Chapter 8 · Designing the study

Why do studies get the wrong answer?

Bias, confounding, validity, reliability, accuracy and precision.

31 TNMGRMU questions answered hereset 31 times, 2004–2025

The problem first

You are a second-year Medicine resident. Your case-control thesis compares 200 patients admitted with myocardial infarction (MI) with 450 controls. Coffee drinkers have nearly four times the odds of MI (odds ratio 3.78), and your discussion calls coffee a risk factor.

Most of the coffee drinkers in your study also smoke. At the viva the examiner asks one question.

“How do you know that odds ratio is due to coffee, and not to smoking or to the way you chose your patients?”

Two types of error

Why does a study give a different answer from the truth?

A result can differ from the true value in two ways. Random error scatters results on both sides of the truth. Systematic error, called bias, shifts results in one direction.

true pressure 120 mmHgRandom errorreadings fall on both sides of the true valueSystematic errorthe cuff reads high, so every reading is shifted up8 mmHg too high100110120130140mmHg
Made-up readings for teaching. Each dot is one reading from the same patient. Random error becomes smaller when you take more readings and average them. Systematic error does not. The average of 100 readings from a cuff that reads high is still too high.
FeatureRandom errorSystematic error (bias)
DirectionEither side of the true valueOne direction
Larger sampleError becomes smallerError stays the same
AffectsPrecision (reliability)Accuracy (validity)
Shown byStandard error and confidence intervalNot shown by the confidence interval
ExampleChance variation between samples or readingsA miscalibrated cuff, hospital-only controls, recall bias
Reduced byLarger sample, repeated measurementsBetter design: sampling, blinding, standard methods

A third reason for a wrong answer is confounding, covered below. The sampling chapter shows sampling error and bias in survey samples.

Errors other than chance variation between samples are called non-sampling errors. The most common is non-response. It does two kinds of harm. Fewer people answer, so the estimate is less precise, and the people who do not answer usually differ from those who do, so the estimate is biased. A larger sample corrects only the first. For the second, make repeated efforts to reach a random subsample of non-responders and compare them with responders. Record a non-response as missing, separately from a true zero and from "not applicable".

Where bias enters

At which stage of a study can bias enter?

Bias is a systematic error in the design, conduct, analysis or reporting of a study that moves the result away from the true value. Name each bias by the stage at which it enters.

Choosewho is studiedselection biasBerkson's biashealthy worker effectMeasureexposure and outcomeinformation biasrecall, interviewerand observer biasAnalysecompare the groupsconfoundinga third factor linkedto exposure and outcomeReportpublish the resultspublication biasstudies with negativeresults unpublished
Most bias cannot be corrected once the data are collected. A confounder that was measured is the main exception, because it can be adjusted for in the analysis. Plan how you will prevent each bias when you write the protocol.

Selection bias

Who got into your study, and who did not?

Selection bias occurs when the people in a study differ systematically from the population you want to describe, so the estimate or the association is wrong.

Community: 10,000 peoplepercentage who also have disease AHas B10.0%No B10.0%odds ratio 1.0In hospital: 628 peoplepercentage who also have disease AHas B29.4%No B15.1%odds ratio 2.34people with bothdiseases areadmitted more often
Teaching example with made-up numbers. In the community, disease A affects 10% of people whether or not they have disease B, so the odds ratio is 1.0. Hospital admission is 30% for people with both diseases, 8% for one disease and 5% for neither. Among the 628 inpatients, 29.4% of people with B have A, against 15.1% of people without B, and the odds ratio becomes 2.34. This is Berkson's bias, also called admission rate bias.

Common types of selection bias

  • Berkson's bias (admission rate bias): an exposure-disease association in hospital patients is distorted because both affect the chance of admission. Take controls from the general population, or use new cases.
  • Healthy worker effect: people must be healthy enough to be employed, so a workforce has lower mortality than the general population, which includes people too ill to work (McMichael, 1976). Compare workers with other workers.
  • Volunteer bias: volunteers differ from the population of interest, in socio-demographic factors and in their attitudes to the study.
  • Non-response bias: people who do not respond differ from those who do in their exposures or outcomes.
  • Attrition bias (loss to follow-up): unequal losses from the study groups change the characteristics of the groups.
  • Prevalence-incidence (Neyman) bias: a study of existing cases misses people who died quickly or recovered. Use new (incident) cases.
  • Healthy user bias: people who take up and continue a preventive treatment tend to be healthier in other ways, such as exercising, not smoking and attending health checks.

Prevent selection bias at the design stage. Define the population, use probability or consecutive sampling, take cases and controls from the same source population, and keep response and follow-up high. Analysis cannot reliably correct it.

Information bias

Was information collected the same way in every group?

Information bias arises from systematic differences in how information is collected, recalled, recorded or handled.

  • Recall bias: people remember past exposures with different accuracy or completeness. Parents of a child with a serious illness may search their memory harder than parents of a healthy child.
  • Interviewer bias: an interviewer who knows who is a case asks or probes cases and controls differently. Use a standard questionnaire, neutral wording, and interviewers who do not know the group or the hypothesis.
  • Observer bias: the person recording an observation, such as a blood pressure or a scan finding, records it with a systematic error, often shaped by what they expect. Use observers who do not know the group.
  • Misclassification: people are put in the wrong exposure or outcome category. If the errors are similar in all groups (non-differential), the association tends to be underestimated. If they differ between groups (differential), it can be overestimated or underestimated.
  • Detection bias: the groups differ in how outcomes are looked for or confirmed.
  • Hawthorne effect: people change their behaviour because they know they are being observed.

To prevent information bias, blind the people who measure outcomes, use standard tools and written protocols, prefer records made before the outcome was known, and train observers.

Regression to the mean

Why do patients chosen for an extreme reading improve without treatment?

A single reading is the true value plus measurement error. People selected because their reading was extreme include many whose error pushed the reading up, so their next reading is, on average, closer to the population mean.

90110130150170190first systolic reading (mmHg)population mean 130selected: 150 or morefirst 155.50second145.55
Teaching example with made-up values. True systolic pressure has mean 130 mmHg and SD 10, and each reading adds a random error with SD 8. The 5.9% of people whose first reading is 150 or more have a mean first reading of 155.50. With no treatment, their mean second reading is expected to be 145.55, a fall of about 10 mmHg.

Expected second reading = mean + r × (first reading − mean)

r = 10² ÷ (10² + 8²) = 0.6098, so 130 + 0.6098 × 25.50 = 145.55

mean
population mean of the readings
r
reliability of one reading: true variance ÷ (true variance + error variance)

Barnett and colleagues (2005) described regression to the mean as a statistical phenomenon that can make natural variation in repeated data look like real change. Its effect is larger when measurement error is larger, and when follow-up measurements are examined only in a subgroup selected on a baseline value. A study that enrols patients because of a high reading and reports the fall at the next visit will show improvement even with a useless treatment.

  • Use a randomised control group. Both groups regress by the same amount, so the difference between them estimates the effect of the treatment.
  • Average several baseline readings. With two readings averaged, r rises to 0.76 and the expected fall in the same example is 6.0 mmHg instead of 10.0.
  • Select on one reading and use a later reading as the baseline. The measurement error that led to selection then does not enter the change.

Improvement through the natural course of a self-limiting illness is a different reason for change without treatment. A randomised control group deals with both.

Screening

Why do screen-detected cancers seem to survive longer?

Survival is counted from the date of diagnosis. Screening moves that date earlier and finds more slow-growing disease, so survival looks longer even if no death is delayed.

death at 65Diagnosed when symptoms appear5 yearsDiagnosed by screening8 yearslead time: 3 years50556065age (years)
Teaching example. The same cancer starts at age 50 and causes death at 65. Grey: disease present without symptoms. Red dot: date of diagnosis. Screening at 57 adds 3 years of lead time, so survival after diagnosis rises from 5 to 8 years with no change in the age at death.
Slow-growing disease4 of 5 foundFast-growing disease1 of 5 foundone screening round
Each bar is one person's disease before symptoms. Slow-growing disease stays in this phase longer, so one screening round (dashed line) finds 4 of the 5 slow-growing cases and 1 of the 5 fast-growing ones. Screen-detected cases therefore have a better prognosis whatever the treatment. This is length-time bias.
FeatureLead-time biasLength-time bias
What happensDiagnosis is moved earlier, but death is not delayedScreening finds slow-growing disease more often than fast-growing disease
Survival after diagnosisLonger by the lead timeLonger because the cases found have a better prognosis
AffectsEvery screen-detected caseThe mix of cases that screening finds
Avoided byA randomised trial counting deaths, with time measured from randomisationAnalysing everyone randomised to screening, not only screen-detected cases

Overdiagnosis is the detection of disease that would never have caused symptoms or death in the person's lifetime. These people are counted as survivors and raise survival figures further. People who attend screening also tend to be healthier than people who do not, which is a form of selection bias.

A screening programme is tested fairly by a randomised trial that compares deaths from the disease in the group invited to screening with the control group.

Reporting

Why do published studies look more positive than all the studies that were done?

Publication bias occurs when the chance of a study being published depends on its results.

no effectfavours treatmentfavours controllarge studiessmall studiesdashed: not published
Made-up meta-analysis for teaching. Each circle is one study, placed by its effect (left of the dashed line: benefit) and its size (large studies at the top). Without bias, studies form a symmetrical funnel around the true effect. Here small studies that found no benefit were not published (dashed circles), so the published studies suggest more benefit than there is. Egger and colleagues (1997) described a regression test for this asymmetry. Asymmetry has other possible causes, so it suggests bias but does not prove it.

In a follow-up of research projects approved by an Oxford ethics committee, 285 had been analysed. Studies with statistically significant results were more likely to be published than studies that found no difference (adjusted odds ratio 2.32) (Easterbrook and colleagues, 1991).

Related reporting biases include outcome reporting bias (only some of the prespecified outcomes are reported, depending on the results) and language bias (a review that reads only English-language studies can reach a biased answer). Trial registration, a published protocol, and searches of trial registries and non-English sources reduce them.

Other sources of error

Which other errors can bias a study?

Bias also enters through how a study is run, recorded and analysed. Each error below has a practical safeguard.

ErrorWhat happensSafeguard
ContaminationControl participants receive some of the intervention, for example by learning from treated neighbours, so the difference between groups is reducedKeep the groups apart, or randomise clusters such as villages or wards
Instrument errorA device reads consistently high or lowCalibrate against a reference standard, and use the same device for all groups
Digit preferenceReadings are rounded to numbers ending in 0 or 5Automated devices, training, and a check of the last digits of recorded values
Social desirabilityRespondents under-report behaviour they see as unacceptable and over-report behaviour they see as acceptableAnonymous self-completed questionnaires, and objective measures where possible
Protocol deviationsParticipants receive the wrong or an incomplete intervention, or ineligible people are enrolledMonitoring, and analysis by the group assigned
Data entry and coding errorsValues are misread, mistyped or miscodedDouble data entry, and range and consistency checks
Decisions about outliersExtreme values are kept or removed to suit the resultState the rule before the analysis, and report results with and without them
Immortal time biasIn a cohort, time before a patient starts a drug is counted as time on the drug, although the outcome could not have occurred during itClassify each person's follow-up time by actual exposure, from the date treatment began

Suissa (2008) defined immortal time as a span of cohort follow-up during which, because of the way exposure is defined, the outcome under study could not occur. The bias was first identified in the 1970s in cohort studies of survival after heart transplantation. It has since appeared in observational studies that reported various medicines to be extremely effective.

Errors of analysis and interpretation, such as testing many outcomes and reporting only the significant ones, removing inconvenient data, or reading a correlation as a cause, are covered in the Practical Statistics chapter on statistical fallacies.

Confounding

Is a third factor mixing into the comparison?

Confounding occurs when the effect of the exposure is mixed with the effect of a third factor that is linked to both the exposure and the outcome.

SmokingCoffeeMI?more coffeemore MIOdds ratio, coffee and MIAll patients3.78Smokers1.0Non-smokers1.00.5125
Teaching example with made-up numbers, using the thesis from the start of this chapter. Among controls, 80% of smokers drink coffee against 10% of non-smokers. Among people who do not drink coffee, 67% of smokers are cases against 20% of non-smokers. The crude odds ratio is 3.78. Within smokers and within non-smokers it is 1.0. Smoking produced the whole association.

Three conditions for a confounder

  1. It is associated with the exposure (smokers drink more coffee).
  2. It is a risk factor for the outcome, including in people who are not exposed (smoking causes MI in non-coffee drinkers).
  3. It is not on the causal pathway: the exposure does not cause it.
GroupCoffee: casesCoffee: controlsNo coffee: casesNo coffee: controlsOdds ratio
Smokers804020101.0
Non-smokers1040903601.0
All patients90801103703.78

How to control confounding

StageMethodHow it works
DesignRandomisationBalances known and unknown confounders between groups, on average
DesignRestrictionEnrol only one level of the confounder (for example, non-smokers only)
DesignMatchingChoose controls with the same confounder values as each case
AnalysisStratificationEstimate the association within each level, then combine (Mantel-Haenszel)
AnalysisMultivariable regressionAdjust for several confounders at once
AnalysisStandardisationCompare rates after applying a common age (or other) structure
AnalysisPropensity scoresCompare people with a similar predicted probability of being exposed

ORMH = Σ(a × d ÷ n) ÷ Σ(b × c ÷ n)

a, b
exposed cases and exposed controls in one stratum
c, d
unexposed cases and unexposed controls in the same stratum
n
total number of people in that stratum

(5.33 + 7.2) ÷ (5.33 + 7.2) = 12.53 ÷ 12.53 = 1.0

Smokers (n = 150): 80 × 10 ÷ 150 = 5.33 and 40 × 20 ÷ 150 = 5.33. Non-smokers (n = 500): 10 × 360 ÷ 500 = 7.2 and 40 × 90 ÷ 500 = 7.2. Only measured confounders can be adjusted for. Randomisation is the only method that also deals with unmeasured ones.

With many confounders, a propensity score, the predicted probability of being exposed given the measured confounders, lets exposed and unexposed people with similar scores be compared. It is described in the Practical Statistics chapter on propensity scores. Like regression, it adjusts only for confounders that were measured.

Confounding by indication is common in studies of treatments. The condition for which a drug is prescribed, not the drug, causes the outcome. Sicker patients receive the treatment, and the treatment appears harmful.

Interactive

Change how smoking is linked to coffee and to MI, and compare the crude and Mantel-Haenszel odds ratios

A third factor confounds only when it is linked to both the exposure and the outcome. Stratifying by the factor shows whether the crude odds ratio was distorted.

Try this: move "Coffee drinkers among smokers" down to 10% and watch the crude odds ratio come down to the odds ratios within each smoking group.

Teaching example with its own made-up numbers: 200 MI cases and 500 controls (100 smokers, 400 non-smokers). Among non-smokers, 10% drink coffee.

80%

Link between smoking and the exposure. Non-smokers: 10%

50%

Link between smoking and the outcome. Controls: 20%

Effect of coffee itself

True odds ratio within each smoking group

GroupCoffee: cases (a)Coffee: controls (b)No coffee: cases (c)No coffee: controls (d)Odds ratio
Smokers808020201.00
Non-smokers1040903601.00
All patients901201103802.59
All patients (crude)2.59Smokers1.00Non-smokers1.00Mantel-Haenszel1.000.51251020

ORMH = (8.00 + 7.20) ÷ (8.00 + 7.20) = 15.20 ÷ 15.20 = 1.00

Smoking is linked to coffee drinking and to MI, so the crude odds ratio (2.59) differs from the odds ratios within smokers and within non-smokers. The Mantel-Haenszel odds ratio (1.00) combines the strata and removes the effect of smoking.

Effect modification

Or is the effect really different in different groups?

Effect modification is present when the strength of an association differs across levels of a third factor. It is a real finding to report, not an error to remove.

Under 50 years1.050 years and over6.0Both ages pooled2.67describes neither group0.512510odds ratio
Teaching example with made-up numbers. An exposure has no effect in people under 50 (odds ratio 1.0) and a large effect in people aged 50 and over (6.0). Pooling both ages gives 2.67, which describes neither group. Report each age group separately.
FeatureConfounderEffect modifier
What it isA third factor that distorts the exposure-outcome associationA third factor across whose levels the association truly differs
NatureA problem in the study, to be removedA finding about the population, to be described
Stratum estimatesSimilar to each other, but different from the crude estimateDifferent from each other
What you doAdjust, and report the pooled (Mantel-Haenszel or regression) estimateReport each stratum separately, and test for interaction
Effect of designRemoved by randomisation, restriction or matchingNot removed by design, because it is a property of the disease or population
ExampleSmoking in coffee and MI (crude 3.78, both strata 1.0)Age above (1.0 under 50, 6.0 at 50 and over)

Decide

Is the third factor a confounder, a mediator or an effect modifier?

Answer these questions about the factor.

Is the third factor more common in the exposed group than in the unexposed group?

Third factor: all possible answers
  • Confounder: Control it. At the design stage use randomisation, restriction or matching. At the analysis stage use stratification (Mantel-Haenszel) or multivariable regression.
  • Mediator (on the causal pathway): Do not adjust for it when you want the total effect of the exposure. Adjusting removes part of the real effect.
  • Effect modifier: Report the association separately for each level of the factor. Do not combine the strata into one adjusted estimate.
  • Neither a confounder nor an effect modifier: The factor does not distort or change this association. It does not need adjustment for this question.

Interactive

Read a study and name the bias

Ask at which stage the error entered: choosing participants, measuring, analysing or reporting.

Try this: read the study, choose the bias, then read the explanation. You can change your answer.

Study 1 of 12 · teaching scenarios

Mothers of babies with a birth defect and mothers of healthy babies are asked, after the birth, which medicines they took in pregnancy. The mothers of affected babies report more medicines.

Choose a bias to see the explanation.

Comparison

Is the result correct for the patients studied, and does it apply to others?

Internal validity asks whether the result is correct for the people studied. External validity asks whether it applies to other people and settings.

FeatureInternal validityExternal validity
QuestionIs the result correct for the people in the study?Does the result apply to other patients and settings?
Threatened bySelection bias, information bias, confounding, loss to follow-upNarrow eligibility criteria, a single centre, unusual care, highly selected participants
Protected byRandomisation, allocation concealment, blinding, complete follow-upBroad eligibility, several centres, usual-care settings
Other nameValidity of the comparisonGeneralisability
OrderNeeded firstJudged only after internal validity is acceptable

Randomisation, allocation concealment and blinding are described in detail in the clinical trials chapter.

Association or causation

When does an association show cause and effect?

After chance, bias and confounding have been considered, Hill (1965) set out nine viewpoints to weigh before deciding that an association is causal.

  1. Strength: how large the association is.
  2. Consistency: whether it has been seen repeatedly, by different people, in different places, circumstances and times.
  3. Specificity: whether the exposure is linked to a particular disease and not to many.
  4. Temporality: whether the exposure came before the disease.
  5. Biological gradient: whether more exposure goes with more disease, a dose-response relation.
  6. Plausibility: whether a biological explanation exists. Hill noted that this depends on the knowledge of the day and cannot be demanded.
  7. Coherence: whether the association fits what is known of the natural history and biology of the disease.
  8. Experiment: whether preventive action taken because of the association does in fact prevent the disease.
  9. Analogy: whether a similar exposure is known to cause a similar effect.

Hill wrote that none of the nine viewpoints can bring indisputable evidence for or against cause and effect, and none is required in every case. They help to answer one question: is there any other way of explaining the facts that is as likely, or more likely, than cause and effect?

Validity of a measurement tool

Does your questionnaire or test measure what it is meant to measure?

A measurement tool is valid if it measures the concept it is meant to measure. Each type of validity is a different kind of evidence for this.

TypeQuestion it answersHow it is checked
Face validityDoes the tool look, to users, as if it measures the concept?Ask people who will use or answer it
Content validityDoes it cover all the important parts of the concept?An expert panel reviews every item
Construct validity: convergentDoes it agree with measures of related concepts?Correlation with an existing related scale
Construct validity: discriminantDoes it disagree with measures of unrelated concepts?Low correlation with an unrelated scale
Construct validity: structureDo the items group into the expected dimensions?Factor analysis
Criterion validity: concurrentDoes it agree with a gold standard measured at the same time?Correlation, or sensitivity and specificity
Criterion validity: predictiveDoes it predict a later outcome?Correlation with, or ROC analysis against, the later outcome

A clinical score or prediction model needs similar evidence. Check its discrimination, how well it separates patients with and without the outcome (the C statistic, which is the area under the ROC curve), and its calibration, how closely predicted risks match observed risks. A score usually performs less well in new patients than in the sample it was developed in, so test it in a separate validation sample, or correct for this optimism. The Practical Statistics chapter on bootstrap validation shows how.

Reliability

Does your tool give the same result when the measurement is repeated?

A tool is reliable if repeated measurements under the same conditions give similar results. Reliability reflects random error. A tool can be reliable and still not valid.

TypeWhat is repeatedUsual statistic
Test-retestSame people, same tool, two occasionsIntraclass correlation coefficient (ICC), or kappa for categories
Inter-raterDifferent raters, same patientsKappa, or ICC
Intra-raterSame rater, same patients, two occasionsKappa, or ICC
Internal consistencyDifferent items of one scale that measure the same conceptCronbach's alpha
Parallel forms and split-halfTwo versions, or two halves, of the same toolCorrelation between the two

κ = (po − pe) ÷ (1 − pe)

p o
observed proportion of agreement
p e
agreement expected by chance, from each rater's totals

Teaching example: two radiologists read the same 100 chest films. Both call 40 abnormal and 45 normal. Radiologist A alone calls 5 abnormal, and radiologist B alone calls 10 abnormal. A calls 45% abnormal and B calls 50%.

po = 85 ÷ 100 = 0.85, pe = 0.45 × 0.50 + 0.55 × 0.50 = 0.50

κ = 0.35 ÷ 0.50 = 0.70

Kappa is 1 when agreement is perfect and 0 when agreement is no better than chance. For two methods that measure the same continuous quantity, a high correlation does not show that they agree. Use the graphical method for agreement described by Bland and Altman (1986).

The intraclass correlation coefficient has several forms, so a report should state which one was used. Koo and Li (2016) suggested reading the 95% confidence interval of an ICC as poor reliability below 0.5, moderate from 0.5 to 0.75, good from 0.75 to 0.9, and excellent above 0.90. Kappa and the ICC are worked through in the Practical Statistics chapters on kappa and agreement and on the ICC and Bland-Altman analysis.

FeatureReliabilityValidity
QuestionDoes the tool give the same result when repeated?Does the tool measure what it is meant to measure?
Error it reflectsRandom errorSystematic error
TypesTest-retest, inter-rater, intra-rater, internal consistencyFace, content, construct, criterion
StatisticsKappa, ICC, Cronbach's alphaCorrelation with a criterion, sensitivity and specificity, factor analysis
RelationshipNecessary for validityRequires reliability, but reliability does not guarantee it

Interactive

Type counts into an agreement table and watch kappa change

Kappa compares the agreement observed with the agreement expected by chance from the totals of each rater.

Try this: press "Abnormal films common". Observed agreement stays at 85%, but kappa falls, because more agreement is expected by chance.

Teaching example: two radiologists read the same chest films. Type any counts into the table.

Radiologist B: abnormalRadiologist B: normal
Radiologist A: abnormal
Radiologist A: normal
0.70kappa

po = (40 + 45) ÷ 100 = 0.850

pe = 0.450 × 0.500 + 0.550 × 0.500 = 0.500

κ = (0.850 − 0.500) ÷ (1 − 0.500) = 0.70

Radiologist A calls 45.0% of films abnormal and radiologist B calls 50.0%.

Observed85.0%By chance50.0%0%100%

Kappa is 1 when agreement is perfect and 0 when agreement is no better than chance. It is below 0 when the raters agree less often than chance would predict.

Internal consistency

Do the items of a scale measure the same thing?

When a questionnaire adds several items into one score, the items should agree with each other. Internal consistency measures how far they do.

Split-half reliability divides the items into two halves, for example odd and even items, and correlates the two half-scores. Each half is only half as long as the full scale, so the correlation is corrected with the Spearman-Brown formula.

Full-scale reliability = 2r ÷ (1 + r) = 2 × 0.60 ÷ 1.60 = 0.75

Cronbach's alpha uses every item instead of one split. In its standardised form it depends only on the number of items and the average correlation between them.

α = k × r̄ ÷ [1 + (k − 1) × r̄]

k
number of items
r̄
average correlation between pairs of items
Items (k)Average inter-item correlationAlpha
50.400.77
100.400.87
200.400.93
100.200.71
  • Adding items raises alpha even when the items correlate no more strongly, so a long scale can have a high alpha without being a better scale.
  • Tavakol and Dennick (2011) noted that a high alpha, above 0.90, may mean that some items are redundant, asking the same question in a different form.
  • Alpha does not show that the items measure a single concept. If a questionnaire has several subscales, calculate alpha for each subscale (Tavakol and Dennick, 2011).
  • For items answered yes or no, the equivalent coefficient is the Kuder-Richardson formula 20 (KR-20).

Teaching numbers, calculated in Python. More on the reliability and validity of scales is in the Practical Statistics chapter on reliability and validity of scales.

Comparison

Accuracy or precision: what is the difference?

Accuracy is how close a measurement is to the true value. Precision is how close repeated measurements are to each other.

Accurate and precisesmall scatter, on targetPrecise, not accuratesmall scatter, off targetAccurate, not precisewide scatter, on targetNeitherwide scatter, off target
Each red dot is one repeated measurement, and the centre is the true value. Accuracy corresponds to validity (little systematic error). Precision corresponds to reliability (little random error).
FeatureAccuracyPrecision
MeaningCloseness of a result to the true valueCloseness of repeated results to each other
Error that reduces itSystematic error (bias)Random error
Expressed asBias: mean result minus the reference valueSD or coefficient of variation of repeated results
Improved byCalibration against a reference standardBetter technique, more replicates
Research termValidityReliability

Coefficient of variation (CV) = SD ÷ mean × 100

Teaching example from a laboratory: repeated glucose measurements have a mean of 100 mg/dL and an SD of 4, so CV = 4.0%. Repeated creatinine measurements have a mean of 1.0 mg/dL and an SD of 0.1, so CV = 10.0%. The SD of glucose looks larger, but creatinine is the less precise test. The SD is in the units of each test and depends on the level measured. The CV is a percentage of the mean, so it compares tests and levels directly.

Interactive

Change the systematic and random error and watch accuracy and precision change

Systematic error moves every reading in one direction. Random error scatters readings on both sides.

Try this: set the systematic error to +8 mg/dL and choose 100 readings. The average is close to 108 mg/dL, not 100, however many readings you take.

Teaching simulation: one laboratory control sample with a true glucose of 100 mg/dL, measured repeatedly.

+5 mg/dL

Removed by calibration against a reference standard

4 mg/dL

Reduced by better technique

Readings
true value 100average 105.0708090100110120130glucose (mg/dL)
Mean of 20 readingsBias (mean − 100)SD of readingsCV
105.0 mg/dL+5.0 mg/dL4.0 mg/dL3.8%

Bias measures accuracy and the SD or CV measures precision. More readings make the average more stable, but its distance from 100 mg/dL stays close to the systematic error you set.

Radiology

Why do experienced radiologists miss or misread findings?

Reading an image means searching it and then deciding what the findings mean. Busby and colleagues (2018) reviewed the cognitive biases that affect both steps, with ways to reduce each.

  • Satisfaction of search: less attention to further abnormalities after the first one is found. Complete a systematic second search.
  • Satisfaction of report (alliterative error): repeating the impression in a previous report, one's own or a colleague's. Form your impression before reading the earlier report.
  • Anchoring bias: staying with the first impression despite later information that contradicts it. Try to disprove the first diagnosis.
  • Confirmation bias: looking for evidence that supports a diagnosis instead of testing alternatives.
  • Framing bias: reaching a different conclusion from the same image depending on how the clinical history is presented. Look at the image first, then read the history.
  • Availability bias: judging a diagnosis as likely because examples come to mind easily, such as a recent missed case.
  • Attribution bias: attributing findings to a patient's characteristics, such as age or sex, given in the history.
  • Premature closure: accepting a diagnosis as final too early in the evaluation.

Kim and Mansfield (2014) reviewed 656 examinations with a delayed diagnosis and classified 1,269 errors. The most common types were under-reading (42%) and satisfaction of search (22%). The missed diagnosis was also not recognised on later examinations in 196 of the 656 cases (30%).

Bias in imaging research

  • Spectrum bias: a test is studied in a different range of patients from the people it is intended for, which changes its sensitivity and specificity.
  • Partial reference (verification or work-up) bias: only some patients receive the reference standard, often those with a positive test.
  • Differential reference bias: patients receive different reference standards.
  • Incorporation bias: the result of the test being studied forms part of the reference standard.

Sensitivity, specificity and the design of diagnostic accuracy studies are covered in the diagnostic tests chapter.

Clinical trials

Which trial methods prevent which bias?

Each major bias in a randomised trial has a matching safeguard, and CONSORT 2025 asks authors to report it.

BiasWhat goes wrongSafeguardCONSORT 2025 item
Selection (allocation) biasThe recruiter knows the next allocation and enrols selectivelyRandom sequence, allocation concealment17a, 18, 19
Performance biasGroups receive different care apart from the interventionBlinding of participants and care providers20a, 20b
Detection biasOutcomes are judged differently by groupBlinded outcome assessors20a
Attrition biasUnequal losses between groupsComplete follow-up, analysis by assigned group, stated handling of missing data21b, 21c, 22b
Reporting biasOnly favourable outcomes are reportedRegistration, prespecified outcomes, reporting of changes2, 10, 14

How easily would a significant result become non-significant?

The Fragility Index (Walsh and colleagues, 2014) is the number of patients in the group with fewer events whose status would have to change from non-event to event for the P-value to reach 0.05 or more. In 399 trials in high-impact journals the median was 8, and 25% had a Fragility Index of 3 or less.

Teaching example: 10 of 100 patients die with a new drug and 25 of 100 with control, Fisher's exact P = 0.0085. Changing one survivor at a time to a death in the drug group gives P = 0.016 at 11 deaths, 0.028 at 12, 0.046 at 13 and 0.073 at 14. The Fragility Index is 4.

By specialty

Bias problems by specialty

A records-based study finds that patients on a newer antihypertensive have more strokes. Doctors had prescribed it mainly to patients with the highest blood pressure.

Confounding by indication. The reason for prescribing is linked to the outcome. Compare patients of similar severity, or randomise.

Common myths

Common mistaken beliefs

  • Myth: A large sample removes bias.

    A larger sample reduces random error only. A biased estimate stays biased, with a narrower confidence interval.

  • Myth: Randomisation removes all bias.

    Randomisation balances confounders at the start. Observer bias, loss to follow-up and selective reporting need blinding, complete follow-up and registration.

  • Myth: Adjusting for every available variable is the safest analysis.

    Adjusting for a factor on the causal pathway removes part of the real effect. Choose adjustment variables before the analysis, using the three conditions for a confounder.

  • Myth: A reliable questionnaire is a valid questionnaire.

    A tool can give the same wrong answer every time. Reliability is needed for validity but does not guarantee it.

  • Myth: Longer survival in screen-detected cases proves that screening works.

    Lead-time and length-time bias lengthen survival after diagnosis without delaying death. Judge screening by deaths from the disease in a randomised trial.

  • Myth: A high correlation between two measurement methods shows that they agree.

    Correlation is misleading for agreement (Bland and Altman, 1986). Two methods can be highly correlated and still give different values.

Why this matters to you

Five consequences of ignoring bias and confounding

  1. Protocol sent back

    A case-control protocol with hospital controls and no plan for confounders is returned by the research committee.

  2. A wrong thesis conclusion

    An unadjusted odds ratio produced by a confounder becomes the main finding of your thesis.

  3. Questions you cannot answer at the viva

    Examiners ask how your controls were chosen, who measured the outcome, and which confounders you adjusted for.

  4. Misreading papers

    Longer survival after screening and benefits seen in observational drug studies are often produced by bias or confounding.

  5. Harm to patients

    Treatments adopted on the basis of biased studies may be ineffective or harmful.

History

History of bias, confounding and validity

  1. 1946

    Joseph Berkson

    Showed that an association between diseases seen in hospital patients may not exist in the population, because of how patients come to be admitted.

  2. 1951

    Lee Cronbach

    Published coefficient alpha and showed that it equals the mean of all possible split-half reliability coefficients of a test.

  3. 1955

    Lee Cronbach and Paul Meehl

    Published "Construct validity in psychological tests", on how to judge a test that measures a concept with no single criterion to compare against.

  4. 1957

    Donald Campbell

    Introduced the distinction between internal validity and external validity.

  5. 1960

    Jacob Cohen

    Proposed kappa, a measure of agreement between two raters that allows for agreement expected by chance.

  6. 1965

    Austin Bradford Hill

    Published "The environment and disease: association or causation?", with nine viewpoints for judging whether an association is causal.

  7. 1976

    Anthony McMichael

    Described the "healthy worker effect": workers must be healthy enough to be employed, so comparing them with the general population underestimates their mortality.

  8. 1978

    Roberts, Spitzer, Delmore and Sackett

    Published an empirical demonstration of Berkson's bias: respiratory and locomotor disease were associated in people who had been in hospital, but essentially independent in the community sample.

  9. 1979

    David Sackett

    Listed 56 biases, grouped by the stage of research at which they arise, and proposed an annotated catalogue of bias.

  10. 1986

    Martin Bland and Douglas Altman

    Showed that correlation is misleading when judging whether two measurement methods agree, and described an alternative graphical approach.

  11. 1991

    Easterbrook and colleagues

    Found that studies with statistically significant results were more likely to be published (adjusted odds ratio 2.32).

  12. 1997

    Egger and colleagues

    Proposed a simple test of funnel plot asymmetry to detect bias in meta-analyses.

  13. 2008

    Samy Suissa

    Described immortal time bias in pharmaco-epidemiology: follow-up time during which, because of how exposure is defined, the outcome could not occur.

  14. 2014

    Walsh and colleagues

    Proposed the Fragility Index for trials with a statistically significant binary or time-to-event outcome.

  15. 2019

    Sterne and colleagues

    Published RoB 2, the revised Cochrane tool for assessing risk of bias in randomised trials.

Guidelines

Bias in guidelines

ICH E9 (1998), glossary and section 2.3

Bias is “the systematic tendency of any factors associated with the design, conduct, analysis and evaluation of the results of a clinical trial to make the estimate of a treatment effect deviate from its true value.” “The most important design techniques for avoiding bias in clinical trials are blinding and randomisation.”

CONSORT 2025, items 18, 20a and 30

Report the “mechanism used to implement the random allocation sequence (eg, central computer/telephone; sequentially numbered, opaque, sealed containers), describing any steps to conceal the sequence until interventions were assigned” (18), “who was blinded after assignment to interventions” (20a), and “trial limitations, addressing sources of potential bias, imprecision, generalisability, and, if relevant, multiplicity of analyses” (30).

STROBE (2007), items 9 and 12(a)

Observational studies must “describe any efforts to address potential sources of bias” (9) and “describe all statistical methods, including those used to control for confounding” (12a).

Cochrane RoB 2 (2019)

Risk of bias in a randomised trial is judged in five domains: bias arising from the randomization process, bias due to deviations from intended interventions, bias due to missing outcome data, bias in measurement of the outcome, and bias in selection of the reported result.

QUADAS-2 (2011)

Diagnostic accuracy studies are assessed in four domains: patient selection, index test, reference standard, and flow and timing.

Reading a paper

Five checks for bias in any study

  • How were participants selected, and were the groups comparable at the start?
  • Were outcome assessors, and where possible participants and carers, blinded to the group?
  • How many were lost to follow-up, and were the losses similar between groups?
  • Which confounders were measured and adjusted for, and was any important one missing?
  • Were the outcomes and analyses prespecified in a registered protocol?

Why is it called that?

Word surgery

“Bias”

In everyday speech

Prejudice, a personal leaning for or against someone.

In research

A systematic error in how a study selects, measures, analyses or reports, which moves the estimate in one direction. The investigator does not have to be prejudiced.

“Confounding”

In everyday speech

Confusing or astonishing.

In research

Mixing of the effect of the exposure with the effect of a third factor that is linked to both the exposure and the outcome.

“Precision”

In everyday speech

Exactness, being correct.

In research

How close repeated measurements are to each other, whether or not they are close to the true value.

Memory trick

A confounder is linked to the exposure, is a risk factor for the outcome, and is not caused by the exposure.

Bias

French biais: "a slant, an oblique"

In the game of bowls, the word was used for balls made heavier on one side, which curve away from a straight line. A biased study moves away from the true value in one direction.

Confound

Latin confundere: "to pour together, mix up"

In confounding, the effect of the exposure and the effect of a third factor are mixed together.

Valid

Latin validus: "strong, effective"

A valid study or tool gives well-founded results: it measures what it claims to measure.

Reliable

rely + -able: "that may be relied on" (in statistics by 1910)

A reliable tool gives the same result when the measurement is repeated.

Accurate

Latin accuratus: "prepared with care"

Close to the true value.

Precision

Latin praecisio: "a cutting off"

Little scatter between repeated measurements.

Exam answers

Model answer outlines

Bias in research: definition, types and how to minimise it

DM Pulmonary Medicine 2025 · DM Clinical Immunology and Rheumatology 2025 · Ph.D. RM 2022 · MCh Surgical Oncology 2009 · MD Community Medicine 2005

Definition: systematic error in the design, conduct, analysis or reporting of a study that moves the estimate away from the true value in one direction. Unlike random error, it does not become smaller as the sample size increases.

StageBiasExamplePrevention
Selecting participantsSelection: Berkson's, healthy worker, volunteer, non-response, attrition, prevalence-incidenceHospital controls in a case-control studySame source population, probability or consecutive sampling, high response and follow-up
MeasuringInformation: recall, interviewer, observer, misclassification, detectionCases recall exposures more completelyBlinding, standard tools, records made before the outcome
AnalysingConfounding (often classed separately)Smoking in coffee and MIRandomisation, restriction, matching, stratification, regression
ReportingPublication, outcome reporting, languageNegative trials left unpublishedRegistration, prespecified outcomes, search of registries
ScreeningLead-time, length-time, overdiagnosisLonger survival after screeningRandomised trial with deaths from the disease as outcome
  • Sackett (1979) listed 56 biases, grouped by the stage of research at which they arise.
  • Assess risk of bias with RoB 2 (randomised trials) or QUADAS-2 (diagnostic accuracy studies).
  • Report efforts to address bias: STROBE item 9 for observational studies, CONSORT 2025 item 30 for trials.
  • Regression to the mean: people selected for an extreme reading move towards the average at the next reading without treatment (teaching example: 155.50 to 145.55 mmHg). Use a randomised control group and several baseline readings (Barnett et al., 2005).
  • Other sources of error: contamination, instrument error, digit preference, social desirability, protocol deviations, data entry errors, decisions about outliers, and immortal time bias in cohort studies of drugs (Suissa, 2008).

Bias in clinical trials, including vaccine trials

MD Pharmacology 2025 · MCh Gynaecological Oncology 2025
  1. Selection (allocation) bias: random sequence and allocation concealment (CONSORT 2025 items 17a, 18, 19).
  2. Performance bias: blinding of participants and care providers, with an identical-looking comparator (item 20b asks for the similarity of interventions).
  3. Detection bias: blinded outcome assessors, and the same case definition and surveillance method in both arms.
  4. Attrition bias: complete follow-up, analysis by assigned group (21b), and a stated method for missing data (21c).
  5. Reporting bias: registration and prespecified outcomes (items 2 and 14).
  6. Chance imbalance of confounders in small trials: stratified randomisation and adjusted analysis for prespecified prognostic factors.
  7. Vaccine trials: if the vaccine causes more injection-site reactions than the comparator, participants and staff may guess the allocation. Keep outcome assessment separate from monitoring of reactions.
  8. Observational vaccine effectiveness studies: healthy user bias, the healthy vaccinee effect, confounding by indication and outcome misclassification (Agampodi and colleagues, 2024).

How selection bias affects study inferences, and methods to minimise it

MD Community Medicine 2021

Definition: the people studied differ systematically from the population of interest.

  • Prevalence or mean values are wrong for the target population.
  • Associations can be created, exaggerated, hidden or reversed (Berkson's bias: odds ratio 1.0 in the community, 2.34 in hospital in the teaching example).
  • Results cannot be generalised, and the direction and size of the error are usually unknown.
  • Analysis cannot reliably correct it, so it must be prevented at the design stage.

Methods to minimise it:

  1. Define the target population and eligibility criteria clearly.
  2. Use probability sampling, or consecutive enrolment of every eligible patient.
  3. In case-control studies, take controls from the same source population as cases, and prefer new cases.
  4. In trials, randomise with allocation concealment and analyse by assigned group.
  5. Maximise response and follow-up, and compare responders with non-responders.
  6. Make repeated efforts to reach a random subsample of non-responders, and compare them with responders.
  7. Report numbers screened, enrolled, lost and analysed, with reasons.

Biases in cross-sectional, case-control and cohort studies, with the steps of a cohort study

MD Community Medicine 2016, 2019, 2021
DesignMain biasesPrevention
Cross-sectionalNon-response, volunteer, prevalence-incidence (only survivors are counted), recall, measurement errorProbability sampling, revisits, validated tools, standard measurement
Case-controlBerkson's, poor choice of controls, prevalence-incidence, recall, interviewer, confoundingPopulation controls, incident cases, blinded interviewers, records, matching or adjustment
CohortHealthy worker and volunteer bias at entry, loss to follow-up, detection bias, misclassification of exposure, confoundingComparable unexposed group, active follow-up, the same outcome checks for all, blinded assessors, adjustment

A cross-sectional study also cannot show whether exposure came before the outcome. This is a limitation of the design, not a bias.

Immortal time bias in cohort studies of treatment: time before a patient starts the drug, during which the outcome could not occur by the way exposure is defined, is counted as treated time (Suissa, 2008). Classify follow-up time by actual exposure.

Steps in the design of a cohort study:

  1. State the research question and hypothesis.
  2. Define exposure and outcome with clear criteria.
  3. Select exposed and unexposed groups (or one cohort with a range of exposure), all free of the outcome at the start.
  4. Measure exposure and possible confounders at baseline.
  5. Follow up both groups for the same period, with the same methods.
  6. Ascertain outcomes, preferably by assessors who do not know the exposure.
  7. Analyse incidence, relative risk and attributable risk, adjusting for confounders.
  8. Report according to STROBE.

Steps in an epidemiological study of the cause of a newly reported syndrome, and possible sources of bias

MD Community Medicine 2012
  1. Confirm that the cases are real: verify diagnoses and exclude laboratory or reporting errors.
  2. Write a case definition (clinical, laboratory, time, place, person).
  3. Search actively for further cases.
  4. Describe cases by time, place and person, and form hypotheses.
  5. Test hypotheses with a case-control study, which is quick and suits a new, uncommon condition. Follow with a cohort study if needed.
  6. Carry out laboratory and environmental studies.
  7. Start control measures and communicate the findings.

Possible sources of bias:

  • Misclassification from a loose or changing case definition.
  • Detection bias: publicity leads exposed people to be examined and reported more often.
  • Selection bias from hospital controls (Berkson's bias) or unrepresentative cases.
  • Recall bias. After publicity alleging a link between MMR vaccine and autism, parents of children diagnosed after the publicity more often recalled onset shortly after MMR than parents of children diagnosed before it (Andrews and colleagues, 2002).
  • Interviewer bias when interviewers know the hypothesis.
  • Confounding by factors linked to both the suspected exposure and the syndrome.

Confounding: definition, conditions and control, including in clinical trials. 'Confounding is not a bias': justify

MD Pharmacology 2025 · MD Community Medicine 2014, 2023 · Ph.D. RM 2021

Definition: distortion of an exposure-outcome association by a third factor linked to both.

Conditions: the factor is associated with the exposure, is a risk factor for the outcome in the unexposed, and is not on the causal pathway.

Example (teaching numbers): coffee and MI, crude odds ratio 3.78, odds ratio 1.0 in smokers and in non-smokers.

  • Design: randomisation, restriction, matching.
  • Analysis: stratification with the Mantel-Haenszel estimate, multivariable regression, standardisation, propensity scores.
  • In clinical trials: randomisation balances known and unknown confounders on average. Chance imbalance can occur in small trials, so use stratified randomisation for strong prognostic factors, check the baseline table, and prespecify adjusted analyses. Confounding by indication affects non-randomised comparisons of treatments.

Why confounding is often treated separately from bias:

  1. Bias is an error introduced by how the investigator selects, measures or reports. Confounding comes from real associations between the third factor, the exposure and the outcome that exist in the population before the study begins.
  2. A measured confounder can be controlled in the analysis. Selection and information bias usually cannot be corrected after data collection.
  3. The crude association correctly describes the data. It is wrong only as an estimate of the causal effect.
  4. Some sources, including the Catalogue of Bias, list confounding among the distortions of research, so state both views and give your reason.

From association to causation: once chance, bias and confounding are unlikely, weigh Hill's nine viewpoints (1965): strength, consistency, specificity, temporality, biological gradient, plausibility, coherence, experiment and analogy. None is required in every case.

Confounders versus effect modifiers, with examples

MD Pharmacology 2025 · MD Community Medicine 2022, 2024, 2025
FeatureConfounderEffect modifier
MeaningThird factor that distorts the associationThird factor across whose levels the association differs
NatureA problem to removeA finding to report
Stratum estimatesSimilar to each other, different from crudeDifferent from each other
ActionAdjust and report the pooled estimateReport each stratum, test for interaction
DesignRemoved by randomisation, restriction, matchingNot removed by design
Example (teaching)Smoking in coffee and MI: crude 3.78, strata 1.0 and 1.0Age: odds ratio 1.0 under 50, 6.0 at 50 and over

How to tell them apart: stratify by the factor. If the stratum estimates are similar to each other but differ from the crude estimate, the factor is a confounder. If the stratum estimates differ from each other, it is an effect modifier. Test this with a test of homogeneity (such as Breslow-Day) or an interaction term in regression. A factor can be both.

Validity and reliability: definitions and forms

Ph.D. RM 2013, 2023, 2024

Validity: the degree to which a tool or study measures what it is intended to measure. Reliability: the degree to which repeated measurements under the same conditions give the same result.

Forms of validity:

  • Study validity: internal (correct for the people studied) and external (generalisable).
  • Face: appears to measure the concept.
  • Content: covers all important parts of the concept, judged by experts.
  • Construct: behaves as the concept should, shown by convergent and discriminant correlations and factor analysis.
  • Criterion: agrees with a gold standard, measured at the same time (concurrent) or later (predictive).

Forms of reliability: test-retest, inter-rater, intra-rater (kappa or ICC), internal consistency (Cronbach's alpha), parallel forms and split-half.

Relationship: reliability concerns random error and validity concerns systematic error. A tool must be reliable to be valid, but a reliable tool can be consistently wrong.

Internal consistency: the split-half correlation is corrected by Spearman-Brown, 2r ÷ (1 + r). Cronbach's alpha = k r̄ ÷ [1 + (k − 1) r̄], for example 5 items with average correlation 0.40 give 0.77. Alpha rises with the number of items, a value above 0.90 may mean redundant items, and it should be calculated for each subscale (Tavakol and Dennick, 2011). KR-20 is used for yes/no items.

ICC: state the form used, and read its 95% CI as poor below 0.5, moderate 0.5 to 0.75, good 0.75 to 0.9 and excellent above 0.90 (Koo and Li, 2016). Validating a clinical score: discrimination (C statistic), calibration, and testing in a separate sample.

Designing a questionnaire for college students, and testing its reliability and validity

MD Community Medicine 2025

Components to consider in design:

  1. Objectives, and the list of variables each question will measure.
  2. Mode: self-administered (paper or online) or interviewer-administered.
  3. Question types: closed questions for analysis, a few open questions where needed.
  4. Wording: short, simple, one idea per question, no leading or double-barrelled questions, no jargon.
  5. Response options: exhaustive and non-overlapping categories, or Likert-type scales.
  6. Order: general before specific, sensitive questions (such as substance use or sexual behaviour) later.
  7. Layout, instructions and length that suit students.
  8. Translation and back-translation if a local language is used.
  9. Pretesting and a pilot study in a similar group of students.
  10. Ethics: informed consent, voluntary participation, anonymity and confidentiality.

Testing validity: face validity (students review it), content validity (expert panel), construct validity (factor analysis, correlation with related and unrelated scales), criterion validity (comparison with a gold standard where one exists).

Testing reliability: test-retest on a subsample (ICC or kappa), internal consistency of multi-item scales (Cronbach's alpha), and inter-rater reliability if interviewers administer it.

Report alpha for each subscale rather than for the whole questionnaire, and treat a value above 0.90 as a sign of possibly redundant items (Tavakol and Dennick, 2011).

Question design and pilot testing are covered in more detail in the data collection chapter.

Accuracy versus precision, and why imprecision is expressed as a coefficient of variation

MD Biochemistry 2014 · Ph.D. RM 2013
FeatureAccuracyPrecision
MeaningCloseness to the true valueCloseness of repeated results to each other
ErrorSystematic (bias)Random
MeasureBias: mean minus reference valueSD, CV
Improved byCalibrationBetter technique, replicates
Research analogueValidityReliability

CV = SD ÷ mean × 100. The SD is in the units of the test and depends on the level measured, so it cannot compare methods or concentrations. The CV is a unit-free percentage.

Example (teaching numbers): glucose mean 100 mg/dL, SD 4, CV 4.0%. Creatinine mean 1.0 mg/dL, SD 0.1, CV 10.0%. The larger SD belongs to the more precise test.

Evaluating a cancer screening test, and biases in screening, including lead-time bias

MCh Surgical Oncology 2004, 2019

Evaluating the test:

  1. Validity: sensitivity and specificity against a reference standard.
  2. Predictive values, which depend on prevalence in the screened population.
  3. Reliability: agreement between and within readers.
  4. Yield: cases detected per number screened, and the stage at detection.
  5. Acceptability, safety and cost.
  6. Effectiveness of the programme: a randomised trial comparing deaths from the cancer in people invited and not invited, analysed by allocation.

Biases:

  • Lead-time bias: earlier diagnosis lengthens survival from diagnosis without delaying death. Teaching example: onset at 50, death at 65. Diagnosis at symptoms (60) gives 5 years of survival, and at screening (57) gives 8 years. Lead time is 3 years.
  • Length-time bias: screening preferentially finds slow-growing tumours with a better prognosis.
  • Overdiagnosis: detection of cancers that would never have caused symptoms or death.
  • Volunteer (healthy screenee) bias: people who attend screening are healthier than those who do not.

Avoiding them: use deaths from the disease, not survival after diagnosis, as the outcome. Measure time from randomisation and analyse everyone randomised. The diagnostic tests chapter covers sensitivity, specificity and predictive values.

Steps in a meta-analysis, sources of bias, and how heterogeneity is addressed

MD Community Medicine 2023

Steps:

  1. Frame the question (population, intervention, comparison, outcome) and register a protocol.
  2. Search databases, trial registries and unpublished sources without language limits.
  3. Screen studies and extract data, with two reviewers working independently.
  4. Assess risk of bias in each study (for example RoB 2 for trials).
  5. Pool effect estimates with a fixed-effect or random-effects model, shown in a forest plot.
  6. Assess heterogeneity and publication bias.
  7. Grade the certainty of the evidence and report according to PRISMA 2020 (27-item checklist).

Sources of bias: publication bias, outcome reporting bias, language bias, double counting of studies published more than once, inclusion criteria chosen after seeing results, and bias within the included studies.

Detecting publication bias: funnel plot and Egger's regression test (Egger and colleagues, 1997).

Heterogeneity:

  • Types: clinical (patients, interventions), methodological (design, risk of bias), statistical (variation in results beyond chance).
  • Detection: inspection of the forest plot, Cochran's Q test, the I² statistic (percentage of variation due to heterogeneity rather than chance).
  • Handling: check the data for errors, use a random-effects model, explore causes with subgroup analysis or meta-regression, run sensitivity analyses (for example excluding studies at high risk of bias), or do not pool and describe the studies instead.

See the systematic review and meta-analysis chapter.

Biases in radiology reporting

MD Radio Diagnosis 2022

Cognitive biases in reporting (Busby and colleagues, 2018):

  • Satisfaction of search: a second abnormality is missed after the first is found. Use a systematic search.
  • Satisfaction of report (alliterative error): the impression of a previous report is repeated. Form your own impression first.
  • Anchoring and confirmation bias: the first impression is kept, and supporting evidence is sought. Try to disprove the first diagnosis.
  • Framing bias: the clinical history changes the reading. Review the images before the history.
  • Availability bias: recent or memorable cases seem more likely. Use known base rates.
  • Attribution bias and premature closure.

Size of the problem: in 656 examinations with delayed diagnoses, under-reading (42% of errors) and satisfaction of search (22%) were the most common errors (Kim and Mansfield, 2014).

Bias in imaging research: spectrum bias, partial and differential reference (verification) bias, incorporation bias, and readers who know the reference result. Inter-observer variation is measured with kappa.

System measures: structured reports and checklists, double reading for selected studies, peer review and discrepancy meetings, and radiologic-pathologic correlation.

Randomised clinical trials: external validity, allocation concealment, stratification, sensitivity analysis, Fragility Index

DM Critical Care Medicine 2022
  1. External validity: the extent to which trial results apply to patients and settings outside the trial. It depends on eligibility criteria, setting, the care delivered and the outcomes chosen. It is judged only after internal validity.
  2. Allocation concealment: keeping the random allocation sequence hidden until each patient is assigned (central computer or telephone, or sequentially numbered, opaque, sealed containers), reported under CONSORT 2025 item 18. It prevents selection bias at enrolment. Blinding differs: it hides the allocation after assignment.
  3. Stratification: randomising separately within strata of a strong prognostic factor (such as centre or disease severity), usually with blocks, so the groups are balanced on that factor. Its type and details are reported under item 17b.
  4. Sensitivity analysis: repeating the analysis under different assumptions or methods (such as different handling of missing data, or excluding protocol violators) to see whether the conclusion changes. Report whether each was prespecified (item 21d).
  5. Fragility Index: the number of patients in the group with fewer events whose status must change from non-event to event to make the P-value 0.05 or more (Walsh and colleagues, 2014). Teaching example: 10/100 against 25/100, P = 0.0085, Fragility Index 4. A small index, especially one smaller than the number lost to follow-up, shows a fragile result.

The one thing to remember

A larger sample reduces random error, but only good design removes bias and confounding.

Worked examples and figures use made-up numbers for teaching unless a study is named.

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Past papers

Every TNMGRMU question on bias, confounding and validity

Each one is answered in this chapter. Tap a sitting to open the university's own question paper.

  1. ×1Confounding factors in clinical trialsMD Pharmacology · Oct 2025
  2. ×1Types of Bias in vaccine trialsMD Pharmacology · Oct 2025
  3. ×1Confounders vs Effect modifiersMD Pharmacology · Oct 2025
  4. ×1Confounder and effect modifier in Epidemiological studiesMD Community Medicine · May 2025
  5. ×1As a health researcher, you are planning to do health research among college students using questionnaire as the only data collecting tool. Discuss various components that you will consider while designing the questionnaire and the methods you will apply to test reliability and validity of a questionnaireMD Community Medicine · Oct 2025
  6. ×1Biases in clinical researchDM Pulmonary Medicine · Aug 2025
  7. ×1Biases in study designDM Clinical Immunology and Rheumatology · Feb 2025
  8. ×1BIAS in clinical trialsMCh Gynaecological Oncology · Feb 2025
  9. ×1Differentiate Effect Modifiers and Confounders in health research with suitable examplesMD Community Medicine · Oct 2024
  10. ×1Reliability & ValidityPh.D. RM · Jul 2024
  11. ×1Describe the steps in conducting Meta-Analysis and sources of bias in Meta - Analysis. How is Heterogenecity between studies addressed during analysis?MD Community Medicine · Oct 2023
  12. ×1Confounding is not a bias –Justify the statementMD Community Medicine · Oct 2023
  13. ×1Reliability and ValidityPh.D. RM · Jul 2023
  14. ×1Biases in radiology reportingMD Radio Diagnosis · May 2022
  15. ×1Effect Modifier vs ConfounderMD Community Medicine · Oct 2022
  16. ×1Regarding randomised clinical trials, explain the following terms: a. External validity. b. Allocation concealment. c. Stratification. d. Sensitivity analysis. e. Fragility IndexDM Critical Care Medicine · Aug 2022
  17. ×1Bias in ResearchPh.D. RM · Jul 2022
  18. ×1Write briefly on how ‘selection bias’ can affect study inferences. List the methods used to minimize itMD Community Medicine · Jul 2021
  19. ×1Describe the biases in cross sectional studiesMD Community Medicine · Jul 2021
  20. ×1ConfoundingPh.D. RM · Aug 2021
  21. ×1Describe the steps in the design of cohort studies. Discuss the biases that may affect cohort studiesMD Community Medicine · May 2019
  22. ×1Elaborate on the concept of lead time bias in cancer screeningMCh Surgical Oncology · Feb 2019
  23. ×1Bias in case control studiesMD Community Medicine · Oct 2016
  24. ×1It is better to express imprecision of an analytical method in terms of coefficient of variation rather than standard deviation. Justify with an exampleMD Biochemistry · Oct 2014
  25. ×1Confounding variablesMD Community Medicine · Oct 2014
  26. ×1Define the term Validity. Discuss the different forms of ValidityPh.D. RM · Jul 2013
  27. ×1Distinguish between “Accuracy” and “Precision”Ph.D. RM · Jul 2013
  28. ×1Give the steps for an epidemiological study to find out cause / risk factors for a newly reported syndrome. What are the possible sources of Bias in this studyMD Community Medicine · Oct 2012
  29. ×1Biases in medical researchMCh Surgical Oncology · Aug 2009
  30. ×1Bias in studiesMD Community Medicine · Feb 2005
  31. ×1How is a screening test for cancer evaluated? Discuss the biases caused as a result of a screening programmeMCh Surgical Oncology · Apr 2004

Further reading

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