Since 1995, America’s pharmaceutical research companies have made great strides in fighting rare diseases. More than 160 new medicines to treat rare or “orphan” diseases have been approved by the Food and Drug Administration (FDA) in the last decade alone.
According to the National Institutes of Health, more than 6,000 rare diseases afflict a total of 25 million Americans. And the FDA says 85 to 90 percent of rare diseases are serious or life-threatening, making the search for new treatments and cures all the more important.
“America’s pharmaceutical research companies have made incredible progress in finding new medicines that offer hope to patients with rare diseases,” says Billy Tauzin, president and CEO of the Pharmaceutical Research and Manufacturers of America (PhRMA). “Continuing innovation by these companies is the key to developing new medicines and offering patients a chance for a brighter, healthier future.”
“A Decade of Innovation: Advances in the Treatment of Rare Diseases”-a new report issued by the National Organization for Rare Disorders, the Genetic Alliance and PhRMA-highlights...