1. C1, L3-4, S1 Design Method of Data Collection Surveys and Polls Experimentation Observational Studies

2. C1, L3-4, S2 Experiments & Observational Studies Two other basic types of statistical study used for collecting data are experimental studies and observational studies. We use these when we are interested in studying the effect of a treatment on individuals or experimental units.

3. C1, L3-4, S3 Experiments & Observational Studies We conduct an experiment when it is (ethically, physically etc) possible for the experimenter to determine which experimental units receive which treatment.

4. C1, L3-4, S4 Experiments & Observational Studies Experiment Terminology Experimental Unit Treatment Response patient drug cholesterol car gasolineknocking tomatoes fertilizer yield mouse radiation mortality

5. C1, L3-4, S5 Experiments & Observational Studies In an observational study, we compare the units that happen to have received each of the treatments.

6. C1, L3-4, S6 e.g. You cannot set up a control (non-smoking) group and treatment (smoking) group. Observational Study patientsmokinglung cancer potatoesweatheryield humanradiationmortality Experiments & Observational Studies UnitTreatmentResponse

7. C1, L3-4, S7 Experiments & Observational Studies Note: Only a well-designed and well-executed experiment can reliably establish causation. An observational study is useful for identifying possible causes of effects, but it cannot reliably establish causation.

8. C1, L3-4, S8 Experimentation Guiding Principle: Make comparisons fair – try to make treatment groups as similar as possible except for treatments being used.

9. C1, L3-4, S9 1. Completely Randomized Design The treatments are allocated entirely by chance to the experimental units.

10. C1, L3-4, S10 1. Completely Randomized Design Example: Which of two varieties of tomatoes (A & B) yield a greater quantity of market quality fruit? Factors that may affect yield: different soil fertility levels exposure to wind/sun soil pH levels soil water content etc.

11. C1, L3-4, S11 Divide the field into plots and randomly allocate the tomato varieties (treatments) to each plot (unit). 8 plots – 4 get variety A (A) (B) 1. Completely Randomized Design What if the field sloped upward from left to right? UPHILL Discuss for ½ Minute

12. C1, L3-4, S12 1. Completely Randomized Design Note: Randomization is an attempt to make the treatment groups as similar as possible — we can only expect to achieve this when there is a large number of plots.

13. C1, L3-4, S13 2. Blocking Group (block) experimental units by some known factor and then randomize within each block in an attempt to balance out the unknown factors. Use: blocking for known factors (e.g. slope of field in previous example) and randomization for unknown factors to try to “balance things out”.

14. C1, L3-4, S14 2. Blocking Example continued: It is recognized that there are two areas in the field – well drained and poorly drained. Partition the field into two blocks and then randomly allocate the tomato varieties to plots within each block.

15. C1, L3-4, S15 Well drained Poorly drained 2. Blocking How should we allocate varieties to plots? Discuss in groups for 1/2 minute. 7 (B) 2 (A) 3 (A) 5 (A) 6 (A) 1 (A) 2 (B) 3 (A) 4 (B) 8 (B) 4 (B) 1 (B) Randomly assign types to 4 well drained plots and then to the 8 poorly drained plots.

16. C1, L3-4, S16 3. People as Experimental Units Example: Cholesterol Drug Study – Suppose we wish to determine whether a drug will help lower the cholesterol level of patients who take it. How should we design our study? Discuss for two minutes in groups.

17. C1, L3-4, S17 Polio Vaccine Example

18. C1, L3-4, S18 Polio Vaccine Example Dr. Jonas Salk, vaccine pioneer 1914-95 Iron Lung

19. C1, L3-4, S19 The Salk Vaccine Field Trial 1954 Public Health Service organized an experiment to test the effectiveness of Salk’s vaccine. Need for experiment: –Polio, an epidemic disease with cases varying considerably from year to year. A drop in polio after vaccination could mean either: Vaccine effective No epidemic that year

20. C1, L3-4, S20 The Salk Vaccine Field Trial Subjects: 2 million, Grades 1, 2, and 3 500,000 were vaccinated –(Treatment Group) 1 million deliberately not vaccinated –(Control Group) 500,000 not vaccinated - parental permission denied

21. C1, L3-4, S21 The Salk Vaccine Field Trial NFIP Design Treatment Group: Grade 2 Control Group: Grades 1 and 3 + No Permission Flaws ? Discuss for 30 seconds. Polio contagious, spreading through contact. i.e. incidence could be greater in Grade 2 (bias against vaccine), or vice-versa. Control group included children without parental permission (usually children from lower income families) whereas Treatment group could not (bias against the vaccine).

22. C1, L3-4, S22 The Salk Vaccine Field Trial Double-Blinded Randomized Controlled Experimental Design Control group only chosen from those with parental permission for vaccination Random assignment to treatment or control group Use of placebo (control group given injection of salted water) Diagnosticians not told which group the subject came from (polio can be difficult to diagnose) i.e., a double-blind randomized controlled experiment

23. C1, L3-4, S23 (NFIP rate) (25) Grade 2 (54) Grade 1/3 (44) Grade 2 The Salk Vaccine Field Trial The double-blind randomized controlled experiment (and NFIP) results Size of group Rate per 100,000 Treatment200,00028 Control200,00071 No consent350,00046

24. C1, L3-4, S24 3. People as Experimental Units control group: –Receive no treatment or an existing treatment blinding: –Subjects don’t know which treatment they receive double blind: –Subjects and administers / diagnosticians are blinded placebo: –Inert dummy treatment

25. C1, L3-4, S25 3. People as Experimental Units placebo effect: –A common response in humans when they believe they have been treated. –Approximately 35% of people respond positively to dummy treatments - the placebo effect

26. C1, L3-4, S26 Design Method of Data Collection Surveys and Polls Experimentation Observational Studies

27. C1, L3-4, S27 Observational Studies There are two major types of observational studies: prospectiveand retrospective studies

28. C1, L3-4, S28 Observational Studies 1. Prospective Studies –(looking forward) –Choose samples now, measure variables and follow up in the future. –E.g., choose a group of smokers and non-smokers now and observe their health in the future.

29. C1, L3-4, S29 Observational Studies –Looks back at the past. –E.g., a case-control study Separate samples for cases and controls (non-cases). Look back into the past and compare histories. E.g. choose two groups: lung cancer patients and non-lung cancer patients. Compare their smoking histories. 2. Retrospective Studies –(looking back)

30. C1, L3-4, S30 Observational Studies Note: 1. Observational studies should use some form of random sampling to obtain representative samples. 2.Observational studies cannot reliably establish causation.

31. C1, L3-4, S31 Controlling for various factors A prospective study was carried out over 11 years on a group of smokers and non- smokers showed that there were 7 lung cancer deaths per 100,000 in the non- smoker sample, but 166 lung cancer deaths per 100,000 in the smoker sample. This still does not show smoking causes lung cancer because it could be that smokers smoke because of stress and that this stress causes lung cancer.

32. C1, L3-4, S32 Controlling for various factors To control for this factor we might divide our samples into different stress categories. We then compare smokers and non-smokers who are in the same stress category. This is called controlling for a confounding factor.

33. C1, L3-4, S33 Observational Studies Surgeon General of United States –Presence of strong relationship (5 x higher) –Strong research design –Dose-response relationship –Temporal relationship - cause should precede effect –Reversible association –Consistency: different studies produce similar results –Biological plausibility

34. C1, L3-4, S34 Example 1 “Home births give babies a good chance” NZ Herald, 1990 –An Australian report was stated to have said that babies are twice as likely to die during or soon after a hospital delivery than those from a home birth. –The report was based upon simple random samples of home births and hospital births. Q: Does this mean hospitals are dangerous places to have babies in Australia? Why or why not? Discuss for 1 minute in groups.

35. C1, L3-4, S35 Example 2 “Lead Exposure Linked to Bad Teeth in Children” ~ USA Today The study involved 24,901 children ages 2 and older. It showed that the greater the child’s exposure to lead, the more decayed or missing teeth. Q: Does this show lead exposure causes tooth decay in children? Why or why not? Discuss for 1 minute.

36. C1, L3-4, S36 Example 2 ~ cont’d “Lead Exposure Linked to Bad Teeth in Children” ~ USA Today Researcher: “We controlled for income level, the proportion of diet due to carbohydrates, calcium in the diet and the number of days since the last dental visit.”