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FDA STANDARDS FOR CANCER APPROVALS ARE NOT PRIME CAUSE OF OFF- LABEL USE, MAYO ONCOLOGIST ARGUES; COMPENDIA APPROACH HAS "SHORTCOMINGS," GAO SAYS

Executive Summary

Attempts to blame restrictive FDA approval standards for the prevalence of off-label uses in the oncology field are debunked by Mayo Clinic oncologist Charles Moertel, MD, in an editorial in the Dec. 4 issue of the Journal of the American Medical Association. A "common argument" for the lag between clinical practice and FDA approval, Moertel notes, "is that current standards for approval, which require demonstration by controlled studies of improved duration or quality of life, are far too stringent and that a reported high objective tumor regression rate should provide a surrogate endpoint." The overly-restrictive argument "is weakened," Moertel contends, "by the remarkable variation of reported regression rates," he said. For example, "interleukin 2-lymphokine-activated killer cell regression rates [were] originally reported at 90% for renal cancer and 45% for melanoma but more recently plummet[ed] to a trivial 13% and 12%, respectively." The dichotomy between regression rates in different trials has been a problem for 30 years, the Mayo oncologist notes. Moertel has an insider's understanding of FDA's cancer drug approval decisions from a long affiliation with the agency's Oncologic Drugs Advisory Committee: initially as a committee member and more recently as a consultant to the committee. The Mayo oncologist notes that the extent of off-label use has been exacerbated by the lack of incentives for companies to pursue additional indications for off-patent products. Pharmaceutical companies also drew sharp comments from Moertel for their roles in off-label promotion. "Commercial interests," he declares, "have been actively promoted through 'educational' vehicles, such as free books and videotapes and, until recently, conferences where oncologists have been rewarded for simple attendance by substantial cash payments." The expense of educational symposia and materials is then "passed on to third- party carriers through inflated drug costs and further compounds the problem of reimbursement for off-label therapy," Moertel contended. The point-of-departure for Moertel's editorial comments is the publication in the Dec. 4 issue of JAMA of an article summarizing the findings of the General Accounting Office's study on the prevalence of off-label cancer drug use and the effect of reimbursement policies on physicians' treatment of cancer patients ("The Pink Sheet" Sept. 30, T&G-5). A survey of responses from 681 oncologists found that one- third of all drug administrations were given off-label and that reimbursement policies cause physicians to alter preferred treatment and sites of care. The article was authored by Thomas Laetz, PhD, and George Silberman from GAO. Noting the current political pressure on third-party payers to pick up off-label uses, Moertel urges restraint in attempts to work out a quick solution to the reimbursement debate. "One would hope," Moertel declared, "that this problem would not be addressed through knee-jerk political reaction to shrill voices." Broad reimbursement coverage of off-label drug use has been included in several major pieces of legislation including the Medicare Catastrophic Care Act. Moertel indicates that physicians, not Congress or government agencies, must take the initiative by removing "the chip from their shoulders" in order to negotiate reasonably with third-party payors for appropriate cancer therapy use. Moertel noted that "efforts by organized oncology groups to curb [gross abuses of off-label uses] are conspicuous by their absence." He dramatizes the current reimbursement situation by noting that "I have recently reviewed a claim for nearly $ 750,000 made for a series of unproven, off-label therapies given to a single patient dying of advanced colon cancer." The resolution of the off-label reimbursement issue, Moertel suggests, "must come through thoughtful and nonpartisan deliberations involving physicians and third-party carriers working together to establish policies that will offer our patient the most ideal standard care." The policies must "allow continued support of the patient care element of clinical research (provided the costs do not substantially exceed those of standard care), and . . . minimize the financial burden on an already fiscally overstressed national health care provision system." A reimbursement policy that relies solely on drug compendia for support of off-label use may have "several shortcomings," Laetz and Siberman cautioned. It might "fail to address those legitimate off-label drug uses that are not yet cited in the compendia because of publication lag." In addition, such a policy, if not monitored, "will miss how these compendia change over time and how they might affect the FDA's drug approval process."

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