PROFIT WINDFALL OR PATIENT WINDFALL? The Orphan Drug Act and Proposals for its Reform

Abstract

The Orphan Drug Act is a statute with a noble purpose but a controversial history. Originally designed to spur research into drugs for conditions with extremely limited patient populations,~ the Act has become a highly successful means of developing new drugs for rare diseases and conditions. In total, these conditions affect a significant percentage of the population -- as many as 1 out of every 13 Americans. In the ten years prior to the Act's passage, only ten drugs had been approved for these conditions, but by December 31, 1993, 569 drugs had received designation as orphan drugs under the Act; of these, over 65 had been approved. Few, then, would dispute that for its intended purpose -- benefit to patients suffering from rare conditions -- the Act has worked successfully.