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Background: Health organizations the world over are required to set priorities and allocate resources within the constraint of limited funding. However, decision makers may not be well equipped to make explicit rationing decisions and as such often rely on historical or political resource allocation processes. One economic approach to priority setting which has gained momentum in practice over the last three decades is program budgeting and marginal analysis (PBMA). Methods: This paper presents a detailed step by step guide for carrying out a priority setting process based on the PBMA framework. This guide is based on the authors' experience in using this approach primarily in the UK and Canada, but as well draws on a growing literature of PBMA studies in various countries. Results: At the core of the PBMA approach is an advisory panel charged with making recommendations for resource re-allocation. The process can be supported by a range of 'hard' and 'soft' evidence, and requires that decision making criteria are defined and weighted in an explicit manner. Evaluating the process of PBMA using an ethical framework, and noting important challenges to such activity including that of organizational behavior, are shown to be important aspects of developing a comprehensive approach to priority setting in health care. Conclusion: Although not without challenges, international experience with PBMA over the last three decades would indicate that this approach has the potential to make substantial improvement on commonly relied upon historical and political decision making processes. In setting out a step by step guide for PBMA, as is done in this paper, implementation by decision makers should be facilitated. [ABSTRACT FROM AUTHOR]

Objectives: It has been estimated that vaccines can accrue a relatively large part of their value from patient and carer productivity. Yet, productivity value is not commonly or consistently considered in health economic evaluations of vaccines in several high-income countries. To contribute to a better understanding of the potential impact of including productivity value on the expected cost-effectiveness of vaccination, we illustrate the extent to which the incremental costs would change with and without productivity value incorporated. Methods: For two vaccines currently under development, one against Cloistridioides difficile (C. difficile) infection and one against respiratory syncytial disease (RSV), we estimated their incremental costs with and without productivity value included and compared the results. Results: In this analysis, reflecting a UK context, a C. difficile vaccination programme would prevent £12.3 in productivity costs for every person vaccinated. An RSV vaccination programme would prevent £49 in productivity costs for every vaccinated person. Conclusions: Considering productivity costs in future cost-effectiveness analyses of vaccines for C. difficile and RSV will contribute to better-informed reimbursement decisions from a societal perspective. [ABSTRACT FROM AUTHOR]

Background: The value of a life is regularly monetised by government departments for informing resource allocation. Guidance documents indicate how economic evaluation should be conducted, often specifying precise values for different impacts. However, we find different values of life and health are used in analyses by departments within the same government despite commonality in desired outcomes. This creates potential inconsistencies in considering trade-offs within a broader public sector spending budget. We provide evidence to better inform the political process and to raise important issues in assessing the value of public expenditure across different sectors. Methods: Our document analysis identifies thresholds, explicitly or implicitly, as observed in government-related publications in the following public sectors: health, social care, transport, and environment. We include both demand-side and supply-side thresholds, understood as societies' and governments' willingness to pay for health gains. We look at key countries that introduced formal economic evaluation processes early on and have impacted other countries' policy development: Australia, Canada, Japan, New Zealand, the Netherlands, and the United Kingdom. We also present a framework to consider how governments allocate resources across different public services. Results: Our analysis supports that identifying and describing the Value of a Life from disparate public sector activities in a manner that facilitates comparison is theoretically meaningful. The optimal allocation of resources across sectors depends on the relative position of benefits across different attributes, weighted by the social value that society puts on them. The value of a Quality-Adjusted Life Year is generally used as a demand-side threshold by Departments of transport and environment. It exceeds those used in health, often by a large enough proportion to be a multiple thereof. Decisions made across departments are generally based on an unspecified rationing rule. Conclusions: Comparing government expenditure across different public sector departments, in terms of the value of each department outcome, is not only possible but also desirable. It is essential for an optimal resource allocation to identify the relevant social attributes and to quantify the value of these attributes for each department. [ABSTRACT FROM AUTHOR]

Background: The use of neonatal screening for cystic fibrosis is widely debated in the United Kingdom and elsewhere, but the evidence available to inform policy is limited. This paper explores the cost-effectiveness of adding screening for cystic fibrosis to an existing routine neonatal screening programme for congenital hypothyroidism and phenylketonuria, under alternative scenarios and assumptions. Methods: The study is based on a decision model comparing screening to no screening in terms of a number of outcome measures, including diagnosis of cystic fibrosis, life-time treatment costs, life years and QALYs gained. The setting is a hypothetical UK health region without an existing neonatal screening programme for cystic fibrosis. Results: Under initial assumptions, neonatal screening (using an immunoreactive trypsin/DNA two stage screening protocol) costs £5,387 per infant diagnosed, or £1.83 per infant screened (1998 costs). Neonatal screening for cystic fibrosis produces an incremental cost-effectiveness of £6,864 per QALY gained, in our base case scenario (an assumed benefit of a 6 month delay in the emergence of symptoms). A difference of 11 months or more in the emergence of symptoms (and mean survival) means neonatal screening is both less costly and produces better outcomes than no screening. Conclusion: Neonatal screening is expensive as a method of diagnosis. Neonatal screening may be a cost-effective intervention if the hypothesised delays in the onset of symptoms are confirmed. Implementing both antenatal and neonatal screening would undermine potential economic benefits, since a reduction in the birth incidence of cystic fibrosis would reduce the cost-effectiveness of neonatal screening. [ABSTRACT FROM AUTHOR]

Background: This paper addresses the question of what a reasonable price for an orphan drug is. The research proposes a way to adjust an established payer/HTA body incremental cost-effectiveness threshold (CET) to take account of differences in patient populations and costs of research and development in order to sustain prices that generate rates of return from investments in developing orphan drugs that are no greater than the industry average. Methods: We investigated the cost of conducting research for orphan drugs as compared to non-orphan drugs, as well as patient population sizes targeted by orphans and non-orphans. We provided an empirical illustration based on novel drug approvals of orphan and non-orphan drugs of the FDA between 2011 and 2015 (N = 182). Results: Using, for illustration, the NICE incremental CET (£20 K per QALY) as an anchor and adjusting by R&D costs and expected market revenue, we estimated the adjusted reasonable CET for orphan drugs to be £39.1 K per QALY at the orphan population cut-off and £78.3 K per QALY at the orphan population mid-point. For ultra-orphan drugs the adjusted CET was £937.1 K. Conclusions: We propose one general method for establishing a reasonable price for an orphan drug, based on the proposition that rates of return for investments in developing orphan drugs should not be greater than the industry average. More research is required on data and assumptions, but with the data and assumptions we use, we find that in order to secure such a reasonable price for an orphan drug, the CET for orphans would need to be higher. This could be one approach for establishing the maximum allowable price society should be willing to pay, although decision-makers may still wish to negotiate a lower price, or refuse to pay such a premium over the value-based price in order to treat these groups of patients. [ABSTRACT FROM AUTHOR]

Introduction: Heart failure (HF) is an unusual heart function that causes reduction in cardiac or pulmonary output. Cardiac resynchronization therapy (CRT) is a mechanical device that helps to recover ventricular dysfunction by pacing the ventricles. This study planned to systematically review cost-effectiveness of CRT combined with an implantable cardioverter-defibrillator (ICD) versus ICD in patients with HF. Methods: We used five databases (NHS Economic Evaluation Database, Cochrane Library, Medline, PubMed, and Scopus) to systematically reviewed studies published in the English language on the cost-effectiveness of CRT with defibrillator (CRT-D) Vs. ICD in patients with HF over 2000 to 2020. Consolidated Health Economic Evaluation Reporting Standards (CHEERS) checklist was applied to assess the quality of the selected studies. Results: Five studies reporting the cost-effectiveness of CRT-D vs ICD were finally identified. The results revealed that time horizon, direct medical costs, type of model, discount rate, and sensitivity analysis obviously mentioned in almost all studies. All studies used quality-adjusted life years (QALYs) as an effectiveness measurement. The highest and the lowest Incremental cost-effectiveness ratio (ICER) were reported in the USA ($138,649per QALY) and the UK ($41,787per QALY), respectively. Conclusion: Result of the study showed that CRT-D compared to ICD alone was the most cost-effective treatment in patients with HF. [ABSTRACT FROM AUTHOR]

Background A healthy diet is associated with reduced risk of diabetes, cardiovascular disease and cancer. The study aimed to evaluate the cost-effectiveness of a universal strategy to promote healthy diet through brief intervention in primary care. Methods The research was informed by a systematic review of randomised trials which found that brief interventions in primary care may be associated with a 0.5 portion per day increase in fruit and vegetable consumption. A Markov model that included five long-term conditions (diabetes, coronary heart disease, stroke, colorectal cancer and depression) was developed. Empirical data from a large cohort of United Kingdom-based participants sampled from the Clinical Practice Research Datalink populated the model. Simulations compared an intervention promoting healthy diet over 5 years in healthy adults, and standard care in which there was no intervention. The annual cost of intervention, in the base case, was one family practice consultation per participant year. Health service costs were included and the model adopted a lifetime perspective. The primary outcome was net health benefit in quality adjusted life years (QALYs). Results A cohort of 262,704 healthy participants entered the model. Intervention was associated with an increase in life years lived free from physical disease of 41.9 (95% confidence interval - 17.4 to 101.0) per 1,000 participants entering the model (probability of increase 88.0%). New incidences of disease states were reduced by 28.4 (18.7 to 75.8) per 1,000, probability reduced 84.6%. Discounted incremental QALYs were 4.3 (-8.8 to 18.0) per 1,000, while incremental costs were £139,755 (£60,466 to 220,059) per 1,000. Net health benefits at £30,000 per QALY were -0.32 (-13.8 to 13.5) QALYs per 1,000 participants (probability cost-effective 47.9%). When the intervention was restricted to adults aged 50 to 74 years, net health benefits were 2.94 (-21.3 to 26.4) QALYs per 1000, probability increased 59.0%. Conclusions A universal strategy to promote healthy diet through brief intervention in primary care is unlikely to be cost-effective, even when delivered at low unit cost. A targeted strategy aimed at older individuals at higher risk of disease might be more cost-effective. More effective dietary change interventions are needed. [ABSTRACT FROM AUTHOR]

Background: To estimate life years and quality-adjusted life years (QALYs) lost and the economic burden of aneurysmal subarachnoid haemorrhage (aSAH) in the United Kingdom including healthcare and non-healthcare costs from a societal perspective. Methods: All UK residents in 2005 with aSAH (International Classification of Diseases 10th revision (ICD-10) code I60). Sex and age-specific abridged life tables were generated for a general population and aSAH cohorts. QALYs in each cohort were calculated adjusting the life tables with health-related quality of life (HRQL) data. Healthcare costs included hospital expenditure, cerebrovascular rehabilitation, primary care and community health and social services. Non-healthcare costs included informal care and productivity losses arising from morbidity and premature death. Results: A total of 80,356 life years and 74,807 quality-adjusted life years were estimated to be lost due to aSAH in the UK in 2005. aSAH costs the National Health Service (NHS) £168.2 million annually with hospital inpatient admissions accounting for 59%, community health and social services for 18%, aSAH-related operations for 15% and cerebrovascular rehabilitation for 6% of the total NHS estimated costs. The average per patient cost for the NHS was estimated to be £23,294. The total economic burden (including informal care and using the human capital method to estimate production losses) of a SAH in the United Kingdom was estimated to be £510 million annually. Conclusion: The economic and disease burden of aSAH in the United Kingdom is reported in this study. Decision-makers can use these results to complement other information when informing prevention policies in this field and to relate health care expenditures to disease categories. [ABSTRACT FROM AUTHOR]

Background: The absence of trial evidence makes it impossible to determine whether or not mass screening for lung cancer would be cost effective and, indeed, whether a clinical trial to investigate the problem would be justified. Attempts have been made to resolve this issue by modelling, although the complex models developed to date have required more real-world data than are currently available. Being founded on unsubstantiated assumptions, they have produced estimates with wide confidence intervals and of uncertain relevance to the United Kingdom. Method: I develop a simple, deterministic, model of a screening regimen potentially applicable to the UK. The model includes only a limited number of parameters, for the majority of which, values have already been established in non-trial settings. The component costs of screening are derived from government guidance and from published audits, whilst the values for test parameters are derived from clinical studies. The expected health gains as a result of screening are calculated by combining published survival data for screened and unscreened cohorts with data from Life Tables. When a degree of uncertainty over a parameter value exists, I use a conservative estimate, i.e. one likely to make screening appear less, rather than more, cost effective. Results: The incremental cost effectiveness ratio of a single screen amongst a high-risk male population is calculated to be around £14,000 per quality-adjusted life year gained. The average cost of this screening regimen per person screened is around £200. It is possible that, when obtained experimentally in any future trial, parameter values will be found to differ from those previously obtained in non-trial settings. On the basis both of differing assumptions about evaluation conventions and of reasoned speculations as to how test parameters and costs might behave under screening, the model generates cost effectiveness ratios as high as around £20,000 and as low as around £7,000. Conclusion: It is evident that eventually being able to identify a cost effective regimen of CT screening for lung cancer in the UK is by no means an unreasonable expectation. [ABSTRACT FROM AUTHOR]

Background: We assessed the cost-effectiveness of adding a quadrivalent (6/11/16/18) human papillomavirus (HPV) vaccine to the current screening programme in the UK compared to screening alone. Methods: A Markov model of the natural history of HPV infection incorporating screening and vaccination was developed. A vaccine that prevents 98% of HPV 6, 11, 16 and 18 -associated disease, with a lifetime duration and 85% coverage, in conjunction with current screening was considered. Results: Vaccination with screening, compared to screening alone, was associated with an incremental cost-effectiveness ratio of £21,059 per quality adjusted life year (QALY) and £34,687 per life year saved (LYS). More than 400 cases of cervical cancer, 6700 cases of cervical intraepithelial neoplasia and 4750 cases of genital warts could be avoided per 100,000 vaccinated girls. Results were sensitive to assumptions about the need for a booster, the duration of vaccine efficacy and discount rate. Conclusion: These analyses suggest that adding a quadrivalent HPV vaccine to current screening in the UK could be a cost-effective method for further reducing the burden of cervical cancer. [ABSTRACT FROM AUTHOR]