The Food, Drug and Cosmetics Act of 1938 and the 1962 amendments to that law charged the Food and Drug Administration (FDA) with approving drugs that are safe and effective. Colchicine is one of many medications that was unapproved and was widely available before the act took effect. In June of 2006, the FDA and its Center for Drug Evaluation and Research (CDER) undertook the Unapproved Drugs Initiative to bring more of these older drugs into compliance.
“Our mission is to ensure that safe and effective drugs are available,” writes Michael Levy, Esq., director of CDER’s Division of New Drugs and Labeling Compliance, in an e-mail response to questions. “Since the initiative began, marketers of unapproved drugs are on notice that addressing risks from unapproved drugs is a high priority, and that the agency plans to ensure all products on the prescription drug market comply with current requirements for safety and efficacy.”
The approval pathways a pharmaceutical company takes to gain approval to market existing unapproved drugs are the same as for new drugs. Generally, all new prescription drugs must go through the New Drug Approval (NDA) process before being legally marketed. Since the initiative started, over 500 unapproved products have been removed from the market and more than 70 older, unapproved drugs have earned approval.
URL Pharma and the Rheumatology Market
URL Pharma, based in Philadelphia, decided to focus on colchicine, among other products. “We looked at the universe of unapproved drugs, searching for medications presenting safety risks or where we had a chance to improve efficacy,” says Richard Roberts, MD, president, chair, and CEO of URL Pharma. “As a physician, I was shocked that colchicine, with all of the toxicities taught [to] every doctor since medical school, was not an approved medication.”
In July 2009, the FDA granted URL Pharma approval to market a form of colchicine for acute gout and familial Mediterranean fever (FMF). An indication for gout flare prophylaxis followed in October. They were given three-year marketing exclusivity for gout and a seven-year term for FMF as an orphan drug. The branded medication called Colcrys sells for about $5.00 a tablet, or roughly 50 times the cost of the unapproved version.
The ACR, responding to member concerns about the new pricing, acted quickly, first consulting with the FDA to discuss its concerns, and then contacting URL Pharma. (A summary of the ACR’s discussion with the FDA is available online at www.rheumatology.org/advocacy/colchicine_ fda.pdf.) The ACR worked closely with URL Pharma on changes to its Colcrys patient assistance program (PAP), resulting in wider coverage. The ACR has also obtained a list of other colchicine makers and has encouraged them to go through the approval process for other indications.