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

Biotech, Drug Discovery & Development, Life Science,
  • Tuesday, September 30, 2025 | 11am EDT (NA) / 4pm BST (UK) / 5pm CEST (EU-Central)
  • 60 min

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

Pam Ventola, Cogstate

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.

Message Presenter

Who Should Attend?

Executives, directors, scientists and managers from pharma, biotech and CROs responsible for global rare disease clinical trials, including:

  • Clinical Operations
  • Clinical Science
  • Clinical Trial Planning and Optimization
  • Medical Affairs
  • Outcomes Research
  • Outsourcing and Procurement
  • Project Management
  • Research and Development

What You Will Learn

Attendees will gain insight into:

  • Strategies to improve data quality monitoring in rare disease clinical trials, including eCOA form optimization, enhanced rater training and the use of combined algorithmic, data-based review and risk-based expert data review
  • The use of central rating with highly trained clinicians
  • Methods for determining meaningful within-patient change thresholds

Xtalks Partner

Cogstate

Cogstate Ltd (ASX:CGS) is a leading science and technology solutions provider dedicated to optimizing the measurement of cognition in clinical trials, academic research and healthcare. Cogstate provides enabling technologies and professional services for higher quality neuropsychological assessments and is a pioneer in commercializing rapid, reliable and highly sensitive computerized cognitive tests. Cogstate customers include the world’s leading biopharmaceutical companies; elite sporting organizations and military; physicians and patients; renowned academic institutions and public-private partnerships. For more information, please visit www.cogstate.com.

You Must Login To Register for this Free Webinar

Already have an account? LOGIN HERE. If you don’t have an account you need to create a free account.

Create Account