FDA Considering New Off-Label Guidance
Under fire from a senior House Democrat, the FDA is reassessing whether it should issue guidance it has drafted re-establishing a "safe harbor" for off-label drug promotion. Off-label promotion of psychopharmacological drugs has been a big issue, both on Capitol Hill and in the courts, most recently reflected in letters sent to federal officials and drug manufacturers by Sen Charles Grassley (R, Iowa). He voiced concern about off-label promotion of atypical antipsychotics for dementia and Alzheimer disease in nursing home patients.
In March 2006, the US Attorney's Office in Brooklyn, NY, indicted a Maryland psychiatrist for giving lectures around the country on behalf of Jazz Pharmaceuticals. The Justice Department indicted Peter Gleason, MD, for urging primary care physicians to use Xyrem (sodium oxybate) off-label for depression. It is currently approved for use with narcolepsy. That case has not come to trial, according to a spokesperson for the US Attorney for the Eastern District of New York.
Off-label drug use is of course permissible; the issue is whether pharmaceutical companies can market drugs to physicians and other medical personnel for off-label uses. Until 1997, that was generally illegal. But Congress passed a law in 1997 allowing off-label marketing if certain conditions were met, including submitting the advertisement to the FDA in advance. That "safe harbor" expired on September 30, 2006.
The FDA prepared a draft guidance document to reinstitute a safe harbor this fall and was set to publish it in the Federal Register, which would have initiated a public comment period, but Rep Henry Waxman (D, Calif), chairman of the House Committee on Oversight and Government Reform, got hold of the draft and wrote to FDA Commissioner Andrew C. von Eschenbach, MD, asking him to reconsider it.
"I urge you to refrain from going forward with this ill-advised guidance," Waxman wrote. "The draft guidance would carve a large loophole in the law and create a pathway by which drug and device manufacturers can promote unapproved (off-label) uses of their products without first obtaining FDA approval by passing out journal articles about the off-label use to physicians."
An FDA spokeswoman declined to say whether the agency was planning to publish the draft guidance as is, was planning to revise it, or was dropping the effort to reinstitute a safe harbor. Ken Johnson, senior vice president of the Pharmaceutical Research and Manufacturers of America, said, "We look forward to reviewing a draft guidance if one is published in the Federal Register. Providing health care professionals with peer-reviewed medical journal articles can provide valuable information to help them make informed pharmaceutical prescribing decisions. This topic is a significant policy issue, and it is one that impacts how patients are treated for diseases."
Some patient advocacy groups want a safe harbor reinstituted. "There are nearly 30 million people in the United States affected by almost 7000 known rare diseases," said Diane Dorman, vice president for public policy at the National Organization for Rare Disorders. "Consequently, most of those disease states are treated off-label because there is no therapy specific for the disease. So getting that information to physicians I would consider to be very, very important for the patient."
The draft guidance the FDA prepared does not exactly mirror the safe harbor established in 1997. For example, it does not require companies to submit off-label promotions to the FDA in advance. But no one could read the draft guidance and say the agency would be giving pharmaceutical companies a wide berth. For example, an article from a peer-reviewed medical journal discussing off-label use of a drug cannot be used if it is "inconsistent with the weight of credible evidence," meaning it cannot run counter to what was found in other, similar clinical investigations. If the article only contradicted the results of one other article, that dissenting article would have to be distributed at the same time.
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SAMHSA Bill Ready for Consideration
Congress may make some changes in the community mental health grant programs funded by the Substance Abuse and Mental Health Services Administration (SAMHSA). That effort began at the end of January when the Senate Committee on Health, Education, Labor, & Pensions was scheduled to vote on a SAMHSA reauthorization bill. SAMHSA includes the Center for Mental Health Services, whose main program is the block grant program, which will distribute $393 million to states in fiscal 2008 (which began October 1, 2007); the states have considerable freedom over how to use those funds.
Senators Edward Kennedy (D, Mass) and Michael Enzi (R, Wyo) have written a draft bill that has substantial support within the committee, according to Joe Faha, director of legislation at SAMHSA. Among the significant changes proposed in the reauthorization bill is to require states to gear local mental health programs to meet national outcome measures and to meet targets based on those measures.
Currently, states voluntarily report system-level data to SAMHSA in a number of national outcome measure domains, such as Access/Capacity, Retention, and Perception of Care. If the reauthorization bill were to pass, the states would have to report client-level data and meet numerical targets in each domain. If the states missed those targets over a 3-year period, SAMHSA would institute a corrective action program and provide technical assistance to the state to help overcome the problems.
A Senate staffer involved in drafting the bill said it will also include a new grant program aimed at funding primary care within community mental health centers and funding mental health services within primary care centers.
As was the case in 2000 when SAMHSA was last reauthorized, "charitable choice" will again be the most controversial issue. That refers to the ability of religious organizations—ie, faith-based providers—to receive and distribute federal funds. In 2000, a charitable choice provision was added to the Substance Abuse Prevention and Treatment Block Grant and the Projects for Assistance in Transition from Homelessness program. Senate sources say Democrats on the committee will attempt to eliminate the charitable choice provisions inserted in 2000.