Office of Health Economics

Objectives: Flash glucose monitoring for patients with T1 diabetes avoids frequent painful finger-prick testing, thus potentially improving frequency of glucose self-monitoring. Our study aimed to explore experiences of young people using Freestyle Libre sensors and their parents, and to identify benefits and challenges to National Health Service (NHS) staff of its adoption in their care provision. Participants: Young people with T1 diabetes, their parents and healthcare professionals were interviewed between February and December 2021. Participants were recruited via social media and through NHS diabetes clinic staff. Design: Semistructured interviews were conducted online and analysed using thematic methods. Staff themes were mapped onto normalisation process theory (NPT) constructs. Results: Thirty-four participants were interviewed: 10 young people, 14 parents and 10 healthcare professionals. Young people reported that life was much easier since changing to flash glucose monitoring, increasing confidence and independence to manage their condition. Parents' quality of life improved and they appreciated access to real-time data. Using the NPT concepts to understand how technology was integrated into routine care proved useful; health professionals were very enthusiastic about flash glucose monitoring and coped with the extra data load to facilitate more tailored patient support within and between clinic visits. Conclusion: This technology empowers young people and their parents to understand their diabetes adherence more completely; to feel more confident about adjusting their own care between clinic appointments; and provides an improved interactive experience in clinic. Healthcare teams appear committed to delivering improving technologies, acknowledging the challenge for them to assimilate new information required to provide expert advice.

Lack of credibility and trust in fund managers has been highlighted as one of the key reasons why people do not join health insurance schemes in low- and middle-income countries, especially in Africa. This work investigates the impact of corruption on households' willingness to participate and pay for health insurance in Sierra Leone. A discrete choice experiment (DCE) method was used to elicit households' willingness to participate in a health insurance scheme with different attributes. The data were collected from 1458 representative households working in the informal sector of the Northern and Western regions. We explore the relationship between household characteristics and experienced (respectively, perceived) corruption with binary and ordered logit models. We use a Mixed Logit model to estimate the association between corruption and participation in a Health Insurance Scheme (HIS) and households' willingness to pay for a HIS. We find that corruption decreases participation in a public HIS and the willingness to pay for it. Our results highlight the perverse spillover effects of corruption. Not only does corruption hinder the effectiveness of healthcare systems and, thus, worsen health outcomes. It also undermines the willingness to pay for them, jeopardizing the sustainability of healthcare systems in the countries that need them most.

Background The EQ VAS component of the EQ-5D questionnaire has been used to assess patients’ valuation of their own health besides its use for self-reporting of overall health status. The objective of the present study was to identify patients’ valuation of EQ-5D-3L health states using the EQ VAS in different patient groups over time and in comparison to the general population. Methods Data were obtained from patients from nine National Quality Registers (n = 172,070 patients) at baseline and at 1-year follow-up and compared with data from the general population (n = 41,761 participants). The correlation between EQ VAS scores and EQ-5D-3L index based on the Swedish experience-based VAS value set was assessed. Ordinary least squares (OLS) regression models were used to determine the association between EQ-5D-3L dimensions and EQ VAS valuation. Results EQ VAS scores showed consistency with severity of health states both at baseline and at 1-year follow-up in the nine selected EQ-5D-3L health states. The regression models showed mostly consistent decrements by severity levels in each dimension at both time points and similar to the general population. The dimension mainly associated with inconsistency was the self-care severity level three. Problems in the anxiety/depression dimension had the largest impact on overall health status in most of the patient groups and the general population. Conclusion The study has demonstrated the important role EQ VAS can play in revealing patients’ valuation of their health and showed the variation in valuation of EQ-5D-3L dimensions and levels of severity across different patient groups.

Background: BMT CTN 1101 was a Phase III randomized controlled trial comparing reduced intensity conditioning followed by double unrelated umbilical cord blood (UCB) versus HLA-haploidentical related donor bone marrow (haplo-BM) transplantation for patients with high-risk hematologic malignancies. Objective: The objective of this study is to report the results of a parallel cost-effectiveness analysis. Study design: Three hundred sixty-eight patients were randomized to unrelated UCB (n=186) or haplo-BM (n=182) transplant. We estimated healthcare utilization and costs using propensity score-matched BMT patients from the OptumLabsⓇ Data Warehouse for trial participants <65 years and Medicare claims for participants ≥65 years. Weibull models were used to estimate 20-year survival. EQ-5D surveys by trial participants were used estimate Quality-Adjusted Life Years (QALYs). Results: At 5-year follow-up, survival was 42% for haplo-BM versus 36% for UCB (P=.06). Over a 20-year time horizon, haplo-BM is expected to be more effective (+0.63 QALY) and more costly +$118,953) for persons under 65. For those over 65, haplo-BM is expected to be more effective and less costly. In one-way uncertainty analyses, for persons <65, the cost per QALY result was most sensitive to life years and health state utilities. For persons ≥65, life years were more influential than costs and health state utilities. Conclusion: Compared to UCB, haplo-BM was moderately cost-effective for patients aged <65 years, and less costly and more effective for persons ≥65 years. Haplo-BM is a fair value choice for commercially insured patients with high-risk leukemia and lymphoma who require HCT. For Medicare enrollees, haplo-BM is a preferred choice when considering costs and outcomes.

An increasing number of prospective parents are experiencing infertility along with associated negative impacts on mental health and life satisfaction that can extend across a network of individuals and family members. Assistive reproductive technologies (ART) can help prospective parents achieve their parenthood goals but, like any health technology, they must demonstrate acceptable 'value for money' to qualify for public funding. We argue that current approaches to understanding the value of ART, including quality-adjusted life-year (QALY) gains based on changes in health-related quality of life (HRQOL) and, more often, cost per live birth, are too narrow to capture the full impact of unmet parenthood goals and ART. We see a fundamental disconnect between measures of HRQOL and broader measures of wellbeing associated with met and unmet parenthood goals. We also suggest that simple concepts such as 'patient' and 'carer' are of limited applicability in the context of ART, where 'spillovers' extend across a wide network of individuals, and the person receiving treatment is often not the infertile individual. Consideration of individual and societal wellbeing beyond HRQOL is necessary to understand the full range of negative impacts associated with unmet parenthood goals and the corresponding positive impacts of successful ART. We suggest moving towards a wellbeing perspective on value to achieve a fuller understanding of value and promote cross-sector allocative efficiency.

Objective: This article estimates the life-cycle value of risperidone as representative of second-generation antipsychotics (SGA) relative to haloperidol (first-generation antipsychotics). Methods: We estimated the number of patients treated with risperidone in Sweden and the United Kingdom, from 1994 to 2017, using data of usage and volume sales. We collected data from the literature on the effectiveness (quality-adjusted life-years per patient per year), direct costs (health services), and indirect costs (productivity) of risperidone and haloperidol. We proxied the incremental value added by the new class (SGA) using a comparator from the inferior class. Next, we modeled the life-cycle uptake of risperidone to estimate the life-cycle incremental cost (ie, direct, indirect, and medicine costs), incremental quality-adjusted life-years, and net monetary benefit of risperidone. We also assessed the life-cycle distribution of the social surplus between the payer (consumer surplus) and the innovator (producer surplus). Results: For the United Kingdom, consumer surplus represents around 72% of the total surplus before patent expiration and around 95% after patent expiration. For Sweden, the consumer surplus represents around 94% of the total surplus before patent expiration and around 99% after generic competition. Conclusion: These results suggest that the value added by SGAs to the system is higher than the expected value estimated using cost-effectiveness analysis at launch. Pricing and reimbursement decisions could recognize the full life cycle of value of innovative medicines. This not only presents a challenge of estimation but also of assessing the appropriate division of shares of social value.

Health technology assessments (HTAs) of vaccines typically focus on the direct health benefits to individuals and healthcare systems. COVID-19 highlighted the widespread societal impact of infectious diseases and the value of vaccines in averting adverse clinical consequences and in maintaining or resuming social and economic activities. Using COVID-19 as a case study, this research work aimed to set forth a conceptual framework capturing the broader value elements of vaccines and to identify appropriate methods to quantify value elements not routinely considered in HTAs. A two-step approach was adopted, combining a targeted literature review and three rounds of expert elicitation based on a modified Delphi method, leading to a conceptual framework of 30 value elements related to broader health effects, societal and economic impact, public finances, and uncertainty value. When applying the framework to COVID-19 vaccines in post-pandemic settings, 13 value elements were consensually rated highly important by the experts for consideration in HTAs. The experts reviewed over 10 methods that could be leveraged to quantify broader value elements and provided technical forward-looking recommendations. Limitations of the framework and the identified methods were discussed. This study supplements ongoing efforts aimed towards a broader recognition of the full societal value of vaccines.

Introduction There are increasing numbers of estimates of opportunity cost to inform the setting of thresholds as ceiling cost-per-quality-adjusted life year (QALY) ratios. To understand their ability to inform policy making, we need to understand the degree of uncertainty surrounding these estimates. In particular, do estimates provide sufficient certainty that the current policy “rules” or “benchmarks” need revision? Does the degree of uncertainty around those estimates mean that further evidence generation is required? Methods We analyse uncertainty and methods from three papers that focus on the use of data from the NHS in England to estimate opportunity cost. All estimate the impact of expenditure on mortality in cross-sectional regression analyses and then translate the mortality elasticities into cost-per-QALY thresholds using the same assumptions. All three discuss structural uncertainty around the regression analysis, and report parameter uncertainty derived from their estimated standard errors. However, only the initial, seminal, paper explores the structural uncertainty involved in moving from the regression analysis to a threshold. We discuss the elements of structural uncertainty arising from the assumptions that underpin the translation of elasticities to thresholds and seek to quantify the importance of some of the effects. Results We find several sets of plausible structural assumptions that would place the threshold estimates from these studies within the current National Institute for Health and Care Excellence (NICE) range of £20,000 to £30,000 per QALY. Heterogeneity, an additional source of uncertainty from variability, is also discussed and reported. Discussion Lastly, we discuss how decision uncertainty around the threshold could be reduced, setting out what sort of additional research is required, notably in improving estimates of disease burden and of the impact of health expenditure on quality of life. Given the likely value to policy makers of this research it should be a priority for health system research funding.

We examine how expenditure changes at retirement during an institutionally and economically uncertain period when a series of pension reforms and cuts were implemented. Overall, we fail to confirm that consumption declines at retirement using data from Greece (2008–2018). Any estimated declines come from turbulent years when major pension cuts were applied. Expenditure drops at retirement were due to pension income shocks, especially for those who were particularly dependent on pension income. Further checks support the presence of an income shock mechanism for retirees who are relatively more treated during the crisis sub-period. Given an aging population and the ongoing global turbulence, our results offer valuable insights.

IntroductionPreference-weighted measures (PWMs)—also referred to as preference-based measures in the literature—of health status/health-related quality of life plays an essential role in estimating quality-adjusted life-years (QALY) for use in economic evaluations of healthcare products and interventions. However, as PWMs are first and foremost intended to accurately reflect respondent health status, they should ideally demonstrate good psychometric properties for the population in question. This study aimed to systematically review published evidence on the measurement properties of commonly used PWMs for children and adolescents.Methods Three electronic databases (PubMed, Medline, and PsycINFO) were searched for articles assessing the psychometric properties (content validity, construct validity—including convergent validity and known-group validity, test-retest reliability, and responsiveness) of the PWMs of interest (AQoL-6D, CHU9D, HUI2, HUI3, and EQ-5D-Y). The COsensus-based Standards for the selection of health Measurement INstruments methodology (COSMIN) guidelines were used to assess (a) the methodological quality of the studies included and (b) the psychometric performance of the instruments covered. Data were analysed overall as well as by population (country and disease group) and perspective (self-report or proxy-report). The study protocol was registered in the International Prospective Register of Systematic Reviews (PROSPERO) database (CRD42021277296).ResultsIn total, 53 articles were included in this systematic review. Health Utilities Index (HUI) was tested only in patient populations, CHU9D was most frequently tested in general population samples, while EQ-5D-Y was tested in both populations. Overall, there was high-quality evidence supporting sufficient construct validity for all instruments except AQoL-6D. Evidence supporting test-retest and responsiveness was scarce. There was high-quality evidence supporting sufficient responsiveness of HUI2 and HUI3, and inconsistent test-retest reliability of CHU9D and EQ-5D-Y. Evidence for content validity was minimal and therefore not extracted and synthesized for any PWMs.Conclusion This review provides updated evidence on the measurement properties of existing generic PWMs for children and adolescents. High-quality evidence for all relevant psychometric properties and across a range of populations was not available for any of the instruments included, indicating that further work is needed in this direction. This study has identified some of the most noticeable evidence gaps for each of the individual measures. Users can use this information to guide their decision on the choice of PWM to administer.

Background Empirical estimates of health system opportunity costs have been suggested as a basis for the cost-effectiveness threshold to use in Health Technology Assessment. Econometric methods have been used to estimate these in several countries based on data on spending and mortality. This study examines empirical evidence on four issues: non-linearity of the relationship between spending and mortality; the inclusion of outcomes other than mortality; variation in the efficiency with which expenditures generate health outcomes; and the relationship among efficiency, mortality rates and outcome elasticities. Methods Quantile Regression is used to examine non-linearities in the relationship between mortality and health expenditures along the mortality distribution. Data Envelopment Analysis extends the approach, using multiple measures of health outcomes to measure efficiency. These are applied to health expenditure data from 151 geographical units (Primary Care Trusts) of the National Health Service in England, across eight different clinical areas (Programme Budget Categories), for 3 fiscal years from 2010/11 to 2012/13. Results The results suggest differences in efficiency levels across geographical units and clinical areas as to how health resources generate outcomes, which indicates the capacity to adjust to a decrease in health expenditure without affecting health outcomes. Moreover, efficient units have lower absolute levels of mortality elasticity to health expenditure than inefficient ones. Conclusions The policy of adopting thresholds based on estimates of a single system-wide cost-effectiveness threshold assumes a relationship between expenditure and health outcomes that generates an opportunity cost estimate which applies to the whole system. Our evidence of variations in that relationship and therefore in opportunity costs suggests that adopting a single threshold may exacerbate the efficiency and equity concerns that such thresholds are designed to counter. In most health care systems, many decisions about provision are not made centrally. Our analytical approach to understanding variability in opportunity cost can help policy makers target efficiency improvements and set realistic targets for local and clinical area health improvements from increased expenditure.

Health systems internationally must prepare for a future of genetic/genomic testing to inform healthcare decision-making while creating research opportunities. High functioning testing services will require additional considerations and health system conditions beyond traditional diagnostic testing. Based on a literature review of good practices, key informant interviews, and expert discussion, this article attempts to synthesize what conditions are necessary, and what good practice may look like. It is intended to aid policymakers and others designing future systems of genome-based care and care prevention. These conditions include creating communities of practice and healthcare system networks; resource planning; across-region informatics; having a clear entry/exit point for innovation; evaluative function(s); concentrated or coordinated service models; mechanisms for awareness and care navigation; integrating innovation and healthcare delivery functions; and revisiting approaches to financing, education and training, regulation, and data privacy and security. The list of conditions we propose was developed with an emphasis on describing conditions that would be applicable to any healthcare system, regardless of capacity, organizational structure, financing, population characteristics, standardization of care processes, or underlying culture.

Background Current guidelines recommend that patients with obesity hypoventilation syndrome (OHS) are electively admitted for inpatient initiation of home non-invasive ventilation (NIV). We hypothesised that outpatient NIV setup would be more cost-effective. Methods Patients with stable OHS referred to six participating European centres for home NIV setup were recruited to an open-labelled clinical trial. Patients were randomised via web-based system using stratification to inpatient setup, with standard fixed level NIV and titrated during an attended overnight respiratory study or outpatient setup using an autotitrating NIV device and a set protocol, including home oximetry. The primary outcome was cost-effectiveness at 3 months with daytime carbon dioxide (PaCO 2 ) as a non-inferiority safety outcome; non-inferiority margin 0.5 kPa. Data were analysed on an intention-to-treat basis. Health-related quality of life (HRQL) was measured using EQ-5D-5L (5 level EQ-5D tool) and costs were converted using purchasing power parities to £(GBP). Results Between May 2015 and March 2018, 82 patients were randomised. Age 59±14 years, body mass index 47±10 kg/m ² and PaCO 2 6.8±0.6 kPa. Safety analysis demonstrated no difference in ∆PaCO 2 (difference −0.27 kPa, 95% CI −0.70 to 0.17 kPa). Efficacy analysis showed similar total per-patient costs (inpatient £2962±£580, outpatient £3169±£525; difference £188.20, 95% CI −£61.61 to £438.01) and similar improvement in HRQL (EQ-5D-5L difference −0.006, 95% CI −0.05 to 0.04). There were no differences in secondary outcomes. Discussion There was no difference in medium-term cost-effectiveness, with similar clinical effectiveness, between outpatient and inpatient NIV setup. The home NIV setup strategy can be led by local resource demand and patient and clinician preference. Trial registration numbers NCT02342899 and ISRCTN51420481 .

Each year there are over 300 natural disasters globally with millions of victims that cost economic losses near USD$100 billion. In the context of climate change, an emerging literature linking extreme weather events to HIV infections suggests that efforts to control the HIV epidemic could be under threat. We used Demographic and Health Survey (DHS) data collected during the 2015–2016 harsh drought that affected several areas of Malawi to provide new evidence on the effect of an unanticipated economic shock on sexual behaviours of young women and men. We find that amongst women employed in agriculture, a six-months drought doubles their likelihood of engaging in transactional sex compared to women who were not affected by the drought and increases their likelihood of having a sexually transmitted infections (STI) by 48% in the past 12 months. Amongst men employed outside of agriculture, drought increases by 50% the likelihood of having a relationship with a woman engaged in transactional sex. These results suggest that women in agriculture experiencing economic shocks as a result of drought use transactional sex with unaffected men, i.e. men employed outside agriculture, as a coping mechanism, exposing themselves to the risk of contracting HIV. The effect was especially observed among non-educated women. A single drought in the last five years increases HIV prevalence in Malawi by around 15% amongst men and women. Overall, the results confirm that weather shocks are important drivers of risky sexual behaviours of young women relying on agriculture in Africa. Further research is needed to investigate the most adequate formal shock-coping strategies to be implemented in order to limit the negative consequences of natural disasters on HIV acquisition and transmission.

The objectives of this research were to produce a macro-level overview of the global COVID-19 burden and estimate the value of access to COVID-19 vaccines. A targeted literature review collated evidence of the burden. Linear modelling and data analysis estimated the health and economic effects of COVID-19 vaccines delivered in 2021, and whether additional value could have been achieved with broader and more equitable access. By 1 December 2020, there had been an estimated 17 million excess deaths due to COVID-19. Low-income countries allocated more than 30% of their healthcare budgets to COVID-19, compared to 8% in high-income countries. All country income groups experienced gross domestic product (GDP) growth lower than predicted in 2020. If all 92 countries eligible for COVAX Advance Market Committee (AMC), access had reached 40% vaccination coverage in 2021, 120% more excess deaths would have been averted, equivalent to USD 5 billion (109) in savings to healthcare systems. Every USD spent by advanced economies on vaccinations for less advanced economies averted USD 28 of economic losses in advanced economies and USD 29 in less advanced economies. The cost to high-income countries when not all countries are vaccinated far outweighs the cost of manufacturing and distributing vaccines globally.

Objectives In situations of excess demand for healthcare, treating one patient means losing the opportunity to treat another. Therefore, each decision bears an opportunity cost. Nevertheless, when assessing the value of health technologies, these opportunity costs are not always fully considered. We present a pragmatic approach for conceptualizing vaccines’ health system capacity value when considering opportunity costs. Methods Our approach proxies opportunity costs through the net monetary benefit forgone as scarce healthcare resources are used to treat a vaccine-preventable disease instead of a patient from the waiting list. We apply this approach to cost the resource “hospital beds” for 3 different scenarios of excess demand. Empirically, we estimate the opportunity costs saved for 4 selected vaccination programs from the national schedule in England during a hypothetical scenario of long-lasting excess demand induced by the pandemic. Results The opportunity cost avoided through vaccination rises with excess demand for treatment. When treating an acute vaccine-preventable outcome is a suboptimal choice compared with treating elective patients, preventing a vaccine-preventable disease from blocking a hospital bed generates opportunity cost savings of approximately twice the direct costs saved by avoiding vaccine-preventable hospitalizations. Conclusions Policy makers should be aware that, in addition to preventing the outcome of interest, vaccines and other preventative health technologies deliver value in maintaining regular healthcare services and clearing the pent-up demand from the pandemic. Therefore, health system capacity value should be a key-value element in health technology assessment. Existing and potential future vaccination programs deliver more value than hitherto quantified.