PhRMA: More than 400 rare disease treatments in development, pending approval

80% of rare diseases affect fewer than 6,000 patients

WASHINGTON — More than 400 drugs are in late-stage clinical development or awaiting Food and Drug Administration approval as treatments for rare diseases, according to a report by the drug industry lobby.

The Pharmaceutical Research and Manufacturers of America said a record 460 drugs were in late stages of the pipeline in a report timed to coincide with Rare Disease Day on Feb. 28. The rare or “orphan” diseases — defined by the government as those that affect fewer than 200,000 Americans — range from autoimmune disorders to cancer to blood disorders to genetic diseases. In total, there are 7,000 known rare diseases, about half of which affect children, and 80% of rare diseases affect fewer than 6,000 patients.

“Once you’ve talked to a desperate parent whose child is suffering or dying, you will be forever convinced of the need for medical innovation, including for terrible diseases that afflict even just a few,” PhRMA president and CEO John Castellani said. “Researchers at biopharmaceutical companies are working every day to bring hope to patients with limited options.”


- 10:33 AM says

Just a shout out to NORD ,Global Genes, Genetic Alliance and all of the wonderful organizations that are fighting for these treatments and for helping get more attention to Rare Diseases. As a Rare Mom, its a full time job finding the name of the disease, educating the medical community about it, and then getting the government to help us. This is an incredible community of intelligent, brave people.Great job moving the needle.

Login or Register to post a comment.