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PhRMA Honors Patient Advocates Ron and Raychel Bartek
Date:3/18/2010

g Act provides seven years of market exclusivity to medicines approved to treat a rare disease. Since its passage, the act has spurred increased research for under-treated patients. According to a recent report by the Tufts University Center for the Study of Drug Development, the 425 orphan drug designations for products in development between 2006 and 2008 represent more than twice the number of designations -- 208 -- for the period from 2000 to 2002.

The illnesses being targeted by current rare disease research include rare cancers, autoimmune disorders and cardiovascular disorders. According to a PhRMA survey, some examples of medicines being developed for rare diseases are: a monoclonal antibody for chronic sarcoidosis, an immune system disorder; a medicine for Lennox-Gastaut syndrome, a severe form of epilepsy; a gene therapy for cystic fibrosis; and a medicine for Friedreich's ataxia.

In fact, a 2006 report released by PhRMA and NORD found that more than 160 new medicines for these illnesses had been approved in the decade between 1995 and 2005, compared with 108 such approvals in the decade prior, and fewer than 10 in the 1970s. Many of the new medicines were the first approved to treat a particular disease.

"This recent progress has given under-treated patients access to treatments they've never had before
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SOURCE Pharmaceutical Research and Manufacturers of America
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