January 4, 1983
I am pleased to sign into law today the Orphan Drug Act.
Over the past century, the United States -- largely through innovative pioneering by private industry and medical researchers in universities -- has led the world in developing new drugs that have saved millions of lives. That is a gift to mankind we can be very proud of.
Yet the sad fact remains that many diseases still cripple or kill hundreds of thousands of Americans, as well as citizens of other countries, because no drugs have yet been developed. These diseases include cystic fibrosis, Wilson's disease, myoclonus, Tourette's syndrome, and certain neuromuscular disorders and cardiac arrhythmias. Statistically, they are rare; yet that is small comfort for those afflicted.
The cost of discovering and developing a new drug is often staggering. By definition, an orphan drug is one that treats a disease that affects 200,000 or fewer individuals -- and, from an economic perspective, groups that small do not now justify the kind of research expenditures that companies must make.
The bill that I am signing today helps to cure that problem and consequently, we hope, some of the diseases as well. The bill provides incentives for the private sector to develop drugs to treat these rare diseases.
It should be pointed out that the Department of Health and Human Services has already made significant progress in this area. Secretary Schweiker established an Orphan Products Board in March 1982, with membership and functions similar to those in the bill. This bill will enhance the steps we have already taken to encourage the development of orphan drugs and ensure that our ongoing program will be permanent. This legislation exemplifies the proper role of government in helping meet legitimate needs in those cases where the free market alone can't do the job.
I am approving this legislation despite the inclusion of a provision about which I have grave reservations. Section 7 of the bill directs the Secretary of Health and Human Services to publish tables showing a causal relationship between radiation exposure and subsequent cancer. The relationship between cancer and low levels of ionizing radiation has long been the subject of research by scientists throughout the world. Despite this intense interest, there is as yet no consensus among radiation experts in relating human cancers and exposure to low levels of radiation. Yet section 7 mandates that probability of causation tables be calculated for even very small dose levels. Accordingly, I am directing the Secretary of Health and Human Services to complete the tables to the extent that may be possible and scientifically responsible, in light of the analysis also mandated by section 7, which requires him to ``assess the credibility, validity, and degree of certainty associated with such tables.''
Despite my reservations on section 7, I am gladly signing the orphan drug bill. I only wish with the stroke of this pen I could also decree that the pain and heartache of people who suffer from these diseases would cease.
Note: As enacted, H.R. 5238 is Public Law 97 - 414, approved January 4.