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.

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.

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.

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.

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.

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.

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.

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.

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.

The European Network for Health Technology Assessment (EUnetHTA) organizes an annual Forum with stakeholders to receive feedback on its activities, processes, and outputs produced. The fourth edition of the EUnetHTA Forum brought together representatives of HTA bodies, patient organizations, healthcare professionals (HCPs), academia, payers, regulators, and industry. The aim of this paper is to provide an overview of the highlights presented at the 2019 EUnetHTA Forum, reporting the main items and themes discussed in the plenary panel and breakout sessions. The leading topic was the concept of unmet medical need seen from different stakeholders' perspectives. Breakout sessions covered the joint production of assessment reports and engagement with payers, patients, and HCPs. Synergies, pragmatism, and inclusiveness across Member States and stakeholders were emphasized as leading factors to put in place a collaboration that serves the interest of patients and public health in a truly European spirit.

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.