title: Safety, dosing, and pharmaceutical quality for studies that evaluate medicinal products (including biological products) in neonates.
abstract: The study of medications among pediatric patients has increased worldwide since 1997 in response to new legislation and regulations, but these studies have not yet adequately addressed the therapeutic needs of neonates. Additionally, extant guidance developed by regulatory agencies worldwide does not fully address the specificities of neonatal drug development, especially among extremely premature newborns who currently survive. Consequently, an international consortium from Canada, Europe, Japan, and the United States was organized by the Critical Path Institute to address the content of guidance. This group included neonatologists, neonatal nurses, parents, regulators, ethicists, clinical pharmacologists, specialists in pharmacokinetics, specialists in clinical trials and pediatricians working in the pharmaceutical industry. This group has developed a comprehensive, referenced White Paper to guide neonatal clinical trials of medicines - particularly early phase studies. Key points include: the need to base product development on neonatal physiology and pharmacology while making the most of knowledge acquired in other settings; the central role of families in research; and the value of the whole neonatal team in the design, implementation and interpretation of studies. This White Paper should facilitate successful clinical trials of medicines in neonates by informing regulators, sponsors, and the neonatal community of existing good practice.
authors.affiliation: Department of Pediatrics, Divisions of Neonatology and Clinical Pharmacology, University of Utah, Salt Lake City, Utah.
authors.forename: Robert M
authors.initials: RM
authors.lastname: Ward
authors.affiliation: Department of Pediatrics and Duke Clinical Research Institute, Duke University, Durham, North Carolina.
authors.forename: Daniel
authors.initials: D
authors.lastname: Benjamin
authors.affiliation: Translational Informatics and Pediatric Network, Sanofi Pharmaceuticals, Bridgewater, New Jersey.
authors.forename: Jeffrey S
authors.initials: JS
authors.lastname: Barrett
authors.affiliation: Intensive Care and Department of Surgery, Erasmus MC-Sophia Children's Hospital, Rotterdam, The Netherlands.|The Neonatal Intensive Care Unit, University of Leuven, Leuven, Belgium.
authors.forename: Karel
authors.initials: K
authors.lastname: Allegaert
authors.affiliation: Pediatric Therapeutic Area, Novartis Pharmaceuticals Corporation, East Hanover, New Jersey.
authors.forename: Ronald
authors.initials: R
authors.lastname: Portman
authors.affiliation: Department of Pediatrics, Floating Hospital for Children, Tufts Medical Center, and Tufts Clinical and Translational Science Institute, Tufts University, Boston, Massachusetts.
authors.forename: Jonathan M
authors.initials: JM
authors.lastname: Davis
authors.affiliation: Institute of Translational Medicine, University of Liverpool, Liverpool, UK.|Critical Path Institute, Tucson, Arizona.
authors.forename: Mark A
authors.initials: MA
authors.lastname: Turner
mesh_terms: D000367:Age Factors; D001685:Biological Factors / Q000008:administration & dosage* / Q000009:adverse effects / Q000493:pharmacokinetics / Q000592:standards; D002986:Clinical Trials as Topic / Q000379:methods* / Q000592:standards; D004305:Dose-Response Relationship, Drug; D054796:Drug Dosage Calculations; D019317:Evidence-Based Medicine; D006801:Humans; D007231:Infant, Newborn; D004364:Pharmaceutical Preparations / Q000008:administration & dosage* / Q000592:standards; D011786:Quality Control; D012107:Research Design / Q000592:standards; D018570:Risk Assessment; D012307:Risk Factors
chemical_list: D001685:Biological Factors; D004364:Pharmaceutical Preparations