--- title: "Analyzing Clinical Significance: The Combined Approach" date: "`r Sys.Date()`" output: rmarkdown::html_vignette vignette: > %\VignetteIndexEntry{5. Combined Approach} %\VignetteEncoding{UTF-8} %\VignetteEngine{knitr::rmarkdown} editor_options: chunk_output_type: console --- ```{r, include = FALSE} knitr::opts_chunk$set( collapse = TRUE, comment = "#>", fig.width = 7, fig.height = 5, fig.align = "center" ) ``` ## Introduction The combined approach represents the most rigorous method for assessing clinical significance. It provides a more nuanced picture of patient outcomes by requiring **two criteria** to be met simultaneously: 1. **Magnitude of Change**: The change must be sufficiently large, either by being statistically reliable (distribution-based) or practically meaningful (anchor-based). 2. **End-State Functioning**: The patient must cross the threshold from a dysfunctional or "clinical" population to a functional or "non-clinical" one (statistical approach). This dual requirement allows for a richer classification of outcomes, distinguishing between patients who have merely "Improved" and those who have truly "Recovered". This vignette demonstrates how to use the `cs_combined()` function to apply these powerful models. ```{r setup} library(clinicalsignificance) ``` ## The Classic Jacobson & Truax (JT) Approach The most common combined method, proposed by Jacobson and Truax (1991), integrates the **distribution-based** and **statistical** approaches. A patient's change is considered clinically significant if: 1. The change is **reliable** (i.e., it exceeds the measurement error of the instrument). 2. The patient's final score falls within the range of a functional population. ### Example Analysis We will again use the `claus_2020` dataset. To perform this analysis, we need summary statistics for a functional (non-clinical) population on the BDI-II (`m_functional`, `sd_functional`) and an estimate of the instrument's reliability. ```{r jt-combined} # Perform the JT combined analysis jt_combined <- claus_2020 |> cs_combined( id = id, time = time, outcome = bdi, pre = 1, post = 4, m_functional = 7.69, sd_functional = 7.52, reliability = 0.801 ) # Display the summary of results summary(jt_combined) ``` ### Visualizing the JT Approach The plot for a combined analysis is particularly informative. It includes both the shaded area for reliable change (from the distribution-based approach) and the cutoff lines for population status (from the statistical approach). * **Dashed Lines**: The cutoff separating the clinical (right/top) and functional (left/bottom) populations. * **Shaded Area**: The zone of "no reliable change". ```{r jt-combined-plot} plot(jt_combined, show_group = "category") ``` The resulting categories are interpreted as follows: - **Recovered** (blue): Patients who showed a reliable improvement *and* ended up in the functional range. - **Improved** (light green): Patients who showed a reliable improvement but remained in the clinical range. - **Unchanged** (yellow): Patients with no reliable change, regardless of their population status. - **Deteriorated** (orange): Patients who showed a reliable worsening but remained in the clinical range. - **Harmed** (dark green): Patients who showed a reliable worsening *and* moved from the functional to the clinical range. ## The Claus, Wager & Bonnet (CWB) Approach As an alternative, `clinicalsignificance` allows you to combine the **anchor-based** and **statistical** approaches. This method is useful when a well-established Minimal Important Difference (MID) is available and may offer a more practically interpretable criterion for change than statistical reliability. Here, a patient's change is clinically significant if: 1. The change is **meaningful** (i.e., it meets or exceeds the MID). 2. The patient's final score falls within the range of a functional population. ### Example Analysis We use the same functional population data as before but replace the `reliability` argument with `mid_improvement`. ```{r anchor-combined} # Perform the anchor-based combined analysis anchor_combined <- claus_2020 |> cs_combined( id = id, time = time, outcome = bdi, pre = 1, post = 4, m_functional = 7.69, sd_functional = 7.52, mid_improvement = 7 ) # Display the summary of results summary(anchor_combined) ``` Notice the subtle shift in classifications: with the MID as the criterion, more patients are categorized as "Improved" or "Recovered" compared to the stricter reliability criterion of the JT approach in this specific example. ### Visualizing the CWB Approach The plot is interpreted similarly, but the shaded area is now defined by the MID instead of the Reliable Change Index. ```{r anchor-combined-plot} plot(anchor_combined, show_group = "category") ``` ## Summary and Next Steps The combined approach offers a powerful and nuanced assessment of treatment outcomes by considering both the magnitude of change and the patient's final functional status. * The **Jacobson & Truax (JT) approach** is the classic standard, requiring a reliable change and a shift in population status. * The **Claus, Wager & Bonnet (CWB) approach** is a valuable alternative when a meaningful MID is known, defining change in practical rather than purely statistical terms. Choosing between them depends on the available information (reliability vs. MID) and the specific research question.