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Discussion

Dynamic incorporation of real world evidence within the framework of adaptive design

ORCID Icon, ORCID Icon & ORCID Icon
Pages 986-998 | Received 21 Dec 2021, Accepted 04 May 2022, Published online: 22 Jun 2022

References

  • Barrett, J. S., C. Bucci-Rechtweg, S. Y. Amy Cheung, M. Gamalo-Siebers, S. Haertter, J. Karres, J. Marquard, Y. Mulugeta, C. Ollivier, A. Strougo, et al. 2020. Pediatric extrapolation in type 2 diabetes: Future implications of a workshop. Clinical Pharmacology and Therapeutics 1080 (1):29–39. doi:10.1002/cpt.1805.
  • ClinicalTrials.gov. 2018. Efficacy and safety of liraglutide in combination with metformin compared to metformin alone, in children and adolescents with type 2 diabetes (Ellipse™). Accessed September 30, 2021. https://clinicaltrials.gov/ct2/show/NCT01541215.
  • ClinicalTrials.gov. 2020a. A pooled analysis of the safety and efficacy of MK-0431A and MK-0431A XR in pediatric participants with type 2 diabetes mellitus with inadequate glycemic control on metformin therapy (Alone or in combination with insulin). (MK-0431A-170/MK-0431A-289). Accessed September 30, 2021. https://clinicaltrials.gov/ct2/show/NCT01760447.
  • ClinicalTrials.gov. 2020b. Study to assess safety efficacy of sitagliptin as initial oral therapy for treatment of type 2 diabetes mellitus in pediatric participants. (MK-0431-083). Accessed September 30, 2021. https://clinicaltrials.gov/ct2/show/NCT01485614.
  • Cui, L., H. M. James Hung, and S.-J. Wang. 1999. Modification of sample size in group sequential clinical trials. Biometrics 550 (3):853–857. doi:10.1111/j.0006-341X.1999.00853.x.
  • EMA. EMA regulatory science to 2025. 2020. Accessed September 30, 2021. https://www.ema.europa.eu/en/documents/regulatory-procedural-guideline/ema-regulatory-science-2025-strategic-reflection_en.pdf.
  • FDA. Rosiglitazone clinical review BPCA, 2005a. https://fda.report/media/90400/21071s015-Rosiglitazone-Clinical-BPCA.pdf [Accessed 2021 September 30].
  • FDA. 2005b. Glimepiride statistical review and evaluation BPCA. Accessed 2021 September 30. https://www.accessdata.fda.gov/drugsatfda_docs/pediatric/020496s015_glimepiride_Amaryl-Statistical_BPCA.pdf.
  • FDA. Critical path opportunities list. 2006. Accessed September 30, 2021. http://wayback.archive-it.org/7993/20180125035449/https://www.fda.gov/downloads/ScienceResearch/SpecialTopics/CriticalPathInitiative/CriticalPathOpportunitiesReports/UCM077258.pdf.
  • FDA. 2018. Complex innovative trial design pilot meeting program. https://www.fda.gov/drugs/development-resources/complex-innovative-trial-design-pilot-meeting-program#goals.
  • FDA. 2019. Submitting documents using real-world data and real-world evidence to fda for drugs and biologics: Guidance for industry: Draft guidance. Rockville, MD: US Food and Drug Administration.
  • Gamalo-Siebers, M., L. Hampson, K. Kordy, S. Weber, R. M. Nelson, and R. Portman. 2019. Incorporating innovative techniques toward extrapolation and efficient pediatric drug development. Therapeutic Innovation & Regulatory Science 530 (5):567–578. doi:10.1177/2168479019842541.
  • Gao, P., J. H. Ware, and C. Mehta. 2008. Sample size re-estimation for adaptive sequential design in clinical trials. Journal of Biopharmaceutical Statistics 180 (6):1184–1196. doi:10.1080/10543400802369053.
  • Gordon Lan, K. K., and D. L. DeMets. 1989. Group sequential procedures: Calendar versus information time. Statistics in Medicine 80 (10):1191–1198.
  • Hainmueller, J. 2012. Entropy balancing for causal effects: A multivariate reweighting method to produce balanced samples in observational studies. Political Analysis 200 (1):25–46. doi:10.1093/pan/mpr025.
  • Hashmi, M., J. Rassen, and S. Schneeweiss. 2021. Single-arm oncology trials and the nature of external controls arms. Journal of Comparative Effectiveness Research 10 (12):1053–1066. doi:10.2217/cer-2021-0003.
  • Imai, K., and M. Ratkovic. 2014. Covariate balancing propensity score. Journal of the Royal Statistical Society. Series B, Statistical Methodology 760 (1):243–263. doi:10.1111/rssb.12027.
  • Joshua Chen, Y. H., D. L. DeMets, and K. K. Gordon Lan. 2004. Increasing the sample size when the unblinded interim result is promising. Statistics in Medicine 230 (7):1023–1038. doi:10.1002/sim.1688.
  • Lehmacher, W., and G. Wassmer. 1999. Adaptive sample size calculations in group sequential trials. Biometrics 550 (4):1286–1290. doi:10.1111/j.0006-341X.1999.01286.x.
  • Li Q., Lin J., Liu M., Wu and Liu Y. 2021. Using surrogate endpoints in adaptive designs with delayed treatment effect. Statistics in Biopharmaceutical Research. doi: 10.1080/19466315.2021.1938203
  • Lin, J., M. Gamalo-Siebers, and R. Tiwari. 2018. Propensity score matched augmented controls in randomized clinical trials: A case study. Pharmaceutical Statistics 170 (5):629–647. doi: 10.1002/pst.1879
  • Lin, J., M. Gamalo-Siebers, and R. Tiwari. 2022. Ensuring exchangeability in data-based priors for a Bayesian analysis of clinical trials. Pharmaceutical Statistics 21 (2):327–344. doi:10.1002/pst.2172.
  • Mehta, C. R., and S. J. Pocock. 2011. Adaptive increase in sample size when interim results are promising: A practical guide with examples. Statistics in Medicine 300 (28):3267–3284.
  • Nadeau, K. J., B. J. Anderson, E. G. Berg, J. L. Chiang, H. Chou, K. C. Copeland, T. S. Hannon, T. T.-K. Huang, J. L. Lynch, J. Powell, et al. 2016. Youth-onset type 2 diabetes consensus report: Current status, challenges, and priorities. Diabetes Care 390 (9):1635–1642. doi:10.2337/dc16-1066.
  • Pulgaron, E. R., A. M. Delamater, C. R. Mehta, and S. J. Pocock. 2014. Obesity and type 2 diabetes in children: Epidemiology and treatment. Current Diabetes Reports 140 (8):508. doi:10.1007/s11892-014-0508-y.
  • Tamborlane, W. V., M. Barrientos-Pérez, U. Fainberg, H. Frimer-Larsen, M. Hafez, P. M. Hale, M. Y. Jalaludin, M. Kovarenko, I. Libman, J. L. Lynch, et al. 2019. Liraglutide in children and adolescents with type 2 diabetes. New England Journal of Medicine 3810 (7):637–646. doi:10.1056/NEJMoa1903822.
  • Wang, S. K., and A. A. Tsiatis. 1987. Approximately optimal one-parameter boundaries for group sequential trials. Biometrics 43 (1):193–199. doi:10.2307/2531959.
  • Wei, L.-J. 1978. The adaptive biased coin design for sequential experiments. The Annals of Statistics 60 (1):92–100.

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