PURPOSE: Workshop attendees will: 1. Learn about the development of a PPI study designed for regulatory purposes 2. Learn about a roadmap designed to identify, select, and translate a PROM into attributes for PPI studies • Specify the regulatory and research question
DESCRIPTION: With increasing interest in the use of PPI to support regulatory decisions, much attention has been paid to attribute selection for regulatory PPI studies. Clinical trial endpoints using PROMs can be an important source for PPI attribute development, as they include concepts relevant to both patients and regulators and are often validated in the target population. The adaptation of a PROM to PPI attributes involves several steps and considerations, as outlined in this roadmap:
• Understand the clinical context: collect, assemble and prioritize all relevant outcomes and endpoints
• Select PROM from confirmatory trials: ensure relevance and saliency to patients and stakeholders
• Convert selected PROM into PPI attributes: select domain(s) of interest and determine measurement model
• Choose appropriate levels for the PROM: encompass clinically-relevant range of change, informed by established meaningful change in domain score when available, and clear definition of the meaning of a change in selected PROM score
• Finalize attributes and levels for survey instrument development
• Pretest the PPI survey instrument to evaluate consistency with PROM items
Using case examples across different therapeutic areas, presenters will discuss issues, challenges and potential solutions associated with the roadmap. Each speaker will present for 10 minutes followed by an interactive discussion with the audience. Attendees will have the opportunity to discuss this challenging issue in an emerging area of interest with academia, regulatory and industry experts.
