Skip to main content Accessibility help
×
Home
Hostname: page-component-99c86f546-7mfl8 Total loading time: 0.895 Render date: 2021-12-07T06:51:05.173Z Has data issue: true Feature Flags: { "shouldUseShareProductTool": true, "shouldUseHypothesis": true, "isUnsiloEnabled": true, "metricsAbstractViews": false, "figures": true, "newCiteModal": false, "newCitedByModal": true, "newEcommerce": true, "newUsageEvents": true }

Chapter 9 - Alternatives to Randomized Trials for Estimating Treatment Effects

Published online by Cambridge University Press:  02 May 2020

Thomas B. Newman
Affiliation:
University of California, San Francisco
Michael A. Kohn
Affiliation:
University of California, San Francisco
Get access

Summary

We said in Chapter 8 that randomized blinded trials are the best way to estimate treatment effects because they minimize the potential for confounding, co-interventions, and bias, thus maximizing the strength of causal inference. However, sometimes observational studies can be attractive alternatives to randomized trials because they may be more feasible, ethical, or elegant. Of course, the issue of inferring causality from observational studies is a major topic in classical risk factor epidemiology. In this chapter, we focus on observational studies of treatments rather than risk factors, describing methods of reducing or assessing confounding that are particularly applicable to such studies.

Type
Chapter
Information
Evidence-Based Diagnosis
An Introduction to Clinical Epidemiology
, pp. 231 - 249
Publisher: Cambridge University Press
Print publication year: 2020

Access options

Get access to the full version of this content by using one of the access options below. (Log in options will check for institutional or personal access. Content may require purchase if you do not have access.)

References

Ye, Z, Song, H. Antioxidant vitamins intake and the risk of coronary heart disease: meta-analysis of cohort studies. Eur J Cardiovasc Prev Rehabil. 2008;15(1):2634.CrossRefGoogle ScholarPubMed
Curtis, AJ, Bullen, M, Piccenna, L, McNeil, JJ. Vitamin E supplementation and mortality in healthy people: a meta-analysis of randomised controlled trials. Cardiovasc Drugs Ther. 2014;28(6):563–73.CrossRefGoogle ScholarPubMed
Warram, JH, Laffel, LM, Valsania, P, Christlieb, AR, Krolewski, AS. Excess mortality associated with diuretic therapy in diabetes mellitus. Arch Intern Med. 1991;151(7):1350–6.CrossRefGoogle ScholarPubMed
Turnbull, F, Neal, B, Algert, C, et al. Effects of different blood pressure-lowering regimens on major cardiovascular events in individuals with and without diabetes mellitus: results of prospectively designed overviews of randomized trials. Arch Intern Med. 2005;165(12):1410–9.Google ScholarPubMed
Halpern, SD, French, B, Small, DS, et al. Randomized trial of four financial-incentive programs for smoking cessation. N Engl J Med. 2015;372(22):2108–17.CrossRefGoogle ScholarPubMed
Tan, HJ, Norton, EC, Ye, Z, et al. Long-term survival following partial vs radical nephrectomy among older patients with early-stage kidney cancer. JAMA. 2012;307(15):1629–35.Google ScholarPubMed
McClellan, M, McNeil, BJ, Newhouse, JP. Does more intensive treatment of acute myocardial infarction in the elderly reduce mortality? Analysis using instrumental variables. JAMA. 1994;272(11):859–66.CrossRefGoogle ScholarPubMed
Neuman, MD, Rosenbaum, PR, Ludwig, JM, Zubizarreta, JR, Silber, JH. Anesthesia technique, mortality, and length of stay after hip fracture surgery. JAMA. 2014;311(24):2508–17.CrossRefGoogle ScholarPubMed
Garabedian, LF, Chu, P, Toh, S, Zaslavsky, AM, Soumerai, SB. Potential bias of instrumental variable analyses for observational comparative effectiveness research. Ann Intern Med. 2014;161(2):131–8.Google ScholarPubMed
Prasad, V, Jena, AB. Prespecified falsification end points: can they validate true observational associations? JAMA. 2013;309(3):241–2.CrossRefGoogle ScholarPubMed
Selby, JV, Friedman, GD, Quesenberry, CP Jr., Weiss, NS. A case-control study of screening sigmoidoscopy and mortality from colorectal cancer. N Engl J Med. 1992;326(10):653–7.CrossRefGoogle ScholarPubMed
Schmidt, C. Colonoscopy vs. sigmoidoscopy: new studies fuel ongoing debate. J Nat Cancer Inst. 2012;104:1350–1.CrossRefGoogle ScholarPubMed
Tuccori, M, Filion, KB, Yin, H, et al. Pioglitazone use and risk of bladder cancer: population based cohort study. BMJ. 2016;352:i1541.CrossRefGoogle ScholarPubMed
Turner, RM, Kwok, CS, Chen-Turner, C, et al. Thiazolidinediones and associated risk of bladder cancer: a systematic review and meta-analysis. Br J Clin Pharmacol. 2014;78(2):258–73.CrossRefGoogle ScholarPubMed
Rimm, EB, Stampfer, MJ, Ascherio, A, et al. Vitamin E consumption and the risk of coronary heart disease in men. N Engl J Med. 1993;328(20):1450–6.CrossRefGoogle ScholarPubMed
Stampfer, MJ, Hennekens, CH, Manson, JE, et al. Vitamin E consumption and the risk of coronary disease in women. N Engl J Med. 1993;328(20):1444–9.CrossRefGoogle ScholarPubMed
Bjelakovic, G, Nikolova, D, Gluud, LL, Simonetti, RG, Gluud, C. Antioxidant supplements for prevention of mortality in healthy participants and patients with various diseases. Cochrane Database Syst Rev. 2012;3:CD007176.Google Scholar
Jena, AB, Prasad, V, Goldman, DP, Romley, J. Mortality and treatment patterns among patients hospitalized with acute cardiovascular conditions during dates of national cardiology meetings. JAMA Intern Med. 2015;175(2):237–44.CrossRefGoogle ScholarPubMed
Gum, PA, Thamilarasan, M, Watanabe, J, Blackstone, EH, Lauer, MS. Aspirin use and all-cause mortality among patients being evaluated for known or suspected coronary artery disease: A propensity analysis. JAMA. 2001;286(10):1187–94.CrossRefGoogle ScholarPubMed
Wei, L, MacDonald, TM, Mackenzie, IS. Pioglitazone and bladdercancer: a propensity scorematched cohort study. Br J Clin Pharmacol. 2012;75(1):254–59.Google Scholar
Suissa, S. Immortal time bias in pharmaco-epidemiology. Am J Epidemiol. 2008;167(4):492–9.CrossRefGoogle ScholarPubMed
Ray, WA. Evaluating medication effects outside of clinical trials: new-user designs. Am J Epidemiol. 2003;158(9):915–20.CrossRefGoogle ScholarPubMed
Hernan, MA, Alonso, A, Logan, R, et al. Observational studies analyzed like randomized experiments: an application to postmenopausal hormone therapy and coronary heart disease. Epidemiology. 2008;19(6):766–79.CrossRefGoogle ScholarPubMed
Zhang, J, Yancey, MK, Klebanoff, MA, Schwarz, J, Schweitzer, D. Does epidural analgesia prolong labor and increase risk of cesarean delivery? A natural experiment. Am J Obstet Gynecol. 2001;185(1):128–34.CrossRefGoogle ScholarPubMed
Taddio, A, Katz, J, Ilersich, AL, Koren, G. Effect of neonatal circumcision on pain response during subsequent routine vaccination. Lancet. 1997;349(9052):599603.CrossRefGoogle ScholarPubMed
Layton, TJ, Barnett, ML, Hicks, TR, Jena, AB. Attention deficit-hyperactivity disorder and month of school enrollment. N Engl J Med. 2018;379(22):2122–30.CrossRefGoogle ScholarPubMed
Roze, JC, Cambonie, G, Marchand-Martin, L, et al. Association between early screening for patent ductus arteriosus and in-hospital mortality among extremely preterm infants. JAMA. 2015;313(24):2441–8.CrossRefGoogle ScholarPubMed
Lindenauer, PK, Pekow, P, Wang, K, Gutierrez, B, Benjamin, EM. Lipid-lowering therapy and in-hospital mortality following major noncardiac surgery. JAMA. 2004;291(17):2092–9.CrossRefGoogle ScholarPubMed
Bui, Q, Miller, CC. The age that women have babies: how a gap divides America. New York Times. August 4, 2018.Google Scholar

Send book to Kindle

To send this book to your Kindle, first ensure no-reply@cambridge.org is added to your Approved Personal Document E-mail List under your Personal Document Settings on the Manage Your Content and Devices page of your Amazon account. Then enter the ‘name’ part of your Kindle email address below. Find out more about sending to your Kindle.

Note you can select to send to either the @free.kindle.com or @kindle.com variations. ‘@free.kindle.com’ emails are free but can only be sent to your device when it is connected to wi-fi. ‘@kindle.com’ emails can be delivered even when you are not connected to wi-fi, but note that service fees apply.

Find out more about the Kindle Personal Document Service.

Available formats
×

Send book to Dropbox

To send content items to your account, please confirm that you agree to abide by our usage policies. If this is the first time you use this feature, you will be asked to authorise Cambridge Core to connect with your account. Find out more about sending content to Dropbox.

Available formats
×

Send book to Google Drive

To send content items to your account, please confirm that you agree to abide by our usage policies. If this is the first time you use this feature, you will be asked to authorise Cambridge Core to connect with your account. Find out more about sending content to Google Drive.

Available formats
×