Strategic Design Decisions in Rare Disease Trials

Life Sciences, Clinical Trials, Patient Recruitment & Retention,
  • Thursday, February 08, 2024

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. Among these are proper endpoint selection, adaptation of endpoints for rare indications, data quality assurance practices (rater training and monitoring) and determining clinical meaningfulness of positive trial results.

In this webinar, Dr. Pam Ventola will share methodological approaches for adapting endpoints for rare disease trials. Specifically, she will outline how sponsors may adapt both standardized endpoints — such as the Vineland-3 — as well as common clinician reported outcomes — such as the clinical global impression – severity/improvement scale (CGI-S/I) — to meet the unique needs of patient populations.

In addition, Dr. Ventola will discuss rater training approaches and targeted data quality monitoring methodologies specific to rare disease trials. These processes are vital for trial success, as rare disease studies have unique challenges in the realm of rater training. In these trials, sponsors typically need to recruit patients from broad geographic areas. This means raters have varied levels of experience and expertise, opening the door for rater variance and errors.

Lastly, determining the clinical meaningfulness of trial results is a critical step in designing trials and interpreting results. Dr. Ventola will outline approaches for supporting the clinical meaningfulness of results in rare disease trials.

Register for this webinar to discover invaluable strategies to effectively navigate rare disease trials, enhancing endpoint adaptation, data quality and clinical interpretation.


Pam Ventola, Cogstate

Pam Ventola, PhD, Chief Science Officer, 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 Associate 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 clinical development of clinical trial solutions in rare disease studies, including:

  • Research and development
  • Clinical operations
  • Clinical science
  • Outcomes research
  • Outsourcing and procurement
  • Project management
  • Clinical trial planning and 
  • Medical affairs

What You Will Learn

Attendees will gain insights into:

  • Approaches for adapting endpoints for rare disease trials
  • Strategies for conducting rater training and data quality monitoring
  • Strategies for supporting clinical meaningfulness

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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