Over 300 drugs to treat rare diseases now in development, say PhRMA, GA and NORD

At present, there are more than 300 medicines in development to treat or prevent hundreds of rare diseases, according to a new report released by the Pharmaceutical Research and Manufacturers of America (PhRMA). The Genetic Alliance and the National Organization for Rare Disorders joined PhRMA in releasing the report.

The US National Institutes of Health estimates that there are approximately 6,000 rare diseases affecting 25 million Americans. A rare disease is defined as a condition affecting fewer than 200,000 people in the USA. In the past, treatment options for such conditions have been non-existent or limited, but approval of several new medicines in this research sector over the last few years has fostered great progress. Since 1995, more than 160 such drugs were approved to treat rare diseases, compared to 108 in the decade before and fewer than 10 in the 1970s, according to the new PhRMA report.

"Advances in science, such as a better understanding of molecular and genetic causes of disease, have given researchers important new insights to better treat rare diseases, which are often more complex than other diseases," said Billy Tauzin, PhRMA president.