Beyond Regulatory Approval: Making Patient Experience Central to HTA Decision Making

Issue: As European healthcare systems shift toward more patient-centric, value-based care, the traditional evidence hierarchy - centered on randomized controlled trials - is under increasing scrutiny. This panel will examine the transition from "evidence of efficacy" to a broader "evidence of value" framework, emphasizing the evolving role of patient experience and preference data in Health Technology Assessment (HTA), pricing, and access decisions. A significant disconnect exists between how regulatory and HTA bodies value patient experience data (PED). Regulatory agencies like the FDA and EMA have made notable progress, incorporating PROs into labeling and using patient input to inform approvals. In contrast, HTA bodies often may lag behind in systematically integrating PED into evaluations. This inconsistency presents challenges for aligning evidence with both regulatory and HTA expectations, particularly given the diverse methodologies used by different HTA agencies. Overview: Building on our ISPOR Montreal panel on value evidence archetypes, we found that PED - especially PRO data - is typically viewed as “supportive” rather than essential in access decisions. This highlights a misalignment in how stakeholders interpret and apply patient-centered evidence, despite its increasing importance. Through real-world case studies, we’ll explore examples where strong PRO data supported regulatory approval but failed to influence HTA outcomes. We’ll identify strategies to better align PED with HTA decision-making by examining current uses of clinical, economic, real-world, and patient-generated evidence. Special attention will be paid to oncology, rare diseases, and chronic care. Panelists from both pharmaceutical and HTA backgrounds will offer practical insights into optimizing evidence generation plans, aligning with payer expectations, and strengthening the patient voice in value assessment frameworks. Attendees will leave with actionable strategies to enhance the impact of PED for decision making.