Rare illness? NIH creating list of approved drugs....

The National Institutes of Health (NIH) has "begun screening the first definitive collection of thousands of approved drugs for clinical use against rare and neglected diseases."

"They are hunting for additional uses of the drugs hoping to find off-label therapies for some of the 6,000 rare diseases that afflict 25 million Americans," says an announcement from the NIH.

The federal government's Chemical Genomic Center is coordinating the effort.

Rare diseases often don't get the attention they would if more people (and thus, more advocates) were affected.

The NIH, led by Dr. Francis Collins, is working to find ways to use already-approved drugs in new ways because it's much easier to go ahead an make use of a drug that's already been through the safety-approval process. Each of the already-marketed drugs is a known entity, with known side effects and known safety guidelines.

"The current focus is on collaborating with disease foundations, industry and academic investigators with disease-relevant assays to screen against the approved drug collection…," the NIH reports. Any new use of an already-approved drug would need confirmatory studies.

Still, years could be cut from the developmental process if drugs are already being used to treat another condition and are coincidentally found to help in the treatment of a rare disease.

"Creating a new drug is expensive," the NIH says. "Recouping the investment can be difficult for rare diseases, due to the small number of patients with the disease or, in the case of tropical neglected diseases, the limited ability of patients to pay for treatments."

Thalidomide, which caused birth defects when pregnant women took it for morning sickness, was "repurposed" for use as a treatment against leprosy and for multiple myeloma patients, the NIH reports. 

The NIH also plans to screen existing drugs to determine their toxicity.

Pulse on Health
By Jeff Hansel, member Association of Health Care Journalists
Health Reporter for the Post-Bulletin newspaper, 18 1st Ave. S.E. in Rochester, Minnesota 55904 
Twitter Hansel's Pulse: @Jeff Hansel