Evaluating Frameworks That Provide Value Measures for Health Care Interventions

The recent acceleration of scientific discovery has led to greater choices in health care. New technologies, diagnostic tests, and pharmaceuticals have widely varying impact on patients and populations in terms of benefits, toxicities, and costs, stimulating a resurgence of interest in the creation of frameworks intended to measure value in health. Many of these are offered by providers and/or advocacy organizations with expertise and interest in specific diseases (e.g., cancer and heart disease). To help assess the utility of and the potential biases embedded in these frameworks, we created an evaluation taxonomy with seven basic components: 1) define the purpose; 2) detail the conceptual approach, including perspectives, methods for obtaining preferences of decision makers (e.g., patients), and ability to incorporate multiple dimensions of value; 3) discuss inclusions and exclusions of elements included in the framework, and whether the framework assumes clinical intervention or offers alternatives such as palliative care or watchful waiting; 4) evaluate data sources and their scientific validity; 5) assess the intervention’s effect on total costs of treating a defined population; 6) analyze how uncertainty is incorporated; and 7) illuminate possible conflicts of interest among those creating the framework. We apply the taxonomy to four representative value frameworks recently published by professional organizations focused on treatment of cancer and heart disease and on vaccine use. We conclude that each of these efforts has strengths and weaknesses when evaluated using our taxonomy, and suggest pathways to enhance the utility of value-assessing frameworks for policy and clinical decision making.     

Validity and Reliability of Value Assessment Frameworks for New Cancer Drugs

Background

Several organizations have developed frameworks to systematically assess the value of new drugs. These organizations include the American Society of Clinical Oncology (ASCO), the European Society for Medical Oncology (ESMO), the Institute for Clinical and Economic Review (ICER), and the National Comprehensive Cancer Network (NCCN).

Challenges with Forecasting Budget Impact: A Case Study of Six ICER Reports

Background

Payers frequently rely on budget impact model (BIM) results to help determine drug coverage policy and its effect on their bottom line. It is unclear whether BIMs typically overestimate or underestimate real-world budget impact.

Toward a Broader Concept of Value: Identifying and Defining Elements for an Expanded Cost-Effectiveness Analysis

This commentary identifies and defines potentially useful expansions to traditional cost-effectiveness analysis as often used in health technology assessment. Since the seminal 1977 article by Weinstein and Stason, the recommended approach has been the use of the incremental cost-effectiveness ratio based on the metric of the cost per quality-adjusted life-year gained, allowing comparisons across different technologies. An expanded framework, incorporating a wider range of the elements of value, is proposed. In addition to the core value drivers of health gain and other health system cost savings (if any), we propose adding other less recognized elements related to the value of knowing and informational externalities. We describe each of five factors related to the value of knowing: 1) a reduction in uncertainty, reflecting the benefit of a companion diagnostic increasing the certainty of a patient?s response to a medicine; 2) insurance value related to greater peace of mind due to protection against catastrophic health and financial loss; 3) the value of hope for a "cure," leading individuals to become risk seekers in some circumstances; 4) real option value due to life extension opening possibilities for individuals to benefit from future innovation; and 5) spillovers or externalities arising from benefits of scientific advances that cannot be entirely appropriated by those making the advances. Further thought and research are needed on how best to measure and integrate these elements into an incremental value framework and on coverage and pricing decisions.     

Assessing the Economic Value of Clinical Artificial Intelligence: Challenges and Opportunities

ObjectivesClinical artificial intelligence (AI) is a novel technology, and few economic evaluations have focused on it to date. Before its wider implementation, it is important to highlight the aspects of AI that challenge traditional health technology assessment methods.MethodsWe used an existing broad value framework to assess potential ways AI can provide good value for money. We also developed a rubric of how economic evaluations of AI should vary depending on the case of its use.ResultsWe found that the measurement of core elements of value—health outcomes and cost—are complicated by AI because its generalizability across different populations is often unclear and because its use may necessitate reconfigured clinical processes. Clinicians’ productivity may improve when AI is used. If poorly implemented though, AI may also cause clinicians’ workload to increase. Some AI has been found to exacerbate health disparities. Nevertheless, AI may promote equity by expanding access to medical care and, when properly trained, providing unbiased diagnoses and prognoses. The approach to assessment of AI should vary based on its use case: AI that creates new clinical possibilities can improve outcomes, but regulation and evidence collection may be difficult; AI that extends clinical expertise can reduce disparities and lower costs but may result in overuse; and AI that automates clinicians’ work can improve productivity but may reduce skills.ConclusionsThe potential uses of clinical AI create challenges for health technology assessment methods originally developed for pharmaceuticals and medical devices. Health economists should be prepared to examine data collection and methods used to train AI, as these may impact its future value.     

Developing a Value Framework: The Need to Reflect the Opportunity Costs of Funding Decisions

A growing number of health care systems internationally use formal economic evaluation methods to support health care funding decisions. Recently, a range of organizations have been advocating forms of analysis that have been termed “value frameworks.” There has also been a push for analytical methods to reflect a fuller range of benefits of interventions through multicriteria decision analysis. A key principle that is invariably neglected in current and proposed frameworks is the need to reflect evidence on the opportunity costs that health systems face when making funding decisions. The mechanisms by which opportunity costs are realized vary depending on the system’s financial arrangements, but they always mean that a decision to fund a specific intervention for a particular patient group has the potential to impose costs on others in terms of forgone benefits. These opportunity costs are rarely explicitly reflected in analysis to support decisions, but recent developments to quantify benefits forgone make more appropriate analyses feasible. Opportunity costs also need to be reflected in decisions if a broader range of attributes of benefit is considered, and opportunity costs are a key consideration in determining the appropriate level of total expenditure in a system. The principles by which opportunity costs can be reflected in analysis are illustrated in this article by using the example of the proposed methods for value-based pricing in the United Kingdom.     

The Impact of Broader Value Elements on Cost-Effectiveness Analysis: Two Case Studies

ObjectivesThis study aimed to explore the impact of including broader value elements in cost-effectiveness analyses by presenting 2 case studies, one on human papillomavirus (HPV) infection and one on early-stage Hodgkin’s lymphoma (ESHL).MethodsWe identified broader value elements (eg, patient and caregiver time, spillover health effects, productivity) from the Second Panel’s Impact Inventory and the ISPOR Special Task Force’s value flower. We then evaluated the cost-effectiveness of HPV vaccination versus no vaccination (case 1) and combined modality therapy (CMT) versus chemotherapy alone for treatment of adult ESHL (case 2) using published simulation models. For each case study, we compared incremental cost-effectiveness ratios considering health sector impacts only (the “base-case” scenario) with incremental cost-effectiveness ratios incorporating broader value elements.ResultsFor vaccination of US girls against HPV before sexual debut versus no vaccination, the base-case result was $38 334 per disability-adjusted life-year averted. Including each broader value element made cost-effectiveness progressively more favorable, with HPV vaccination becoming cost-saving (ie, reducing costs and averting more disability-adjusted life-years) when the analysis incorporated productivity costs. For CMT versus chemotherapy alone in patients with ESHL, the base-case result indicated that CMT was cost-saving. Including all elements made this treatment’s net monetary benefits (the sum of its averted resource costs and the net value of its health impacts) less favorable, even as the contribution from CMT’s near-term health benefits grew.ConclusionsIncluding broader value elements can substantially influence cost-effectiveness ratios, although the direction and the magnitude of their impact can differ across interventions and disease context.     

Cost-effectiveness analysis of day care for patients with dementia disorders

In a cost-effectiveness analysis, the costs of 55 demented patients in day care (DC) and 45 patients on a waiting list were related to the effects on indices measuring quality of life. Based on one year's study, the costs per patient per day, was USD 55 for the DC-group and USD 63 for the controls (p > 0.05). Both groups deteriorated in the indices during the study year. The trend was that DC was both cheaper and better in the indices, and some of the underlying variables for the used indices also showed significantly better results in favour of DC. However, since the changes between the groups were not significant regarding the basic question at issue, it cannot be stated that DC is more cost-effective than a care organisation without DC. Different interpretations of the results are presented.     

Value of Research and Value of Development in Early Assessments of New Medical Technologies

ObjectivesIn early stages of development of new medical technologies, there are conceptually separate but related societal decisions to be made concerning adoption, further development (i.e., technical improvement), and research (i.e., clinical trials) of new technologies. This article presents a framework to simultaneously support these three decisions from a societal perspective. The framework is applied to the 70-gene signature, a gene-expression profile for breast cancer, deciding which patients should receive adjuvant systemic therapy after surgery. The “original” signature performed on fresh frozen tissue (70G-FFT) could be further developed to a paraffin-based signature (70G-PAR) to reduce test failures.MethodsA Markov decision model comparing the “current” guideline Adjuvant Online (AO), 70G-FFT, and 70G-PAR was used to simulate 20-year costs and outcomes in a hypothetical cohort in The Netherlands. The 70G-PAR strategy was based on projected data from a comparable technology. Incremental net monetary benefits were calculated to support the adoption decision. Expected net benefit of development for the population and expected net benefit of sampling were calculated to support the development and research decision.ResultsThe 70G-PAR had the highest net monetary benefit, followed by the 70G-FFT. The population expected net benefit of development amounted to €91 million over 20 years (assuming €250 development costs per patient receiving the test). The expected net benefit of sampling amounted to €61 million for the optimal trial (n = 4000).ConclusionsWe presented a framework to simultaneously support adoption, development, and research decisions in early stages of medical technology development. In this case, the results indicate that there is value in both further development of 70G-FFT into 70G-PAR and further research.     

Cost Effectiveness of the Angiotensin Receptor Neprilysin Inhibitor Sacubitril/Valsartan for Patients with Chronic Heart Failure and Reduced Ejection Fraction in the Netherlands: A Country Adaptation Analysis Under the Former and Current Dutch Pharmacoeconomic Guidelines

Objectives

To describe the adaptation of a global health economic model to determine whether treatment with the angiotensin receptor neprilysin inhibitor LCZ696 is cost effective compared with the angiotensin-converting enzyme inhibitor enalapril in adult patients with chronic heart failure with reduced left ventricular ejection fraction in the Netherlands; and to explore the effect of performing the cost-effectiveness analyses according to the new pharmacoeconomic Dutch guidelines (updated during the submission process of LCZ696), which require a value-of-information analysis and the inclusion of indirect medical costs of life-years gained.

医疗保健公平:价值理念选择与实现路径

公平、正义和平等作为公平价值理念的常规表达方式,其含义看似相近,但其价值内核却存在根本差异。平等理念从人权伦理出发,主张医疗资源配置的结果均等,理应成为医疗保健领域公平价值理念的最优选择;囿于现实条件的约束,医疗资源的配置无法做到绝对的结果均等,必须要进行保健优先权的设置。     

Evaluating the Consistency Between Conceptual Frameworks and Factors Influencing the Safe Behavior of Iranian Workers in the Petrochemical Industry: Mixed Methods Study

BackgroundUnsafe worker behavior is often identified as a major cause of dangerous incidents in the petrochemical industry. Behavioral safety models provide frameworks that may help to prevent such incidents by identifying factors promoting safe or unsafe behavior. We recently conducted a qualitative study to identify factors affecting workers'' unsafe behaviors in an Iranian petrochemical company.ObjectiveThe aims of this study were to (1) conduct a review of the relevant research literature between the years 2000 and 2019 to identify theoretical models proposed to explain and predict safe behavior in the workplace and (2) to select the model that best reflects our qualitative findings and other evidence about the factors influencing safe behaviors among petrochemical workers.MethodsThis research used mixed methods. Initially, we conducted a qualitative study of factors that Iranian petrochemical workers believed affected their safety behavior. Four themes emerged from the semistructured interviews: (1) poor direct safety management and supervision; (2) unsafe workplace conditions; (3) workers’ perceptions, skills, and training; and (4) broader organizational factors. Electronic databases, including PubMed, Embase, Scopus, Google Scholar, EBSCOhost, and Science Direct, were then searched for eligible studies on models to explain and predict safe behaviors, which were published between the years 2000 and 2019. Medical subject headings were used as the primary analytical element. Medical subject headings and subheadings were then extracted from the literature. One researcher conducted the search and 3 researchers performed screening and data extraction. Then, constructs described in each study were assessed to determine which were the most consistent with themes derived from our qualitative analysis.ResultsA total of 2032 publications were found using the search strategy. Of these, 142 studies were assessed and 28 studies met the inclusion criteria and were included in the review. The themes identified in the qualitative study most closely matched 3 scales included in Wu et al''s model that measured safety behavior and performance, safety leadership, and safety climate in petrochemical industries. Poor direct safety management and supervision matched with safety leadership and its subscales; unsafe workplace conditions matched with safety climate and its subscales; workers'' perceptions, skills, and training matched with safety performance and its subscales; and broader organizational factors matched with some subscales of the model.ConclusionsThis is the first literature review to identify models intended to explain and predict safe behavior and select the model most consistent with themes elicited from a qualitative study. Our results showed that effective safety leadership and management and safety climate and culture systems are the most frequently identified factors affecting safe behaviors in the petrochemical industry. These results can further help safety researchers and professionals design effective behavior-based safety interventions, which can have a more sustainable and persistent impact on workers’ safety behaviors.Trial RegistrationIranian Registry of Clinical Trials IRCT20170515033981N2; https://www.irct.ir/trial/26107International Registered Report Identifier (IRRID)RR2-10.1186/s12889-019-7126-1     

The ICER Value Framework: Integrating Cost Effectiveness and Affordability in the Assessment of Health Care Value

What should be the relationship between the concepts of cost effectiveness and affordability in value assessments for health care interventions? This question has received greater attention in recent years given increasing financial pressures on health systems, leading to different views on how assessment reports and decision-making processes can provide the best structure for considering both elements. In the United States, the advent of explicit value frameworks to guide drug assessments has also focused attention on this issue, driven in part by the prominent inclusion of affordability within the value framework used to guide reports from the Institute for Clinical and Economic Review. After providing a formal definition of affordability for health care systems, this article argues that, even after using empirical estimates of true health system opportunity cost, cost-effectiveness thresholds cannot by themselves be set in a way that subsumes questions about short-term affordability. The article then presents an analysis of different approaches to integrating cost effectiveness and budget impact assessments within information to guide decision making. The evolution and experience with the Institute for Clinical and Economic Review value framework are highlighted, providing lessons learned and guiding principles for future efforts to bring measures of affordability within the scope of value assessment.     

Model-Based Cost-Effectiveness Analyses for the Treatment of Acute Stroke Events: A Review and Summary of Challenges

Objective:  To summarize the methodological approaches used in published decision-analytic models evaluating interventions for acute stroke treatment, to highlight key components of decision-analytic models of stroke treatment, and to discuss challenges for developing stroke decision models.
Methods:  A review of the published literature was performed using Medline, to identify studies involving mathematical decision models to evaluate interventions for acute stroke treatment. Articles were analyzed to determine key components of a stroke model and to note areas in which data are lacking.
Results:  We identified 13 published models of acute stroke treatment. These models typically possessed a short-term treatment module and a long-term post-treatment module. The following aspects of economic modeling were found to be relevant for developing a stroke model: modeling approach and health state; health state transition probabilities; estimation of short-term, long-term, and indirect costs; health state utilities; poststroke mortality; time horizon; model validation; and estimation of parameter uncertainty.
Conclusions:  Data gaps have limited the development of economic models in stroke to date. In order to more accurately assess the long-term incremental impact of a new treatment of stroke, future research is needed to address these data gaps. We recommend that the complexity of models for examining the cost-effectiveness of an acute stroke treatment be kept to a minimum such that it can incorporate the currently available data without making a large number of assumptions around the data.     

Integrating Alternative Social Value Judgments Into Cost-Effectiveness Analysis of Vaccines: An Application to Varicella-Zoster Virus Vaccination

ObjectivesCost-effectiveness analyses (CEA) are based on the value judgment that health outcomes (eg, quantified in quality-adjusted life-years; QALYs) are all equally valuable irrespective of their context. Whereas most published CEAs perform extensive sensitivity analysis on various parameters and assumptions, only rarely is the influence of the QALY-equivalence assumption on cost-effectiveness results investigated. We illustrate how the integration of alternative social value judgments in CEA can be a useful form of sensitivity analysis.MethodsBecause varicella-zoster virus (VZV) vaccination affects 2 distinct diseases (varicella zoster and herpes zoster) and likely redistributes infections across different age groups, the program has an important equity dimension. We used a cost-effectiveness model and disentangled the share of direct protection and herd immunity within the total projected QALYs resulting from a 50-year childhood VZV program in the UK. We use the UK population’s preferences for QALYs in the vaccine context to revalue QALYs accordingly.ResultsRevaluing different types of QALYs for different age groups in line with public preferences leads to a 98% change in the projected net impact of the program. The QALYs gained among children through direct varicella protection become more important, whereas the QALYs lost indirectly through zoster in adults diminish in value. Weighting of vaccine-related side effects made a large difference.ConclusionsOur study shows that a sensitivity analysis in which alternative social value judgments about the value of health outcomes are integrated into CEA of vaccines is relatively straightforward and provides important additional information for decision makers to interpret cost-effectiveness results.     

A Comparison of Coverage Restrictions for Biopharmaceuticals and Medical Procedures

Background

Differences in payer evaluation and coverage of pharmaceuticals and medical procedures suggest that coverage may differ for medications and procedures independent of their clinical benefit. We hypothesized that coverage for medications is more restricted than corresponding coverage for nonmedication interventions.

Uncertainty in Decision-Making: Value of Additional Information in the Cost-Effectiveness of Lifestyle Intervention in Overweight and Obese People

Objectives:  To quantify the uncertainty in the cost-effectiveness of lifestyle intervention versus standard care in overweight and obese people in Switzerland and to determine if further research is necessary based on current information. There has been an increasing interest in using value of information analysis in medical decision-making to quantify the uncertainty in decision-making, and to identify the need for further research.
Methods:  Value of information analysis was applied on a probabilistic cost-effectiveness model to evaluate the uncertainty by calculating the patient expected value of perfect information (EVPI), population EVPI, and partial EVPI. The costs were expressed in Swiss Francs (CHF), price year 2006.
Results:  Overall, the EVPI was higher in overweight than in obese people. The maximum population EVPI was CHF 6.8 million in overweight people and CHF 3.2 million in moderate obese people, representing the upper limit on costs associated with decision uncertainty. The partial EVPI estimated a higher uncertainty in the model parameters such as utilities, body mass index, cardiovascular risk factors, and systolic blood pressure in overweight and moderate obese subjects.
Conclusions:  The EVPI analysis indicates that there is some uncertainty regarding the choice between lifestyle intervention and standard care. The parameter EVPI suggests that if further research is commissioned, this should focus on the effectiveness of lifestyle intervention on cardiovascular risk factors and utilities.     

中国卫生改革的形势:问题和挑战

分析了当前中国卫生改革的形势，认为存在的主要问题是卫生系统绩效差，医院市场化、医疗费用增长、需求受抑制，城乡之间在卫生服务覆盖率、可及性和健康结果等方面的差异拉大。总结了三医联动改革的成就和今后改革的方向，认为中国需要做更多的循证政策研究来解决自身的问题并迎接挑战。     

Cost-Effectiveness Analysis of a Pharmacist-Led Medication Therapy Management Program: Hypertension Management

ObjectivesUncontrolled hypertension is a common cause of cardiovascular disease, which is the deadliest and costliest chronic disease in the United States. Pharmacists are an accessible community healthcare resource and are equipped with clinical skills to improve the management of hypertension through medication therapy management (MTM). Nevertheless, current reimbursement models do not incentivize pharmacists to provide clinical services. We aim to investigate the cost-effectiveness of a pharmacist-led comprehensive MTM clinic compared with no clinic for 10-year primary prevention of stroke and cardiovascular disease events in patients with hypertension.MethodsWe built a semi-Markov model to evaluate the clinical and economic consequences of an MTM clinic compared with no MTM clinic, from the payer perspective. The model was populated with data from a recently published controlled observational study investigating the effectiveness of an MTM clinic. Methodology was guided using recommendations from the Second Panel on Cost-Effectiveness in Health and Medicine, including appropriate sensitivity analyses.ResultsCompared with no MTM clinic, the MTM clinic was cost-effective with an incremental cost-effectiveness ratio of $38 798 per quality-adjusted life year (QALY) gained. The incremental net monetary benefit was $993 294 considering a willingness-to-pay threshold of $100 000 per QALY. Health-benefit benchmarks at $100 000 per QALY and $150 000 per QALY translate to a 95% and 170% increase from current reimbursement rates for MTM services.ConclusionsOur model shows current reimbursement rates for pharmacist-led MTM services may undervalue the benefit realized by US payers. New reimbursement models are needed to allow pharmacists to offer cost-effective clinical services.     

攀枝花市某三甲医院腰椎间盘突出患者住院费用因子分析

目的探讨腰椎间盘住院患者医疗费用的主要支配因子,为控制住院患者的医疗费用过度增长提供参考依据。方法以攀枝花市某三甲医院骨科2018年就诊的腰椎间盘突出患者954例作为研究样本,使用因子分析法分析各项医疗费用。结果医疗服务药品因子、诊断相关因子、材料血液制品因子和检查手术因子是支配腰椎间盘突出患者住院费用的四个公因子。结论控制医疗费用的过度增长,根据支配因子,应着力控制材料费和药费。