Abstract
When analyzing the results of a trial, the primary outcome variable must be kept in clear focus. In the analysis plan, consideration must be given to comparing the characteristics of the subjects, taking account of differences in these characteristics, intention-to-treat analysis, interim analyses and stopping rules, mortality comparisons, composite outcomes, research design including run-in periods, factorial, stratified and crossover designs, number needed to treat, power issues, multivariate modeling, subgroup analysis, competing risks, and hypothesis-generating analyses.
Access this chapter
Tax calculation will be finalised at checkout
Purchases are for personal use only
Similar content being viewed by others
References
Miller AB, Wall C, Baines CJ, Sun P, To T, Narod SA (2014) Twenty five year follow-up for breast cancer incidence and mortality of the Canadian National Breast Screening Study: randomised screening trial. BMJ 348:366–376
Pocock SJ (1997) Group sequential methods in the design and analysis of clinical trials. Biometrika 64:191–200
Haybittle JL (1971) Repeated assessment of results in clinical trials of cancer treatment. Br J Radiol 44:793–797
O’Brien PC, Fleming TR (1979) A multiple testing procedure for clinical trials. Biometrics 35:549–556
Diabetes Control and Complications Trial Research Group, Nathan DM, Genuth S, Lachin J, Cleary P, Crofford O, Davis M, Rand L, Siebert C (1993) The effect of intensive treatment of diabetes on the development and progression of long-term complications in insulin-dependent diabetes mellitus. N Engl J Med 329:977–986
Singh AK, Szczech L, Tang KL, Barnhart H, Sapp S, Wolfson M, Reddan D, Investigators CHOIR (2006) Correction of anemia with epoetin alfa in chronic kidney disease. N Engl J Med 355:2085–2098
Packer M, McMurray JJ, Desai AS, Gong J, Lefkowitz MP, Rizkala AR, Rouleau JL, Shi VC, Solomon SD, Swedberg K et al (2014) Angiotensin-neprilysin inhibition versus enalapril in heart failure. N Engl J Med 371:993–1004
Walter SD, Guyatt GH, Bassler D, Briel M, Ramsay T, Han HD (2019) Randomized trials with provision for early stopping for benefit (or harm): the impact on the estimated treatment effect. Stat Med 38:2524–2543
Frick MH, Elo O, Haapa K, Heinonen OP, Heinsalmi P, Helo P, Huttunen JK, Kaitaniemi P, Koskinen P, Manninen V et al (1987) Helsinki Heart Study: Helsinki Heart Study: primary-prevention trial with gemfibrozil in middle-aged men with dyslipidemia. Safety of treatment, changes in risk factors, and incidence of coronary heart disease. N Engl J Med 317:1237–1245
Austin PC, Fine JP (2017) Accounting for competing risks in randomized controlled trials: a review and recommendation for improvement. Stat Med 36:1203–1209
Wheeler DC, London GM, Parfrey PS et al (2014) Effects of Cinacalcet on atherosclerotic and non-atherosclerotic cardiovascular events in patients receiving hemodialysis: The Evaluation of Cinacalcet HCI Therapy to Lower CardioVascular Events (EVOLVE) trial. I Am Heart Assoc 3(6):e001363. https://doi.org/10.1161/JAHA.114.001363
Freemantle N, Calvert M, Wood J, Eastaugh J, Griffin C (2003) Composite results in randomized trials: Greater precision but with bigger uncertainty? JAMA 289:2554–2559
Packer M, Bristow MR, Cohn JN, Colucci WS, Fowler MB, Gilbert EM, Shusterman NH (1996) The effect of carvedilol on morbidity and mortality in patients with chronic heart failure. U.S. Carvedilol Heart Failure Study Group. N Engl J Med 334:1349–1355
Grizzle JE (1965) The two-period change-over design and its use in clinical trials. Biometrics 21:461–480
Sibbald B, Roberts C (1998) Understanding controlled trials Crossover trials. BMJ 316:1719
Freeman PR (1989) The performance of the two-stage analysis of two-treatment two-period crossover trials. Stat Med 8:1421–1432
SPRINT Research Group, Wright JT Jr, Williamson JD, Whelton PK, Snyder JK et al (2015) A randomized trial of intensive versus standard blood pressure control. N Engl J Med 373:2103–2116
ACCORD Study Group, Cushman WC, Evans GW, Byington RP, Goff DC et al (2010) Effects of intensive blood-pressure control in type 2 diabetes mellitus. N Engl J Med 362:1575–1585
Laupacis A, Sackett DL, Roberts RS (1988) An assessment of clinically useful measures of the consequences of treatment. N Engl J Med 318:1728–1733
Parfrey PS, Block GA, Correa-Rotter R et al (2016) lessons learned from EVOLVe for planning of the future randomized trials in patients on dialysis. Clin J Am Soc Nephrol 11:534–546
Sun X, Briel M, Busse IW et al (2012) Credibility of claims of subgroup effects in randomized controlled trials: systematic review. Brit Med J 344:e1553. https://doi.org/10.1136/bmj.e1553
Parfrey PS, Drueke TB, Block GA et al (2015) The effects of Cinacalcet in younger and older patients on hemodialysis: The Evaluation of Cinacalcet HCI Therapy to Lower CardioVascular Events (EVOLVE) Trial. Clin J Am Soc Nephrol 10:791–799
Hansson L, Zanchetti A, Carruthers SG, Dahlof B, Elmfeldt D, Julius S, Menard J, Rahn KH, Wedel H, Westerling S, HOT Study Group (1998) Effects of intensive blood-pressure lowering and low-dose aspirin in patients with hypertension: major results of the Hypertension Optimal Treatment (HOT) randomised trial. Lancet 351:1755–1762
Author information
Authors and Affiliations
Corresponding author
Editor information
Editors and Affiliations
Rights and permissions
Copyright information
© 2021 Springer Science+Business Media, LLC, part of Springer Nature
About this protocol
Cite this protocol
Foley, R.N., Parfrey, P.S. (2021). Randomized Controlled Trials 2: Analysis. In: Parfrey, P.S., Barrett, B.J. (eds) Clinical Epidemiology. Methods in Molecular Biology, vol 2249. Humana, New York, NY. https://doi.org/10.1007/978-1-0716-1138-8_12
Download citation
DOI: https://doi.org/10.1007/978-1-0716-1138-8_12
Published:
Publisher Name: Humana, New York, NY
Print ISBN: 978-1-0716-1137-1
Online ISBN: 978-1-0716-1138-8
eBook Packages: Springer Protocols