Objectives: Reported is the importance of properly reflecting uncertainty associated with prognostic model estimates when calculating the survival benefit of a treatment or technology, using liver transplantation as an example.

Methods: Monte Carlo simulation techniques were used to account for the uncertainty of prognostic model estimates using the standard errors of the regression coefficients and their correlations. These methods were applied to patients with primary biliary cirrhosis undergoing liver transplantation using a prognostic model from a historic cohort who did not undergo transplantation. The survival gain over 4 years from transplantation was estimated.

Results: Ignoring the uncertainty in the prognostic model, the estimated survival benefit of liver transplantation was 16.7 months (95 percent confidence interval [CI], 13.5 to 20.1), and was statistically significant (p<.001). After adjusting for model uncertainty using the standard errors of the regression coefficients, the estimated survival benefit was 17.5 months (95 percent CI, −3.9 to 38.5) and was no longer statistically significant. An additional adjustment for the correlation between regression coefficients widened the 95 percent confidence interval slightly: the estimated survival benefit was 17.0 months (95 percent CI: −4.6 to 38.6).

Conclusions: It is important that the precision of regression coefficients is available for users of published prognostic models. Ignoring this additional information substantially underestimates uncertainty, which can then impact misleadingly on policy decisions.

For patients who have had the misfortune to develop terminal renal failure, three basic modes of treatment can be offered. One is renal transplantation, with a graft taken from either a living or a cadaveric donor. When successful, it offers the best quality of life and treatment for patients with end-stage kidney disease. However, the elderly and those with severe cardiovascular or systemic complications are not always suitable candidates for renal transplantation (1).

Less developed countries (LDCs) are limited in medical resources. Medical technology and the management talent required to handle it play a particularly major role in their national health care and has significant economic, political, and ethical ramifications. This study of the assessment process of medical technology in the LDCs proposes a limited framework for the analysis of the major parameters involved, i.e., stakeholders, boundaries and constraints, goals and objectives, criteria to be met, performance measures, and measurement of performance. The importance of the intangible factors is elucidated.

Objective: To review the assessment of screening in the United Kingdom, focusing on three methods: mammography for breast cancer, screening for prostate cancer, and routine use of ultrasound in pregnancy.

Method: To review policy documents and published papers dealing with prevention and screening in the United Kingdom.

Results: Indicate that the United Kingdom has an active policy concerning the assessment of screening methods. Generally speaking, this assessment policy is part of the national program for health technology assessment (HTA). The government has given HTA an important place within health care in the United Kingdom, and prevention and screening is no exception to this general rule. The assessment of screening is now implemented through the National Screening Committee, established in 1997. The three issues reviewed in this paper have all been assessed within the context of the Department of Health. In the case of mammography, the assessment was done more than 10 years ago and was followed by a rational implementation of a national screening program for breast cancer. In the case of prostate cancer screening, two systematic reviews have concluded that screening should not be carried out. In general, this recommendation has been accepted in the United Kingdom. Use of ultrasound in pregnancy has been assessed by the National Screening Committee. This complex technology is difficult to assess, and the screening procedure is deeply embedded in clinical practice in the United Kingdom, so assessment has not had much impact on the frequency of screening.

Conclusion: HTA and the assessment of screening are well established in the United Kingdom. Policy is generally based on the assessments done, and practice generally follows the results of assessment. Assessment of screening is expected to become increasingly important in the United Kingdom during the next years.

Objectives: The aim of this study was to evaluate the success of search strategies in retrieving key documents for a technology assessment report (TAR) on a social care topic.

Methods: This study measured the differential yield of relevant studies from various information sources and evaluated strategies in different databases, with particular reference to capturing randomized controlled trials (RCTs) as a study design.

Results: A combination of four major databases would have found all thirty-two key references. One database alone would have found 78 percent, with another two each locating 59 percent. Sixteen percent of the trials were unique references. In non–health care databases, more sensitive search strategies would have resulted in a higher yield of relevant studies, in part due to inconsistent indexing and in part to attempts to restrict searches to RCTs. Although additional terms could be used to increase the sensitivity of the original strategies, this raises the question of trading off time against exhaustiveness, given the greater number of irrelevant references likely to be retrieved.

Conclusions: A successful search for evidence on this social care topic would be possible using a combination of MEDLINE, EMBASE, the Cochrane Library and PsycINFO, supplemented by only limited use of supplementary databases. In areas such as social care where evidence-based research is not yet well established, attempts to replicate searches based on study design do not seem to be advisable, although this may be an area for future research.

Objectives: To describe the adoption and take up of thrombolytic agents for acute myocardial infarction since 1980 in England and compare use with the estimated ceiling of need.

Methods: Data on national sales and use of thrombolysis since 1980 (supplied by IMS Health) was used to draw an adoption and diffusion curve. The epidemiological ceiling of acute myocardial infarction, from hospital activity statistics, was modified to an estimated clinical need by accounting for diagnostic difficulty and contraindications using information from published surveys of thrombolysis use in the United Kingdom.

Results: There was a rapid uptake of thrombolytic agents in the first 2 years after availability in 1987, then a plateau, followed by a rise to a peak use in 1995. The shortfall in doses resulting from the difference between estimated ceiling of clinical need and doses purchased and provided in the 14 years since availability is estimated as 167,800 (95 percent confidence range 94,000 to 241,700).

Conclusions: Although there was a rapid initial uptake of thrombolysis in England, usage took 8 years to reach the ceiling of clinical need of 65 percent of patients with acute myocardial infarction, with many patients missing the opportunity to benefit. Monitoring of uptake of innovations known to be cost-effective is required to identify those developments that need additional stimulus for change to ensure that patients do not miss out on the opportunity to benefit.

In A Theory of Justice, John Rawls demands from citizens who decide upon principles of justice and the rules derived from such principles that they abstract from all particularities that constitute their identity as unique individuals. This demand is unrealistic in policy settings where actual policy-makers convene to provide guidance, establish rules regarding public good, and enact legislation. In practice, I argue, policy-makers, legislators, and others involved in developing social rules that pertain to distributive justice formulate such rules as reasonably partial spectators. To illustrate, I show how clinical practice guidelines are established and mediated by a reasonably partial expert panel whose partial action is publicly justifiable, yet whose claims to impartiality are not.

The delivery of rehabilitation services requires an infrastructure if service provision is to be given any priority. This article discusses some characteristics of handicaps, technical aids, the service delivery process, and habilitation/rehabilitation, and recommends developments of national structures for service delivery based upon the characteristics of rehabilitation technology, user need, and the country frameworks.

Objectives: An example of technology assessment in dental care by evaluating the (cost-)effectiveness of types of three-surface inlays (gold, laboratory-fabricated ceramic, and chairside CAD/CAM ceramic) is provided.

Methods: MEDLINE, EMBASE, and the Cochrane Library were searched for studies published between 1966 and June 2003 that reported annual survival probabilities and annual observations. The longevity of different types of inlays was measured by the number of failure-free years. Annual survival rates from different studies were pooled by weighing the rates of each study by the inverse of the variance of the effect estimate. A cost-effectiveness analysis from the perspective of German private health insurers was performed using billing charges.

Results: Three, five, and two case series on laboratory-fabricated ceramic, chairside CAD/CAM ceramic, and gold inlays, respectively, were included. Over a 9-year observation period, the number of undiscounted failure-free years was 8.62 (95 percent confidence interval, 8.40–8.85), 8.65 (8.58–8.73), and 8.76 (8.72–8.80) for laboratory-fabricated ceramic, chairside CAD/CAM ceramic, and gold inlays, respectively. Laboratory-fabricated ceramic inlays were the most expensive.

Conclusions: While laboratory-fabricated ceramic, chairside CAD/CAM ceramic, and gold inlays had a strikingly similar failure-free survival rate, laboratory-fabricated ceramic inlays had the highest costs and, thus, were less cost-effective than chairside CAD/CAM ceramic and gold inlays.

The launch of innovative technologies has been credited with significant improvements in health indicators, but it comes at a high financial impact, and the value of certain innovations may not be well documented. Health technology assessment (HTA) is a universally established process to assess the incremental value of innovations. Despite its acknowledged value, almost one-third of the countries around the globe have not established yet a formal HTA in their health systems. The UAE is one of the pioneering countries worldwide in adopting innovative health technologies. This emphasizes the importance of exploring the key elements in the UAE’s journey toward the establishment of HTA. Our study aims to articulate an academic insight that can support the ongoing endeavors to establish the HTA in the UAE. This case study was guided by an analytical framework. Data was collected from document review and semistructured interviews, then analyzed by applying the codebook thematic analysis technique. The findings outline multiple facilitators and challenges in the perspective process, as they show a multidimensional interlink between all identified elements. Markedly, leveraging the role of specialized academia and building HTA genuine knowledge are the areas that need the most attention. The originality of this research is associated with analyzing the three health policy pillars: the context, actors, and content in a prospective HTA establishment process. The main practical implications generated from this study are supporting global health organizations, HTA policy entrepreneurs, and academics in improving their strategies and designing more effective HTA policy learning programs.

Current thinking about the methodology of health technology assessment (HTA) seems to be dominated by two fundamental tensions: [1] between maintaining a tight focus on quality-adjusted life-years and broadening its concern out to pay attention to a broader range of factors, and [2] between thinking of the evaluative dimensions that matter as being objectively important factors or as ones that are ultimately of merely subjective importance. In this study, I will argue that health is a tremendously important all-purpose means to enjoying basic human capabilities, but a mere means, and not an end. The ends to which health is a means are manifold, requiring all those engaged in policy making to exercise intelligence in a continuing effort to identify them and to think through how they interrelate. Retreating to the subjective here would be at odds with the basic idea of HTA, which is to focus on certain objectively describable dimensions of what matters about health and to collect empirical evidence rigorously bearing on what produces improvements along those dimensions. To proceed intelligently in doing HTA, it is important to stay open to reframing and refashioning the ends we take to apply to that arena. The only way for that to happen, as an exercise of public, democratic policy making, is for the difficult value questions that arise when ends clash not to be buried in subjective preference information, but to be front-and-center in the analysis.

Objectives: Kids in Control OF Food (KICk-OFF) is a 5-day structured education program for 11- to 16-year-olds with type 1 diabetes mellitus (T1DM) who are using multiple daily insulin injections. This study evaluates the cost-effectiveness of the KICk-OFF education program compared with the usual care using data from the KICk-OFF trial.

Methods: The short-term within-trial analysis covers the 2-year postintervention period. Data on glycated hemoglobin (HbA1c), severe hypoglycemia, and diabetic ketoacidosis (DKA) were collected over a 2-year follow-up period. Sub-group analyses have been defined on the basis of baseline HbA1c being below 7.5 percent (58.5 mmol/mol) (low group), between 7.5 percent and 9.5 percent (80.3 mmol/mol) (medium group), and over 9.5 percent (high group). The long-term cost-effectiveness evaluation has been conducted by using The Sheffield Type 1 Diabetes Policy Model, which is a patient-level simulation model on T1DM. It includes long-term microvascular (retinopathy, neuropathy, and nephropathy) and macrovascular (myocardial infarction, stroke, revascularization, and angina) diabetes-related complications and acute adverse events (severe hypoglycemia and DKA).

Results: The most favorable within-trial scenario for the KICk-OFF arm led to an incremental cost-effectiveness ratio (ICER) of £23,688 (base year 2009) with a cost-effectiveness probability of 41.3 percent. Simulating the long-term complications using the full cohort data, the mean ICER for the base case was £28,813 (base year 2011) and the probability of the KICk-OFF intervention being cost-effective at £20,000/QALY threshold was 42.6 percent, with considerable variation due to treatment effect duration. For the high HbA1c sub-group, the KICk-OFF arm was “dominant” (meaning it provided better health gains at lower costs than usual care) over the usual care arm in each scenario considered.

Conclusions: For the whole study population, the cost-effectiveness of KICk-OFF depends on the assumption for treatment effect duration. For the high baseline HbA1c sub-group, KICk-OFF arm was estimated to be dominant over the usual care arm regardless of the assumption on the treatment effect duration.

The number of publications on health technology assessment (HTA) from Central, Eastern, and Southern Europe (CESE countries) is still low compared with the north and west of Europe. It is not surprising, as the idea of HTA originated from high-income Western economies and was afterward adopted by the south-eastern part of Europe, which mostly consists of middle-income countries. These CESE countries, with less capacity and experience with HTA processes, must deal with even tougher decisions on financing health technologies than north-western Europe. There may even be a lack of confidence to open discussions on their specific needs for HTA.

Ethiopia’s commitment to achieving universal health coverage (UHC) requires an efficient and equitable health priority-setting practice. The Ministry of Health aims to institutionalize health technology assessment (HTA) to support evidence-based decision making. This commentary highlights key considerations for successful formulation, adoption, and implementation of HTA policies and practices in Ethiopia, based on a review of international evidence and published normative principles and guidelines. Stakeholder engagement, transparent policymaking, sustainable financing, workforce education, and political economy analysis and power dynamics are critical factors that need to be considered when developing a national HTA roadmap and implementation strategy. To ensure ownership and sustainability of HTA, effective stakeholder engagement and transparency are crucial. Regulatory embedding and sustainable financing ensure legitimacy and continuity of HTA production, and workforce education and training are essential for conducting and interpreting HTA. Political economy analysis helps identify opportunities and constraints for effective HTA implementation. By addressing these considerations, Ethiopia can establish a well-designed HTA system to inform evidence-based and equitable resource allocation toward achieving UHC and improving health outcomes.