Here's why this is particularly bad news for rural hospitals: many pharmaceuticals classified as orphan drugs do not actually treat rare diseases. "The Food and Drug Administration gives the orphan drug designation to a medicine as a first step when it agrees with a drugmaker's request to study whether the medicine can be used to treat a specific rare disease. And this can happen even if a drug is already FDA-approved and on the market for use in treating a common condition," Tribble reports. So widely used drugs such as the clot-busting Activase, which must be used immediately to help stroke victims, are too expensive to keep on hand. Activase has not been approved to treat a rare disease, but the FDA granted it orphan status in 2003 and 2014 because its manufacturer, Genentech, said it is researching possible ways for Activase to treat rare diseases.
"If we don't keep this drug [in stock], people will die," hospital pharmacist Mandy Langston told Tribble. Where she works, Stone County Medical Center in Mountain View, Ark., a single dose of Activase costs $8,010. But just miles down the road, regional hospital White River Medical Center pays only $1,600 per dose.
The orphan drug clause affects cancer drugs too. At Cass County Memorial Hospital in Atlantic, Iowa, cancer patients may have to wait a few days to begin treatment so the hospital can order the medications, otherwise too expensive to keep on hand. And "in Vermont, North Country Hospital closed its infusion center this spring due to the soaring cost of medicines," Tribble reports. Cancer patients who live nearby must now drive 45 minutes for treatment.
Some drugmakers, such as Janssen Pharmaceuticals, voluntarily offer discounts to rural hospitals on all of their orphan drugs. "In contrast, drugmaker Genentech sent letters to rural hospitals on Jan. 1 listing dozens of drugs that would not qualify for discounts — including Activase and cancer drug Avastin," Tribble reports.