The 30Th Anniversary of the Orphan Drug Actby Former Representative Henry A. Waxman
Posted on 2013-01-04
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 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, 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 [[Page E14]] 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.