Session (CTI)

Purpose To equip attendees with tools to engage FDA in developing and validating clinical outcome assessments (COAs). Drawing on a six-year FDA collaboration, we share insights and strategies for sponsors navigating the regulatory landscape of patient-reported and performance outcome measures. Description Developing COAs for regulatory purposes requires strategic decision-making throughout the research lifecycle. Disease-specific COA development demands substantial investment and multi-year timelines, creating barriers for rare diseases and small populations. While FDA guidance provides frameworks, sponsors face practical questions: What evidence demonstrates cross-disease utility? How should rigor be balanced with regulatory pragmatism? How can sponsors optimize limited FDA interactions? This interactive workshop draws on NUCOAT's (Northwestern University Clinical Outcome Assessment Team) position as an FDA-funded collaborative grant (PI: David Cella, PhD), enabling six years of near-monthly FDA consultation while developing physical function measures across rare diseases and sarcopenia of aging. This offered opportunities to test assumptions, refine methods, and understand which evidence most influenced FDA discussions. Attendees will navigate key "forks in the road"—decision points where methodological or strategic choices shaped NUCOAT's trajectory and FDA input proved pivotal, spanning: concept elicitation and content validity, measurement properties and validation, meaningful change determination, and qualification pathway planning. For each fork, speakers present real-world challenges. Attendees vote on preferred approaches via live polling, discuss with neighbors, then learn what pathway NUCOAT selected and how FDA engagement informed the decision. Each section concludes with practical "pearls" on anticipating regulatory concerns, prioritizing evidence, and preparing Drug Development Tool submissions. The workshop features study leadership and an FDA representative offering complementary perspectives to help attendees prepare for their own FDA engagements.