[Congressional Record Volume 140, Number 35 (Thursday, March 24, 1994)]
[Extensions of Remarks]
[Page E]
From the Congressional Record Online through the Government Printing Office [www.gpo.gov]


[Congressional Record: March 24, 1994]
From the Congressional Record Online via GPO Access [wais.access.gpo.gov]

 
         INTRODUCTION OF THE ORPHAN DRUG ACT AMENDMENTS OF 1994

                                 ______


                          HON. HENRY A. WAXMAN

                             of california

                    in the house of representatives

                        Thursday, March 24, 1994

  Mr. WAXMAN. Mr. Speaker, I am pleased to introduce the Orphan Drug 
Act Amendments of 1994. This legislation will amend the Orphan Drug Act 
of 1983, which was designed to stimulate research on drugs for rare 
diseases and disorders.
  The Orphan Drug Act has been a resounding success. During the past 10 
years, more than 500 orphan drugs have been designated by FDA and more 
than 100 of these have been approved for patient use. This compares to 
a mere 10 orphan drugs approved in the decade before the law was 
passed. As a result of the act, many thousands of rare-disease patients 
who had previously felt abandoned now have hope--and, in many cases, 
they also have a new lease on life.
  In fact, on some occasions the act has been almost too successful. In 
recent years there have been several drugs with sales of $200 million 
per year or more than have qualified as orphan drugs under the act. The 
Orphan Drug Act was never intended to grant marketing exclusivity to 
these blockbuster drugs. The commercial market for these drugs provides 
many incentives for their development. Instead it was intended for 
drugs for rare diseases, which have so little prospect of profit that 
they would not have been developed without the incentives of the Orphan 
Drug Act.
  The bill am introducing today will correct this imbalance. Under the 
legislation, orphan drugs will be limited to 4 years of exclusivity. 
However, sponsors of the drugs will be able to obtain an additional 3 
years of exclusivity if they can demonstrate to the Food and Drug 
Administration that their drug has limited commercial potential. In 
addition, the bill provides that where drug is so profitable that more 
than one company is interested in developing it, both companies can get 
their drugs on the market if they filed their applications for 
marketing within 1 year of each other and if they meet certain other 
conditions.
  Mr. Speaker, striking the appropriate balance for the Orphan Drug Act 
has been a delicate issue. On the one hand, we do not want the act used 
for blockbuster drugs which clearly would have been developed without 
the additional marketing exclusivity that it provides. On the other 
hand, we want the incentives in the act to continue to be generous so 
that companies will continue to invest in orphan drugs.
  I believe that this bill strikes the right balance. I am pleased to 
announce that it has been endorsed by the Biotechnology Industry 
Organization [BIO], the trade association for the biotechnology 
industry, which includes most of the companies that have used the act. 
It has also been endorsed by the National Organization for Rare 
Disorders, which represents patients who have the diseases which these 
drugs are designed to treat.
  I am also pleased that my colleague, Mr. Studds, has joined me as a 
cosponsor of the bill. Mr. Studds has been a tireless advocate of 
disadvantaged patients and has worked hard to help make drugs more 
affordable by amending the Orphan Drug Act.
  Finally I would like to note for the record that a companion bill has 
been introduced in the other body by Senators Kassebaum, Metzenbaum, 
and Kennedy.
  I have prepared a summary of the bill. I ask that it and the bill be 
included in the Record after this statement.

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