Advances in Addressing Patient Heterogeneity in Economic Evaluation: A Review of the Methods Literature.

Cost-effectiveness analyses commonly use population or sample averages, which can mask key differences across subgroups and may lead to suboptimal resource allocation. Despite there being several new methods developed over the last decade, there is no recent summary of what methods are available to researchers. This review sought to identify advances in methods for addressing patient heterogeneity in economic evaluations and to provide an overview of these methods.

Risk Prediction Using Bayesian Networks: An Immunotherapy Case Study in Patients With Metastatic Renal Cell Carcinoma.

Purpose: To address the need for more accurate risk stratification models for cancer immuno-oncology, this study aimed to develop a machine-learned Bayesian network model (BNM) for predicting outcomes in patients with metastatic renal cell carcinoma (mRCC) being treated with immunotherapy.

Methods: Patient-level data from the randomized, phase III CheckMate 025 clinical trial comparing nivolumab with everolimus for second-line treatment in patients with mRCC were used to develop the BNM. Outcomes of interest were overall survival (OS), all-cause adverse events, and treatment-related adverse events (TRAE) over 36 months after treatment initiation.

An empirical investigation of time-varying cost-effectiveness across the product life cycle.

Cost-effectiveness is traditionally treated as a static estimate driven by clinical trial efficacy and drug price at launch. Prior studies suggest that cost-effectiveness varies over the drug's lifetime. We examined the impact of "learning by doing," one of the least studied drivers of changes in cost-effectiveness across the product life cycle.

One size does not always fit all in value assessment.

Laying a clear path for incorporating reliable evidence on heterogeneity in value assessments could improve their applicability for healthcare decision making.

Maternal and Infant Adverse Outcomes Associated with Mild and Severe Preeclampsia during the First Year after Delivery in the United States.

Objective: The burden of preeclampsia severity on the health of mothers and infants during the first year after delivery is unclear, given the lack of population-based longitudinal studies in the United States.

Study Design: We assessed maternal and infant adverse outcomes during the first year after delivery using population-based hospital discharge information merged with vital statistics and birth certificates of 2,021,013 linked maternal-infant births in California. We calculated sampling weights using the National Center for Health Statistics data to adjust for observed differences in maternal characteristics between California and the rest of the United States.

An empirical tool for estimating the share of unmet need due to healthcare inefficiencies, suboptimal access, and lack of effective technologies.

Background: Although there has been growing attention to the measurement of unmet need, which is the overall epidemiological burden of disease, current measures ignore the burden that could be eliminated from technological advances or more effective use of current technologies.

Methods: We developed a conceptual framework and empirical tool that separates unmet need from met need and subcategorizes the causes of unmet need into suboptimal access to and ineffective use of current technologies and lack of current technologies. Statistical models were used to model the relationship between health-related quality of life (HR-QOL) and treatment utilization using data from the National Health and Wellness Survey (NHWS).

The Economic and Social Burden of the 2014 Ebola Outbreak in West Africa.

Background: The 2014 Ebola virus disease (EVD) outbreak in West Africa was more devastating than all previous EVD outbreaks combined; however, many estimates of its economic burden did not capture its significant social costs. This study aimed to review all currently available estimates, identify social components missed by these estimates, and generate a comprehensive cost of the 2014 EVD outbreak.

Methods: We conducted a systematic review of the grey (reports produced by nonprofit or nongovernmental organizations, government, or industry) and published literature to identify current estimates of the burden of the outbreak.

The Lifetime Health Burden of Delayed Graft Function in Kidney Transplant Recipients in the United States.

Although delayed graft function (DGF) is associated with an increased risk of acute rejection and decreased graft survival, there are no estimates of the long-term or lifetime health burden of DGF. To estimate the long-term and lifetime health burden of DGF, defined as the need for at least one dialysis session within the first week after transplantation, for a cohort representative of patients who had their first kidney transplant in 2014. Data from the United States Renal Data System (USRDS; 2001-2014) were used to estimate a semi-Markov parametric multi-state model with three disease states.

Assessing Variation in the Cost of Palivizumab for Respiratory Syncytial Virus Prevention in Preterm Infants.

Background: The variability in cost of palivizumab treatment, indicated for prevention of respiratory syncytial virus (RSV) infections in high-risk infants, has not been robustly estimated in prior studies. This study aimed to determine the cost variations of palivizumab from a US payer perspective for otherwise healthy preterm infants born 29-35 weeks gestational age (wGA) using infant characteristics and applied dosing regimens.

Methods: Fenton Growth Charts were merged with World Health Organization Child Growth Standards to estimate preterm infant growth patterns.

Amazon plant diversity revealed by a taxonomically verified species list.

Recent debates on the number of plant species in the vast lowland rain forests of the Amazon have been based largely on model estimates, neglecting published checklists based on verified voucher data. Here we collate taxonomically verified checklists to present a list of seed plant species from lowland Amazon rain forests. Our list comprises 14,003 species, of which 6,727 are trees.

Short-term costs of preeclampsia to the United States health care system.

Background: Preeclampsia is a leading cause of maternal morbidity and mortality and adverse neonatal outcomes. Little is known about the extent of the health and cost burden of preeclampsia in the United States.

Objective: This study sought to quantify the annual epidemiological and health care cost burden of preeclampsia to both mothers and infants in the United States in 2012.

Value of improved lipid control in patients at high risk for adverse cardiac events.

Objectives: Lipid-lowering therapy (LLT) is suboptimally used in patients with hyperlipidemia in the 2 highest statin benefit groups (SBGs), as categorized by the American College of Cardiology and the American Heart Association. This study estimated the social value of reducing low-density lipoprotein cholesterol (LDL-C) levels by 50% for patients in SBGs 1 and 2 who have been treated with standard LLT but have not reached LDL-C goal, as well as the potential value of PCSK9 inhibitors for patients in these groups.

Study Design: Simulation model.

The wider public health value of HCV treatment accrued by liver transplant recipients.

Objectives: Organs for transplantation are scarce, but new medical therapies can prevent organ failure and the need for transplants. We sought to describe the unique value created by treatments that spare organs from failure and thus conserve donated organs for transplant into others, using hepatitis C virus (HCV) as a case study.

Study Design: Epidemiologic-economic model.

Estimating the future burden of cardiovascular disease and the value of lipid and blood pressure control therapies in China.

Background: Lifestyle and dietary changes reflect an ongoing epidemiological transition in China, with cardiovascular disease (CVD) playing an ever-increasing role in China's disease burden. This study assessed the burden of CVD and the potential value of lipid and blood pressure control strategies in China.

Methods: We estimated the likely burden of CVD between 2016 and 2030 and how expanded use of lipid lowering and blood pressure control medication would impact that burden in the next 15 years.

The Rising Burden of Preeclampsia in the United States Impacts Both Maternal and Child Health.

Objective: Preeclampsia is one of the top six causes of maternal mortality in the United States (US) and is associated with considerable perinatal morbidity and mortality. Evidence suggests the US incidence of preeclampsia has increased dramatically over the past two decades. This study aims to compile, summarize, and critique the literature on the health and economic burden of preeclampsia and early-onset preeclampsia.

Cancer mortality reductions were greatest among countries where cancer care spending rose the most, 1995-2007.

Health care spending and health outcomes vary markedly across countries, but the association between spending and outcomes remains unclear. This inevitably raises questions as to whether continuing growth in spending is justified, especially relative to the rising cost of cancer care. We compared cancer care across sixteen countries over time, examining changes in cancer spending and two measures of cancer mortality (amenable and excess mortality).

Turning evidence into practice under payment reform: the new frontier of translational science.

Momentum is building to replace the current fee-for-service payment system with value-based reimbursement models that aim to deliver high quality care at lower costs. Although the goals of payment and delivery system reforms to improve quality and reduce costs are clear, the actual path by which provider groups can achieve these goals is not well understood, in large part because the role of identifying and discouraging the use of low-value, high-cost services and encouraging the use of high-value, low-cost services has traditionally fallen to health plans, not provider groups. The shifting focus towards provider accountability for costs and quality promises to expand the role of provider organizations from mainly delivering care to both delivering and prioritizing it based on costs and quality.

A Cost-Benefit Analysis of Using Evidence of Effectiveness in Terms of Progression Free Survival in Making Reimbursement Decisions on New Cancer Therapies.

Payers increasingly require evidence of a statistically significant difference in overall survival (OS) for reimbursement of new cancer therapies. At the same time, it becomes increasingly costly to design clinical trials that measure OS endpoints instead of progression-free survival (PFS) endpoints. While PFS is often an imperfect proxy for OS effects, it is also faster and cheaper to measure accurately.

Modeling health impact of global health programs implemented by Population Services International.

Background: Global health implementing organizations benefit most from health impact estimation models that isolate the individual effects of distributed products and services - a feature not typically found in intervention impact models, but which allow comparisons across interventions and intervention settings. Population Services International (PSI), a social marketing organization, has developed a set of impact models covering seven health program areas, which translate product/service distribution data into impact estimates. Each model's primary output is the number of disability-adjusted life-years (DALYs) averted by an intervention within a specific country and population context.

Epidemiological and economic burden of metabolic syndrome and its consequences in patients with hypertension in Germany, Spain and Italy; a prevalence-based model.

Background: The presence of metabolic syndrome in patients with hypertension significantly increases the risk of cardiovascular disease, type 2 diabetes and mortality. Our aim is to estimate the epidemiological and economic burden to the health service of metabolic syndrome in patients with hypertension in three European countries in 2008 and 2020.

Methods: An age, sex and risk group structured prevalence based cost of illness model was developed using the United States Adult Treatment Panel III of the National Cholesterol Education Program criteria to define metabolic syndrome.

Modelling the costs of care of hypertension in patients with metabolic syndrome and its consequences, in Germany, Spain and Italy.

The presence of metabolic syndrome in patients with hypertension significantly increases the risk of cardiovascular disease, type 2 diabetes and mortality. Our aim is to estimate the economic burden to the health service of metabolic syndrome (MetS) in patients with hypertension and its consequences, in three European countries in 2008, and to forecast future economic burden in 2020 using projected demographic estimates and assumptions around the growth of MetS. An age-, sex- and risk group-structured prevalence-based cost of illness model was developed using the United States Adult Treatment Panel III of the National Cholesterol Education Program criteria to define MetS.

Meeting oxygen needs in Africa: an options analysis from the Gambia.

Objective: To compare oxygen supply options for health facilities in the Gambia and develop a decision-making algorithm for choosing oxygen delivery systems in Africa and the rest of the developing world.

Methods: Oxygen cylinders and concentrators were compared in terms of functionality and cost. Interviews with key informants using locally developed and adapted WHO instruments, operational assessments, cost-modelling and cost measurements were undertaken to determine whether oxygen cylinders or concentrators were the better choice.

Access to health care and mortality of children under 5 years of age in the Gambia: a case-control study.

Objective: To assess whether traditional measures of access to health care (distance and travel time to a facility) and non-traditional measures (social and financial support indicators) are associated with mortality among children under 5 years of age in the Gambia.

Methods: We conducted a case-control study in a population under demographic surveillance. Cases (n = 140) were children under 5 years of age who died between 31 December 2003 and 30 April 2006.