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Off-Label Use of Prescription Drugs
February 19, 2009 • Volume 9, Issue 7
For cancer, but also for other diseases, doctors often prescribe medicines that were approved by the FDA as treatments for other conditions. A drug approved by the FDA for colon cancer is used to treat breast cancer, for example.
Sometimes, the prescribing of medicines for these off-label indications is based on strong evidence of efficacy from clinical studies published in respected journals that are peer-reviewed—vetted by leading doctors and researchers in the field. In other cases, the off-label use is based on weak evidence—a poorly designed study or case reports of how the medicine worked in one or two patients—or is the result of illegal marketing campaigns by the drug manufacturer.In the first scenario, the patient can receive a treatment that will save her life or help her live a full life despite a chronic and debilitating disease. In the second scenario, the treatment may put the patient’s life at risk, either from the side effects of the medicine or because the treatment is ineffective, or both.
Policymakers have tried to design Medicare coverage policy so that Medicare will pay for off-label treatments when there is good evidence of safety and efficacy, and not pay for treatments that are unsafe and unsupported by clinical evidence. To distinguish the two, Medicare had relied primarily on privately published reference manuals known as compendia.
Newly published research shows that the compendia are not doing a good job keeping up with the most current research and incorporating it into their recommendations. There is also widespread concern that compendia editorial decisions—which off-label uses they support and which they do not—are unduly influenced by drug manufacturers, and that publishers of the compendia do not have adequate policies to guard against conflicts of interests in editorial decisions.
Because of these twin failings, much attention has focused on the possibility that Medicare is paying for expensive off-label treatments that are ineffective and potentially unsafe. That is a legitimate concern.
But these deficiencies in the compendia can also mean that Medicare is denying coverage for off-label uses that have strong support in the peer-reviewed medical journals. If the compendia are not keeping up with the research, then they may miss published studies’ support an off-label use of a particular medicine.
It may seem counterintuitive, but undue influence by drug manufacturers may also lead the compendia to omit off-label uses that have been shown to be effective in clinical studies. If drug manufacturers can influence the decision to review a drug for a particular off-label indication, then there is much less chance that a use for a rare disease that has little market potential will be reviewed. Similarly, brand-name drug manufacturers have much less incentive to push for compendia support for drugs with generic competition than for drugs that still have patent protection.
What this means is that it is in the interest of consumers for the Centers for Medicare & Medicaid Services to hold the compendia to high standards of performance and to require rigorous and effective conflict-of-interest policies, including over which off-label indications are brought up for review.
It also means that there needs to be a safety valve allowing case-by-case coverage decisions when the compendia may have missed the mark. Such a safety valve exists for cancer drugs covered under either Part B (delivered in the oncologist’s office) or Part D (generally, purchased at the pharmacy). Where there is no support in the compendia for an off-label use, Part B contractors can review the peer-reviewed journals for studies that demonstrate effectiveness.
The safety valve also exists for other drugs covered under Part B. Contractors are instructed by CMS to look at both the compendia and the medical literature on a case-by-case basis.
But under Part D, for drugs that do not treat cancer, there is no safety valve. If there is no compendia support for an off-label use, the drug cannot be covered. The freedom that state Medicaid departments and private insurers have to look past the compendia at the research and make a case-specific coverage decision does not exist for Part D plans.
It is high time that policy makers in Congress and the Obama administration recognize the shortcomings of the compendia as arbiters of coverage, and allow coverage decisions to be made on an individual basis on the strength of sound clinical research published in peer-reviewed medical journals.
“The compendia's stated methods varied greatly from their actual practices. Compendia cited little of the available evidence, often neither the most recent nor that of highest methodological quality. Compendia differed in evidence cited, terminology, detail, presentation, and referencing. For the 14 off-label indications studied, the compendia differed in the indications included and whether and how they recommended particular agents for particular types of cancer.” (Reliability of Compendia Methods for Off-Label Oncology Indications, Annals of Internal Medicine, March 2009)
“FDA-approved drugs used for indications other than what is indicated on the official label may be covered under Medicare [Part B] if the carrier determines the use to be medically accepted, taking into consideration the major drug compendia, authoritative medical literature and/or accepted standards of medical practice.” (Medicare Benefit Policy Manual, Centers for Medicare & Medicaid Services, December 2008)
“If CMS fails to adjust the current regulations appropriately, Congress should revise the language of the Part D statute to more clearly allow for the consideration of other evidence of medical necessity in Medicare Part D coverage determinations and appeals, including peer-reviewed medical literature and the individual’s medical history.” (Off-Base: The Exclusion of Off-Label Prescriptions from Medicare Part D Coverage, Medicare Rights Center, August 2007)
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