References
- Institute of Medicine. Initial national priorities for comparative effectiveness research. Washington, DC: National Academy Press; 2009. Available online at: http://www.nap.edu/catalog/12648.html, accessed 10 Oct 2014
- Sox HC, Goodman SN. The methods of comparative effectiveness research. Annu Rev Public Health 2012;33:425–45
- Steinbrook R. Health care and the American Recovery and Reinvestment Act. N Engl J Med 2009;360:1057–60
- Patient Protection and Affordable Care Act, 42 U.S.C. § 18001 et seq. (2010)
- Patient-Centered Outcomes Research Institute. National priorities for research and research agenda [Internet]. Washington, DC: PCORI; 2012. Available online at: http://www.pcori.org/assets/PCORI-National-Priorities-and-Research-Agenda-2012-05-21-FINAL.pdf, accessed 10 Oct 2014
- Luce BR, Kramer JM, Goodman SN, et al. Rethinking randomized clinical trials for comparative effectiveness research: the need for transformational change. Ann Intern Med 2009;151:206–9
- Tunis SR, Benner J, McClellan M. Comparative effectiveness research: policy, context, methods development, and research infrastructure. Stat Med 2010;29:1963–76
- Sullivan P, Goldman D. The promise of comparative effectiveness research. JAMA 2011;305:400–1
- Chalkidou K, Tunis S, Whicher D, et al. The role for pragmatic randomized controlled trials (pRCTs) in comparative effectiveness research. Clin Trials 2012;9:436–46
- Zwarenstein M, Treweek S, Gagnier J, et al. Improving the reporting of pragmatic trials: an extension of the CONSORT statement. BMJ 2008;337:a2390
- Thorpe KE, Zwarenstein M, Oxman AD, et al. A pragmatic–explanatory continuum indicator summary (PRECIS): a tool to help trial designers. J Clin Epidemiol 2009;62:464–75
- Davis BR, Cutler JA, Gordon DJ, et al. Rationale and design for the antihypertensive and lipid lowering treatment to prevent heart attack trial (ALLHAT). Am J Hypertens 1996;9:342–60
- Davis BR, Cutler JA, Gordon DJ. Major outcomes in high risk hypertensive patients randomized to angiotensin-converting enzyme inhibitor or calcium channel blocker vs diuretic: the Antihypertensive and Lipid Lowering treatment to prevent Heart Attack Trial (ALLHAT). JAMA 2002;288:2981–97
- Stafford RS, Monti V, Furberg CD, Ma J. Long-term and short-term changes in antihypertensive prescribing by office-based physicians in the United States. Hypertension 2006;48:213–18
- Morton SC, Ellenberg JH. Infusion of statistical science in comparative effectiveness research. Clin Trials 2012;9:6–12
- Food and Drug Administration. Guidance for industry: adaptive design clinical trials for drugs and biologics draft guidance. 2010. Available online at: http:/www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/default.htm, accessed 10 Oct 2014
- Gallo P, Chuang-Stein C, Dragalin V, et al. Adaptive designs in clinical drug development: an executive summary of the PhRMA working group. J Biopharm Stat 2006;16:275–83
- Kairalla JA, Coffey CS, Thomann MA, Muller KE. Adaptive trial designs: a review of barriers and opportunities. Trials 2012;13:145
- Lai TL, Lavori PW, Shih MC. Adaptive trial designs. Ann Rev Pharmacol Toxicol 2012;52:101–10
- Bauer P, Kieser M. Combining different phases in the development of medical treatments within a single trial. Stat Med 1999;18:1833–48
- Lehmacher W, Wassmer G. Adaptive sample size calculations in group sequential trials. Biometrics 1999;55:1286–90
- Bretz F, Koenig F, Brannath W, et al. Adaptive designs for confirmatory clinical trials. Stat Med 2009;28:1181–217
- Whitehead J, Valdes-Marquez E, Lissmats A. A simple two-stage design for quantitative responses with application to a study in diabetic neuropathic pain. Pharm Stat 2009;8:125–35
- Wason JMS, Magirr D, Law M, Jaki T. Some recommendations for multi-arm multi-stage trials. Stat Methods Med Res 2012;Published online December 12, 2012
- Menon S, Chang M. Optimization of adaptive designs: efficiency evaluation. J Biopharm Stat 2012;22:641–61
- Liu Q, Li G, Anderson KM, Lim P. On efficient two-stage adaptive designs for clinical trials with sample size adjustment. J Biopharm Stat 2012;22:617–40
- Connor JT, Elm JJ, Broglio KR. Bayesian adaptive trials offer advantages in comparative effectiveness trials: an example in status epilepticus. J Clin Epidemiol 2013;66:S130–7
- Connor JT, Luce BR, Broglio KR, et al. Do Bayesian adaptive trials offer advantages for comparative effectiveness research? Protocol for the RE-ADAPT study. Clin Trials 2013;10:807–27
- Tsiatis AA, Mehta C. On the inefficiency of the adaptive design for monitoring clinical trials. Biometrika 2003;90:367–78
- Jennison C, Turnbull BW. Efficient group sequential designs when there are several effect sizes under consideration. Stat Med 2006;25:917–32
- Pocock SJ. Group sequential methods in the design and analysis of clinical trials. Biometrika 1977;64:191–9
- O’Brien PC, Fleming TR. A multiple testing procedure for clinical trials. Biometrics 1979;35:549–56
- Whitehead J, Stratton I. Group sequential clinical trials with triangular continuation regions. Biometrics 1983;39:227–36
- Whitehead J, Todd S. The double triangular test in practice. Pharm Stat 2004;3:39–49
- Lan KKG, DeMets DL. Discrete sequential boundaries for clinical trials. Biometrika 1983;70:659–63
- Jennison C, Turnbull BW. Group sequential methods with applications to clinical trials. Boca Raton, FL: Chapman & Hall/CRC; 2000
- Cui L, Hung HMJ, Wang S. Modifications of sample size in group sequential clinical trials. Biometrics 1999;55:853–7
- Wittes J, Brittain E. The role of internal pilot studies in increasing the efficiency of clinical trials. Stat Med 1990;9:65–72
- Mehta CR, Tsiatis AA. Flexible sample size considerations using information-based interim monitoring. Drug Inf J 2001;35:1095–112
- Tsiatis AA. Information-based monitoring of clinical trials. Stat Med 2006;25:3236–44
- Kairalla JA, Muller KE, Coffey CS. Combining an internal pilot with an interim analysis for single degree of freedom tests. Commun Stat Theory Methods 2010;39:3717–38
- Kairalla JA, Coffey CS, Muller KE. Achieving the benefits of both an internal pilot and interim analysis in large and small samples. JSM Proceedings, ENAR Section. Vancouver, BC (August 2010): 2010:5239–52
- Miller F. Variance estimation in clinical studies with interim sample size re-estimation. Biometrics 2005;61:355–61
- Mehta CR, Patel NR. Adaptive, group sequential, and decision theoretic approaches to sample size determination. Stat Med 2006;25:3250–69
- Hamilton M. Rating depressive patients. J Clin Psychiatry 1980;41:21–4
- Thall PF, Simon R, Ellenberg SS. Two-stage selection and testing designs for comparative clinical trials. Biometrika 1988;75:303–10
- Stallard N, Todd S. Sequential designs for phase III clinical trials incorporating treatment selection. Stat Med 2003;22:689–703
- Magirr D, Jaki T, Whitehead J. A generalized Dunnett test for multi-arm multi-stage clinical studies with treatment selection. Biometrika 2012;99:494–501
- Wason JM, Mander AP. Minimizing the maximum expected sample size in two-stage phase II clinical trials with continuous outcomes. J Biopharm Stat 2012;22:836–52