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medicines-in-development-drug-list-rare-diseases.pdf
May 9th, 2016
A report by an industry group says 566 drugs designed to treat rare diseases are now in clinical testing by pharmaceutical companies. The report, prepared by Pharmaceutical Research and Manufacturers of America, or PhRMA, and the ALS Association, credits many of these new drugs to incentives in legislation encouraging research and development on treatments for rare diseases.
These 7,000 rare disorders, also known as orphan diseases, each affect less than 200,000 individuals in the U.S. But taken together, says the report, orphan diseases affect about 30 million, or 1 in 10, Americans. Because of the small numbers — sometimes just a few hundred people — affected by each disease, diagnostics and development of new drugs are difficult. Few clinicians have experience with these diseases, and the biological pathways for treating the disorders are often complicated. In addition, the small numbers of potential patients make it difficult to recruit participants for clinical trials.....
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