Is FDA approval for safety ... or price gouging?

Kaiser Health News

Doris Webb was diagnosed last year with a rare disease called familial Mediterranean fever. It’s a hereditary condition tied to her French ancestry, causing recurrent fevers, arthritis attacks and inflammation of the lining of the lungs and abdomen. The drug colchicine, prescribed by her doctor, was able to relieve her debilitating joint and bone pain, and Webb now takes two to three tablets a day.

She pays only $12 at Wal-Mart for a 90-day supply, according to her daughter Tina Martin, who also takes colchicine for the condition. Colchicine has been prescribed for centuries, more commonly for gout. The form the two women use is cheap because, like thousands of prescription drugs on the U.S. market, it predates modern drug laws and has never been evaluated or approved by the U.S. Food and Drug Administration.

Now, Webb may have trouble getting the drug. In July, Philadelphia-based URL Pharma won FDA approval for a branded version called Colcrys, which sells for about $4.50 a tablet. This will dramatically boost costs for patients, like Webb, without drug coverage. The 66-year-old Morristown, Tenn., resident is on Medicare but can’t afford the Part D drug benefit, her daughter says. Even insured patients like Martin will feel the pinch; her co-payment 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 and easier for patients to tolerate than the unapproved forms of colchicine. The agency said it had received reports of 120 patient deaths from unapproved colchicine’s interactions 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 familial Mediterranean fever.

But some rheumatologists and patient groups charge that the FDA is letting URL Pharma gouge the public on a drug that’s essentially the same as one doctors have found safe and effective for millions of Americans over decades. They say Colcrys is no better or safer than the much cheaper unapproved forms of colchicine. There were about 3.5 million colchicine prescriptions filled in 2009, according to IMS Health, a data research firm.

Colchicine is the latest drug targeted by the FDA under a 2006 initiative aimed at prodding drug makers to take unapproved but medically valuable drugs through the agency’s lengthy and costly approval process. The agency already has removed many prescription products and ingredients from the market. But some observers ask whether replacing a cheap, unapproved product with a similar, branded one benefits the public in all cases.

“If [URL Pharma] can show their medicine is superior, that’s fine,” said Dr. Chris Morris, a rheumatologist in Kingsport, Tenn., who has many patients treated with colchicine. “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.”

In December, the American College of Rheumatology sent a letter to the FDA seeking to discuss how to keep colchicine affordable. “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, a Dallas rheumatologist who is president of the college, in an interview. “That doesn’t make sense in the setting of healthcare reform.”

URL Pharma plan

Part of URL Pharma’s business plan is to take advantage of the FDA’s campaign. “Four years ago we decided to join the FDA in this effort,” said Dr. Richard Roberts, chief executive 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 up to several thousand drugs -- including nearly 2% of all prescription drugs -- marketed illegally without required FDA approval. These include compounds with unapproved active ingredients such as antihistamines, narcotics and sedatives. Federal law first required approval of new drugs for safety in 1938 and didn’t require approval for effectiveness until 1962. Many drugs currently on the market preceded those laws.

Unapproved products the FDA has forced off the market since the policy was announced include injectable colchicine, topical papain, timed-release guaifenesin and trimethobenzamide hydrochloride suppositories, plus some versions of codeine sulfate, carbinoxamine, ergotamine and hydrocodone.

Given its limited resources, the FDA said it would target unapproved drugs with potential safety risks or lack of evidence of effectiveness, those marketed fraudulently and those directly competing with an approved drug. But the agency also said it would consider whether halting sales would leave patients with no good therapeutic alternatives or otherwise burden patients.

With the FDA approval of Colcrys, however, cheap colchicine probably won’t be on the market for long. That will affect lots of Americans, given that there are an estimated 5 million gout sufferers in the U.S.; fewer than 200,000 have familial Mediterranean fever.

In August, URL Pharma filed suit in federal court in Sacramento to force unapproved colchicine products off the market; the court denied a motion for a preliminary injunction to halt their sales and transferred the case to federal court in New Jersey.

Whatever that court rules, the FDA policy guide suggests the drug agency will give other colchicine makers one year from its approval of Colcrys before moving to halt sales. FDA spokeswoman Karen Riley declined to comment on the agency’s enforcement plans.

Cheap drug scarce

Nancy Sparks Morrison, a 70-year-old retiree in Cross Lanes, W.Va., who operates a website for fellow familial Mediterranean fever sufferers, says she and other patients already are having trouble finding cheap colchicine. Her pharmacist no longer could get her old brand, made by Corona, Calif.-based Watson Pharmaceuticals. So she started ordering a Canadian-made version called Odan, which costs about $100 a month through an online pharmacy. She previously was paying a $10 co-payment for a month’s supply of colchicine under her Medicare supplemental drug coverage.

“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 Watson and other colchicine producers, including Estero, Fla.-based Vision Pharma and Fairfield, N.J.-based Excellium Pharmaceutical, and their attorneys, 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. We’ve revolutionized how colchicine can be used.”

His company distributed an unapproved form of the drug until 2006 but decided several years ago to stop producing or distributing non-FDA approved products, Roberts said. “We’re very hopeful the FDA will take action against unapproved [colchicine] products soon.”

Some pharmaceutical experts say the higher price of Colcrys is a necessary trade-off for greater public protection. “It costs a lot to come out with a new drug that meets the standards of safety and efficacy,” said Dr. Tom Hazlet, an associate professor at the University of Washington School of Pharmacy. “The manufacturer goes to the trouble to seek FDA approval, and that allows them to charge what they want to charge.”

Still, 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 U.S. patients with incomes of up to three times the poverty level. Familial Mediterranean fever patients with private insurance will qualify for coupons limiting their co-payment to $25 per 30-day prescription. And Roberts said his company soon will announce a program for familial Mediterranean fever patients who don’t qualify for the other two, including Medicare beneficiaries who lack drug coverage.

But Tina Martin isn’t convinced URL Pharma’s aid programs will help her mother, and she doesn’t want their help anyway. “My mother can’t afford Colcrys at all. They’ve made the price astronomical, and it’s not right.”

Kaiser Health News is an editorially independent news service and is a program of the Kaiser Family Foundation, a nonpartisan healthcare policy research organization. Neither Kaiser Health News nor the foundation is affiliated with Kaiser Permanente.