Healthcare resource allocation decisions made under conditions of uncertainty may turn out to be suboptimal. In a resource constrained system in which there is a fixed budget, these suboptimal ...decisions will result in health loss. Consequently, there may be value in reducing uncertainty, through the collection of new evidence, to make better resource allocation decisions. This value can be quantified using a value of information (VOI) analysis. This report, from the ISPOR VOI Task Force, introduces VOI analysis, defines key concepts and terminology, and outlines the role of VOI for supporting decision making, including the steps involved in undertaking and interpreting VOI analyses. The report is specifically aimed at those tasked with making decisions about the adoption of healthcare or the funding of healthcare research. The report provides a number of recommendations for good practice when planning, undertaking, or reviewing the results of VOI analyses.
•Decision uncertainty, although not relevant to a risk-neutral decision maker identifying the optimal choice in the current circumstances, is of interest for addressing the question of whether to collect additional information to better inform future decisions. As such, probability distributions should be assigned to parameters to characterize uncertainty in the current evidence base, with probabilistic analysis (PA) used to assess the uncertainty. Parameters excluded from the PA will be excluded from the analysis of uncertainty.•A value of information (VOI) analysis provides a formal assessment of the value of research, based on the extent to which the information generated through research would improve the expected payoffs associated with a decision by reducing the uncertainty surrounding it. This value can then be compared with the cost of acquiring the information to determine whether the research is potentially worthwhile and of value to undertake.•This report was written to provide decision makers who have been tasked with making decisions about the adoption of healthcare or the funding of healthcare research with an introduction to the concept of VOI analysis and to the decisions that can be supported by this type of analysis, including: (1) research prioritization, (2) efficient research design, (3) reimbursement, and (4) efficient decision making over the life cycle.•The report describes the process of VOI analysis, providing a top-level description of the methods and steps involved in undertaking and interpreting the results of such an analysis, from conceptualizing the decision problem to developing the decision model, parameterizing the model, running the probabilistic analysis, calculating the value of information (perfect, partial perfect, and sample), and determining the worth of research (expected net benefit of sampling).•This report provides 9 recommendations for good practice when planning, undertaking, or reviewing the results of VOI analyses with the aim to improve accessibility of VOI analysis for all stakeholders.
The allocation of healthcare resources among competing priorities requires an assessment of the expected costs and health effects of investing resources in the activities and of the opportunity cost ...of the expenditure. To date, much effort has been devoted to assessing the expected costs and health effects, but there remains an important need to also reflect the consequences of uncertainty in resource allocation decisions and the value of further research to reduce uncertainty. Decision making with uncertainty may turn out to be suboptimal, resulting in health loss. Consequently, there may be value in reducing uncertainty, through the collection of new evidence, to better inform resource decisions. This value can be quantified using value of information (VOI) analysis. This report from the ISPOR VOI Task Force describes methods for computing 4 VOI measures: the expected value of perfect information, expected value of partial perfect information (EVPPI), expected value of sample information (EVSI), and expected net benefit of sampling (ENBS). Several methods exist for computing EVPPI and EVSI, and this report provides guidance on selecting the most appropriate method based on the features of the decision problem. The report provides a number of recommendations for good practice when planning, undertaking, or reviewing VOI analyses. The software needed to compute VOI is discussed, and areas for future research are highlighted.
•Value of information (VOI) analysis provides a framework for quantifying the value of acquiring additional information to reduce uncertainty in decision making. Quantifying the expected improvement with new information requires an assessment of the scale and consequences of uncertainty in terms of payoffs. Acquiring information, however, can be costly. Therefore, the value of new information is compared with the cost of acquiring the information to determine whether it is worthwhile.•This report provides practical guidance on the methods and reporting of VOI analysis. The methods are presented in generic form to allow them to be adapted to any specific decision-making context. This means that even in healthcare systems in which economic considerations are not explicitly incorporated into decision making, the same methods can be applied.•This report provides 8 recommendations for good practice when planning, undertaking, or reviewing VOI analyses. The primary audience for the report is methodologists or analysts who are responsible for undertaking VOI analysis to inform decision making.
Overview of Cost-effectiveness Analysis Sanders, Gillian D; Maciejewski, Matthew L; Basu, Anirban
JAMA : the journal of the American Medical Association,
04/2019, Letnik:
321, Številka:
14
Journal Article
Recenzirano
This JAMA Guide to Statistics and Methods reviews the use of cost-effectiveness analysis to quantify the tradeoffs in costs, harms, and benefits of new health care interventions compared with ...existing interventions.
Cost-Effectiveness Analysis 2.0 Neumann, Peter J; Sanders, Gillian D
The New England journal of medicine,
01/2017, Letnik:
376, Številka:
3
Journal Article
Recenzirano
As prominent groups in U.S. health care ramp up use of cost-effectiveness analysis to measure and communicate the value of new drugs and other interventions, an expert panel has released updated ...guidelines for such analysis.
Cost-effectiveness analysis in U.S. health care seems poised for a second act of sorts. Although it has never actually gone away, efforts to apply it have encountered resistance, and the federal government and some health care organizations have sometimes prohibited its use or relegated it to a minor role. But several developments are helping to recharge the field. One is the embrace of its methods by prominent groups that are using it to measure and communicate the value of new drugs and other interventions. Another is the publication of new guidelines for such analysis from a national panel that is . . .
The need for consistent and current data describing the true incidence of sudden cardiac arrest (SCA) and/or sudden cardiac death (SCD) was highlighted during the most recent Sudden Cardiac Arrest ...Thought Leadership Alliance's (SCATLA) Think Tank meeting of national experts with broad representation of key stakeholders, including thought leaders and representatives from the American College of Cardiology, American Heart Association, and the Heart Rhythm Society. As such, to evaluate the true magnitude of this public health problem, we performed a systematic literature search in MEDLINE using the MeSH headings, “death, sudden” OR the terms “sudden cardiac death” OR “sudden cardiac arrest” OR “cardiac arrest” OR “cardiac death” OR “sudden death” OR “arrhythmic death.” Study selection criteria included peer-reviewed publications of primary data used to estimate SCD incidence in the U.S. We used Web of Science's Cited Reference Search to evaluate the impact of each primary estimate on the medical literature by determining the number of times each “primary source” has been cited. The estimated U.S. annual incidence of SCD varied widely from 180,000 to >450,000 among 6 included studies. These different estimates were in part due to different data sources (with data age ranging from 1980 to 2007), definitions of SCD, case ascertainment criteria, methods of estimation/extrapolation, and sources of case ascertainment. The true incidence of SCA and/or SCD in the U.S. remains unclear, with a wide range in the available estimates that are badly dated. As reliable estimates of SCD incidence are important for improving risk stratification and prevention, future efforts are clearly needed to establish uniform definitions of SCA and SCD and then to prospectively and precisely capture cases of SCA and SCD in the overall U.S. population.
Abstract
Background
Atrial fibrillation (AF) is a common cardiac arrhythmia that increases the risk of stroke. Medical therapy for decreasing stroke risk involves anticoagulation, which may increase ...bleeding risk for certain patients. In determining the optimal therapy for stroke prevention for patients with AF, clinicians use tools with various clinical, imaging and patient characteristics to weigh stroke risk against therapy-associated bleeding risk.
Aim
This article reviews published literature and summarizes available risk stratification tools for stroke and bleeding prediction in patients with AF.
Methods
We searched for English-language studies in PubMed, Embase and the Cochrane Database of Systematic Reviews published between 1 January 2000 and 14 February 2018. Two reviewers screened citations for studies that examined tools for predicting thromboembolic and bleeding risks in patients with AF. Data regarding study design, patient characteristics, interventions, outcomes, quality, and applicability were extracted.
Results
Sixty-one studies were relevant to predicting thromboembolic risk and 38 to predicting bleeding risk. Data suggest that CHADS
2
, CHA
2
DS
2
-VASc and the age, biomarkers, and clinical history (ABC) risk scores have the best evidence for predicting thromboembolic risk (moderate strength of evidence for limited prediction ability of each score) and that HAS-BLED has the best evidence for predicting bleeding risk (moderate strength of evidence).
Limitations
Studies were heterogeneous in methodology and populations of interest, setting, interventions and outcomes analysed.
Conclusion
CHADS
2
, CHA
2
DS
2
-VASc and ABC scores have the best prediction for stroke events, and HAS-BLED provides the best prediction for bleeding risk. Future studies should define the role of imaging tools and biomarkers in enhancing the accuracy of risk prediction tools.
Primary Funding Source
Patient-Centered Outcomes Research Institute (PROSPERO #CRD42017069999)
IMPORTANCE: Since publication of the report by the Panel on Cost-Effectiveness in Health and Medicine in 1996, researchers have advanced the methods of cost-effectiveness analysis, and policy makers ...have experimented with its application. The need to deliver health care efficiently and the importance of using analytic techniques to understand the clinical and economic consequences of strategies to improve health have increased in recent years. OBJECTIVE: To review the state of the field and provide recommendations to improve the quality of cost-effectiveness analyses. The intended audiences include researchers, government policy makers, public health officials, health care administrators, payers, businesses, clinicians, patients, and consumers. DESIGN: In 2012, the Second Panel on Cost-Effectiveness in Health and Medicine was formed and included 2 co-chairs, 13 members, and 3 additional members of a leadership group. These members were selected on the basis of their experience in the field to provide broad expertise in the design, conduct, and use of cost-effectiveness analyses. Over the next 3.5 years, the panel developed recommendations by consensus. These recommendations were then reviewed by invited external reviewers and through a public posting process. FINDINGS: The concept of a “reference case” and a set of standard methodological practices that all cost-effectiveness analyses should follow to improve quality and comparability are recommended. All cost-effectiveness analyses should report 2 reference case analyses: one based on a health care sector perspective and another based on a societal perspective. The use of an “impact inventory,” which is a structured table that contains consequences (both inside and outside the formal health care sector), intended to clarify the scope and boundaries of the 2 reference case analyses is also recommended. This special communication reviews these recommendations and others concerning the estimation of the consequences of interventions, the valuation of health outcomes, and the reporting of cost-effectiveness analyses. CONCLUSIONS AND RELEVANCE: The Second Panel reviewed the current status of the field of cost-effectiveness analysis and developed a new set of recommendations. Major changes include the recommendation to perform analyses from 2 reference case perspectives and to provide an impact inventory to clarify included consequences.
Oral contraceptives may influence the risk of certain cancers. As part of the AHRQ Evidence Report, Oral Contraceptive Use for the Primary Prevention of Ovarian Cancer, we conducted a systematic ...review to estimate associations between oral contraceptive use and breast, cervical, colorectal, and endometrial cancer incidence. We searched PubMed, Embase, and Cochrane Database of Systematic Reviews. Study inclusion criteria were women taking oral contraceptives for contraception or ovarian cancer prevention; includes comparison group with no oral contraceptive use; study reports quantitative associations between oral contraceptive exposure and relevant cancers; controlled study or pooled patient-level meta-analyses; sample size for nonrandomized studies ≥100; peer-reviewed, English-language; published from January 1, 2000 forward. Random-effects meta-analyses were conducted by estimating pooled ORs with 95% confidence intervals (CIs). We included 44 breast, 12 cervical, 11 colorectal, and 9 endometrial cancers studies. Breast cancer incidence was slightly but significantly increased in users (OR, 1.08; CI, 1.00-1.17); results show a higher risk associated with more recent use of oral contraceptives. Risk of cervical cancer was increased with duration of oral contraceptive use in women with human papillomavirus infection; heterogeneity prevented meta-analysis. Colorectal cancer (OR, 0.86; CI, 0.79-0.95) and endometrial cancer incidences (OR, 0.57; CI, 0.43-0.77) were significantly reduced by oral contraceptive use. Compared with never use, ever use of oral contraceptives is significantly associated with decreases in colorectal and endometrial cancers and increases in breast cancers. Although elevated breast cancer risk was small, relatively high incidence of breast cancers means that oral contraceptives may contribute to a substantial number of cases.
To model the long-term clinical and economic outcomes of potential cord blood therapy in autism spectrum disorder (ASD).
Markov microsimulation of ASD over the lifespan was used to compare two ...strategies: 1) standard of care (SOC), including behavioral and educational interventions, and 2) novel cord blood (CB) intervention in addition to SOC. Input data reflecting behavioral outcomes included baseline Vineland Adaptive Behavior Scale (VABS-3), monthly VABS-3 changes, and CB intervention efficacy on adaptive behavior based on a randomized, placebo-controlled trial (DukeACT). Quality-adjusted life-years (QALYs) were correlated to VABS-3. Costs for children with ASD ($15,791, ages 2-17 years) and adults with ASD ($56,559, ages 18+ years), and the CB intervention (range $15,000-45,000) were incorporated. Alternative CB efficacy and costs were explored.
We compared model-projected results to published data on life-expectancy, mean VABS-3 changes, and lifetime costs. Undiscounted lifetime QALYs in the SOC and CB strategies were 40.75 and 40.91. Discounted lifetime costs in the SOC strategy were $1,014,000, and for CB ranged from $1,021,000-$1,058,000 with CB intervention cost ($8,000-$45,000). At $15,000 cost, CB was borderline cost-effective (ICER = $105,000/QALY). In one-way sensitivity analysis, CB cost and efficacy were the most influential parameters on CB ICER. CB intervention was cost-effective at costs<$15,000 and efficacies ≥2.0. Five-year healthcare payer projected budgetary outlays at a $15,000 CB cost were $3.847B.
A modestly effective intervention designed to improve adaptive behavior in autism can be cost-effective under certain circumstances. Intervention cost and efficacy most affected the cost-effectiveness results and should be targeted to increase economic efficiency.
IMPORTANCE: Patients need to consider both benefits and harms of breast cancer screening. OBJECTIVE: To systematically synthesize available evidence on the association of mammographic screening and ...clinical breast examination (CBE) at different ages and intervals with breast cancer mortality, overdiagnosis, false-positive biopsy findings, life expectancy, and quality-adjusted life expectancy. EVIDENCE REVIEW: We searched PubMed (to March 6, 2014), CINAHL (to September 10, 2013), and PsycINFO (to September 10, 2013) for systematic reviews, randomized clinical trials (RCTs) (with no limit to publication date), and observational and modeling studies published after January 1, 2000, as well as systematic reviews of all study designs. Included studies (7 reviews, 10 RCTs, 72 observational, 1 modeling) provided evidence on the association between screening with mammography, CBE, or both and prespecified critical outcomes among women at average risk of breast cancer (no known genetic susceptibility, family history, previous breast neoplasia, or chest irradiation). We used summary estimates from existing reviews, supplemented by qualitative synthesis of studies not included in those reviews. FINDINGS: Across all ages of women at average risk, pooled estimates of association between mammography screening and mortality reduction after 13 years of follow-up were similar for 3 meta-analyses of clinical trials (UK Independent Panel: relative risk RR, 0.80 95% CI, 0.73-0.89; Canadian Task Force: RR, 0.82 95% CI, 0.74-0.94; Cochrane: RR, 0.81 95% CI, 0.74-0.87); were greater in a meta-analysis of cohort studies (RR, 0.75 95% CI, 0.69 to 0.81); and were comparable in a modeling study (CISNET; median RR equivalent among 7 models, 0.85 range, 0.77-0.93). Uncertainty remains about the magnitude of associated mortality reduction in the entire US population, among women 40 to 49 years, and with annual screening compared with biennial screening. There is uncertainty about the magnitude of overdiagnosis associated with different screening strategies, attributable in part to lack of consensus on methods of estimation and the importance of ductal carcinoma in situ in overdiagnosis. For women with a first mammography screening at age 40 years, estimated 10-year cumulative risk of a false-positive biopsy result was higher (7.0% 95% CI, 6.1%-7.8%) for annual compared with biennial (4.8% 95% CI, 4.4%-5.2%) screening. Although 10-year probabilities of false-positive biopsy results were similar for women beginning screening at age 50 years, indirect estimates of lifetime probability of false-positive results were lower. Evidence for the relationship between screening and life expectancy and quality-adjusted life expectancy was low in quality. There was no direct evidence for any additional mortality benefit associated with the addition of CBE to mammography, but observational evidence from the United States and Canada suggested an increase in false-positive findings compared with mammography alone, with both studies finding an estimated 55 additional false-positive findings per extra breast cancer detected with the addition of CBE. CONCLUSIONS AND RELEVANCE: For women of all ages at average risk, screening was associated with a reduction in breast cancer mortality of approximately 20%, although there was uncertainty about quantitative estimates of outcomes for different breast cancer screening strategies in the United States. These findings and the related uncertainty should be considered when making recommendations based on judgments about the balance of benefits and harms of breast cancer screening.
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