16 pediatric subjects suffering from a aare skin disease (epidermolysis bullosa simplex) treated with placebo or diacerin cream in a longitudinal cross-over trial (14 paired).
<ul>
<li><code>Id</code>: study participant.</li>
<li><code>Time</code>: end of the 4 weeks treatment period (t4) or 12 weeks after treatment ended (t12)</li>
<li><code>treatment</code>: treatment arm: diacerin cream (V) or placebo (P).</li>
<li><code>StdDiffCount</code>: blister uncertainty</li>
<li><code>Bin</code>: indicator of 40</li>
<li><code>DiffQoL</code>: Change in quality of life since baseline.</li>
<li><code>period</code>: same as time but coded as numeric: 1 for t4 and 2 for t12</li>
</ul>

datasets

Implementation of the Generalized Pairwise Comparisons (GPC) as defined in Buyse (2010) <doi:10.1002/sim.3923> for complete observations, and extended in Peron (2018) <doi:10.1177/0962280216658320> to deal with right-censoring. GPC compare two groups of observations (intervention vs. control group) regarding several prioritized endpoints to estimate the probability that a random observation drawn from one group performs better/worse/equivalently than a random observation drawn from the other group. Summary statistics such as the net treatment benefit, win ratio, or win odds are then deduced from these probabilities. Confidence intervals and p-values are obtained based on asymptotic results (Ozenne 2021 <doi:10.1177/09622802211037067>), non-parametric bootstrap, or permutations. The software enables the use of thresholds of minimal importance difference, stratification, non-prioritized endpoints (O Brien test), and can handle right-censoring and competing-risks.

EB: Rare disease trial

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