References
  1. Rieder MJ. If children ruled the pharmaceutical industry: The need for pediatric formulations. Drug News Perspect 2010; 23: 458-64.
  2. Bideau A. Desjardins B, Pérez Brignoli H (eds). Infant and Child Mortality in the Past. Claredon Press, Oxford, 1997.
  3. Kermack W, McKendrick AG, McKinley PL. Death Rates in Great Britain and Sweden: Some general regularities and their significance.Lancet 1934; 31: 698-703.
  4. UN Inter-agency Group for Child Mortality Estimation, https://childmortality.org
  5. Fleming A. On the antibacterial action of cultures of a penicillium with special reference to their use in the isolation of B. influenzae. Br J Exper Path 1929; 10: 226-36.
  6. Weinshilboum RM. The therapeutic revolution. Clin Pharmacol Ther 1987; 42: 481-5.
  7. Domagk G. Eine neue klasse von desinfektionshmittelm. Deutsch Med Wochenschr 1935; 61: 829-32.
  8. Tréfouël J, Tréfouël J, Nitti F, Bovet D. Activité du p-aminophényl-sulfamide sur les infections stretococciques expérimentales de la souris et du lapin. Cr Scanc Soc Biol1935; 120: 756-8.
  9. Thomas L. The youngest science: Notes of a medicine-watcher. Viking Press. New York, 1983.
  10. O’Leary M, Krailo M, Reaman GH. Progress in childhood cancer: 50 years of research collaboration, a report from the Children’s Oncology Group. Semin Oncol 2008; 35: 484-93.
  11. Connolly CA. Drug Therapy: From ‘Baby Killers’ to Baby Savers, 1906–1933.” In: Children and Drug Safety: Balancing Risk and Protection in Twentieth-Century America , by Cynthia A Connolly, Rutgers University Press, New Brunswick, Camden, Newark, New Jersey; London, 2018, pp. 1–15.
  12. Wax PM. Elixirs, diluents and the passage of the 1938 Federal Food, Drug and Cosmetic Act. Ann Intern Med 1995; 122: 456-61.
  13. Sutherland JM. Fatal cardiovascular collapse of infants receiving large amounts of chloramphenicol. Am J Dis Child 1959; 97: 761-7.
  14. Mulhall A, de Louvois J, Hurley R. Chloramphenicol toxicity in neonates Its incidence and prevention. Br Med J 1983; 287: 1424-6.
  15. Kearns GL, Abdel-Rahman SM, Alander SW et al. Developmental pharmacology – drug disposition, action and therapy in infants and children. N Engl J Med 2003; 349: 1157-67.
  16. Burke EC. Abraham Jacobi, MD: The man and his legacy. Pediatr1998; 101: 309-12.
  17. Burgio GR. The thalidomide disaster revisited. Eur J Pediatr1981; 136: 229-30.
  18. Vargesson N. Thalidomide induced teratogenesis: History and mechanisms. Birth Defects Res C Embryo Today 2015; 105: 140-56.
  19. McBride WG. Thalidomide and congenital malformations.. Lancet1961; 2: 1358.
  20. Lenz W. Kindliche missbildungen nach medikameteinnahme wahrend der graviditat? Dtsch Med Wochensche 1961; 86: 2555-6.
  21. Kauffman RE. Status of drug approval processes and regulation of medications for children. Curr Opin Pediatr 1995; 7: 195-8.
  22. Shirkey HC. Therapeutic orphans J Pediatr 1968; 72: 119-20.
  23. Kauffman RE. Essential drug for infants and children: North American perspective. Pediatr 1999; 104 (S3):603-5.
  24. Committee on Drugs, American Academy of Pediatrics. Use of drugs not described in the package insert (off-label uses). Pediatr 2002; 110: 181-3.
  25. Pandolfini C, Bonatii M. A literature review on off-label drug use in children. Eur J Pediatr 2005; 164: 522-8.
  26. MacLeod SM. Current environment for drug development, regulation and use. In: Improving Medicines for Children in Canada. Council of Canadian Academies, Ottawa, Ontario, 2014.
  27. Rieder MJ, Matsui D, MacLeod S: Myths and challenges – Drug utilization by Canadian children. Paed Child Health 2003; 8: 7A-8A
  28. Khaled LA, Ahmed F, Brogan T et al. Prescription drug use by one million Canadian children. Paed Child Health 2003; 8 (Supple A), 1-51.
  29. Lasky T. Estimates of pediatric medication use in the United States: current abilities and limitations. Clin Ther 2009;31:436-45.
  30. Turner MA, Lewis S, Hawcutt DB, Field D. Prioritising neonatal medicines research: UK Medicines for Children Research Network scoping survey. BMC Pediatr 2009 Aug 12;9:50.
  31. Vernacchio L, Kelly JP, Kaufman DW, Mitchell AA. Medication use among children <12 years of age in the United States: results from the Slone Survey. Pediatr 2009;124:446-54.
  32. Clavenna A, Bonati M. Drug prescriptions to outpatient children: a review of the literature. Eur J Clin Pharmacol 2009;65:749-55.
  33. Chai G, Governale L, McMahon AW, Trinidad AW Trinidad JP, Staffa J, Murphy D. Trends of outpatient prescription drug use in US children, 2002-2010. Pediatr 2012 ;130:23-31.
  34. Rashed AN, Wong ICK, Wilton L, Tomlin S, Neubert A. Drug utilization patterns in children admitted to a paediatric general medical ward in five countries. Drugs Real World Outcomes 2015; 2: 397-410.
  35. Rosli R, Dali AF, Abd Aziz N, Abdullah AH, Ming LC, Manan MM. Drug utilization on neonatal Wards: A systematic review of observational Studies. Front Pharmacol 2017; 8:27.
  36. Martin CB, Hales CM, Gu Q, Ogden CL. Prescription drug use in the United States, 2015-2016. NCHS Data Brief 2019; 334.
  37. Swart J, Giancane G, Horneff G et al. Pharmacovigilance in juvenile idiopathic arthritis patients treated with biologic or synthetic drugs: Combined data of more than 15,000 patients from Pharmachild and national registries. Arthritis Res Ther 2018; 20: 285.
  38. Griesenauer RH, Kinch MS. 2016 in review: FDA approvals of new molecular entitites. Drug Discov Today 2017; Jul 4. pii: S1359-6446(17)30129-0.
  39. Mahmood I. Pharmacokinetic considerations in designing pediatric studies of proteins, antibiotics and plasma-derived products. Am J Ther 2016;23:e1043-56.
  40. Wilson JT. Update on the therapeutic orphan. Pediatr 1999; 104: 585-9.
  41. Aronson JK, Ferner RE. Unlicensed and off-label uses of medicine: Definitions and clarification of terminology. Br J Clin Pharmacol 2017; 83: 2615-25.
  42. Cohen SN. The pediatric pharmacology research unit (PPRU) network and its role in meeting pediatric labeling needs. Pediatr 1999; 104: 644-5.
  43. Institute of Medicine (US) Forum on Drug Discovery, Development, and Translation. Addressing the Barriers to Pediatric Drug Development: Workshop Summary. Washington (DC): National Academies Press (US); 2008. 2, Regulatory Framework.
  44. Wharton GT, Murphy MD, Avant D et al. Impact of pediatric exclusivity on drug labelling and demonstrations of efficacy.Pediatr 2014; 134: e512-8.
  45. Penkov D, Tomasi P, Eichler I et al. Pediatric medicine development: An overview and comparison of regulatory processes in the European Union and the United States. Ther Inno Regul Sci 2017; 51: 360-71.
  46. Thomsen MDT. Global pediatric drug development. Curr Ther Res2019; 90: 135-42.
  47. Moore Hepburn C, Gilpin A, Autmizguine J et al. Improving paediatric medications: A prescription for Canadian chidren and youth.Paeds Child Health 2019; 24: 333-5.
  48. Saitou H, Nakatani D, Myoui A, Kubota , Ozono K. Pediatric drug development in Japan: Current issues and perspectives. Clin Pediatr Endocrinol 2020; 29: 1-7.
  49. Committee on Drugs, American Academy of Pediatrics. Off-label use of drugs in children. Pediatr 2014, 133 563-567.
  50. Corny J, Lebel D, Bailey B, Bussières JF. Unlicensed and off-label drug use in children before and after pediatric government incentives.J Pediatr Pharmacol Ther 2015; 20: 316-28.
  51. Rieder MJ. Size and Taste Matters: Recent Progress in the Development of Age-Appropriate Medicines for Children. Pharmaceut Med2018; 32:21-30.
  52. Gerrard SE, Walsh J, Bowers N, Salunka S, Hershenson S. Innovations in pediatric drug formulations and administration technologies for low resource settings. Pharmaceutics 2019; 11: doi:10.3390/pharmaceutics 11100518.
  53. Thabet Y, Klingmann V, Breitkreutz J. Drug formulation: Standards and novel strategies for drug administration in pediatics. J Clin Pharmacol 2018;58 Suppl 10:S26-S35.
  54. Teixeira T, Kweder SL, Saint-Raymond A. Are the European Medicine Agency, US Food and Drug Administration, and other international regulators talking to each other? Clin Pharmacol Ther 2020: 107: 507-13.
  55. Rieder M, Hawcutt D. Design and conduct of early phase drug studies in children. Br J Clin Pharmacol 2016;82:1308-1314. PMID: 27353241
  56. Duffett M, Swinton M, Brouwers M, Meade M, Cook DJ. Advancing randomized controlled trials in pediatric critical care: The perspectives of trialists. Pediatr Crit Care Med2018;19:e595-e602.
  57. Cohen JW, Akshintala S, Kane E, et al. A systematic review of pediatric Phase I trails in oncology: Toxicity and outcomes in the era of targeted therapies. Oncologist 2020 Jan 14. doi: 10.1634/theoncologist.2019-0615.
  58. Collingnon O, Gartner C, Haidich A-B et al. Current statistical considerations and regulatory perspectives on the planning of confimatory basket, umbrella and platform trials. Clin Pharmacol Ther 2020; 107: 1059-67.
  59. Breckenridge A, Feldschreiber P. Impact of Brexit on UK and EU Drug Regulation and Patient Access. Clin Pharmacol Ther 2019; 105: 923-5.
  60. MacLeod SM, Greff M, Knoppert DC, Ito S, Rieder MJ. An international asset map of clinicians, educators and researchers pursing better medicine use in children: Initial findings. Clin Pharmacol Ther2017; 101: 274-80.