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Investigators commissioned by the US Centers for Medicare & Medicaid Services (CMS) said compendia of medications used to justify off-label use for cancer treatments appear not to use systematic methods to review or update evidence. As a result, physicians using these compendia to determine treatment regimens for patients with cancer may not be prescribing the best medications for a particular case, and Medicare and other insurers may be paying for suboptimal care for such patients. Researchers say oncology drug compendia that update off-label prescribing for patients with cancer lack rigorous evidence of efficacy for certain indications. The findings, appearing in the March 3 issue of the Annals of Internal Medicine and presented by investigators with Duke University Medical Center in Durham, NC, and Tufts Medical Center in Boston (Abernethy AP et al. Ann Intern Med. 2009;150[5]:336-343), are part of a series of technology assessments commissioned by the CMS and funded by the Agency for Healthcare Research and Quality. The CMS is compiling data to help it determine whether it has sufficient evidence that the use of compendia is reasonable and necessary. The researchers are also looking at potential conflicts of interest in the production of cancer drug compendia, the effects of third-party payment on clinical trial accrual and retention that can make it more difficult to produce the evidence that would be needed for the drugs to be tested for a particular indication, and off-label indications for high-cost monoclonal antibody and small-molecule anticancer therapies whose efficacy is difficult to quantify as relevant data continue to be published. Getting a handle on the off-label use of medications for cancer treatment is important for the CMS because up to an estimated 75% of all cancer therapy now involves off-label prescriptions. And the drugs are not cheap, with some costing as much as $10 000 per month, straining the Medicare budget. A recent report found that from 1997 to 2007, Medicare spending increased 47%, from $210 billion to $309 billion, but spending for Medicare Part B drugs, the great majority of which goes to cancer drugs, rose 267%, from $3 billion to $11 billion (Bach PB. N Engl J Med 2009;360[6]:626-633). While Medicare tends to not pay for off-label drug use, a law passed in 1993 required such coverage in the oncology setting. If an off-label use of a drug or biologic is determined to have a “medically accepted indication” in cancer treatment, supported by citation in a compendium, then it must be covered by Medicare unless the Secretary of the Department of Health and Human Services determines the therapy to be medically inappropriate or if one or more compendia cite it as not indicated. But Amy P. Abernethy, MD, an assistant professor at Duke and coauthor of the Annals article, was not confident that the compendia are providing the rigorous data needed for Medicare to make informed payment decisions. “The most critical question to ask is, ‘Do the compendia do what they say they do?’” said Abernethy. “And it turns out they do not do nearly the kind of job they said they would do.” Abernethy and colleagues performed a systematic review of available studies of 14 different off-label uses of cancer drugs appearing in 6 compendia, such as American Hospital Formulary Service Drug Information and Clinical Pharmacology, in 2006. Only 1 compendium included all 14 of the off-label indications, while the others discussed from 2 to 9. (Only gemcitabine for bladder cancer and gemcitabine for ovarian cancer were covered in all compendia.) The researchers repeated their systematic review for one indication, gemcitabine for bladder cancer, to determine whether 2008 compendia used the latest literature. They identified an additional 25 reports on this indication published since 2006, but only 1 compendium increased its citations—from three to 11—while the others had little or no change. The authors also found that all but one of the compendia cited literature only through 2001 and some did not provide any citations for certain drugs and indications. “How do the compendia stand up against basic evidence-based practices?” asked Abernethy. “We found there is a lot more out there that is not being summarized in the compendia.” J. Leonard Lichtenfeld, MD, deputy chief medical officer for the American Cancer Society, said the current method of assessing off-label drug use can be improved, but he cautions that at a time when pharmaceutical manufacturers are reluctant to conduct large-scale trials that cost millions of dollars and take years to complete, compendia are the best tools available for physicians and payers. “We're left relying on experts reviewing the evidence to determine appropriate and inappropriate use,” Lichtenfeld said. “With these drugs so darn expensive, we have to have some structure in place that is reasonable in terms of advancing treatment without becoming so permissive that we don't review the evidence.”
JAMA – American Medical Association
Published: Apr 22, 2009
Keywords: antineoplastic agents,off-label use
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