Study: Hundreds of rare disease drugs in testing
WASHINGTON - Good news for nearly 30 million Americans with a rare disease: Drugmakers are doing much more research on rare, often-deadly diseases with few or no treatment options.
They're running patient tests of more than 5,400 potential new medicines, many for multiple conditions. Nearly 1,800 research projects are for rare diseases. Hundreds more are for disorders with no new treatments in many years.
That's according to a study being released Thursday by the trade group Pharmaceutical Research and Manufacturers of America.
It found that thousands of those experimental drugs could be the first in a new medicine class with a unique target or effect. Nearly 600 use new technologies, from gene therapy to cloned antibodies, to precisely target the disease site. Others are for tiny subgroups of patients with genetic variations.