Abstract
Clinical trials are the standard approach for evaluating new treatments, but may lack the power to assess rare outcomes. Trial results are also necessarily restricted to the population considered in the study. The availability of routinely collected healthcare data provides a source of information on the performance of treatments beyond that offered by clinical trials, but the analysis of this type of data presents a number of challenges. Hierarchical methods, which take advantage of known relationships between clinical outcomes, while accounting for bias, may be a suitable statistical approach for the analysis of this data. A study of direct oral anticoagulants in Scotland is discussed and used to motivate a modeling approach. A Bayesian hierarchical model, which allows a stratification of the population into clusters with similar characteristics, is proposed and applied to the direct oral anticoagulant study data. A simulation study is used to assess its performance in terms of outcome detection and error rates.
Original language | English |
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Pages (from-to) | 2639-2654 |
Number of pages | 16 |
Journal | Statistics in Medicine |
Volume | 39 |
Issue number | 20 |
Early online date | 7 May 2020 |
DOIs | |
Publication status | Published - 10 Sept 2020 |
Externally published | Yes |
Keywords
- Bayesian hierarchy
- direct oral anticoagulants
- multiple outcomes
- observational study
- safety outcomes