A Systematic Review of Demand-Side Methods of Estimating the Societal Monetary Value of Health Gain.

OBJECTIVES: Although many reviews of the literature on cost-effectiveness thresholds (CETs) exist, the availability of new studies and the absence of a fully comprehensive analysis warrant a new review. This study systematically reviews demand-side methods for estimating the societal monetary value of health gain. METHODS: Several electronic databases were searched from inception to October 2019. To be included, a study had to be an original article in any language, with a clearly described method for estimating the societal monetary values of health gain and with all estimated values reported. Estimates were converted to US dollars ($), using purchasing power parity (PPP) exchange rates and the gross domestic product (GDP) per capita (2019). RESULTS: We included 53 studies; 45 used direct approach and 8 used indirect approach. Median estimates from the direct approach were PPP$ 24 942 (range 554-1 301 912) per quality-adjusted life-year (QALY), which were typically 0.53 (range 0.02-24.08) GDP per capita. Median estimates using the indirect approach were PPP$ 310 051 (range 36 402-7 574 870) per QALY, which accounted for 7.87 (range 0.68-116.95) GDP per capita. CONCLUSIONS: Our review found that the societal values of health gain or CETs were less than GDP per capita. The great variety in methods and estimates suggests that a more standardized and internationally agreed methodology for estimating CET is warranted. Multiple CETs may have a role when QALYs are not equally valued from a societal perspective (eg, QALYs accruing to people near death compared with equivalent QALYs to others).

Avoiding health technology assessment: a global survey of reasons for not using health technology assessment in decision making.

INTRODUCTION: Despite the documented benefits of using health technology assessments (HTA) to inform resource allocation in health care systems, HTA remains underused, especially in low- and middle-income countries. A survey of global health practitioners was conducted to reveal the top reasons ("excuses") that they had heard from colleagues, policymakers or other stakeholders for not using HTA in their settings. METHODS: There were 193 respondents to the survey. Most responses were from individuals in research organisations (37%), ministries of health (27%) and other government agencies (14%). Participants came from Southeast Asia (40%), the Western Pacific (30%), Africa (15%), Europe (7%), the Americas (7%) and the Eastern Mediterranean region (2%). RESULTS: The top five reasons encountered by respondents related to lack of data, lack of technical skills for HTA, the technocratic nature of the work, the lack of explicit decision rules and the perception that HTA puts a "price on life". CONCLUSIONS: This study aimed to understand and address the top reasons for not using HTA. They fall into three categories: (1) misconceptions about HTA; (2) feasibility issues; and (3) values, attitudes and politics. Previous literature has shown that these reasons can be addressed when identified, and even imperfect HTA analyses can provide useful information to a decision-maker.

Disability and multidimensional quality of life: A capability approach to health status assessment.

This paper offers an approach to assessing quality of life, based on Sen's (1985) theory, which it uses to understand loss in quality of life due to mobility impairment. Specifically, it provides a novel theoretical analysis that is able to account for the possibility that some functionings may increase when a person's capabilities decrease, if substitution effects are large enough. We then develop new data consistent with our theoretical framework that permits comparison of quality of life between those with a disability (mobility impairment) and those without. Empirical results show that mobility impairment has widespread rather than concentrated impacts on capabilities and is associated with high psychological costs. We also find evidence that a small number of functionings are higher for those with a disability, as our theory allows. The paper concludes by discussing possible implications for policy and health assessment methods.

Economic Evaluation for Health Investments En Route to Universal Health Coverage: Cost-Benefit Analysis or Cost-Effectiveness Analysis?

BACKGROUND: It is an unresolved issue as to whether cost-benefit analysis (CBA) or cost-effectiveness analysis (CEA) is the preferable analytical toolkit for use in health technology assessment (HTA). The distinction between the two and an expressed preference for CEA go back at least to 1980 in the USA and, most recently, a Harvard-based group has been reappraising the case for CBA. OBJECTIVES: This article seeks to answer the question: would the use of cost-benefit analysis rather than the more usual cost-effectiveness analysis be an improvement, specifically in appraising health and health-related investments in low and middle-income countries (LMICs) as they transition to Universal Health Coverage?. METHODS/RESULTS: A selective literature review charts the welfare economics (welfarism and extra-welfarism) roots of both approaches. The principal distinguishing feature of the two is the monetary valuation of health outcomes under CBA compared with the use of health constructs such as the Quality-Adjusted Life-Year (QALY) or Disability-Adjusted Life-Year (DALY) under CEA. The former enables direct comparison of the outcomes of health investments with the monetized outcomes of other investments, while the CEA approach facilitates direct comparisons with other health investments. Seven challenges in using CBA in developing countries arise, including ethical issues in outcome valuation, practical challenges in the acquisition of data, intrinsic bias in data on values, and some of the practical issues of implementation for either CBA or CEA. CONCLUSIONS: We conclude with a list of nine issues that both CBA and CEA need to settle if they are to be useful in LMICs. For the immediate future we judge CBA to be the less practicable.

Economic Analysis of Vaccination Programs: An ISPOR Good Practices for Outcomes Research Task Force Report.

This report provides recommendations for budget holders and decision makers in high-, middle, and low-income countries requiring economic analyses of new vaccination programs to allocate scarce resources given budget constraints. ISPOR's Economic Evaluation of Vaccines Designed to Prevent Infectious Disease: Good Practices Task Force wrote guidelines for three analytic methods and solicited comments on them from external reviewers. Cost-effectiveness analyses use decision-analytic models to estimate cumulative changes in resource use, costs, and changes in quality- or disability-adjusted life-years attributable to changes in disease outcomes. Constrained optimization modeling uses a mathematical objective function to be optimized (e.g. disease cases avoided) for a target population for a set of interventions including vaccination programs within established constraints. Fiscal health modeling estimates changes in net present value of government revenues and expenditures attributable to changes in disease outcomes. The task force recommends that those designing economic analyses for new vaccination programs take into account the decision maker's policy objectives and country-specific decision context when estimating: uptake rate in the target population; vaccination program's impact on disease cases in the population over time using a dynamic transmission epidemiologic model; vaccination program implementation and operating costs; and the changes in costs and health outcomes of the target disease(s). The three approaches to economic analysis are complementary and can be used alone or together to estimate a vaccination program's economic value for national, regional, or subregional decision makers in high-, middle-, and low-income countries.

COST, CONTEXT, AND DECISIONS IN HEALTH ECONOMICS AND HEALTH TECHNOLOGY ASSESSMENT.

OBJECTIVES: This study is an attempt to demystify and clarify the idea of cost in health economics and health technology assessment (HTA). METHODS: Its method draws on standard concepts in economics. Cost is a more elusive concept than is commonly thought and can be particularly elusive in multidisciplinary territory like HTA. RESULTS: The article explains that cost is more completely defined as opportunity cost, why cost is necessarily associated with a decision, and that it will always vary according to the context of that decision: whether choice is about inputs or outputs, what the alternatives are, the timing of the consequences of the decision, the nature of the commitment to which a decision maker is committed, who the decision maker is, and the constraints and discretion limiting or liberating the decision maker. Distinctions between short and long runs and between fixed and variable inputs are matters of choice, not technology, and are similarly context-dependent. Harms or negative consequences are, in general, not costs. Whether so-called "clinically unrelated" future costs and benefits should be counted in current decisions again depends on context. CONCLUSIONS: The costs of entire health programs are context-dependent, relating to planned rates of activity, volumes, and timings. The implications for the methods of HTA are different in the contexts of low- and middle-income countries compared with high-income countries, and further differ contextually according to the budget constraints (fixed or variable) facing decision makers.

Priority-setting for achieving universal health coverage.

Governments in low- and middle-income countries are legitimizing the implementation of universal health coverage (UHC), following a United Nation's resolution on UHC in 2012 and its reinforcement in the sustainable development goals set in 2015. UHC will differ in each country depending on country contexts and needs, as well as demand and supply in health care. Therefore, fundamental issues such as objectives, users and cost-effectiveness of UHC have been raised by policy-makers and stakeholders. While priority-setting is done on a daily basis by health authorities - implicitly or explicitly - it has not been made clear how priority-setting for UHC should be conducted. We provide justification for explicit health priority-setting and guidance to countries on how to set priorities for UHC.

Les gouvernements des pays à revenu faible et intermédiaire sont en train de légitimer la mise en place de la couverture sanitaire universelle (CSU), suite à une résolution des Nations Unies de 2012 sur la CSU et à son entérinement dans les objectifs de développement durable fixés en 2015. La CSU variera selon les pays, en fonction de leur contexte et de leurs besoins, ainsi qu'en fonction de la demande et de l'offre de soins. Des questions fondamentales ont ainsi été soulevées par les responsables politiques et les parties prenantes, portant notamment sur les objectifs, les utilisateurs et le rapport coût-efficacité de la CSU. Si les autorités sanitaires déterminent quotidiennement des priorités, de façon implicite ou explicite, la marche à suivre pour définir les priorités en matière de CSU n'a pas été clairement établie. Nous justifions ici la nécessité de définir explicitement les priorités dans le domaine de la santé tout en donnant des orientations aux pays pour définir les priorités en matière de CSU.

Los gobiernos de países con ingresos bajos y medios están legitimando la implementación de una cobertura sanitaria universal (CSU) tras un acuerdo de las Naciones Unidas acerca de la cobertura sanitaria universal en 2012 y su consolidación en los objetivos de desarrollo sostenible establecidos en 2015. Cada país tendrá una cobertura sanitaria universal distinta, según el contexto y las necesidades de cada uno, así como la oferta y la demanda de atención sanitaria. Por tanto, los responsables políticos y partes interesadas han abordado los asuntos fundamentales como los objetivos, los usuarios y la rentabilidad de la cobertura sanitaria universal. A pesar de que las autoridades sanitarias han establecido prioridades diarias (de forma implícita o explícita), no se ha aclarado cómo se debería gestionar el establecimiento de prioridades para la cobertura sanitaria universal. Se ofrece una justificación para el establecimiento de prioridades sanitarias explícitas y orientación a los países en la definición de prioridades para la cobertura sanitaria universal.

The International Decision Support Initiative Reference Case for Economic Evaluation: An Aid to Thought.

BACKGROUND: Policymakers in high-, low-, and middle-income countries alike face challenging choices about resource allocation in health. Economic evaluation can be useful in providing decision makers with the best evidence of the anticipated benefits of new investments, as well as their expected opportunity costs-the benefits forgone of the options not chosen. To guide the decisions of health systems effectively, it is important that the methods of economic evaluation are founded on clear principles, are applied systematically, and are appropriate to the decision problems they seek to inform. METHODS: The Bill and Melinda Gates Foundation, a major funder of economic evaluations of health technologies in low- and middle-income countries (LMICs), commissioned a "reference case" through the International Decision Support Initiative (iDSI) to guide future evaluations, and improve both the consistency and usefulness to decision makers. RESULTS: The iDSI Reference Case draws on previous insights from the World Health Organization, the US Panel on Cost-Effectiveness in Health Care, and the UK National Institute for Health and Care Excellence. Comprising 11 key principles, each accompanied by methodological specifications and reporting standards, the iDSI Reference Case also serves as a means of identifying priorities for methods research, and can be used as a framework for capacity building and technical assistance in LMICs. CONCLUSIONS: The iDSI Reference Case is an aid to thought, not a substitute for it, and should not be followed slavishly without regard to context, culture, or history. This article presents the iDSI Reference Case and discusses the rationale, approach, components, and application in LMICs.

Causes for concern: is NICE failing to uphold its responsibilities to all NHS patients?

Organisations across diverse health care systems making decisions about the funding of new medical technologies face extensive stakeholder and political pressures. As a consequence, there is quite understandable pressure to take account of other attributes of benefit and to fund technologies, even when the opportunity costs are likely exceed the benefits they offer. Recent evidence suggests that NICE technology appraisal is already approving drugs where more health is likely to be lost than gained. Also, NICE recently proposed increasing the upper bound of the cost-effectiveness threshold to reflect other attributes of benefit but without a proper assessment of the type of benefits that are expected to be displaced. It appears that NICE has taken a direction of travel, which means that more harm than good is being, and will continue to be, done, but it is unidentified NHS patients who bear the real opportunity costs.

FROM TALK TO ACTION: POLICY STAKEHOLDERS, APPROPRIATENESS, AND SELECTIVE DISINVESTMENT.

OBJECTIVES: There is widespread commitment--at least in principle--to "evidence-informed" clinical practice and policy development in health care. The intention is that only "appropriate" care ought to be delivered at public expense. Although the rationale for an appropriateness agenda is widely endorsed, and methods have been proposed for addressing it, few published studies exist of contemporary policy initiatives which have actually led to successful disinvestment. Our objective was to explore whether the direct involvement of policy stakeholders could advance appropriateness and disinvestment. METHODS: Several collaborative engagements with policy stakeholders were undertaken to adapt and combine conceptual and empirical material related to appropriateness and disinvestment from the literature to create tools and processes for use in Canada and the province of Ontario in particular. RESULTS: By combining inputs from the literature with colloquial evidence from policy stakeholders, a definition of appropriateness was developed and, importantly, endorsed by all the provincial and territorial ministers of health in Canada. Second, a reassessment framework was successfully implemented for identifying priorities for selective disinvestment. CONCLUSIONS: When scientific evidence was combined with colloquial evidence from policy stakeholders, progress was made on the design and successful implementation of policies for appropriateness and disinvestment.

Systematic review of the impact of health care expenditure on health outcome measures: implications for cost-effectiveness thresholds.

OBJECTIVE: Empirical estimates of the impact of healthcare expenditure on health outcome measures may inform the cost-effectiveness threshold (CET) for guiding funding decisions. This study aims to systematically review studies that estimated this, summarize and compare the estimates by country income level. METHODS: We searched PubMed, Scopus, York Research database, and [anonymized] for Reviews and Dissemination database from inception to 1 August 2023. For inclusion, a study had to be an original article, estimating the impact of healthcare expenditure on health outcome measures at a country level, and presented estimates, in terms of cost per quality-adjusted life year (QALY) or disability-adjusted life year (DALY). RESULTS: We included 18 studies with 385 estimates. The median (range) estimates were PPP$ 11,224 (PPP$ 223 - PPP$ 288,816) per QALY gained and PPP$ 5,963 (PPP$ 71 - PPP$ 165,629) per DALY averted. As ratios of Gross Domestic Product per capita (GDPPC), these estimates were 0.376 (0.041-182.840) and 0.318 (0.004-37.315) times of GDPPC, respectively. CONCLUSIONS: The commonly used CET of GDPPC seems to be too high for all countries, but especially low-to-middle-income countries where the potential health losses from misallocation of the same money are greater. REGISTRATION: The review protocol was published and registered in PROSPERO (CRD42020147276).

Reinforcing Science and Policy, With Suggestions for Future Research Comment on "Evidence-Informed Deliberative Processes for Health Benefit Package Design - Part II: A Practical Guide".

Oortwijn et al continue their guide to good practice in the use of deliberative processes in health technology assessment (HTA) based on a survey of international practice. This is useful, and I applaud their care in maintaining objectivity, especially regarding the treatment of moral and politically controversial issues, in reporting how jurisdictions have handled such matters in designing HTA procedures and in their execution. To their suggestions for future research, I add: the historical development of deliberation in healthcare decision-making and in other fields of public choice, with comparisons of methods, successes and failures; development of guidance on the design and use of deliberative processes that enhance decision-making when there is no consensus amongst the decision-makers; ways of identifying and managing context-free and context-sensitive evidence; and a review of high-level capacity building to raise awareness of HTA and the use of knowledge translation and exchange (KTE) and deliberation amongst policy makers, especially in low and middle-income countries.

Expanding access to high-cost medicines under the Universal Health Coverage scheme in Thailand: review of current practices and recommendations.

BACKGROUND: There has been an increasing demand to reimburse high-cost medicines, through public health insurance schemes in Thailand. METHODS: A mixed method approach was employed. First, a rapid review of select high-income countries was conducted, followed by expert consultations and an in-depth review of three countries: Australia, England and Republic of Korea to understand reimbursement mechanisms of high-cost medicines. In Thailand, current pathways for reimbursing high-cost medicines reviewed, the potential opportunity cost estimated, and stakeholder consultations were conducted to identify context specific considerations. RESULTS: High-income countries reviewed have implemented a variety of pathways and mechanisms for reimbursing high-cost medicines under specific eligibility criteria, listing processes, varying cost-effectiveness thresholds and special funding arrangements. In Thailand, high-cost medicines that do not offer good value-for-money are excluded from the reimbursement process. A framework for reimbursing high-cost medicines that are not cost-effective at the current willingness-to-pay threshold was proposed for Thailand. Under this framework, specific criteria are proposed to determine their eligibility for reimbursement such life-saving nature, treatment of conditions with no alternative treatment options, and affordability. CONCLUSION: High-cost medicines may become eligible for reimbursement through alternative mechanisms based on specific criteria which depend on each context. The application of HTA methods and processes is important in guiding these decisions to support sustainable access to affordable healthcare in pursuit of Universal Health Coverage (UHC).

Nudging and not nudging: the difference doesn't matter.

The characteristics that make a nudge a nudge, and a not-nudge a not-nudge, are too important to be assumed or left to economists and other analysts to determine. They warrant specific appraisal. Likewise, both nudges and not-nudges ought to be required to pass the same tests of effectiveness and acceptability to which all healthcare technologies ought to be subjected and through similar open and participative processes. In this sense, they are not to be differentiated.

Discounting and decision making in the economic evaluation of health-care technologies.

Discounting costs and health benefits in cost-effectiveness analysis has been the subject of recent debate - some authors suggesting a common rate for both and others suggesting a lower rate for health. We show how these views turn on key judgments of fact and value: on whether the social objective is to maximise discounted health outcomes or the present consumption value of health; on whether the budget for health care is fixed; on the expected growth in the cost-effectiveness threshold; and on the expected growth in the consumption value of health. We demonstrate that if the budget for health care is fixed and decisions are based on incremental cost effectiveness ratios (ICERs), discounting costs and health gains at the same rate is correct only if the threshold remains constant. Expecting growth in the consumption value of health does not itself justify differential rates but implies a lower rate for both. However, whether one believes that the objective should be the maximisation of the present value of health or the present consumption value of health, adopting the social time preference rate for consumption as the discount rate for costs and health gains is valid only under strong and implausible assumptions about values and facts.

"Perspectives" in Health Technology Assessment.

This article considers 2 types of standard by which health technology assessment (HTA) studies should be judged: methodological and social. Methodological desiderata specify characteristics of a good quality analysis and should be met regardless of context. Transparency about an HTA study's perspective (eg, specifying whose costs and whose benefits from an intervention should be counted) is one such desideratum. Whether any particular perspective is the right one is, by contrast, contingent upon conditions in which the analysis is to be applied. A perspective ought always to be treated as context sensitive. Recently, it has been advocated that an HTA study's perspective should always be "societal" (ie, including consequences, good or bad, for anyone affected in any way by a technology's use). This article argues that this is a mistake, ethically attractive though it might appear.

Organising Research and Development for evidence-informed health care: some universal characteristics and a case study from the UK.

Research and Development (R&D) in health and health care has several intriguing characteristics which, separately and in combination, have significant implications for the ways in which it is organised, funded and managed. We review the characteristics, some of which apply under most circumstances and others of which may be context-specific, explore their implications for the organisation and management of health-related R&D, and illustrate the main features from the UK experience in the 1990s.