaboration among federal agencies and academic institutions on scientific investigation, clinical-study design, and consideration of the weight of evidence and address ethical issues related to the performance of drug studies.www.pediatrics.org/cgi

Off-Label Prescribing to Children in the United States Outpatient Setting

“…Few medications are tested or approved in this age group, 43 giving physicians limited choices for medications approved for young children. Given the difficulties in enrolling young children in studies, this group likely will continue to be understudied by pharmaceutical manufacturers.…”

Pharmacotherapy in Pediatric Critical Illness

“…[15,16] These data underscore earlier calls for more comprehensive regulatory standards and postmarketing studies in children. [3,40] …”

“…[1][2][3] Medications are employed to treat primary disease, to facilitate initiation of mechanical organ support (most commonly respiratory, cardio-circulatory, renal, or hepatic), and to enable the continued use of these therapies. Medications are also used to counteract unwanted or excessive effects of other medications.…”

Federal Legislation and the Advancement of Neonatal Drug Studies

“…In recognition of the paucity of children-specific pharmacologic data on medication prescribed to children, the federal government, through the National Institute of Health (NIH) and the Food and Drug Administration (FDA), have taken several steps towards generating new knowledge about medicines prescribed to children [9, 10]. These resulted in the creation of the Pediatric Pharmacology Research Units (PPRU) Network, the FDA Modernization Act (FDAMA), the Best Pharmaceuticals for Children Act (BPCA), and the Pediatric Research Equity Act (PREA) [11–14].…”