Delay in Patients’ Access Is the Price of Misalignment: Why Evidence Must Speak Also the Language of Payers and Patients

Biotech companies, regulators, payers, physicians, and patients all seek to understand the value new medicines may bring to current treatment pathways. However, each stakeholder defines "value" differently. For regulators, value typically centers around efficacy, safety, and manufacturing quality. Physicians consider treatment’s place in therapy and implementation feasibility. Budget holders focus on comparative clinical effectiveness, population size, affordability, and value for money. Meanwhile, patients prioritize also out-of-pocket costs, convenience, impact on daily functioning and independence, and implications for families. However, clinical trials are often designed primarily to meet regulatory requirements with new medicines unable to articulate a compelling value story to the diverse stakeholders, resulting in delayed or even denied access and patient uptake. Increasingly, RWE, advisory boards, and stakeholder engagement strategies are used to bridge this gap. Yet critical questions remain: Are these engagements occurring early enough in the product lifecycle? Do they meaningfully and equally incorporate the insights of all relevant stakeholders? Are feedback loops structured to enable tangible influence on evidence generation plans? And are opportunities for collaborative research being explored, resourced adequately, and successfully implemented? This panel, moderated by Cristina Masseria, will bring together diverse perspectives from across the health ecosystem -with Adrian Cassidy from industry, Entela Xoxi as payer, and a patient advocate- to explore how companies can better integrate the voices and needs of different stakeholders into evidence generation strategies. Panelists will reflect on current practices, share examples, and debate what changes are needed to more effectively align evidence planning with the broader definitions of value held across the system also in consideration of EU-JCA. Audience engagement will play a key role, with time for Q&A and experience sharing. The goal is to identify actionable strategies to align evidence generation with broader definitions of value, supporting timely and equitable access to innovation.