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The High Price of FDA Approval

Several months ago, Doris Webb was diagnosed with a rare disease called Familial Mediterranean Fever, a hereditary condition tied to her French ancestry that causes fevers, arthritis attacks and inflammation of the lining of the lungs and abdomen. Debilitating pain in her joints and bones was relieved by a prescription drug called colchicine.

Webb, of Morristown, Tenn., takes two to three tablets a day, paying $11 at Wal-Mart for a 90-day supply, according to her daughter Tina Martin, who also takes colchicine for FMF. The drug, which has long been used to treat gout, is cheap because, like thousands of prescription drugs, it pre-dates modern drug laws and has never been approved by the Food and Drug Administration.

Now, however, Webb, 66, who’s on Medicare but can’t afford the Part D drug benefit, her daughter says, faces a problem in getting her colchicine. In July, Philadelphia-based URL Pharma won FDA approval for a branded version called Colcrys, which sells for about $4.50 a tablet – nearly 50 times the price of the unapproved version. While the uninsured will be hit hardest by the cost increase, even insured patients, like Martin, will face higher costs. She says her copayment will rise from $10 to $35 for a 90-day supply.

The FDA found that Colcrys’ drug interaction labeling and recommended dosing regimen make it safer than the unapproved forms of colchicine. The agency said it had received reports of 120 patient deaths from interactions of unapproved colchicine with other drugs. It granted URL Pharma and Colcrys three years exclusivity for treatment of gout – a recurrent arthritic inflammatory disease caused by uric acid buildup – and seven years for FMF under orphan drug rules.

But some rheumatologists and patients’ groups charge that the FDA is letting URL Pharma overcharge the public for a drug that’s no better or safer than the unapproved form. “If URL Pharma can show their medicine is superior, that’s fine,” said Dr. Chris Morris, a rheumatologist in Kingsport , Tenn. who has many FMF patients and gouty arthritis patients. “But I don’t think they can. They’re charging an outlandish amount for a medicine that’s available for a fraction of the price.”

There were about 3.5 million colchicine prescriptions filled in 2009, according to IMS Health. There are an estimated five million gout suffers in the U.S. and fewer than 200,000 FMF patients.

Colchicine is the latest unapproved drug targeted by the FDA under a 2006 initiative aimed at prodding drugmakers to go through the agency’s lengthy and costly approval process. The agency already has removed a number of unapproved prescription products and ingredients from the market.

Recently, the American College of Rheumatology sent a letter to the FDA seeking a meeting to discuss how to keep colchicine affordable to patients. “We want to express our concern that a medicine used for centuries to treat gout and rare conditions, which costs pennies, will now cost patients quite a bit more,” said Dr. Stanley Cohen, who is the Dallas-based president of the group, in an interview. “That doesn’t make sense in the setting of health care reform.”

Part of URL Pharma’s business plan is to take advantage of the FDA’s campaign against unapproved drugs.

“Four years ago we decided to join the FDA in this effort,” said Dr. Richard Roberts, CEO of URL Pharma. “We are focusing on a few of the unapproved products where there are significant safety and medical issues, applying a lot of science and creativity to bring them into compliance and make them safer.”

In 2005, the company won FDA approval for Qualaquin, a brand-name formulation of unapproved quinine sulfate, long used for treating malaria. The following year, after it sued to force unapproved quinine sulfate products off the market, the FDA halted sales of those drugs.

In its 2006 policy guide, the FDA estimated there are several thousand drugs being marketed without the agency’s approval. Federal law required approval of new drugs for safety beginning in 1938, and for effectiveness in 1962. Many drugs currently on the market preceded those laws.

Unapproved products the FDA has forced off the market include codeine sulfate, carbinoxamine, ergotamine and trimethobenzamide hydrochloride suppositories.

The FDA is targeting unapproved drugs with potential safety risks or lack of evidence of effectiveness, as well as those being marketed fraudulently or competing with an approved drug. But the agency has also said it would consider whether halting sales would leave patients without good alternatives.

Cheap colchicine probably won’t be on the market for long. URL Pharma is suing to force the unapproved products off the market. Whatever the outcome of that lawsuit, the FDA policy guide suggests the drug agency will give other colchicine makers a year from Colcrys’ approval before moving to halt sales. FDA spokeswoman Karen Riley declined to comment on the agency’s enforcement plans.

Nancy Sparks Morrison, 70-year-old resident of Cross Lanes, W.V. who operates a Web site for fellow FMF sufferers, says she and other patients already are having trouble finding cheap colchicine. Her pharmacist no longer can get her old brand, which cost about $10 a month, so she started ordering a Canadian-made version, which costs about $100 a month, through an online pharmacy.

“With Colcrys coming out, all the other makers have stopped production, and when supplies run out there will be no more generic colchicine,” Morrison said. “Then Colcrys will be the only product and they can charge whatever they want. It’s price gouging, there’s absolutely no excuse.”

Attempts to reach colchicine producers were unsuccessful.

Roberts argues that it’s unfair to compare the price of Colcrys and unapproved colchicine. “The companies pushing out illegal products with no regard for safety issues didn’t add value for patients and doctors that we’ve now created,” he said. “We’ve revolutionized how colchicine can be used. We don’t compare ourselves to illegal products.”

He acknowledged that his company distributed an unapproved form of the drug until 2006 but decided several years ago to stop producing or distributing any non-FDA approved products.

Some experts say the higher price of Colcrys is a necessary tradeoff for greater public protection. “It costs a lot to come out with a new drug that meets the standards of safety and efficacy, and someone has to pay for that,” said Dr. Tom Hazlet, an associate professor at the University of Washington School of Pharmacy.

URL Pharma is reaching out to calm the consternation over the price of its new product. It’s offering a three-month supply of Colcrys for $15 to any U.S. patients with incomes under three times the poverty level. FMF patients with private insurance will qualify for coupons limiting their copayment to $25 per prescription. And Roberts said his company soon will announce a third assistance program for FMF patients who don’t qualify for the other two, including Medicare beneficiaries who lack drug coverage.

But Tina Martin remains upset. “They’ve made the price astronomical,” she said, “and it’s not right.”

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Cost and Quality