EBHC Library > EBP > Clinical Trials > Articles

Page last update: 08/01/2008

	General
	Design
	Reporting
	Bias
	Search Strategies
	Critical Appraisal
	Measures of Efficacy
	Economic Issues
	Miscellanea

Design

• General

  •  

• Ethical issues

  • Moving from compliance to conscience: why we can and should improve on the ethics of clinical research. Arch Intern Med 2001
  • Scientific and Ethical Issues in Equivalence Trials. JAMA 2001;
  • What makes clinical research ethical? JAMA 2000;
  • Marquis D. How to resolve an ethical dilemma concerning randomized clinical trials. N Engl J Med 1999;341:691-3
  • Edwards SJ, Lilford RJ, Hewison J. The ethics of randomised controlled trials from the perspectives of patients, the public, and healthcare professionals. BMJ 1998;317:1209-12
  • Rothman KJ, Michels KB. The continuing unethical use of placebo controls. N Engl J Med 1994;331:394-8

• Randomization

  • General
    • Gebski VJ, Beller EM, Keech AC. Randomisation in clinical trials. Med J Aust 2002;177:565-567
    • Schulz KF, Grimes DA. Generation of allocation sequences in randomised trials: chance, not choice. Lancet 2002;359:515-9
    • Schulz KF, Grimes DA. Unequal group sizes in randomised trials: guarding against guessing. Lancet 2002;359:966-70
    • Altman DG, Bland JM. How to randomise BMJ 1999;319:703-44.
    • Roberts C, Torgerson D. Randomisation methods in controlled trials. BMJ 1998;317:1301
    • Schulz KF. Randomised trials, human nature, and reporting guidelines. Lancet 1996;348:596-98
    • Schulz KF. Subverting randomization in controlled trials. JAMA 1995;274:1456-8.
  • Allocation Concealment
    • Pildal J, Chan AW, Hrobjartsson A, et al. Comparison of descriptions of allocation concealment in trial protocols and the published reports: cohort study. BMJ 2005;330:1049
    • Hewitt C, Hahn S, Torgerson DJ, et al. Adequacy and reporting of allocation concealment: review of recent trials published in four general medical journals. BMJ 2005;330:1057-8.
    • Forder PM, Gebski VJ, Keech AC. Allocation concealment and blinding: when ignorance is bliss. Med J Aust 2005;182:87-9
    • Schulz KF, Grimes DA. Allocation concealment in randomised trials: defending against deciphering. Lancet 2002;359:614-8
    • Altman DG, Schulz KF. Statistics notes: Concealing treatment allocation in randomised trials. BMJ 2001;323:446-7.

    • Schulz KF. Assessing allocation concealment and blinding in randomized controlled trials: why bother? Evidence-Based Medicine [EBM Notebook] 2000;5:36-7

    • Haynes RB. Incorporating allocation concealment and blinding in randomized controlled trials. Evidence-Based Medicine [EBM Notebook] 2000;5:38

    • Roberts C, Torgerson D. Randomisation methods: concealment. BMJ 1999;319:375-6
  • Others
    • Hewitt CE, Torgerson DJ. Is restricted randomisation necessary? BMJ 2006;332:1506-8
    • Altman DG, Bland JM. Treatment allocation by minimisation. BMJ 2005;330:843.
    • Roberts C, Sibbald B. Randomising groups of patients BMJ 1998;316:1898

• Sample size

• Blinding

  • Boutron I, Estellat C, Guittet L, et al. Methods of blinding in reports of randomized controlled trials assessing pharmacologic treatments: a systematic review. PLoS Med 2006;3:e425
  • Forder PM, Gebski VJ, Keech AC. Allocation concealment and blinding: when ignorance is bliss. Med J Aust 2005;182:87-9
  • Devereaux PJ, Choi PT, El-Dika S, et al. An observational study found that authors of randomized controlled trials frequently use concealment of randomization and blinding, despite the failure to report these methods. J Clin Epidemiol 2004;57:1232-1236 [abstract]
  • Boutron I, Tubach F, Giraudeau B, et al. Blinding was judged more difficult to achieve and maintain in nonpharmacologic than pharmacologic trials. J Clin Epidemiol 2004;57:543-50 [abstract]
  • Fergusson D, Glass KC, Waring D, et al. Turning a blind eye: the success of blinding reported in a random sample of randomised, placebo controlled trials. BMJ 2004;328:432
  • Montori VM, Bhandari M, Devereaux PJ, et al. In the dark: the reporting of blinding status in randomized controlled trials. J Clin Epidemiol. 2002;55:787-90 [abstract]
  • Schulz KF, Grimes DA. Blinding in randomised trials: hiding who got what. Lancet 2002;359:696-700.
  • Schulz KF, Chalmers I, Altman DG. The landscape and lexicon of blinding in randomized trials. Ann Intern Med 2002;136:254-9
  • Devereaux PJ, Manns BJ, Ghali WA, et al. Physician interpretations and textbook definitions of blinding terminology in randomized controlled trials. JAMA 2001;285:2000-3
  • Day SJ, Altman DG. Statistics notes: blinding in clinical trials and other studies. BMJ 2000;321:504
  • Meinert CL. Masked monitoring in clinical trials--blind stupidity? N Engl J Med 1998;338:1381-2
  • Jadad AR, Moore RA, Carroll D, et al. Assessing the quality of reports of randomized clinical trials: is blinding necessary? Control Clin Trials 1996;17:1-12  [abstract]
• Placebo
• End-points
  • Surrogate end-point
    • Hauptman PJ. Measurement of end points in heart failure trials: jousting at windmills? Mt Sinai J Med 2004;71:298-304
    • Johnson JR, Williams G, Pazdur R. End points and United States FDA approval of oncology drugs. J Clin Oncol 2003;21:1404-1411
    • Baker SG, Kramer BS.  A perfect correlate does not a surrogate make. BMC Med Res Methodol 2003;3:16
    • Hughes MD. Evaluating surrogate endpoints. Control Clin Trials 2002;23:703-7
    • Molenberghs G, Buyse M, Geys H, et al. Statistical challenges in the evaluation of surrogate endpoints in randomized trials. Control Clin Trials 2002;23:607-25 [abstract]
    • De Gruttola VG, Clax P, DeMets DL, et al. Considerations in the evaluation of surrogate endpoints in clinical trials. summary of a National Institutes of Health workshop. Control Clin Trials 2001;22:485-502 [abstract]
    • Biomarkers Definitions Working Group. Biomarkers and surrogate endpoints: preferred definitions and conceptual framework. Clin Pharmacol Ther 2001;69:89-95
    • Kuller LH. Clinical trials: surrogate endpoints or hard endpoints? Am J Cardiol 2001;88(2-A):59E-61E [abstract]
    • Kaplan NM. Should new drugs be used without outcome data? Implications of ALLHAT and ELITE II. Arch Intern Med 2001;161:511-2
    • Fleming TR. Surrogate end points in cardiovascular disease trials. Am Heart J 2000;139:S193-6.
    • Bobbio M. Gli end point surrogati. It Hearth J 2000;1:877-9
    • D’Agostino Jr RB. Debate: The slippery slope of surrogate outcomes. Curr Control Trials Cardiovasc Med 2000, 1:76–78
    • Demets DL. The role of surrogate outcome measures in evaluating medical devices. Surgery 2000;128:379-85
    • Bucher HC, Guyatt GH, Cook DJ, et al. Users' Guides to the Medical Literature: XIX. Applying Clinical Trial Results A. How to Use an Article Measuring the Effect of an Intervention on Surrogate End Points. JAMA 1999;282:771-8
    • Temple R. Are surrogate markers adequate to assess cardiovascular disease drugs? JAMA 1999;282:790-5
    • Psaty BM, Weiss NS, Furberg CD, et al. Surrogate end points, health outcomes, and the drug-approval process for the treatment of risk factors for cardiovascular disease. JAMA 1999;282:786-90
    • Fleming TR, DeMets DL. Surrogate end points in clinical trials: are we being misled? Ann Intern Med 1996;125:605-13.
    • Johnston K. What are surrogate outcome measures and why do they fail in clinical research? Neuroepidemiology 1999;18:167-73
    • Sobel BE, Furberg CD. Surrogates, semantics, and sensible public policy. Circulation. 1997;95:1661-1663
    • Surrogate measures in clinical trials. Lancet 1990;335:261-2
  • Composite end-points
    • Montori VM, Permanyer-Miralda G, Ferreira-Gonzalez I, et al. Validity of composite end points in clinical trials. BMJ 2005;330:594-6.
    • Montori VM, Busse JW, Permanyer-Miralda G, et al. How should clinicians interpret results reflecting the effect of an intervention on composite endpoints: should I dump this lump? Evid Based Med 2005;10:162-3
    • Freemantle N, Calvert M, Wood J, et al. Composite outcomes in randomized trials: greater precision but with greater uncertainty? JAMA 2003;289:2554-9.
      • Lauer MS, Topol EJ. Clinical trials--multiple treatments, multiple end points, and multiple lessons. JAMA 2003;289:2575-7.
• Statistical Significancy and P value
  • Primer on: Type I and Type II Errors. Eff Clin Pract, Nov-Dec 2001
  • Primer on: 95% Confidence Intervals. Eff Clin Pract, Sep-Oct 2001
  • Primer on: Statistical Significance and P Values. Eff Clin Pract Jul-Aug 2001
  • Sterne JA, Davey Smith G. Sifting the evidence - what's wrong with significance tests? BMJ 2001;322:226-231
  • Goodman SN. Toward evidence-based medical statistics. 1: The P value fallacy. Ann Intern Med 1999;130:995-1004.
  • Goodman SN. Toward evidence-based medical statistics. 2: The Bayes factor. Ann Intern Med 1999;130:1005-13.
  • Guyatt GH,  Jaeschke R,  Heddle N, et al. Hypothesis testing. CMAJ 1995;152:27-32.
• Class Effect
  • Zhou Z, Rahme E, Abrahamowicz M, Tu JV, et al. Effectiveness of statins for secondary prevention in elderly patients after acute myocardial infarction: an evaluation of class effect. CMAJ 2005;172:1187-94.
    • Wright JM. Are the benefits of statins a class effect? CMAJ 2005;172:1195-6.
  • Pilote L, Abrahamowicz M, Rodrigues E, et al. Mortality rates in elderly patients who take different angiotensin-converting enzyme inhibitors after acute myocardial infarction: a class effect? Ann Intern Med 2004;141:102-12.
  • Antman EM, Ferguson JJ. Should evidence-based proof of efficacy as defined for a specific therapeutic agent be extrapolated to encompass a therapeutic class of agents? Circulation 2003;108:2604-7
  • Furberg CD, Psaty BM. Should evidence-based proof of drug efficacy be extrapolated to a "class of agents"?  Circulation 2003;108:2608-2610
  • Kereiakes DJ, Willerson JT. Therapeutic substitution: guilty until proven innocent  Circulation 2003;108:2611-2612
  • Brown MJ. A rational basis for selection among drugs of the same class. Heart 2003;89:687-94.
  • Kennedy HL, Rosenson RS. Physicians' interpretation of "class effects": a need for thoughtful re-evaluation. J Am Coll Cardiol. 2002;40:19-26.
  • McAlister FA, Sackett DL. Active-control equivalence trials and antihypertensive agents. Am J Med 2001;111:553-8.
    • Class and equivalence. Bandolier, 2002
  • Furberg CD. Class effects and evidence-based medicine. Clin Cardiol 2000;23(7 Suppl 4):IV15-9
  • McAlister FA, Laupacis A, Wells GA, et al. Users' Guides to the Medical Literature: XIX. Applying clinical trial results B. Guidelines for determining whether a drug is exerting (more than) a class effect. JAMA 1999;282:1371-7.
  • Furberg CD, Herrington DM, Psaty BM. Are drugs within a class interchangeable? Lancet 1999;354:1202-4.
  • Kessler DA, Rose JL, Temple RJ, et al. Therapeutic-class wars--drug promotion in a competitive marketplace. N Engl J Med 1994;331:1350-3.
• Intention to Treat Analysis
  • Heritier SR, Gebski VJ, Keech AC. Inclusion of patients in clinical trial analysis: the intention-to-treat principle. Med J Aust 2003;179:438-40
  • Wright CC, Sim J. Intention-to-treat approach to data from randomized controlled trials: a sensitivity analysis. J Clin Epidemiol 2003;56:833-42 [abstract]
  • Kruse RL, Alper BS, Reust C, et al. Intention-to-treat analysis: who is in? Who is out? J Fam Pract 2002;51:969-71
  • Fergusson D, Aaron SD, Guyatt G, et al. Post-randomisation exclusions: the intention to treat principle and excluding patients from analysis. BMJ 2002;325:652-4
  • Goetghebeur E, Loeys T. Beyond intention to treat. Epidemiol Rev 2002;24:85-90
  • Montori VM, Guyatt GH. Intention-to-treat principle. CMAJ 2001;165:1339-41
  • Ruiz-Canela M, Martinez-Gonzalez MA, de Irala-Estevez J. Intention to treat analysis is related to methodological quality. BMJ 2000;320:1007-8.
  • Hollis S, Campbell F. What is meant by intention to treat analysis? Survey of published randomised controlled trials. BMJ 1999;319:670-4
• Subgroup Analysis
  • Lagakos SW. The challenge of subgroup analyses--reporting without distorting. N Engl J Med 2006;354:1667-9.

  • Parker AB, Naylor CD. Interpretation of subgroup results in clinical trial publications: insights from a survey of medical specialists in Ontario, Canada. Am Heart J 2006;151:580-8.

  • Hernandez AV, Boersma E, Murray GD, et al. Subgroup analyses in therapeutic cardiovascular clinical trials: are most of them misleading? Am Heart J 2006;151:257-64.

  • Rothwell PM. Subgroup analysis in randomised controlled trials: importance, indications, and interpretation. Lancet 2005;365:176-86

  • Schulz KF, Grimes DA. Multiplicity in randomised trials II: subgroup and interim analyses. Lancet 2005;365:1657-61

  • Rothwell PM, Mehta Z, Howard SC, et al. 3. From subgroups to individuals: general principles and the example of carotid endarterectomy. Lancet 2005;365:256-65

  • Simes RJ, Gebski VJ, Keech AC. Subgroup analysis: application to individual patient decisions. Med J Aust 2004;180:467-9
  • Brookes ST, Whitely E, Egger M, et al. Subgroup analyses in randomized trials: risks of subgroup-specific analyses; power and sample size for the interaction test. J Clin Epidemiol 2004;57:229-36
  • Cook DI, Gebski VJ, Keech AC. Subgroup analysis in clinical trials. Med J Aust 2004;180:289-91
  • Brookes ST, Whitley E, Peters TJ, et al. Subgroup analyses in randomised controlled trials: quantifying the risks of false-positives and false-negatives. Health Technol Assess 2001;5:1-56
  • Freemantle N. Interpreting the results of secondary end points and subgroup analyses in clinical trials: should we lock the crazy aunt in the attic? BMJ 2001;322:989-91
  • Assmann SF, Pocock SJ, Enos LE, et al. Subgroup analysis and other (mis)uses of baseline data in clinical trials. Lancet 2000;355:1064-9
  • Horton R. From star signs to trial guidelines. Lancet 2000;355:1033-4
  • Parker AB, Naylor CD. Subgroups, treatment effects, and baseline risks: some lessons from major cardiovascular trials. Am Heart J 2000;139:952-961.
  • Adams KF Jr. Post hoc subgroup analysis and the truth of a clinical trial. Am Heart J 1998;136:753-8
  • Detsky AS, Naglie IG. Subgroup analyses: primary and secondary. ACP J Club 1995;122(3):A12-A14
  • Oxman AD, Guyatt GH. A consumer's guide to subgroup analyses. Ann Intern Med 1992;116:78-84 [abstract]
  • Yusuf S, Wittes J, Probstfield J, Tyroler HA. Analysis and interpretation of treatment effects in subgroups of patients in randomized clinical trials. JAMA 1991;266:93-8 [abstract]
  • Bulpitt CJ. Subgroup analysis. Lancet 1988;ii:31-4.

Reporting

• CONSORT Statement
  • Altman DG, Schulz KF, Moher D, et al; CONSORT Group (Consolidated Standards of Reporting Trials). The revised CONSORT statement for reporting randomized trials: explanation and elaboration. Ann Intern Med 2001;134:663-94
  • Moher D, Schulz KF, Altman DG; CONSORT Group (Consolidated Standards of Reporting Trials). The CONSORT statement: revised recommendations for improving the quality of reports of parallel-group randomized trials. Ann Intern Med 2001;134:657-62.
  • Begg C, Cho M, Eastwood S, et al. Improving the quality of reporting of randomized controlled trials. The CONSORT statement JAMA 1996;276:637-9.
• Extensions of CONSORT Statement
  • Piaggio G, Elbourne DR, Altman DG, et al; CONSORT Group. Reporting of noninferiority and equivalence randomized trials: an extension of the CONSORT statement. JAMA 2006;295:1152-60.
  • Gagnier JJ, Boon H, Rochon P, et al; CONSORT Group. Reporting randomized, controlled trials of herbal interventions: an elaborated CONSORT statement. Ann Intern Med 2006;144:364-7. 
  • Ioannidis JP, Evans SJ, Gotzsche PC, et al; CONSORT Group. Better reporting of harms in randomized trials: an extension of the CONSORT statement. Ann Intern Med 2004;141:781-8.
  • Campbell MK, Elbourne DR, Altman DG; CONSORT group. CONSORT statement: extension to cluster randomised trials. BMJ 2004;328:702-8.
• CONSORT Statement implementation
  • Plint AC, Moher D, Morrison A, et al. Does the CONSORT checklist improve the quality of reports of randomised controlled trials? A systematic review. Med J Aust 2006;185:263-7.
  • Altman DG. Endorsement of the CONSORT statement by high impact medical journals: survey of instructions for authors. BMJ 2005;330:1056-7.
  • Moher D, Jones A, Lepage L; CONSORT Group. Use of the CONSORT statement and quality of reports of randomized trials: a comparative before-and-after evaluation. JAMA 2001;285:1992-5.
    • Rennie D. CONSORT revised--improving the reporting of randomized trials. JAMA 2001;285:2006-7. 
  • Egger M, Juni P, Bartlett C; CONSORT Group. Value of flow diagrams in reports of randomized controlled trials. JAMA 2001;285:1996-9.
• Comments
  • Moher D, Altman DG, Schulz KF, et al. Opportunities and challenges for improving the quality of reporting clinical research: CONSORT and beyond. CMAJ 2004;171:349-50.
  • Campbell MJ. Extending CONSORT to include cluster trials. BMJ 2004;328:654-5.
  • Shapiro S. The revised CONSORT statement: honing the cutting edge of the randomized controlled trial. CMAJ 2001;164:1157-8.
  • Moher D. CONSORT: an evolving tool to help improve the quality of reports of randomized controlled trials. Consolidated Standards of Reporting Trials. JAMA 1998;279:1489-91.
  • Meinert CL. Beyond CONSORT: need for improved reporting standards for clinical trials. Consolidated Standards of Reporting Trials. JAMA 1998;279:1487-9.
  • Freemantle N, Mason JM, Haines A, et al. CONSORT: an important step toward evidence-based health care. Consolidated Standards of Reporting Trials. Ann Intern Med 1997;126:81-3.
  • Huston P, Hoey J. CMAJ endorses the CONSORT statement. CONsolidation of Standards for Reporting Trials. CMAJ 1996;155:1277-82.
  • Altman DG. Better reporting of randomised controlled trials: the CONSORT statement. BMJ 1996;313:570-1
  • Schulz KF. Randomised trials, human nature, and reporting guidelines. Lancet 1996;348:596-98.
  • Rennie D. How to Report Randomized Controlled Trials. The CONSORT Statement. JAMA 1996;276:64

Bias

Search Strategies

• Wilczynski NL, et al; Hedges Team. Optimal search strategies for retrieving scientifically strong studies of treatment from Medline: analytical survey. BMJ 2005;330:1179

• Wong SS, Wilczynski NL, Haynes RB. Related Articles, Links Developing optimal search strategies for detecting clinically sound treatment studies in EMBASE. J Med Libr Assoc 2006;94:41-7.

• Wong SS, Wilczynski NL, Haynes RB. Optimal CINAHL search strategies for identifying therapy studies and review articles. J Nurs Scholarsh 2006;38:194-9.

Critical Appraisal

• General

  • Users' guide to detecting misleading claims in clinical research reports

  • Guyatt GH, Sackett DL, Cook DJ. Evidence-Based Medicine Working Group. Users' guides to the medical literature. II. How to use an article about therapy or prevention.

    • A. Are the results of the study valid? Evidence-Based Medicine Working Group. JAMA 1993;270:2598-601.

    • B. What were the results and will they help me in caring for my patients? Evidence-Based Medicine Working Group. JAMA 1994;271:59-63.

  • Greenalgh T. Papers that report drug trials. BMJ 1997;315:480-483
• Internal validity
• Analysis of results
  • Different interpretation
    • Fahey T, Griffiths S, Peters TJ. Evidence based purchasing: understanding results of clinical trials and systematic reviews. BMJ 1995;311:1056-1060.
• Applicability
  • General
    • Rothwell PM. Factors that can affect the external validity of randomised controlled trials. PLoS Clin Trials 2006;1:e9.
    • Bonell C, Oakley A, Hargreaves J, et al. Assessment of generalisability in trials of health interventions: suggested framework and systematic review. BMJ 2006;333:346-9.

    • Thompson SG, Higgins JP. Treating Individuals 4. Can meta-analysis help target interventions at individuals most likely to benefit? Lancet 2005;365:341-6

    • Rothwell PM. Treating individuals 1. External validity of randomised controlled trials: "to whom do the results of this trial apply?". Lancet 2005;365:82-93

    • Summerskill W. Evidence-based practice and the individual. Lancet 2005;365:13-4

    • Senn S. Individual response to treatment: is it a valid assumption? BMJ 2004;329:966-8.
    • Simes RJ, Gebski VJ, Keech AC. Subgroup analysis: application to individual patient decisions. Med J Aust 2004;180:467-9

    • McAlister FA. Applying evidence to patient care: from black and white to shades of grey. Ann Intern Med 2003;138:938-9

    • Are my patients like this? Bandolier 2002

    • Straus SE, McAlister F. Applying the results of trials and systematic reviews to our individual patients. Evid Based Ment Health 2001 4: 6-7

    • Doust JA, Silagy CA. Applying the results of a systematic review in general practice. Med J Aust 2000;172:153-6 [abstract]

    • McAlister FA, Straus SE, Guyatt GH, et al. Users' guides to the medical literature: XX. Integrating research evidence with the care of the individual patient. Evidence-Based Medicine Working Group. JAMA 2000;283:2829-36.

    • Rosser WW. Application of evidence from randomized controlled trials to general practice. Lancet 1999;353:661-4

    • Mant D. Can randomised trials inform clinical decisions about individual patients? Lancet 1999;353:743-6

    • Straus SE, Sackett DL. Applying evidence to the individual patient. Ann Oncol 1999;10:29-32
    • Britton A, McKee M, Black N, et al. Threats to applicability of randomised trials: exclusions and selective participation. J Health Serv Res Policy 1999;4:112-21 [abstract]
    • Glasziou P, Guyatt GH, Dans AL,et al. Applying the results of trials and systematic reviews to individual patients. ACP J Club 1998;129(3):A15-A16
    • Ross JM. Commentary on applying the results of trials and systematic reviews to individual patients. ACP J Club 1998;129(3):A17
    • Godlee F. Applying research evidence to individual patients. Evidence based case reports will help. BMJ 1998;316:1621-1622
    • Dans AL, Dans LF, Guyatt GH, Richardson S. Users' guides to the medical literature: XIV. How to decide on the applicability of clinical trial results to your patient. JAMA 1998;27;279:545-9
    • Fahey T. Applying the results of clinical trials to patients to general practice: perceived problems, strengths, assumptions, and challenges for the future. Br J Gen Pract 1998;48:1173-8

    • Sullivan FM, MacNaughton RJ. Evidence in consultations: interpreted and individualised. Lancet 1996;348:941-3

    • Glasziou P, Irwig L. An evidence based approach to individualising patient treatment. BMJ 1995;311:1356-9
    • Rothwell PM. Can overall results of clinical trials be applied to all patients? Lancet 1995;345:1616-9 [abstract]
  • Diseases specific

    • Jackson R, Lawes CM, Bennett DA, et al. Treating Individuals 5. Treatment with drugs to lower blood pressure and blood cholesterol based on an individual's absolute cardiovascular risk. Lancet 2005;365:434-41

    • Rothwell PM, Mehta Z, Howard SC, et al. Treating individuals 3. From subgroups to individuals: general principles and the example of carotid endarterectomy. Lancet 2005;365:256-65

    • Rathore SS, Weinfurt KP, Gross CP,et al. Validity of a simple ST-elevation acute myocardial infarction risk index: are randomized trial prognostic estimates generalizable to elderly patients? Circulation 2003;107:811-6

    • Evans A, Kalra L. Are the results of randomized controlled trials on anticoagulation in patients with atrial fibrillation generalizable to clinical practice? Arch Intern Med 2001;161:1443-7

    • Pignone M, Mulrow CD. Evidence based management of hypertension: Using cardiovascular risk profiles to individualise hypertensive treatment. BMJ 2001;322:1164-6

    • Cook DJ, Guyatt GH. Interpreting, integrating, and individualizing evidence about the prevention of diabetic nephropathy. Ann Intern Med 1999;131:707-8

    • Walker PM. Carotid endarterectomy: applying trial results in clinical practice. CMAJ 1997;157:693-4

Measures of Efficacy

• General

  • Centre for Evidence-based Medicine. NNT
  • Jaeschke R, et al. Assessing the effects of treatment: measures of association Can Med Assoc J 1995;152:351-357
  • Primer on 95% CIs for the Number Needed To Treat. Effective Clinical Practice, May/June 1999
  • Sackett DL, Haynes RB. Summarizing the effects of a therapy: a new table and some more terms. Evidence-Based Medicine [EBM Notebook] 1997;2:103-4.
  • Steiner JF. Talking about Treatment: The  Language of Populations and the  Language of Individuals. Ann Intern Med 1999;130:618-622
  • Sackett DL, Deeks JJ, Altman DG. Down with odds ratios! Evidence-Based Medicine [EBM Notebook] 1996;2:164-6
  • Davies HTO, Crombie IK, Tavakoli M. When can odds ratios mislead? BMJ 1998;316:989-991
  • Swots corner: What is an odds ratio? Bandolier 1996;25:6

• Number Needed to Treat (NNT)

• Confidence intervals

  • Guyatt GH, et al. Interpreting study results: confidence intervals. Can Med Assoc J 1995;152:169-173
  • Altman DG. Use of confidence intervals to indicate uncertainty in research findings. Evidence-Based Medicine [EBM Notebook] 1996;2:102-4

Economic Issues

Miscellanea

• Acronyms
  • Stanbrook MB, Austin PC, Redelmeier DA. Acronym-named randomized trials in medicine--the ART in medicine study. N Engl J Med 2006;355:101-2.
  • Sundar S, Lawton P. International register of trial acronyms. Lancet 2004;363:171.
  • Fallowfield L, Jenkins V. Acronymic trials: the good, the bad, and the coercive. Lancet. 2002;360:1622.
  • Orlowski JP, Christensen JA. The potentially coercive nature of some clinical trial research acronyms. Chest 2002;121:2023-2028.
  • Berkwits M. Capture! Shock! Excite! Clinical trial acronyms and the "branding" of clinical research. Ann Intern Med 2000;133:755-62.
  • Mansel RE. Duplicate acronyms. Lancet 1999;354:1564.
  • Lader EW. Acronym mania. Lancet 2002;360:576.
• Compliance
  • Hewitt CE, Torgerson DJ, Miles JN. Is there another way to take account of noncompliance in randomized controlled trials? CMAJ 2006;175:347-8
  • Giuffrida A, Torgerson DJ. Should we pay the patient? Review of financial incentives to enhance patient compliance. BMJ 1997;315:703-7.
• Statistic analysis
  • Lagakos SW. Time-to-event analyses for long-term treatments--the APPROVe trial. N Engl J Med 2006;355:113-7.
• Non-inferiority/Equivalence Trials
  • Kaul S, Diamond GA. Good enough: a primer on the analysis and interpretation of noninferiority trials. Ann Intern Med 2006;145:62-9.
  • Gotzsche PC. Lessons from and cautions about noninferiority and equivalence randomized trials. JAMA 2006;295:1172-4.
  • Le Henanff A, Giraudeau B, Baron G, et al. Quality of reporting of noninferiority and equivalence randomized trials. JAMA 2006;295:1147-51.
  • Piaggio G, Elbourne DR, Altman DG, et al; CONSORT Group. Reporting of noninferiority and equivalence randomized trials: an extension of the CONSORT statement. JAMA 2006;295:1152-60.
  • McAlister FA, Sackett DL. Active-control equivalence trials and antihypertensive agents. Am J Med 2001;111:553-8.
    • Class and equivalence. Bandolier, 2002
  • Djulbegovic B, Clarke M. Scientific and ethical issues in equivalence trials. JAMA 2001;285:1206-8.
  • For and against: Clinical equipoise and not the uncertainty principle is the moral underpinning of the randomised controlled trial
  • Djulbegovic B, et al. The uncertainty principle and industry-sponsored research. Lancet 2000;356:635-8
  • Tramer MR, Reynolds DJ, Moore RA, et al. When placebo controlled trials are essential and equivalence trials are inadequate. BMJ 1998;317:875-80.
  • Jones B, Jarvis P, Lewis JA, et al. Trials to assess equivalence: the importance of rigorous methods. BMJ 1996;313:36-9.
• Interviews
  • Chalmers TC.  Dr. Tom Chalmers, 1917-1995: the trials of a randomizer. Interview by Malcolm Maclure. CMAJ 1996;155:757-60
• Open label trials
  • Open label extension studies: research or marketing?

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