According to the Orphan Drug Act, a rare disease affects less than 200,000 people in the United States. Support from FDA, EMA and other regulatory agencies, combined with technological and scientific advances, have made it more feasible for pharmaceutical companies to give dedicated attention to orphan drug development. In doing so, they’re developing medications for rare diseases that often have no cure and no available treatment. To ensure novel therapies advance from the lab to regulatory approval, a blockbuster-drug approach won’t work. Managing rare disease studies to completion requires innovative thinking in nearly every phase of program development. Those willing to become trailblazers have the highest odds of success.