Joseph PD, Craig JC, Caldwell PH. Clinical trials in children. Br J Clin Pharmacol. 2015;79:357–69.
Article PubMed PubMed Central Google Scholar
Pandolfini C, Bonati M. A literature review on off-label drug use in children. Eur J Pediatr. 2005;164:552–8.
Czaja AS, Reiter PD, Schultz ML, et al. Patterns of off-label prescribing in the pediatric intensive care Unit and prioritizing future research. J Pediatr Pharmacol Ther. 2015;20:186–96.
PubMed PubMed Central Google Scholar
Gore R, Chugh PK, Tripathi CD, et al. Pediatric off-label and unlicensed drug use and its implications. Curr Clin Pharmacol. 2017;12:18–25.
Horen B, Montastruc JL, Lapeyre-Mestre M. Adverse drug reactions and off-label drug use in paediatric outpatients. Br J Clin Pharmacol. 2002;54:665–70.
Article PubMed PubMed Central Google Scholar
Cuzzolin L, Atzei A, Fanos V. Off-label and unlicensed prescribing for newborns and children in different settings: a review of the literature and a consideration about drug safety. Expert Opin Drug Saf. 2006;5:703–18.
Pediatric research equity act of 2003. Public Law 108–55. https://www.govinfo.gov/content/pkg/PLAW-108publ155/pdf/PLAW-108publ155.pdf. Accessed 20 Aug 2024.
FDA reauthorization act of 2017. Public Law 115–52. https://www.govinfo.gov/content/pkg/PLAW-115publ52/pdf/PLAW-115publ52.pdf. Accessed 20 Aug 2024.
Ueyama E, Kaneko M, Narukawa M. Pediatric drug development in Japan: a comparison of the current situation and characteristics between Japan and Europe. Ther Innov Regul Sci. 2020;54:1152–8.
Hirota S, Yamaguchi T. Timing of pediatric drug approval and clinical evidence submitted to regulatory authorities: international comparison among Japan, the United States, and the European Union. Clin Pharmacol Ther. 2020;108:985–94.
Japan Pharmaceuticals and Medical Devices Agency website. https://www.pmda.go.jp/PmdaSearch/iyakuSearch/. Accessed 20 Aug 2024.
Drug@FDA. https://www.accessdata.fda.gov/scripts/cder/daf/index.cfm. Accessed 20 Aug 2024.
ATC/DDD Index 2024. https://www.whocc.no/atc_ddd_index/. Accessed 20 Aug 2024.
Clinical investigation of medicinal products in the pediatric population; E11:(R1). https://database.ich.org/sites/default/files/E11_R1_Addendum.pdf. Accessed 20 Aug 2024.
Nakayama H, Matsumaru N, Tsukamoto K. The drug lag and associated factors for orphan anticancer drugs in Japan compared to the United States. Invest New Drugs. 2019;37:1086–93.
Article CAS PubMed Google Scholar
Kanda Y. Investigation of the freely available easy-to-use software “EZR” for medical statistics. Bone Marrow Transplant. 2013;48:452–8.
Article CAS PubMed Google Scholar
Enya K, Lim Y, Sengoku S, et al. Increasing orphan drug loss in Japan: trends and R&D strategy for rare diseases. Drug Discov Today. 2023;28:1037–55.
Ohashi T, Nagashima M, Kawai N, et al. A narrative review on drug development for the management of antimicrobial- resistant infection crisis in Japan: the past, present, and future. Expert Rev Anti-Infect Ther. 2022;20:1603–14.
Article CAS PubMed Google Scholar
Laughon MM, Avant D, Tripathi N, et al. Drug labeling and exposure in neonates. JAMA Pediatr. 2014;168:130–6.
Article PubMed PubMed Central Google Scholar
Bell SA, Tudur SC. A comparison of interventional clinical trials in rare versus non-rare diseases: an analysis of ClinicalTrials.gov. Orphanet J Rare Dis. 2014;9:170.
Article PubMed PubMed Central Google Scholar
Tsuji K, Tsutani K. Approval of new drugs 1999–2007: comparison of the US, the EU and Japan situations. J Clin Pharm Ther. 2010;35:289–301.
Article CAS PubMed Google Scholar
Tanaka M, Idei M, Sakaguchi H, et al. Evolving landscape of new drug approval in Japan and lags from international birth dates: retrospective regulatory analysis. Clin Pharmacol Ther. 2021;109:1265–73.
Tajima G, Matsumaru N, Tsukamoto K. Impact of expedited programs in the United States, as foreign regulatory factors, on clinical development time in Japan. J Clin Pharm Ther. 2022;47:1395–401.
Hudgins JD, Bacho MA, Olsen KL, et al. Pediatric drug information available at the time of new drug approvals: a cross-sectional analysis. Pharmacoepidemiol Drug Saf. 2018;27:161–7.
Christiansen H, De Bruin ML, Frokjaer S, et al. Guidance for pediatric use in prescription information for novel medicinal products in the EU and the US. PLoS ONE. 2022;17:e0266353.
Article CAS PubMed PubMed Central Google Scholar
Zettler ME. The RACE for children act at one year: progress in pediatric development of molecularly targeted oncology drugs. Expert Rev Anticancer Ther. 2022;22:317–21.
Article CAS PubMed Google Scholar
Samuels S, Park K, Bhatt-Mehta V, Sun H, et al. Pediatric efficacy extrapolation in drug development submitted to the US food and drug administration 2015–2020. J Clin Pharmacol. 2023;63:307–13.
Article CAS PubMed Google Scholar
U.S. Food and Drug Administration: Guidance for industry: Drugs for treatment of partial onset seizures: Full extrapolation of efficacy from adults to pediatric patients 2 years of age and older. 2019. https://www.fda.gov/media/130449/download. Accessed 20 Aug 2024.
European Medicines Agency. Reflection paper on the use of extrapolation in the development of medicines for paediatrics. 2018. https://www.ema.europa.eu/en/documents/scientificguideline/adopted-reflection-paper-use-extrapolationdevelopment-medicines-paediatrics-revision-1_en.pdf. Accessed 20 Aug 2024.
Pediatric Extrapolation; E11A. https://database.ich.org/sites/default/files/ICH_E11A_Document_Step2_Guideline_2022_0404_0.pdf. Accessed 20 Aug 2024.
Momper JD, Mulugeta Y, Burckart GJ. Failed pediatric drug development trials. Clin Pharmacol Ther. 2015;98:245–51.
Comments (0)