Here is an excerpt from an article we found in the Wall Street Journal, “Push to Cure Rare Diseases, by Amy Docker Marcus:

CLAREMONT, Calif.—Staff members at the Food and Drug Administration are doing something unusual. They are leaving Washington to help drug makers take a crucial step in developing drugs for rare diseases.

The staffers help administer the Orphan Drug Act, which provides incentives to create therapies for so-called orphan diseases—those that affect fewer than 200,000 Americans. There are about 7,000 such maladies, most of them serious, that have few or no drugs to treat them, from adenoid cystic carcinoma, a rare head and neck cancer, to Zollinger-Ellison syndrome, which is associated with a tumor that causes the production of high levels of stomach acid.

As a result, doctors may end up prescribing drugs developed for other diseases off-label, but not all insurers will cover this kind of use.

Getting an orphan-drug designation opens the door to incentives once the FDA approves a medicine for sale in the U.S., including seven years’ marketing exclusivity and tax breaks. Last year, just 250 requests for orphan-drug designation were filed, and 160 received it.

Read this extensive and important article by going to: FDA Officials Go to New Lengths to Encourage Applications for Orphan-Drug Status.

Feel good and keep smiling! Pat & Pattie

Leave a Reply