Linder BL, Fradkin JE, Rodgers GP. The TODAY study: an NIH perspective on its implications for research. Diabetes Care. 2013;36(6):1775–6. https://doi.org/10.2337/dc13-0707.
Article PubMed PubMed Central Google Scholar
Copeland KC, Silverstein J, Moore KR, Prazar GE, Raymer T, Shiffman RN, et al. Management of newly diagnosed type 2 diabetes mellitus (T2DM) in children and adolescents. Pediatrics. 2013;131(2):364–82. https://doi.org/10.1542/peds.2012-3494.
21CFR314.55. https://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?fr=314.55.
Pediatric Research Equity Act. https://www.fda.gov/drugs/development-resources/pediatric-research-equity-act-prea#:~:text=PREA%20gives%20FDA%20the%20authority,pediatric%20labeling%20for%20the%20product.
Tamborlane WV, Barrientos-Pérez M, Fainberg U, Frimer-Larsen H, Hafez M, Hale PM, et al. Liraglutide in children and adolescents with type 2 diabetes. N Engl J Med. 2019;381(7):637–46. https://doi.org/10.1056/NEJMoa1903822.
Article CAS PubMed Google Scholar
Arslanian SA, Hannon T, Zeitler P, Chao LC, Boucher-Berry C, Barrientos-Pérez M, et al. Once-weekly dulaglutide for the treatment of youths with type 2 diabetes. N Engl J Med. 2022;387(5):433–43. https://doi.org/10.1056/NEJMoa2204601.
Article CAS PubMed Google Scholar
Laffel LM, Danne T, Klingensmith GJ, Tamborlane WV, Willi S, Zeitler P, et al. Efficacy and safety of the SGLT2 inhibitor empagliflozin versus placebo and the DPP-4 inhibitor linagliptin versus placebo in young people with type 2 diabetes (DINAMO): a multicentre, randomised, double-blind, parallel group, phase 3 trial. Lancet Diabetes Endocrinol. 2023;11(3):169–81. https://doi.org/10.1016/S2213-8587(22)00387-4.
Article CAS PubMed PubMed Central Google Scholar
Supplemental NDAs 201280/S-027, 201281/S-035, 208026/S-024 Clinical Review. https://www.fda.gov/media/172628/download
Food and Drug Administration. E11A pediatric extrapolation. 2022. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/e11a-pediatric-extrapolation.
Travis J, Rothmann M, Thomson A. Perspectives on informative Bayesian methods in pediatrics. J Biopharm Stat. 2023. https://doi.org/10.1080/10543406.2023.2170405.
Food and Drug Administration. E11(R1) addendum: clinical investigation of medicinal products in the pediatric population. 2018. https://www.fda.gov/media/101398/download.
ADAPP. Children and adolescents: standards of care in diabetes—2024. Diabetes Care. 2024;47(Supplement 1):S258–81. https://doi.org/10.2337/dc24-S014.
Copeland KC, Zeitler P, Geffner M, Guandalini C, Higgins J, Hirst K, et al. Characteristics of adolescents and youth with recent-onset type 2 diabetes: the TODAY cohort at baseline. J Clin Endocrinol Metab. 2011;96(1):159–67.
Article CAS PubMed Google Scholar
Arslanian S, Bacha F, Grey M, Marcus MD, White NH, Zeitler P. Evaluation and management of youth-onset type 2 diabetes: a position statement by the American diabetes association. Diabetes Care. 2018;41(12):2648.
Article CAS PubMed PubMed Central Google Scholar
VICTOZA product label. https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/022341s044lbl.pdf
BYDUREON Product Label. https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/022200s036lbl.pdf
TRULICITY Product Label. https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/125469s063lbl.pdf
JARDIANCE Product Label: https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/204629s040lbl.pdf
FARXIGA Product Label: https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/202293s031lbl.pdf
INVOKANA Product Label. https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/204042s043lbl.pdf
BLA 125370/s-064 and BLA 761043/s-007 multi-disciplinary review and evaluation benlysta® (belimumab) for intravenous infusion in children 5 to 17 years of age with SLE. https://www.fda.gov/media/127912/download.
NDA multi-disciplinary review and evaluation . https://www.accessdata.fda.gov/drugsatfda_docs/nda/2023/214070Orig1s000TOC.cfm.
Wang Y, Tu W, Kim Y, Sinks S, He J, Cambon A, et al. Statistical methods for handling missing data to align with treatment policy strategy. Pharm Stat. 2023;22(4):650–70. https://doi.org/10.1002/pst.2299.
Neuenschwander B, Capkun-Niggli G, Branson M, Spiegelhalter DJ. Summarizing historical information on controls in clinical trials. Clin Trials (London, England). 2010;7(1):5–18. https://doi.org/10.1177/1740774509356002.
Schmidli H, Gsteiger S, Roychoudhury S, O’Hagan A, Spiegelhalter D, Neuenschwander B. Robust meta-analytic-predictive priors in clinical trials with historical control information. Biometrics. 2014;70(4):1023–32. https://doi.org/10.1111/biom.12242.
Röver C, Friede T. Meta-analytic-predictive priors based on a single study. 2025. arXiv preprint arXiv:2505.15502.
Neuenschwander B, Weber S, Schmidli H, O’Hagan A. Predictively consistent prior effective sample sizes. Biometrics. 2020;76(2):578–87. https://doi.org/10.1111/biom.13252.
Wiesenfarth M, Calderazzo S. Quantification of prior impact in terms of effective current sample size. Biometrics. 2020;76(1):326–36. https://doi.org/10.1111/biom.13124.
Morita S, Thall PF, Müller P. Determining the effective sample size of a parametric prior. Biometrics. 2008;64(2):595–602. https://doi.org/10.1111/j.1541-0420.2007.00888.x.
Zhang H, Anderson KM, Zimmer Z, Golm G, Sapre A, Ibrahim JG.. Prior Effective sample size when borrowing on the treatment effect scale. 2024. arXiv preprint arXiv:2404.13366.
JANUMET Product Label. https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/022044s052lbl.pdf
JANUMET XR Product Label. https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/202270s026lbl.pdf
Schmidli H, Häring DA, Thomas M, Cassidy A, Weber S, Bretz F. Beyond randomized clinical trials: use of external controls. Clin Pharmacol Ther. 2020;107(4):806–16. https://doi.org/10.1002/cpt.1723.
Majumdar A, Rothwell R, Reaman G, Ahlberg C, Roy P. Utility of propensity score-based Bayesian borrowing of external adult data in pediatric trials: a pragmatic evaluation through a case study in acute lymphoblastic leukemia (ALL). J Biopharm Stat. 2023. https://doi.org/10.1080/10543406.2022.2162069.
Sebastien B, Cheung SA, Corriol-Rohou S, Gamalo-Siebers M, Jreich R, Krishna R, et al. Use of pharmacodynamic modeling for Bayesian information borrowing in pediatric clinical trials. J Biopharm Stat. 2022. https://doi.org/10.1080/10543406.2022.2149772.
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