Substitution of lost anatomical structure or physiological function by various devices was one of the earliest technologies in medicine. The wooden leg, the spectacle lens, and the ear trumpet were antecedents of today's powered limb prostheses, pacemakers, valves, vessels, and hearts. Transplantation of tissues or organs from humans and animals represents a further step along the same path. The replacement of deficient organic chemicals such as insulin and coagulation factors might be considered as part of the same continuum. Such substitutes may be wholly external. These include the extension of extremities foreshortened by congenital or acquired abnormality and various communication aids that allow the transmission or reception of information. Replacing the chemical activity of the kidney by dialysis is another external device.

For several decades, major advances in medicine have increased dramatically our ability to prevent, diagnose, and manage a wide variety of chronic and acute health problems. However, medical innovations also pose significant economic, social, and ethical questions. How do we best utilize scarce medical resources? How do we balance anticipated benefits against the dollar cost or associated risk? What is the impact on total health care spending now and in future years? As concern over these issues grows concomitant with increased health care spending, technology assessment is often promoted as a method for determining whether a biomedical innovation should be encouraged to disseminate rapidly in order to maximize its benefits to individual patients, or constrained to develop slowly in order to minimize collective costs or risks. But, the art and practice of evaluating medical technologies, especially innovations still in the research and development phase, are not well developed (6;8;11;14;16;34;35).

Viewed in relation to the vast amount of clinical research in the United States, the number of total artificial heart (TAH) implants is so small as to be statistically invisible. And while the results of those implants to date may seem dubious in terms of the recipients' outcomes, theyare in fact very similar to those of many other innovative therapies when they are first tried ondesperately ill patients. Why, then, has the artificial heart been the object and subject of suchextraordinary interest and controversy? It has been the subject of special federal studies, reports, and hearings for 20 years; has been perceived and portrayed as a unique chapter in the annals of human experimentation and therapeutic innovation; has attracted medically unprecedented media attention; and, in the case of permanent implants, has received unusually exhaustive and restrictive regulatory supervision.

For some observers, the artificial heart represents the latest, and perhaps the most flagrant example of the health system's tendency to favor the rapid introduction of expensive but ineffective technologies over efforts to prevent disease and to improve access to care (5;6;19;44;45). Even if it can be perfected, they argue, its opportunity cost in terms of other foregone health benefits would be exorbitant. The ultimate failing of the health care system, it would seem, is its failure to establish mechanisms to select among alternative uses of resources. If such mechanisms had existed, some critics believe that the quest for an artificial heart never would have begun and certainly its premature clinical uses could have been prevented (6;45).

From the perspectives of coverage and reimbursement, heart transplantation has been a serious concern of policymakers in the U.S. since 1980, the year it was decided that a comprehensive study was required before the Health Care Financing Administration (HCFA), the federal agency responsible for the administration of the Medicare program, would be able to decide the status of the procedure (14;15;30;35). It was well acknowledged that the issues surrounding this decision were complex and that initial attempts to resolve the underlying coverage issue seemed to be too narrowly construed. It was at this time that the late Patricia Roberts Harris, then Secretary of the Department of Health and Human Services (DHHS), declared that DHHS would require new technologies to pass muster on the basis of their “social consequences” before “financing their wide distribution” (30). In a very special sense, although not appreciated at the time, a new era of health care technology assessment was ushered in, as Harris proclaimed that the then-conceived study of heart transplantation should serve as the “prototype” of technology assessment.

Liver transplantation is an exciting new therapeutic procedure that over the past few years has shown signs of increasing interest among physicians and health care providers (26;36). The history of liver transplantation dates back to the immediate post-World War II years when early experiments showed the technical feasibility of transplanting a functioning liver into a recipient, either heterotopically as an accessory organ or orthotopically, i.e., by complete replacement of the original organ (29).

Since the National Institutes of Health (NIH) Consensus Development Conference (1983) concluded that liver transplantation was a procedure deserving of wider application to the treatment of end-stage liver disease, there has been a very considerable increase in the number of centers performing liver transplantation, in Europe as well as in the United States. The number of operations performed has increased logarithmically (Figure 1, in Höckerstedt and Kankaanpää, p. 453). With the detailed overall assessment of liver transplantation in Europe by Höckerstedt and Kankaanpää, we will take the opportunity to review the position in Great Britain from a physician's, i.e., non-surgeon's, viewpoint as seen from one of the two centers (Cambridge/King's College and Birmingham) currently recognized in this country. This is based on an experience of 255 cases dating from the first liver transplant performed in Great Britain by Professor Roy Y. Calne in May 1968.

The validity of technology assessment may depend largely upon who performs the job, and when they get involved. The options are many. Industry may hire a small private laboratory to perform animal trials on a completed device or drug; manufacturers may support their own in-house laboratories or even study beds; industry may contract with hospitals or private medical groups to evaluate a potential product. It is the thesis of this discussion that there is a different option which brings together the strengths of three often stubbornly independent institutions: industry, university-based investigators, and federal science granting agencies.

Insulin dependent diabetes mellitus ranks as one of the major disease entities in the world. It is widespread in industrialized countries as well as in developing countries. In spite of the considerable improvements in conventional diabetes therapy during the past few years, severe secondary complications still develop in most patients. The National Commission on Diabetes in the United States has reported that patients with insulin dependent diabetes are 25 times more prone to blindness, 17 times more prone to kidney disease, 5 times more often afflicted with gangrene and twice as often afflicted with heart disease than are non-diabetic individuals. In Sweden diabetes is now the second most common cause of uremia and it is the most common cause of blindness in adults. The life expectancy for the diabetic patient is approximately one-third less than that of the general population. Since the metabolic abnormalities that prevail in diabetes mellitus are due to abolished beta-cell function, it would seem logical to treat this disease by the transplantation of normally functioning islets of Langerhans. This operation should correct the metabolic abnormalities and presumably prevent or delay the severe secondary complications. There are two ways of transplanting the endocrine pancreas, either by means of a vascularized graft or by providing free, isolated islets. Unfortunately it has not been possible, so far, to correct diabetes in humans with islet transplantation. However, during the last years there has been substantial progress in the field of vascularized pancreatic transplantation and the results now begin to approach those obtained with other organs such as the kidney, heart, and liver.

The National Health Service (NHS) supplies the majority of health care in Great Britain but is virtually the monopoly provider of high technology and disaster services such as treatment for chronic renal failure or end-stage renal disease (ESRD).

Bone marrow transplantation has become an accepted treatment for severe aplastic anemia, acute myelogenous leukemia, and acute lymphocytic leukemia (20;35). Less commonly thalassemia (23), multiple myeloma (25), severe combined immune deficiencies (10), and chronic granulocytic leukemia (14) are also treated with marrow transplantation. Most donors are siblings whose HLA phenotype is identical to the patient's. Such donors, however, are available to only one third of patients (6;16;27;28;35). In the absence of an HLA-identical sibling, partially matched, haplo-identical, related donors (differing by only one major histocom-patibility antigen) can be found for another 7% of patients (1;6;27;37). A variety of lower quality matches also can be found but have not been used extensively. Advances in tissue typing and the treatment of graft versus host disease now allow physicians to consider matched unrelated individuals as potential donors when a suitable related donor is not available (11;12;13;15;17;22).

The Netherlands is a small country of about 15 million inhabitants. With 400 inhabitants per square kilometer, it is one of the most densely populated areas of the world (19). Here, government is not seen as negative, but as a tool to solve societal problems. Ordinarily, the public and private sectors work cooperatively together, but the government will step in when needed. (19, 20).

In 1958 Joseph Murray and associates reported the first successful kidney transplantation between two identical twins at Peter Bent Brigham Hospital in Boston (14). Since then organ transplantation has advanced rapidly. Renal transplantation is now a standard treatment for irreversible renal failure, and liver transplantation is an accepted method for treating end-stage liver disease. A number of patients have survived more than 20 years after renal transplantation and more than 10 years after liver transplantation (20). Very few liver transplantations have been performed in Sweden but this method is expected to become more common in the near future.

This article describes issues that are clinically significant when assessing quality of life of patients whose organ disease can be treated only through transplantation or reliance upon an artificial organ. Emphasis is placed on stresses that affect the family as well as the immediate patient, because, over time, these are inextricably interwoven. The transplant recipient who experiences a “good” medical outcome may perceive that he has little to live for if his family is severely disrupted or broken apart by the strain of the illness. The family of a patient who dies after unremitting complications while being sustained by an artificial organ may experience both relief at the lifting of an unmanageable burden, and continuing guilt that they were unable to provide unconditional love and support to a dying family member.

The economic, political, ethical, and medical issues surrounding organ transplantation are complex, and coverage policies for it vary considerably among insurers. Some insurers cover virtually all transplants, while others cover few or none (Wyoming Medicaid does not cover any transplants) (3;4;5;6;7). In the past, insurers have followed Medicare's lead regarding coverage of new technologies. This has not been the case with organ transplantation. Although Medicare does not cover heart transplants, almost all Blue Cross/Blue Shield plans and commercial health insurers, as well as one-half of the state Medicaid plans and one-third of the Health Maintenance Organizations (HMOs), do cover heart transplants. (See note, p. 570.) In addition, although Medicare does not cover heart–lung transplants, a substantial proportion of Blue Cross/Blue Shield plans, commercial health insurers, state Medicaid plans, and HMOs cover it. At present, Medicare covers kidney and cornea transplants, selected pediatric liver transplants, and selected bone marrow transplants.