The use of cost-effectiveness analysis for health benefit package design - should countries follow a sectoral, incremental or hybrid approach?

BACKGROUND: Countries around the world are increasingly rethinking the design of their health benefit package to achieve universal health coverage. Countries can periodically revise their packages on the basis of sectoral cost-effectiveness analyses, i.e. by evaluating a broad set of services against a 'doing nothing' scenario using a budget constraint. Alternatively, they can use incremental cost-effectiveness analyses, i.e. to evaluate specific services against current practice using a threshold. In addition, countries may employ hybrid approaches which combines elements of sectoral and incremental cost-effectiveness analysis - a country may e.g. not evaluate the comprehensive set of all services but rather relatively small sets of services targeting a certain condition. However, there is little practical guidance for countries as to which kind of approach they should follow. METHODS: The present study was based on expert consultation. We refined the typology of approaches of cost-effectiveness analysis for benefit package design, identified factors that should be considered in the choice of approach, and developed recommendations. We reached consensus among experts over the course of several review rounds. RESULTS: Sectoral cost-effectiveness analysis is especially suited in contexts with large allocative inefficiencies in current service provision and can, in theory, realize large efficiency gains. However, it may be challenging to implement a comprehensive redesign of the package in practice. Incremental cost-effectiveness analysis is especially relevant in contexts where specific new services may impact the sustainability of the health system. It may potentially support efficiency improvement, but its focus has typically been on new services while existing inefficiencies remain unchallenged. The use of hybrid approach may be a way forward to address the strengths and weaknesses of sectoral and incremental analysis areas. Such analysis may be especially useful to target disease areas with suspected high inefficiencies in service provision, and would then make good use of the available research capacity and be politically rewarding. However, disease-specific analyses bear the risk of not addressing resource allocation inefficiencies across disease areas. CONCLUSIONS: Countries should carefully select their approach of cost-effectiveness analyses for benefit package design, based on their decision-making context.

Estimating and Comparing Health and Financial Risk Protection Outcomes in Economic Evaluations.

OBJECTIVES: Improving health and financial risk protection (FRP, the prevention of medical impoverishment) and their distributions is a major objective of national health systems. Explicitly describing FRP and disaggregated (eg, across socioeconomic groups) impact of health interventions in economic evaluations can provide decision makers with a broader set of health and financial outcomes to compare and prioritize interventions against each other. METHODS: We propose methods to synthesize such a broader set of outcomes by estimating and comparing the distributions in both health and FRP benefits procured by health interventions. We build on benefit-cost analysis frameworks and utility-based models, and we illustrate our methods with the case study of universal public finance (financing by government regardless of whom an intervention is targeting) of disease treatment in a low- and middle-income country setting. RESULTS: Two key findings seem to emerge: FRP is critical when diseases are less lethal (eg, case fatality rates <1% or so), and quantitative valuation of inequality aversion across income groups matters greatly. We recommend the use of numerous sensitivity analyses and that all distributional health and financial outcomes be first presented in a disaggregated form (before potential subsequent aggregation). CONCLUSIONS: Estimation approaches such as the one we propose provide explicit disaggregated considerations of equity, FRP, and poverty impact for the development of health sector policies, with high relevance for population-based preventive measures.

Public participation: healthcare rationing in the newspaper media.

BACKGROUND: It is impossible to meet all healthcare demands, but an open and fair rationing process may improve the public acceptability of priority setting in healthcare. Decision-making is subject to scrutiny by newspaper media, an important public institution and information source for discussions about rationing. In Norway, healthcare rationing has been subject to public debate both before and after the establishment of "The National System for Managed Introduction of New Health Technologies within the Specialist Health Service" (New Methods) in 2013. AIM: To describe and assess the development of the public debate on Norwegian healthcare rationing through three cases in print media. METHODS: We purposively sampled Norwegian newspaper articles between 2012 and 2018 concerning three reimbursement decisions in the Norwegian system. The reimbursement decisions were ipilimumab (Yervoy, n = 45) against metastatic melanoma, nivolumab (Opdivo, n = 23) against non-small cell lung cancer, and nusinersen (Spinraza, n = 68) against spinal muscular atrophy. Cases were analysed separately using the qualitative method of systematic text condensation. RESULTS: Our analysis highlighted four common themes-money, rationales, patient stories, and process-and a unique theme for each case. Ipilimumab was uniquely themed by rationing rejection, nivolumab by healthcare two-tiering, and Spinraza by patients' rights. We found wide media deliberation among a multitude of stakeholders in all cases. Perceptions of rationing were found to be chiefly aligned with previous empirical research. We found that the media reported more frequently on opposition to rationing compared to findings from previous studies on Norwegian healthcare decision-making attitudes. We think this was influenced by our selection of cases receiving extraordinary media attention, and from media sources being subject to political communication from special interest groups. CONCLUSION: We observed that the introduction of New Methods institutionalised Norwegian healthcare rationing and isolated the public debate into conversations between stakeholders and decision makers outside the political sphere. The findings from these three extraordinary debates are not generalisable and should be seen as a stakeholder learning opportunity regarding media coverage and engagement with expensive specialist healthcare decision-making in Norway.

The magnitude and perceived reasons for childhood cancer treatment abandonment in Ethiopia: from health care providers' perspective.

BACKGROUND: Treatment abandonment is one of major reasons for childhood cancer treatment failure and low survival rate in low- and middle-income countries. Ethiopia plans to reduce abandonment rate by 60% (2019-2023), but baseline data and information about the contextual risk factors that influence treatment abandonment are scarce. METHODS: This cross-sectional study was conducted from September 5 to 22, 2021, on the three major pediatric oncology centers in Ethiopia. Data on the incidence and reasons for treatment abandonment were obtained from healthcare professionals. We were unable to obtain data about the patients' or guardians' perspective because the information available in the cancer registry was incomplete to contact adequate number of respondents. We used a validated, semi-structured questionnaire developed by the International Society of Pediatric Oncology Abandonment Technical Working Group. We included all (N = 38) health care professionals (physicians, nurses, and social workers) working at these centers who had more than one year of experience in childhood cancer service provision (a universal sampling and 100% response rate). RESULTS: The perceived mean abandonment rate in Ethiopia is 34% (SE 2.5%). The risk of treatment abandonment is dependent on the type of cancer (high for bone sarcoma and brain tumor), the phase of treatment and treatment outcome. The highest risk is during maintenance and treatment failure or relapse for acute lymphoblastic leukemia, and during pre- or post-surgical phase for Wilms tumor and bone sarcoma. The major influencing risk factors in Ethiopia includes high cost of care, low economic status, long travel time to treatment centers, long waiting time, belief in the incurability of cancer and poor public awareness about childhood cancer. CONCLUSIONS: The perceived abandonment rate in Ethiopia is high, and the risk of abandonment varies according to the type of cancer, phase of treatment or treatment outcome. Therefore, mitigation strategies to reduce the abandonment rate should include identifying specific risk factors and prioritizing strategies based on their level of influence, effectiveness, feasibility, and affordability.

Reducing regional health inequality: a sub-national distributional cost-effectiveness analysis of community-based treatment of childhood pneumonia in Ethiopia.

BACKGROUND: Increasing the coverage of community-based treatment of childhood pneumonia (CCM) is part of the strategy to improve child survival, increase life-expectancy at birth and promote equity in Ethiopia. However, full coverage of CCM has not been reached in any regions of the country. There are no sub-national cost-effectiveness analyses available to inform decision makers on the most equitable scale up strategy. OBJECTIVES: Our first objective is to estimate the sub-national cost-effectiveness and the interindividual inequality impacts of scaling up CCM coverages to 90% in each region. Our second objective is to explore the costs, health effects, and geographical inequality impacts associated with three scale-up scenarios promoting different policy-aims: maximizing health, reducing geographical inequalities, and achieving 90% universal coverage. METHODS: We used Markov modelling to estimate the sub-national cost-effectiveness of CCM in each region. All data were collected through literature review and adjusted to the region-specific proportions of the rural population. Health effects were modeled as life years gained and under-five deaths averted. Interindividual and geographical inequality impacts were measured by the GINI index applied to health. In scenario analysis we explored three different scale-up strategies: 1) maximizing health by prioritizing the regions where the intervention was the most cost-effective, 2) reducing geographical inequalities by prioritizing the regions with high baseline under-five mortality rate (U5MR), and 3) universal upscaling to 90% coverage in all the regions. RESULTS: The regional incremental-cost effectiveness ratio (ICER) of scaling up the intervention coverage varied from 26 USD per life year gained in Addis to 199 USD per life year gained in the Southern Nations, Nationalities, and Peoples' region. Universal upscaling of CCM in all regions would cost about 1.3 billion USD and prevent about 90,000 under-five deaths. This is less than 15,000 USD per life saved and translates to an increase in life expectancy at birth of 1.6 years across Ethiopia. In scenario analysis, we found that prioritizing regions with high U5MR is effective in reducing geographical inequalities, although at the cost of fewer lives saved as compared to the health maximizing strategy. CONCLUSIONS: Our model results illustrate a trade-off between maximizing health and reducing health inequalities, two common policy-aims in low-income settings.

Health equity impact of community-initiated kangaroo mother care: a randomized controlled trial.

BACKGROUND: Kangaroo mother care (KMC) can substantially enhance overall survival of low birthweight babies. In a large randomized controlled trial, we recently showed that supporting mothers to provide community initiated KMC (ciKMC) can reduce mortality among infants up to 180 days of life by 25% (hazard ratio (HR) 0.75). With the current analysis, we aimed to explore if ciKMC promotion leads to increased inequity in survival. METHODS: In the trial we randomized 8402 low birthweight babies to a ciKMC (4480 babies) and a control (3922 babies) arm, between 2015 and 2018 in Haryana, India. We estimated the difference in concentration indices, which measure inequality, between babies in the ciKMC and control arms for survival until 180 days of life. Further, we compared the effect of ciKMC promotion across subgroups defined by socioeconomic status, caste, maternal literacy, infant's sex, and religion. RESULTS: Our intervention did not increase survival inequity, as the concentration index in the ciKMC arm of the trial was 0.05 (95% CI -0.07 to 0.17) lower than in the control arm. Survival impact was higher among those belonging to the lower two wealth quintiles, those born to illiterate mothers and those belonging to religions other than Hindu. CONCLUSIONS: We found that ciKMC promotion did not increase inequity in survival associated with wealth. The beneficial impact of ciKMC tended to be larger among vulnerable groups. Supporting mothers to provide KMC at home to low birthweight babies will not increase and could indeed reduce inequities in infant survival. TRIAL REGISTRATION: ClinicalTrials.gov, NCT02653534 . Registered January 12, 2016-Retrospectively registered.

Contextualization of cost-effectiveness evidence from literature for 382 health interventions for the Ethiopian essential health services package revision.

BACKGROUND: Cost-effectiveness of interventions was a criterion decided to guide priority setting in the latest revision of Ethiopia's essential health services package (EHSP) in 2019. However, conducting an economic evaluation study for a broad set of health interventions simultaneously is challenging in terms of cost, timeliness, input data demanded, and analytic competency. Therefore, this study aimed to synthesize and contextualize cost-effectiveness evidence for the Ethiopian EHSP interventions from the literature. METHODS: The evidence synthesis was conducted in five key steps: search, screen, evaluate, extract, and contextualize. We searched MEDLINE and EMBASE research databases for peer-reviewed published articles to identify average cost-effectiveness ratios (ACERs). Only studies reporting cost per disability-adjusted life year (DALY), quality-adjusted life year (QALY), or life years gained (LYG) were included. All the articles were evaluated using the Drummond checklist for quality, and those with a score of at least 7 out of 10 were included. Information on cost, effectiveness, and ACER was extracted. All the ACERs were converted into 2019 US dollars using appropriate exchange rates and the GDP deflator. RESULTS: In this study, we synthesized ACERs for 382 interventions from seven major program areas, ranging from US$3 per DALY averted (for the provision of hepatitis B vaccination at birth) to US$242,880 per DALY averted (for late-stage liver cancer treatment). Overall, 56% of the interventions have an ACER of less than US$1000 per DALY, and 80% of the interventions have an ACER of less than US$10,000 per DALY. CONCLUSION: We conclude that it is possible to identify relevant economic evaluations using evidence from the literature, even if transferability remains a challenge. The present study identified several cost-effective candidate interventions that could, if scaled up, substantially reduce Ethiopia's disease burden.

Cycle-network expansion plan in Oslo: Modeling cost-effectiveness analysis and health equity impact.

Physical inactivity is the leading cause of non-communicable diseases, and further research on the cost-effectiveness of interventions that target inactivity is warranted. Socioeconomic status is vital in this process. We aim to evaluate the cost-effectiveness of a cycle-network expansion plan in Oslo compared to the status quo by income quintiles. We applied a Markov model using a public payer perspective. Health outcomes were measured by quality-adjusted life years (QALYs) gained from the prevention of coronary heart disease, stroke, type 2 diabetes, and cancer. We measured equity impact by the concentration index and social welfare using the achievement index. We conducted sensitivity analyses. The intervention was generally more costly and more effective than the status quo. Incremental cost per QALY falls with income quintile, ranging from $10,098 in the richest quintile to $23,053 per QALY gained in the poorest quintile. The base-case intervention increased health inequality. However, a scenario targeting low-income quintiles reduced inequality and increased social welfare. In conclusion, the cycle-network expansion is likely to be cost-effective, but with equity concerns. If decision makers care about health inequalities, the disadvantaged groups could be targeted to produce more equitable and socially desirable outcomes instead of a uniform intervention across income quintiles.

Geographic health inequalities in Norway: a Gini analysis of cross-county differences in mortality from 1980 to 2014.

BACKGROUND: This study aims at quantifying the level and changes over time of inequality in age-specific mortality and life expectancy between the 19 Norwegian counties from 1980 to 2014. METHODS: Data on population and mortality by county was obtained from Statistics Norway for 1980-2014. Life expectancy and age-specific mortality rates (0-4, 5-49 and 50-69 age groups) were estimated by year and county. Geographic inequality was described by the absolute Gini index annually. RESULTS: Life expectancy in Norway has increased from 75.6 to 82.0 years, and the risk of death before the age of 70 has decreased from 26 to 14% from 1980 to 2014. The absolute Gini index decreased over the period 1980 to 2014 from 0.43 to 0.32 for life expectancy, from 0.012 to 0.0057 for the age group 50-69 years, from 0.0038 to 0.0022 for the age group 5-49 years, and from 0.0009 to 0.0006 for the age group 0-4 years. It will take between 2 and 32 years (national average 7 years) until the counties catch up with the life expectancy in the best performing county if their annual rates of increase remain unchanged. CONCLUSION: Using the absolute Gini index as a metric for monitoring changes in geographic inequality over time may be a valuable tool for informing public health policies. The absolute inequality in mortality and life expectancy between Norwegian counties has decreased from 1980 to 2014.

How can MCDA tools improve priority setting? Four critical questions.

This comment argues that four critical questions needs to be resolved before MCDA tools can improve priority setting in health: how to merge the quantitative and deliberative elements of MCDA; how to select criteria; how to weigh them, and whom to bring to the table.

Increased risk of peripartum perinatal mortality in unplanned births outside an institution: a retrospective population-based study.

BACKGROUND: Births in midwife-led institutions may reduce the frequency of medical interventions and provide cost-effective care, while larger institutions offer medically and technically advanced obstetric care. Unplanned births outside an institution and intrapartum stillbirths have frequently been excluded in previous studies on adverse outcomes by place of birth. OBJECTIVE: The objective of the study was to assess peripartum mortality by place of birth and travel time to obstetric institutions, with the hypothesis that centralization reduces institution availability but improves mortality. STUDY DESIGN: This was a national population-based retrospective cohort study of all births in Norway from 1999 to 2009 (n = 648,555) using data from the Medical Birth Registry of Norway and Statistics Norway and including births from 22 gestational weeks or birthweight ≥500 g. Main exposures were travel time to the nearest obstetric institution and place of birth. The main clinical outcome was peripartum mortality, defined as death during birth or within 24 hours. Intrauterine fetal deaths prior to start of labor were excluded from the primary outcome. RESULTS: A total of 1586 peripartum deaths were identified (2.5 per 1000 births). Unplanned birth outside an institution had a 3 times higher mortality (8.4 per 1000) than institutional births (2.4 per 1000), relative risk, 3.5 (95% confidence interval, 2.5-4.9) and contributed 2% (95% confidence interval, 1.2-3.0%) of the peripartum mortality at the population level. The risk of unplanned birth outside an institution increased from 0.5% to 3.3% and 4.5% with travel time <1 hour, 1-2 hours, and >2 hours, respectively. In obstetric institutions the mortality rate at term ranged from 0.7 per 1000 to 0.9 per 1000. Comparable mortality rates in different obstetric institutions indicated well-functioning routines for referral. CONCLUSION: Unplanned birth outside an institution was associated with increased peripartum mortality and with long travel time to obstetric institutions. Structural determinants have an important impact on perinatal health in high-income countries and also for low-risk births. The results show the importance of skilled birth attendance and warrant attention from clinicians and policy makers to negative consequences of reduced access to institutions.

Costs and expected gain in lifetime health from intensive care versus general ward care of 30,712 individual patients: a distribution-weighted cost-effectiveness analysis.

BACKGROUND: Clinicians, hospital managers, policy makers, and researchers are concerned about high costs, increased demand, and variation in priorities in the intensive care unit (ICU). The objectives of this modelling study are to describe the extra costs and expected health gains associated with admission to the ICU versus the general ward for 30,712 patients and the variation in cost-effectiveness estimates among subgroups and individuals, and to perform a distribution-weighted economic evaluation incorporating extra weighting to patients with high severity of disease. METHODS: We used a decision-analytic model that estimates the incremental cost per quality-adjusted life year (QALY) gained (ICER) from ICU admission compared with general ward care using Norwegian registry data from 2008 to 2010. We assigned increasing weights to health gains for those with higher severity of disease, defined as less expected lifetime health if not admitted. The study has inherent uncertainty of findings because a randomized clinical trial comparing patients admitted or rejected to the ICU has never been performed. Uncertainty is explored in probabilistic sensitivity analysis. RESULTS: The mean cost-effectiveness of ICU admission versus ward care was €11,600/QALY, with 1.6 QALYs gained and an incremental cost of €18,700 per patient. The probability (p) of cost-effectiveness was 95% at a threshold of €22,000/QALY. The mean ICER for medical admissions was €10,700/QALY (p = 97%), €12,300/QALY (p = 93%) for admissions after acute surgery, and €14,700/QALY (p = 84%) after planned surgery. For individualized ICERs, there was a 50% probability that ICU admission was cost-effective for 85% of the patients at a threshold of €64,000/QALY, leaving 15% of the admissions not cost-effective. In the distributional evaluation, 8% of all patients had distribution-weighted ICERs (higher weights to gains for more severe conditions) above €64,000/QALY. High-severity admissions gained the most, and were more cost-effective. CONCLUSIONS: On average, ICU admission versus general ward care was cost-effective at a threshold of €22,000/QALY (p = 95%). According to the individualized cost-effectiveness information, one in six ICU admissions was not cost-effective at a threshold of €64,000/QALY. Almost half of these admissions that were not cost-effective can be regarded as acceptable when weighted by severity of disease in terms of expected lifetime health. Overall, existing ICU services represent reasonable resource use, but considerable uncertainty becomes evident when disaggregating into individualized results.

Avoiding 40% of the premature deaths in each country, 2010-30: review of national mortality trends to help quantify the UN sustainable development goal for health.

BACKGROUND: The UN will formulate ambitious Sustainable Development Goals for 2030, including one for health. Feasible goals with some quantifiable, measurable targets can influence governments. We propose, as a quatitative health target, "Avoid in each country 40% of premature deaths (under-70 deaths that would be seen in the 2030 population at 2010 death rates), and improve health care at all ages". Targeting overall mortality and improved health care ignores no modifiable cause of death, nor any cause of disability that is treatable (or also causes many deaths). 40% fewer premature deaths would be important in all countries, but implies very different priorities in different populations. Reinforcing this target for overall mortality in each country are four global subtargets for 2030: avoid two-thirds of child and maternal deaths; two-thirds of tuberculosis, HIV, and malaria deaths; a third of premature deaths from non-communicable diseases (NCDs); and a third of those from other causes (other communicable diseases, undernutrition, and injuries). These challenging subtargets would halve under-50 deaths, avoid a third of the (mainly NCD) deaths at ages 50-69 years, and so avoid 40% of under-70 deaths. To help assess feasibility, we review mortality rates and trends in the 25 most populous countries, in four country income groupings, and worldwide. METHODS: UN sources yielded overall 1970-2010 mortality trends. WHO sources yielded cause-specific 2000-10 trends, standardised to country-specific 2030 populations; decreases per decade of 42% or 18% would yield 20-year reductions of two-thirds or a third. RESULTS: Throughout the world, except in countries where the effects of HIV or political disturbances predominated, mortality decreased substantially from 1970-2010, particularly in childhood. From 2000-10, under-70 age-standardised mortality rates decreased 19% (with the low-income and lower-middle-income countries having the greatest absolute gains). The proportional decreases per decade (2000-10) were: 34% at ages 0-4 years; 17% at ages 5-49 years; 15% at ages 50-69 years; 30% for communicable, perinatal, maternal, or nutritional causes; 14% for NCDs; and 13% for injuries (accident, suicide, or homicide). INTERPRETATION: Moderate acceleration of the 2000-10 proportional decreases in mortality could be feasible, achieving the targeted 2030 disease-specific reductions of two-thirds or a third. If achieved, these reductions avoid about 10 million of the 20 million deaths at ages 0-49 years that would be seen in 2030 at 2010 death rates, and about 17 million of the 41 million such deaths at ages 0-69 years. Such changes could be achievable by 2030, or soon afterwards, at least in areas free of war, other major effects of political disruption, or a major new epidemic. FUNDING: UK Medical Research Council, Norwegian Agency for Development Cooperation, Centre for Global Health Research, and Bill & Melinda Gates Foundation.

Ethical priority setting for universal health coverage: challenges in deciding upon fair distribution of health services.

Priority setting is inevitable on the path towards universal health coverage. All countries experience a gap between their population's health needs and what is economically feasible for governments to provide. Can priority setting ever be fair and ethically acceptable? Fairness requires that unmet health needs be addressed, but in a fair order. Three criteria for priority setting are widely accepted among ethicists: cost-effectiveness, priority to the worse-off, and financial risk protection. Thus, a fair health system will expand coverage for cost-effective services and give extra priority to those benefiting the worse-off, whilst at the same time providing high financial risk protection. It is considered unacceptable to treat people differently according to their gender, race, ethnicity, religion, sexual orientation, social status, or place of residence. Inequalities in health outcomes associated with such personal characteristics are therefore unfair and should be minimized. This commentary also discusses a third group of contested criteria, including rare diseases, small health benefits, age, and personal responsibility for health, subsequently rejecting them. In conclusion, countries need to agree on criteria and establish transparent and fair priority setting processes.

Inequalities in utilization of maternal and child health services in Ethiopia: the role of primary health care.

BACKGROUND: Health systems aim to narrow inequality in access to health care across socioeconomic groups and area of residency. However, in low-income countries, studies are lacking that systematically monitor and evaluate health programs with regard to their effect on specific inequalities. We aimed to measure changes in inequality in access to maternal and child health (MCH) interventions and the effect of Primary Health Care (PHC) facilities expansion on the inequality in access to care in Ethiopia. METHODS: The Demographic and Health Survey datasets from Ethiopia (2005 and 2011) were used. We calculated changes in utilization of MCH interventions and child morbidity. Concentration and horizontal inequity indices were estimated. Decomposition analysis was used to calculate the contribution of each determinant to the concentration index. RESULTS: Between 2005 and 2011, improvements in aggregate coverage have been observed for MCH interventions in Ethiopia. Wealth-related inequality has remained persistently high in all surveys. Socioeconomic factors were the main predictors of differences in maternal and child health services utilization and child health outcome. Utilization of primary care facilities for selected maternal and child health interventions have shown marked pro-poor improvement over the period 2005-2011. CONCLUSIONS: Our findings suggest that expansion of PHC facilities in Ethiopia might have an important role in narrowing the urban-rural and rich-poor gaps in health service utilization for selected MCH interventions.