Parkinson's disease (PD), following Alzheimer's disease, is the second‐most common neurodegenerative disorder in the United States. A lack of treatment options for changing the trajectory of disease ...progression, in combination with an increasing elderly population, portends a rising economic burden on patients and payers. This study combined information from nationally representative surveys to create a burden of PD model. The model estimates disease prevalence, excess healthcare use and medical costs, and nonmedical costs for each demographic group defined by age and sex. Estimated prevalence rates and costs were applied to the U.S. Census Bureau's 2010 to 2050 population data to estimate current and projected burden based on changing demographics. We estimate that approximately 630,000 people in the United States had diagnosed PD in 2010, with diagnosed prevalence likely to double by 2040. The national economic burden of PD exceeds $14.4 billion in 2010 (approximately $22,800 per patient). The population with PD incurred medical expenses of approximately $14 billion in 2010, $8.1 billion higher ($12,800 per capita) than expected for a similar population without PD. Indirect costs (e.g., reduced employment) are conservatively estimated at $6.3 billion (or close to $10,000 per person with PD). The burden of chronic conditions such as PD is projected to grow substantially over the next few decades as the size of the elderly population grows. Such projections give impetus to the need for innovative new treatments to prevent, delay onset, or alleviate symptoms of PD and other similar diseases. 2013 Movement Disorder Society
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BFBNIB, FZAB, GIS, IJS, KILJ, NLZOH, NUK, OILJ, SBCE, SBMB, UL, UM, UPUK
Abstract
Background
U.S. cost-effectiveness recommendations suggest that analyses should include all costs and effects relevant to the decision problem 1. However, in many diseases, including spinal ...muscular atrophy (SMA), few studies have evaluated bereaved family outcomes after a child has died, neglecting potential impacts on their health-related quality of life (HRQoL), work productivity, and mental health. Additionally, grief-related outcomes are rarely included in economic evaluations. This manuscript outlines the protocol of a study that will estimate the HRQoL, work functioning, and mental health of bereaved parents of children with SMA type 1 to determine how outcomes vary based on parent’s sex and the time since a child’s death.
Methods
This study will involve two phases. In Phase 1, we will conduct a literature review to identify prior research that has measured how parental grief impacts HRQoL, work productivity, and mental health. We will also interview four bereaved parents of children with SMA type 1, stratified by parent sex and time since their child’s death, and analyze findings using a thematic analysis. In Phase 2, we will develop a survey draft based on Phase 1 findings. Parents bereaved from SMA type 1 will review our survey draft and we will revise the survey based on their feedback. We will send a cross-sectional survey to approximately 880 parents bereaved from SMA type 1. We will analyze findings from the survey to investigate whether the severity of grief symptoms is correlated with HRQoL, productivity, depression and anxiety symptom severity. We will also evaluate whether the mean scores of grief and each of the outcomes vary significantly when stratified by parent sex and the time since the child’s death.
Discussion
Our results will provide preliminary information on how parental grief can impact HRQoL, productivity, and mental health outcomes over time. Increasing the availability of family outcomes data will potentially assist organizations performing health economic evaluations, such as the Institute of Clinical and Economic Review (ICER) in the U.S. This research will also help to inform the development of future economic guidelines on this topic.
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CEKLJ, IZUM, KILJ, NUK, PILJ, PNG, SAZU, UL, UM, UPUK
Abstract Objective We sought to compare the cost-effectiveness of different interventions that have been shown to improve adherence with antihypertensive and lipid-lowering therapy, by combining a ...burden of nonadherence model framework with literature-based data on adherence-improving interventions. Methods MEDLINE was reviewed for studies that evaluated ≥1 adherence intervention compared with a control, used an adherence measure other than self-report, and followed patients for ≥6 months. Effectiveness was assessed as Relative Improvement, ratio of adherence with an intervention versus control. Costs, standardized to 12 months and adjusted to 2007 US$, and effectiveness estimates for each intervention were entered into a previously published model designed to measure the burden of nonadherence with antihypertensive and lipid-lowering medications, in a hypertensive population. Outputs included direct medical costs and incremental costs per quality-adjusted life-year (QALY) gained. Results After screening, 23 eligible adherence-improving interventions were identified from 18 studies. Relative Improvement ranged from 1.13 to 3.60. After eliminating more costly/less effective interventions, two remained. Self-monitoring, reminders, and educational materials incurred total health-care costs of $17,520, and compared with no adherence intervention, had an incremental cost-effectiveness ratio (ICER) of $4984 per QALY gained. Pharmacist/nurse management incurred total health-care costs of $17,896, and versus self-monitoring, reminders, and education had an ICER of $6358 per QALY gained. Conclusions Of published interventions shown to improve adherence, reminders and educational materials, and a pharmacist/nurse management program, appear to be cost-effective and should be considered before other interventions. Understanding relative cost-effectiveness of adherence interventions may guide design and implementation of efficient adherence-improving programs
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BFBNIB, DOBA, FZAB, GEOZS, GIS, IJS, IMTLJ, IZUM, KILJ, KISLJ, NLZOH, NUK, OILJ, PILJ, PNG, SAZU, SBCE, SBJE, SBMB, UILJ, UKNU, UL, UM, UPCLJ, UPUK, ZAGLJ, ZRSKP
This study updates previous estimates of the economic burden of diagnosed diabetes and quantifies the increased health resource use and lost productivity associated with diabetes in 2012.
The study ...uses a prevalence-based approach that combines the demographics of the U.S. population in 2012 with diabetes prevalence, epidemiological data, health care cost, and economic data into a Cost of Diabetes Model. Health resource use and associated medical costs are analyzed by age, sex, race/ethnicity, insurance coverage, medical condition, and health service category. Data sources include national surveys, Medicare standard analytical files, and one of the largest claims databases for the commercially insured population in the U.S.
The total estimated cost of diagnosed diabetes in 2012 is $245 billion, including $176 billion in direct medical costs and $69 billion in reduced productivity. The largest components of medical expenditures are hospital inpatient care (43% of the total medical cost), prescription medications to treat the complications of diabetes (18%), antidiabetic agents and diabetes supplies (12%), physician office visits (9%), and nursing/residential facility stays (8%). People with diagnosed diabetes incur average medical expenditures of about $13,700 per year, of which about $7,900 is attributed to diabetes. People with diagnosed diabetes, on average, have medical expenditures approximately 2.3 times higher than what expenditures would be in the absence of diabetes. For the cost categories analyzed, care for people with diagnosed diabetes accounts for more than 1 in 5 health care dollars in the U.S., and more than half of that expenditure is directly attributable to diabetes. Indirect costs include increased absenteeism ($5 billion) and reduced productivity while at work ($20.8 billion) for the employed population, reduced productivity for those not in the labor force ($2.7 billion), inability to work as a result of disease-related disability ($21.6 billion), and lost productive capacity due to early mortality ($18.5 billion).
The estimated total economic cost of diagnosed diabetes in 2012 is $245 billion, a 41% increase from our previous estimate of $174 billion (in 2007 dollars). This estimate highlights the substantial burden that diabetes imposes on society. Additional components of societal burden omitted from our study include intangibles from pain and suffering, resources from care provided by nonpaid caregivers, and the burden associated with undiagnosed diabetes.
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Background: Survival benefit from current anticancer treatments, even if modest, improves a patient’s chances of accessing and benefiting from future innovations currently in clinical ...development. No studies to date have evaluated the potential impact of future innovations on oncologists’ treatment recommendations. Methods: We conducted a national survey of medical and hematologic oncologists (n = 200) on how expectations about future innovations may influence their treatment recommendations today. The survey described a hypothetical patient with metastatic cancer, whose median overall survival (OS) was approximately 6 months with most dying within 2 years. We presented four alternative hypothetical scenarios of new innovations in the clinical development pipeline with varying (a) expected efficacy—“modest” (improvement in median OS of 2 months} or “breakthrough” (improvement in median OS of 6 months) and (b) time of arrival (in 6 months or 1 year). We asked the oncologists about the likelihood (using a 5-point Likert scale) (1) that they would discuss these new innovations with the hypothetical metastatic patient, and (2) that these new innovations would influence their recommended treatment for the patient today. We descriptively summarized percentages of oncologists who responded “likely” or “very likely” and examined their associations (using multivariate logistic regression) with the expected efficacy and time of arrival of future innovations as well as the characteristics of oncologists and their practice. Results: When future innovations were expected to have modest efficacy and arrive in 6 months, 61% of oncologists reported that they would be likely or very likely discuss them with their patients, and 52% reported new innovations would influence their treatment recommendations today. With modest efficacy and time of arrival in 1 year, percentages dropped to 49% and 41%, respectively. With breakthrough efficacy and arrival in 6 months, 76% would discuss and 68% would consider new innovations. With breakthrough efficacy and arrival in 1 year, percentages dropped to 62% and 55%, respectively. Greater expected efficacy and shorter time to arrival were associated with a significantly greater likelihood of discussing a future innovations with patients across oncologist and practice characteristics (adjusted odds ratios: breakthrough vs. modest = 1.75; 6 month vs. 1 year = 1.72). Conclusions: Future treatment innovations in clinical development are relevant for decision-making today even when anticipated time to arrival is two times the current median OS. results indicate that it is important to include future innovations in shared decision-making in addition to the benefit-risk balance of current treatments.
Introduction
The COVID-19 pandemic is a global crisis impacting population health and the economy. We describe a cost-effectiveness framework for evaluating acute treatments for hospitalized patients ...with COVID-19, considering a broad spectrum of potential treatment profiles and perspectives within the US healthcare system to ensure incorporation of the most relevant clinical parameters, given evidence currently available.
Methods
A lifetime model, with a short-term acute care decision tree followed by a post-discharge three-state Markov cohort model, was developed to estimate the impact of a potential treatment relative to best supportive care (BSC) for patients hospitalized with COVID-19. The model included information on costs and resources across inpatient levels of care, use of mechanical ventilation, post-discharge morbidity from ventilation, and lifetime healthcare and societal costs. Published literature informed clinical and treatment inputs, healthcare resource use, unit costs, and utilities. The potential health impacts and cost-effectiveness outcomes were assessed from US health payer, societal, and fee-for-service (FFS) payment model perspectives.
Results
Viewing results in aggregate, treatments that conferred at least a mortality benefit were likely to be cost-effective, as all deterministic and sensitivity analyses results fell far below willingness-to-pay thresholds using both a US health payer and FFS payment perspective, with and without societal costs included. In the base case, incremental cost-effectiveness ratios (ICER) ranged from $22,933 from a health payer perspective using bundled payments to $8028 from a societal perspective using a FFS payment model. Even with conservative assumptions on societal impact, inclusion of societal costs consistently produced ICERs 40–60% lower than ICERs for the payer perspective.
Conclusion
Effective COVID-19 treatments for hospitalized patients may not only reduce disease burden but also represent good value for the health system and society. Though data limitations remain, this cost-effectiveness framework expands beyond current models to include societal costs and post-discharge ventilation morbidity effects of potential COVID-19 treatments.
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EMUNI, FIS, FZAB, GEOZS, GIS, IJS, IMTLJ, KILJ, KISLJ, MFDPS, NLZOH, NUK, OILJ, PNG, SAZU, SBCE, SBJE, SBMB, SBNM, UKNU, UL, UM, UPUK, VKSCE, ZAGLJ
Introduction
A recent proliferation of value frameworks, as well as the emergence of innovative approaches to treating disease (e.g., cell/gene therapies) have been accompanied by an increased focus ...on nontraditional elements of value. We sought to understand whether and how health technology assessment (HTA) agencies consider novel aspects of treatment in value assessments.
Methods
We defined treatment novelty as follows: (i) a new mechanism of action or administration; (ii) addresses an unmet need; or (iii) confers a distinct benefit that transforms clinical practice or that is difficult to quantify. We reviewed technical guidance and peer-reviewed literature to investigate how organizations in eight countries (Australia, Canada, England, France, Norway, the Netherlands, Sweden, and the United States) consider aspects of this definition.
Results
All (n = 8) organizations give special consideration to interventions that address an unmet need, particularly in cancer, rare diseases, and other severe conditions. Nearly all (n = 5) organizations consider whether an intervention produces benefits that may not be adequately quantified. Organizations in England, Norway, and France sometimes recommend drugs with less favorable cost-effectiveness estimates than traditionally considered if the drug addresses rare or severe conditions, or if its quality-of-life benefit is thought to be inadequately quantified. The Institute for Clinical and Economic Review in the United States models cost-effectiveness in rare diseases using both a modified societal and health care system perspective. Importantly, the benefits of novel treatments are frequently considered uncertain, particularly treatments with a new mechanism of action. When uncertainty is high, organizations in Canada, England, France, the Netherlands, and Sweden sometimes issue conditional recommendations until additional evidence is submitted. England and Australia have used risk sharing agreements for drugs determined to be novel but uncertain.
Conclusions
The most widely considered aspects of treatment novelty in HTA are unmet needs and potential benefits that are not easily measured. The willingness to pay for novel treatments is often greater, despite inherent uncertainties about benefit and cost-effectiveness.
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