FCPS Part 1 Biostatistics and epidemiology

Sampling and sample size

Sampling Methods, Sample Size, Power and Type I and II Errors

Random, systematic, stratified, cluster and non-probability sampling, sample size, drop-out adjustment, power, and type I and type II errors. Explained with the FCPS scenarios that test each method, plus intention-to-treat analysis and the link between sample size and standard error.

Key terms

  • Population: the whole group of interest. The target population is who you want to draw conclusions about; the study (accessible) population is who you can actually reach.
  • Sample: the subset studied. Sampling frame: the list of all units from which the sample is drawn (electoral roll, household register, clinic list). Sampling unit: individual, household, village or school.
  • Parameter (true population value, such as μ) versus statistic (sample value, such as x̄).
  • Sampling error: chance difference between a sample statistic and the population parameter; it shrinks as the sample grows. Sampling bias is systematic and does not shrink with size.

Probability (random) sampling

  • Every unit has a known, non-zero chance of selection, so results can be generalised and sampling error calculated.
MethodHow it worksWhen and why
Simple randomEvery unit has an equal chance (random numbers, computer, lottery)Needs a complete frame; may by chance miss small subgroups
SystematicEvery kth unit after a random start (k = N ÷ n), such as every 10th patientEasy in clinics and streets; biased if the list has a repeating pattern
StratifiedSplit the population into homogeneous strata (age, sex, urban/rural), then sample randomly within eachGuarantees each subgroup is represented; more precise
ClusterRandomly choose groups (villages, schools), then study all or some membersCheap and practical when no list of individuals exists; larger sampling error (design effect)
MultistageRandom selection at several levels (districts, then villages, then households)National surveys such as DHS
MultiphaseBasic data from everyone, more detail from a subsampleScreening surveys
  • The WHO EPI 30-cluster survey (30 clusters × 7 children) estimates immunisation coverage quickly.
  • Stratified versus cluster: strata are internally similar and all strata are sampled; clusters are internally mixed (each a mini-population) and only some clusters are chosen.

Exam trap Stratified sampling is chosen for representation of subgroups; cluster sampling for convenience and cost when no list of individuals exists.

Non-probability sampling

MethodDescription
ConvenienceWhoever is available (patients on today's ward); prone to selection bias
ConsecutiveEvery eligible patient over a set period; better than convenience
QuotaFixed numbers from subgroups, chosen non-randomly within them
Purposive (judgemental)Researcher picks information-rich cases; used in qualitative research
SnowballParticipants recruit others; reaches hidden populations (drug users, sex workers)
  • Results from non-probability samples cannot be generalised with confidence because selection chances are unknown.

Hypothesis testing errors

  • The null hypothesis says there is no difference. A study can make two kinds of mistake:
Null hypothesis true (no real difference)Null hypothesis false (real difference)
Study finds a difference (rejects null)Type I error (α): false positiveCorrect: power (1 − β)
Study finds no differenceCorrectType II error (β): false negative
  • α is set before the study, usually 0.05; it is the risk of claiming an effect that is not there.
  • β is usually 0.2 or 0.1; power = 1 − β = the probability of detecting a real difference of the chosen size, conventionally 80% or 90%.
  • Small studies are prone to type II error: a non-significant result from an underpowered study is absence of evidence, not evidence of absence.

Remember Type I sees an effect that is not there; type II misses an effect that is.

What decides sample size

  • A larger sample is needed when:
  • the effect size to detect is smaller;
  • variability (SD) is greater;
  • α is stricter (0.01 rather than 0.05);
  • power wanted is higher (90% rather than 80%);
  • a cluster design is used (multiply by the design effect), several subgroups will be analysed, or drop-outs are expected.
  • For a prevalence survey: n = Z² × p(1 − p) ÷ d², where Z = 1.96 for 95% confidence, p = expected prevalence and d = acceptable margin of error.
  • Sample size and precision: SE = SD ÷ √n, so precision improves only with the square root of n. Halving the standard error requires four times the sample.
  • Adjusting for drop-outs: divide the required number by the proportion expected to remain. Needing 200 completers with 20% loss: 200 ÷ 0.8 = 250 to recruit. Adding 20% (240) leaves you short, because 20% of 240 is 48 and only 192 remain.
  • Power is calculated before the study (a priori); post hoc power adds nothing once results are known.
  • Power increases with larger samples, larger effects, less variability, a more lenient α and one-tailed tests.

Exam trap To allow for drop-outs divide by the retention rate; do not just add the percentage.

Randomisation and analysis in trials

  • Allocation methods: simple, block (keeps group sizes equal), stratified (balances key prognostic factors), cluster randomisation and minimisation.
  • Allocation concealment (central telephone or web system, sealed opaque envelopes) stops recruiters predicting the next assignment.
  • Intention-to-treat analysis: every participant is analysed in the group to which they were randomised, whether or not they took the treatment, switched or dropped out. It preserves the balance created by randomisation and gives a realistic, conservative estimate. Per-protocol analysis includes only compliers and can overstate benefit.

Sampling and measurement quality

  • A representative sample protects external validity; good measurement protects internal validity.
  • Reliability (precision, repeatability) means a test gives the same result when repeated on the same subject, by the same or different observers. Validity (accuracy) means it measures what it claims to measure. A test can be reliable but not valid.

How FCPS asks about this

  • Every 10th patient after a random start: systematic sampling.
  • Villages selected at random and all children in them studied: cluster sampling; WHO 30 × 7 survey.
  • Population divided by age group and each sampled randomly: stratified sampling.
  • Recruiting injecting drug users through other users: snowball sampling.
  • Power: 1 − β, usually 80%; type II error is missing a real difference.
  • 200 needed with 20% expected drop-out: recruit 250 (200 ÷ 0.8).
  • Intention-to-treat analysis: participants analysed in the group they were randomised to.

Test yourself on this topic

5 of the 25 questions in our bank on this topic. Choose an answer to see why every option is right or wrong.

Question 1Medium

A study must estimate prevalence separately in urban and rural areas, where rural households are fewer. Which method ensures both groups are adequately represented?

Question 2Medium

What does the reliability of a test describe?

Question 3Medium

Which change requires a larger sample size in a trial?

Question 4Medium

Forty villages are randomly allocated so that half receive chlorinated piped water, and diarrhoea rates are compared. What type of study is this?

Question 5Medium

A trial concludes a new drug is no better than placebo when it actually is. What error has occurred, and what reduces it?

Practise all 25 questions on this topic

Where this sits in the CPSP syllabus

  • Paper 1: Biostatistics, Sampling methods and sample size; power

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