The assortment of clinical trials focused on symptom management and related costs does not accurately reflect the reality of clinically managing terminally ill cancer patients. Aside from the pain management studies that included associated costs, the costs of other frequently occurring debilitating symptoms of dyspnea, diarrhea, constipation, seizures, and terminal delirium have not been assessed adequately. Not only do we know little about how much it costs to treat these symptoms, we know equally little about the costs to society when they are mismanaged, as they often are.

An interesting associated issue arises with the off-label use of therapies that are thought to be helpful to suffering patients. An intriguing example might be erythropoietin alfa (Epo), which is used for cancer-related anemia. Epo is approved by the Food and Drug Administration (FDA) for various indications, but the only cancer indication for Epo is for patients with nonmyeloid malignancies who are concomitantly receiving myelosuppressive chemotherapy. Physician prescribing is not limited to FDA-approved indications, however, and many cancer patients not receiving chemotherapy, or receiving chemotherapy that is not myelosuppressive, are prescribed Epo for anemia. Although lack of FDA approval is not always synonymous with a lack of evidence of effectiveness, in this case, there have been no trials in the general population of cancer patients, so effectiveness has not been demonstrated. Only one active National Cancer Institute (NCI) Phase III clinical trial is exploring the effect of Epo in anemic patients with advanced cancer undergoing platinum-containing chemotherapy. Nonetheless, it is an approved drug that is covered by Medicare, which paid $210 per injection in 1998. In 1998, about 35 million injections were given to about 2 million Medicare patients, at a cost of about $7 billion. If Epo is given in the hospital, it is part of the diagnosis-related group (DRG) payment for each stay, but if it is given in doctor’s offices, it is a separate covered expense. Either way, it is free to the patient. It has few side effects, beyond the cost.

METHODOLOGICAL CONSIDERATIONS CONCERNING STUDIES REPORTING COSTS

Assessing Costs and Effectiveness

For a few discrete advanced cancers and their symptoms, we know that certain treatments will not provide an improved quantity or quality of life and may be costly as well (Smith, 2001) (Table 2-1). One example of this is second-line chemotherapy for metastatic lung cancer, which entails the use of fairly expensive drugs and a number of toxicities. On the other hand, we know very little about the most efficacious, least costly treatment for the full spectrum of advanced cancers. Indeed, little discussion illuminates the



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