The problem is that after submitting such drugs to expensive testing, drug makers typically jack up the prices, in a position to do so under congressional patent incentives aimed at producing innovative drug research. The FDA has no say in how a drug is priced.
Such was the case with colchicine. Most patients can treat their acute gout symptoms with steroids or nonsteroidal anti-inflammatories like aspirin or ibuprofen. But patients who can't tolerate those drugs rely on colchicine. In addition, an estimated 100,000 Americans who suffer from Familial Mediterranean Fever take colchicine every day to prevent the terrible chest and abdominal pains the rare genetic disorder causes.
Despite the fact that colchicine has been in use since at least the sixth century—but also largely because of it—the drug had never been submitted to rigorous clinical trials.
In 2009, after winning FDA approval, URL Pharma won exclusive rights to sell colchicine as a treatment for acute gout for three years, and seven-year exclusivity to sell it for Familial Mediterranean Fever, under the Orphan Drugs Act. No other companies sought FDA approval—presumably because after spending money to win a new drug license, they would have had to split the market with URL Pharma, forcing both parties to sell at a lower price.
URL Pharma says it tested the drug on 1,000 patients. It did pharmacokinetic studies that helped establish important drug-drug interactions that make colchicine dangerous in some cases. But its license seemed to rest primarily on a trial of just 184 people who got placebo, or colchicine at high or low dosage levels. This was hardly the kind of long, expensive trial that would justify a big price markup.
The company and senior FDA officials maintain that the studies made colchicine much safer than before. For the first time, the drug's labeling contains dosing and drug-interaction information that informs doctors and patients how to use the drug safely. This is important. The question, however, is whether these benefits were worth the drug's increased cost.
In September 2010, the FDA warned all other makers of colchicine to remove their drugs from the shelves by January. Dr. John J. Cush, director of clinical rheumatology at Baylor University in Dallas, is one of an undetermined number of physicians who responded by deciding to boycott Colcrys.
"This is a travesty, a joke that is not funny to patients or rheumatologists, and the FDA has endorsed it and that's unfortunate," said Cush, who has served on FDA drug approval panels and is generally positive about the agency's programs.
In their response to criticism, FDA officials have noted that colchicine had been associated with 117 non-overdose deaths, half of them involving an interaction between colchicine and the antibiotic clarithromycin. As a result of the FDA's review and approval of Colcrys, drug interactions and dosage warnings are now included in the drug's label.
But the dosage information was already part of the American College of Rheumatology's guidelines for gout treatment. Dr. Edward Fudman, an Austin rheumatologist, told a medical newsletter last year that no one in his specialty had used high doses of colchicine in years. URL Pharma "set up a straw man to knock it down," he said. "Doing one trial in patients and a few drug interaction studies doesn't justify marketing exclusivity and a 50-fold increase in price."