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Orphan Drug Act at 20: Big gains, some strains

The law has given physicians new tools for treating patients with rare diseases, but some believe the act has not reached its full potential.

By Larry Stevens, amednews correspondent. Aug. 4, 2003.

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In 1991 neurologist Roscoe O. Brady, MD, and his colleagues enjoyed the satisfaction of watching their discovery of the cause of Gaucher disease culminate in a government-approved drug to treat the rare metabolic disorder.

But just a few years later, the unfolding AIDS crisis threatened to undo all of their work.

The treatment -- an enzyme replacement therapy -- relied on limited supplies of human placenta as a source for the deficient enzyme. But worries about HIV/AIDS cast doubt on the safety of products derived from placentas. Another source had to be developed.

So in 1995, Dr. Brady turned to the therapy's maker, Genzyme Corp., for funding to manufacture the enzyme using recombinant DNA. He accomplished his goal, but only because of a federal law, the Orphan Drug Act, which provides companies with financial incentives to produce drugs for rare diseases.

"Genzyme could never have sold this idea to investors or stockholders without the government protection they got from the act," says Dr. Brady, chief of the developmental and metabolic neurology branch at the National Institute of Neurological Disorders and Stroke.

Now 20 years old, the act has succeeded in encouraging drugmakers to invest in uncommon conditions, such as Gaucher disease, that they would have ignored previously because demand never would have been great enough to make treatments profitable.

Before 1983, only a handful of drugs for "orphan" diseases were developed -- 10 by most estimates. Since then the Food and Drug Administration has designated 1,100 drugs as orphan products, of which 231 were approved for marketing.