NCPA - National Center for Policy Analysis


February 9, 2006

In 1982, when the Orphan Drug Act was passed as an amendment to the Federal, Food, Drug and Cosmetic Act, few suspected the extent to which this law would alleviate the plight of patients with rare diseases, says Marlene Haffner, director of the Office of Orphan Products Development. The law defines an orphan drug as one with efficacy against a disease affecting fewer than 200,000 people in the United States.

Whether a disease is rare or common, however, the discovery, development, and clinical testing of a drug that can treat it represent a long, arduous, and expensive process. The Orphan Drug Act created government incentives to encourage academic researchers to participate in research on drugs for the treatment of rare diseases.

  • Under this law a company or a researcher can qualify for benefits such as tax credits for clinical development, seven years of exclusive marketing and development assistance from the Orphan Products Office.
  • In the 24 years since this law was passed, 282 such drugs and biologic products providing treatment for more than 14 million patients in the United States have come to market under its aegis.
  • Some 38 of the orphan drugs approved are for cancer, 23 are for infectious disease and nine are for pulmonary diseases.

By most measures, the Orphan Drug Act has been successful in enabling patients with rare diseases to receive treatments that would otherwise never have been developed. Moreover, the Orphan Drug Act has had not only domestic but also global benefits for patients with serious and rare diseases -- benefits that will only expand as genomic medicine fuels more rapid progress in alleviating the effects of devastating diseases, says Haffner.

Source: Marlene E. Haffner, "Adopting Orphan Drugs - Two Dozen Years of Treating Rare Diseases," New England Journal of Medicine, February 2, 2006.


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