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).

The treatment of End-Stage Renal Disease (ESRD) represents a victory for medical technology. Dialysis and kidney transplantation, developed in the early 1960s, offer alternative treatments to patients whose own kidneys no longer function; before, these patients faced a terminal diagnosis. Dialysis is a mechanical treatment in which the patient is connected to a machine that cleanses the blood of impurities and returns it to the body. Although recent innovations (e.g., continuous ambulatory peritoneal dialysis—CAPD) facilitate patient independence from a machine, replacement of the diseased kidneys is the most desirable and least expensive treatment for many patients (33;39). Kidney transplantation remains the most effective and common type of transplantation, and a new kidney (from a living-related or cadaver donor) often dramatically improves the recipient's health and general well-being (20;39). Now, in the mid-1980s, these technologies are no longer new and innovative. Further analysis of these established but costly technologies provides a perspective on the long-range implications of innovations in patient care: while some new issues have emerged, many problems originally associated with these treatments seem to have intensified. Access to treatment remains a central issue, closely linked to the dilemma of equity versus cost. The contrast in the access provided by the United States and Great Britain is dramatic (40); in 1982, the rate of ESRD treatment within the U.S. was twice that of the U.K. (353 versus 160 patients per million) (37). The U.S. policy is basically one of unlimited access, whereas the U.K. has restricted access.

The term “assessment of medical technologies” does not appear to have a unique meaning that always applies to its use. It seems wise, then, to state what will be meant by the term in this chapter. “Medical technology” is regarded here as including drugs, devices, procedures, (whether preventive, diagnostic, or therapeutic) that bear more or less directly on patients. Thus, an educational program for patients might be a technology, but a training program for dietitians would not. A surgical procedure or a prosthesis would; but a scale for assessing quality of life would not. “Assessment,” as used here, refers to a complex set of activities that comprise data acquisition, analysis, and interpretation concerning properties of medical technologies. Assessment tends to be comparative, either explicitly (as to a control group) or implicitly (as looking toward “usual experience”). In any case, the goal of assessment is to increase understanding of such properties of a medical technology as its applicability, convenience, efficacy, safety, effectiveness, cost, etc.

French drug policy is closely controlled by the government to achieve two main purposes: to ensure therapeutic efficacy and safety, and to control pricing. Manufacturers usually price new drugs at the amount the French Sécurité Sociale (Social Security) agrees to reimburse the consumer. The specific steps a new ethical drug must go through in order to be released on the French market, and the process of negotiation between the government and the manufacturer are discussed in this paper.

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.

In a recent article in Commentary, American medical sociologist Florence Ruderman observes that the evolution of medical practice and medical attitudes in the United States has been guided largely by the autonomous development of biomedical science and technology. “Science”, she asserts:

… is a profoundly unsettling force … it causes endless dislocations and conflicts. Most basically, science is a source of independent values, motives, norms of conduct, and criteria of judgment. It sets its own course, defines its own goals. As for technology, its impact on medicine is even more obvious and direct; again, not just in producing tools or power but in transmitting values and in shaping the field from within. One need only think of the artificial-heart cases now in the news to realize that the physicians involved … are impelled by a technological drive that has its own logic and values, its own momentum—and its own dangers (24,45).

All drug evaluation and regulation policy in the Federal Republic of Germany is based on the Drug Law of 1976. In accordance with the pharmaceutical directives of the European Community, this law provided that all drug products must be of good pharmaceutic quality, therapeutically effective, and safe according to the current state of knowledge. It is aimed at guaranteeing safety in the use of drugs without hindering the plurality of thought in medical science.

Few would argue that the randomized clinical trial is the most definitive instrument available for measuring the safety and efficacy of a new medical technology, be it drug, device, or procedure. Randomization removes the potential for bias in the allocation of subjects to the intervention group or to the control group. In addition, randomization tends to produce comparable groups which can be further strengthened by stratification and by stratified analyses. The randomized controlled trial guarantees validity of statistical tests, as long as the sample size is large enough to avoid type-II error.

If medicine is becoming mechanized, as many indications suggest, then those interested in policy making for medical matters have much to learn from the history of technology. The mechanization of medicine, as in the case of the mechanization of production, will accelerate the transfer of skill and knowledge from people to machines and the transition of health care to a capital intensive industry (19, 196–226). Furthermore, mechanization and increasing capital intensification may bring the increased systematization of health care. If the development of mechanized medicine follows the precedent of the mechanization of production, then our society must deal with the evolution of another set of extremely large systems, systems that will become virtually impervious to social control. Historians of technology are currently providing a better understanding of the evolution of large systems of production (3;9;10); there are lessons to be learned from this history by policy makers in health care.

At about the time of national confederation in Canada, in the 1860s, profound changes had begun to take place in the field of chemical science, although most of these had not yet been translated into any advances in drug treatment. This was the era of chemical brilliance in Germany. Paul Ehrlich (1854–1915), often referred to as the father of modern pharmacology, had applied, with great foresight and considerable courage, the 1865 insights of August Kekulé, the chemist who had accomplished the difficult transition between inorganic and organic chemistry. Indeed, it was Kekulé who made possible the chemical –magic bullet— that could reach body organs to effect therapeutic changes, a realistic goal for Ehrlich, especially in his work associated with the treatment of syphilis.

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.

Economic techniques have been used for several years to evaluate health care expenditures, and there is reasonable agreement amongst economists as to the appropriate methodology. Comprehensive reviews of techniques and applications can be found in Drummond (3,4), Culyer and Horisberger (2) and a report from the Office of Technology Assessment (7). More recently Williams (13) has addressed the problems of evaluating a developing technology and Wagner (10) the specific problems of diagnostic technologies.

The basic aim of a drug policy is to ensure that effective and safe drugs of good quality are available to cover the health needs of a country. A national drug policy should be considered an integral part of any comprehensive health-care policy. The formulation of national drug policies varies even between similar countries because of conflicting interests and different political, economic, and social pressures. It is influenced by such factors as:

the health situation of the country;

the medical care system;

the education and training of health personnel;

the social security and health-insurance schemes;

drug research and development possibilities;

the domestic production of drugs;

the determination of the demand for drugs;

the system of drug distribution;

the possibilities for evaluation and control of drugs; and

international policies on medicinal products.

There are three phenomena unique to medical research. First, unlike most historic events and their time- and culture-specific relationships, determination of the efficacy of medical care cuts across national and cultural boundaries in the Western world. Second, most of the study methods currently used to determine whether a specific medical intervention works have a long tradition, usually stretching back over centuries. Third, it is difficult to predict whether, and how readily, research results will be incorporated into medical practice.

The standard way to assess medical technologies is to conduct a randomized clinical trial. Patients are randomly assigned to groups receiving alternative treatments, and outcomes are monitored over a long period of time. For example, some victims of left main coronary artery disease may undergo coronary artery bypass surgery, and others may receive medical treatment with nitroglycerine and beta blockers. Comparison of five-year mortality and morbidity in the two groups helps to determine the relative appropriateness of the two procedures. In addition, information about quality of life and cost can also be collected and compared.

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 consensus development program for health care is an American invention which dates back to 1977. It is derived out of concern for the appropriate use of medical technology and designed to formalize the process by which research pertinent to health care is disseminated and used.

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).

The assessment of drugs in Sweden has a long tradition that goes back to the seventeenth century when the Collegium Medicorum was established in 1663. One of its duties was to control the production of drugs in pharmacies. In 1688, Sweden got its first pharmacopoeia. In 1917, the Pharmaceutical Laboratory was founded as an advisory body of the National Board of Health and Welfare. The laboratory, now called the Department of Drugs, became responsible for evaluating drug applications, including information on their pharmaceutical and chemical properties, pharmacological actions, and clinical effects.

It has been suggested that efficacy and safety are the core issues for technology assessment in health care (1). To this must be added the issue of appropriateness (2). Efficacy remains the first issue nonetheless, for surely no one would knowingly evaluate the safety of an ineffective technology.