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Title Extensions to the design and analysis of case-control studies
Course number 2991
Programme Epidemiologi
Language English
Credits 1.5
Date 2017-11-22 -- 2017-12-01
Responsible KI department Institutionen för medicinsk epidemiologi och biostatistik
Specific entry requirements Epidemiology I, Introduction to epidemiology; Epidemiology II, Design of epidemiological studies; Biostatistics I, Introduction for epidemiologists; Biostatistics II, Logistic regression for epidemiologists; and Biostatistics III: Survival analysis for epidemiologists, or equivalent courses
Purpose of the course This course aims to enable practicing epidemiologists to make more efficient use of already-available case-control data and to design case-control studies that will extend the possibilities for future analysis.
Intended learning outcomes After successfully completing this course you as a student are expected to be able to:

- select a suitable epidemiological design for addressing a specified research question and justify the choice of design compared to other options.
- compare the risk estimates obtained by different sampling strategies from the same underlying cohort and interpret these estimates for common designs.
- compare and contrast the purpose of time-matching and confounder-matching in (nested) case-control studies, and generalise the resulting risk sets to a wide range of standard and non-standard designs.
- compute weights that enable the reconstruction of an underlying cohort from a (nested) case-control sample and recognise that two-stage designs, re-use of case-control data, and extended/extreme case-control designs can all be analysed using appropriate weights to reflect the sampling
- discuss the designs of published studies with particular attention to the choice of controls and devise more efficient alternatives.
Contents of the course The overall aim of this course is to present statistical approaches that enable researchers to design more efficient case-control studies and to exploit more efficiently the data provided by nested case-control studies conducted in well-defined cohorts (such as national registers). In particular, the course will focus on different sampling designs in terms of their (biased) representation of the underlying cohort, and how to reconstruct the correct numbers at-risk to produce unbiased parameter estimates, including several important quantities (other than the odds ratio). The course will demonstrate the application of these methods to re-use controls from a prior study or after breaking the matching in a matched case-control study, conduct more flexible and informative analysis, and make efficient use of costly data.
Teaching and learning activities Lectures interspersed with tutorials consisting of workshops and journal club sessions. In the workshops, participants will develop and refine a study design to address a clinical/epidemiological research question which will be presented and discussed. Journal clubs will consist of discussion and debate concerning key papers that will be assigned.
Compulsory elements The individual examination
Examination The course grade will be based on a take-home assignment involving a proposed epidemiological study. The participant will submit a short written report and an oral presentation where they will present and defend their proposal. A passing grade must be obtained for both the written and oral section in order to obtain a passing grade for the course. Students who obtain a passing grade on one of these sections will be allowed to revise that part of their work and be re-examined under the same conditions. The exam will have a strong emphasis on intuitive understanding and ability to explain/communicate rather than on technical or mathematical detail. The take-home examination will be explained on the first day of the course, assigned on the last day, and due within ten days of the end of the course. Students who do not obtain a passing grade in the first examination will be offered a second examination within 2 months of the final day of the course.
Literature and other teaching material Suggested course literature:
As this is an advanced course, it is not covered in a text book, but involves material from several decades of scientific publications concerning case-control designs. The participants will be asked to reread papers 1-5 below which are assumed familiar, and to read papers 6-10 before the course. Teaching material will also consist of published code for Stata and SAS that enable the implementation of the methods introduced in the course.

1. Miettinen O. Design options in epidemiologic research. An update. Scand J Work Environ Health. 1982;8 Suppl 1:7-14.
2. Vandenbroucke JP, Pearce N. Case-control studies: basic concepts. Int J Epidemiol. 2012 Oct;41(5):1480-9.
3. Knol MJ, Vandenbroucke JP, Scott P, Egger M. What do case-control studies estimate? Survey of methods and assumptions in published case-control research. Am J Epidemiol. 2008 Nov 1;168(9):1073-81.
4. Ernster VL. Nested case-control studies. Prev Med. 1994 Sep;23(5):587-90.
5. Pearce N. Analysis of matched case-control studies. BMJ. 2016 Feb 25;352:i969.
6. Borgan O, Samuelsen SO. A review of cohort sampling designs for Cox regression model: potentials in epidemiology. Norsk Epi. 2003, 13(2), 239-248.
7. Hanley JA. The Breslow estimator of the nonparametric baseline survivor function in Cox regression model: some heuristics. Epidemiology. 2008 Jan;19(1):101-2.
8. Heller RF, Dobson AJ, Attia J, Page J. Impact numbers: measures of risk factor impact on the whole population from case-control and cohort studies. J Epidemiol Community Health. 2002 Aug;56(8):606-10.
9. Salim A, et al. A maximum likelihood method for secondary analysis of nested case-control data.Stat Med. 2014 May 20;33(11):1842-52.
10. Arnold BF, Ercumen A, Benjamin-Chung J, Colford JM Jr. Brief Report: Negative Controls to Detect Selection Bias and Measurement Bias in Epidemiologic Studies. Epidemiology. 2016 ;27(5):637-41.
Number of students 8 - 25
Selection of students Eligible doctoral students, with required prerequisite knowledge, will be selected based on 1) the relevance of the syllabus for the applicant's doctoral project (according to written motivation), and 2) date for registration as doctoral student (priority given to earlier registration date). To be considered, submit a completed application form. Give all information requested, including a description of current research and motivation for attending, and an account of previous courses taken.
More information Course dates are November 22, 24, 27, 29 and December 1. The course is extended over time, but still five full course days, in order to promote reflection and reinforce learning.
Additional course leader
Latest course evaluation Not available
Course responsible Marie Reilly
Institutionen för medicinsk epidemiologi och biostatistik
08-524 839 82
Marie.Reilly@ki.se
Contact person Gunilla Nilsson Roos
Institutionen för medicinsk epidemiologi och biostatistik
08-524 822 93
gunilla.nilsson.roos@ki.se