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In economic evaluation, the healthcare perspective has gradually given way to use of the societal perspective, as this perspective is often advocated for support in making optimal societal decisions. In practice, economic evaluations conducted from the societal perspective ignore, fail to measure and/or fail to monetize many of the costs that fall outside of the healthcare sector. To limit bias and increase decision-supportive power, researchers could strengthen their evaluations by adhering to a few basic principles. Five “pillars for the societal perspective” are proposed. First, who bears the cost and who does not is irrelevant. Second, it is imperative to consider including costs for sectors outside the healthcare sector. Third, both high frequent costs and costs with high unit prices should be considered. Fourth, double counting should be avoided. And fifth, researchers should reflect on choices related to costs, i.e. cost omission and problems with identifying, measuring, and valuing costs.
The International Network of Agencies for Health Technology Assessment (INAHTA) spans the globe as a network of 50 publicly-funded health technology assessment (HTA) agencies supporting health system decision making for 1.4 billion people in thirty countries. Agency members are non-profit HTA organizations that are part of, or directly support, regional or national governments. Recently, INAHTA surveyed its members to gather perspectives from agency leadership on the most important issues in HTA today. This paper describes the top 10 challenges identified by INAHTA members. Addressing these challenges requires a call for action from INAHTA member agencies and the many other actors involved in the HTA ecosystem. In opening this call for action, INAHTA will lead the way; however, a comprehensive undertaking from all players is needed to effectively address these challenges and to continue to evolve HTA in its role as a strong and effective contributor to health systems.
There are several techniques for estimating health state utility values, each of which presents pros and cons in the context of rare diseases (RDs). Direct approaches (e.g. standard gamble and time trade-off) may be too demanding for patients with RDs, since most of them affect young children or cause cognitive impairment. The alternatives are using “vignettes” that describe hypothetical health states for the general public, which may not reflect the heterogeneous manifestations of RDs, or multi-attribute utility instruments (i.e. indirect techniques), such as EQ-5D, which may be less sensitive in capturing the specificities of RDs. The “rule of rescue” approach is a promising alternative in RDs, since it prioritizes identifiable patients with life-threatening or disabling conditions. However, it raises measurement challenges and ethical issues. Furthermore, the literature reports on relevant implications of choosing a technique over others for health technology assessment, which should be considered in relation to individual RDs.
Health technology assessment (HTA) conducted to inform developers of health technologies (development-focused HTA, DF-HTA) has a number of distinct features when compared to HTA conducted to inform usage decisions (use-focused HTA). To conduct effective DF-HTA, it is important that analysts are aware of its distinct features as analyses are often not published. We set out a framework of ten features, drawn from the literature and our own experience: a target audience of developers and investors; an underlying user objective to maximize return on investment; a broad range of decisions to inform; wide decision space; reduced evidence available; earlier timing of analysis; fluid business model; constrained resources for analysis; a positive stance of analysis; and a “consumer”-specific burden of proof. This paper presents a framework of ten features of DF-HTA intended to initiate debate as well as provide an introduction for analysts unfamiliar with the field.
There is no consensus on who might be qualified to conduct ethical analysis in the field of health technology assessment (HTA). Is there a specific expertise or skill set for doing this work? The aim of this article is to (i) clarify the concept of ethics expertise and, based on this, (ii) describe and specify the characteristics of ethics expertise in HTA.
Based on the current literature and experiences in conducting ethical analysis in HTA, a group of members of the Health Technology Assessment International (HTAi) Interest Group on Ethical Issues in HTA critically analyzed the collected information during two face-to-face workshops. On the basis of the analysis, working definitions of “ethics expertise” and “core competencies” of ethics experts in HTA were developed. This paper reports the output of the workshop and subsequent revisions and discussions online among the authors.
Expertise in a domain consists of both explicit and tacit knowledge and is acquired by formal training and social learning. There is a ubiquitous ethical expertise shared by most people in society; nevertheless, some people acquire specialist ethical expertise. To become an ethics expert in the field of HTA, one needs to acquire general knowledge about ethical issues as well as specific knowledge of the ethical domain in HTA. The core competencies of ethics experts in HTA consist of three fundamental elements: knowledge, skills, and attitudes.
The competencies described here can be used by HTA agencies and others involved in HTA to call attention to and strengthen ethical analysis in HTA.
Innovative health technologies, like telemedicine, offer advantageous telediagnostic apps that can improve the health care of populations in remote regions. However, evidence on how these developments can enhance universal coverage for electrocardiographic (ECG) diagnosis to support a cardiovascular disease prevention program is limited. The utility of telemedicine for attaining universal coverage for ECG diagnosis according to the national cardiovascular disease prevention program in Paraguay was investigated.
This cross-sectional survey included adults (aged 19 to 80 years) and children (aged 1 to 18 years) with a medical prescription. The study was carried out by the Telemedicine Unit to evaluate the utility of a telemedicine net for a countrywide detection and prevention program for cardiovascular disease. The results obtained by the tele-ECG net, which was implemented in sixty public hospitals countrywide, were analyzed and used to verify adherence to the cardiovascular prevention program.
Between 2014 and 2019, 331,418 remote ECG diagnoses were performed. Of these, eighty-two percent (n = 270,539) were in adults and eighteen percent (n = 60,879) were in children. Among the adult diagnoses, the majority (52%) were pathological and included sinus bradycardia (13%), right bundle branch block (6%), left ventricular hypertrophy (5%), and ventricle repolarization disorder (5%). Among the children, only twenty percent of diagnoses pathological and included sinus bradycardia (11%) and sinus tachycardia (4%). The mean rate of adherence to the prevention program was 38.2 per 1,000 diagnoses performed.
The results showed that telemedicine can significantly enhance coverage for universal ECG diagnosis to support cardiovascular disease prevention and health programs. However, before carrying out the systematic implementation of such a program contextualization using the regional epidemiological profile must be performed.
An international joint task group co-led by the International Network of Agencies for Health Technology Assessment (INAHTA) and Health Technology Assessment International (HTAi) has developed a new and internationally accepted definition of HTA.
The task group, consisting of representatives of leading HTA networks, societies and global organizations, developed guiding principles for the process and followed an established consultation plan with the broader HTA community to develop the definition.
The consensus achieved by the international joint task group brings the collective weight of the participating networks, societies, and organizations behind the new definition.
The new definition of HTA is an historic achievement and it is offered to the current and emerging HTA world as a cornerstone reference for today and into the future.
A key working session, held as part of the Health Technology Assessment international (HTAi) Global Policy Forum meeting asks members to share “What's Keeping Me Up At Night.” Members—senior thought leaders from health technology assessment (HTA) agencies, payer organizations, industry, and the HTAi Board—share without fear or favor the thorny issues related to HTA that are challenging them now or likely to do so in the near future. This article contains a reflection on the discussions at this session over the last 2 years and focuses on the recurrent and repeated themes: internal and external stakeholder involvement in HTA processes; globalization of HTA and the future of HTA (namely innovative technologies, tide of data and the “war for talent”). While the aim of these informal sessions is not to produce solutions, it reinforces the importance of developing a truly multi-stakeholder HTA community with working relationships built on mutual trust and long-standing engagement.
Pragmatic or practice-oriented comparative effectiveness trials may be conducted to fill the evidence gaps that are revealed after the private sector has performed the trials needed for bringing their product to the market. A tool of increasing importance to identify such evidence gaps is resulting from health technology assessments (HTA) whereby the data derived from clinical research are examined in a systematic manner with reference to effect, safety, as well as additional parameters. Practice-oriented trials are informative for healthcare decision makers, practice-changing and may even be cost-saving for the healthcare payers. There are however only a limited number of funding sources for such trials. Public and private healthcare payers should stimulate the conduct of practice-oriented trials in their effort to maximize patient benefit within the limitation of the available resources. Pragmatic randomized trials can be performed at low cost when based on existing coded electronic health records and as well health registries. Public health decision makers are increasingly taking advantage of results from health technology assessments to support priority setting. In accordance with this it would appear reasonable that decision makers should get more involved in priority setting and funding also in the field of clinical research in order to provide further evidence needed for assessments, reassessments, and subsequent qualified decisions and resource allocations in health care. A closer dialogue and collaboration between the clinical research and HTA communities would facilitate a more efficient utilization of such opportunities.
When setting priorities for health, there is broad agreement that a range of social values and ethical principles beyond clinical and cost-effectiveness matter, but exactly how health technology assessment (HTA) should account for a broader set of criteria remains an area of ongoing debate. In light of this, we welcome a recent review paper by Baltussen et al. evaluating the potential of different multi-criteria decision analysis (MCDA) approaches to enable HTA agencies to incorporate a broader set of values in their appraisals. The authors describe three approaches to MCDA—qualitative MCDA, quantitative MCDA, and MCDA with decision rules—laying out their relative advantages and disadvantages and providing recommendations for how they can best be implemented. While we endorse many of the authors' assessments and conclusions, including the critical role of deliberation in any MCDA approach and the undertaking of qualitative MCDA at a minimum, we take a stronger position regarding the flaws of quantitative MCDA and strongly caution against it. We find quantitative MCDA antithetical to at least two of the ways MCDA is intended to improve HTA recommendations: (i) enhancing quality and (ii) promoting transparency. Quantitative MCDA may mask the complex tradeoffs that exist within and between decision criteria and remain generally inaccessible to those who are not well-versed in its technical methods of appraisal. We advocate for a predominantly qualitative approach to MCDA appraisal centered around deliberation and supplemented with decision aids to help account for health opportunity costs.
There is growing interest globally in using real-world data (RWD) and real-world evidence (RWE) for health technology assessment (HTA). Optimal collection, analysis, and use of RWD/RWE to inform HTA requires a conceptual framework to standardize processes and ensure consistency. However, such framework is currently lacking in Asia, a region that is likely to benefit from RWD/RWE for at least two reasons. First, there is often limited Asian representation in clinical trials unless specifically conducted in Asian populations, and RWD may help to fill the evidence gap. Second, in a few Asian health systems, reimbursement decisions are not made at market entry; thus, allowing RWD/RWE to be collected to give more certainty about the effectiveness of technologies in the local setting and inform their appropriate use. Furthermore, an alignment of RWD/RWE policies across Asia would equip decision makers with context-relevant evidence, and improve timely patient access to new technologies. Using data collected from eleven health systems in Asia, this paper provides a review of the current landscape of RWD/RWE in Asia to inform HTA and explores a way forward to align policies within the region. This paper concludes with a proposal to establish an international collaboration among academics and HTA agencies in the region: the REAL World Data In ASia for HEalth Technology Assessment in Reimbursement (REALISE) working group, which seeks to develop a non-binding guidance document on the use of RWD/RWE to inform HTA for decision making in Asia.
As COVID-19 ravages the world, many countries are faced with the grim reality of not having enough critical-care resources to go around. Knowing what could be in store, the Thai Ministry of Public Health called for the creation of an explicit protocol to determine how these resources are to be rationed in the situation of demand exceeding supply. This paper shares the experience of developing triage criteria and a mechanism for prioritizing intensive care unit resources in a middle-income country with the potential to be applied to other low- and middle-income countries (LMICs) faced with a similar (if not more of a) challenge when responding to the global pandemic. To the best of our knowledge, this locally developed guideline would be among the first of its kind from an LMIC setting. In summary, the experience from the Thai protocol development highlights three important lessons. First, stakeholder consultation and public engagement are crucial steps to ensure the protocol reflects the priorities of society and to maintain public trust in the health system. Second, all bodies and actions proposed in the protocol must not conflict with existing laws to ensure smooth implementation and adherence by professionals. Last, all components of the protocol must be compatible with the local context including medical culture, physician–patient relationship, and religious and societal norms.
Health technology assessment (HTA) is a cost-effective resource allocation tool in healthcare decision-making processes; however, its use is limited in low-income settings where countries fall short on both absorptive and technical capacity. This paper describes the journey of the introduction of HTA into decision-making processes through a case study revising the National Essential Medicines List (NEMLIT) in Tanzania. It draws lessons on establishing and strengthening transparent priority-setting processes, particularly in sub-Saharan Africa.
The concept of HTA was introduced in Tanzania through revision of the NEMLIT by identifying a process for using HTA criteria and evidence-informed decision making. Training was given on using economic evidence for decision making, which was then put into practice for medicine selection for the NEMLIT. During the revision process, capacity-building workshops were held with reinforcing messages on HTA.
Between the period 2014 and 2018, HTA was introduced in Tanzania with a formal HTA committee being established and inaugurated followed by the successful completion and adoption of HTA into the NEMLIT revision process by the end of 2017. Consequently, the country is in the process of institutionalizing HTA for decision making and priority setting.
While the introduction of HTA process is country-specific, key lessons emerge that can provide an example to stakeholders in other low- and middle-income countries (LMICs) wishing to introduce priority-setting processes into health decision making.
Due to the nature of Alzheimer's disease (AD), health technology assessment (HTA) agencies might face considerable challenges in choosing appropriate outcomes and outcome measures for drugs that treat the condition. This study sought to understand which outcomes informed previous HTAs, to explore possible reasons for prioritizations, and derive potential implications for future assessments of AD drugs.
We conducted a literature review of studies that analyzed decisions made in HTAs (across disease areas) in three European countries: England, Germany, and The Netherlands. We then conducted case studies of technology assessments conducted for AD drugs in these countries.
Overall, outcomes measured using clinical scales dominated decisions or recommendations about whether to fund AD drugs, or price negotiations. HTA processes did not always allow the inclusion of outcomes relevant to people with AD, their carers, and families. Processes did not include early discussion and agreement on what would constitute appropriate outcome measures and cut-off points for effects.
We conclude that in order to ensure that future AD drugs are valued appropriately and timely, early agreement with various stakeholders about outcomes, outcome measures, and cut-offs is important.
Ghana is one of the few African countries to enact legislation and earmark significant funding to establish universal health coverage (UHC) through the National Health Insurance Scheme, although donor funds have declined recently. Given a disproportionate level of spending on medicines, health technology assessment (HTA) can support resource allocation decisions in the face of highly constrained budgets, as commonly found in low-resource settings. The Ghanaian Ministry of Health, supported by the International Decision Support Initiative (iDSI), initiated a HTA study in 2016 to examine the cost-effectiveness of antihypertensive medicines. We aimed to summarize key insights from this work that highlights success factors beyond producing purely technical outputs. These include the need for capacity building, academic collaboration, and ongoing partnerships with a broad range of experts and stakeholders. By building on this HTA study, and with ongoing interactions with iDSI, HTAi, WHO, and others, Ghana will be well positioned to institutionalize HTA in resource allocation decisions and support progress toward UHC.
India has introduced health technology assessment (HTA) as a tool for improving the allocation of health resources. The core mandate of HTA in India (HTAIn) is to undertake critical appraisal of available technologies, identify cost-effective interventions, and help the government pursue evidence-informed decisions regarding public health expenditures. We conducted a systematic review to assess economic evaluation studies published in the last four years from India.
Economic evaluations published from September 2015 to September 2019 were identified by searching various databases, including PubMed, Scopus, Embase, The Cochrane Library, and CINAHL according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guideline. Cost-effectiveness studies and HTAs reported or conducted in India were included. Two independent reviewers performed the final selection of studies by assessing the full-text articles and conducted the data extraction. Differences of opinions were resolved through discussion and mutual consensus.
After screening 2,837 articles, seventy met the inclusion criteria and were selected. The articles predominantly used secondary data (70%) to evaluate the cost effectiveness of an innovation. Among the technologies assessed, fifty-seven percent were curative in nature and most commonly addressed infectious diseases (27%), closely followed by non-communicable diseases, and maternal and child health. Principally, the cost effectiveness of a technology was expressed in terms of disability-adjusted or quality-adjusted life-years. Only two studies reported negative findings.
HTA can play a pivotal role in equipping policy makers and public health payers to make appropriate decisions for healthcare budget allocations when mapped with the true disease burden of the population. It is important to highlight negative results and to create a national repository of HTA studies to facilitate faster adoption of best practices in India.
Early health technology assessment (HTA), which includes all methods used to inform industry and other stakeholders about the potential value of new medical products in development, including methods to quantify and manage uncertainty, has seen many applications in recent years. However, it is still unclear how such early value assessments can be integrated into the technology innovation process. This commentary contributes to the discussion on the purposes early HTA can serve. Similarities and differences in the perspectives of five stakeholders (i.e., the hospital, the patient, the assessor, the medical device industry, and the policy maker) on the purpose, value, and potential challenges of early HTA are described. All five stakeholders agreed that integrating early HTA in the innovation process has the possibility to shape and refine an innovation, and inform research and development decisions. The early assessment, using a variety of methodologies, can provide insights that are relevant for all stakeholders but several challenges, for example, feasibility and responsibility, need to be addressed before early HTA can become standard practice. For early evaluations to be successful, all relevant stakeholders including patients need to be involved. Also, nimble, flexible assessment methods are needed that fit the dynamics of medical technology. Best practices should be shared to optimize both the innovation process and the methods to perform an early value assessment.