Skip to main content


Fig. 2 | Orphanet Journal of Rare Diseases

Fig. 2

From: Recent advances in methodology for clinical trials in small populations: the InSPiRe project

Fig. 2

Optimal adaption rules of adaptive enrichment designs, optimized for a sponsor (left graph) and a societal perspective (right graph). Depending on the observed standardized treatment effects in the biomarker positive (plotted on the x-axes) and negative (plotted on the y-axes) population, the graph indicates the optimal second stage design option: futility stop (white area), enrichment design, recruiting biomarker positive patients only (red area), or partially enriched design (grey area). In addition, the second stage sample sizes are optimized (not shown in the graph). The optimisation is based on an a priori distribution on the effect sizes corresponding to the assumption that the treatment effect is either independent of the biomarker or that it is larger (or only present) in biomarker positive patients. See Ondra et al. [23] for details. Figure reproduced from [23] under CC BY-NC License [50]

Back to article page