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).

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).

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.

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.

"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.

Use of Evidence-Informed Deliberative Processes - Learning by Doing Comment on "Use of Evidence-informed Deliberative Processes by Health Technology Assessment Agencies Around the Globe".

The article by Oortwijn, Jansen, and Baltussen (OJB) is much more important than it appears because, in the absence of any good general theory of "evidence-informed deliberative processes" (EDP) and limited evidence of how they might be shaped and work in institutionalising health technology assessment (HTA), the best approach seems to be to accumulate the experience of a variety of countries, preferably systematically, from which some general principles might subsequently be inferred. This comment reinforces their arguments and provides a further example.

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.

Expanding HTA - Correcting a Misattribution, Clarifying the Scope of HTA and CEA Comment on "Ethics in HTA: Examining the 'Need for Expansion'".

Abrishami, Oortwijn, and Hofman (AOH) attribute to me a position I do not hold and an argument I did not make. The purpose of this note is make clear what my position actually is and to clarify the main differences between health technology assessment (HTA) and cost-effectiveness analysis (CEA).

How can we make better health decisions a Best Buy for all?: Commentary based on discussions at iDSI roundtable on 2 nd May 2019 London, UK.

The World Health Organization (WHO) resolution calling on Member States to work towards achieving universal health coverage (UHC) has increased the need for prioritizing health spending. Such need will soon accelerate as low- and middle-income countries transition from external aid. Countries will have to make difficult decisions on how best to integrate and finance previously donor-funded technologies and health services into their UHC packages in ways that are equitable, and operationally and financially sustainable. The International Decision Support Initiative (iDSI) is a global network of health, policy and economic expertise which supports countries in making better decisions about how best and how much to spend public money on healthcare. iDSI core partners include Center For Global Development, China National Health Development Research Center, Clinton Health Access Initiative, Health Intervention and Technology Assessment Program, Thailand / National Health Foundation, Imperial College London, Kenya Medical Research Institute, and the Norwegian Institute of Public Health. In May 2019, iDSI convened a roundtable entitled Why strengthening health systems to make better decisions is a Best Buy. The event brought together members of iDSI, development partners and other organizations working in the areas of evidence-informed priority-setting, resource allocation and purchasing. The roundtable participants identified key challenges and activities that could be undertaken by the broader health technology assessment (HTA) community to further country-led capacity building, as well to foster deeper collaboration between the community itself. HTA is a tool which can assist governments and development partners with evaluating alternative investment options in a defensible and accountable fashion. The definition and scope of HTA, and what it can achieve and support, can be presented more clearly and cohesively to stakeholders. Organizations engaging in HTA must develop deeper collaboration, and integrate existing collaborations, to ensure progress in developing HTA institutionalization globally is well organized and sustainable.

Cost-Effectiveness of Docetaxel and Paclitaxel for Adjuvant Treatment of Early Breast Cancer: Adaptation of a Model-Based Economic Evaluation From the United Kingdom to South Africa.

OBJECTIVES: Transferability of economic evaluations to low- and middle-income countries through adaptation of models is important; however, several methodological and practical challenges remain. Given its significant costs and the quality-of-life burden to patients, adjuvant treatment of early breast cancer was identified as a priority intervention by the South African National Department of Health. This study assessed the cost-effectiveness of docetaxel and paclitaxel-containing chemotherapy regimens (taxanes) compared with standard (non-taxane) treatments. METHODS: A cost-utility analysis was undertaken based on a UK 6-health-state Markov model adapted for South Africa using the Mullins checklist. The analysis assumed a 35-year time horizon. The model was populated with clinical effectiveness data (hazard ratios, recurrence rates, and adverse events) using direct comparisons from clinical trials. Resource use patterns and unit costs for estimating cost parameters (drugs, diagnostics, consumables, personnel) were obtained from South Africa. Uncertainty was assessed using probabilistic and deterministic sensitivity analyses. RESULTS: The incremental cost per patient for the docetaxel regimen compared with standard treatment was R6774. The incremental quality-adjusted life years (QALYs) were 0.24, generating an incremental cost-effectiveness ratio of R28430 per QALY. The cost of the paclitaxel regimen compared with standard treatment was estimated as -R578 and -R1512, producing an additional 0.03 and 0.025 QALYs, based on 2 trials. Paclitaxel, therefore, appears to be a dominant intervention. The base case results were robust to all sensitivity analyses. CONCLUSIONS: Based on the adapted model, docetaxel and paclitaxel are predicted to be cost-effective as adjuvant treatment for early breast cancer in South Africa.

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.

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.

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.