Why the study?
Appropriate end points and trial designs need to be identified for evaluating new and emerging therapies in pulmonary arterial hypertension clinical trials.
Comparison
New and emerging therapies versus existing combination therapies
Design
Discussion of various trial designs including placebo-controlled, add-on, noninferiority, and withdrawal trials
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May guide end point selection for PAH combination trials; leaves open optimal designs pending prospective validation.
New and emerging therapies might provide benefit in patients with pulmonary arterial hypertension. Their efficacy and safety will be compared with existing combination therapies in randomized clinical trials. Appropriate end points for these trials need to be identified: these will include exercise testing, the composite end point of time to clinical worsening, and hemodynamic markers, including advanced imaging modalities and biomarkers. Quality-of-life questionnaires are useful and important secondary end points; pulmonary arterial hypertension-specific questionnaires are currently being developed. Advantages and disadvantages of various trial designs, including placebo-controlled monotherapy or add-on trials, noninferiority studies, and withdrawal trials are also discussed.
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McLaughlin et al. (2009) studied this question.
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