The patient’s perspective in the pharmaceutical regulatory and access pathway: considerations and recommendations from the 2025 Patient Forum

Introduction: Despite the growing attention on Patient-Reported Outcome (PRO) and Patient-Reported Experience (PRE), their use for drugs approval and, especially, price and reimbursement is still limited. The present paper illustrates the discussion and recommendations of three interdisciplinary and multi-stakeholder Working Groups (WGs) on this topic.Method: The discussion, oriented by a report from the coordinators, focused on the role of PRO in the comparative evaluation of efficacy and risk, of PRE as an expression of preference for the drugs value components and of PRO and PRE in the regulatory process and national and regional access.Results: The results are reported as reflections and recommendations on the three topics.Conclusions: There is widespread consensus on the need to enhance the use of PRO and PRE, provided that (i) methodological and operational issues are addressed, (ii) PRO and PRE role in the regulatory and access process is clarified, (iii) representatives of patients and institutions are involved in a more structured way (iv) the actual role played in decision-making is clarified.