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Join us at Symposium 4:

Optimizing Power and Precision in Rare Disease PRO Endpoints

MODERATOR:

Pronabesh Dasmahapatra, MD MPH, Head, Heath Economics and Value Assessment Strategy, Rare Disease and Rare Blood Disorder, Cambridge, Massachusetts, United States

INDIVIDUAL PRESENTATIONS:

  • Most Bothersome Symptom in Rare Disease: A Look at Endpoint Precision and Validity
    R. J. Wirth, PhD, Vector Psychometric Group, LLC, Chapel Hill, North Carolina, United States
  • Hypothesis Tests to Evaluate Treatment Efficacy that Optimize Power and Precision in Rare Disease Settings
    Charles Iaconangelo, PhD, Pharmerit – an OPEN Health Company, Brooklyn, New York, United States
  • A New Method for Testing Significance of eCDF Separation for Meaningful Change in Rare Diseases
    Daniel Serrano, PhD, Pharmerit – an OPEN Health Company, Bethesda, Maryland, United States