Federal administrators who run the Medicare program have begun to closely scrutinize the off-label use of prescription drugs, using clinical reviews to determine which prescriptions will get into the hands of American consumers and which won't.
The move is part of a pattern of growing influence for the Medicare agency, called the Centers for Medicare and Medicaid Services, or CMS. By conducting more clinical reviews of its own to decide whether to pay for drugs, it is adopting activities that previously had been the exclusive domain of the Food and Drug Administration.
Off-label uses of drugs, meaning they are used in treatments not approved by the FDA, are common in US healthcare. Medicare has traditionally covered these prescriptions based on a doctor's judgment. But the agency is beginning to demand evidence of health benefits in response to expensive cutting-edge treatments, more off-label prescribing, and marketing abuses by drug companies.
Although the aim is to reduce expenses for off-label uses that are least effective, the changes have not necessarily gone against drug companies. In a rare move, Medicare, after reviewing clinical data, recently approved coverage nationwide for an off-label use of a Novartis International AG drug called Visudyne, which can prevent blindness from macular degeneration in the elderly.
Meanwhile, the agency is considering revoking coverage for off-label uses for some cancer drugs, including Biogen Idec's Zevalin, an expensive treatment for non-Hodgkins lymphoma that is used off-label to treat other forms of cancer.
"This is the direction the agency is headed, and companies need to take account of this as they plan their clinical trials and research," said Nick Littlefield, a lawyer at Foley Hoag in Boston who represented Novartis in its effort to win coverage for Visudyne. "All that FDA does is say you can market a product, but CMS provides the coverage -- CMS decides whether or not the product is going to be paid for."
CMS could wield even more clout -- and depend to an even greater extent on scientific evidence -- under the Medicare prescription benefit that takes effect in 2006. The agency likely will weigh health claims to judge which drugs are being fairly or unfairly excluded from coverage lists maintained by the government's Medicare prescription contractors. The result, said Thomas Scully, a former CMS administrator who now works as a consultant to drug companies, will be greater public focus and lobbying attention from industry.
"The drug companies I know couldn't tell you where CMS headquarters is in Baltimore," said Scully. By 2006, he said, the map to the front door will be etched on their brains.
Novartis lost a battle in 2001 when CMS decided not to cover off-label uses of Visudyne, following the lead of the FDA, which had decided trial data submitted by Novartis did not support approval for use on some types of macular degeneration. Macular degeneration breaks down the part of the retina responsible for direct, sharp vision needed for activities like driving or reading. Last year, Novartis, in an effort parallel to its continuing attempts to win expanded approval from FDA, presented new clinical data to CMS and enlisted doctor and patient groups to help make its case. It worked.
"The rules have changed," said Dr. George A. Williams, chairman of the department of ophthalmology at the William Beaumont Hospital in Royal Oak, Mich., who worked with Novartis on the application.
Williams said the move could benefit between 40,000 and 100,000 elderly patients a year. The drug costs about $1,300 per treatment and requires multiple treatments.
On the flip side of the equation, the agency is evaluating the health benefits of off-label uses of several cancer drugs to determine if coverage should be revoked. The review was reported by the New York Times last month.
Dr. Sean Tunis, chief health officer at CMS, said the review of cancer drugs grew partly out of a feeling that doctors were prescribing expensive medications based on "non-clinical concerns," including promotions by drug companies.
Tunis said he supports another form of review that could be used in the future: "head-to-head" trials within a class of similar drugs to see which brand or generic is the most cost-effective. Medicare reimbursement rates could then be pegged to the least costly of the effective drugs, he said. "One of the key gaps now is head-to-head trials, which for the most part are not required for FDA approval but which I think are increasingly important for reasons of reimbursement," he said. The approach would be similar to limitations private health insurers already place on their drug lists.
Christopher Rowland can be reached at firstname.lastname@example.org.