International Epidemiological Association 6th International Course on Epidemiological Principles and Methods Vilnius, Lithuania, 5-16 May 2014 Week 2, Day 1. Conducting population-based research: the Eastern European experience Practical Session 39: Response Rates, Selection Bias and Representativeness by Vladimir M. Shkolnikov, David A. Leon, Domantas Jasilionis May 12, 2014 1 Outline • A short introduction. • Preparatory work in groups. Issue of representativeness. Discussion in the International Journal of Epidemiology 2013; 42: 1012-1028. Rothman et al. vs. Ebrahim&Davey Smith. • Discussion between groups (20 min). Arguments pro and contra representativeness. Considerations and examples in support of R or E&DS • Joint discussion (30 min). All groups together. 2 Sequence of work Short explanation in the conference room 10 min Preparatory work of six groups in three rooms (40 min) plus presentations pro and contra 2x10 min Joint discussion in the conference room 20 min Introduction Y N Y N Y N Summary 3 K.Rothman et al: Representativeness to be avoided • Against a view that a study is not worth undertaking if representativeness cannot be achieved. • John Snow. Cholera and water source in London. A non-representative population sample with comparable characteristics except for the source of their water consumption. • Doll and Hill. Mortality of male British doctors depending on smoking. An unrepresentative sample of all tobacco users. Clear research question and use of samples allowing to built statistical evidence to answer the question. • Scientific generalization relates to circumstances in which a finding applies. • A study of therapeutic efficacy of a drug. Alternatives: A-subjects aged 40 to 49 only; B-equal numbers of subjects from groups 20-29, 40-49, and 60-69; C-according to age structure of the general population. To see how the effect varies by age, option B is preferable. • If a study population is representative, it does itself deliver a valid scientific evidence. The overall associations observed in the study population may not apply to every subgroup. Result obtained from a two-sex population, 4 does not necessarily apply to males or to females. K.Rothman et al: Representativeness to be avoided (2) • Representative sampling is needed: a) for control sampling in some casecontrol studies; b) descriptive purposes – e.g. prevalence studies; c) population impact studies. • A divide between a goal of understanding a causal relationship and a practical goal of applying this knowledge to a population. The first goal is not enhanced by representativeness. It depends on tightly controlled comparisons drawn over a variety of relevant settings. It is the second goal of the application of science that may require representativeness. • Surveys of opinions, of the prevalence of disease, of habits or of environmental exposures may be informative, but they are not science in the same way that causal studies about how nature works. 5 E.Shah&G.Davey Smith: we should not be deliberately non-representative • We are concerned that the notion of Rothman et al will become an accepted wisdom in epidemiology. • The purpose of epidemiology is not simply to assess causal hypotheses. Rothman et al.: causal hypotheses testing is a “science” and descriptive epidemiology is not a “science”. • Non-representative study groups may produce biased associations. Multiple risk factors may be associated with participation in a study. • In a large volunteer cohort of the American Cancer Society, high alcohol consumption was associated with reduced risk of stroke. What heavy drinker would volunteer for a study about the health effects of their lifestyle? Heavy drinkers in the cohort are unlikely to be representative of all heavy drinkers (they may be more non-smoking, exercising etc.) and (therefore) exposed to a lower risk of stroke. • Randomised trials: internal validity may be achieved by limiting recruitment to narrowly defined group. Criticized for not recruiting adults of older age with multiple morbidities (potential users). 6 E.Shah&G.Davey Smith: we should not be deliberately non-representative • UK Biobank Study. 500 thousand participants, reponse rate = 5.5%. Genetic variants are unlikely to be associated with self-selection to the study. Leads to unbiased purely genetic associations but non-genetic associations should be interpreted with caution. • Age of “Big data”. It might be possible to predict insurance risk from hundreds of variables including amount of TV watching, use of various Internet sites etc. instead of performing lab tests. But epidemiologists are not ready to abandon the difficult business of characterizing causality. 7 References - Rothman K.J., Gallacher J.E.J., Hatch E. 2013. Why representativeness should be avoided. International Journ Epidemiology 42: 1012-14. - Elwood J.M. 2013. On representativeness. International Journ Epidemiology 42: 1014-15. - Nohr E.A., Olsen J. 2013. Commentary: epidemiologists have debated representativeness for more than 40 years – has the time come to move one? International Journ Epidemiology 42: 1016-17. - Richardi L., Pizzi C., Pearce N. 2013. Commentary: Representativeness is usually not necessary and often should be avoided. International Journ Epidemiology 42: 1018-22. - Ebrahim S., Davey Smith G. 2013. Commentary: should we always deliberately be non-representative? International Journ Epidemiology 42: 1022-26. - Rothman K.J., Gallacher J.E.J., Hatch E. 2013. Rebuttal: when it comes to scientific inference, sometimes a cigar is just a cigar. International Journ Epidemiology 42: 1026-28. 8 An example. How to capture risk of alcohol drinking 9
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