From the Executive Director: When Will They Ever Learn? Reflections on the FDA and Cancer Drugs

by Barbara A. Brenner

Back in March 2007, two developments at the FDA regarding cancer drugs occurred close on the heels of each other: the issuance of a “black box” warning on drugs approved for treatment of cancer-related anemia and the approval of lapatinib (Tykerb) to treat metastatic breast cancer (see news clipping in this issue). In conjunction, these developments seem to indicate that folks at the FDA aren’t seeing the forest for the trees.

The anemia-fighting drugs—known as erythropoietins and marketed as Procrit, Epogen, and Aranesp—are often given to cancer patients. The black box warning resulted from recent studies showing that some cancer patients receiving the drugs died more quickly than similarly situated patients who weren’t receiving them. In one study, women with metastatic breast cancer who took an erythropoietin died more quickly than those who did not.1 One of the FDA’s concerns is that doctors are prescribing the drugs at doses greater than what the agency has approved. The new warning on the drugs addresses this problem, as well as the practice—never approved by the FDA—of giving the drugs to people with cancer who are not being actively treated for their disease, either because their disease is no longer treatable or because they have chosen to stop treatment. These people can become anemic as a result of cancer progression.

Erythropoietins generated $10 billion in sales in the United States in 2006. While Procrit is marketed by Johnson & Johnson, all of the erythropoietin drugs sold in the United States are made by Amgen, which notified investors of problems with a trial of Aranesp in head and neck cancer patients on January 25, 2007, almost two months after the company halted the trial.2 In a news conference held by the FDA on March 9, 2007, about the problems with how erythropoietins are being used in cancer patients, one FDA official said that she thinks “there is considerable off-label use of this drug.” Off-label use, which involves the use of a drug for a purpose outside the scope of the drug’s approved label, is not regulated by the FDA.

Procrit was first approved by the FDA in 1993. In 1997, BCA published a newsletter article entitled “Most Cancer-Related Fatigue Can Be Treated,” reprinting an article from the Boston Globeabout the Cancer Fatigue Coalition, which turned out to be a group funded by Ortho Biotech, the Johnson & Johnson company that markets Procrit.3 (More on Ortho Biotech’s marketing of Procrit…)

If you watch television, you’ve probably seen the ads for these drugs. One ad highlights the benefits of the drug for fighting chemotherapy-induced anemia. It tells you that “life doesn’t stop during chemotherapy” and shows people engaged in activities meant to suggest that the drug will help reverse the fatigue that commonly accompanies chemotherapy. Here’s the kicker: the FDA has never had any data showing that Procrit or the other erythropoietins improve energy levels or decrease the fatigue experienced by chemotherapy patients.4 Despite the lack of evidence of benefit, drugs like Procrit are prescribed to many breast cancer patients to address chemotherapy-induced fatigue. In May, the FDA’s Oncologic Drug Advisory Committee recommended further restrictions on the marketing of these drugs and noted the need for studies of the benefits of these drugs as yet unproven in chemotherapy patients.5

The erythropoietin story highlights several problems in the FDA’s oversight of cancer drugs: the agency’s lack of authority to regulate off-label uses of drugs it has approved, the failure to monitor how drugs are used once they reach the market, and the agency’s inability to effectively monitor televised drug advertisements. Ironically, none of the lessons from the erythropoietin debacle seems to have been taken to heart by the FDA, as revealed by the agency’s approval of Tykerb four days after the erythropoietin black box warning was issued in March.

Tykerb is a new drug approved for use in patients with metastatic breast cancer whose tumors overexpress HER2/neu and who have been unsuccessfully treated with an anthracycline (such as Adriamycin), a taxane (such as Taxol), and trastuzumab (Herceptin). For patients in these dire circumstances, BCA has long taken the position with the FDA that a new drug should either extend the patient’s life, improve her quality of life, or both. Otherwise, given the number of breast cancer drugs on the market, the drug should not be approved. Since the trial that was the basis of GlaxoSmithKline’s (GSK) application for approval did not measure quality of life and did not continue long enough to determine whether patients on the drug lived longer, BCA opposed approval at this time. The short duration of the single trial that was the basis for the FDA’s approval of Tykerb also means that many of the drug’s side effects remain unknown.

GSK has already made it clear that it wants to see whether Tykerb will be a good drug in the adjuvant setting.6 But because the drug has now been approved and the off-label use of drugs is not regulated, doctors can and almost certainly will start using Tykerb in settings other than the one for which the drug is approved. At $2,900 for a month’s prescription, GSK will make a good deal of money on Tykerb despite the limited population for whom the drug has been FDA approved and the minimal information available on the drug’s side effects. The off-label use of Tykerb also will improve the company’s bottom line.

The number of drugs for which the FDA has issued warnings, or which the agency has forced off the market because of adverse effects discovered after the drug went on the market, has grown enormously in the past few years. BCA, with our allies in women’s health and public health, is supporting FDA reform legislation in Congress that will provide more resources for the agency and more authority to assure drug safety and control drug marketing. To find out more about this effort, contact BCA at

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