A range of fecal occult blood tests are presently on the market and could potentially be used in population screening programs for the detection of colorectal neoplasms. This paper estimates the relative cost-effectiveness of alternative tests and concludes that the unhydrated Hemoccult II is the most cost-effective. However, the incremental costs per life-year of the HemeSelect test and the rehydrated Hemoccult II test are in line with incremental costs observed in breast cancer and cervical cancer programs.

The aim of this paper is to review the concept of patient-based evidence in health technology assessment (HTA), drawing on philosophical ideas of knowledge in order to judge whether current approaches to the use of evidence for HTA are complete. We draw on a number of key sources, including key papers and book chapters, discussion forums, agency reports, and conference presentations. We develop the potential dimensions of patient-based evidence, describe its key attributes, and consider its future development. Patient-based evidence has the potential to be a key concept in HTA, comprised of a series of related elements of importance to patients. We recognize that we raise more questions than can be answered, but as an emerging concept, recognition and understanding of patient-based evidence is still developing. The concepts and methods that support its application in HTA require urgent development. We conclude that clinical and economic forms of evidence are not enough for HTA. For HTA to be complete, we need to consider all relevant aspects of the phenomena, including patient-based evidence. There is now an urgent need for the global research and HTA community to work together to realize the full potential of patient-based evidence through conceptual and methodological development and wider recognition. We advocate that a task force be set up to address these urgent issues.

Healthcare systems account for a substantial proportion of global carbon emissions and contribute to wider environmental degradation. This scoping review aimed to summarize the evidence currently available on incorporation of environmental and sustainability considerations into health technology assessments (HTAs) and guidelines to support the National In stitute for Health and Care Excellence and analogous bodies in other jurisdictions developing theirown methods and processes. Overall, 7,653 articles were identified, of which 24 were included in this review and split into three key areas – HTA (10 studies), healthcare guidelines (4 studies), and food and dietary guidelines (10 studies). Methodological reviews discussed the pros and cons of different approaches to integrate environmental considerations into HTAs, including adjustments to conventional cost-utility analysis (CUA), cost–benefit analysis, and multicriteria decision analysis. The case studies illustrated the challenges of putting this into practice, such as lack of disaggregated data to evaluate the impact of single technologies and difficulty in conducting thorough life cycle assessments that consider the full environmental effects. Evidence was scant on the incorporation of environmental impacts in clinical practice and public health guidelines. Food and dietary guidelines used adapted CUA based on life cycle assessments, simulation modeling, and qualitative judgments made by expert panels. There is uncertainty on how HTA and guideline committees will handle trade-offs between health and environment, especially when balancing environmental harms that fall largely on society with health benefits for individuals. Further research is warranted to enable integration of environmental considerations into HTA and clinical and public health guidelines.

Objectives: The aim of this study was to demonstrate how value of information analysis can measure the upper limit on returns to future research and identify the research priorities for computer-assisted total knee replacement (CAS-TKR).

Methods: Using a previous economic analysis of CAS-TKR compared with conventional TKR, the population expected value of perfect information (EVPI) was calculated using Monte Carlo simulation to provide an estimate of the upper limit on returns to future research. The population expected value of partial perfect information (EVPPI) for both individual parameters and groups of parameters was estimated to inform specific future research priorities.

Results: The UK individual EVPI would be £21.4 if the willingness to pay for one QALY (quality-adjusted life-year) were £30,000. The population EVPPI would be £8.3 million, assuming a 10-year time horizon for CAS-TKR. In this instance, the expected value of information is positively related to willingness to pay for one QALY for the range of £0 to £50,000. Although each individual parameter had an EVPPI of £0, groups of utility parameters had positive EVPPI. Population EVPPI was £5.6 million for utility parameters, £20,000 for transition probabilities relating to CAS-TKR, and £5,000 for transition probabilities related to conventional TKR.

Conclusions: The study provides evidence on which parameters further information may be of most value. Focusing research on the utility values associated with health states relating to TKR would be of greatest value.

Objectives: In April 1998, the Medical Services Advisory Committee (MSAC) was established by the Australian federal government. Since that time, all new medical procedures must be evaluated for safety, effectiveness, and cost-effectiveness as a condition of the surgeon receiving public funding by means of the Medicare Benefits Schedule (MBS). Over these first 8 years, a significant number of applications for the public funding of new procedures have been given negative recommendations by the MSAC based on insufficient clinical evidence or lack of cost-effectiveness. In August 2006, after almost 2 years of processing, the MSAC made the decision to fund the new procedure, laparoscopic remotely assisted radical prostatectomy (LRARP). However, they stated that there was still uncertainty about the comparative cost-effectiveness.

Methods: An observational study using provisional cost-utility data for LRARP based on a combination of costs taken from consecutive patients at the Epworth Hospital, Melbourne, Australia, and utilities from the prospectively collected data on all patients undergoing surgery for prostate cancer over a 4-year period at the Vattikuti Urology Institute, Michigan, United States.

Results: The incremental cost for LRARP compared with the open surgery alternative is A$2,264 or A$24,457 per quality-adjusted life-year, well below the range accepted by the Australian pharmaceutical equivalent of the MSAC (the PBAC) of A$42,000 and A$76,000. This figure does not take into account additional benefits such as reduced time away from employment, reduced blood loss, reduced possibility of infection, and reduced scarring.

Conclusions: This case study of LRARP demonstrates that there is sufficient crude evidence to show that this new procedure is likely to be superior to the existing procedure in terms of safety, effectiveness, and cost-effectiveness. The decision to allow MBS funding was correct and will allow for the collection of additional evidence, on both economic and clinical outcomes.

Objectives: To improve processes of ambulatory care for patients with type 2 diabetes in a nationwide program.

Methods: Interrupted time-series analysis with audits of practice. To implement selected recommendations of national guidelines, educational outreach visits (office visits or phone discussions) were offered to all French physicians who diagnosed one case of type 2 diabetes during a six-month intervention period. Outcome measures were the number of HBA1c measurements recorded monthly in the medical insurance computer database and the proportion of diabetic patients for whom one test had been reimbursed during the previous six months (HBA1c, fasting blood glucose) or previous twelve months (serum cholesterol, serum creatinine, urine microalbumin, electrocardiogram, ophthalmologic examination).

Results: A total of 15,522 office visits and 9,062 telephone discussions were performed among 22,940 physicians. The increase in the monthly proportion of the number of HBA1c tests to the total number of laboratory tests was higher during the intervention period than during the preintervention (p value<.0001) and postintervention periods (p value<.001). Between the first audit (n=651,574) and the third audit (n=911,871), HBA1c measurements increased from 41.2% to 60.5% and blood glucose measurements performed alone decreased from 38.8% to 18.7%. Urine microalbumin measurements increased from 10.6% before to 15.3% after intervention. Only a slight increase was observed for other tests.

Conclusions: Physician to physician outreach visits can be an effective way to improve the processes of care for diabetes and to routinize nationwide use of practice guidelines.

Including information and values from patients in HTA has the potential to improve both the process and outcomes of health technology policy decisions. Accordingly, funding and structural incentives to include patients in HTA activities have increased over the past several years. Unfortunately, these incentives have not yet been accompanied by a corresponding increase in resources, time, or commitment to responsiveness. In this Perspectives piece, we reflect on our collective experiences participating in, conducting, and overseeing patient engagement activities within HTA to highlight the ethical challenges associated with this area of activity. While we remain committed to the idea that patient engagement activities strengthen the findings, relevance, and legitimacy of health technology policy, we are deeply concerned about the potential for these activities to do ethical harm. We use this analysis to call for action to introduce strong protections against ethical violations that may harm patients participating in HTA engagement activities.

Objective: The aim of this study was to present a clear process of synthesizing test accuracy data when conducting economic evaluations of diagnostic tests for health technology assessment (HTA) assessors and health economists.

Methods: We appraised the methods advocated for using diagnostic test accuracy data in economic evaluations. We used a case study of fetal anemia in which data from a screening test are used in combination with a confirmatory test.

Results: We developed a step-by-step guide and consider two scenarios: when data on test accuracy from several studies are based on (i) the same test threshold for positivity and (ii) different test thresholds.

Conclusions: We conclude that each approach has its strengths and limitations. We show that the optimal operating point of the test should be identified to determine the true cost-effectiveness of the test. We advocate that these issues require a multidisciplinary team of health economists, decision modelers and statisticians.

Objective: Economic evaluations such as cost-effectiveness and cost-utility analyses generally fail to incorporate elements of intangible costs and benefits, such as anxiety and discomfort associated with the screening test and diagnostic test, as well as the magnitude of utility associated with a reduction in the risk of dying from cancer. This paper seeks to include all costs and effects incurred by introducing mammography screening through the application of discrete ranking modeling.

Methods: In the present analysis, 207 women were interviewed and asked to rank, according to priority, a number of alternative breast cancer screening setups. The alternative programs varied with respect to number of tests performed, risk reduction obtained, probability of a false-positive outcome, and extent of copayment. Using discrete ranking modeling, the stated preferences were analyzed and the relative weighting of the program attributes identified. For a range of hypothetical breast cancer programs, relative utilities and corresponding willingness-to-pay estimates were derived.

Results: A comparison of cost and willingness to pay for each of the programs suggested that net benefits are maximized when screening person aged 50–74 years biennially. More intensive screening produces lower or similar levels of utility at a higher cost.

Conclusion: Discrete ranking modeling can aid decision making by identifying inferior healthcare programs, i.e., programs that are more costly but less beneficial.

Objectives: There are very few descriptions on how familial hypercholesterolemia (FH), a genetic disorder with an increased risk of coronary heart disease (CHD), affects older patients. The aim of this study was to analyze the health-related quality of life (HRQoL) of elderly patients with FH.

Methods: All FH patients with the same FH North Karelia-mutation aged 65 and over living in North Karelia province in Eastern Finland were asked to participate in the study in 2003. They were compared with age-standardized controls from the Finnish general population. A total of forty-three elderly FH patients were identified and thirty-seven of them (aged 65 to 84 years, M/F 10/27) agreed to participate. Most of them (27/37) had been diagnosed with CHD. All but one of these FH patients had been using statins for approximately 15 years. HRQoL was assessed with the RAND-36 (SF-36) and 15D instruments.

Results: Despite the clear cardiovascular morbidity and low socioeconomic status, these FH patients enjoyed a similar HRQoL as the age-standardized controls in the general population.

Conclusions: Lifelong hypercholesterolemia was not associated with poorer HRQoL in elderly survivors of FH. These elderly FH individuals were characterized by their healthy lifestyle and long-term statin treatment.

Introduction: The supplementation of alanyl-glutamine dipeptide in critically ill patients necessitating total parenteral nutrition (TPN) improves clinical outcomes, reducing mortality, infection rate, and shortening intensive care unit (ICU) hospital lengths of stay (LOSs), as compared to standard TPN regimens.

Methods: A Discrete Event Simulation model that incorporates outcomes rates from 200 Italian ICUs for over 60,000 patients, alanyl-glutamine dipeptide efficacy data synthesized by means of a Bayesian random effects meta-analysis, and national cost data has been developed to evaluate the alternatives from the cost perspective of the hospital. Simulated clinical outcomes are death and infection rates in ICU, death rate in general ward, and hospital LOSs. Sensitivity analyses are performed by varying all uncertain parameter values in a plausible range.

Results: The internal validation process confirmed the accuracy of the model in replicating observed clinical data. Alanyl-glutamine dipeptide on average results more effective and less costly than standard TPN: reduced mortality rate (24.6% ± 1.6% vs. 34.5% ± 2.1%), infection rate (13.8% ± 2.9% vs. 18.8% ± 3.9%), and hospital LOS (24.9 ± 0.3 vs. 26.0 ± 0.3 days) come at a lower total cost per patient (23,409 ± 3,345 vs. 24,161 ± 3,523 Euro).Treatment cost is completely offset by savings on ICU and antibiotic costs. Sensitivity analyses confirmed the robustness of these results.

Conclusions: Alanyl-glutamine dipeptide is expected to improve clinical outcomes and to do so with a concurrent saving for the Italian hospital.

Health systems are placing more and more emphasis on designing and delivering services that are focused on the patient, and there is a growing interest in patient aspects of health policy research and health technology assessment (HTA). Only a few HTA agencies use and invest in scientific methods to generate knowledge and evidence about the patient aspects of a given technology. This raises questions about how knowledge is produced in HTA reports and what kind of knowledge is considered relevant. This article uses a Danish HTA on patient education from 2009 as empirical material for a critical examination and discussion of knowledge and knowledge production about the patient aspects of HTA.

We conducted a study to acquire information on the current behavior of a sample of Italian surgeons and anesthesiologists about prescribing, interpreting, and using routine preoperative investigations. Consultants in surgery and anesthesiology in 60 hospitals in northern, central, and southern Italy were interviewed. Prescription of these procedures by doctors were driven more by personal experience than by updated scientific knowledge. This practice often led to ineffective and inefficient clinical practice, with healthy patients undergoing useless, time-consuming, costly, and sometimes harmful procedures.

That new health technologies often diffuse into the health care systems of developed countries without adequate evaluation has long been a cause of concern (I). In addition, where clinical or economic assessments have been carried out, they often contain methodological weaknesses which reduce their usefulness to health policy makers or clinical practitioners (5,6).

Objectives: The University of Minnesota has maintained a home monitoring program for over 10 years for lung and heart–lung transplant patients. A cost analysis was completed to assess the impact of home monitoring on the cost of post-transplant medical care.

Methods: Clinical information gathered with the monitoring system includes spirometry, vital signs, and symptom data. To estimate the impact of this system on medical costs, we completed a retrospective analysis of the effects of home monitoring on the cost of post-lung transplant medical care. The cost analysis used multivariate linear regression with inpatient, outpatient, and total medical care costs as the dependent variables. The independent variables for the regression include home monitoring adherence, underlying disease, ambulatory diagnostic group mapping variables, transplant type, and patient demographics.

Results: The multivariate regression of the overall cost results predicts a 52.4 percent reduction in total costs with 100 percent patient adherence; this rate includes a 72.24 percent reduction in inpatient costs and a 46.6 percent increase in outpatient costs. The actual first year average patient adherence was 74 percent.

Conclusions: Adherence to home monitoring increases outpatient costs and reduces inpatient costs and provides an overall cost savings. The break-even point for patient adherence was 25.28 percent, where the net savings covered the cost of home monitoring. This is well within the actual first year adherence rates (74 percent) for subjects in the lung transplant home monitoring program, providing a net savings with adherence to home monitoring.

This study provides a sociological commentary on the current debates within health technology assessment (HTA), specifically in response to the approaches taken in France, The Netherlands, Sweden, and the United Kingdom. It argues that HTA is part of a wider reflexive innovation system that seeks to order current and prospective technologies. The study discusses the socio-political process of HTA priority setting, the rhetorical role of HTA, the localised and contingent use of HTA, and the policy gap between guidelines and practice. It argues for the development of new types of methodologies for assessment and for a stronger social embedding of HTA practice.

Objectives: The aim of the study was to investigate the feasibility and effectiveness of searching selected databases to identify information required to populate a decision-analytic model.

Methods: Methods of searching for information to populate a decision-analytic model were piloted using a case study of prophylactic antibiotics to prevent recurrent urinary tract infections in children. This study explored how the information requirements for a decision-analytic model could be developed into searchable questions and how search strategies could be derived to answer these questions. The study also assessed the usefulness of three published search filters and explored which resources might produce relevant information for the various model parameters.

Results: Based on the data requirements for this case study, 42 questions were developed for searching. These questions related to baseline event rates, health-related quality of life and outcomes, relative treatment effects, resource use and unit costs, and antibiotic resistance. A total of 1,237 records were assessed by the modeler, and of these, 48 were found to be relevant to the model. Search precision ranged from 0 percent to 38 percent, and no single database proved the most useful for all the questions.

Conclusions: The process of conducting specific searches to address each of the model questions provided information that was useful in populating the case study model. The most appropriate resources to search were dependent on the question, and multiple database searching using focused search strategies may prove more effective in finding relevant data than thorough searches of a single database.