Presentation (CTI)

OBJECTIVES: As therapeutic landscapes evolve, structured evaluation of patient-reported outcomes (PRO) is essential to support market access, regulatory alignment, and competitive differentiation. This study conducted a retrospective review to assess the concept-level breadth and strength of PRO evidence used across clinical trials and real-world studies for a marketed treatment in a rare, chronic condition. The objective was to identify strengths, gaps, and differentiation potential based on how well key patient-prioritized concepts were captured, using a methodology that integrates psychometric rigor with stakeholder expectations.
METHODS: A structured, multi-phase framework was applied, incorporating a multi-dimensional weighting methodology to evaluate how well key concepts were captured by PRO instruments across diverse evidence sources. Evidence Compilation: Systematic review of clinical trials, observational studies, and regulatory documentation to identify PROs used to assess symptoms, impacts, and treatment experience. Psychometric & Conceptual Evaluation: Evaluation of instruments for reliability, validity, responsiveness, and alignment with disease-relevant concepts. Conceptual Mapping and Weighted Tiering: Mapping of PROs to key patient-centered concepts (e.g., pain, fatigue, gastrointestinal [GI] symptoms, emotional well-being) and weighting based on psychometric strength, regulatory alignment, and contextual relevance. Strategic Application: Tiered summaries and concept-level maps highlighted strengths and evidence gaps in PRO coverage, supporting alignment with evolving regulatory and HTA expectations.
RESULTS: Concept mapping showed that core concepts—such as fatigue, GI symptoms, pain, and cognitive function—were consistently represented, while others (e.g., emotional well-being, daily functioning) were underrepresented. The multi‑dimensional weighting and tiering analysis revealed instruments with strong psychometric properties and regulatory alignment, while pinpointing where further evidence could support differentiation.
CONCLUSIONS: This multi-dimensional weighting methodology enables scalable, concept-level evaluation of PRO instruments to optimize evidence and support strategic differentiation. By integrating psychometric rigor with regulatory alignment and coverage of patient-prioritized concepts, the framework supports both the differentiation of marketed treatments and informs future endpoint selection in clinical development.