ABMT: a microcosm of the U.S. health care system.

HEALTH CARE TECHNOLOGY

The continuing controversy over appropriate utilization of high-dose chemotherapy (HCDT)-autologous bone marrow transplantation (ABMT) in the treatment of cancer epitomizes the debate in this country over increasing expenditures for the application of health care technology. The debate includes all imaginable constituencies--patients, physicians, hospitals, payers, employers, lawyers, economists, and the media. The issue is fascinating, as it continually presents new twists and turns. Additionally, the way in which the HCDT-ABMT controversy is resolved (or not resolved) will presage the manner in which similar issues are addressed in the 1990s.

The high expense of HDCT-ABMT is a major reason for the attention that has been given decisions regarding its clinical application. Charges for the process of bone marrow harvest, high-dose chemotherapy, and bone marrow reinfusion range from $75,000 to $150,000, with most being in the vicinity of the latter. The cost of an individual procedure is magnified by the approximately one million new cases of cancer each year, including 135,000 new cases of breast cancer. The potential for high-volume use of HDCT-ABMT is being realized by an expanding list of indications and by the application of HDCT-ABMT earlier in therapeutic regimens.

HDCT-ABMT continues to be used for the established indications of acute leukemia, Hodgkin's disease, non-Hodgkin's lymphoma, and stage III and IV neuroblastoma. It is also being applied in metastatic breast cancer and multiple myeloma. Finally, a variety of new applications have been observed, including stages II and III breast cancer, brain tumors in adults and children, ovarian cancer, testicular cancer, Ewing's sarcoma, metastatic melanoma, etc. The move to the use of HDCT-ABMT in earlier stages of the disease process is exemplified by the use of the regimen in stages II and III breast cancer and the earlier stages of multiple myeloma. Additional factors influencing utilization include the use of regimens that involve repeating HDCT-ABMT at some predetermined interval after the first treatment and applying HDCT-AMBT to treat the recurrence of a cancer two or three years after initial HDCT-ABMT improve.

Thus, the high expense, the size of the potential patient population, and rapidly expanding applications clearly identify HDCT-ABMT as a clinical issue that has substantial policy implications.

Outcomes

The debate over the expanded use of HDCT-ABMT is only accentuated by lingering and justifiable concerns over whether HDCT-ABMT improves final health outcomes (i.e., survival) in comparison to standard chemotherapeutic regimens. For example, the results for improvement in survival for metastatic breast cancer overlap in large measure for HDCT-ABMT and repetitive conditional dose chemotherapy. Some studies suggest improvement in overall survival but this usually is measured in months. This measure of improvement is counterbalanced by the association of HDCT-ABMT with the occurrence of death in 5 percent of cases. Thus, the question of improvement in survival and quality of life is a very real one for many of the cancers in which HDCT-ABMT is being applied.

Patient Need

Of course, the most pressing and most real issue is the attempt to address the needs of an individual with a life-threatening disease. Often, that individual's family or personal circumstances dictate that the individual attempt almost anything in order to achieve a cure. For some, individual circumstances and preferences will argue less persuasively for attempts at therapy with significant risks. The opportunity for individuals' full and informed participation in decision-making is essential all along the continuum of individual needs and preferences.

The shared decision-making model being developed by Mulley and Kasper may have application in terminal disease. [1] The initial introduction of this model was for a "quasinecessary, quasi-elective" procedure--transurethral resection of the prostate (TURP) in benign prostatic hypertrophy (BPH). The applicability of the model and the need for enhanced participation in the decision-making process was heightened by the Wennberg study that showed that "watchful waiting" was indeed a viable option for many patients with BPH. [2] In first applying the shared decision-making model, Mulley, Wennberg, and others provided patients with a synopsis of outcomes (beneficial and adverse) associated with the options of TURP and "watchful waiting." This video presentation of data and of patients discussing their own decision-making processes provided a new vehicle for patients to become more actively involved in therapeutic decisions.

Although the severity of the disease and sensitivity of all involved is greatly magnified in terminal illness, increased participation of patients in therapeutic decisions is needed here as well. Thus, the shared decision-making model may have positive application in difficult decisions, such as whether or not to undergo HDCT-ABMT, and will help ensure that the ultimate decision is an informed one

Policy Debate, Hyperbole and

Posturing

The high expense, potentially large volume of use, and critical patient circumstances have raised this issue to the national public policy level. A number of court cases have been heard regarding payer denials of HDCT-ABMT. In a clear majority of these cases, the plaintiff has prevailed. In most of these cases, court decisions turned not so much on the merits (i.e., data) available to support the use of HDCT-ABMT as on the coverage decision-making process and accurate translation of terms (e.g., "investigational) and criteria used in this process into specific contractual language.

The education of the courts on the methods used by payers, physicans, and patients to reach these difficult decision must continue. Because the courts will most likely continue as a forum for debate on the effectiveness and appropriateness of HDCT-ABMT for specific indications for at least the short term, the courts must achieve a greater understanding of clinical and coverage decision-making processes that are relying increasingly on health outcomes data.

Additionally, there has been hyperbole and posturing suggesting that payer cooperation with the National Cancer Institute (NCI) in trials on breast cancer represents an attempt on the part of payers to avoid payment for a larger number of cases. Such posturing ignores the fact that trials would not be sponsored by the NCI if outcomes data had convincingly demonstrated a therapeutic advantage for HDCT-ABMT over conventional therapy in breast cancer. Indeed, if such an advantage had been shown, it would be unethical for such trials to continue. Rather, the NCI trials represent attempt by responsible parties to obtain objective data to incorporate into their processes for making these difficult decisions.

Other Issues

There are a number of other interesting and important issues that cannot be discussed in depth here. What is the cost-effectiveness of HDCT-ABMT in the various cancers? more established indications, such as acute nonlymphocytic leukemia, the case has been argued that HDCT-ABMT is as cost-effective as conventional chemotherapy. Related to this issue is the ultimate impact of colony-stimulating factors in reducing the length of stay and, thus, the costs associated with HDCT-ABMT. More practical issues involve the views of employers. On a national level, employers are voicing strong concerns over the need to decrease the rate of rise of health care expenditures and the desire to pay only for technologies that have proven to be safe and effective. On a local level, when an employee who has served a company well for 25 years presents with terminal cancer, the company appropriately feels obligated to provide what it can for the patient.

Summary

HDCT-ABMT crystallizes the issues and concerns that have surrounded the deate over ways to deliver high-quality health care while controlling the rate of rise in health care expenditures. Is this country's health care system moving to decisions based on data about a technology's safety and effectiveness? The HDCT-ABMT issue would suggest that it is at a painstakingly and begrudgingly show pace. HDCT-ABMT epitomizes the difficulty that individuals have applying data developed in the aggregate to their own circumstances, particularly when the circumstances are dire. Thus, it would be naive to think that the development of even significant amounts of data will completely eliminate the sometimes adversarial relationship between payers and providers. However, even these adversarial relationships must be viewed as a positive tention that will pull the delivery system to more informed decision making on all levels.

The debate over appropriate use of HDCT-ABMT will continue for the foreseeable future. At the end of this decade, we will look back and view the HDCT-ABMT issue as a landmark that helped determine the extent to which health outcomes data and patient preferences truly influence difficult utilization decisions.

William T. McGivney, PhD, is Head, Technology Assessment and Policy, Aetna Health Plans, Hartford, Conn.