Presentation (CTI)

OBJECTIVES: Patient experience data (PED) are increasingly recognized as essential for Health Technology Assessment (HTA) and Health Economics and Outcomes Research (HEOR). The FDA's Patient-Focused Drug Development (PFDD) guidance and the European Medicines Agency's (EMA) 2025 Reflection Paper emphasize rigorous methods for collection and analysis of PED. However, standardized frameworks harmonizing FDA and EMA requirements and leveraging data collected for other healthcare decision-making settings (HCDM), such as HTA and HEOR, remain limited.
METHODS: A targeted review was conducted of FDA PFDD guidance documents, EMA reflection papers, ISPOR Task Force guidance, and peer-reviewed literature on PED collection within HCDM. Sources were reviewed to identify methodological approaches, current standards, and quality assurance requirements for PED for FDA, EMA and HCDM. Data collection methods and best practices identified were categorized and synthesized.
RESULTS: Current literature supports utilizing rigorous qualitative, quantitative and mixed methods data collection in representative samples to fully understand relevant patient experience. Data collection methodologies and best practice identified included: qualitative approaches (in-depth interviews, focus groups, in-trial interviews), quantitative methods (PROs and COAs, surveys, patient preference studies, registries, health records databases, safety monitoring), digital health technology (mobile apps, wearables), and mixed methods approaches (e.g. Delphi studies, expert elicitation). Our review highlighted variability in selection and execution of PED for HTA. Based on our review, PED collection leveraging best practice regulatory guidance ensures a defensible and systematic foundation, aligned with HEOR and HTA expectations. We propose a framework comprising several interconnected domains including: external stakeholder identification, PED methodology selection aligned with regulatory and HTA requirements and quality assurance.
CONCLUSIONS: This framework provides HEOR practitioners with actionable guidance for selecting among the myriad of PED collection methods throughout drug development, to generate high-quality PED aligned with regulatory expectations. Implementation of this will support emerging PFDD requirements, enhance HTA submissions, and advance patient-centered decision-making.