A drug called colchicine is all that keeps some 2 million American gout patients from suffering debilitating pain in their toes, elbows, wrists, and fingers. Doctors have prescribed the compound, derived from the seeds of the autumn crocus, for centuries. But patients who take colchicine woke up with a new symptom recently: a giant pain in the wallet.
Until January, colchicine was sold by many companies and cost as little as 10 cents a pill. Now it's available only under the trade name Colcrys, sold by a Philadelphia company called URL Pharma—for five dollars per pill. * The colchicine story, and a few others like it, have provoked ire among some patients and doctors about an otherwise praiseworthy effort by the FDA to get rid of old, untested, potentially harmful drugs.
Take the case of 17 alpha-hydroxyprogesterone caproate (17OHP), a synthetic hormone designed to prevent preterm births in pregnant women. An unapproved form of the drug was previously formulated at specialized pharmacies. In February, after K-V Pharmaceutical Co. submitted its version of the drug to rigorous testing, FDA approved the new drug under the trade name Makena. Pharmacies that try to sell their own forms of 170HP now risk FDA enforcement action such as fines or raids.
The drug's retail price jumped from $15 to $1,440 per treatment, a shock to obstetricians and their patients, as well as to those who write checks at insurance companies and Medicaid. The tens of thousands of women who receive the treatment each year typically require 20 injections during the course of their pregnancies.
Sens. Sherrod Brown, D-Ohio, and Amy Klobuchar, D-Minn., have called for a price-gouging investigation of K-V Pharmaceuticals, and they may be on to something. Several physicians and patients who use colchicine have also asked for congressional investigations.
One can't fault the FDA for the price increases for Makeda and Colcrys; the agency is doing what it's supposed to do, clearing out old drugs and improving the standards of the national Pharmacopoeia. (Disclosure: I worked as a speechwriter for FDA in early 2010 but had no involvement in the colchicine or 17OHP issues.) But it seems unjust—if legal—for companies to take unscrupulous advantage of patients and doctors who rely on already well-established drugs.
The colchicine and 17OHP stories have their roots in the FDA's historically complex relationship with the drug industry. Since 1962, the agency has required that all new drugs be proven safe and efficacious before hitting the market. Many drugs marketed before 1962, however, remain on sale without having been formally approved by the FDA and are technically illegal. In 2006, the FDA launched the Unapproved Drugs Initiative, aimed at getting rid of as many of these drugs as possible.
When the FDA launched its initiative, unapproved drugs made up about 2 percent of the prescriptions written in the United States, estimates Michael Levy, director of the FDA's New Drugs and Labeling Compliance office. Today, they account for slightly more than 1 percent. The agency has removed more than 1,000 unapproved drugs from the market.
Not all drugs are targeted equally: The agency takes enforcement action based on a hierarchy of needs, starting with safety. Some of the agency's biggest targets, for this reason, have been 120 cold medicines containing carbinoxamine, an antihistamine linked to 21 infant deaths. And it tends not to go after unapproved drugs if banning them would create a gap in medical care; the FDA, for instance, has not moved to withdraw phenobarbital, an old anti-epilepsy drug that is still the treatment of choice for certain types of seizures.
The FDA campaign has two approaches. In some cases, the agency simply warns companies to stop producing and shipping unlicensed drugs by a given date. In other cases the FDA warns a group of companies producing a particular class of drug, notifying them that it plans to crack down on their unapproved substances. The idea here is to give the companies an opportunity to submit their drugs to the rigorous testing required for FDA approval. This is what happened with Colcrys and Makeda, and at least 86 other newly approved drugs.