A story in The Wall Street Journal last Thursday, “Medicare Eye Study Finds Untapped Savings” (subscription required), reflects the “do as I say, not as I do” nature of federal regulation of the off-label use of medical products.  Contradictions already abound in the regulatory scheme: FDA, with punishing support from prosecutors, has shut down most communication about off-label uses between health product companies and physicians.  Off-label regulators don’t distinguish between truthful, peer-reviewed information and snake-oil marketing; both are equally suspect to them.  At the same time, the Centers for Medicare and Medicaid Studies (CMS) reimburses for many off-label uses.

But is CMS now drifting beyond its financial stamp of approval for off-label uses into the realm of promotion?  As the Journal story relates, three CMS employees, together with a private retina specialist, have produced a study concluding that Medicare could save the feds millions in care for age-related macular degeneration by more aggressively reimbursing for the off-label use of a cancer drug (Avastin) to treat the eye ailment, instead of paying for a drug FDA has specifically approved for that condition (Lucentis).  The study has not been peer-reviewed, nor has it been published.  It’s eye specialist-author accuses CMS of “suppressing” the study, and Senator Herb Kohl is quoted as finding it “baffling” that CMS is delaying its release.

What is truly baffling is CMS’s response to the reporter’s inquiries on the study.  Its chief medical officer asserts that he “has no problem with the data” but is waiting for information from the authors on “how the data can be translated into policy.”  He then went on to note that CMS is “prohibited from doing cost-effectiveness analysis.”  If federal law prevents CMS from “translating” the data “into policy,” then how can CMS lawfully place its official imprimatur on its employees’ work?  Was CMS aware of these employees’ involvement in the study?  Were they conducting research during government work hours?  Elected officials responsible for CMS oversight, and perhaps the HHS inspector general, should ask these questions, especially given CMS’s “too cute by half” response to the leaked study.

The even larger question here is whether this leaked study and CMS’s non-denial denials constitute “promotion” of an off-label use — motivated by cost savings rather than patient care.  The company which produces both Avastin and Lucentis has expressed concern about the off-label use of Avastin.  So has FDA.  FDA related through a letter to retinal specialists (reprinted in part by the Patent Docs blog) that

Avastin is a sterile product and  . . . its off-label use for ophthalmic diseases sometimes involves repackaging Avastin into multiple smaller doses for administration.  The agency is concerned about the manipulation of sterile products because of the increased risk of product contamination.

The Patent Docs story also notes that FDA had inspected the Avastin production plant and issued a “verbal warning about promotion of off-label use of the drug.”

WLF certainly doesn’t take issue with the government’s reimbursement for off-label uses, though doing so when there is a drug actually approved for the medical condition being targeted does give us pause.  CMS policy has taken this approach with other medical conditions such as fibromyalgia, for which there is an approved drug, but the agency won’t reimburse for that drug until practitioners try other, older drugs in a off-label fashion (see this WLF Legal Opinion Letter for more on this controversy).

What troubles us, though, is that if the producer of Avastin and Lucentis asserted its First Amendment rights and shared peer-reviewed, third-party authored and published studies about Avastin with doctors, it would be subject to punitive civil and criminal sanctions.  Venomous headlines about how it peddled unapproved uses of drugs to doctors for treating the elderly and their eye conditions would likely follow.

But the media and some in Congress applaud when CMS employees apparently promote such an off-label use through an unpublished, unreviewed study which was curiously leaked to The Wall Street Journal.  In this age of health care cost reduction at just about any cost, double standards for the regulators will increasingly become the norm.