Targeting drug shortages

Patients and government agencies are using a growing array of tools to mitigate the scope and effects of drug shortages, which continue to rise.

The Food and Drug Administration released draft guidance last week that builds on an executive order President Barack Obama issued in October to address the growing problem.

Companies that are the sole source of certain drugs should be required to notify the government of any manufacturing disruptions, according to the guidance. The FDA said it “originally interpreted this requirement to apply only to permanent manufacturing discontinuances” rather than other temporary manufacturing suspensions.

The FDA also announced moves to ease the shortages of two cancer drugs, approving methotrexate from a new manufacturer and the importation of LipoDox, an unapproved drug in the U.S., to replace Doxil. The agency is showing an “increased, new flexibility,” said Albert Wertheimer, a professor of pharmacy at Temple University in Philadelphia.

Those moves alone won't allay concerns over the shortages, which have worsened over the last six months, said Michael Cohen, president of the Institute for Safe Medication Practices.

There were 267 reported drug shortages in 2011, up 26.5% compared with 211 in 2010 and up 281.4% compared with 70 in 2006, according to the University of Utah Drug Information Service.

“This is not a resolved situation,” Cohen said. “We've got a long way to go.”

The shortages have turned a spotlight on the relatively unknown market of secondary drug distributors, which often sell drugs at prices that are higher than what a hospital with a discount from its group purchasing organization may pay. Allegations of price-gouging prompted Rep. Elijah Cummings (D-Md.) to launch an investigation into several distributors.

Last week, the U.S. Justice Department announced the resolution of a case suggesting the government is using criminal enforcement to stem the flow of drugs into the secondary market. A Florida physician pleaded guilty to one count of conspiracy to commit mail and wire fraud as part of a scheme to buy and resell a prostate cancer drug to at least two drug wholesalers in Ohio and New Jersey.

Dr. Michael Schoenwald of Hollywood, Fla., sold Lupron to another individual, who created false drug pedigrees and sold the drugs to wholesalers from 2007 to 2009, according to documents filed in Cincinnati.

Assistant U.S. Attorney Anne Porter said it was the first case in which a physician pleaded guilty to a drug diversion scheme with the U.S. attorney's office in Cincinnati. She declined to say whether the office is pursuing similar investigations.

Also this month, 25 patients filed a lawsuit in Washington against HHS, the FDA, and the National Institutes of Health, alleging that the way the FDA handled shortages of Fabrazyme, a drug used to treat a rare genetic disease and which costs $200,000 for a year's supply, and aquasol A, a drug used to treat vitamin A deficiency, violated the patients' constitutional rights.

The plaintiffs allege that Fabrazyme patients, who purchase the drug directly from Genzyme Corp., the drug's only manufacturer in the U.S., received diluted doses after the shortage began in 2009. They allege the company has provided full doses to patients in Europe, where there is a competing drug. The patients say that the courts should supervise the allocation of a drug in short supply and that FDA licenses issued to drug companies that cause shortages should be invalidated and the patents declared unenforceable.

“The FDA stepped back when there was a shortage and let the corporations make all the medical decisions for the patient,” said Allen Black, the patients' lawyer. Fourteen plaintiffs filed a related suit in Boston last year against Genzyme and the Mount Sinai School of Medicine, New York, which owns the patent for Fabrazyme. Black said a second suit will be filed by about 50 plaintiffs in New York against both organizations this year.

Although the Fabrazyme case differs from that of many of the other drugs in short supply—most are generic injectibles purchased by hospitals and GPOs, not directly by patients—Black said that he expects to see other lawsuits related to shortages. “The common denominator for all of the shortages is FDA licenses,” he said.