The introduction of noninvasive imaging techniques allowed diagnosis of intracranial pathology in the newborn infant during life, which previously was only possible at postmortem. This has stimulated new interest in correlating pathology with the pathophysiological changes that lead to these events, the neurological signs associated with them, and their ultimate effect on the neurological state of the survivors.

While subcutaneous heparin is a standard prophylaxis for death from pulmonary embolism following general surgery, it has been suggested that adding the vasoconstricting drug dihydroergotamine would improve survival compared to heparin alone. Dihydroergotamine may be associated with rare but life-threatening side effects; thus, reduced mortality from pulmonary embolism could be offset by increased mortality from other causes. Because a clinical trial to examine this possibility would be impractical, we performed a cost-effectiveness analysis to evaluate the effects of prophylactic dihydroergotamine on mortality. Based on published data, despite its favorable effects on the prevention of deep vein thrombosis, the addition of dihydroergotamine did not appear to save lives when added to heparin as prophylaxis. Probabilistic sensitivity analysis demonstrated that even if published risk estimates are in error, substantial changes would still not support the conclusion that dihydroergotamine is lifesaving. In the absence of clear potential for improved survival, the increased costs associated with dihydroergotamine provide reason to question its routine prophylactic use in general surgery.

One of the common ingredients in all attempts to slow escalating health care costs is to control the utilization of services that provide little or uncertain benefit. To reform existing delivery systems, the organization, provision, and financing of health care need to be evaluated critically. Successful reform requires that more rational methods be used to determine which services to provide. The preferences of physicians, patients, and payers of care do not appear optimal from a societal perspective in choosing health care services. While reducing the use of interventions of little or unknown benefit should save money, a policy to restrict the use of medical services may lead to an unwanted result: the underutilization of interventions of proven clinical benefit. Through the determination of the value—by rigorous assessment of both costs and benefits of available alternatives—in a context sensitive to the unique cultural, political, and economic characteristics of individual nations, the health of the population should be improved and growth of health expenditures constrained. This is the first step in health care reform.

Countries worldwide spend huge sums on health—about $1,700 billion a year, or roughly 8% of global income. But the World Development Report 1993: Investing in Health shows that these monies could be spent much more wisely, in the process doing a great deal to help the world's 1 billion poor. Essential national public health and clinical packages are proposed based on assessment of the burden of disease (measured in disability adjusted life years) and the cost-effectiveness of interventions. Governments can play a central role in improving the health of their citizens: they can foster an environment that enables households to improve health and they can also improve their own spending on health, targeting it to support universal access to essential national public health and clinical packages based on the above methods. This is a good example of the concept of needs-based technology assessment, combining the disciplines of epidemiology, economics, and policy formulation. When applied, it should lead to improved effectiveness, efficiency, and equity.

Objectives: As health care budgets continue to face close scrutiny, any new acquisition must be evaluated for both costs and outcomes. This study was undertaken to demonstrate the application of an economic framework for the evaluation of a multileaf collimator as an example of a new technology that can have both quantifiable and nonquantifiable benefits for patients, staff, and cancer care institutions.

Methods: Using financial data from the Northeastern Ontario Regional Cancer Centre (NEORCC) and a recognized staffing model, a commercial spreadsheet, developed to economically characterize the principal radiotherapy processes has been used to determine the net incremental annual cost of a multileaf collimator (MLC).

Results: The incremental annual cost of purchasing an MLC is estimated at approximately $85,000 (1997 CDN $). Without increasing patient throughput, this increases the average cost of a course of radiotherapy by approximately CDN $200. Savings can be accrued by decreasing mold room activity, increasing the hourly patient capacity on each treatment machine, and decreasing sick time due to strain injuries.

Conclusions: Although the clinical outcome of techniques facilitated by MLCs, such as intensity-modulated radiation therapy, are unknown at this time, an economic context within which to objectively evaluate this technology is presented. The framework presented suggests a method of quantifying outcome-justified expenditures, such as improved patient outcome and greater treatment flexibility, which, when offset against the incremental annual equipment cost, may be used to help justify the acquisition of multileaf technology.

Objectives: The aim of this study was to determine the aspects of expert advice that decision makers find most useful in the development of evidence-based guidance and to identify the characteristics of experts providing the most useful advice.

Methods: First, semi-structured interviews were conducted with seventeen members of the Interventional Procedures Advisory Committee of the UK's National Institute of Health and Care Excellence. Interviews examined the usefulness of expert advice during guidance development. Transcripts were analyzed inductively to identify themes. Second, data were extracted from 211 experts’ questionnaires for forty-one consecutive procedures. Usefulness of advice was scored using an index developed through the qualitative work. Associations between usefulness score and characteristics of the expert advisor were investigated using univariate and multivariate analyses.

Results: Expert opinion was seen as a valued complement to empirical evidence, providing context and tacit knowledge unavailable in published literature, but helpful for interpreting it. Interviewees also valued advice on the training and experience required to perform a procedure, on patient selection criteria and the place of a procedure within a clinical management pathway. Limitations of bias in expert opinion were widely acknowledged and skepticism expressed regarding the anecdotal nature of advice on safety or efficacy outcomes. Quantitative analysis demonstrated that the most useful advice was given by clinical experts with direct personal experience of the procedure, particularly research experience.

Conclusions: Evidence-based guidance production is often characterized as a rational, pipeline process. This ignores the valuable role that expert opinion plays in guidance development, complementing and supporting the interpretation of empirical data.

The Austrian healthcare system relies mainly on physicians in private practice and on various services provided by hospitals. The social health insurance scheme is compulsory, covering 99% of the population. The system is very decentralized. While the federal state provides the framework, the nine autonomous provinces are responsible for administering health and social services. There is ongoing public discussion about centralizing the healthcare system to make it more efficient and to enforce structural reforms. Because of concerns about healthcare expenditures, in 1997 the Performance-Related Hospital Financing System (LKF), a system similar to the diagnosis-related group system, was introduced for hospitals, including a plan for large medical devices. It is too early to evaluate the success of this new system, although some effects of the LKF system that could have been anticipated, such as shortened lengths of stay and more hospitalizations, have been seen. Previously, health technologies have been almost uncontrolled in Austria. The evaluation of health technologies as an instrument to support or to control their dissemination and use or to help define policies is not institutionalized or systematically used. It seems clear that structural reforms of the Austrian healthcare system are needed. Health technology assessment should be part of such reforms.

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.

The assessment of value within healthcare is undergoing a major transformation. Gone are the days when a new mechanism of action alone would be regarded as a high value innovation. Today, there is much more of an emphasis on what are the outcomes (i.e., mortality and morbidity benefits), how does this compare with the current standard of care and what is the impact to the usage of healthcare resources. This emphasis is quite understandable given the financial crisis we are experiencing with an ever growing and aging population that is placing considerable strain on the healthcare system.

Target Product Profiles (TPPs) outline the characteristics that new health technologies require to address an unmet clinical need. To date, published TPPs for medical tests have focused on infectious diseases, mostly in the context of low- and middle-income countries. Recently, there have been calls for a broader use of TPPs as a mechanism to ensure that diagnostic innovation is aligned with clinical needs, yet the methodology underpinning TPP development remains suboptimal. Here, we propose that early economic evaluation (EEE) should be integrated within the TPP methodology to create a more rigorous framework for the development of “fit-for-purpose” tests. We discuss the potential benefits that EEE could bring to the core activities underpinning TPP development—scoping, drafting, consensus building, and updating—and argue that using EEE to help inform TPPs provides a more objective, evidence-based, and transparent approach to defining test specifications.

Patient engagement in health technology assessment (HTA) has become increasingly important over the past 20 years. Academic and practitioner literature has produced numerous case studies and best practice accounts of patient involvement practices around the world. This text analyzes the experience of being involved in an Institute for Clinical and Economic Review (ICER) HTA review in the United States. The analysis comes from the joint perspective of three patient organizations: Lupus and Allied Diseases Association, Inc.; Lupus Foundation of America; and Black Women’s Health Imperative, as well as ICER. We suggest that meaningful, patient-centered engagement, where patient communities are systematically integrated throughout the review, can be a way of returning to the discipline’s roots focusing on technologies’ societal and ethical impact. It is a process that requires robust commitment from all involved but produces assessments relevant to those directly affected by them.

We studied the role of economic incentives for private providers in explaining Spain's disproportionately large number of extracorporeal shock wave lithotripters (ESWLs) and low number of linear accelerators (linacs) per million population (pmp) in comparison to other countries of the Organization for Economic Cooperation and Development (OECD). We found that the reimbursement policy for 1990 allowed an average profit per private ESWL of $732,000, but no profit for linacs. Regional per-capita income was positively correlated with ESWLs pmp in Spain (R2 = 0.49; p =.001), but not with linacs. Sixty-nine percent of ESWLs were privately held versus only 16% of linacs. To avoid these types of distortions, financial incentives should be based on a reasonable relationship between cost and charges and should be associated with the appropriateness of medical care.

Objectives: The object of this study was to determine, taking into account uncertainty on cost and outcome parameters, the cost-effectiveness of high-dose chemotherapy (HDC) compared with conventional chemotherapy for advanced breast cancer patients.

Methods: An analysis was conducted for 300 patients included in a randomized clinical trial designed to evaluate the benefits, in terms of disease-free survival and overall survival, of adding a single course of HDC to a four-cycle conventional-dose chemotherapy for breast cancer patients with axillary lymph node invasion. Costs were estimated from a detailed observation of physical quantities consumed, and the Kaplan–Meier method was used to evaluate mean survival times. Incremental cost-effectiveness ratios were evaluated successively considering disease-free survival and overall survival outcomes. Handling of uncertainty consisted in construction of confidence intervals for these ratios, using the truncated Fieller method.

Results: The cost per disease-free life year gained was evaluated at 13,074€, a value that seems to be acceptable to society. However, handling uncertainty shows that the upper bound of the confidence interval is around 38,000€, which is nearly three times higher. Moreover, as no difference was demonstrated in overall survival between treatments, cost-effectiveness analysis, that is a cost minimization, indicated that the intensive treatment is a dominated strategy involving an extra cost of 7,400€, for no added benefit.

Conclusions: Adding a single course of HDC led to a clinical benefit in terms of disease-free survival for an additional cost that seems to be acceptable, considering the point estimate of the ratio. However, handling uncertainty indicates a maximum ratio for which conclusions have to be discussed.

Fueled by high returns on its investments, the pharmaceutical industry in the United States has flourished for the past 50 years. The regulatory strategy of demanding stringent testing then allowing market-based pricing has allowed private companies to fund ambitious research and development activities with the assurance that these investments will be recovered. However, aggressive managed-care cost-containment strategies threaten the companies' ability to recoup research and development expenses and may affect their willingness to invest in future innovative research.