According to the PharmaTimes, a new PhRMA report says that US drug companies are currently working (all in clinical trials or under FDA review) on 452 new medicines for the treatment of rare diseases. Rare diseases are defined as those with less than 200,000 afflicted in the US. But there are more than 7000 rare diseases and in aggregate this represents about 30 million afflicted or approximately 10% of the US population. The FDA accordingly has approved 1 in 3 new drugs to combat rare diseases in the past five years, with 13 such drugs already having been approved in 2013.
In breaking down the diseases addressed by the 452 rare disease drugs, we find:
- 105 for cancer
- 85 for genetic disorders
- 65 for blood disorders
- 32 for neurological disorders
- 28 for infectious diseases
- 20 for respiratory conditions
Posted by Bruce Lehr Oct 10th 2013.