Global Harmonization of Pediatric Drug Development: Critical for Progress for Developing Safe and Effective Therapeutic Agents for Children

Current Therapeutic Research - Tập 90 - Trang 109-112 - 2019
Andrew E. Mulberg1, Timothy Cripps1
1Amicus Therapeutics Inc, Cranbury, NJ

Tài liệu tham khảo

Rose, 2019, Questionable Industry-sponsored Studies in Children and Adolescents in Slovenia, Curr Ther Res, 10.1016/j.curtheres.2019.01.002 2013 2012 Shirkey, 1999, Therapeutic orphans, Pediatrics, 104, 583, 10.1542/peds.104.S3.583 Stafford, 2012, Off-label use of drugs and medical devices: a review of policy implications, Clin Pharmacol Ther, 91, 920, 10.1038/clpt.2012.22 Kimland, 2012, Off-label drug use in pediatric patients, Clin Pharmacol Ther, 91, 796, 10.1038/clpt.2012.26 Personal communication, Lily Mulugeta, Division of Pediatrics and Maternal Health, U.S. Food and Drug Administration. Analysis was completed using the data sets from: [email protected] (adult NDA) and FDA New Pediatric Labeling Information Database (pediatric label changes). Accessed June 2014. Sample size N = 118 pediatric labels randomly selected). Wertheimer, 2011, Off-label prescribing of drugs for children, Curr Drug Saf, 6, 46, 10.2174/157488611794479973 Radley, 2006, Off-label prescribing among office-based physicians, Arch Intern Med, 166, 1021, 10.1001/archinte.166.9.1021 Pediatric Research Equity Act of 2007 and Best Pharmaceuticals for Children Act of 2007. Available at: http://www.fda.gov/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm049867.htm. Accessed September 21, 2012. Karesh, 2013, Off-label use of medicine in pediatrics: focus on gastrointestinal diseases, Curr Opin Pediatr, 25, 612, 10.1097/MOP.0b013e328363ed4e Karesh, 2013, US Perspective on Off-label Use in Pediatrics, JPGN, 56 Mulberg, 2008, Pharmaceutical Industry in Pediatric Drug Development: Partners and Collaborators With Academia and the FDA, J Pediatr Gastroenterol Nutr, 47, 10.1097/01.mpg.0000338970.57088.89 Wynn, 2012, Off Label Use of Drugs for Gastrointestinal Diseases in Children: Focus on EoE, J PGN Benjamin, 2009, Safety and transparency of pediatric drug trials, Arch Pediatr Adolesc Med, 163, 1080, 10.1001/archpediatrics.2009.229 Prevacid (lansoprazole) Delayed-Release Capsules, Prevacid SoluTab (lansoprazole) Delayed-Release Orally Disintegrating Tablets labeling. Available at: http://www.accessdata.fda.gov/drugsatfda_docs/label/2012/020406s079,021428s026lbl.pdf. Label approved May 3, 2012. Accessed September 27, 2012. Chai, 2012, Trends of outpatient prescription drug utilization in US children, 2002–2010, Pediatrics, 130, 23, 10.1542/peds.2011-2879 Rose, 2019, Questionable Industry-sponsored Studies in Children and Adolescents in Slovenia, Curr Ther Res, 10.1016/j.curtheres.2019.01.002 Regulation (EC) No 1901/2006 of the European Parliament and of the Council of 12 December2006 on medicinal products for pediatric use and amending Regulation (EEC) No 1768/92, Directive 2001/20/ EC, Directive 2001/83/EC and Regulation (EC) No 726/2004. Official Journal L 378, 27/12/2006, 1-19, 2006. Available at: http://ec.europa.eu/health/files/eudralex/vol-1/reg_2006_1901/reg_2006_1901_en.pdf; https://www.ema.europa.eu/en/human-regulatory/overview/paediatric-medicines/paediatric-regulation. 10-Year Report to the European Commission; General report on the experience acquired as a result of the application of the Paediatric Regulation. EMA231225/2015 27th Oct 2016. State of Paediatric Medicines in the EU 10 years of the EU Paediatric Regulation: Report from the Commission to the European Parliament and the Council COM (2017) 626. https://ec.europa.eu/health/sites/health/files/files/paediatrics/2016_pc_report_2017/ema_10_year_report_for_consultation.pdf