Abstract

Composite scale data is widely used in many therapeutic areas and consists of several categorical questions/items that are usually summarized into a total score (TS). Such data is discrete and bounded by nature. The gold standard to analyse composite scale data is item response theory (IRT) models. However, IRT models require item-level data while sometimes only TS is available. This work investigates models for TS. When an IRT model exists, it can be used to derive the information as well as expected mean and variability of TS at any point, which can inform TS-analyses. We propose a new method: IRT-informed functions of expected values and standard deviation in TS-analyses. The most common models for TS-analyses are continuous variable (CV) models, while bounded integer (BI) models offer an alternative that respects scale boundaries and the nature of TS data. We investigate the method in CV and BI models on both simulated and real data. Both CV and BI models were improved in fit by IRT-informed disease progression, which allows modellers to precisely and accurately find the corresponding latent variable parameters, and IRT-informed SD, which allows deviations from homoscedasticity. The methodology provides a formal way to link IRT models and TS models, and to compare the relative information of different model types. Also, joint analyses of item-level data and TS data are made possible. Thus, IRT-informed functions can facilitate total score analysis and allow a quantitative analysis of relative merits of different analysis methods.

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