Improving Clinical Response Signaling in Rare Disease Studies with Targeted Endpoint Data Quality Programs

Rare disease clinical trials face unique challenges due to small patient populations, heterogeneous disease presentations and complex neurocognitive assessments. These factors exacerbate the difficulty of detecting meaningful treatment effects, making it crucial for study teams to adopt innovative strategies to enhance signal detection and improve decision-making both during and after rare disease studies.

In this webinar, the speaker, a rare disease expert, will share proven tactics and clinical trial case examples of how rare disease study teams are improving clinical response signaling using targeted approaches to endpoint data quality.

In particular, the speaker will share details regarding:

• eCOA form optimization to address common rater errors before they occur
• Optimizing rater qualification and training programs for rare disease trials
• Data quality monitoring using both algorithmic, data-based review as well as risk-based expert review
• Central rating using small groups of highly trained clinicians to administer/score scales remotely
• Methods for determining meaningful within-patient change thresholds

Register for this webinar to gain insights into how teams are overcoming the unique challenges of rare disease studies and improving clinical response signaling.

Speaker

Pamela Ventola, PhD, Chief Science Officer, Cogstate

Dr. Pamela Ventola is Chief Science Officer at Cogstate, an Associate Professor at the Yale Child Study Center and a licensed clinical neuropsychologist. Dr. Ventola leads Cogstate’s science team, providing Cogstate customers with strategic oversight and expert guidance throughout all stages of their study planning and execution — from endpoint selection, rater training and strategic monitoring to final statistical analysis.

Dr. Ventola earned her Doctor of Philosophy in Clinical Psychology from the University of Connecticut and completed her clinical training and Postdoctoral Fellowship at Yale University School of Medicine. She serves on the editorial review board of multiple academic journals and has authored numerous peer-reviewed manuscripts, book chapters and scientific presentations.

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