Edward M. Kennedy: “I join in sponsoring the Orphan Drug Act amendments of 1994. This legislation provides important and timely improvements…”

“I join in sponsoring the Orphan Drug Act amendments of 1994. This legislation provides important and timely improvements to a law which has led to the development of vital new medicines for the treatment of rare diseases. It is widely agreed that the Orphan Drug Act, originally enacted in 1983, has been a tremendous success. The purpose of the act was to create incentives for the development of drugs for diseases so rare that the drugs have little commercial value. Innovative companies, particularly biotechnology companies, need the market exclusivity offered by the Orphan Drug Act to develop drugs for such diseases, since patent protection may be unavailable for their products. There are 102 orphan drugs currently on the market, and over 500 under investigation. However, a handful of orphan drugs now on the market may reach $1 billion in multiyear sales--a record reached by few drugs in history, much less drugs of supposedly limited commercial value. For several years, the perception that the Orphan Drug Act can be a shelter for high-priced drugs has fueled debate which threatened to undermine the strong support for the act by the public, industry, and Congress. Changes are needed to maintain the integrity of the act while assuring that its protections are not misused to produce unreasonable prices and profits. The legislation offered today resolves any uncertainties over the future of orphan drug development.”