ISSUE: Of the 7,000 recognized rare diseases, only ~400 have currently licensed treatments, meaning clinicians often have no choice but to prescribe drugs off-label. Although providing a short-term solution for some individuals, off-label use can theoretically have a negative impact on long-term patient access, by potentially reducing the incentive to conduct time-consuming and costly evidence generation processes in order to inform licensing or reimbursement applications. The potential failings in the current system in terms of off-label drug use in rare diseases, and their impact on patient access will be discussed, and strategies suggested to improve the process at the stages of drug development, clinical practice and reimbursement. Annabel Griffiths will moderate and provide an overview of the current system. Jack Scannell will present the financial perspective and the role of off-label treatments in drug development for rare diseases. Oliver Timmis will voice the patient perspective, highlighting challenges faced by the rare diseases patient community due to the use of off-label treatments. Pilar Pinilla-Dominguez will represent the HTA perspective, and discuss the impact of off-label drugs on the methodologies NICE uses for evaluations. OVERVIEW: Off-label prescriptions are particularly common in rare diseases, with estimates suggesting that 90% of all prescribed treatments for rare diseases lack specific FDA approval. The use of off-label treatments can, however, be detrimental to long-term patient access, limiting the patient population eligible for inclusion in clinical trials and possibly reducing the incentive for formal licensing and reimbursement. This issue panel will explore the impact of off-label prescriptions on patient access in rare diseases, highlighting issues faced by industry, clinicians and payers, and asking what changes could improve the system for all stakeholders. The audience will vote on their opinion on the title question at the beginning and end of the issue panel; discussion time will be included, and questions from the audience encouraged throughout.