GAO General Accounting Office
Washington, D.C. 20548
Health, Education, and Human Services Division
April 24, 1996
The Honorable Ron Wyden
United States Senate
Dear Senator Wyden:
Rapid advances in biomedical research and technology are producing a continuous stream of new, and often expensive, medical devices, drugs, and therapies. Health insurers' decisions about whether and when to provide coverage for these new medical products and treatments play a pivotal role in determining their availability for use in general medical practice. In recent years, conflict over insurers' coverage decisions of new medical treatments has led to litigation and to a variety of federal and state legislation and regulations that mandates insurance coverage of some new medical treatments.
Some of the most visible and contentious coverage decisions have involved the treatment of breast cancer with high-dose chemotherapy supported by autologous bone marrow transplantation (ABMT). In this procedure, bone marrow or stem cells from the blood are taken from the patient and then reinfused after high doses of chemotherapy have been administered. The high-dose chemotherapy is toxic to the bone marrow which produces the blood cells that fight infections. ABMT following the chemotherapy treatment helps restore the patient's ability to produce the blood cells that fight infection.
Most experts say that more research is needed before definitive conclusions can be reached about the treatment's effectiveness compared with conventional chemotherapy. Proponents of insurance coverage of the procedure say it provides breast cancer patients with access to a promising, potentially life-saving treatment. Critics say that the public is not well-served by the proliferation of an unproven treatment that is costly and possibly harmful, and that such proliferation hinders clinical research to determine if the treatment is effective.
To illustrate the issues raised when demand grows for coverage of a new treatment in advanced clinical trials, you asked that we provide you with information regarding insurance coverage of ABMT for breast cancer. Specifically, you asked that we address (1) the factors that have influenced insurers in deciding whether to cover the treatment, (2) the status of the research on ABMT for breast cancer and the consensus on what is known about its effectiveness, and (3) the consequences of the increased use and insurance coverage of the treatment while it is still being evaluated in clinical trials.
To develop this information, we conducted structured interviews with officials responsible for medical coverage decisions at 12 health insurance companies, including some of the nation's largest insurers. These companies also included a mix of indemnity and managed care plans. We also obtained information from researchers and oncologists at major research centers, large urban hospitals, and community hospitals. Others we obtained information from included the American Society of Clinical Oncology; the National Association of Insurance Commissioners; patient and women's health advocates, including the National Breast Cancer Coalition; state health officials; technology assessment organizations; and the National Cancer Institute (NCI). We also reviewed state and federal legislation and regulations regarding insurance coverage of ABMT, as well as relevant scientific literature, and visited a large, private transplant center. The National Institutes of Health (NIH) and the Office of Personnel Management (OPM) provided formal comments on a draft of this report. We did our fieldwork and analysis from April to December 1995 in accordance with generally accepted government auditing standards.
As agreed with your office, unless you release its contents earlier, we plan no further distribution of this report for 30 days. At that time, we will send copies to other congressional committees and members with an interest in this matter, the Secretary of Health and Human Services; the Director, NIH; and the Director, OPM.
This report was prepared by William Reis, Assistant Director; Joan Mahagan; and Jason Bromberg under the direction of Mark Nadel, Associate Director. Please contact me on (202) 512-7119 or Mr. Reis on (617) 565-7488 if you or your staff have any questions on this report.
Sarah F. Jaggar
Director, Health Financing
and Public Health Issues
[The text of the report follows. Ed.]
Results in Brief
Although it is widely considered an experimental therapy, many health insurers are covering ABMT following high-dose chemotherapy for breast cancer. The 12 insurers we spoke with said they based their decision to cover the treatment on the preliminary clinical evidence, but also on factors like fear of litigation and adverse public relations.
The use of ABMT for breast cancer has increased rapidly in recent years, from an estimated 522 patients in 1989 to an estimated 4,000 in 1994. At least seven states now require insurers to cover ABMT for breast cancer, and other states have such legislation under consideration. Medicaid covers the treatment in some states, and OPM has required that all beneficiaries of the Federal Employees Health Benefits Program be covered.
Despite its increased coverage and use, most experts say they do not yet know whether ABMT for breast cancer is effective, and for which patients, compared with conventional therapy. Randomized clinical trials sponsored by NCI are expected to provide the most definitive answers, but these will not be completed for several years. In the meantime, there have been sharp disagreements among researchers, physicians, NCI, insurers, and patients about the appropriate use of the treatment before definitive research results are available. At one end are those who argue that the preliminary evidence supports a policy of widespread use and universal insurance coverage of the treatment. At the other end are those who feel that the treatment should largely be restricted to patients enrolled in randomized clinical trials until the treatment's effectiveness has been clinically proven.
The NCI-sponsored trials have been slow to accrue patients. Many experts expressed concern to us that the wide availability of ABMT has impeded the ability to complete these randomized clinical trials, which require a control group of patients who receive conventional therapy. There is also concern that a substantial portion of patients receiving ABMT are doing so outside of any research setting, which may further slow down the effort to learn whether the treatment is effective.
If ABMT is ultimately shown to be preferable to conventional therapy for some groups of breast cancer patients, then those patients will have benefited from the early diffusion of this technology. If it is not, however, then the widespread availability of the treatment before its effectiveness has been established will mean that many patients may have been unnecessarily subjected to an aggressive and toxic treatment. The diffusion of the treatment also has implications for health care costs: ABMT typically costs anywhere from $80,000 to over $150,000, compared with approximately $15,000 to $40,000 for conventional chemotherapy.
Breast cancer is the second leading cause of cancer deaths among American women. The American Cancer Society estimates that there will be 184,300 new cases of breast cancer diagnosed in US women in 1996 and that 44,300 women will die from the disease. One in eight women will develop breast cancer during her lifetime.
Breast cancer is generally classified into four main stages based on the size of the tumor and the spread of the cancer at the time of diagnosis. Mortality rates are strongly related to the stage of the disease at the time of detection. Stage I patients have an excellent chance of long-term survival, while stage IV (metastatic) breast cancer is usually fatal. A wide variety of treatments exists for breast cancer patients, including surgery, chemotherapy, radiation therapy, and hormone therapy. The particular treatments used depend on the stage and characteristics of the cancer and other aspects of the patient and her health.
ABMT is a therapy that allows a patient to receive much higher dosages of chemotherapy than is ordinarily possible. Because high-dose chemotherapy is toxic to the bone marrow (which supports the immune system), methods have been developed for restoring the bone marrow by reinfusing stem cells (the bone marrow cells that mature into blood cells) taken from the patient before chemotherapy. Stem cells are removed from the patient's blood or bone marrow, then concentrated, frozen, and sometimes purged in an attempt to remove any cancerous cells. The patient then undergoes chemotherapy at dosages 2 to 10 times the standard dosage. To restore the ability to produce normal blood cells and fight infections, the patient's concentrated stem cells are thawed and reinfused after chemotherapy. When the transplant is done from the blood rather than the bone marrow, the procedure is often referred to as peripheral blood stem cell transplantation.
ABMT is an expensive treatment although the cost per patient has been falling in recent years. Aside from financial costs, the treatment is usually very unpleasant for the patient and may pose significant risks. The high doses of chemotherapy are very toxic, leading to treatment-related morbidity and mortality rates that, while declining, are still higher than for conventional chemotherapy. There may also be problems in restoring the patient's ability to produce normal blood cells and thereby fight infections. ABMT is being evaluated in the treatment of a number of types of cancer other than breast cancer and is considered standard therapy for treating certain types of leukemia and lymphoma under certain conditions.
Many clinical trials have been conducted to assess ABMT for breast cancer, but most of these studies have been phase I and phase II trials, which most experts agree have been of limited use in firmly establishing the effectiveness of ABMT compared with conventional therapy. NCI is currently sponsoring three randomized clinical trials that seek to determine whether ABMT is better than current standard therapy in comparable breast cancer patients. These trials seek to ultimately involve a total of about 2,000 women at more than 70 institutions around the country.