Medicare patients who rely on "off-label" use of drugs for pain, rare diseases and other conditions are often barred from getting them through the new prescription drug program, says a patient advocacy group in a report out today. Under rules issued last year by the Bush administration for the Medicare Part D program, so- called off-label prescriptions written by doctors for their patients can be denied by insurers.

"Off-label" means a drug is used to treat a condition for which it is not specifically approved by the FDA. "For the first time in more than 40 years, we have a Medicare statute interpreted as not covering medically necessary care," says Robert Hayes, president of the Medicare Rights Center. But Medicare officials say Medicare rules allow patients to get doctor-prescribed off-label drugs if the drugs are listed in one of three drug reference guides as useful for the condition they are suffering. "There has to be some reason for using (the drugs)," says Jeffrey Kelman, chief medical officer for Medicare's Center for Beneficiary Choices. Hayes wants other, peer-reviewed scientific evidence allowed for exceptions to the regulations, as is allowed under many state Medicaid programs for low- income patients. But Kelman says the three guides are "very, very broad" in outlining what drugs can be used off-label. He says patients should be cautious about using a drug off-label, as labeling requirements are one way the FDA tries to ensure safety and efficacy.

In its report, the center highlights troubles faced by some patients in getting drugs their doctors order. Based on calls to the center, the most commonly denied off-label drugs include: powerful painkillers Actiq and Fentora, approved for cancer-related pain but also sought by patients with other types of pain; anti-nausea drug Zofran, approved for patients undergoing chemotherapy but sought by patients with other causes of nausea; and Lotronex, a bowel syndrome drug approved for women, but sought by some men. Referencing a 2006 article in the Archives of Internal Medicine, the center says more than 20% of prescriptions written for the 500 most commonly used prescription drugs in the USA are for off-label use. The group does not have a tally of how many Medicare patients have had a doctor-prescribed treatment denied because of the rules, or of how much it would cost the Medicare program to cover all such uses of drugs.

Morris Hilton, 42, whose head was crushed when his apartment collapsed around him during a 1997 tornado in Chattanooga, Tenn., is among the patient stories published in the center's report. He suffers from traumatic brain syndrome and says he experiences debilitating headaches almost daily. For six years, he took pain medication Actiq, which the state Medicaid program covered. When Medicare began its prescription program last year, Morris, like other disabled residents eligible for both programs, was switched from his state's Medicaid drug program into Medicare. For six months, his Medicare insurer paid for the drug, then denied it. His doctor switched him to another painkiller approved for use in cancer patients, Fentora, but that was denied also. Both drugs are expensive: Hilton says his Fentora cost $60 a day. "They're saying to me, 'You're going to have pain all the time, and we're not going to pay for your medication, although if you had cancer pain, we would,' " Hilton says. "What's the difference?" On Tuesday, Hilton learned that an administrative law judge had ruled in his favor in an appeal of his insurer's decision. On Wednesday, he still did not have the medication, and his lawyer has said the ruling could be further appealed by the insurer.

August 13, 2007