Improving Signal Detection in Rare Disease Clinical Trials

Life Sciences, Clinical Trials,
  • Wednesday, November 11, 2020

High-quality data are critical when seeking to measure treatment outcomes, but this is especially challenging in rare disease trials where the stakes are high. In rare disease trials, the assessments are complex, and the eligible participants are limited. When the trial sites are numerous and geographically dispersed, inter-rater variability can further compromise data quality. As such, high-quality rater training and data monitoring programs are essential for rare disease clinical trials.

Complicating the rare disease trial landscape, though, are the efficiencies required for these smaller trials. Sites are typically highly specialized, and raters participate in many similar trials simultaneously. As such, efficient rater training and data quality monitoring approaches that minimize the budgetary impact and site burden are required.

In this webinar, Dr. Pam Ventola will describe customized rater training approaches that meet sponsors’ needs but also are flexible and efficient so as to be feasible for sites. Dr. Ventola will discuss a “Rater Academy” approach, where raters can be trained and certified on scales across trials as well as tailored training programs for both novice and highly experienced raters. Lastly, Dr. Ventola will describe several options for customized data-quality monitoring, including review of data, review of source documents, and centralized data collection.



Pamela Ventola, PhD, Senior Science Director of Pediatrics and Rare Disease, Cogstate

Dr. Pam Ventola leads Cogstate’s Rare Disease and Pediatric Center for Excellence, providing strategic oversight and expert guidance throughout all stages of study planning and execution. She is also an Assistant Professor at the Yale Child Study Center. As a licensed clinical psychologist, Dr. Ventola’s primary clinical expertise is in pediatric neuropsychology and specifically developmental and genetic disorders.

Message Presenter

Who Should Attend?

Executives, directors, scientists and managers from pharma, biotech and CROs responsible for the clinical development of CNS or rare disease therapeutics, including:

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

What You Will Learn

In this webinar, participants will learn about:

  • Critical considerations for data quality in rare disease clinical trials
  • Customized, high-quality, efficient rater training approaches
  • Customized data quality monitoring programs

Xtalks Partner


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

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