In recent years, rare diseases have attracted significant interest by drug manufacturers. Legislative and financial incentives, combined with scientific and technological advances, have stimulated the development of novel rare disease treatments. Today, almost 600 orphan drugs are approved in over 700 indication. However, most of the estimated 8,000 rare diseases, cumulatively effecting approximately 350 million people globally, remain without effective treatments. The pathway from identifying a rare disease to sustainable patient access to an effective treatment is still fraught with significant challenges, some of which are unique to rare diseases. The Rare Disease Special Interest Group offers a platform for ISPOR members to address these challenges in support of patients, clinicians, researchers and drug developers concerned with rare diseases. Join us for a brief presentation of the Special Interest Group and the opportunity to propose and discuss topics that the group should advance or volunteer for any future activities.