In bringing novel therapeutics to market, clinical development programs face intense pressures to meet speed and efficiency goals. Enter innovative adaptive study designs that enable modification of the clinical program, drawing from early efficacy readouts. Such approaches aim to avoid sunken costs and reallocate resources to the most viable candidate therapeutics or research questions of interest with the aim of accelerating the availability of efficacious treatments to patients. As a consequence, clinical study designs are gaining complexity to answer as many questions within a single study.
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Exploratory Prognostic Biomarkers of Complement-Mediated Thrombotic Microangiopathy (CM-TMA) in Adults with Atypical Hemolytic Uremic Syndrome (aHUS): Analysis of a Phase III Study of Ravulizumab
Cammett, Garlo, Millman et al Mol Diagn Ther (2022)
Progress: Responsiveness Validation of an Electronic Patient Reported Outcome (ePRO) in Adults with Sickle Cell Disease (NCT05407805)
Minniti, Bronte-Hall, Marraffino et al Blood (2022) 140 (Supplement 1)
