608
Views
0
CrossRef citations to date
0
Altmetric
Articles

Homogeneity assessment for pivotal medical device clinical studies

, , , , , , , , , , , & show all
Pages 749-759 | Received 05 Sep 2018, Accepted 10 Jan 2019, Published online: 05 Sep 2019

References

  • Agresti, A., and J. Hartzel. 2000. Strategies for comparing treatments on a binary response with multi-centre data. Statistics in Medicine 19 (8):1115–1139.
  • Alosh, M., K. Fritsch, M. Huque, K. Mahjoob, G. Pennello, M. Rothmann, E. Russek-Cohen, F. Smith, S. Wilson, and L. Yue. 2015. Statistical considerations on subgroup analysis in clinical trials. Statistics in Biopharmaceutical Research 7 (4):286–304. doi:10.1080/19466315.2015.1077726.
  • Arshad, A., A. J. Moss, E. Foster, L. Padeletti, A. Barsheshet, I. Goldenberg, H. Greenberg, W. J. Hall, S. McNitt, W. Zareba, et al.; MADIT-CRT Executive Committee. 2011. Cardiac resynchronization therapy is more effective in women than in men: the MADIT-CRT (Multicenter Automatic Defibrillator Implantation Trial with Cardiac Resynchronization Therapy) trial. Journal of the American College of Cardiology 57 (7):813–820. doi:10.1016/j.jacc.2010.06.061.
  • Austin, P. 2011. An introduction to propensity score methods for reducing the effects of confounding in observational studies. Multivariate Behavioral Research 46 (3):399–424. doi:10.1080/00273171.2011.568786.
  • Bradburn, M. J., J. J. Deeks, J. A. Berlin, and L. A. Russel. 2007. Much ado about nothing: a comparison of the performance of meta-analytical methods with rare events. Statistics in Medicine 26:53–77. doi:10.1002/(ISSN)1097-0258.
  • Chen, J., and H. Quan. 2016. Multiregional clinical trials for simultaneous global new drug development. New York, NY: CRC Press.
  • Cox, D. R. 1984. Interaction. International Statistical Review 52:1–31. doi:10.2307/1403235.
  • Cuzick, J. 2005. Forest plots and the interpretation of subgroups. The Lancet 365:1308. doi:10.1016/S0140-6736(05)61026-4.
  • Dixon, D., and R. Simon. 1991. Bayesian subset analysis. Biometrics 47 (3):871–881. doi:10.2307/2532645.
  • Food and Drug Administration (FDA). 2013. Guidance for industry, clinical investigators, and food and drug administration staff: Design considerations for pivotal clinical investigations for medical devices. https://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/ucm373766.pdf
  • Food and Drug Administration (FDA). 2014. Evaluation of sex-specific data in medical device clinical studies. https://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/UCM283707.pdf
  • Food and Drug Administration (FDA). 2017. Evaluation and reporting of age-, race-, and ethnicity-specific data in medical device clinical studies. https://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/UCM507278.pdf
  • Gail, M., and R. Simon. 1985. Tests for qualitative interactions between treatment effects and patient subsets. Biometrics 41:361–372.
  • Grizzle, J. E. 1987. Letter to the editor. Controlled Clinical Trials 8:392–393.
  • Hsu, Y., J. Zalkikar, and R. Tiwari. 2019. Hierarchical Bayes approach for subgroup analysis. Statistical Methods in Medical Research vol. 28(1): 275-288.
  • Huang, L., J. Zalkikar, and R. Tiwari. 2011. A likelihood ratio test based method for signal detection with application to FDA’s drug safety data. Journal of the American Statistical Association 106 (496):1230–1241. doi:10.1198/jasa.2011.ap10243.
  • International Conference on Harmonisation (ICH). 1998. E9: Statistical principles for clinical trials. http://www.fda.gov/cber/gdlns/ichclinical.txt
  • International Conference on Harmonisation (ICH). 2017. E17: General principles for planning and design of multi-regional clinical trials. http://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/Guidelines/Efficacy/E17/E17EWG_Step4_2017_1116.pdf
  • Kahan, B. C. 2014. Accounting for centre-effects in multicentre trials with a binary outcome – when, why, and how? BMC Medical Research Methodology 14:20. doi:10.1186/1471-2288-14-20.
  • Kahan, B. C., and T. P. Morris. 2012a. Improper analysis of trials randomised using stratified blocks or minimisation. Statistics in Medicine 31 (4):328–340. doi:10.1002/sim.4431.
  • Kahan, B. C., and T. P. Morris. 2012b. Reporting and analysis of trials using stratified randomisation in leading medical journals: review and reanalysis. BMJ 345:e5840. doi:10.1136/bmj.e5840.
  • Kahan, B. C., and T. P. Morris. 2013. Analysis of multicentre trials with continuous outcomes: when and how should we account for centre effects? Statistics in Medicine 32 (7):1136–1149. doi:10.1002/sim.v32.7.
  • Li, H., V. Mukhi, N. Lu, Y. Xu, and L. Yue. 2016. A note on good practice of objective propensity score design for premarket nonrandomized medical device studies with an example. Statistics in Biopharmaceutical Research 8 (3):282–286. doi:10.1080/19466315.2016.1148071.
  • Lipsitz, S. R., K. B. G. Dear, N. M. Laird, and G. Molenberghs. 1998. Tests for homogeneity of the risk difference when data are sparse. Biometrics 54:148–160. doi:10.2307/2534003.
  • Najjar, S. S. 2014. Late bleeding and neurological sequelae after HeartMate II left ventricular assist device: Risk factors from the prequel. Journal of the American College of Cardiology 63:889–890. doi:10.1016/j.jacc.2013.11.018.
  • Pennello, G., and L. Thompson. 2008. Experience with reviewing Bayesian medical device trials. Journal of Biopharmaceutical Statistics 18 (1):81–115. doi:10.1080/10543400701668274.
  • Permutt, T. 2003. Probability models and computational models for ANOVA in multicenter clinical trials. Journal of Biopharmaceutical Statistics 13 (3):495–505. doi:10.1081/BIP-120022769.
  • Peto, R. 1982. Statistical aspects of cancer trials. In Treatment of cancer, ed. K. E. Halnan, 867–871. London: Chapman and Hall.
  • Piantadosi, S., and M. Gail. 1993. A comparison of the power of two tests for qualitative interactions. Statistics in Medicine 12:1239–1248.
  • Potthoff, R., B. Peterson, and S. George. 2001. Detecting treatment-by-centre interaction in multi-centre clinical trials. Statistics in Medicine 20:193–213.
  • Rosenbaum, P. R., and D. B. Rubin. 1983. The central role of the propensity score in observational studies for causal effects. Biometrika 70:41–55. doi:10.1093/biomet/70.1.41.
  • Rosenbaum, P. R., and D. B. Rubin. 1984. Reducing bias in observational studies using subclassification on the propensity score. Journal of the American Statistical Association 79:516–524. doi:10.1080/01621459.1984.10478078.
  • Rothwell, P. 2005. Subgroup analysis in randomised controlled trials: importance, indications, and interpretation. The Lancet 365:176–186. doi:10.1016/S0140-6736(05)17709-5.
  • Scott, P., and G. Campbell. 1998. Interpretation of subgroup analyses in medical device clinical trials. Drug Information Journal 32:213–220. doi:10.1177/009286159803200129.
  • Senn, S. 1998. Some controversies in planning and analysing multi-centre trails. Statistics in Medicine 17:1753–1765. doi:10.1002/(SICI)1097-0258(19980815/30)17:15/16<1753::AID-SIM977>3.0.CO;2-X.
  • Simon, R. 2008. Subgroup analyses. Wiley Encyclopedia of Clinical Trials.
  • Sneha, R., M. L. Zych, M. Jane, F. Billia, and V. Rao. 2017. Gender differences in Left Ventricular Assist Device (LVAD) support. International Journal of Surgery Research and Practice 4:53. doi:10.23937/2378-3397/1410053.
  • Zareba, W., H. Klein, I. Cygankiewicz, W. J. Hall, S. McNitt, M. Brown, D. Cannom, J. P. Daubert, M. Eldar, M. R. Gold, et al.; MADIT-CRT Investigators. 2011. Effectiveness of cardiac resynchronization therapy by QRS morphology in the Multicenter Automatic Defibrillator Implantation Trial-Cardiac Resynchronization Therapy (MADIT-CRT). Circulation 123 (10):1061–1072. doi:10.1161/CIRCULATIONAHA.110.960898.
  • Zhang, L., H. Yang, and I. Cho. 2009. Test homogeneity of risk difference across subgroups in clinical trials. Journal of Biopharmaceutical Statistics 19 (1):67–76. doi:10.1080/10543400802527874.

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.