Orphan drug products generally are used in treating or preventing rare diseases. The small number of patients available for study may create special problems in the evaluation of these products. This paper examines some of the special problems that are associated with the design and implementation of studies to evaluate the safety and efficacy of orphan drugs. The U.S. Food and Drug Administration (FDA) has not established special criteria for evaluating orphan drugs per se, but the FDA has been flexible in evaluating drug products that present special problems, especially when these products are for treatment of serious of life-threatening illnesses. The FDA and other U.S. governmental agencies also have taken steps to promote the development and availability of drugs for rare diseases, including making these products available to patients who are in need, even before the drugs have full FDA marketing approval.

The rapidly changing health care system is still largely organized according to old, and increasingly outdated models. The contemporary demands of patient care and residency training call for an experimental ward, which can develop and test new techniques in hospital organization and the delivery of care in a comprehensive way.

The advantages and limitations of national registers containing individual identification numbers in clinical epidemiological research are reviewed. The author concludes that such registers are very valuable for epidemiological research and for formulation of research questions of public health importance.

Different aspects of the process of evaluating research and education are discussed, using the discipline of medicine as a model. The focus is primarily on potential problems in the design of an evaluation. The most important aspects of an assessment are: to create confidence in the evaluation among scientists and/or teachers who are being assessed before beginning; to find experts for whom the scientists and/or teachers have professional respect; to choose assessment methods in relation to the focus, level, and objectives of the evaluation; and to make the report of the evaluation's findings short and explicit.

Failures are inevitable in health care. When failure occurs as a result of practitioner error, quality in patient care may be compromised. This article proposes that analyzing clinical errors may contribute to quality assurance. Examples from physical therapy illustrate how information gained from analyzing errors can enhance patient care.

Economic evaluations can be used to compare alternative antenatal care programs in terms of cost and outcome. The cost of routine antenatal care is small compared to the total cost associated with pregnancy and childbirth. The main problem in economic evaluations is related to outcome measurement.

Despite the need for more safe and effective contraceptive drugs and devices, enormous barriers to contraceptive research and development have been raised in the United States. The designation of contraceptives as orphan drugs, with concomitant incentives, may be warranted to encourage private manufacturers to reenter the field.

Cardiac rehabilitation outcome assessment has reflected the isolated concerns of different professionals, each addressing different factors of the problems of patients. Physical, medical, psychosocial, and epidemiological approaches have been taken. Discrepancies in findings may relate more to problems with assessment than to the technology itself, and can undermine the usefulness of the intervention.

Firms research combines parallel providers of care, ongoing patient randomization, and continuous experimental changes and improvement. This form of efficient research now has a substantial track record of published studies by a growing number of hospitals.

In general, technically demanding medical procedures are associated with better outcomes when they are carried out in institutions and by physicians with higher volumes of practice. This paper examines the evidence for a volume–outcome relationship in the case of organ transplantation. Although few studies have been done on this subject, existing evidence is consistent with improved outcomes at higher volumes. Therefore, evidence supports policies that regionalize transplantation services.

The development and assessment, over a 10-year period, of a technology for group treatment of patients with chronic pain and anxiety is described. Positive results, such as a decrease of symptoms and improved self-confidence, have stimulated diffusion of the technology to colleagues and have increased our own involvement in the organization of services.

Hospital-based “firms” provide a means for combatting the fragmentation experienced by both patients and caregivers in the modern teaching hospital environment. A “firm” is an academic group practice that includes attending physicians, physician trainees, nurses, other staff, and patients. Each person's relationship with a firm lasts throughout his or her association with a particular institution. This article describes the firm system that was recently implemented on the Medical Service of the Cleveland VAMC. This system incorporates both inpatient and outpatient general medical services and provides for unbiased assignment of patients, physicians, and nurses.

Hypertension during pregnancy, defined as a diastolic blood pressure of at least 90 mm Hg, occurs in about 7% of Western countries. Primiparity and familial factors are the most important risk factors. Fifty percent of women experienced blood pressure elevation as late as the last month of pregnancy.

A computer simulation model shows that the cost-effectiveness of treating hypertension is highly sensitive to different assumptions about the effectiveness of treatment, the outcome measure, the cost concept, the discounting of effects, and the duration of therapy. Cost-effectiveness analysis should be supplemented by another approach–cost-benefit analysis based on the contingent valuation (CV) method (the measurement, by survey, of willingness to pay). The CV method is tested in two empirical applications that indicate that it is possible to use the method in this area. Its results should be interpreted with caution, however, since the reliability and validity of the method is not yet established.

With the growing international literature in economic evaluation and the rapid spread of new health technologies, there is a need to undertake, or at least interpret, economic evaluations on the international level. However, the ways in which cross-national differences affect the cost-effectiveness of health technologies or their evaluations have never been studied. This paper explores these issues by taking advantage of a unique situation in which the same economic evaluation of a new indication for a health technology was conducted simultaneously in four countries using an identical methodology. The study showed that if prior agreement on methods can be reached and local data applied, economic evaluations can be undertaken in a way that facilitates the extrapolation of results from country to country.

The literature dealing with screening for hypertension in pregnancy was reviewed. No level of blood pressure or any other factor provides a guarantee of no risk for the development of preeclampsia. However, higher blood pressure in early pregnancy and a failure to decrease blood pressure in midpregnancy are both associated with the development of preeclampsia. The development of proteinuria, rather than the level of blood pressure, is the best predictor of poor pregnancy outcome. Multiparas, especially those with severe chronic hypertension who develop preeclampsia, are at greatest risk of poor pregnancy outcome.

The treatment of prostate cancer was reviewed at a U.S. National Institutes of Health Consensus Development Conference in June 1987. Data from the U.S. National Cancer Institute's Surveillance, Epidemiology, and End Results tumor registries were analyzed and showed that the proportion of eligible prostate cancer patients receiving the recommended therapies did not increase at a faster rate after the conference than before.

Health care technology assessment is broadly and ambiguously interpreted, diluting its role. It is time to recast the field. We must elucidate it in the contemporary context of health inquiry, explicitly relating it to such pursuits as quality assurance, health services research, effectiveness research, medical informatics, and technological innovation.

A hospital's quality of care is generally assessed by a review of individual records. This study used unadjusted and risk-adjusted complication rates to measure the quality of care for hospitals that perform coronary artery bypass surgery or angioplasty. Hospitals differed greatly in their complication rates. Only a small percentage of this difference was due to differences in the risks that patients faced.

A pilot study was done to assess the feasibility of reducing the hospital stays of patients with total hip replacement (THR). The length of hospital stay for these patients depends largely on how rehabilitation, mostly physical therapy, is organized. This study shows that not more than a half hour per postoperative day was devoted to care services and rehabilitation activities. It is feasible and less expensive to reduce substantially hospital stay by planned physical therapy in the patient's home. These results have prompted a randomized controlled clinical trial to assess hospital versus home rehabilitation.