Sky-High Prices for Orphan Drugs Slam American Families and Insurers

January 17, 2017

(Kaiser Health News) – Cerezyme is an “orphan drug” which means it was created to treat a rare disease, one that affects fewer than 200,000 people in the U.S. The orphan drug program overseen by the Food and Drug Administration is loaded with government incentives and has helped hundreds of thousands of patients like Luke feel better or even stay alive. But the 34-year-old program has opened the door to almost unlimited price tags and created incentives among drugmakers to cash in, and to cash in repeatedly, a Kaiser Health News investigation shows.