[Congressional Record (Bound Edition), Volume 159 (2013), Part 1]
[Extensions of Remarks]
[Pages 102-103]
[From the U.S. Government Publishing Office, www.gpo.gov]




              THE 30TH ANNIVERSARY OF THE ORPHAN DRUG ACT

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                          HON. HENRY A. WAXMAN

                             of california

                    in the house of representatives

                        Friday, January 4, 2013

  Mr. WAXMAN. Mr. Speaker, as we begin a new Congress with many 
disappointments behind us and many challenges before us, I'd like to 
take a moment to note legislation that has proven successful beyond all 
expectations--the Orphan Drug Act. And I'd also like to note the recent 
passing of one of that bill's first champions--Jack Klugman.
  Before 1983, people suffering from a rare disease had little or no 
hope that a treatment or cure would be developed, despite the fact that 
many potential treatments for those diseases existed. By definition, 
the market for a drug for rare disease was too small to attract drug 
companies to do the research and go through the approval process. It 
was a classic case of market failure. And, for better or for worse, 
pharmaceutical research usually depends on the market.
  So we had to invent an incentive for the pharmaceutical industry to 
do something that was squarely in the public interest. We had to make 
the development of orphan treatments something companies themselves 
wanted to do.
  To do so, we created incentives: intellectual-property protections, 
tax benefits, and regulatory advantages. But, like many good bills on 
complicated issues, the Orphan Drug Act got bogged down.
  And that's when the unexpected champion emerged--the Hollywood actor 
Jack Klugman. He was a big star and he had read about the problems of 
people with rare diseases. He used his television show, Quincy, to 
highlight the issue. (The show even hired people with rare diseases to 
serve as extras in a fictional demonstration outside the Capitol.) He 
was a witness before the Subcommittee on Health and the Environment, 
which I chaired. He went on to bring national attention to this problem 
that affected many, many people--but people in groups so small that 
they could never on their own get the help they needed.
  The bill passed the House. Ultimately, with the help of my friend and 
colleague, Senator Orrin Hatch, it also passed the Senate. President 
Reagan signed it into law on January 4, 1983.
  And the rest, as they say, is history--good history. The Act has been 
very successful. Over the thirty years between then and now,

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hundreds of orphan drugs have been approved and millions of Americans 
with rare diseases have been helped.
  There is still a lot to be done. There are thousands of rare diseases 
without treatments. The cost of many orphan drugs and many other drugs 
is still far too high. But this Act and the amendments made to it since 
then have made great progress.
  I'm sad to say that Jack Klugman died last week. He'd had a great 
career in entertainment and he will be remembered for that. But we can 
also all be grateful to him for his starring role in bringing this bill 
to the public and helping it become law. And we in the Congress can be 
proud of what the Orphan Drug Act has done.

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