Search

Background: Several aspects of the opioid epidemic and of public health care organization in the United States (US) make the conduct of economic evaluation and the design of policies to respond to this crisis particularly challenging. Objectives: This commentary offers suggestions for how economic evaluation may address and overcome four key features of the opioid epidemic: 1) its magnitude and geographical distribution, 2) its intersection with multiple epidemics, 3) its rapidly changing dynamics, 4) its multi-sectoral causes and consequences. Results: We first offer pragmatic suggestions to address the difficulties in delivering a coordinated response given the fragmented nature of health care in the US. In view of the broad suite of responses required to address opioid use disorder and its associated comorbidities, we highlight the need for economic evaluations which consider interventions throughout the continuum of care (i.e. primary, secondary and tertiary levels of prevention). We examine how the use of predictive modelling alongside economic evaluation might be adopted to address the rapidly evolving situation affecting distinct populations and geographic areas and encourage investments in epidemic preparedness. Finally, we propose methods to capture the interdependence of various sectors of government affected by the opioid crisis in economic evaluations to ensure optimal levels of investment towards a comprehensive response. Conclusions: The opioid epidemic in the US represents an unprecedented public health challenge, but sound epidemiological modelling and economic analysis can help to guide use of limited resources committed to addressing it in ways that can have greatest impact in limiting its adverse consequences.

Objective A ‘lite’ version of the EQ-5D-5L valuation protocol, which requires a smaller sample by collecting more data from each participant, was proposed and used to develop an EQ-5D-5L value set for Uganda. Methods Adult respondents from the general Ugandan population were quota sampled based on age and sex. Eligible participants were asked to complete 20 composite time trade-off tasks in the tablet-assisted personal interviews using the offline EuroQol Portable Valuation Technology software under routine quality control. No discrete choice experiment task was administered. The composite time trade-off data were modelled using four additive and two multiplicative regression models. Model performance was evaluated based on face validity, prediction accuracy in cross-validation and in predicting mild health states. The final value set was generated using the best-performing model. Results A representative sample (N = 545) participated in this study. Responses to composite time trade-off tasks from 492 participants were included in the primary analysis. All models showed face validity and generated comparable prediction accuracy. The Tobit model with constrained intercepts and corrected for heteroscedasticity was considered the preferred model for the value set on the basis of better performance. The value set ranges from − 1.116 (state 55555) to 1 (state 11111) with ‘pain/discomfort’ as the most important dimension. Conclusions This is the first EQ-5D-5L valuation study using a ‘lite’ protocol involving composite time trade-off data only. Our results suggest its feasibility in resource-constrained settings. The established EQ-5D-5L value set for Uganda is expected to be used for economic evaluations and decision making in Uganda and the East Africa region.

Background Prolonged acute hospital stays are a major problem for older people and for health services. Failure to effectively manage the psychological and social aspects of illness is an important cause of prolonged hospital stays. Proactive Psychological Medicine (PPM) is a new way of providing psychiatry services to medical wards. PPM is proactive, focussed, intensive and integrated with medical care. A major aim of PPM is to reduce the time older people spend in hospital because of unmanaged psychological and social problems. The HOME Study will test the effectiveness and cost-effectiveness of PPM. Methods/design A two-arm parallel-group randomised controlled superiority trial, with a linked health economic analysis and an embedded process evaluation, will be conducted at three sites. A total of 3588 participants will be recruited and randomised to usual care or usual care plus PPM. The primary outcome is the number of days spent as an inpatient in a general hospital in the month (30 days) post-randomisation. Secondary outcomes for each participant (measured at 1 and 3 months) include quality of life, independent functioning, symptoms of anxiety and depression, cognitive function, and their experience of the hospital stay. Discussion The trial has been designed to produce findings that are generalisable to all older medical inpatients (including those with cognitive impairment). It will provide information on the effectiveness and cost-effectiveness of PPM, which we hope will be of value to patients, clinicians, managers and service planners. Trial registration ISRCTN86120296. Registered on 3 January 2018. Electronic supplementary material The online version of this article (10.1186/s13063-019-3502-5) contains supplementary material, which is available to authorized users.

In most societies, resources are distributed by individuals acting in markets and by governments through some form of collective decision-making process. Economic evaluation offers a set of tools to inform collective decisions by examining the resource requirements and outcomes of alternative policies. The ‘societal perspective’ has been advocated, but less consideration has been given to what this should include and its practical implementation. This paper presents a framework for economic evaluation of policies with costs and outcomes falling on different sectors (e.g. health, criminal justice, education) and involving different decision makers. It extends the ‘impact inventory’ developed by the Second Panel on Cost-Effectiveness in Health and Medicine by considering all affected individuals and reflecting how outcomes attributed to an intervention can be compared with outcomes forgone as a result of resources not being available for other purposes. The framework sets out the series of assessments to be made, distinguishing points at which value judgements feed into the evaluation, and the implications of alternative judgements. These assessments reflect the institutional arrangements of public bodies, for example, their funding, the outcomes they consider important and their relative valuations of these outcomes. By avoiding the use of an abstract ‘societal perspective’, the contribution of the framework is to inform multiple decision makers with different objectives and provide practical guidance on overall societal impact. Electronic supplementary material The online version of this article (10.1007/s40258-019-00481-8) contains supplementary material, which is available to authorized users.

Background Results from large randomised controlled trials have shown that adding docetaxel to the standard of care (SOC) for men initiating hormone therapy for prostate cancer (PC) prolongs survival for those with metastatic disease and prolongs failure-free survival for those without. To date there has been no formal assessment of whether funding docetaxel in this setting represents an appropriate use of UK National Health Service (NHS) resources. Objective To assess whether administering docetaxel to men with PC starting long-term hormone therapy is cost-effective in a UK setting. Design, setting, and participants We modelled health outcomes and costs in the UK NHS using data collected within the STAMPEDE trial, which enrolled men with high-risk, locally advanced metastatic or recurrent PC starting first-line hormone therapy. Intervention SOC was hormone therapy for ≥2 yr and radiotherapy in some patients. Docetaxel (75 mg/m2) was administered alongside SOC for six three-weekly cycles. Outcome measurements and statistical analysis The model generated lifetime predictions of costs, changes in survival duration, quality-adjusted life years (QALYs), and incremental cost-effectiveness ratios (ICERs). Results and limitations The model predicted that docetaxel would extend survival (discounted quality-adjusted survival) by 0.89 yr (0.51) for metastatic PC and 0.78 yr (0.39) for nonmetastatic PC, and would be cost-effective in metastatic PC (ICER £5514/QALY vs SOC) and nonmetastatic PC (higher QALYs, lower costs vs SOC). Docetaxel remained cost-effective in nonmetastatic PC when the assumption of no survival advantage was modelled. Conclusions Docetaxel is cost-effective among patients with nonmetastatic and metastatic PC in a UK setting. Clinicians should consider whether the evidence is now sufficiently compelling to support docetaxel use in patients with nonmetastatic PC, as the opportunity to offer docetaxel at hormone therapy initiation will be missed for some patients by the time more mature survival data are available. Patient summary Starting docetaxel chemotherapy alongside hormone therapy represents a good use of UK National Health Service resources for patients with prostate cancer that is high risk or has spread to other parts of the body., Take Home Message Docetaxel is a cost-effective intervention in patients with metastatic and nonmetastatic prostate cancer starting hormone therapy. Clinicians and National Health Service funding bodies should consider whether the evidence is now sufficiently compelling to support initiating docetaxel in patients with nonmetastatic disease.

Background Prolonged acute hospital stays are a problem for older people and for health services. Failure to effectively manage the psychological and social aspects of illness is an important cause of prolonged hospital stay. Proactive Psychological Medicine (PPM) is a new way of providing psychiatry services to medical wards which is proactive, focussed, intensive and integrated with medical care. The primary aim of PPM is to reduce the time older people spend in hospital because of unmanaged psychological and social problems. The HOME Study will test the effectiveness and cost-effectiveness of PPM. Methods/design The study is a two-arm, parallel-group, randomised, controlled superiority trial with linked health economic analysis and an embedded process evaluation. The target population is people aged 65 years and older admitted to acute hospitals. Participants will be randomly allocated to either usual care plus PPM or usual care alone. The primary outcome is the number of days spent as an inpatient in a general hospital in the month following randomisation. Secondary outcomes include quality of life, cognitive function, independent functioning, symptoms of anxiety and depression, and experience of hospital stay. The cost-effectiveness of usual care plus PPM compared with usual care alone will be assessed using quality-adjusted life-years as an outcome as well as costs from the NHS perspective. Discussion This update to the published trial protocol gives a detailed plan of the statistical and economic analysis of The HOME Study. Trial registration ISRCTN registry, ISRCTN86120296. Registered on 3 January 2018.

The United States is one of the few high-income countries not to apply economic evaluation routinely to health care decision making on a national level, yet it excels at spending least efficiently on health care. In the interest of continuing to develop new solutions to curb spending on health care and reduce waste in the United States, perhaps now is an important moment to reconsider the benefits of economic evaluation and the barriers that must be overcome to have it emerge as a solution for health care institutions and the patients they serve. This article offers several distinct considerations to make economic evaluation methods (such as cost-effectiveness analysis) an effective component of value-based decision making in the United States. These considerations include overcoming the barriers presented by opportunity costs, spending on health care services versus biomedical technologies, phasing out low-value care, using value of information to prioritize resources, and determining what to do with the quality-adjusted life-year. These issues need to be addressed to achieve a collective purpose for economic evaluation at state and national levels.

ObjectiveTo assess the cost-effectiveness of management strategies for patients presenting with chest pain and suspected coronary heart disease (CHD): (1) cardiovascular magnetic resonance (CMR); (2) myocardial perfusion scintigraphy (MPS); and (3) UK National Institute for Health and Care Excellence (NICE) guideline-guided care.MethodsUsing UK data for 1202 patients from the Clinical Evaluation of Magnetic Resonance Imaging in Coronary Heart Disease 2 trial, we conducted an economic evaluation to assess the cost-effectiveness of CMR, MPS and NICE guidelines. Health outcomes were expressed as quality-adjusted life-years (QALYs), and costs reflected UK pound sterling in 2016–2017. Cost-effectiveness results were presented as incremental cost-effectiveness ratios and incremental net health benefits overall and for low, medium and high pretest likelihood of CHD subgroups.ResultsCMR had the highest estimated QALY gain overall (2.21 (95% credible interval 2.15, 2.26) compared with 2.07 (1.92, 2.20) for NICE and 2.11 (2.01, 2.22) for MPS) and incurred comparable costs (overall £1625 (£1431, £1824) compared with £1753 (£1473, £2032) for NICE and £1768 (£1572, £1989) for MPS). Overall, CMR was the cost-effective strategy, being the dominant strategy (more effective, less costly) with incremental net health benefits per patient of 0.146 QALYs (−0.18, 0.406) compared with NICE guidelines at a cost-effectiveness threshold of £15 000 per QALY (93% probability of cost-effectiveness). Results were similar in the pretest likelihood subgroups.ConclusionsCMR-guided care is cost-effective overall and across all pretest likelihood subgroups, compared with MPS and NICE guidelines.

Alternative strategies can reduce road vehicle emissions, with differential effects on exposure across population groups. We compare alternative strategies in West Yorkshire using a framework for economic evaluation that considers multiple perspectives and that takes account of the distribution of health outcomes. Exposure to pollutants by area is converted, via dose response relationships, into disease averted. Health benefits and National Health Service costs from diseases are estimated conditional on population demographics and index of multiple deprivation. The net health benefits from alternative strategies are expressed as distributions of quality-adjusted life expectancy (QALE), which are compared using dominance criteria and societal aversion to health inequality. Net production is estimated from intervention costs and the effects of health improvement on production and consumption. Social care outcomes are estimated from health improvement among care recipients and changes in care expenditure. A switch to less polluting private vehicles is dominant in terms of the distribution of QALE and social care outcomes but not consumption. Inclusion of health inequality aversion alters the rank order compared with prioritisation on health maximisation. The results were sensitive to the magnitude of health opportunity costs, the level of inequality aversion, and the proportion of intervention cost that generates health opportunity cost.

The National Institute for Health and Care Excellence (NICE) is only one of several organisations internationally that uses economic evaluation as part of decision making regarding funding and pricing of new medical technologies. However, it can be argued that NICE has developed a more prominent international profile than most in their use of economics. After 20 years of operation, it is timely to assess the extent of NICE’s achievements, including the economic evaluation methods it has used and its willingness to adapt these as new evaluative approaches emerge and when NICE faces particular policy challenges. This paper considers some of the important policy and contextual developments in the UK over the last 20 years and how these may have shaped NICE’s approach to economic evaluation. It then assesses key areas of NICE methods, including perspective, defining benefits, modelling and uncertainty. The paper concludes that NICE has provided important support for the development of new methods, in particular through its role in identifying priorities for methods research funding and its sponsorship of the NICE Decision Support Unit. However, potentially important developments in methods in a number of important areas have yet to be formally included in NICE’s methods guidance and this should be addressed in the Institute’s 2019/2020 methods review.

The potential for multi-criteria decision analysis (MCDA) to support health technology assessment (HTA) has been much discussed, and various HTA agencies are piloting or applying MCDA. Alongside these developments, good practice guidelines for the application of MCDA in health care have been developed. An assessment of current applications of MCDA to HTA in light of good practice guidelines reveals, however, that many have methodologic flaws that undermine their usefulness. Three challenges are considered: the use of additive models, a lack of connection between criteria scales and weights, and the use of MCDA in economic evaluation. More attention needs to be paid to MCDA good practice by researchers, journal editors, and decision makers and further methodologic developments are required if MCDA is to achieve its potential to support HTA.

BACKGROUND: Policymakers in high-, low-, and middle-income countries alike face challenging choices about resource allocation in health. Economic evaluation can be useful in providing decision makers with the best evidence of the anticipated benefits of new investments, as well as their expected opportunity costs-the benefits forgone of the options not chosen. To guide the decisions of health systems effectively, it is important that the methods of economic evaluation are founded on clear principles, are applied systematically, and are appropriate to the decision problems they seek to inform. METHODS: The Bill and Melinda Gates Foundation, a major funder of economic evaluations of health technologies in low- and middle-income countries (LMICs), commissioned a "reference case" through the International Decision Support Initiative (iDSI) to guide future evaluations, and improve both the consistency and usefulness to decision makers. RESULTS: The iDSI Reference Case draws on previous insights from the World Health Organization, the US Panel on Cost-Effectiveness in Health Care, and the UK National Institute for Health and Care Excellence. Comprising 11 key principles, each accompanied by methodological specifications and reporting standards, the iDSI Reference Case also serves as a means of identifying priorities for methods research, and can be used as a framework for capacity building and technical assistance in LMICs. CONCLUSIONS: The iDSI Reference Case is an aid to thought, not a substitute for it, and should not be followed slavishly without regard to context, culture, or history. This article presents the iDSI Reference Case and discusses the rationale, approach, components, and application in LMICs.

Healthcare systems in low- and middle-income countries (LMICs) face considerable population healthcare needs with markedly fewer resources than those in developed countries. The way in which available resources are allocated across competing priorities is crucial in affecting how much health is generated overall, who receives healthcare interventions and who goes without. Cost-effectiveness analysis (CEA) is one tool that can assist policy-makers in resource allocation. The central concern in CEA is whether the health gains offered by an intervention are large enough relative to its costs to warrant adoption. This requires some notion of the value that must be realized by an intervention, which is most frequently represented using a cost-effectiveness threshold (CET). CETs should be based on estimates of the forgone benefit associated with alternative priorities which consequently cannot be implemented as a result of the commitment of resources to an alternative. For most health care systems these opportunity costs fall predominantly on health as a result of fixed budgets or constraints on health systems’ abilities to increase expenditures. However, many CEAs to inform decisions in LMICs have used aspirational expressions of value, such as the World Health Organization’s (WHO) recommended CETs (of 1-3 times GDP per capita in a country) which are not based upon opportunity costs. In contrast, we estimate CETs for a number of countries based upon recent empirical estimates of foregone benefit (from the English NHS) and international income elasticities of the value of health. The resulting CETs are much lower than those previously posited by WHO. There is no intention to provide definitive CETs; rather, the study is intended to provoke further research in this area of crucial policy importance and outlines how more robust estimates of CETs could be generated.

Introduction As prevalence of undiagnosed HIV declines, it is unclear whether testing programmes will be cost‐effective. To guide their HIV testing programmes, countries require appropriate metrics that can be measured. The cost‐per‐diagnosis is potentially a useful metric. Methods We simulated a series of setting‐scenarios for adult HIV epidemics and ART programmes typical of settings in southern Africa using an individual‐based model and projected forward from 2018 under two policies: (i) a minimum package of “core” testing (i.e. testing in pregnant women, for diagnosis of symptoms, in sex workers, and in men coming forward for circumcision) is conducted, and (ii) core‐testing as above plus additional testing beyond this (“additional‐testing”), for which we specify different rates of testing and various degrees to which those with HIV are more likely to test than those without HIV. We also considered a plausible range of unit test costs. The aim was to assess the relationship between cost‐per‐diagnosis and the incremental cost‐effectiveness ratio (ICER) of the additional‐testing policy. The discount rate used in the base case was 3% per annum (costs in 2018 U.S. dollars). Results There was a strong graded relationship between the cost‐per‐diagnosis and the ICER. Overall, the ICER was below $500 per‐DALY‐averted (the cost‐effectiveness threshold used in primary analysis) so long as the cost‐per‐diagnosis was below $315. This threshold cost‐per‐diagnosis was similar according to epidemic and programmatic features including the prevalence of undiagnosed HIV, the HIV incidence and a measure of HIV programme quality (the proportion of HIV diagnosed people having a viral load

A recent systematic review identified few papers on the economic evaluation of systems for emergency transport of acutely ill or injured patients. In addition, we found no articles dealing with the methodological challenges posed by such studies in low-income or middle-income countries. We therefore carried out an analysis of issues that are of particular salience to this important topic. This is an intellectual study in which we develop models, identify their limitations, suggest potential extensions to the models and discuss priorities for empirical studies to populate models. First, we develop a general model to calculate changes in survival contingent on the reduced time to treatment that an emergency transport system is designed to achieve. Second, we develop a model to estimate transfer times over an area that will be served by a proposed transfer system. Third, we discuss difficulties in obtaining parameters with which to populate the models. Fourth, we discuss costs, both direct and indirect, of an emergency transfer service. Fifth, we discuss the issue that outcomes other than survival should be considered and that the effects of a service are a weighted sum over all the conditions and severities for which the service caters. Lastly, based on the above work, we identify priorities for research. To our knowledge, this is the first study to identify and frame issues in the health economics of acute transfer systems and to develop models to calculate survival rates from basic parameters, such as time delay/survival relationships, that vary by intervention type and context.

Background Bevacizumab is used extensively in the treatment of cancer, including advanced ovarian cancer, for which results of the International Collaborative Ovarian Neoplasm (ICON) 7 trial have been recently reported. The National Institute for Health and Care Excellence's (NICE's) recent decision not to recommend bevacizumab for advanced ovarian cancer was not based on evidence related to the unlicensed lower dosage (7.5 mg/kg) of the drug despite its use in the English National Health Service (NHS) and the ICON7 trial. Objective To report on the findings of an analysis that considered whether the lower dose is cost-effective. Methods Cost-effectiveness analysis is assessed from the perspective of the English NHS and health outcomes expressed in terms of quality-adjusted life-years (QALYs). The analysis focuses on a clinically predefined high-risk subgroup of the ICON7 trial. The price at which the lower dose of bevacizumab could be considered cost-effective for the English NHS is presented for a range of scenarios to inform decisions about price negotiations by international health systems. Results In the base-case analysis, bevacizumab has an incremental cost-effectiveness ratio of £48,975 per additional QALY, which is above NICE's standard cost-effectiveness threshold (£20,000–£30,000 per QALY). The official price of bevacizumab in 2013 was between £2.31 and £2.63 per milligram. A price reduction of between 46% and 67%, dependent on the NICE threshold, would be required for the product to be cost-effective in the high-risk subgroup. Conclusions The lower dose of bevacizumab for advanced ovarian cancer is not cost-effective based on the product's list price and using NICE's cost-effectiveness thresholds. Significant price discounts would be needed to make the drug affordable to the NHS.

When making decisions regarding the funding of new health technologies (e.g. pharmaceuticals), a key consideration should be the opportunity cost associated with a positive funding decision in terms of other patients’ health. Lambda (k) is a term that has been used to refer to a maximum acceptable value (cost-effectiveness threshold) for the incremental cost-effectiveness ratio (ICER) of a new technology, which should reflect the opportunity cost of funding decisions. In 2006, Gafni and Birch [1] discussed the ‘silence of the k’ with respect to the justification of its numerical value within the decision-making process. They noted that the use of threshold values, which do not represent the true opportunity cost of new and generally more expensive technologies, can lead to increased health expenditures with little evidence of increases in population health. In this commentary, we interpret the ‘silence of the k0 with respect to the lack of explicit recognition of the use of a cost-effectiveness threshold by national funding bodies that use the threshold in the decision-making process. We also discuss the potential impact of this silence on health systems. As an example, the Australian Pharmaceutical Benefits Advisory Committee (PBAC) makes recommendations regarding the public funding of pharmaceuticals, on the basis of clinical effectiveness, safety and cost effectiveness (value for money). No official statement has ever been made about the use of a cost-effectiveness threshold to determine whether a pharmaceutical provides value for money. Although our focus is on the Australian PBAC decisionmaking process, the discussion can be applied more widely—first, to many other national funding bodies that use economic evaluation to inform funding decisions but do not explicitly acknowledge the use of a threshold, e.g. the Medical Services Advisory Committee in Australia (MSAC) or the Canadian Agency for Drugs and Technologies in Health, and second, to any collectively funded health system that fails to consider the opportunity costs of its decisions.

Objectives. In 2016, the Second Panel on Cost-effectiveness in Health and Medicine updated the seminal work of the original panel from 2 decades earlier. The Second Panel had an opportunity to reflect on the evolution of cost-effectiveness analysis (CEA) and to provide guidance for the next generation of practitioners and consumers. In this article, we present key topics for future research and policy. Methods. During the course of its deliberations, the Second Panel discussed numerous topics for advancing methods and for improving the use of CEA in decision making. We identify and consider 7 areas for which the panel believes that future research would be particularly fruitful. In each of these areas, we highlight outstanding research needs. The list is not intended as an exhaustive inventory but rather a set of key items that surfaced repeatedly in the panel’s discussions. In the online Appendix , we also list and expound briefly on 8 other important topics. Results. We highlight 7 key areas: CEA and perspectives (determining, valuing, and summarizing elements for the analysis), modeling (comparative modeling and model transparency), health outcomes (valuing temporary health and path states, as well as health effects on caregivers), costing (a cost catalogue, valuing household production, and productivity effects), evidence synthesis (developing theory on learning across studies and combining data from clinical trials and observational studies), estimating and using cost-effectiveness thresholds (empirically representing 2 broad concepts: opportunity costs and public willingness to pay), and reporting and communicating CEAs (written protocols and a quality scoring system). Conclusions. Cost-effectiveness analysis remains a flourishing and evolving field with many opportunities for research. More work is needed on many fronts to understand how best to incorporate CEA into policy and practice.

Background Men with suspected prostate cancer usually undergo transrectal ultrasound (TRUS)-guided prostate biopsy. TRUS-guided biopsy can cause side effects and has relatively poor diagnostic accuracy. Multiparametric magnetic resonance imaging (mpMRI) used as a triage test might allow men to avoid unnecessary TRUS-guided biopsy and improve diagnostic accuracy. Objectives To (1) assess the ability of mpMRI to identify men who can safely avoid unnecessary biopsy, (2) assess the ability of the mpMRI-based pathway to improve the rate of detection of clinically significant (CS) cancer compared with TRUS-guided biopsy and (3) estimate the cost-effectiveness of a mpMRI-based diagnostic pathway. Design A validating paired-cohort study and an economic evaluation using a decision-analytic model. Setting Eleven NHS hospitals in England. Participants Men at risk of prostate cancer undergoing a first prostate biopsy. Interventions Participants underwent three tests: (1) mpMRI (the index test), (2) TRUS-guided biopsy (the current standard) and (3) template prostate mapping (TPM) biopsy (the reference test). Main outcome measures Diagnostic accuracy of mpMRI, TRUS-guided biopsy and TPM-biopsy measured by sensitivity, specificity, positive predictive value (PPV) and negative predictive value (NPV) using primary and secondary definitions of CS cancer. The percentage of negative magnetic resonance imaging (MRI) scans was used to identify men who might be able to avoid biopsy. Results Diagnostic study – a total of 740 men were registered and 576 underwent all three tests. According to TPM-biopsy, the prevalence of any cancer was 71% [95% confidence interval (CI) 67% to 75%]. The prevalence of CS cancer according to the primary definition (a Gleason score of ≥ 4 + 3 and/or cancer core length of ≥ 6 mm) was 40% (95% CI 36% to 44%). For CS cancer, TRUS-guided biopsy showed a sensitivity of 48% (95% CI 42% to 55%), specificity of 96% (95% CI 94% to 98%), PPV of 90% (95% CI 83% to 94%) and NPV of 74% (95% CI 69% to 78%). The sensitivity of mpMRI was 93% (95% CI 88% to 96%), specificity was 41% (95% CI 36% to 46%), PPV was 51% (95% CI 46% to 56%) and NPV was 89% (95% CI 83% to 94%). A negative mpMRI scan was recorded for 158 men (27%). Of these, 17 were found to have CS cancer on TPM-biopsy. Economic evaluation – the most cost-effective strategy involved testing all men with mpMRI, followed by MRI-guided TRUS-guided biopsy in those patients with suspected CS cancer, followed by rebiopsy if CS cancer was not detected. This strategy is cost-effective at the TRUS-guided biopsy definition 2 (any Gleason pattern of ≥ 4 and/or cancer core length of ≥ 4 mm), mpMRI definition 2 (lesion volume of ≥ 0.2 ml and/or Gleason score of ≥ 3 + 4) and cut-off point 2 (likely to be benign) and detects 95% (95% CI 92% to 98%) of CS cancers. The main drivers of cost-effectiveness were the unit costs of tests, the improvement in sensitivity of MRI-guided TRUS-guided biopsy compared with blind TRUS-guided biopsy and the longer-term costs and outcomes of men with cancer. Limitations The PROstate Magnetic resonance Imaging Study (PROMIS) was carried out in a selected group and excluded men with a prostate volume of > 100 ml, who are less likely to have cancer. The limitations in the economic modelling arise from the limited evidence on the long-term outcomes of men with prostate cancer and on the sensitivity of MRI-targeted repeat biopsy. Conclusions Incorporating mpMRI into the diagnostic pathway as an initial test prior to prostate biopsy may (1) reduce the proportion of men having unnecessary biopsies, (2) improve the detection of CS prostate cancer and (3) increase the cost-effectiveness of the prostate cancer diagnostic and therapeutic pathway. The PROMIS data set will be used for future research; this is likely to include modelling prognostic factors for CS cancer, optimising MRI scan sequencing and biomarker or translational research analyses using the blood and urine samples collected. Better-quality evidence on long-term outcomes in prostate cancer under the various management strategies is required to better assess cost-effectiveness. The value-of-information analysis should be developed further to assess new research to commission. Trial registration Current Controlled Trials ISRCTN16082556 and NCT01292291. Funding This project was funded by the National Institute for Health Research (NIHR) Health Technology Assessment programme and will be published in full in Health Technology Assessment; Vol. 22, No. 39. See the NIHR Journals Library website for further project information. This project was also supported and partially funded by the NIHR Biomedical Research Centre at University College London (UCL) Hospitals NHS Foundation Trust and UCL and by The Royal Marsden NHS Foundation Trust and The Institute of Cancer Research Biomedical Research Centre and was co-ordinated by the Medical Research Council’s Clinical Trials Unit at UCL (grant code MC_UU_12023/28). It was sponsored by UCL. Funding for the additional collection of blood and urine samples for translational research was provided by Prostate Cancer UK.

Objective: To determine the impact of endobronchial coils on health-related quality-of-life (HRQoL). This paper utilizes trial data to identify the predictors of HRQoL in patients with severe emphysema, and subsequently estimates the impact of a new treatment on HRQoL (measured by utilities). These utility estimates are used to generate indicative long-term QALY estimates for a range of clinically plausible scenarios as a precursor to cost-effectiveness analyses.Methods: Patient level HRQoL data from RENEW and the National Emphysema Treatment Trial (NETT) were combined and mapped to generic EuroQol 5-dimension health utility questionnaire (EQ-5D) values using a published algorithm. Multilevel statistical models were developed using treatment, time, response, and baseline characteristics (EQ-5D, age, gender, FEV1, lung RV) to predict EQ-5D over time. Lifetime QALY estimates were generated using published survival data from NETT (assuming no impact of treatment on mortality) and four clinically plausible response profiles. Each response profile was combined with assumptions around treatment impact (constant or time varying).Results: After controlling for baseline characteristics, both treatment and response had a statistically significant impact (p Conclusions: Coils and SoC resulted in statistically significant improvements in HRQoL compared to SoC alone in patients with severe emphysema.

Background Fenestrated endovascular aneurysm repair (fEVAR) is a new approach for complex abdominal aortic aneurysms, limited to a few specialist centers, with limited evidence base. We developed a cost-effectiveness decision model of fEVAR compared to open surgical repair (OSR) to investigate the likely direction of costs and benefits and inform further research projects on this technology. Methods A systematic review with meta-analysis and a four-state Markov model were used to estimate the cost-effectiveness of fEVAR versus OSR. We used a recent coverage with evidence development framework to characterize the main sources of uncertainty and inform decisions about the type of further research that would be most worthwhile and feasible. Results Seven observational comparative studies were identified, of which four presented odds ratios adjusted for confounders. The odds ratios for operative mortality varied widely between studies. Assuming a central estimate of the odds ratio of 0.54 (95% CI 0.05–6.24), the decision model estimated that the incremental cost per quality adjusted life year (QALY) was £74,580/QALY with a probability of 9 and 16% of being cost-effective at standard cost-effectiveness thresholds of £20,000/QALY and £30,000/QALY, respectively. The Expected Value of Perfect Information over 10 years at a threshold of £20,000/QALY was £11.2 million. Operative mortality contributed to most of the uncertainty in the decision model. Conclusions In the case of “maturing technologies”, decision modelling indicates the likely direction of costs and benefits and guides the development of further research projects. In our analysis of fEVAR versus OSR, decision uncertainty, particularly around operative mortality, might be effectively resolved by a short-term RCT, or possibly a well-conducted comparative observational study. Decision makers may consider that a conditional coverage decision is warranted with assessments required to make this type of recommendation depending on local priorities and circumstances. Electronic supplementary material The online version of this article (10.1186/s12962-018-0098-7) contains supplementary material, which is available to authorized users.

BACKGROUND: The WHO HIV Treatment Guidelines suggest routine viral-load monitoring can be used to differentiate antiretroviral therapy (ART) delivery and reduce the frequency of clinic visits for patients stable on ART. This recommendation was informed by economic analysis that showed the approach is very likely to be cost-effective, even in the most resource constrained of settings. The health benefits were shown to be modest but the costs of introducing and scaling up viral load monitoring can be offset by anticipated reductions in the costs of clinic visits, due to these being less frequent for many patients. KEY ISSUES FOR ECONOMIC EVALUATION: The cost-effectiveness of introducing viral-load informed differentiated care depends upon whether cost reductions are possible if the number of clinic visits is reduced and/or how freed clinic capacity is used for alternative priorities. Where freed resources, either physical or financial, generate large health gains (e.g. if committed to patients failing ART or to other high value health care interventions), the benefits of differentiated care are expected to be high; if however these freed physical resources are already under-utilized or financial resources are used less efficiently and would not be put to as beneficial an alternative use, the policy may not be cost-effective. The implication is that the use of conventional unit costs to value resources may not well reflect the latter's value in contributing to health improvement. Analyses intended to inform resource allocated decisions in a number of settings may therefore have to be interpreted with due consideration to local context. In this paper we present methods of how economic analyses can reflect the real value of health care resources rather than simply applying their unit costs. The analyses informing the WHO Guidelines are re-estimated by implementing scenarios using this framework, informing how differentiated care can be prioritized to generate greatest gains in population health. IMPLICATIONS: The findings have important implications for how economic analyses should be undertaken and reported in HIV and other disease areas. Results provide guidance on conditions under which viral load informed differentiated care will more likely prove to be cost effective when implemented.

Background: Survival rates in lung cancer in England are significantly lower than in many similar countries. A range of Be Clear on\ud Cancer (BCOC) campaigns have been conducted targeting lung cancer and found to improve the proportion of diagnoses at the\ud early stage of disease. This paper considers the cost-effectiveness of such campaigns, evaluating the effect of both the regional\ud and national BCOC campaigns on the stage distribution of non-small-cell lung cancer (NSCLC) at diagnosis.\ud Methods: A natural history model of NSCLC was developed using incidence data, data elicited from clinical experts and model\ud calibration techniques. This structure is used to consider the lifetime cost and quality-adjusted survival implications of the early\ud awareness campaigns. Incremental cost-effectiveness ratios (ICERs) in terms of additional costs per quality-adjusted life-years\ud (QALYs) gained are presented. Two scenario analyses were conducted to investigate the role of changes in the ‘worried-well’\ud population and the route of diagnosis that might occur as a result of the campaigns.\ud Results: The base-case theoretical model found the regional and national early awareness campaigns to be associated with QALY\ud gains of 289 and 178 QALYs and ICERs of d13 660 and d18 173 per QALY gained, respectively. The scenarios found that increases\ud in the ‘worried-well’ population may impact the cost-effectiveness conclusions.\ud Conclusions: Subject to the available evidence, the analysis suggests that early awareness campaigns in lung cancer have the\ud potential to be cost-effective. However, significant additional research is required to address many of the limitations of this study.\ud In addition, the estimated natural history model presents previously unavailable estimates of the prevalence and rate of disease\ud progression in the undiagnosed population.

Organisations across diverse health care systems making decisions about the funding of new medical technologies face extensive stakeholder and political pressures. As a consequence, there is quite understandable pressure to take account of other attributes of benefit and to fund technologies, even when the opportunity costs are likely exceed the benefits they offer. Recent evidence suggests that NICE technology appraisal is already approving drugs where more health is likely to be lost than gained. Also, NICE recently proposed increasing the upper bound of the cost-effectiveness threshold to reflect other attributes of benefit but without a proper assessment of the type of benefits that are expected to be displaced. It appears that NICE has taken a direction of travel, which means that more harm than good is being, and will continue to be, done, but it is unidentified NHS patients who bear the real opportunity costs.

AIMS: New sequencing technologies allow increased opportunities to use genomic-based diagnostic tests (genomic tests) in routine clinical practice, which will impact healthcare budgets and patients' outcomes. This article aims to generate a list of recommendations on how the principles and methods of cost-effectiveness analysis (CEA) can be used to quantify the costs and benefits of genomic tests. METHODS: A systematic literature search identified publications describing the use of CEA to evaluate genomic tests. Data were extracted as key concepts to produce a thematic list of previously described challenges and solutions to using CEA to evaluate genomic tests. Defining features of evaluating genomic tests were categorized into a list of key recommendations for applying methods in practice and for research needs. RESULTS: Features producing challenges in the implementation of CEA to evaluate genomic tests were as follows: the ability of the tests to diagnose multiple disorders; potential consequences for future generations suggesting an infinite time horizon; and the potential need to consider nonhealth benefits. CONCLUSIONS: CEA was identified as an appropriate evaluative framework for genomic tests, although standard methods may need modification and important method research questions remain. Key recommendations suggest a need for research to reflect: sharing genomic information across generations; genomic tests for multiple disorders; and health and nonhealth benefits.

Evidence about cost-effectiveness is increasingly being used to inform decisions about the funding of new technologies that are usually implemented as guidelines from centralized decision-making bodies. However, there is also an increasing recognition for the role of patients in determining their preferred treatment option. This paper presents a method to estimate the value of implementing a choice-based decision process using the cost-effectiveness analysis toolbox. This value is estimated for 3 alternative scenarios. First, it compares centralized decisions, based on population average cost-effectiveness, against a decision process based on patient choice. Second, it compares centralized decision based on patients' subgroups versus an individual choice-based decision process. Third, it compares a centralized process based on average cost-effectiveness against a choice-based process where patients choose according to a different measure of outcome to that used by the centralized decision maker. The methods are applied to a case study for the management of acute coronary syndrome. It is concluded that implementing a choice-based process of treatment allocation may be an option in collectively funded health systems. However, its value will depend on the specific health problem and the social values considered relevant to the health system.

BACKGROUND: There is limited information on the costs and benefits of alternative adjunct non-pharmacological treatments for knee osteoarthritis and little guidance on which should be prioritised for commissioning within the NHS. This study estimates the costs and benefits of acupuncture, braces, heat treatment, insoles, interferential therapy, laser/light therapy, manual therapy, neuromuscular electrical stimulation, pulsed electrical stimulation, pulsed electromagnetic fields, static magnets and transcutaneous electrical nerve Stimulation (TENS), based on all relevant data, to facilitate a more complete assessment of value. METHODS: Data from 88 randomised controlled trials including 7,507 patients were obtained from a systematic review. The studies reported a wide range of outcomes. These were converted into EQ-5D index values using prediction models, and synthesised using network meta-analysis. Analyses were conducted including firstly all trials and secondly only trials with low risk of selection bias. Resource use was estimated from trials, expert opinion and the literature. A decision analytic model synthesised all evidence to assess interventions over a typical treatment period (constant benefit over eight weeks or linear increase in effect over weeks zero to eight and dissipation over weeks eight to 16). RESULTS: When all trials are considered, TENS is cost-effective at thresholds of £20-30,000 per QALY with an incremental cost-effectiveness ratio of £2,690 per QALY vs. usual care. When trials with a low risk of selection bias are considered, acupuncture is cost-effective with an incremental cost-effectiveness ratio of £13,502 per QALY vs. TENS. The results of the analysis were sensitive to varying the intensity, with which interventions were delivered, and the magnitude and duration of intervention effects on EQ-5D. CONCLUSIONS: Using the £20,000 per QALY NICE threshold results in TENS being cost-effective if all trials are considered. If only higher quality trials are considered, acupuncture is cost-effective at this threshold, and thresholds down to £14,000 per QALY.

A growing number of health care systems internationally use formal economic evaluation methods to support health care funding decisions. Recently, a range of organizations have been advocating forms of analysis that have been termed "value frameworks." There has also been a push for analytical methods to reflect a fuller range of benefits of interventions through multicriteria decision analysis. A key principle that is invariably neglected in current and proposed frameworks is the need to reflect evidence on the opportunity costs that health systems face when making funding decisions. The mechanisms by which opportunity costs are realized vary depending on the system's financial arrangements, but they always mean that a decision to fund a specific intervention for a particular patient group has the potential to impose costs on others in terms of forgone benefits. These opportunity costs are rarely explicitly reflected in analysis to support decisions, but recent developments to quantify benefits forgone make more appropriate analyses feasible. Opportunity costs also need to be reflected in decisions if a broader range of attributes of benefit is considered, and opportunity costs are a key consideration in determining the appropriate level of total expenditure in a system. The principles by which opportunity costs can be reflected in analysis are illustrated in this article by using the example of the proposed methods for value-based pricing in the United Kingdom.

Background: This review scopes the evidence on the effectiveness and costeffectiveness of interventions to improve suboptimal use of medicines in order to determine the evidence gaps and help inform research priorities. Sources of data: Systematic searches of the National Health Service (NHS) Economic Evaluation Database, the Cochrane Database of Systematic Reviews and the Database of Abstracts of Reviews of Effects. Areas of agreement: The majority of the studies evaluated interventions to improve adherence, inappropriate prescribing and prescribing errors. Areas of controversy: Interventions tend to be specific to a particular stage of the pathway and/or to a particular disease and have mostly been evaluated for their effect on intermediate or process outcomes. Growing points: Medicines optimization offers an opportunity to improve health outcomes and efficiency of healthcare. Areas timely for developing research: The available evidence is insufficient to assess the effectiveness and cost-effectiveness of interventions to address suboptimal medicine use in the UK NHS. Decision modelling, evidence synthesis and elicitation have the potential to address the evidence gaps and help prioritize research.

Background\ud\udBreast, cervical and colorectal cancers are the three most frequent cancers in women, while lung, prostate and colorectal cancers are the most frequent in men. Much attention has been given to the economic evaluation of pharmaceuticals for treatment of cancer by the National Institute for Health and Care Excellence (NICE) in the UK and similar authorities internationally, while economic analysis developed for other types of anti-cancer interventions, including radiotherapy and surgery, are less common.\udObjectives\ud\udOur objective was to review methods used in published cost-effectiveness studies evaluating radiotherapy for breast, cervical, colorectal, head and neck and prostate cancer, and to compare the economic evaluation methods applied with those defined in the guidelines used by the NICE technology appraisal programme.\udMethods\ud\udA systematic search of seven databases (MEDLINE, EMBASE, CDSR, NHSEED, HTA, DARE, EconLit) as well as research registers, the NICE website and conference proceedings was conducted in July 2012. Only economic evaluations of radiotherapy interventions in individuals diagnosed with cancer that included quality-adjusted life-years (QALYs) or life-years (LYs) were included. Included studies were appraised on the basis of satisfying essential, preferred and UK-specific methods requirements, building on the NICE Reference Case for economic evaluations and on other methods guidelines.\udResults\ud\udA total of 29 studies satisfied the inclusion criteria (breast 14, colorectal 2, prostate 10, cervical 0, head and neck 3). Only two studies were conducted in the UK (13 in the USA). Among essential methods criteria, the main issue was that only three (10 %) of the studies used clinical-effectiveness estimates identified through systematic review of the literature. Similarly, only eight (28 %) studies sourced health-related quality-of-life data directly from patients with the condition of interest. Other essential criteria (e.g. clear description of comparators, patient group indication and appropriate time horizon) were generally fulfilled, while most of the UK-specific requirements were not met.\udConclusion\ud\udBased on this review there is a dearth of up-to-date, robust evidence on the cost effectiveness of radiotherapy in cancer suitable to support decision making in the UK. Studies selected did not fully satisfy essential method standards currently recommended by NICE.

Despite expansion of services for prevention of mother-to-child transmission of HIV (PMTCT), about 700 infants acquire HIV every day. Early initiation of antiretroviral therapy for HIV-infected infants reduces mortality but requires diagnosis by virological testing, which is complex, expensive, and inaccessible in many settings. Little cost-effectiveness evidence exists about different strategies to deliver early infant diagnosis services. Cost-effectiveness will vary depending on entry points for testing, underlying prevalences of HIV, PMTCT coverage, treatment availability, programme attrition, and other factors. Appropriate policy responses are therefore context-specific. In most cases, early infant diagnosis should be concentrated at entry points where underlying infant HIV prevalence is highest (eg, malnutrition wards). This strategy contrasts with the tendency at present to test mainly within PMTCT programmes. If testing is undertaken in PMTCT programmes with high coverage, addition of a virological test at birth might have advantages, including greater predictive value, earlier diagnosis, and better infant follow-up. National programme managers should recognise the opportunity costs of the limited resources available, acknowledge the changing scenario of PMTCT scale-up, ensure implementation of provider-initiated testing and counselling, and tailor early infant diagnosis programmes to maximise health gains for children.

This article develops a general framework to guide the use of subgroup cost-effectiveness analysis for decision making in a collectively funded health system. In doing so, it addresses 2 key policy questions, namely, the identification and selection of subgroups, while distinguishing 2 sources of potential value associated with heterogeneity. These are 1) the value of revealing the factors associated with heterogeneity in costs and outcomes using existing evidence (static value) and 2) the value of acquiring further subgroup-related evidence to resolve the uncertainty given the current understanding of heterogeneity (dynamic value). Consideration of these 2 sources of value can guide subgroup-specific treatment decisions and inform whether further research should be conducted to resolve uncertainty to explain variability in costs and outcomes. We apply the proposed methods to a cost-effectiveness analysis for the management of patients with acute coronary syndrome. This study presents the expected net benefits under current and perfect information when subgroups are defined based on the use and combination of 6 binary covariates. The results of the case study confirm the theoretical expectations. As more subgroups are considered, the marginal net benefit gains obtained under the current information show diminishing marginal returns, and the expected value of perfect information shows a decreasing trend. We present a suggested algorithm that synthesizes the results to guide policy.

Background A number of published economic evaluations of elective endovascular aneurysm repair (EVAR) versus open repair for abdominal aortic aneurysm (AAA) have come to differing conclusions about whether EVAR is cost-effective. This paper reviews the current evidence base and presents up-to-date cost-effectiveness analyses in the light of results of four randomized clinical trials: EVAR-1, DREAM, OVER and ACE. Methods Markov models were used to estimate lifetime costs from a UK perspective and quality-adjusted life-years (QALYs) based on the results of each of the four trials. The outcomes included in the model were: procedure costs, surveillance costs, reintervention costs, health-related quality of life, aneurysm-related mortality and other-cause mortality. Alternative scenarios about complications, reinterventions and deaths beyond the trial were explored. Results Models based on the results of the EVAR-1, DREAM or ACE trials did not find EVAR to be cost-effective at thresholds used in the UK (up to £30 000 per QALY). EVAR seemed cost-effective according to models based on the OVER trial. These results seemed robust to alternative model scenarios about events beyond the trial intervals. Conclusion These analyses did not find that EVAR is cost-effective compared with open repair in the long term in trials conducted in European centres. EVAR did appear to be cost-effective based on the OVER trial, conducted in the USA. Caution must be exercised when transferring the results of economic evaluations from one country to another.

BackgroundGestational diabetes mellitus (GDM) is associated with a higher risk of important adverse outcomes. Practice varies and the best strategy for identifying and treating GDM is unclear.AimTo estimate the clinical effectiveness and cost-effectiveness of strategies for identifying and treating women with GDM.MethodsWe analysed individual participant data (IPD) from birth cohorts and conducted systematic reviews to estimate the association of maternal glucose levels with adverse perinatal outcomes; GDM prevalence; maternal characteristics/risk factors for GDM; and the effectiveness and costs of treatments. The cost-effectiveness of various strategies was estimated using a decision tree model, along with a value of information analysis to assess where future research might be worthwhile. Detailed systematic searches of MEDLINE®and MEDLINE In-Process & Other Non-Indexed Citations®, EMBASE, Cumulative Index to Nursing and Allied Health Literature Plus, Cochrane Central Register of Controlled Trials, Cochrane Database of Systematic Reviews, Database of Abstracts of Reviews of Effects, Health Technology Assessment database, NHS Economic Evaluation Database, Maternity and Infant Care database and the Cochrane Methodology Register were undertaken from inception up to October 2014.ResultsWe identified 58 studies examining maternal glucose levels and outcome associations. Analyses using IPD alone and the systematic review demonstrated continuous linear associations of fasting and post-load glucose levels with adverse perinatal outcomes, with no clear threshold below which there is no increased risk. Using IPD, we estimated glucose thresholds to identify infants at high risk of being born large for gestational age or with high adiposity; for South Asian (SA) women these thresholds were fasting and post-load glucose levels of 5.2 mmol/l and 7.2 mmol/l, respectively and for white British (WB) women they were 5.4 and 7.5 mmol/l, respectively. Prevalence using IPD and published data varied from 1.2% to 24.2% (depending on criteria and population) and was consistently two to three times higher in SA women than in WB women. Lowering thresholds to identify GDM, particularly in women of SA origin, identifies more women at risk, but increases costs. Maternal characteristics did not accurately identify women with GDM; there was limited evidence that in some populations risk factors may be useful for identifying low-risk women. Dietary modification additional to routine care reduced the risk of most adverse perinatal outcomes. Metformin (Glucophage,®Teva UK Ltd, Eastbourne, UK) and insulin were more effective than glibenclamide (Aurobindo Pharma – Milpharm Ltd, South Ruislip, Middlesex, UK). For all strategies to identify and treat GDM, the costs exceeded the health benefits. A policy of no screening/testing or treatment offered the maximum expected net monetary benefit (NMB) of £1184 at a cost-effectiveness threshold of £20,000 per quality-adjusted life-year (QALY). The NMB for the three best-performing strategies in each category (screen only, then treat; screen, test, then treat; and test all, then treat) ranged between –£1197 and –£1210. Further research to reduce uncertainty around potential longer-term benefits for the mothers and offspring, find ways of improving the accuracy of identifying women with GDM, and reduce costs of identification and treatment would be worthwhile.LimitationsWe did not have access to IPD from populations in the UK outside of England. Few observational studies reported longer-term associations, and treatment trials have generally reported only perinatal outcomes.ConclusionsUsing the national standard cost-effectiveness threshold of £20,000 per QALY it is not cost-effective to routinely identify pregnant women for treatment of hyperglycaemia. Further research to provide evidence on longer-term outcomes, and more cost-effective ways to detect and treat GDM, would be valuable.Study registrationThis study is registered as PROSPERO CRD42013004608.FundingThe National Institute for Health Research Health Technology Assessment programme.

BackgroundThe iDSI reference case, originally published in 2014, aims to improve the quality and comparability of cost-effectiveness analyses (CEAs). This study assesses whether the development of the guideline has improved the reporting and methodology for CEAs using disability-adjusted life-years (DALYs).MethodsWe analyzed the Tufts Medical Center Global Health CEA Registry to identify cost-per-DALY averted studies published from 2011 to 2017. Among each of 11 principles in the iDSI reference case, we translated all reporting standards and methodological specifications into quantifiable yes/no questions and awarded articles one point for each item satisfied. We then separately calculated reporting and methods scores, measured as percent adherence (0%=no adherence, 100%=full adherence). Using the year 2014 as the dissemination period, we conducted a pre-post analysis. Additionally, we conducted an analysis stratified by the 11 principles and examined different scoring strategies and dissemination periods in sensitivity analyses.ResultsArticles averaged 74% adherence to reporting standards and 60% adherence to methodological specifications. Adherence to reporting standards increased slightly over time (72% pre-2014 vs. 75% post-2014, pConclusionsThe iDSI reference case has substantial potential to serve as a useful resource for researchers and policy-makers in global health settings, but greater effort to promote adherence and awareness is needed to achieve its potential.

The study’s objective was to assess the cost-effectiveness of preoperative transfusion compared with no preoperative transfusion in patients with sickle cell disease undergoing low- or medium-risk surgery. Seventy patients with sickle cell disease (HbSS/Ss 0 thal genotypes) undergoing elective surgery participated in a multicentre randomised trial, Transfusion Alternatives Preoperatively in Sickle Cell Disease (TAPS). Here, a cost-effectiveness analysis based on evidence from that trial is presented. A decision-analytic model is used to incorporate long-term consequences of transfusions and acute chest syndrome. Costs and health benefits, expressed as quality-adjusted life years (QALYs), are reported from the ‘within-trial’ analysis and for the decision-analytic model. The probability of cost-effectiveness for each form of management is calculated taking into account the small sample size and other sources of uncertainty. In the range of scenarios considered in the analysis, preoperative transfusion was more effective, with the mean improvement in QALYs ranging from 0.018 to 0.206 per patient, and also less costly in all but one scenario, with the mean cost difference ranging from � £813 to £26. All scenarios suggested preoperative transfusion had a probability of cost-effectiveness >0.79 at a cost-effectiveness threshold of £20 000 per QALY.

Background Laparoscopic fundoplication surgery has been shown to be a cost-effective alternative to continued medical management over 1 year for patients with gastro-oesophageal reflux disease (GORD). The longer-term cost-effectiveness is, however, uncertain. This study evaluated the long-term health benefits, costs and cost-effectiveness of laparoscopic fundoplication compared with continued medical management in patients with GORD. Methods Individual patient data were used from the 5-year follow-up of the REFLUX trial, a large multicentre, pragmatic, randomized trial in which 357 patients with GORD for at least 12 months at trial entry were allocated randomly to early laparoscopic fundoplication or continued medical management. Health outcomes were expressed in quality-adjusted life-years (QALYs). A UK National Health Service perspective was used for costs. Results The group randomized to surgery experienced better health outcomes in each year of follow-up, but the difference narrowed over time. At 5 years, the surgery group had experienced 0·216 (95 per cent confidence interval 0·021 to 0·412) more QALYs but also accrued €1832 (1214 to 2448) more costs. The incremental cost-effectiveness ratio was €8481 per QALY gained. The probability that surgery is the most cost-effective intervention was 0·932 at a threshold of €24 134/QALY (£20 000/QALY). Results were robust to most sensitivity analyses, except where patients with missing data randomized to surgery were assumed to have worse health outcomes. Conclusion Laparoscopic fundoplication is a cost-effective alternative to continued medical management over 5 years. No evidence was found to suggest that the cost-effectiveness of laparoscopic fundoplication diminishes over time.

To evaluate the long-term cost-effectiveness of 12-months treatment with prasugrel vs clopidogrel from four European healthcare systems' perspectives (Germany, Sweden, the Netherlands, and Turkey).In the TRITON-TIMI 38 trial, patients with an acute coronary syndrome (ACS) undergoing percutaneous coronary intervention (PCI) were treated with prasugrel or clopidogrel. Prasugrel reduced the composite end-point (cardiovascular death, MI, or stroke), but increased TIMI major bleeding. A Markov model was constructed to facilitate a lifetime horizon for the analysis. A series of risk equations constructed using individual patient data from TRITON-TIMI 38 was used to estimate risks of clinical events. Quality-adjusted life-years (QALYs) were derived by weighting survival time by estimates of health-related quality-of-life. Incremental cost-effectiveness is presented based on differences in treatments' mean costs and QALYs for the licensed population in TRITON-TIMI 38, and the sub-groups of UA-NSTEMI, STEMI, diabetes, and the 'core clinical cohort' (75 years, ≥60 kg, no history of stroke or TIA).Mean cost of study drug was €364 (Turkey) to €818 (Germany) higher for prasugrel vs clopidogrel. Rehospitalization costs at 12 months were lower for prasugrel due to reduced rates of revascularization, although hospitalization costs beyond 12 months were higher due to longer life expectancy associated with lower rates of non-fatal MI in the prasugrel group. The incremental cost per QALY saved with prasugrel in the licensed population ranged from €6520 (for Sweden) to €14,350 for (Germany). Prasugrel's cost per QALY was more favourable still in the STEMI and diabetes sub-groups of the licensed population.Probabilistic analyses of the whole trial population is impractical due to the number of individual patient profiles over which population level results are calculated.Among patients undergoing PCI for ACS, treatment with prasugrel compared with clopidogrel resulted in favourable cost-effectiveness profiles from these healthcare systems' perspectives.

Harris has been a vocal critic of CEA and the QALY for over 20 years. In this paper we attempt to summarise and evaluate both Harris’ criticisms of CEA and the alternative procedures he commends to health care decision makers. Harris’ basic position is that all health benefits are indivisible and, unless a strong argument can be made, of equal worth. He argues individuals have a right to treatment that cannot be denied by a decision maker on the basis of their ability to benefit and therefore that life saving treatments dominate life enhancing treatments in all circumstances, regardless of the QALY benefits in both cases. In this paper we review Harris’ arguments against the use of CEA and QALYs and critically appraise his suggestions for alternative approaches to health care resource allocation. We conclude that whilst his work has challenged the proponents of CEA and QALYs to be explicit about the method’s discriminatory characteristics, his arguments are largely based upon the flawed assumptions that lives can be saved, rather than death postponed; and that opportunity cost can be sidestepped by attempting to impose the same outcome for all.

Objective To evaluate the cost-effectiveness of implantable cardioverter defibrillators (ICDs), cardiac resynchronisation therapy pacemakers (CRT-Ps) and combination therapy (CRT-D) in patients with heart failure with reduced ejection fraction based on a range of clinical characteristics.\ud \ud Methods Individual patient data from 13 randomised trials were used to inform a decision analytical model. A series of regression equations were used to predict baseline all-cause mortality, hospitalisation rates and health-related quality of life and device-related treatment effects. Clinical variables used in these equations were age, QRS duration, New York Heart Association (NYHA) class, ischaemic aetiology and left bundle branch block (LBBB). A UK National Health Service perspective and a lifetime time horizon were used. Benefits were expressed as quality-adjusted life-years (QALYs). Results were reported for 24 subgroups based on LBBB status, QRS duration and NYHA class.\ud \ud Results At a threshold of £30 000 per QALY gained, CRT-D was cost-effective in 10 of the 24 subgroups including all LBBB morphology patients with NYHA I/II/III. ICD is cost-effective for all non-NYHA IV patients with QRS duration 120 ms and for NYHA I/II non-LBBB morphology patients with QRS duration between 120 ms and 149 ms. CRT-P was also cost-effective in all NYHA III/IV patients with QRS duration 120 ms. Device therapy is cost-effective in most patient groups with LBBB at a threshold of £20 000 per QALY gained. Results were robust to altering key model parameters.\ud \ud Conclusions At a threshold of £30 000 per QALY gained, CRT-D is cost-effective in a far wider group than previously recommended in the UK. In some subgroups ICD and CRT-P remain the cost-effective choice.

Objectives- T identify the literature on primary total hip replacement(THR) surgery that is relevant to the question of whether prostheses differ in their medium to longer term outcomes, and to synthesise this evidence. To use evidence regarding both costs and outcomes of primary THR to model how much more effective newer prostheses must be to justify higher costs

There is increasing recognition that decision modelling is central to health technology assessment and, in particular, to analyses to support formal decision making regarding the funding of the use of new technologies. In part, the key role of decision analysis stems from the need to handle multiple sources of uncertainty in the available evidence. The use of probabilistic decision analysis is a means of reflecting the parameter uncertainty in models and presenting this in a comprehensible manner to decision makers. In this article, we demonstrate the potential role of probabilistic models using the case study of total hip replacement surgery.A cost-effectiveness model was constructed to compare the Charnley and Spectron hip prostheses in terms of lifetime costs and quality-adjusted life-years (QALYs). Revision rates were estimated from the Swedish National Total Hip Arthroplasty Register (1992-2000); the risk of revision with the Spectron prosthesis relative to the Charnley prosthesis was 0.67 (95% confidence interval [CI] 0.32, 1.02) for early revisions and 0.26 (95% CI 0.07, 0.46) for late revisions. This lower revision risk resulted in the Spectron generating more QALYs than the Charnley prosthesis. Based on mean costs and QALYs, the Spectron results in cost savings in younger patients, and generates incremental cost-effectiveness ratios of between pound1000 and pound16 000 in older patient groups. The probabilistic results from the model indicated that, if it is assumed that decision makers are willing to pay up to pound20 000 per additional QALY, the probability of the Spectron being the more cost-effective prosthesis ranged between 70% and 100%, depending on the age and sex of the patient.This article looks at the application of probabilistic decision modelling using total hip replacement as a case study to emphasis the need for decision models to quantify all sources of parameter uncertainty and to clearly distinguish parameter uncertainty from subgroup heterogeneity.

This paper presents a conceptual framework to analyse the design of the cost-effectiveness appraisal process of new healthcare technologies. The framework characterises the appraisal processes as a diagnostic test aimed at identifying cost-effective (true positive) and non-cost-effective (true negative) technologies. Using the framework, factors that influence the value of operating an appraisal process, in terms of net gain to population health, are identified. The framework is used to gain insight into current policy questions including (a) how rigorous the process should be, (b) who should have the burden of proof, and (c) how optimal design changes when allowing for appeals, price reductions, resubmissions, and re-evaluations. The paper demonstrates that there is no one optimal appraisal process and the process should be adapted over time and to the specific technology under assessment. Optimal design depends on country-specific features of (future) technologies, for example, effect, price, and size of the patient population, which might explain the difference in appraisal processes across countries. It is shown that burden of proof should be placed on the producers and that the impact of price reductions and patient access schemes on the producer's price setting should be considered when designing the appraisal process.

BACKGROUND: It is unknown what properties would be required to make an intervention in low income countries that can eradicate or control human immunodeficiency virus (HIV) without antiretroviral therapy (ART) cost-effective.METHODS: We used a model of HIV and ART to investigate the effect of introducing an ART-free viral suppression intervention in 2022 using Zimbabwe as an example country. We assumed that the intervention (cost: $500) would be accessible for 90% of the population, be given to those receiving effective ART, have sufficient efficacy to allow ART interruption in 95%, with a rate of viral rebound of 5% per year in the first 3 months, and a 50% decline in rate with each successive year.RESULTS: An ART-free viral suppression intervention with these properties would result in >0.53 million disability-adjusted-life-years averted over 2022-2042, with a reduction in HIV program costs of $300 million (8.7% saving). An intervention of this efficacy costing anything up to $1400 is likely to be cost-effective in this setting.CONCLUSIONS: Interventions aimed at curing HIV infection have the potential to improve overall disease burden and to reduce costs. Given the effectiveness and cost of ART, such interventions would have to be inexpensive and highly effective.

SummaryBackground The new direct-acting anti-virals (DAAs) for hepatitis C virus (HCV) infection offer higher cure rates, but at a much higher cost than the standard interferon-based treatments. Aim To identify the cost-effective treatment for patients with HCV infection with F3 liver fibrosis who are at high risk of progression to cirrhosis. Methods A decision-analytic Markov model compared the health benefits and costs of all currently licensed treatments as single treatments and in sequential therapy of up to three lines. Costs were expressed in pound sterling from the perspective of the UK National Health Service. Health benefits were expressed in quality-adjusted life years. Results Treatment before progression to cirrhosis always offers the most health benefits for the least costs. Sequential therapy with multiple treatment lines cures over 89% of patients across all HCV genotypes while ensuring a cost-effective use of resources. Cost-effective regimes for HCV genotype 1 patients include first-line oral therapy with sofosbuvir–ledipasvir while peginterferon continues to have a role in other genotypes. Conclusions The cost-effective treatment for HCV can be established using decision analytic modelling comparing single and sequential therapies. Sequential therapy with DAAs is effective and cost-effective in HCV patients with F3 fibrosis. This information is of significant benefit to health care providers with budget limitations and provides a sound scientific basis for drug treatment choices.

Background: Protease inhibitor (PI) monotherapy can maintain virological suppression in the majority of patients once it has been established on triple therapy and may also have the potential for substantial cost savings arising from the use of fewer drugs. However, the cost effectiveness of PI monotherapy has yet to be demonstrated. Objectives: In this study we examine the cost effectiveness of PI monotherapy with prompt return to combination therapy in the event of viral load rebound compared with ongoing triple therapy (OT) in patients with suppressed viral load on combination antiretroviral therapy (ART) in the UK. Methods: The analysis used data from the PIVOT trial in which HIV-positive adults with suppressed viral load for ≥24 weeks on combination ART were randomised to maintain OT or to a strategy of PI monotherapy with prompt return to combination therapy if viral load rebounded. A cost-effectiveness analysis including long-term modelling was conducted. Main outcomes included UK National Health Service (NHS) costs and quality-adjusted life-years (QALYs) with comparative results presented as incremental cost-effectiveness ratios. Results: PI monotherapy was cost saving as a result of large savings in ART drug costs while being no less effective in terms of QALYs in the within-trial analysis and marginally less effective with lifetime modelling. In the base-case analysis over 3 years, the incremental total cost per patient was −£6424.11 (95 % confidence interval −7418.84 to −5429.38) and incremental QALYs were 0.0051 (95 % CI −0.0479 to 0.0582), resulting in PI monotherapy ‘dominating’ OT. Multiple scenario analyses found that PI monotherapy was cost saving with no marked differences in QALYs. Modelling of lifetime costs and QALYs showed that PI monotherapy was associated with significant cost savings and was marginally less effective; PI monotherapy was cost effective at accepted cost-effectiveness thresholds in all but one scenario analysis. Conclusions: Under most assumptions, PI monotherapy appears to be a cost-effective treatment strategy compared with OT for HIV-infected patients who have achieved sustained virological suppression.