Suit opposes Medicare denials of off-label, non-compendia drugs

Washington -- Cancer patient Judith M. Layzer has come to the conclusion that the administrators of the Medicare drug benefit are forcing her to choose between her money and her life.

Layzer, a 66-year-old retired widow from New York, has a granulosa cell tumor, a rare form of ovarian cancer that no longer responds to the more traditional treatments for the condition. So for the past eight years, her doctor has had her on daily doses of Cetrotide (cetrorelix acetate), a drug that is normally used to prevent premature ovulation in women undergoing fertility treatments. She and her doctor are convinced that this off-label use of the drug has kept her alive.

The trouble started in 2006, when Layzer enrolled in Medicare Part D after leaving her former employer's health plan. Her Medicare drug plan denied coverage of the costly medication on the basis that it was not an approved therapy for ovarian cancer. Even though she, her doctor and several other physicians eventually were able to convince an administrative law judge and the Medicare Appeals Council that her course of treatment was her only medical option, the officials hearing the appeal were forced to rule that federal regulations did not allow the Medicare drug plan to pay for the medication. Layzer cannot afford it on her own.

"Even the [administrative law judge] and the MAC said that this was medically necessary, but Medicare wants me to go on the private market for this drug because it costs over $300,000," Layzer said. "What else can you think about this except that it seems to be coming down to money? It could cost me my life."

Having exhausted her administrative appeals options, Layzer enlisted the assistance of the Medicare Rights Center and filed suit in November 2007 against Health and Human Services Secretary Michael Leavitt in the U.S. District Court for the Southern District of New York. She seeks an injunction ordering Medicare to cover the denied drug and to reimburse her the more than $100,000 she has paid out of pocket to maintain her treatment. She has been able to receive help with her drug bills from other sources in the meantime, but that coverage is set to run out soon.

A complicated situation

Layzer's case is trickier than the average Medicare Part D appeal involving off-label medication use.

In most cases, when a Medicare drug plan denies coverage of a drug for a particular off-label use, the patient and the prescribing physician can pursue up to five levels of appeal starting with the plan and ending in U.S. District Court. If the appellants can prove at any point along the way that the drug in question is medically necessary and has no covered alternative, the officials hearing the appeal can reverse the original coverage determination and order the plan to pay for the medication.

But in this case, the prescribed use of Cetrotide is not only absent from Food and Drug Administration labeling but also from the three drug compendia that serve as medical reference guides for the use of Medicare Part D medications. An off-label treatment can make it into one or more of the compendia only if it is supported by numerous clinical studies or other pieces of medical literature.

Under Centers for Medicare & Medicaid Services regulations, if no approved compendium lists the use, the Medicare drug benefit will not cover it even if the patient appeals the original denial on the basis of medical necessity.

Gary D. Smith, the administrative law judge who heard Layzer's appeal, said his hands were tied by the federal regulations, which CMS drafted in advance of the drug benefit's launch in 2006.

"In light of the overwhelming expert medical testimony and the unique circumstances presented in this appeal, it is frustrating to have to deny this appeal merely because the use of Cetrotide to treat ovarian cancer is not listed in one of the three statutorily recognized compendia," he wrote. "The evidence clearly establishes that the use of Cetrotide to treat the appellant's ovarian cancer is reasonable and necessary."

Smith surmised that the reason why this off-label treatment is not in any of the compendia is that Layzer's form of cancer is too rare to generate sufficient clinical trials or other supporting medical literature.

The Medicare Rights Center complains that the compendia are incomplete, are updated too slowly and are inconsistent with each other. Coverage of injectable drugs under Medicaid and Medicare Part B refer to different sets of compendia than Medicare Part D. This means an off-label use approved for one program may not be approved for another.

More cases out there?

A CMS spokesman said the agency would not comment on the pending litigation in the Layzer case nor on any of the issues that it raises. But in response to an earlier Medicare Rights Center report criticizing frequent Medicare denials of off-label medications, Jeffrey Kelman, MD, chief medical officer at the CMS Center for Beneficiary Choices, said patient-safety concerns dictated that medication coverage follow medically acceptable standards. Off-label, non-compendia treatments, for the most part, should not be used by Medicare beneficiaries, he said.

But even for the relatively rare instances in which non-compendia is the only way to go for a patient, the CMS regulations leave absolutely no room for exception, said Robert Hayes, the Medicare Rights Center president. The organization insists that the agency is misreading Medicare law and ignoring the will of the lawmakers who drafted it. The center is looking to the courts and Congress to force a change in the administration's policy. Such a change would not cause Medicare Part D's spending to balloon, he said.

"This is a manifest injustice. We're talking about a few dollars, a comparably few people and great need," he said.

Layzer said that although her case is likely an extreme one, many more Medicare drug plan enrollees could be out there who are facing similar predicaments. She already has picked up one co-plaintiff in her case, and she said more people in the same situation may have come into the pipeline since she first encountered a problem at the start of Medicare Part D.

Physicians whose patients have rare conditions and require off-label treatments need to be more outspoken about Medicare's flawed drug coverage policy because it affects them, too, Layzer said.

"There is an effort to undermine the doctors here, and it's making them very uneasy about prescribing medicines," she said. "It comes back to the doctor's judgment versus what's in the cookbook."