Session (CTI)

Issue While patient experience data (PED) - including PROs, quality-of-life measures, treatment preferences, and patient-generated data - are central to evidence expectations in U.S. regulatory decisions, their use in payer decision-making remains inconsistent. The FDA’s Patient-Focused Drug Development (PFDD) Guidance Series and the European Medicines Agency draft Reflection Paper on PED signal that patient experience data are considered part of the totality of evidence required for regulatory and access decisions. European HTA bodies increasingly treat QoL and patient-relevant outcomes as core determinants of added benefit and price negotiation. In contrast, the use of PED in formulary and access decisions by payers remains variable, limited to PRO data and coverage exceptions. Overview The panel will explore 3 questions: 1. Why does PED matter now? Panelists will discuss how PED strengthens clinical credibility, captures health outcomes beyond survival, and reflects elements of total cost of care such as functioning, productivity, and caregiver burden. Drawing on ISPOR Europe 2025 themes, the FDA PFDD Guidance Series, and the EMA PED Reflection Paper, the session will highlight expectations that PED be proactively incorporated into regulatory and reimbursement submissions. 2. Why hasn’t PED meaningfully influenced payer decisions? Despite increasing availability of PROs and PED, analyses show limited uptake in coverage and utilization management. Panelists will address methodological uncertainty, lack of standardization, perceived operational burden, and the belief that PED does not materially influence comparative effectiveness. 3. What would it take to change the dynamic? The discussion will identify practical actions for key stakeholders: • Industry: embed fit-for-purpose PED in pivotal trials and real-world studies; develop concise, payer-ready evidence packages. • Regulators/HTA bodies: clarify evidentiary standards; signal PED relevance beyond labeling; align approaches with EMA and FDA initiatives. • Payers: define how PED could influence access thresholds and contracting; increase transparency when PED informs decisions.