Improving Signal Detection in Rare Disease Clinical Trials

November 11, 2020

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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 with complex assessments and a limited number of eligible participants. 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 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.

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


Pam 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.

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