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December 13, 1993

The Orphan Drug Act: Should It Be Changed?

Author Affiliations

Glaxo Inc Research Triangle Park, NC 27709

Arch Intern Med. 1993;153(23):2623-2625. doi:10.1001/archinte.1993.00410230025005

IT IS A FACT of political life that much legislation, no matter how well intended and well crafted, often has unforeseen effects. That is one of the reasons the Orphan Drug Act of 1983 is such an outstanding example of successful law making. It has done exactly what its supporters intended it to do. The act represents sound and responsible public policy. But, more importantly, it has served millions of people suffering from rare diseases.

The Congress, therefore, should deliberate very carefully before changing it. The act seeks to encourage development of pharmaceutical therapies for disorders that afflict fewer than 200 000 people by providing incentives for drug companies to undertake the task. Recognizing that market incentives alone are inadequate given the relatively small patient size, it provides tax credits and 7 years of market exclusivity to pharmaceutical firms willing to "adopt" and develop a drug.

A look at the