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. In this webinar, Dr. Pam Ventola will share methodological approaches for adapting endpoints for rare disease trials and discuss rater training approaches and targeted data quality monitoring methodologies specific to rare disease trials. Dr. Ventola will also outline approaches for supporting the clinical meaningfulness of results in rare disease trials.