In the past and, perhaps to some extent, today, the specialized care and medications required for the appropriate management of patients with rare diseases, such as hemophilia and SLE, was neither readily accessible nor affordable. A collaborative effort for advocacy by patients and physicians has gradually pushed the evolution of legislation and regulations to improve access to optimal management. Two such legislation are the Orphan Drug Act of 1983 and the Rare Diseases Act of 2002, which provided the incentives and means necessary to drive the pharmaceutical research and development to grow a flourishing drug market and pipeline for rare diseases. As a result, managed care policies continue to evolve in many ways, implementing mechanisms and policies to balance treatment access and costs with appropriate medication use and patient outcomes. Patient and physician advocacy for rare diseases prompted a wave of change, but the momentum will need to be maintained.