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Arvid Sjölander

Publications and source records attributed to Arvid Sjölander.

20 records · Page 2Linked to original sources

Aim for clinical utility, not just predictive accuracy

The predictions from an accurate prognostic model can be of great interest to patients and clinicians. When predictions are reported to individuals, they may decide to take action to improve their health or they may simply be comforted by the knowledge. However, if there is a clearly defined space of actions in the clinical context, a formal decision rule based on the prediction has the potential to have a much broader impact. Even if it is not the intended use of a developed prediction model, informal decision rules can often be found in practice. The use of a prediction-based decision rule should be formalized and compared to the standard of care in a randomized trial to assess its clinical utility, however, evidence is needed to motivate such a trial. We outline how observational data can be used to propose a decision rule based on a prognostic prediction model. We then propose a framework for emulating a prediction driven trial to evaluate the utility of a prediction-based decision rule in observational data. A split-sample structure can and should be used to develop the prognostic model, define the decision rule, and evaluate its clinical utility.

q-bio.QM↗

Analysis of 1:1 Matched Cohort Studies and Twin Studies, with Binary Exposures and Binary Outcomes

To improve confounder adjustments, observational studies are often matched on potential confounders. While matched case-control studies are common and well covered in the literature, our focus here is on matched cohort studies, which are less common and sparsely discussed in the literature. Matched data also arise naturally in twin studies, as a cohort of exposure-discordant twins can be viewed as being matched on a large number of potential confounders. The analysis of twin studies will be given special attention. We give an overview of various analysis methods for matched cohort studies with binary exposures and binary outcomes. In particular, our aim is to answer the following questions: (1) What are the target parameters in the common analysis methods? (2) What are the underlying assumptions in these methods? (3) How do the methods compare in terms of statistical power?

stat.ME↗