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EM TopicsResearch, EBM and biostatistics

EM · Research, EBM and biostatistics

Research, evidence-based medicine and biostatistics — appraising and applying evidence at the bedside

Also known as Evidence-based medicine · EBM · Critical appraisal · PICO · Number needed to treat · NNT · Likelihood ratio · Sensitivity and specificity · Confidence interval · Type I error · Type II error · Statistical power · Forest plot · GRADE · CONSORT · PRISMA

Research, evidence-based medicine and biostatistics — Sackett's definition of evidence-based medicine as the integration of best research evidence, clinical expertise and patient values; the hierarchy of evidence from meta-analysis and systematic review through randomised controlled trial, cohort, case-control, cross-sectional and ecological designs down to case series and expert opinion; formulating the focused clinical question with PICO (Population, Intervention, Comparator, Outcome); the three-question critical appraisal of validity, results and applicability; the measures of treatment effect — absolute and relative risk reduction, number needed to treat and to harm (NNT and NNH, Laupacis); the measures of diagnostic test performance — sensitivity, specificity, predictive values and likelihood ratios (Jaeschke); statistical inference — confidence intervals, p-values, type I and type II errors and power; synthesis of evidence in meta-analysis with heterogeneity (I-squared, Higgins), forest and funnel plots and publication bias; grading the body of evidence with GRADE and the risk-of-bias tools (Cochrane RoB 2, ROBINS-I) and the reporting guidelines (CONSORT, PRISMA, STROBE, QUADAS-2); and the differential of good versus poor quality evidence. ACEM-primary, globally tagged.

medium16 referencesUpdated 26 July 202618 min readVerification in progress

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ACEMFRCEMABEMFRCPCCCFPEMEBEEM

Red flags

  • A relative risk reduction reported without the absolute risk reduction is the commonest vehicle for exaggerating a treatment effect — always compute the number needed to treat
  • A diagnostic test with excellent sensitivity and specificity yields a poor positive predictive value when the disease is rare — base-rate neglect at the bedside
  • A p value is the probability of the observed data under the null hypothesis, not the probability that the null hypothesis is true — a small p value is not proof of a meaningful effect
  • A meta-analysis with high heterogeneity (I-squared above 50 per cent) does not deliver a single trustworthy pooled estimate — explore the heterogeneity, do not over-read the diamond
  • An underpowered trial reporting no difference has not shown no effect — absence of evidence is not evidence of absence
  • Surrogate endpoints (blood pressure, lactate clearance) are not patient-centred outcomes — a drug that moves the number may not change mortality, morbidity or quality of life
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Related topics

  • Quality and ED metrics — performance measurement, the four-hour target and quality improvement
  • Medical error and patient safety in the emergency department
  • Patient disposition and safety-netting in the emergency department
  • Team-based care and crisis resource management in the emergency department
  • Consent, capacity and the medico-legal framework in the emergency department
  • Arterial blood gas interpretation — the systematic emergency department approach
Study tools

Your progress

Saved on this device.

Practise this topic8 MCQs with explanations

Target exams

ACEMFRCEMABEMFRCPCCCFPEMEBEEM

Red flags

  • A relative risk reduction reported without the absolute risk reduction is the commonest vehicle for exaggerating a treatment effect — always compute the number needed to treat
  • A diagnostic test with excellent sensitivity and specificity yields a poor positive predictive value when the disease is rare — base-rate neglect at the bedside
  • A p value is the probability of the observed data under the null hypothesis, not the probability that the null hypothesis is true — a small p value is not proof of a meaningful effect
  • A meta-analysis with high heterogeneity (I-squared above 50 per cent) does not deliver a single trustworthy pooled estimate — explore the heterogeneity, do not over-read the diamond
  • An underpowered trial reporting no difference has not shown no effect — absence of evidence is not evidence of absence
  • Surrogate endpoints (blood pressure, lactate clearance) are not patient-centred outcomes — a drug that moves the number may not change mortality, morbidity or quality of life
The one-line answer

Evidence-based medicine, in Sackett's own words, is the conscientious, explicit and judicious use of current best evidence in making decisions about the care of individual patients — the integration of best research evidence with clinical expertise and the patient's values and circumstances. The discipline is a three-question appraisal — validity, results, applicability — applied to a question framed in PICO.[1]

Meet the patient

A registrar hands you a journal-club paper at the end of a night shift. A new anticoagulant, tested in two thousand patients with acute coronary syndrome, reports a "50 per cent reduction in death or stroke" — a headline that reads as transformative. She asks whether the drug should replace the current standard on the department protocol.[1]

Before you answer, reach for the absolute numbers behind the relative one. The control event rate is 2 per cent and the treated rate is 1 per cent: a 50 per cent relative reduction that is a 1 per cent absolute benefit, a number needed to treat of one hundred. The headline and the bedside decision are not the same sentence, and the appraisal is the discipline that converts one into the other.[1]

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References16ShowHide
  1. [1]Sackett DL, Rosenberg WM, Gray JA, Haynes RB, Richardson WS. Evidence based medicine: what it is and what it isn't BMJ, 1996.PMID 8555924
  2. [2]Richardson WS, Wilson MC, Nishikawa J, Hayward RS. The well-built clinical question: a key to evidence-based decisions ACP J Club, 1995.PMID 7582737
  3. [3]Laupacis A, Sackett DL, Roberts RS. An assessment of clinically useful measures of the consequences of treatment N Engl J Med, 1988.PMID 3374545
  4. [4]Jaeschke R, Guyatt GH, Sackett DL. Users' guides to the medical literature. III. How to use an article about a diagnostic test. B. What are the results and will they help me in caring for my patients? The Evidence-Based Medicine Working Group JAMA, 1994.PMID 8309035
  5. [5]Schulz KF, Altman DG, Moher D; CONSORT Group. CONSORT 2010 statement: updated guidelines for reporting parallel group randomised trials Int J Surg, 2011.PMID 22019563
  6. [6]Moher D, Liberati A, Tetzlaff J, Altman DG; PRISMA Group. Preferred reporting items for systematic reviews and meta-analyses: the PRISMA statement BMJ, 2009.PMID 19622551
  7. [7]von Elm E, Altman DG, Egger M, Pocock SJ, Gøtzsche PC, Vandenbroucke JP; STROBE Initiative. The Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) statement: guidelines for reporting observational studies Lancet, 2007.PMID 18064739
  8. [8]Whiting PF, Rutjes AWS, Westwood ME, et al. QUADAS-2: a revised tool for the quality assessment of diagnostic accuracy studies Ann Intern Med, 2011.PMID 22007046
  9. [9]Guyatt GH, Oxman AD, Kunz R, et al. What is quality of evidence and why is it important to clinicians? BMJ, 2008.PMID 18456631
  10. [10]Higgins JP, Thompson SG, Deeks JJ, Altman DG. Measuring inconsistency in meta-analyses BMJ, 2003.PMID 12958120
  11. [11]Higgins JPT, Altman DG, Gøtzsche PC, et al. The Cochrane Collaboration's tool for assessing risk of bias in randomised trials BMJ, 2011.PMID 22008217
  12. [12]Sterne JAC, Savović J, Page MJ, et al. RoB 2: a revised tool for assessing risk of bias in randomised trials BMJ, 2019.PMID 31462531
  13. [13]Sterne JA, Hernán MA, Reeves BC, et al. ROBINS-I: a tool for assessing risk of bias in non-randomised studies of interventions BMJ, 2016.PMID 27733354
  14. [14]Shakur H, Roberts I, Bautista R, et al. Effects of tranexamic acid on death, vascular occlusive events, and blood transfusion in trauma patients with significant haemorrhage (CRASH-2): a randomised, placebo-controlled trial Lancet, 2010.PMID 20554319
  15. [15]Horby P, Lim WS, Emberson JR, et al. Dexamethasone in Hospitalized Patients with Covid-19 N Engl J Med, 2021.PMID 32678530
  16. [16]Emberson J, Lees KR, Lyden P, et al. Effect of treatment delay, age, and stroke severity on the effects of intravenous thrombolysis with alteplase for acute ischaemic stroke: a meta-analysis of individual patient data from randomised trials Lancet, 2014.PMID 25106063

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Related topics

  • Quality and ED metrics — performance measurement, the four-hour target and quality improvement
  • Medical error and patient safety in the emergency department
  • Patient disposition and safety-netting in the emergency department
  • Team-based care and crisis resource management in the emergency department
  • Consent, capacity and the medico-legal framework in the emergency department
  • Arterial blood gas interpretation — the systematic emergency department approach