Real-world impact of disease on functioning and activity: what is missed when using general instruments to estimate quality-adjusted life years.

OBJECTIVE: Economic evaluations conducted to inform healthcare resource allocation often rely on quality-adjusted life years (QALYs) to measure therapeutic benefit. However, QALYs, with underlying health utilities estimated using the EQ-5D or SF-36, may fail to capture the impact of disease for all patients. How well-being and heath utility differ across several common conditions was explored. METHODS: This study examined eight diseases: arthritis, asthma, cancer, depression, diabetes, heart disease, lung disease and stroke. Health utilities for each disease were obtained from published literature. Other measures of disease burden, including physical functioning, cognitive functioning and physical activity, were estimated from the National Health and Nutrition Examination Survey (NHANES). Group rankings by these measures were compared to rankings by health utility. RESULTS: Health utilities were lowest for patients with depression (0.44), and highest for those with cancer (0.81). Physical functioning was most limited (higher score) among those with stroke (28.2) and had the least impact for cancer (24.4). Physical activity was most impacted by heart disease (27.3) and least impacted by depression (40.7). Cognitive functioning was lowest in stroke (41.6) and highest in asthma (52.0). CONCLUSION: Differences in rankings of disease severity by metric indicate that the results of cost-utility analyses might be biased against treatments for certain diseases. As patient preferences for clinical outcomes vary, the full burden of disease should be considered in evaluations. Restricting access to treatments based on an incomplete estimate of burden could lead to misallocation of resources and a withholding of therapies that patients find valuable.

The economic burden of caregiving in epilepsy: An estimate based on a survey of US caregivers.

OBJECTIVE: The burden of caregiving for persons with epilepsy (PWEs) has not been examined previously in the United States. We assessed the clinical impact and direct and indirect economic costs for caregivers of PWEs. METHODS: An internet survey of 500 caregivers of PWEs was conducted from May to July 2015 using a combination of validated instruments and questions designed specifically for this survey. Caregivers were stratified by PWE age (adult/child) and disease severity (low: 0 vs high: 1 + seizures in the prior month). Annual self-reported direct and indirect costs were reported per caregiver and extrapolated to all US caregivers. The economic burden of caregiving for PWEs was defined as the difference between costs for caregivers and the general population. RESULTS: Caregivers reported that PWEs averaged 11.4 seizures in the prior month. Eighty percent of respondents were female and the average age was 44.3. Since becoming a caregiver, many reported anxiety (52.8%), depression (41.0%), and insomnia (30.8%). Annual mean direct medical costs for caregivers of children with low vs high seizure frequency were $4344 and $10 162, respectively. Costs for caregivers of adult PWEs were $4936 and $8518. Mean indirect costs associated with caregiving for a child with low vs high seizure frequency were $20 529 and $40 137; those for caregivers of an adult were $13 981 and $28 410. The cost estimates are higher vs the general US population; annual per-person healthcare utilization costs were $2740 and productivity loss costs were $5015. When extrapolating to the US population of PWE caregivers, annual costs exceeded $62 billion vs $14 billion for the general population, resulting in a caregiver burden of nearly $48 billion. SIGNIFICANCE: The clinical and economic burden of caregivers for PWE were substantial, and greatest for those caring for children with frequent seizures. The impact on caregivers should be considered when estimating the value of interventions that control epilepsy.

Healthcare Policy Changes in Osteoporosis Can Improve Outcomes and Reduce Costs in the United States.

In the United States, osteoporosis affects over 10 million adults, has high societal costs ($22 billion in 2008), and is currently being underdiagnosed and undertreated. Given an aging population, this burden is expected to rise. We projected the fracture burden in US women by modeling the expected demographic shift as well as potential policy changes. With the anticipated population aging and growth, annual fractures are projected to increase from 1.9 million to 3.2 million (68%), from 2018 to 2040, with related costs rising from $57 billion to over $95 billion. Policy-driven expansion of case finding and treatment of at-risk women could lower this burden, preventing 6.1 million fractures over the next 22 years while reducing payer costs by $29 billion and societal costs by $55 billion. Increasing use of osteoporosis-related interventions can reduce fractures and result in substantial cost-savings, a rare and fortunate combination given the current landscape in healthcare policy. © 2019 The Authors. JBMR Plus published by Wiley Periodicals, Inc. on behalf of American Society for Bone and Mineral Research.

Costs associated with unplanned readmissions among patients with heart failure with and without hyponatremia.

PURPOSE: Costs associated with unplanned readmissions among patients with heart failure with and without hyponatremia were studied. METHODS: This study estimated the costs of patients hospitalized for heart failure (HF) discharged with or without corrected sodium. A model was developed to monetize the 30-day readmission risk based on hyponatremia correction. Costs of discharging patient with corrected versus uncorrected hyponatremia were estimated using readmission rates from a previously published study and hospitalization costs from the Healthcare Costs and Utilization Cost Project and the Premier Healthcare Database. RESULTS: Discharging patients with HF and hyponatremia increased costs from $488-$569 per discharge compared to patients with corrected hyponatremia. This range reflected differences in readmission rates and sources of hospitalization costs. Sensitivity analyses showed hospitalization costs and readmission rates had the largest impact on model results. CONCLUSION: A retrospective study supports the value of upfront monitoring and correction of low serum sodium levels before discharge among patients with HF and hyponatremia by presenting an economic argument in addition to the clinical rational for reducing risk of readmission.

Protecting the gains: What changes are needed to prevent a reversal of the downward cardiovascular disease mortality trend?

AIMS: Cardiovascular disease (CVD) mortality has decreased over 60% over the past 50 years in the United States; however, emerging data indicate CVD incidence may be rising because of shifting demographics, increasing risk factor prevalence, and competing needs for limited resources. We projected CVD mortality from 2015 to 2040 given varying informed assumptions regarding changes in risk factor prevalence, uptake of current therapeutic options, and future innovations. METHODS: A microsimulation model was used to project US CVD mortality trends. National Health and Nutrition Examination Survey data were used to estimate population-level trends in CVD risk factors. Risk factors were used to generate Framingham Risk Scores for cohorts of 1 000 000 individuals from the general population to determine each individuals' CVD risk. Annual cardiovascular incidence, prevalence, and mortality were projected for scenarios differing by uptake of current therapies, anticipated pharmaceutical innovations with variable efficacy, risk factor prevalence, and changes in health disparities. RESULTS: When incorporating a demographic shift, continued changes in risk factors, current treatment utilization, and no major innovations, we predicted the CVD mortality rate would increase 41% by 2040. If innovations providing incremental benefits equal to those associated with the introduction of statins are identified and widely utilized, CVD mortality could remain constant through 2040. With more efficacious innovations, CVD mortality could be further reduced. CONCLUSIONS: Given demographic and risk prevalence changes, increasing access and adherence to current preventative therapeutics could slow the expected mortality increase, but new therapies may be needed to maintain the downward trend in CVD deaths.

Utilization and budget impact of tolvaptan in the inpatient setting among patients with heart failure and hyponatremia.

OBJECTIVE: Assess characteristics of patients with heart failure (HF) and hyponatremia (HN) using tolvaptan, a selective vasopressin V2-receptor antagonist, for sodium correction, and estimate the budget impact of tolvaptan use in a hospital. METHODS: The Premier hospital database was analyzed to assess the utilization of tolvaptan, characteristics of users and non-users, and hospitalization costs among patients with HF and HN. Using these findings, a model was developed to estimate tolvaptan costs in proportion to total medical costs of managing patients with HF and HN, and the budget impact of tolvaptan use. Results were regenerated using data from the Healthcare Cost and Utilization Project (HCUP) database, and robustness was assessed in sensitivity analyses. RESULTS: Tolvaptan was used in 4.96% of inpatient visits among patients with HF and HN, more commonly among sicker patients as reflected in high utilization during intensive care stays (30.46%). Additionally, utilization increased by length of stay, which can serve as a proxy for disease severity. The model estimated that tolvaptan costs accounted for 0.3% of total hospitalization-related costs for patients with HF and HN, and the budget impact was $52.42 per visit. CONCLUSIONS: Results demonstrate that tolvaptan is used infrequently among patients with HF and HN, and is utilized among sicker patients. Tolvaptan accounted for 0.3% of total spending on management of inpatient visits with HF and HN, and had a marginal impact on hospital budget when compared with fluid restriction for HN correction. Availability of tolvaptan can provide an additional therapeutic option for sodium correction.

A systematic literature review of methods of incorporating mortality in cost-effectiveness analyses of lipid-lowering therapies.

AIMS: Cost effectiveness analysis (CEA) is a useful tool for estimating the value of an intervention in relation to alternatives. In cardiovascular disease (CVD), CEA is especially important, given the high economic and clinical burden. One key driver of value is CVD mortality prevention. However, data used to inform CEA parameters can be limited, given the difficulty in demonstrating statistically significant mortality benefit in randomized clinical trials (RCTs), due in part to the frequency of fatal events and limited trial durations. This systematic review identifies and summarizes whether published CVD-related CEAs have incorporated mortality benefits, and the methodology among those that did. MATERIALS AND METHODS: A systematic literature review was conducted of CEAs of lipid-lowering therapies published between 2000-2017. Health technology assessments (HTA) and full-length manuscripts were included, and sources of mortality data and methods of applying mortality benefits were extracted. Results were summarized as proportions of articles to articulate common practices in CEAs of CVD. RESULTS: This review identified 100 studies for inclusion, comprising 93 full-length manuscripts and seven HTA reviews. Among these, 99% assumed a mortality benefit in the model. However, 87 of these studies that incorporated mortality differences did so despite the trials used to inform model parameters not demonstrating statistically significant differences in mortality. None of the 12 studies that used statistically significant findings from an individual RCT were based on active control studies. In a sub-group analysis considering the 60 CEAs that incorporated a direct mortality benefit, 48 (80%) did not have RCT evidence for statistically significant benefit in CVD mortality. LIMITATIONS AND CONCLUSIONS: The finding that few CEA models included mortality inputs from individual RCTs of lipid-lowering therapy may be surprising, as one might expect that treatment efficacy should be based on robust clinical evidence. However, regulatory requirements in CVD-related RCTs often lead to insufficient sample sizes and observation periods for detecting a difference in CVD mortality, which results in the use of intermediate outcomes, composite end-points, or meta-analysis to extrapolate long-term mortality benefit in a lifetime CEA.