Pulmonary arterial hypertension

Enhancing patient outcomes and statistical efficiency in rare-disease phase-II trials. The StratosPHere 2 study (Invited Talk @ Italian Bayesian Day 2023)

Designing early- and late-phase clinical trials for rare conditions pose unique challenges, ranging from ethical considerations to generating robust inferences from limited sample sizes. In this work, guided by a rare-disease precision-medicine case …

Enhancing patient outcomes and statistical efficiency in rare-disease phase-II trials. The StratosPHere 2 study (Poster @ ISCB2023 - Joint conference with the IBS)

Designing early- and late-phase clinical trials for rare conditions pose unique challenges, ranging from ethical and practical enrolment considerations to generating robust evidence from limited sample sizes. Pulmonary arterial hypertension (PAH), …