Will Your Health Care Plan Pay For Tamoxifen?

By Ronald L. Scott

For the first time, a drug (tamoxifen) has been shown to prevent breast cancer among women at high risk for the disease. Women who took tamoxifen in a large study had 45 percent fewer cases of breast cancer than a control group of women who took a placebo. Results of the study are still being analyzed, and women have been cautioned not to rush to demand the drug until the findings have been tailored to individual women, which analysis will take about two months.

A month’s supply of tamoxifen costs $80 to $100 in the United States, based on the dosage of women who took tamoxifen in the study, i.e., two pills a day, or 20 milligrams. However, women may encounter a number of barriers to having their health care plans pay for the drug. Most managed care plans and insurance policies do not cover treatment that is experimental or investigational. However, virtually every treatment is "experimental" when first introduced, so the issue is really whether the proposed treatment is experimental based on current information. If a physician believes his or her patient needs a treatment that the health care plan has denied as experimental, the plan will need to be convinced that the treatment is recommended by experts in the field, that the patient is in a class of patients shown to have benefited from the treatment, and that the treatment is for the patient’s benefit -- not just for the benefit of furthering scientific research. When the analysis of the study’s findings has been completed, it should be more difficult for a plan to argue the treatment is experimental. Plans define experimental in various ways.

The most restrictive plans view any "off-label" use of a drug as experimental. When the Food and Drug Administration first approves a drug, its labeling is also approved, signifying that the FDA has determined that the drug is both safe and effective to treat the conditions stated in the labeling. However, physicians are allowed to prescribe drugs for conditions not listed in the FDA-approved labeling, i.e., off-label use. Physicians have used tamoxifen to treat breast cancer for 25 years and are free to prescribe it for prevention even without approval from the FDA. It is expensive and time-consuming for a pharmaceutical company to amend a drug’s labeling. Essentially, the company must conduct sufficient clinical trials to convince the FDA that a drug is safe and effective to treat a new condition to be included in the labeling. However, this will not pose a problem in the case of tamoxifen, since demand for the drug is likely to be high, and since this study should be able to form the basis for a decision by the FDA. A news release issued by the FDA advised it could not approve tamoxifen for prevention of breast cancer until it had thoroughly reviewed the information from the study

Finally, even if the drug receives FDA approval for use in breast cancer prevention, a health care plan could deny coverage on the basis of "medical necessity," i.e., that the drug was not an appropriate choice for a particular patient. Dr. Leslie Ford of the National Cancer Institute was quoted by the New York Times as saying: "As with any medication, the decision to begin tamoxifen therapy is a very complex one," and that "even if a woman is at increased risk of breast cancer, tamoxifen therapy may not be appropriate for her" and "there are no simple answers." A woman denied coverage on the basis of medical necessity might have recourse to appeal the denial, depending on state law. For example, Texas requires that any denial of treatment based on medical necessity be appealable to an independent review organization that has the power to order a health care plan to provide the requested treatment.

04/10/98