Moderator
Cheryl Coon, PhD, Critical Path Institute, Tucson, AZ, United States
Speakers
Heather Gelhorn, PhD, PPD Evidera Patient-Centered Research, Thermo Fisher Scientific, Winter Park, CO, United States; Katelyn N Cutts, MS, PPD Evidera Patient-Centered Research, Thermo Fisher Scientific, Waltham, MA, United States; Brooke Harrow, PhD, Deciphera Pharmaceuticals, Waltham, MA, United States
Navigating the path to successful Clinical Outcome Assessment (COA) label claims is a formidable challenge compounded by stringent but evolving regulatory expectations and complex methodological hurdles. We will present a successful case study in Tenosynovial Giant Cell Tumor that included 4 COAs and resulted in 3 labeling claims. This session will delve into some of the key challenges including: a rare and heterogeneous disease indication, multiple regulatory interactions and requests, successful use of an item-response theory-based approach to COA measurement and scoring with the Patient Reported Outcomes Measurement System (PROMIS) Physical Function (PF) item bank, the use of mixed-methods to define meaningful change, and the design and implementation and use of data from in-trial exit interviews.
Sponsored by Corporate Partner, Thermo Fisher Scientific
Code
031
Topic
Methodological & Statistical Research, Patient-Centered Research, Study Approaches