Strategic Design Decisions in Rare Disease Trials

Research2/8/2024 4:00 PM

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Adapting Endpoints, Ensuring Data Quality, and Determining Clinical Meaningfulness of Trial Results. Testing new therapeutic candidates in patients with rare diseases presents considerable challenges for clinical trial teams. In this webinar, Dr. Pam Ventola will share methodological approaches for adapting endpoints for rare disease trials and discuss rater training approaches and targeted data quality monitoring methodologies specific to rare disease trials. Dr. Ventola will also outline approaches for supporting the clinical meaningfulness of results in rare disease trials.


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Cogstate is a neuroscience technology company dedicated to simplifying the measurement of cognition. With a mission to democratize cognitive health, Cogstate offers digital cognitive assessment solutions, endpoint data quality services, remote assessment capabilities, healthcare cognitive screening tools, and scientific consulting. They have over 20 years of experience supporting biopharmaceutical companies, academic institutions, physicians, and individuals around the world. Cogstate's easy-to-use digital tests provide rapid, reliable, and science-backed results, enabling better brain health outcomes.