title: Paediatric drug development: the impact of evolving regulations.
abstract: Children deserve medicines that are adapted to their needs. The need to include children in drug development has been recognised increasingly over the past few decades. Legal and regulatory frameworks are well established in the EU and US. The amount of work done to study medicines for children is significantly greater than it was 10 years go. Proof-of-concept has been demonstrated for all segments of the paediatric drug development pipeline. It is now time to examine how the practice of developing medicines for children has evolved within those frameworks and to determine how that work should be generalised. This review describes the development of medicines for children and critically appraises the work that has been done within those frameworks. Significant effort is needed to realize the potential provided by the current regulatory framework. Using the work programme of the Global Research in Paediatrics (GRiP) Network of Excellence as a template we outline current work and future growing points.
authors.affiliation: University of Liverpool, Department of Women's and Children's Health, Institute of Translational Medicine, Liverpool Women's NHS Foundation Trust, Crown Street, Liverpool L8 7SS, UK. Electronic address: mark.turner@liv.ac.uk.
authors.forename: M A
authors.initials: MA
authors.lastname: Turner
authors.affiliation: University of Pavia, Italian Group for the Study of Pharmacoeconomics (GISF), Via Luigi Porta 14, 27100 Pavia, Italy. Electronic address: mariana.c@libero.it.
authors.forename: M
authors.initials: M
authors.lastname: Catapano
authors.affiliation: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), 31 Center Drive, Building 31, Room 2A32, Bethesda, MD 20892-2425, USA. Electronic address: hirschfs@mail.nih.gov.
authors.forename: S
authors.initials: S
authors.lastname: Hirschfeld
authors.affiliation: Azienda Ospedaliera di Padova (AOPD), Department of Paediatrics, Via Giustiniani 1, 35128 Padova, Italy. Electronic address: carlog@pediatria.unipd.it.
authors.forename: C
authors.initials: C
authors.lastname: Giaquinto
mesh_terms: D000818:Animals; D002648:Child; D055808:Drug Discovery / Q000331:legislation & jurisprudence*; D005062:European Union; D006801:Humans; D007880:Legislation, Drug; D010372:Pediatrics; D014481:United States
keywords: Children's medicine; Extrapolation; Pharmaceutical development; Pharmacodynamics; Pharmacokinetics; Regulatory framework; Risk benefit ratio