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Paeds Topicsprofessional-practice-and-evidence

Paeds · professional-practice-and-evidence

Clinical epidemiology and measures of effect

Also known as Measures of effect in paediatric research · Relative risk, odds ratio and hazard ratio · Absolute risk reduction and number needed to treat · Attributable risk and population attributable fraction · Interpreting effect estimates and confidence intervals

Fellowship guide to clinical epidemiology and measures of effect in paediatrics: incidence and prevalence, the 2x2 contingency table, relative risk, odds ratio and hazard ratio, risk difference, absolute and relative risk reduction, number needed to treat and harm, attributable risk and population attributable fraction, interpretation of confidence intervals, effect modification and confounding, with worked examples and ANZ, UK, US and Canada guidance.

high14 referencesUpdated 11 July 202625 min readVerification in progress

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Target exams

RACP DWEMRCPCH Theory

Red flags

  • Reading an odds ratio as a relative risk when the outcome is common, which overstates the apparent effect
  • Quoting a relative risk reduction without the absolute baseline risk or number needed to treat
  • Treating a confidence interval that crosses the null as a positive result
  • Applying a number needed to treat from a trial to a child whose baseline risk differs, without recomputing
  • Pooling odds ratios across trials with disparate baseline risks and believing the summary applies to one patient
  • Confusing effect modification with confounding, or hiding a true subgroup difference inside a single pooled estimate

Life stages

fetalneonateinfanttoddlerpreschoolschool-ageadolescentyoung-adult-transition

Care settings

outpatient

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written-only

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  • Clinical epidemiology and measures of effect
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Target exams

RACP DWEMRCPCH Theory

Red flags

  • Reading an odds ratio as a relative risk when the outcome is common, which overstates the apparent effect
  • Quoting a relative risk reduction without the absolute baseline risk or number needed to treat
  • Treating a confidence interval that crosses the null as a positive result
  • Applying a number needed to treat from a trial to a child whose baseline risk differs, without recomputing
  • Pooling odds ratios across trials with disparate baseline risks and believing the summary applies to one patient
  • Confusing effect modification with confounding, or hiding a true subgroup difference inside a single pooled estimate

Life stages

fetalneonateinfanttoddlerpreschoolschool-ageadolescentyoung-adult-transition

Care settings

outpatient

Clinical exam formats

written-only

Board mappings

  • Clinical epidemiology and measures of effect
The fellowship answer

Every clinical claim about a treatment or exposure is a number, and the measure that produced that number decides whether the claim is honest. Clinical epidemiology gives you the toolkit: build the 2x2 contingency table, then read off the relative measures (relative risk, odds ratio, hazard ratio) and the absolute measures (risk difference, absolute and relative risk reduction, number needed to treat). The single most examinable trap is the odds ratio read as a relative risk when the outcome is common, because the odds ratio then overstates the effect. Always report the absolute effect alongside the relative one, read the confidence interval against its null value (1 for ratios, 0 for differences), and tailor the number needed to treat to the child's own baseline risk rather than the trial's average. A measure of association is not a measure of how the child will respond; it is a probability you weigh with the family.

[1][8]

Two families you must never blur

Relative measures (ratio, null = 1)

    Absolute measures (difference, null = 0)

      Overview & Definition

      A parent hands you an abstract claiming a therapy halves their child's risk of a complication, and you must decide whether to start it. The word "halves" is a relative risk reduction, and on its own it tells you almost nothing useful, because the same 50 percent reduction means a very different absolute benefit when the baseline risk is 2 in 100 than when it is 20 in 100. Clinical epidemiology is the set of methods that turns a study's raw counts into the numbers that drive that decision, and its measures of effect are the vocabulary in which the answer must be given. [1] [8]

      Clinical epidemiology applies epidemiological methods to the questions clinicians ask at the bedside — therapy, harm, prognosis, and aetiology. Its currency is the measure of effect, the statistic that quantifies the strength of the link between an exposure (or a treatment) and an outcome. Two facts about any outcome anchor everything else: incidence counts new cases over time, while prevalence counts existing cases at a single point. A risk is a probability of the outcome, between 0 and 1, while an odds is a ratio of the probability of the event to the probability of no event, which can run from 0 to infinity. [8]

      The central distinction you must hold is between relative and absolute measures. A relative measure tells you how many times more likely the outcome is in one group than another, while an absolute measure tells you how many extra or fewer events the exposure actually produces. They answer different questions, they can disagree about whether an effect is large, and a report that gives only the relative figure is hiding the more useful number. This page owns the computation and interpretation of these measures; the appraisal process and hierarchy of evidence belong to the evidence-based medicine leaf, and sensitivity, specificity, and likelihood ratios belong to the diagnostic accuracy leaf. [1] [14]

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      References14ShowHide
      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]Guyatt GH, Sackett DL, Cook DJ Users' guides to the medical literature. II. How to use an article about therapy or prevention. B. What were the results and will they help me in caring for my patients? Evidence-Based Medicine Working Group JAMA, 1994.PMID 8258890
      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]Cook RJ, Sackett DL The number needed to treat: a clinically useful measure of treatment effect BMJ, 1995.PMID 7873954
      5. [5]Davies HT, Crombie IK, Tavakoli M When can odds ratios mislead? BMJ, 1998.PMID 9550961
      6. [6]Zhang J, Yu KF What's the relative risk? A method of correcting the odds ratio in cohort studies of common outcomes JAMA, 1998.PMID 9832001
      7. [7]Spruance SL, Reid JE, Grace M, Samore M Hazard ratio in clinical trials Antimicrob Agents Chemother, 2004.PMID 15273082
      8. [8]Greenhalgh T How to read a paper. Statistics for the non-statistician. II: Significant relations and their pitfalls BMJ, 1997.PMID 9277611
      9. [9]Egger M, Davey Smith G, Schneider M, Minder C Bias in meta-analysis detected by a simple, graphical test BMJ, 1997.PMID 9310563
      10. [10]Altman DG, Bland JM Absence of evidence is not evidence of absence BMJ, 1995.PMID 7647644
      11. [11]Altman DG, Bland JM Interaction revisited: the difference between two estimates BMJ, 2003.PMID 12543843
      12. [12]Grimes DA, Schulz KF Bias and causal associations in observational research Lancet, 2002.PMID 11812579
      13. [13]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
      14. [14]Murad MH, Montori VM, Ioannidis JP, et al. How to read a systematic review and meta-analysis and apply the results to patient care: users' guides to the medical literature JAMA, 2014.PMID 25005654

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