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Research Article

Power and sample size calculation for the win odds test: application to an ordinal endpoint in COVID-19 trials

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References

  • Bebu, I., and J. M. Lachin. 2016. Large sample inference for a win ratio analysis of a composite outcome based on prioritized components. Biostatistics 17 (1):178–187. doi:10.1093/biostatistics/kxv032.
  • Beigel, J. H., K. M. Tomashek, L. E. Dodd, A. K. Mehta, B. S. Zingman, A. C. Kalil, E. Hohmann, H. Y. Chu, A. Luetkemeyer, and S. Kline, al. 2020. Remdesivir for the treatment of Covid-19 — Final report. New England Journal of Medicine 383 (19):1813–1826. doi:10.1056/NEJMoa2007764.
  • Brunner, E., M. Vandemeulebroecke, and M. Tobias. 2021. Win odds: An adaptation of the win ratio to include ties. Statistics in Medicine 40 (14):3367–3384. doi:10.1002/sim.8967.
  • Buyse, M. 2010. Generalized pairwise comparisons of prioritized outcomes in the two-sample problem. Statistics in Medicine 29 (30):3245–3257. doi:10.1002/sim.3923.
  • Carr, G. J., K. B. Hafner, and G. G. Koch. 1989. Analysis of rank measures of association for ordinal data from longitudinal studies. Journal of the American Statistical Association 84 (407):797–804. doi:10.1080/01621459.1989.10478840.
  • Casey, J. D., N. J. Johnson, M. W. Semler, S. P. Collins, N. R. Aggarwal, R. G. Brower, S. Y. Chang, J. Eppensteiner, M. Filbin, K. W. Gibbs, et al. 2020. Rationale and design of ORCHID: A randomized placebo-controlled trial of hydroxychloroquine for adults hospitalized with COVID-19. Annals of the American Thoracic Society 17 (9):1144–1153. doi:10.1513/AnnalsATS.202005-478SD.
  • Dodd, L. E., D. Follmann, J. Wang, F. Koenig, L. L. Korn, C. Schoergenhofer, M. Proschan, S. Hunsberger, T. Bonnett, M. Makowski, et al. 2020. Endpoints for randomized controlled clinical trials for COVID-19 treatments. Clinical Trials 17 (5):472–482. doi:10.1177/1740774520939938.
  • Dong, G., D. C. Hoaglin, J. Qiu, R. A. Matsouaka, Y. W. Chang, J. Wang, and M. Vandemeulebroecke. 2020. The win ratio: On interpretation and handling of ties. Statistics in Biopharmaceutical Research 12 (1):99–106. doi:10.1080/19466315.2019.1575279.
  • FDA. 2020. COVID-19: Developing drugs and biological products for treatment or prevention guidance for industry. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/covid-19-developing-drugs-and-biological-products-treatment-or-prevention
  • Finkelstein, D. M., and D. A. Schoenfeld. 1999. Combining mortality and longitudinal measures in clinical trials. Statistics in Medicine 18 (11):1341–1354. doi:10.1002/(SICI)1097-0258(19990615)18:11<1341::AID-SIM129>3.0.CO;2-7.
  • Fligner, M. A., and G. E. Policello. 1981. Robust rank procedures for the Behrens-Fisher problem. Journal of the American Statistical Association 76 (373):162–168. doi:10.1080/01621459.1981.10477623.
  • Gasparyan, S. B., F. Folkvaljon, O. Bengtsson, J. Buenconsejo, and G. G. Koch. 2021. Adjusted win ratio with stratification: Calculation methods and interpretation. Statistical Methods in Medical Research 30 (2):580–611. doi:10.1177/0962280220942558.
  • Harrell, F. E. 2015. Regression modeling strategies: With applications to linear models, logistic and ordinal regression, and survival analysis, vol. 3. New York: Springer.
  • Hoeffding, W. 1948. A class of statistics with asymptotically normal distribution. The Annals of Mathematical Statistics 19 (3):293–325. doi:10.1214/aoms/1177730196.
  • Hollander, M., D. A. Wolfe, and E. Chicken. 2013. Nonparametrics: Statistical methods based on ranks. Hoboken, NJ: John Wiley & Sons.
  • ICH. 2020. E9 (R1) addendum on estimands and sensitivity analysis in clinical trials to the guideline on statistical principles for clinical trials. https://www.ema.europa.eu/en/documents/scientific-guideline/ich-e9-r1-addendum-estimands-sensitivity-analysis-clinical-trials-guideline-statistical-principles_en.pdf
  • Kawaguchi, A., and G. G. Koch. 2015. sanon: An R package for stratified analysis with nonparametric covariable adjustment. Journal of Statistical Software 67 (9):9. doi:10.18637/jss.v067.i09.
  • Kawaguchi, A., G. G. Koch, and X. Wang. 2011. Stratified multivariate Mann–Whitney estimators for the comparison of two treatments with randomization based covariance adjustment. Statistics in Biopharmaceutical Research 3 (2):217–231. doi:10.1198/sbr.2010.10007.
  • Koch, G. G., C. M. Tangen, J. ‐. W. Jung, and I. A. Amara. 1998. Issues for covariance analysis of dichotomous and ordered categorical data from randomized clinical trials and non‐parametric strategies for addressing them. Statistics in Medicine 17 (15–16):1863–1892. doi:10.1002/(SICI)1097-0258(19980815/30)17:15/16<1863::AID-SIM989>3.0.CO;2-M.
  • Kosiborod, M., O. Berwanger, G. G. Koch, F. Martinez, O. Mukhtar, S. Verma, V. Chopra, A. Javaheri, P. Ambery, S. B. Gasparyan, et al. 2021. Effects of dapagliflozin on prevention of major clinical events and recovery in patients with respiratory failure because of COVID‐19: Design and rationale for the DARE‐19 study. Diabetes, Obesity and Metabolism 23 (4):886–896.
  • Lehmann, E. L., and H. J. M. D’Abrera. 1975. Nonparametrics: Statistical methods based on ranks. San Francisco, CA: Holden-Day.
  • Mendes, D., C. Alves, and F. Batel-Marques. 2017. Number needed to treat (NNT) in clinical literature: An appraisal. BMC Medicine 15 (1):1–13. doi:10.1186/s12916-017-0875-8.
  • Natanegera, F., N. Zariffa, J. Buenconsejo, R. Liao, F. Cooner, D. Lakshminarayanan, S. Ghosh, and M. Gamalo. 2021. Statistical opportunities to accelerate development for COVID-19 therapeutics. Statistics in Biopharmaceutical Research 1–17. doi:10.1080/19466315.2020.1865195.
  • O’Brien, R. G., and J. M. Castelloe. 2006. Exploiting the link between the Wilcoxon–Mann–Whitney test and a simple odds statistic. Cary, NC: SAS Institute Inc. http://www2.sas.com/proceedings/sugi31/209-31.pdf.
  • Packer, M. 2016. Development and evolution of a hierarchical clinical composite end point for the evaluation of drugs and devices for acute and chronic heart failure: A 20-year perspective. Circulation 134 (21):1664–1678. doi:10.1161/CIRCULATIONAHA.116.023538.
  • Peng, L. 2020. The use of the win odds in the design of non-inferiority clinical trials. Journal of Biopharmaceutical Statistics 30 (5):941–946. doi:10.1080/10543406.2020.1757690.
  • Pocock, S. J., C. A. Ariti, T. J. Collier, and D. Wang. 2012. The win ratio: A new approach to the analysis of composite endpoints in clinical trials based on clinical priorities. European Heart Journal 33 (2):176–182. doi:10.1093/eurheartj/ehr352.
  • R Core Team. 2019. R: A language and environment for statistical computing, version 4.0.3. Vienna, Austria: R Foundation for Statistical Computing. https://www.R-project.org.
  • Rauch, G., A. Jahn-Eimermacher, W. Brannath, and M. Kieser. 2014. Opportunities and challenges of combined effect measures based on prioritized outcomes. Statistics in Medicine 33 (7):1104–1120. doi:10.1002/sim.6010.
  • SAS Institute Inc. 2018. SAS/STAT®: 15.1 user’s guide. Cary, NC: SAS Institute Inc.
  • Stokes, M. E., C. S. Davis, and G. G. Koch. 2012. Categorical data analysis using SAS®. 3rd ed. Cary, NC: SAS Institute Inc. http://support.sas.com/rnd/app/stat/cat/edition3/samples
  • Van der Vaart, A. W. 2000. Asymptotic statistics, vol. 3. Cambridge, UK: Cambridge University Press.
  • Verbeeck, J., V. Deltuvaite-Thomas, B. Berckmoes, T. Burzykowski, M. Aerts, O. Thas, M. Buyse, and G. Molenberghs. 2021. Unbiasedness and efficiency of non-parametric and UMVUE estimators of the probabilistic index and related statistics. Statistical Methods in Medical Research 30 (3):747–768.
  • Whitehead, J. 1993. Sample size calculations for ordered categorical data. Statistics in Medicine 12 (24):2257–2271. doi:10.1002/sim.4780122404.
  • WHO, R&D. 2020. WHO R&D blueprint novel coronavirus (COVID-19) therapeutic trial synopsis. WHO. https://www.who.int/blueprint/priority-diseases/key-action/COVID-19_Treatment_Trial_Design_Master_Protocol_synopsis_Final_18022020.pdf

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