--- title: "Analyzing Clinical Significance: The Percentage-Change Approach" date: "`r Sys.Date()`" output: rmarkdown::html_vignette vignette: > %\VignetteIndexEntry{3. Percentage-Change 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 percentage-change approach defines clinical significance based on the **relative change** in a patient's score from baseline. This method is common in clinical trials where a specific reduction in symptoms, such as "a 50% improvement," is a primary endpoint for defining treatment response. A change is considered clinically significant if it meets or exceeds a predefined **Percentage-Change Cutoff (PCC)**. For example, if the PCC is 50% (or 0.5), any patient who shows at least a 50% reduction in symptoms would be classified as "Improved". A key consideration for this method is that it is **highly dependent on the patient's baseline score**. A 10-point drop is a 50% improvement for a patient starting at 20 points, but only a 25% improvement for a patient starting at 40 points. This characteristic should be kept in mind when interpreting the results. This vignette demonstrates how to use the `cs_percentage()` function to apply this approach. ```{r setup} library(clinicalsignificance) ``` ## Basic Analysis Let's analyze the `claus_2020` dataset. A common benchmark for depression treatment trials is a 50% reduction in symptom scores. We will set this as our `pct_improvement` cutoff. ```{r pct-basic} # Analyze change using a 50% improvement cutoff pct_results <- claus_2020 |> cs_percentage( id = id, time = time, outcome = bdi, pre = 1, post = 4, pct_improvement = 0.5 ) summary(pct_results) ``` The summary shows that based on this 50% criterion, about 28% of patients are classified as having improved. ### Visualizing the Results The plot for the percentage-change approach looks similar to those for the anchor- and distribution-based methods. However, the shaded area is now determined by the percentage-change cutoff relative to each individual's starting score. This means the boundaries of the "unchanged" area are not parallel lines. ```{r pct-basic-plot} plot(pct_results) ``` ## Grouped Analysis We can also explore if the proportion of "responders" differs between the treatment groups (TAU vs. PA). ```{r pct-grouped} # Grouped analysis with a 50% improvement cutoff pct_grouped <- claus_2020 |> cs_percentage( id = id, time = time, outcome = bdi, pre = 1, post = 4, pct_improvement = 0.5, group = treatment ) summary(pct_grouped) ``` The results suggest that a much higher proportion of patients in the Placebo Amplification (PA) group (42.9%) achieved a 50% symptom reduction compared to the Treatment as Usual (TAU) group (10.5%). The plot clearly visualizes this difference: ```{r pct-grouped-plot} plot(pct_grouped) ``` ## Using Different Cutoffs for Improvement and Deterioration A useful feature of `cs_percentage()` is the ability to set different cutoffs for improvement and deterioration via the `pct_deterioration` argument. For instance, in some contexts, a small worsening of symptoms (e.g., 20%) might already be considered a significant deterioration, while a larger change (e.g., 50%) is required for improvement. ```{r pct-different-cutoffs} pct_asymmetric <- claus_2020 |> cs_percentage( id = id, time = time, outcome = bdi, pre = 1, post = 4, pct_improvement = 0.5, pct_deterioration = 0.2 # A smaller threshold for worsening ) summary(pct_asymmetric)