FDA statistician outlines adaptive trial designs to address rare-disease evidence gaps At FDA Grand Rounds, Natalie Morris of the agency's Office of Biostatistics reviewed adaptive clinical-trial designs for rare diseases, presenting simulations that show an adaptive treatment-duration approach can preserve type I error and offer similar power to a fixed two-year trial in some settings.

Natalie Morris from the FDA reveals groundbreaking adaptive trial designs that could revolutionize drug development for rare diseases, tackling significant evidence gaps in the process.

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#US #CitizenPortal #AdaptiveMethods #StatisticalEvidence #RareDiseaseTrials

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💊 Clinical Trial Spotlight: Rare Bone Disorders 💊

Prof Stuart Ralston (University of Edinburgh) will explore groundbreaking research on rare bone disorders in the TOPAZ/ZIPP trials. A must-attend session! #RareDiseaseTrials

📅 Register here: www.eventbrite.ie/e/...

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🚨 Clinical Trial Spotlight: IMPALA 2 🚨

Prof Elisabeth Bendstrup (Aarhus University) presents on Autoimmune Pulmonary Alveolar Proteinosis & IMPALA 2 – a game-changing trial for this rare condition! #RareDiseaseTrials #RareLung

Register here: www.eventbrite.ie/e/...

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📑 Clinical Trial Identification Project 📑
Ms Sarah Forde (RDCTN) will present an overview of the Clinical Trial Identification Project, a vital initiative for improving trial accessibility. #ClinicalTrialAccess #RareDiseaseTrials 📅 Register here: www.eventbrite.ie/e/...