Colonoscopy vs the Fecal Immunochemical Test: Which is Best?

Although there is no debate around the effectiveness of colorectal cancer screening in reducing disease burden, there remains a question regarding the most effective and cost-effective screening modality. Current United States guidelines present a panel of options that include the 2 most commonly used modalities, colonoscopy and stool testing with the fecal immunochemical test (FIT). Large-scale comparative effectiveness trials comparing colonoscopy and FIT for colorectal cancer outcomes are underway, but results are not yet available. This review will separately state the "best case" for FIT and colonoscopy as the screening tool of first choice. In addition, the review will examine these modalities from a health economics perspective to provide the reader further context about the relative advantages of these commonly used tests.

Challenges of Health Technology Assessment in Pluralistic Healthcare Systems: An ISPOR Council Report.

Health technology assessment (HTA) has been growing in use over the past 40 years, especially in its impact on decisions regarding the reimbursement, adoption, and use of new drugs, devices, and procedures. In countries or jurisdictions with "pluralistic" healthcare systems, there are multiple payers or sectors, each of which could potentially benefit from HTA. Nevertheless, a single HTA, conducted centrally, may not meet the needs of these different actors, who may have different budgets, current standards of care, populations to serve, or decision-making processes. This article reports on the research conducted by an ISPOR Health Technology Assessment Council Working Group established to examine the specific challenges of conducting and using HTA in countries with pluralistic healthcare systems. The Group used its own knowledge and expertise, supplemented by a narrative literature review and survey of US payers, to identify existing challenges and any initiatives taken to address them. We recommend that countries with pluralistic healthcare systems establish a national focus for HTA, develop a uniform set of HTA methods guidelines, ensure that HTAs are produced in a timely fashion, facilitate the use of HTA in the local setting, and develop a framework to encourage transparency in HTA. These efforts can be enhanced by the development of good practice guidance from ISPOR or similar groups and increased training to facilitate local use of HTA.

A Value Framework for the Assessment of Diagnostic Technologies: A Proposal Based on a Targeted Systematic Review and a Multistakeholder Deliberative Process in Latin America.

OBJECTIVES: there are very few value frameworks (VFs) to assess health technologies that are focused on diagnostic tests; they usually do not reflect a multistakeholder process; and they are all developed in high-income countries. Our project performed a targeted systematic review, with the objective of proposing an evidence-based, up-to-date VF informed by a multinational multistakeholder group working in the health technology assessment (HTA) space. METHODS: (1) A targeted systematic review, with the aim to identify existing VFs and their dimensions; and (2) generation a VF proposal through a mixed-methods, qualitative-quantitative approach. RESULTS: From 73 citations identified, 20 met our inclusion criteria and served to provide the initial list of dimensions for our VF. An initial list of criteria and subcriteria for a preliminary VF was proposed. After a full-day deliberative face-to-face meeting with 30 relevant stakeholders from seven Latin American countries and the United Kingdom, the final VF was defined, consisting of 15 criteria: five "essential or core," six highly relevant, three moderately relevant, and one of low relevance. Barriers and facilitators of value assessment of diagnostic technologies were also discussed. CONCLUSIONS: We propose a VF oriented to diagnostic technologies based on a targeted systematic review and a participatory process with key HTA stakeholders. It is the first to be produced in a lower and middle income setting but can also be potentially useful in other contexts aimed to assist decision-making processes with these particularly complex health technologies.

Measurement Instruments of Productivity Loss of Paid and Unpaid Work: A Systematic Review and Assessment of Suitability for Health Economic Evaluations From a Societal Perspective.

OBJECTIVES: This study aimed (1) to perform a systematic literature review of instruments for measuring productivity loss of paid and unpaid work and (2) to assess the suitability (in terms of identification, measurement, and valuation) of these instruments for use in health economic evaluations from a societal perspective. METHODS: Articles published from 2018 were sourced from PubMed/Medline, PsycInfo, Embase, and Econlit. Using 2 separate search strategies, eligible economic evaluations and validation studies were selected and unique measurement instruments identified. A data-extraction form was developed by studying previous literature and consulting an international panel of experts in the field of productivity costs. This data-extraction form was applied to assess the suitability of instruments for use in economic evaluations. RESULTS: A total of 5982 articles were retrieved from the databases, of which 99 economic evaluations and 9 validation studies were included in the review. A total of 42 unique measurement instruments were identified. Nine instruments provided quantified measures of absenteeism, presenteeism, and unpaid work. Five instruments supplied the necessary information to enable the use of at least 1 common valuation method. The Health and Labour Questionnaire-Short Form, Health and Labour Questionnaire, and Institute for Medical Technology Assessment Productivity Cost Questionnaire met both criteria. Nevertheless, the developers replaced the Health and Labour Questionnaire-Short Form and Health and Labour Questionnaire by the more recently developed Institute for Medical Technology Assessment Productivity Cost Questionnaire. CONCLUSIONS: Although many instruments for measuring productivity loss were identified, most were not suitable for capturing productivity changes for economic evaluations from a societal perspective. Future research can benefit from this study by making an informed instrument choice for the measurement of productivity loss of paid and unpaid work.

Do Social Values and Institutional Context Shape the Use of Economic Evaluation in Reimbursement Decisions? An Empirical Analysis.

OBJECTIVES: To investigate whether the use of economic evaluation (EE) in healthcare decision making is influenced by the social values and institutional context in a given country. METHODS: We developed and tested a conceptual framework for the 36 Organisation for Economic Co-operation and Development (OECD) countries. The countries were divided into two groups based on the extent of their use of EE in drug reimbursement. The key social values were efficiency, equity, and personal responsibility, measured in an international survey. Countries were classified based on their institutional context in terms of their general welfare paradigm/type of healthcare system and the administrative tradition to which they belong. We performed correlation tests and ran path analysis regression models to test our hypotheses. RESULTS: EE high users included significantly more Beveridge-type systems (50% vs 31%) and fewer Bismarck-type (15% vs 56%). Napoleonic tradition countries seemed to reject personal responsibility in health (r = -0.511, P = .009), whereas Germanic tradition countries embraced it (r = 0.572, P = .003); Anglo-American tradition countries exhibited a significant association with efficiency (r = 0.444, P = .026), whereas Scandinavian tradition countries appeared to reject it as a criterion for rationing in healthcare (r = -0.454, P = .023). No significant direct association was found between social values and use of EE. CONCLUSION: Our exploratory analysis suggests that institutional context and, indirectly, social values may play a role in shaping the use of EE in healthcare decision making. Because of the differences among countries in terms of institutional context, which may in part be influenced by social values, it is unlikely that there will ever be a single, harmonious approach to the use of EE.

Analytic Considerations in Applying a General Economic Evaluation Reference Case to Gene Therapy.

The concept of a reference case, first proposed by the US Panel on Cost-Effectiveness in Health and Medicine, has been used to specify the required methodological features of economic evaluations of healthcare interventions. In the case of gene therapy, there is a difference of opinion on whether a specific methodological reference case is required. The aim of this article was to provide a more detailed analysis of the characteristics of gene therapy and the extent to which these characteristics warrant modifications to the methods suggested in general reference cases for economic evaluation. We argue that a completely new reference case is not required, but propose a tailored checklist that can be used by analysts and decision makers to determine which aspects of economic evaluation should be considered further, given the unique nature of gene therapy.

Real-World Evidence in Healthcare Decision Making: Global Trends and Case Studies From Latin America.

BACKGROUND: Real-world evidence (RWE) is increasingly used to inform health technology assessments for resource allocation, which are valuable tools for emerging economies such as in America. Nevertheless, the characteristics and uses in South America are unknown. OBJECTIVES: To identify sources, characteristics, and uses of RWE in Argentina, Brazil, Colombia, and Chile, and evaluate the context-specific challenges. The implications for future regulation and responsible management of RWE in the region are also considered. METHODS: A systematic literature review, database mapping, and targeted gray literature search were conducted to identify the sources and characteristics of RWE. Findings were validated by key opinion leaders attending workshops in 4 South American countries. RESULTS: A database mapping exercise revealed 407 unique databases. Geographic scope, database type, population, and outcomes captured were reported. Characteristics of national health information systems show efforts to collect interoperable data from service providers, insurers, and government agencies, but that initiatives are hampered by fragmentation, lack of stewardship, and resources. In South America, RWE is mainly used for pharmacovigilance and as pure academic research, but less so for health technology assessment decision making or pricing negotiations and not at all to inform early access schemes. CONCLUSIONS: The quality of real-world data in the case study countries vary and RWE is not consistently used in healthcare decision making. Authors recommend that future studies monitor the impact of digitalization and the potential effects of access to RWE on the quality of patient care.

Objectives, Budgets, Thresholds, and Opportunity Costs-A Health Economics Approach: An ISPOR Special Task Force Report [4].

The fourth section of our Special Task Force report focuses on a health plan or payer's technology adoption or reimbursement decision, given the array of technologies, on the basis of their different values and costs. We discuss the role of budgets, thresholds, opportunity costs, and affordability in making decisions. First, we discuss the use of budgets and thresholds in private and public health plans, their interdependence, and connection to opportunity cost. Essentially, each payer should adopt a decision rule about what is good value for money given their budget; consistent use of a cost-per-quality-adjusted life-year threshold will ensure the maximum health gain for the budget. In the United States, different public and private insurance programs could use different thresholds, reflecting the differing generosity of their budgets and implying different levels of access to technologies. In addition, different insurance plans could consider different additional elements to the quality-adjusted life-year metric discussed elsewhere in our Special Task Force report. We then define affordability and discuss approaches to deal with it, including consideration of disinvestment and related adjustment costs, the impact of delaying new technologies, and comparative cost effectiveness of technologies. Over time, the availability of new technologies may increase the amount that populations want to spend on health care. We then discuss potential modifiers to thresholds, including uncertainty about the evidence used in the decision-making process. This article concludes by discussing the application of these concepts in the context of the pluralistic US health care system, as well as the "excess burden" of tax-financed public programs versus private programs.

Approaches to Aggregation and Decision Making-A Health Economics Approach: An ISPOR Special Task Force Report [5].

The fifth section of our Special Task Force report identifies and discusses two aggregation issues: 1) aggregation of cost and benefit information across individuals to a population level for benefit plan decision making and 2) combining multiple elements of value into a single value metric for individuals. First, we argue that additional elements could be included in measures of value, but such elements have not generally been included in measures of quality-adjusted life-years. For example, we describe a recently developed extended cost-effectiveness analysis (ECEA) that provides a good example of how to use a broader concept of utility. ECEA adds two features-measures of financial risk protection and income distributional consequences. We then discuss a further option for expanding this approach-augmented CEA, which can introduce many value measures. Neither of these approaches, however, provide a comprehensive measure of value. To resolve this issue, we review a technique called multicriteria decision analysis that can provide a comprehensive measure of value. We then discuss budget-setting and prioritization using multicriteria decision analysis, issues not yet fully resolved. Next, we discuss deliberative processes, which represent another important approach for population- or plan-level decisions used by many health technology assessment bodies. These use quantitative information on CEA and other elements, but the group decisions are reached by a deliberative voting process. Finally, we briefly discuss the use of stated preference methods for developing "hedonic" value frameworks, and conclude with some recommendations in this area.

A Health Economics Approach to US Value Assessment Frameworks-Summary and Recommendations of the ISPOR Special Task Force Report [7].

This summary section first lists key points from each of the six sections of the report, followed by six key recommendations. The Special Task Force chose to take a health economics approach to the question of whether a health plan should cover and reimburse a specific technology, beginning with the view that the conventional cost-per-quality-adjusted life-year metric has both strengths as a starting point and recognized limitations. This report calls for the development of a more comprehensive economic evaluation that could include novel elements of value (e.g., insurance value and equity) as part of either an "augmented" cost-effectiveness analysis or a multicriteria decision analysis. Given an aggregation of elements to a measure of value, consistent use of a cost-effectiveness threshold can help ensure the maximization of health gain and well-being for a given budget. These decisions can benefit from the use of deliberative processes. The six recommendations are to: 1) be explicit about decision context and perspective in value assessment frameworks; 2) base health plan coverage and reimbursement decisions on an evaluation of the incremental costs and benefits of health care technologies as is provided by cost-effectiveness analysis; 3) develop value thresholds to serve as one important input to help guide coverage and reimbursement decisions; 4) manage budget constraints and affordability on the basis of cost-effectiveness principles; 5) test and consider using structured deliberative processes for health plan coverage and reimbursement decisions; and 6) explore and test novel elements of benefit to improve value measures that reflect the perspectives of both plan members and patients.

International lessons in new methods for grading and integrating cost effectiveness evidence into clinical practice guidelines.

Economic evidence is influential in health technology assessment world-wide. Clinical Practice Guidelines (CPG) can enable economists to include economic information on health care provision. Application of economic evidence in CPGs, and its integration into clinical practice and national decision making is hampered by objections from professions, paucity of economic evidence or lack of policy commitment. The use of state-of-art economic methodologies will improve this. Economic evidence can be graded by 'checklists' to establish the best evidence for decision making given methodological rigor. New economic evaluation checklists, Multi-Criteria Decision Analyses (MCDA) and other decision criteria enable health economists to impact on decision making world-wide. We analyse the methodologies for integrating economic evidence into CPG agencies globally, including the Agency of Health Research and Quality (AHRQ) in the USA, National Health and Medical Research Council (NHMRC) and Australian political reforms. The Guidelines and Economists Network International (GENI) Board members from Australia, UK, Canada and Denmark presented the findings at the conference of the International Health Economists Association (IHEA) and we report conclusions and developments since. The Consolidated Guidelines for the Reporting of Economic Evaluations (CHEERS) 24 item check list can be used by AHRQ, NHMRC, other CPG and health organisations, in conjunction with the Drummond ten-point check list and a questionnaire that scores that checklist for grading studies, when assessing economic evidence. Cost-effectiveness Analysis (CEA) thresholds, opportunity cost and willingness-to-pay (WTP) are crucial issues for decision rules in CEA generally, including end-of-life therapies. Limitations of inter-rater reliability in checklists can be addressed by including more than one assessor to reach a consensus, especially when impacting on treatment decisions. We identify priority areas to generate economic evidence for CPGs by NHMRC, AHRQ, and other agencies. The evidence may cover demand for care issues such as involved time, logistics, innovation price, price sensitivity, substitutes and complements, WTP, absenteeism and presentism. Supply issues may include economies of scale, efficiency changes, and return on investment. Involved equity and efficiency measures may include cost-of-illness, disease burden, quality-of-life, budget impact, cost-effective ratios, net benefits and disparities in access and outcomes. Priority setting remains essential and trade-off decisions between policy criteria can be based on MCDA, both in evidence based clinical medicine and in health planning.

Estimation of Societal Values of Health States Preferences at the National Level for Low- and Middle-Income Countries.

OBJECTIVES: Social preference values of health states are a fundamental input for the preparation of studies in health economics. Several countries have undertaken studies to obtain these values. Our objective was to conduct a structured and systematic literature review of articles that calculates this set of representative values at the national level in low- and middle-income countries (LMICs). METHODS: In this systematic review, we searched the Embase, MEDLINE, Ovid, SciELO, and LILACS databases, among others, for studies published up to June 2022 that estimated nationally representative health states preferences values for LMICs. We summarized the information qualitatively and assessed the risk of bias in each article using the consensus-based standards for selecting health measurement instruments checklist tool. RESULTS: Of the 23 663 articles identified, 35 studies were eligible for inclusion. The studies were from 19 countries in Latin-American, Europe, Africa, and Asia. No studies were found for low-income countries. The most commonly applied generic instrument for measuring health-related quality of life was the 5-level version of EQ-5D and 3-level version of EQ-5D. Preference was given to face-to-face administration of these instruments. The sociodemographic variables with the most significant negative correlation versus utility were older adults, marital status (widowed or divorced), and low educational level and income. CONCLUSIONS: Worldwide, there have been few studies that have estimated, in a nationally representative manner, the social values of health states preferences in LMICs. We consider the local estimate of this set of societal values relevant for any society to improve decision making in allocating resources in health budgets.

Transparency, openness, and reproducible research practices are frequently underused in health economic evaluations.

OBJECTIVES: To investigate the extent to which articles of economic evaluations of healthcare interventions indexed in MEDLINE incorporate research practices that promote transparency, openness, and reproducibility. STUDY DESIGN AND SETTING: We evaluated a random sample of health economic evaluations indexed in MEDLINE during 2019. We included articles written in English reporting an incremental cost-effectiveness ratio in terms of costs per life years gained, quality-adjusted life years, and/or disability-adjusted life years. Reproducible research practices, openness, and transparency in each article were extracted in duplicate. We explored whether reproducible research practices were associated with self-report use of a guideline. RESULTS: We included 200 studies published in 147 journals. Almost half were published as open access articles (n = 93; 47%). Most studies (n = 150; 75%) were model-based economic evaluations. In 109 (55%) studies, authors self-reported use a guideline (e.g., for study conduct or reporting). Few studies (n = 31; 16%) reported working from a protocol. In 112 (56%) studies, authors reported the data needed to recreate the incremental cost-effectiveness ratio for the base case analysis. This percentage was higher in studies using a guideline than studies not using a guideline (72/109 [66%] with guideline vs. 40/91 [44%] without guideline; risk ratio 1.50, 95% confidence interval 1.15-1.97). Only 10 (5%) studies mentioned access to raw data and analytic code for reanalyses. CONCLUSION: Transparency, openness, and reproducible research practices are frequently underused in health economic evaluations. This study provides baseline data to compare future progress in the field.