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).

We need to talk about values: a proposed framework for the articulation of normative reasoning in health technology assessment.

It is acknowledged that health technology assessment (HTA) is an inherently value-based activity that makes use of normative reasoning alongside empirical evidence. But the language used to conceptualise and articulate HTA's normative aspects is demonstrably unnuanced, imprecise, and inconsistently employed, undermining transparency and preventing proper scrutiny of the rationales on which decisions are based. This paper - developed through a cross-disciplinary collaboration of 24 researchers with expertise in healthcare priority-setting - seeks to address this problem by offering a clear definition of key terms and distinguishing between the types of normative commitment invoked during HTA, thus providing a novel conceptual framework for the articulation of reasoning. Through application to a hypothetical case, it is illustrated how this framework can operate as a practical tool through which HTA practitioners and policymakers can enhance the transparency and coherence of their decision-making, while enabling others to hold them more easily to account. The framework is offered as a starting point for further discussion amongst those with a desire to enhance the legitimacy and fairness of HTA by facilitating practical public reasoning, in which decisions are made on behalf of the public, in public view, through a chain of reasoning that withstands ethical scrutiny.

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.

Fifteen Lessons from Fifteen Years of the Health Intervention and Technology Assessment Program in Thailand.

The Health Intervention and Technology Assessment Program (HITAP) was established in 2007. This article highlights 15 lessons from over 15 years of experience, noting five achievements about what HITAP has done well, five areas that it is currently working on, and five aims for work in the future. HITAP built capacity for HTA and linked research to policy and practice in Thailand. With collaborators from academic and policy spheres, HITAP has mobilized regional and global support, and developed global public goods to enhance the field of HTA. HITAP's semi-autonomous structure has facilitated these changes, though they have not been without their challenges. HITAP aims to continue its work on HTA for public health interventions and disinvestments, effectively engaging with stakeholders and strategically managing its human resources. Moving forward, HITAP will develop and update global public goods on HTA, work on emerging topics such as early HTA, address issues in digital health, real-world evidence and equity, support HTA development globally, particularly in low-income settings, and seek to engage more effectively with the public. HITAP seeks to learn from its experience and invest in the areas identified so that it can grow sustainably. Its journey may be relevant to other countries and institutions that are interested in developing HTA programs.

International Partnerships to Develop Evidence-informed Priority Setting Institutions: Ten Years of Experience from the International Decision Support Initiative (iDSI).

All health systems must set priorities. Evidence-informed priority-setting (EIPS) is a specific form of systematic priority-setting which involves explicit consideration of evidence to determine the healthcare interventions to be provided. The international Decision Support Initiative (iDSI) was established in 2013 as a collaborative platform to catalyze faster progress on EIPS, particularly in low- and middle-income countries. This article summarizes the successes, challenges, and lessons learned from ten years of iDSI partnering with countries to develop EIPS institutions and processes. This is a thematic documentary analysis, structured by iDSI's theory of change, extracting successes, challenges, and lessons from three external evaluations and 19 internal reports to funders. We identified three phases of iDSI's work-inception (2013-15), scale-up (2016-2019), and focus on Africa (2019-2023). iDSI has established a global platform for coordinating EIPS, advanced the field, and supported regional networks in Asia and Africa. It has facilitated progress in securing high-level commitment to EIPS, strengthened EIPS institutions, and developed capacity for health technology assessments. This has resulted in improved decisions on service provision, procurement, and clinical care. Major lessons learned include the importance of sustained political will to develop EIPS; a clear EIPS mandate; inclusive governance structures appropriate to health financing context; politically sensitive and country-led support to EIPS, taking advantage of policy windows for EIPS reforms; regional networks for peer support and long-term sustainability; utilization of context appropriate methods such as adaptive HTA; and crucially, donor-funded global health initiatives supporting and integrating with national EIPS systems, not undermining them.

Redefining Health Technology Assessment: A Comment on "The New Definition of Health Technology Assessment: A Milestone in International Collaboration".

A new definition of health technology assessment (HTA), developed by an International Joint Task Group claims to be a "milestone," "an historic achievement," and "a cornerstone reference"-claims that we think to be unjustified. We too favor clear definitions, especially when confusion abounds. However, the Task Group seems to have developed a definition without the help of usual conventions regarding definitions and, in our view, through an ill-described process. A definition ought to differentiate the entity defined from other entities. This one fails to do so. It states traits that are true of HTA (e.g., that is interdisciplinary) but HTA is not alone in this. There are other concerns: examples of HTA's use are embodied in the definition, precluding other uses; the adjectives used, although generally true of HTA, are not differentiating features; and attributing to HTA specific purposes, thereby excluding other purposes. We have sympathy for these purposes but cannot consider them HTA's only purposes or even, its main purpose. A newcomer to HTA, on reading this definition, will have no idea of HTA's true potential. These numerous failings, we feel, send all the wrong signals, and could ultimately weaken, rather than strengthen perceptions of HTA's legitimacy and objectivity. The production of a good definition remains, therefore, a work in progress.

Designing and Implementing Deliberative Processes for Health Technology Assessment: A Good Practices Report of a Joint HTAi/ISPOR Task Force.

OBJECTIVES: Deliberative processes for health technology assessment (HTA) are intended to facilitate participatory decision making, using discussion and open dialogue between stakeholders. Increasing attention is being given to deliberative processes, but guidance is lacking for those who wish to design or use them. Health Technology Assessment International (HTAi) and ISPOR-The Professional Society for Health Economics and Outcomes Research initiated a joint Task Force to address this gap. METHODS: The joint Task Force consisted of 15 members with different backgrounds, perspectives, and expertise relevant to the field. It developed guidance and a checklist for deliberative processes for HTA. The guidance builds upon the few, existing initiatives in the field, as well as input from the HTA community following an established consultation plan. In addition, the guidance was subject to 2 rounds of peer review. RESULTS: A deliberative process for HTA consists of procedures, activities, and events that support the informed and critical examination of an issue and the weighing of arguments and evidence to guide a subsequent decision. Guidance and an accompanying checklist are provided for (i) developing the governance and structure of an HTA program and (ii) informing how the various stages of an HTA process might be managed using deliberation. CONCLUSIONS: The guidance and the checklist contain a series of questions, grouped by 6 phases of a model deliberative process. They are offered as practical tools for those wishing to establish or improve deliberative processes for HTA that are fit for local contexts. The tools can also be used for independent scrutiny of deliberative processes.

Designing and Implementing Deliberative Processes for Health Technology Assessment: A Good Practices Report of a Joint HTAi/ISPOR Task Force.

OBJECTIVES: Deliberative processes for health technology assessment (HTA) are intended to facilitate participatory decision making, using discussion and open dialogue between stakeholders. Increasing attention is being given to deliberative processes, but guidance is lacking for those who wish to design or use them. Health Technology Assessment International (HTAi) and ISPOR-The Professional Society for Health Economics and Outcomes Research initiated a joint Task Force to address this gap. METHODS: The joint Task Force consisted of fifteen members with different backgrounds, perspectives, and expertise relevant to the field. It developed guidance and a checklist for deliberative processes for HTA. The guidance builds upon the few, existing initiatives in the field, as well as input from the HTA community following an established consultation plan. In addition, the guidance was subject to two rounds of peer review. RESULTS: A deliberative process for HTA consists of procedures, activities, and events that support the informed and critical examination of an issue and the weighing of arguments and evidence to guide a subsequent decision. Guidance and an accompanying checklist are provided for (i) developing the governance and structure of an HTA program and (ii) informing how the various stages of an HTA process might be managed using deliberation. CONCLUSIONS: The guidance and the checklist contain a series of questions, grouped by six phases of a model deliberative process. They are offered as practical tools for those wishing to establish or improve deliberative processes for HTA that are fit for local contexts. The tools can also be used for independent scrutiny of deliberative processes.

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.

Charging for the use of survey instruments on population health: the case of quality-adjusted life years.

A trend towards charging for access to research findings, tools and databases is becoming more prominent globally. But charging for the use of research tools and databases that are vital to research supporting national and international policy development might be unjustified. Financial barriers to accessing these tools and databases disproportionately affect low- and middle-income countries, who may have greater need for information that fuels research in their areas of concern. However, changing this trend is potentially possible. One example is the experience with the EuroQol-five-dimensional questionnaire (EQ-5D), a generic measure of health status used in economic evaluations for resource allocation decisions. Increasingly, governments and health-care providers are using the EQ-5D tool in patient-reported outcome measures to monitor quality of health-care provision, diagnose and track disease progression, and involve patients in their health care. The EuroQol Group, which owns the intellectual property rights to the EQ-5D, recently terminated their policy of charging for noncommercial, nonresearch uses of the tool. We share a brief history of this development and examine these charging policies in the context of the EQ-5D's use in national health-care research and policies, reflecting the trends and developments in the use of survey instruments on population health.

On observe à l'échelle mondiale une tendance de plus en plus marquée à la tarification de l'accès aux résultats, aux outils et aux bases de données de recherche. Cependant, la tarification de l'utilisation d'outils et de bases de données essentiels aux recherches appuyant l'élaboration de politiques nationales et internationales n'est pas toujours justifiée. Les obstacles financiers qui entravent l'accès à ces outils et bases de données affectent de manière disproportionnée les pays à revenu faible et intermédiaire, qui peuvent avoir davantage besoin d'informations pour alimenter des recherches sur leurs sujets de préoccupation. Il est toutefois possible d'infléchir cette tendance. Le questionnaire EuroQol-five-dimensions (EQ-5D), une mesure générale de l'état de santé utilisée dans les évaluations économiques pour les décisions relatives à l'affectation des ressources, constitue un exemple. Les gouvernements et les prestataires de soins de santé utilisent de plus en plus l'outil EQ-5D dans les mesures des résultats notifiés par les patients pour surveiller la qualité de la prestation des soins de santé, diagnostiquer et suivre l'évolution d'une maladie, et associer les patients à leurs soins de santé. Le groupe EuroQol, qui détient les droits de propriété intellectuelle relatifs à l'EQ-5D, a récemment mis fin à sa politique de tarification pour l'utilisation de l'outil à des fins non commerciales et dans un contexte autre que celui de la recherche. Nous retraçons brièvement ce cheminement et examinons ces politiques de tarification dans le contexte de l'utilisation de l'EQ-5D dans les recherches et politiques nationales sur les soins de santé, en rendant compte de l'évolution de l'utilisation du matériel d'enquête sur la santé de la population.

La tendencia a cobrar por el acceso a los resultados de las investigaciones, las herramientas y las bases de datos está cobrando cada vez más importancia en todo el mundo. Sin embargo, puede que no esté justificado cobrar por el uso de herramientas de investigación y bases de datos que son vitales para la investigación en apoyo del desarrollo de políticas nacionales e internacionales. Las barreras financieras para acceder a estas herramientas y bases de datos afectan desproporcionadamente a los países de ingresos bajos y medios, que pueden tener una mayor necesidad de información que impulse la investigación en sus áreas de interés. Sin embargo, es posible cambiar esta tendencia. Un ejemplo es la experiencia con el cuestionario de cinco dimensiones EuroQol (EQ-5D), una medida genérica del estado de salud utilizada en las evaluaciones económicas para las decisiones de asignación de recursos. Cada vez más, los gobiernos y los proveedores de la atención de la salud están utilizando la herramienta EQ-5D en las medidas de resultado informadas por el paciente para supervisar la calidad de la prestación de la atención de la salud, diagnosticar y hacer un seguimiento de la progresión de la enfermedad e involucrar a los pacientes en su cuidado de la salud. El Grupo EuroQol, propietario de los derechos de propiedad intelectual de EQ-5D, puso fin recientemente a su política de cobrar por los usos no comerciales y no relacionados con la investigación de la herramienta. En este artículo se presenta una breve historia de este desarrollo y se examinan estas políticas de cobro en el contexto del uso de EQ-5D en la investigación y las políticas nacionales de atención de la salud, reflejando las tendencias y los avances en el uso de los instrumentos de las encuestas sobre la salud de la población.

What next after GDP-based cost-effectiveness thresholds?

Public payers around the world are increasingly using cost-effectiveness thresholds (CETs) to assess the value-for-money of an intervention and make coverage decisions. However, there is still much confusion about the meaning and uses of the CET, how it should be calculated, and what constitutes an adequate evidence base for its formulation. One widely referenced and used threshold in the last decade has been the 1-3 GDP per capita, which is often attributed to the Commission on Macroeconomics and  WHO guidelines on Choosing Interventions that are Cost Effective (WHO-CHOICE). For many reasons, however, this threshold has been widely criticised; which has led experts across the world, including the WHO, to discourage its use. This has left a vacuum for policy-makers and technical staff at a time when countries are wanting to move towards Universal Health Coverage . This article seeks to address this gap by offering five practical options for decision-makers in low- and middle-income countries that can be used instead of the 1-3 GDP rule, to combine existing evidence with fair decision-rules or develop locally relevant CETs. It builds on existing literature as well as an engagement with a group of experts and decision-makers working in low, middle and high income countries.