Assessing the Impacts of Cluster Effects and Covariate Imbalance in Cluster Randomized Equivalence Trials

References

• Acuna, S. A., Dossa, F., and Baxter, N. (2020), “Meta-Analysis of Noninferiority and Equivalence Trials: Ignoring Trial Design Leads to Differing and Possibly Misleading Conclusions,” Journal of Clinical Epidemiology, 127, 134–141. DOI: 10.1016/j.jclinepi.2020.05.034.
   PubMed Web of Science ®Google Scholar
• Anderson, S., and Hauck, W. W. (1983), “A New Procedure for Testing Equivalence in Comparative Bioavailability and Other Clinical Trials,” Communications in Statistics - Theory and Methods, 12, 2663–2692. DOI: 10.1080/03610928308828634.
   Web of Science ®Google Scholar
• Bates, D., Mächler, M., Bolker, B., and Walker, S. (2015), “Fitting Linear Mixed-Effects Models Using Lme4,” Journal of Statistical Software, 67, 1– 48. DOI: 10.18637/jss.v067.i01.
   Web of Science ®Google Scholar
• Berger, R., and Hsu, J. (1996), “Bioequivalence Trials, Intersection-Union Tests and Equivalence Confidence Sets,” Statistical Science, 11, 283–319. DOI: 10.1214/ss/1032280304.
   Web of Science ®Google Scholar
• Bowalekar, S. K. (2011), “Non-Inferiority and Equivalence Trials: Need for a Standardized Process,” Perspectives in Clinical Research, 2, 115–118. DOI: 10.4103/2229-3485.86868.
   PubMedGoogle Scholar
• Brown, H., and Prescott, R. (2015), Applied Mixed Models in Medicine, Chichester: Wiley.
   Google Scholar
• Campbell, M. J., Donner, A., and Klar, N. (2007), “Developments in Cluster Randomized Trials and Statistics in Medicine,” Statistics in Medicine, 26, 2–19. DOI: 10.1002/sim.2731.
   PubMed Web of Science ®Google Scholar
• Campbell, M. J., and Walters, S. J. (2014), How to Design, Analyse and Report Cluster Randomized Trials in Medicine and Health Related Research, Chichester: Wiley.
   Google Scholar
• Chan, I. S. (2004), “Noninferiority and Equivalence Trials,” Journal of Biopharmaceutical Statistics, 14, 261–262. DOI: 10.1081/BIP-120037177.
   PubMedGoogle Scholar
• Chervoneva, I., Hyslop, T., and Hauck, W. W. (2007), “A Multivariate Test for Population Bioequivalence,” Statistics in Medicine, 26, 1208–1223. DOI: 10.1002/sim.2605.
   PubMed Web of Science ®Google Scholar
• Ciolino, J. D., Martin, R. H., Zhao, W., Hill, M. D., Jauch, E. C., and Palesch, Y. Y. (2011), “Measuring Continuous Baseline Covariate Imbalances in Clinical Trial Data,” Statistical Methods in Medical Research, 24, 255–272. DOI: 10.1177/0962280211416038.
   PubMedGoogle Scholar
• Ciolino, J. D., Palac, H. L., Yang, A., Vaca, M., and Belli, H. M. (2019), “Ideal Vs. Real: A Systematic Review on Handling Covariates in Randomized Controlled Trials,” BMC Medical Research Methodology, 19, 1–14. DOI: 10.1186/s12874-019-0787-8.
   PubMedGoogle Scholar
• Committee for Proprietary Medicinal Products. (2004), “Committee for Proprietary Medicinal Products (Cpmp) Points to Consider on Adjustment for Baseline Covariates,” Statistics in Medicine, 23, 701–709.
   PubMed Web of Science ®Google Scholar
• Dasgupta, A., Lawson, K. A., and Wilson, J. P. (2010), “Evaluating Equivalence and Noninferiority Trials,” American Journal of Health-System Pharmacy, 67, 1337–1343. DOI: 10.2146/ajhp090507.
   PubMed Web of Science ®Google Scholar
• Dunn, D. T., Copas, A. J., and Brocklehurst, P. (2018), “Superiority and Non-Inferiority: Two Sides of the Same Coin?” Trials, 19, 1–5. DOI: 10.1186/s13063-018-2885-z.
   Google Scholar
• Eldridge, S. M., Ashby, D., Feder, G. S., Rudnicka, A. R., and Ukoumunne, O. C. (2004), “Lessons for Cluster Randomized Trials in the Twenty-First Century: A Systematic Review of Trials in Primary Care,” Clinical Trials, 1, 80–90. DOI: 10.1191/1740774504cn006rr.
   PubMedGoogle Scholar
• Feng, Z., Diehr, P., Peterson, A., and McLerran, D. (2001), “Selected Statistical Issues in Group Randomized Trials,” Annual Review of Public Health, 22, 167–187. DOI: 10.1146/annurev.publhealth.22.1.167.
   PubMed Web of Science ®Google Scholar
• Flight, L., and Julious, S. A. (2016), “Practical Guide to Sample Size Calculations: Non-Inferiority and Equivalence Trials,” Pharmaceutical Statistics, 15, 80–89. DOI: 10.1002/pst.1716.
   PubMed Web of Science ®Google Scholar
• Ganju, J., and Rom, D. (2017), “Non-Inferiority Versus Superiority Drug Claims: The (Not So) Subtle Distinction,” Trials, 18, 1–5. DOI: 10.1186/s13063-017-2024-2.
   PubMedGoogle Scholar
• Ghosh, P., Nahum-Shani, I., Spring, B., and Chakraborty, B. (2020), “Noninferiority and Equivalence Tests in Sequential, Multiple Assignment, Randomized Trials (Smarts),” Psychological Methods, 25, 182–205. DOI: 10.1037/met0000232.
   PubMed Web of Science ®Google Scholar
• Hua, S. Y., Xu, S., and D’Agostino, R. B. Sr.(2015), “Multiplicity Adjustments in Testing for Bioequivalence,” Statistics in Medicine, 34, 215–231. DOI: 10.1002/sim.6247.
   PubMed Web of Science ®Google Scholar
• Irena, A. H., Bahwere, P., Owino, V. O., Diop, E. I., Bachmann, M. O., Mbwili-Muleya, C., Dibari, F., Sadler, K., and Collins, S. (2015), “Comparison of the Effectiveness of a Milk-Free Soy-Maize-Sorghum-Based Ready-to-Use Therapeutic Food to Standard Ready-to-Use Therapeutic Food with 25% Milk in Nutrition Management of Severely Acutely Malnourished Zambian Children: An Equivalence Non-Blinded Cluster Randomised Controlled Trial,” Maternal and Child Nutrition, 11, 105–119.
   PubMed Web of Science ®Google Scholar
• Ivers, N. M., Halperin, I. J., Barnsley, J., Grimshaw, J. M., Shah, B. R., Tu, K., Upshur, R., and Zwarenstein, M. (2012), “Allocation Techniques for Balance at Baseline in Cluster Randomized Trials: A Methodological Review,” Trials, 13, 1–9. DOI: 10.1186/1745-6215-13-120.
   PubMed Web of Science ®Google Scholar
• Jaffar, S., Amuron, B., Foster, S., Birungi, J., Levin, J., Namara, G., Nabiryo, C., Ndembi, N., Kyomuhangi, R., Opio, A., Bunnell, R., Tappero, J. W., Mermin, J., Coutinho, A., and Grosskurth, H. (2009), “Rates of Virological Failure in Patients Treated in a Home-Based Versus a Facility-Based HIV-Care Model in Jinja, Southeast Uganda: A Cluster-Randomised Equivalence Trial,” The Lancet, 374, 2080–2089. DOI: 10.1016/S0140-6736(09)61674-3.
   PubMed Web of Science ®Google Scholar
• Kahan, B. C., Jairath, V., Doré, C. J., and Morris, T. P. (2014), “The Risks and Rewards of Covariate Adjustment in Randomized Trials: An Assessment of 12 Outcomes from 8 Studies,” Trials, 15, 139. DOI: 10.1186/1745-6215-15-139.
   PubMed Web of Science ®Google Scholar
• Kerai, S. (2017), “Equivalence and Non-Inferiority Trials in a Snapshot,” Journal of Ayub Medical College Abbottabad, 29, 371–372.
   PubMedGoogle Scholar
• Kuznetsova, A., Brockhoff, P., and Christensen, R. (2017), “Lmertest Package: Tests in Linear Mixed Effects Models,” Journal of Statistical Software, 82, 1–26. DOI: 10.18637/jss.v082.i13.
   Web of Science ®Google Scholar
• Lee, K. J., and Thompson, S. G. (2005), “The Use of Random Effects Models to Allow for Clustering in Individually Randomized Trials,” Clinical Trials, 2, 163–173. DOI: 10.1191/1740774505cn082oa.
   PubMed Web of Science ®Google Scholar
• Leon, A. C., Demirtas, H., Li, C., and Hedeker, D. (2013), “Subject-Level Matching for Imbalance in Cluster Randomized Trials with a Small Number of Clusters,” Pharmaceutical Statistics, 12, 268–274. DOI: 10.1002/pst.1580.
   PubMed Web of Science ®Google Scholar
• Luke, S. G. (2017), “Evaluating Significance in Linear Mixed-Effects Models in R,” Behavior Research Methods, 49, 1494–1502. DOI: 10.3758/s13428-016-0809-y.
   PubMed Web of Science ®Google Scholar
• Mayer, J. M., Childs, J. D., Neilson, B. D., Chen, H., Koppenhaver, S. L., and Quillen, W. S. (2016), “Effect of Lumbar Progressive Resistance Exercise on Lumbar Muscular Strength and Core Muscular Endurance in Soldiers,” Military Medicine, 181, e1615–e1622. DOI: 10.7205/MILMED-D-15-00543.
   PubMed Web of Science ®Google Scholar
• Moerbeek, M., and Teerenstra, S. (2015), Power Analysis of Trials with Multilevel Data, Boca Raton, FL: CRC Press.
   Google Scholar
• Moerbeek, M., and van Schie, S. (2016), “How Large Are the Consequences of Covariate Imbalance in Cluster Randomized Trials: A Simulation Study with a Continuous Outcome and a Binary Covariate at the Cluster Level,” BMC Medical Research Methodology, 16, 1–10. DOI: 10.1186/s12874-016-0182-7.
   PubMed Web of Science ®Google Scholar
• Moher, D., Hopewell, S., Schulz, K. F., Montori, V., Gøtzsche, P. C., Devereaux, P. J., Elbourne, D., Egger, M., and Altman, D. G. (2010), “Consort 2010 Explanation and Elaboration: Updated Guidelines For reporting Parallel Group Randomised Trials,” Journal of Clinical Epidemiology, 63, e1–e37. DOI: 10.1016/j.jclinepi.2010.03.004.
   PubMed Web of Science ®Google Scholar
• Murray, D. M., Pals, S. L., George, S. M., Kuzmichev, A., Lai, G. Y., Lee, J. A., Myles, R. L., and Nelson, S. M. (2018), “Design and Analysis of Group-Randomized Trials in Cancer: A Review of Current Practices,” Preventive Medicine, 111, 241–247. DOI: 10.1016/j.ypmed.2018.03.010.
   PubMed Web of Science ®Google Scholar
• Murray, D. M., Varnell, S. P., and Blitstein, J. L. (2004), “Design and Analysis of Group-Randomized Trials: A Review of Recent Methodological Developments,” American Journal of Public Health, 94, 423–432. DOI: 10.2105/ajph.94.3.423.
   PubMed Web of Science ®Google Scholar
• Pallmann, P., and Jaki, T. (2017), “Simultaneous Confidence Regions for Multivariate Bioequivalence,” Statistics in Medicine, 36, 4585–4603. DOI: 10.1002/sim.7446.
   PubMed Web of Science ®Google Scholar
• Phillips, K. F. (1990), “Power of the Two One-Sided Tests Procedure in Bioequivalence,” Journal of Pharmacokinetics and Biopharmaceutics, 18, 137–144. DOI: 10.1007/BF01063556.
   PubMedGoogle Scholar
• Piaggio, G., Carroli, G., Villar, J., Pinol, A., Bakketeig, L., Lumbiganon, P., Bergsjø, P., Al-Mazrou, Y., Ba’aqeel, H., Miguel Belizán, J., Farnot, U., & Berendes, H. (2001), “Methodological Considerations on the Design and Analysis of an Equivalence Stratified Cluster Randomization Trial,” Statistics in Medicine, 20, 401–416.
   PubMed Web of Science ®Google Scholar
• Pocock, S. J., Assmann, S. E., Enos, L. E., and Kasten, L. E. (2002), “Subgroup Analysis, Covariate Adjustment and Baseline Comparisons in Clinical Trial Reporting: Current practice and problems,” Statistics in Medicine, 21, 2917–2930. DOI: 10.1002/sim.1296.
   PubMed Web of Science ®Google Scholar
• Powers, J. H. (2008), “Noninferiority and Equivalence Trials: Deciphering ‘Similarity’ of Medical Interventions,” Statistics in Medicine, 27, 343–352. DOI: 10.1002/sim.3138.
   PubMed Web of Science ®Google Scholar
• Ranstam, J., and Cook, J. A. (2017), “Non-Inferiority and Equivalence Trials,” The British Journal of Surgery, 104, 1578–1579.
   PubMed Web of Science ®Google Scholar
• Rief, W., and Hofmann, S. G. (2019), “The Limitations of Equivalence and Non-Inferiority Trials,” Psychological Medicine, 49, 349–350. DOI: 10.1017/S0033291718002891.
   PubMed Web of Science ®Google Scholar
• Rutterford, C., Copas, A., and Eldridge, S. (2015), “Methods for Sample Size Determination in Cluster Randomized Trials,” International Journal of Epidemiology, 44, 1051–1067. DOI: 10.1093/ije/dyv113.
   PubMed Web of Science ®Google Scholar
• Schober, P., and Vetter, T. R. (2020), “Noninferiority and Equivalence Trials in Medical Research,” Anesthesia and Analgesia, 131, 208–209. DOI: 10.1213/ANE.0000000000004783.
   PubMed Web of Science ®Google Scholar
• Schuirmann, D. (1987), “A Comparison of the Two One-Sided Tests Procedure and the Power Approach for Assessing the Equivalence of Average Bioavailability,” Journal of Pharmacokinetics and Biopharmaceutics, 15, 657–680. DOI: 10.1007/BF01068419.
   PubMedGoogle Scholar
• Schuirmann, D. L. (1981), “On Hypothesis-Testing to Determine If the Mean of a Normal-Distribution Is Contained in a Known Interval,” Biometrics, 37, 617–617.
   Web of Science ®Google Scholar
• Stefanos, R., Graziella, D., and Giovanni, T. (2020), “Methodological Aspects of Superiority, Equivalence, and Non-Inferiority Trials,” Internal and Emergency Medicine, 15, 1085–1091. DOI: 10.1007/s11739-020-02450-9.
   PubMed Web of Science ®Google Scholar
• Turner, E. L., Li, F., Gallis, J. A., Prague, M., and Murray, D. M. (2017a), “Review of Recent Methodological Developments in Group-Randomized Trials: Part 1-Design,” American Journal of Public Health, 107, 907–915. DOI: 10.2105/AJPH.2017.303706.
   PubMed Web of Science ®Google Scholar
• Turner, E. L., Perel, P., Clayton, T., Edwards, P., Hernández, A. V., Roberts, I., Shakur, H., and Steyerberg, E. W. (2012), “Covariate Adjustment Increased Power in Randomized Controlled Trials: An Example in Traumatic Brain Injury,” Journal of Clinical Epidemiology, 65, 474–481. DOI: 10.1016/j.jclinepi.2011.08.012.
   PubMed Web of Science ®Google Scholar
• Turner, E. L., Prague, M., Gallis, J. A., Li, F., and Murray, D. M. (2017b), “Review of Recent Methodological Developments in Group-Randomized Trials: Part 2-Analysis,” American Journal of Public Health, 107, 1078–1086. DOI: 10.2105/AJPH.2017.303707.
   PubMed Web of Science ®Google Scholar
• Wang, W. W., Mehrotra, D. V., Chan, I. S., and Heyse, J. F. (2006), “Statistical Considerations for Noninferiority/Equivalence Trials in Vaccine Development,” Journal of Biopharmaceutical Statistics, 16, 429–441. DOI: 10.1080/10543400600719251.
   PubMed Web of Science ®Google Scholar
• Westlake, W. J. (1981), “Bioequivalence Testing – a Need to Rethink – Reply,” Biometrics, 37, 591–593.
   Web of Science ®Google Scholar
• Wright, N., Ivers, N., Eldridge, S., Taljaard, M., and Bremner, S. (2015), “A Review of the Use of Covariates in Cluster Randomized Trials Uncovers Marked Discrepancies between Guidance and Practice,” Journal of Clinical Epidemiology, 68, 603–609. DOI: 10.1016/j.jclinepi.2014.12.006.
   PubMed Web of Science ®Google Scholar
• Yang, S., Starks, M. A., Hernandez, A. F., Turner, E. L., Califf, R. M., O’Connor, C. M., Mentz, R. J., and Roy Choudhury, K. (2020), “Impact of Baseline Covariate Imbalance on Bias in Treatment Effect Estimation in Cluster Randomized Trials: Race as an Example,” Contemporary Clinical Trials, 88, 105775. DOI: 10.1016/j.cct.2019.04.016.
   PubMed Web of Science ®Google Scholar