By Marie Rosenthal
LAS VEGAS—Rare diseases affect more than 30 million people—nearly 10% of the U.S. population—and represent a significant unmet medical need for patients and caregivers. The FDA has approved more than 650 orphan drugs for rare diseases. The number and range of products have challenged payors to balance patient access to innovative medicines with the need to manage rapidly rising costs, so utilization management (UM) is going to be an important concept for