1. What is a sample? Epidemiology matters: a new introduction to methodological foundations Chapter 4

2. Seven steps 1.Define the population of interest 2.Conceptualize and create measures of exposures and health indicators 3.Take a sample of the population 4.Estimate measures of association between exposures and health indicators of interest 5.Rigorously evaluate whether the association observed suggests a causal association 6.Assess the evidence for causes working together 7.Assess the extent to which the result matters, is externally valid, to other populations Epidemiology Matters – Chapter 12

3. 1.Why take a sample? 2.How to take a representative sample 3.Quantifying sampling variability 4.How to take a purposive sample 5.Study design 6.Summary Epidemiology Matters – Chapter 43

4. 1.Why take a sample? 2.How to take a representative sample 3.Quantifying sampling variability 4.How to take a purposive sample 5.Study design Epidemiology Matters – Chapter 44

5. Why take a sample?  Epidemiologists take samples to answer health- related research questions efficiently  A full census is the epidemiologic ideal  Reasons not to take a census all the time include lack of time, lack of money, and waste of resources Epidemiology Matters – Chapter 45

6. To take a sample 1.Specify population of interest 2.Specify a research question of interest Epidemiology Matters – Chapter 46

7. Specify population of interest  What are the characteristics of the population in which we would like to understand health?  Example: Do we want to know what the prevalence of diabetes is within New York City? New York State? The United States? Do we want to know the causes of diabetes?  The population of interest has to be specified before the sampling strategy defined Epidemiology Matters – Chapter 47

8. Specifying a question  Question of interest can help clarify appropriate way to sample population of interest  Questions asked can include estimating population parameters, or estimating causal effects of exposures on outcomes Epidemiology Matters – Chapter 48

9. Example, estimating population parameters Questions concerned with population parameters include  What proportion of individuals in the population of interest has breast cancer?  What is the mean blood pressure in the population?  How many new cases of HIV are diagnosed in the population over three years? Population parameters include estimates of  Proportions  Means  Standard deviations Sample required  Representative sample 9

10. Example, estimating causal effects of exposures on outcomes Questions for which these measures are needed are  Does exposure to pollution cause lung cancer?  Does suffering abuse in childhood cause depression in adulthood?  Does a specific genetic marker cause Alzheimer’s disease? Parameter of interest  Causal effect of an exposure on a health outcome Sampling concerns  Not representativeness (as in population parameters)  Whether individuals exposed to hypothesized cause of interest are comparable to individuals not exposed  Purposive sample sufficient 10

11. Representative and purposive  A representative sample is one where the sample that is taken has characteristics similar to the overall population  A purposive sample selects from the population base on some criterion  A representative sample may or may not include individuals who are comparable with respect to causal identification  A purposive sample may or may not be representative of a particular population of interest Epidemiology Matters – Chapter 411

12. 1.Why take a sample? 2.How to take a representative sample 3.Quantifying sampling variability 4.How to take a purposive sample 5.Study design 6.Summary Epidemiology Matters – Chapter 412

13. How to take a representative sample  The simplest approach: a simple random sample  Each member of the population has an equal probability of being selected into the sample  A successful simple random sample should have the same basic characteristics as the original population Epidemiology Matters – Chapter 413

14. Taking a simple random sample 1.Enumerate all potential members of population of interest 2.Assign each member a probability of selection 3.Ensure selection of members are independent Epidemiology Matters – Chapter 414

15. Example: Sampling Farrlandia Epidemiology Matters – Chapter 415 30 residents in Farrlandia Options for random selection: --Every 4th home, dice roll for selection within home Challenges include (a) clustered exposures, (b) unequal ‘home’ size Selected for sample

16. Example: Sampling Farrlandia Epidemiology Matters – Chapter 416 30 residents in Farrlandia Select every Nth person in phone book Challenges include that not everyone is in phone book Selected for sample

17. Epidemiology Matters – Chapter 417  There is no perfect sample  The goal in epidemiology is to understand limitations of sampling methods and account for them The perfect sample?

18. Sampling Farrlandia Epidemiology Matters – Chapter 418

19. Sampling Farrlandia We want to collect our sample in such a way that the sample also has 50% exposed and 30% dotted. Epidemiology Matters – Chapter 419

20. Sampling Farrlandia We can use a simple random sample  ½ the population (25)  Probability of selection 1/50 or 2%  Random number generator Epidemiology Matters – Chapter 420

21. Sampling Farrlandia 21 Original Population Black solid1530% Black dots1020% Total black2550% Gray solid2040% Gray dots510% Total gray2550% Epidemiology Matters – Chapter 4

22. Sampling Farrlandia 22 Original Population Sample Black solid1530% Black dots1020% Total black2550% Gray solid2040% Gray dots510% Total gray2550% Black solid832% Black dots520% Total black1352% Gray solid1040% Gray dots28% Total gray1248% Epidemiology Matters – Chapter 4

23. 1.Why take a sample? 2.How to take a representative sample 3.Quantifying sampling variability 4.How to take a purposive sample 5.Study design 6.Summary Epidemiology Matters – Chapter 423

24. Quantifying sampling variability  Sampled population will not have the exact same population parameters as complete population census  The ‘truth’, i.e., the population parameter of original population is called the true population parameter Epidemiology Matters – Chapter 424

25. Epidemiology Matters – Chapter 425 Variations in possible samples

26. Epidemiology Matters – Chapter 426

27. Epidemiology Matters – Chapter 427 Variations in possible samples

28. Epidemiology Matters – Chapter 428 38,760 different possible samples of 5 Variations in possible samples

29. Quantifying uncertainty, Central Limit Theorem (CLT) 1.Average proportion across all possible samples = true population proportion  Example:  50% of true population has diabetes  Sample 1 has 100% diabetes  Sample 2 has 0% diabetes  Average of all samples will have 50% diabetes Epidemiology Matters – Chapter 429

30. Quantifying uncertainty, CLT 2.Variance around average sample proportions (standard error) p = sample proportion n = sample size Epidemiology Matters – Chapter 430

31. Quantifying uncertainty, CLT 3.Large samples will have normally distributed samples  > 30 people  No group < 5 people Epidemiology Matters – Chapter 431

32. Quantifying uncertainty, CLT Therefore the principal drivers of uncertainty are 1.Prevalence in the sample 2.Sample size The larger the sample size, the smaller the amount of uncertainty in the sample estimate Epidemiology Matters – Chapter 432

33. 1.Why take a sample? 2.How to take a representative sample 3.Quantifying sampling variability 4.How to take a purposive sample 5.Study design 6.Summary Epidemiology Matters – Chapter 433

34. Purposive sample  Eligibility criteria for study is the central design element; entry is based on exposure status, or sometimes on health outcome status Epidemiology Matters – Chapter 434

35. 1.Why take a sample? 2.How to take a representative sample 3.Quantifying sampling variability 4.How to take a purposive sample 5.Study design 6.Summary Epidemiology Matters – Chapter 435

36. Study design  Study design considerations are similar for representative or purposive sample  Study design reflects decisions made at one time point or over time  Timing of disease process can inform the study design Epidemiology Matters – Chapter 436

37. Study design options 1.Sample one moment in time, irrespective of disease status, measure disease and potential cause simultaneously 2.Sample over time, start with disease free individuals only, measure disease over time 3.Sample one moment in time, based on disease status Epidemiology Matters – Chapter 437

38. Farrlandia population Epidemiology Matters – Chapter 438

39. Farrlandia population Epidemiology Matters – Chapter 439

40. Farrlandia population Epidemiology Matters – Chapter 440

41. Farrlandia population Epidemiology Matters – Chapter 441

42. Farrlandia population Epidemiology Matters – Chapter 442

43. Farrlandia population Epidemiology Matters – Chapter 443

44. Farrlandia population Epidemiology Matters – Chapter 444

45. Farrlandia population Epidemiology Matters – Chapter 445

46. Farrlandia population Epidemiology Matters – Chapter 446

47. Option 1, Cross-sectional Epidemiology Matters – Chapter 447

48. Option 2, Cohort Epidemiology Matters – Chapter 448

49. Option 3, Case-control Epidemiology Matters – Chapter 449

50. 1.Why take a sample? 2.How to take a representative sample 3.Quantifying sampling variability 4.How to take a purposive sample 5.Study design 6.Summary Epidemiology Matters – Chapter 450

51. Summary 1.Samples are efficient, representative or purposive 2.Representative sample; e.g., simple random sample 3.Sampling variability, standard error 4.Purposive sample, selection on exposure or disease status 5.Study designs can be cross-sectional, cohort, case- control Epidemiology Matters – Chapter 451

52. Seven steps 1.Define the population of interest 2.Conceptualize and create measures of exposures and health indicators 3.Take a sample of the population 4.Estimate measures of association between exposures and health indicators of interest 5.Rigorously evaluate whether the association observed suggests a causal association 6.Assess the evidence for causes working together 7.Assess the extent to which the result matters, is externally valid, to other populations Epidemiology Matters – Chapter 152

53. epidemiologymatters.org 53Epidemiology Matters – Chapter 1