2024 - Strategic Design Decisions in Rare Disease Trials

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.
Keywords: Clinical Trials, Rare Disease, Clinical Research, Patient Recruitment, Clinical Data, Clinical Trial Design, Rare Disease Clinical Trials, Clinical Trial Endpoints, Rater Training, Rare Diseases/Orphan Drugs