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Design choices for observational studies of the effect of exposure on disease incidence

Gail, Mitchell H, Altman, Douglas G, Cadarette, Suzanne M, Collins, Gary, Evans, Stephen JW, Sekula, Peggy, Williamson, Elizabeth, Woodward, Mark·2019·BMJ Open

Abstract

The purpose of this paper is to help readers choose an appropriate observational study design for measuring an association between an exposure and disease incidence. We discuss cohort studies, sub-samples from cohorts (case-cohort and nested case-control designs), and population-based or hospital-based case-control studies. Appropriate study design is the foundation of a scientifically valid observational study. Mistakes in design are often irremediable. Key steps are understanding the scientific aims of the study and what is required to achieve them. Some designs will not yield the information required to realise the aims. The choice of design also depends on the availability of source populations and resources. Choosing an appropriate design requires balancing the pros and cons of various designs in view of study aims and practical constraints. We compare various cohort and case-control designs to estimate the effect of an exposure on disease incidence and mention how certain design features can reduce threats to study validity.

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Figure 1 Key points.
Figure 1 Key points.CC BY-NC · PubMed Central
Figure 2 Designs for estimating an association between an exposure and disease incidence.
Figure 2 Designs for estimating an association between an exposure and disease incidence.CC BY-NC · PubMed Central

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Cites

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Metadata

Authors
Gail, M. H., Altman, D. G., Cadarette, S. M., Collins, G., Evans, S. J., Sekula, P., Williamson, E., & Woodward, M.
Affiliations
  • Biostatistics Branch, National Cancer Institute, Rockville, Maryland, USA
  • Nuffield Department of Orthopaedics, Centre for Statistics in Medicine, Oxford, UK
  • Faculty of Pharmacy and School of Public Health, University of Toronto, Toronto, Ontario, Canada
  • Centre for Statistics in Medicine, University of Oxford, Oxford, UK
  • Medical Statistics Unit, London School of Hygiene and Tropical Medicine, London, UK
  • Institute of Genetic Epidemiology and Faculty of Medicine, Medical Center, University of Freiburg, Freiburg, Germany
  • Department of Medical Statistics, London School of Hygiene and Tropical Medicine, London, UK
  • The George Institute for Global Health, Oxford University UK and University of New South Wales, Sydney, New South Wales, Australia
Venue
BMJ Open
Publisher
BMJ
Published
2019-12
Volume
9
Issue
12
Pages
e031031
ISSN
2044-6055
Cited by
36
References
3
Language
en

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