To send this article to your account, please select one or more formats and confirm that you agree to abide by our usage policies. If this is the first time you use this feature, you will be asked to authorise Cambridge Core to connect with your account.
Find out more about sending content to .
To send this article to your Kindle, first ensure email@example.com is added to your Approved Personal Document E-mail List under your Personal Document Settings on the Manage Your Content and Devices page of your Amazon account. Then enter the ‘name’ part of your Kindle email address below. Find out more about sending to your Kindle.
Find out more about sending to your Kindle.
Note you can select to send to either the @free.kindle.com or @kindle.com variations. ‘@free.kindle.com’ emails are free but can only be sent to your device when it is connected to wi-fi. ‘@kindle.com’ emails can be delivered even when you are not connected to wi-fi, but note that service fees apply.
As health technology assessment (HTA) seeks to combine complex sets of evidence, values, and perspectives to support open, accountable, and transparent decision making, uncertainty is inherent. Uncertainty is present in the clinical and economic inputs that inform HTA and is a critical factor during context-specific deliberations where the evidence is weighed and decisions are made, taking uncertainty into account through either financial or evidence-generation mechanisms. The presence and impact of uncertainty must also be communicated to all relevant stakeholders during the HTA output stage. This article summarizes the 2021 HTAi Global Policy Forum discussion on “Considering and Communicating Uncertainty in HTA” that debated some of the key challenges and opportunities regarding uncertainty in HTA. Through a combination of small and large group discussions, core themes related to the topic of uncertainty in HTA were identified. These discussions revealed that: utilization of a life cycle/HTA management approach helps manage uncertainty; genuine stakeholder input and engagement (and not just consultation) can clarify uncertainty; tolerance of risk, the relationship of risk to uncertainty, and the context in which uncertainty is considered is critical; transparent and early dialogues could be increased to further reduce the uncertainty during HTA; and communicating uncertainty in HTA outputs is critical. The paper ends with suggested next steps that HTA agencies and stakeholders (such as industry, patients, regulators, payers, and others) might take to move the field forward. The paper promotes further discussion on aspects of uncertainty that should be more openly discussed, debated, and addressed.
Deliberative processes are a well-established part of health technology assessment (HTA) programs in a number of high- and middle-income countries, and serve to combine complex sets of evidence, perspectives, and values to support open, transparent, and accountable decision making. Nevertheless, there is little documentation and research to inform the development of effective and efficient deliberative processes, and to evaluate their quality. This article summarizes the 2020 HTAi Global Policy Forum (GPF) discussion on deliberative processes in HTA.
Through a combination of small and large group discussion and successive rounds of polling, the GPF members reached strong agreement on three core principles for deliberative processes in HTA: transparency, inclusivity, and impartiality. In addition, discussions revealed other important principles, such as respect, reviewability, consistency, and reasonableness, that may supplement the core set. A number of associated supporting actions for each of the principles are also described in order to make each principle realizable in a given HTA setting. The relative importance of the principles and actions are context-sensitive and must be considered in light of the political, legislative, and operational factors that may influence the functioning of any particular HTA environment within which the deliberative process is situated. The paper ends with suggested concrete next steps that HTA agencies, researchers, and stakeholders might take to move the field forward. The proposed principles and actions, and the next steps, provide a springboard for further research and better documentation of important aspects of deliberation that have historically been infrequently studied.
The 2019 Health Technology Assessment International (HTAi) Asia Policy Forum (HAPF) discussed the role of horizon scanning (HS) and health technology reassessment (HTR) in supporting sustainable healthcare in Asia.
Discussions and presentations at the 2019 HAPF, informed by a literature review, results of a premeeting survey, and case studies of HTA agencies from the region form the basis of this paper.
Five of the fourteen HTA agencies surveyed have established or are developing HS systems (HSSs), and six reported some experience with HTR. Although there were many differences in the respective HSSs, all were proactive and identified technologies within a 1–3-year time horizon. Commonalities included the criteria used to prioritize the technologies including burden and rarity of the disease, and cost to the health system and patients. Experience in HTR was not as extensive with most HTR activity being conducted on an ad hoc basis. Challenges for both HS and HTR include a lack of HTA capacity and good quality data, as well as a need for transparency and collaboration with industry.
With developing universal health care and HTA capacity in many countries in Asia, HS and HTR are in their infancy. Whilst several countries in the region are experienced in all facets of HTA, other countries are developing their HTA skill base. As such, there was a clear appetite for setting up a collaborative network in the Asia region to facilitate information sharing around HTA activities, and more specifically, HS and HTR methodologies, experiences, and assessments.
One of the good practice principles for health technology assessment (HTA) is having a clear link between the assessment and decision making. The objective of the 2019 Latin American Policy Forum (LatamPF) of Health Technology Assessment International was to explore different models of connection between HTA and decision making and to discuss the potential applicability of such models in Latin America.
This paper is based on a background document and the deliberations of the members of the LatamPF (fifty-four participants from twelve countries) where a design-thinking methodology was used.
The participants agreed that insufficient links between HTA and decision making undermine the legitimacy of decisions, expose the HTA process to excessive political and judicial influence, and promote the exclusion of some stakeholders from participating in the assessment process and decision making. High priority aspects of the HTA process that could feasibly be improved and which hold the greatest potential to generate positive changes in the health systems in the region were identified. The majority of these aspects were associated with the appropriate institutionalization of HTA, a greater degree of participation by different stakeholders, and improved transparency in the HTA process.
The LatamPF identified barriers and recommended actions to strengthen the link between HTA and decision making. Participants emphasized that there is now a window of opportunity in the region as many societal actors see this as a priority. For this reason, health system stakeholders must take this opportunity to increase efforts toward strengthening the link between HTA and decision making.
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.
Real-world evidence (RWE), derived from real-world data (RWD), is already used, to some extent, for health technology assessment (HTA) purposes. With the increased availability of RWD, there is potential for more widespread use but also challenges ensuring reliable RWE for HTA. Opportunities to overcome key challenges, identified at a scoping meeting, were discussed during the 2019 HTA international (HTAi) Global Policy Forum (GPF).
Reflection of discussions using Design Thinking (an interactive process aimed to solve complex problems) between seventy-three representatives from not-for-profit, for-profit organizations, and HTAi leadership. The discussions were informed by a background paper, and presentations from three invited keynote speakers and eleven GPF members.
Several options were listed for addressing the identified key challenges: quality and acceptability, governance and accountability, transferability, and informing decision making. The GPF emphasized that the HTA community should first understand what questions could be answered with RWE. Additionally, more clarity on methods, standards, streamlining RWD collection, data sharing across jurisdictions, replication of RWD, and expert analysis were mentioned as important priorities.
The HTA community is currently standing at a cross-road as it is not yet fully equipped to address these key challenges. It is, therefore, time for action. The community should start aligning on what is the best source of evidence according to purpose and how the data should be collected to create reliable evidence. It should also initiate the development of actions and guidance to properly develop and manage RWD/RWE to inform decision making across the technology lifecycle.
Discussions at the Health Technology Assessment International (HTAi) Asia Policy Forum (HAPF) aimed to understand the meaning of “high-cost technologies,” and to explore mechanisms to increase access to these technologies in publicly funded health systems in the Asia region.
Discussions and presentations at the 2018 HAPF, informed by a literature review and a premeeting survey of HTA agencies and industry, form the basis of this paper.
Challenges payers in the public health system face when investing in high-cost technologies include a lack of data, especially real-world data, affordability, and the budgetary impact of high-cost technologies. Managed entry schemes (MES) are one means to enable earlier access to high-cost technologies, or at reduced cost to the system. Most countries surveyed had used an MES to introduce a new health technology and most industry representatives had experience with financial-based MES, such as discounts or rebates, with most put in place to increase access to pharmaceuticals. Little experience of outcome-based or evidence-generation MES was reported.
Although it is early days in the implementation of MES in Asia, they have the potential to play an important role enabling access to new, mainly pharmaceutical, health technologies. The development of a “road map” of MES in the region should outline the intent and need for a MES, articulating the “rules of engagement” for all stakeholders—patients, providers, payers, and industry—which will assist countries to clearly identify the problem trying to be solved, and how an MES can be part of the solution.
The recent development of value frameworks to inform healthcare resource allocation responds to a demand to make the decision-making process more inclusive and explicit. The objectives of the 2018 Latin American (LAtam) Health Technology Assessment International (HTAi) Policy Forum were to explore the current international experiences and to discuss the potential application of value frameworks in Latin America.
A background paper, presentations, and group discussions of Policy Forum members (43 participants, 12 LAtam countries represented) at the 2018 HTAi Policy Forum meeting informed this paper.
Participants agreed that HTA and decision making based on more comprehensive and inclusive value frameworks could improve health system effectiveness, efficiency, sustainability, and equity; promote transparency in the decision process; sustain a more comprehensive assessment of technologies; and facilitate stakeholder participation as well as accountability of decisions. Criteria that were identified as essential to be included in a value framework for LAtam were burden of illness and severity of the disease, effectiveness and safety of the technology, quality of the evidence, cost-effectiveness, and budget impact. Potential challenges identified for the application of value frameworks in LAtam, included scarcity of human resources and delays in the assessment process.
Forum participants agreed that the next steps should be to identify appropriate processes and methodologies, adapted to the context of each country, regarding the application of value frameworks to improve the link between HTA and decision making.
The Health Technology Assessment International (HTAi) Asia Policy Forum (HAPF) met to discuss the challenges of achieving universal health care (UHC) in Asia.
Group discussions and presentations at the 2017 HAPF, informed by a background paper, including a literature review and the results of pre-meeting surveys of health technology assessment (HTA) agencies and industry, formed the basis of this article.
Affordability was identified as the greatest barrier to establishing UHC; however, other impediments include the lack of political will to implement UHC, and the cultural issue of deference to expert opinion instead of evidence-based assessments. Although HTA was identified as an important prioritization tool when adding new technologies to benefit packages, it is used inconsistently in the region, resulting in a less transparent decision-making process for stakeholders. Although regional challenges exist around real-world data (RWD), including a lack of capacity to enable information and data sharing, most policy or funding decision makers in the region have access to data. However, there appears to be a disconnect with the experience of industry, whose representatives identify the lack of RWD as their primary issue. To overcome these issues, both HTA agencies and industry agree that collaboration and transparency should be fostered to support the development of robust evidence generation in the region.
There is a willingness for HTA agencies and industry to collaborate to develop HTA methodology for the prioritization of technologies in the Asia region that support healthcare systems to achieve the ultimate outcome of UHC.
The aim of this study was to identify good practice principles for health technology assessment (HTA) that are the most relevant and of highest priority for application in Latin America and to identify potential barriers to their implementation in the region.
HTA good practice principles proposed at the international level were identified and then explored during a deliberative process in a forum of assessors, funders, and product manufacturers.
Forty-two representatives from ten Latin American countries participated. Good practice principles proposed at the international level were considered valid and potentially relevant to Latin America. Five principles were identified as priority and with the greatest potential to be strengthened at this time: transparency in the production of HTA, involvement of relevant stakeholders in the HTA process, mechanisms to appeal decisions, clear priority-setting processes in HTA, and a clear link between HTA and decision making. The main challenge identified was to find a balance between the application of these principles and the available resources in a way that would not detract from the production of reports and adaptation to the needs of decision makers.
The main recommendation was to progress gradually in strengthening HTA and its link to decision making by developing appropriate processes for each country, without trying to impose, in the short-term, standards taken from examples at the international level without adequate adaptation of these to local contexts.
For many years, several health technology assessment (HTA) agencies scanned the horizon to identify health technologies that were safe, effective and offer value for money. However, there is limited evidence regarding its impact. The role of horizon scanning in preparing health systems for the uptake of new and emerging health technologies was discussed during the 2018 HTA International (HTAi) Global Policy Forum Meeting.
Reflection of the discussion between seventy-two senior representatives from for-profit, not-for-profit organizations, and HTAi leadership. It was informed by a background paper, and presentations from four invited experts and seventeen Policy Forum members.
Current horizon scanning systems (HSS) mainly identify health technologies in the late stage of development, aiming to inform topic selection for HTA. Areas for improvement included the need for a clearer definition of the end user(s), purpose, scope, and focus of HSS, the long-term full health system effects, including all relevant stakeholders as early as possible, and considering smart data systems and international collaboration to improve HSS's efficiency. The way in which HSS could be further optimized and better shaped to prepare health systems was also discussed and good practice examples were presented.
HSS have not yet reached their full potential in preparing health systems. To improve the current situation, the HTA community could act as convenors, bringing together all relevant stakeholders and providing the information that decision makers need. This would require a new, more integrative approach to define and use HSS and HTA, and requires new skills.
Latin American countries are taking important steps to expand and strengthen universal health coverage, and health technology assessment (HTA) has an increasingly prominent role in this process. Participation of all relevant stakeholders has become a priority in this effort. Key issues in this area were discussed during the 2017 Latin American Health Technology Assessment International (HTAi) Policy Forum.
The Forum included forty-one participants from Latin American HTA agencies; public, social security, and private insurance sectors; and the pharmaceutical and medical device industry. A background paper and presentations by invited experts and Forum members supported discussions. This study presents a summary of these discussions.
Stakeholder involvement in HTA remains inconsistently implemented in the region and few countries have established formal processes. Participants agreed that stakeholder involvement is key to improve the HTA process, but the form and timing of such improvements must be adapted to local contexts. The legitimization of both HTA and decision-making processes was identified as one of the main reasons to promote stakeholder involvement; but to be successful, the entire system of assessment and decision making must be properly staffed and organized, and certain basic conditions must be met, including transparency in the HTA process and a clear link between HTA and decision making.
Participants suggested a need for establishing clear rules of participation in HTA that would protect HTA producers and decision makers from potentially distorting external influences. Such rules and mechanisms could help foster trust and credibility among stakeholders, supporting actual involvement in HTA processes.
Background: In recent years, there has been a surge in the development of frameworks to assess the value of different types of health technologies to inform healthcare resource allocation. The reasons for, and the potential of, these value frameworks were discussed during the 2017 Health Technology Assessment International (HTAi) Policy Forum Meeting.
Methods: This study reflects the discussion, drawing on presentations from invited experts and Policy Forum members, as well as a background paper.
Results: The reasons given for a proliferation of value frameworks included: rising healthcare costs; more complex health technology; perceived disconnect between price and value in some cases; changes in societal values; the need for inclusion of additional considerations, such as ethical issues; and greater empowerment of clinicians and patients in defining and using value frameworks. Many Policy Forum participants recommended learning from existing frameworks. Furthermore, there was a desire to agree on the core components of value frameworks, defining the additional value elements as necessary and considering how they might be measured and used in practice. Furthermore, adherence to the principles of transparency, predictability, broad stakeholder involvement, and accountability were widely supported, along with being forward looking, explicit, and consistent across decisions.
Conclusions: Value frameworks continue to evolve with significant implications for global incentives for innovation and access to health technologies. There is a role for the HTA community to address some of the key areas discussed during the meeting, such as defining the core components for assessing the value of a health technology.
Objectives: The aim of this study was to explore definitions of value and the use of budget impact and affordability considerations in health technology assessment (HTA) in the Asia region, particularly in relation to high cost technologies.
Methods: Issues were debated by senior representatives from HTA and payer systems in Asian countries, delegates from industry, and invited experts at the 2016 meeting of the HTAi Asia Policy Forum (HAPF). A premeeting survey was used to gather data on how value is assessed and budget impact calculations are used within current processes, as well as current approaches to managing affordability.
Results: All systems consider health benefit to be the key component of value. There is little consensus around “wider” elements of value that should be included. All systems use budget impact in decision making, although meeting attendees noted the challenges in making accurate estimates. The most common strategies used to address affordability concerns to date have been: restricting coverage, for example, to patients who are likely to get the highest value; discounts; and revenue caps. It was noted that these “solutions” may have unintended consequences of creating inequitable access to therapies and failing to provide adequate rewards for innovation.
Conclusions: Decision makers, HTA agencies, and industry need to continue to work together to find mutually agreeable solutions to ensure that patients continue to get equitable access to effective therapies at costs that can be afforded throughout the Asia region.
Objectives: Health technology assessment (HTA) has to innovate to best support changing health system environments and to help provide access to valuable innovation under fiscal constraint.
Methods: Issues associated with changing HTA paradigms were identified through scoping and explored through deliberation at a meeting of industry and HTA leaders.
Results: Five broad areas of change (engagement, scientific dialogue, research prioritization, adaptive approaches, and real world data) were identified. The meeting focused on two themes derived from these: re-thinking scientific dialogue and multi-stakeholder engagement, and re-thinking value, affordability, and access. Earlier and ongoing engagement to steer the innovation process and help achieve appropriate use across the technology lifecycle was perceived as important but would be resource intensive and would require priority setting. Patients need to be involved throughout, and particularly at the early stages. Further discussion is needed on the type of body best suited to convening the dialogue required. There was agreement that HTA must continue to assess value, but views differed on the role that HTA should play in assessing affordability and on appropriate responses to challenges around affordability. Enhanced horizon scanning could play an important role in preparing for significant future investments.
Conclusions: Early and ongoing multi-stakeholder engagement and revisiting approaches to valuing innovation are required. Questions remain as to the most appropriate role for HTA bodies. Changing HTA paradigms extend HTA's traditional remit of being responsive to decision-makers demands to being more proactive and considering whole system value.
Objectives: Health Technology Assessment (HTA) needs to address the challenges posed by high cost, effective technologies, expedited regulatory approaches, and the opportunities provided by collaborative real-world evaluation of technologies. The Health Technology Assessment International (HTAi) Policy Forum met to consider these issues and the implications for evidence production to inform HTA. This paper shares their discussion to stimulate further debate.
Methods: A background paper, presentations, group discussions, and stakeholder role play at the 2015 HTAi Policy Forum meeting informed this paper.
Results: HTA has an important role to play in helping improve evidence production and ensuring that the health service is ready to adopt effective technologies. It needs to move from simply informing health system decisions to also working actively to align stakeholder expectations about realistic evidence requirements. Processes to support dialogue over the health technology life cycle need to be developed that are mindful of limited resources, operate across jurisdictions and learn from past processes. Collaborations between health technology developers and health systems in different countries should be encouraged to develop evidence that will inform decision making. New analytical techniques emerging for real-world data should be harnessed to support modeling for HTA.
Conclusions: A paradigm shift (to “Health Innovation System 2.0”) is suggested where HTA adopts a more central, proactive role to support alignment within and amongst stakeholders over the whole life cycle of the technology. This could help ensure that evidence production is better aligned with patient and health system needs and so is more effective and efficient.
Background: Adaptive approaches to the introduction of drugs and medical devices involve the use of an evolving evidence base rather than conventional single-point-in-time evaluations as a proposed means to promote patient access to innovation, reduce clinical uncertainty, ensure effectiveness, and improve the health technology development process.
Methods: This report summarizes a Health Technology Assessment International (HTAi) Policy Forum discussion, drawing on presentations from invited experts, discussions among attendees about real-world case examples, and background paper.
Results: For adaptive approaches to be understood, accepted, and implemented, the Forum identified several key issues that must be addressed. These include the need to define the goals of and to set priorities for adaptive approaches; to examine evidence collection approaches; to clarify the roles and responsibilities of stakeholders; to understand the implications of adaptive approaches on current legal and ethical standards; to determine costs of such approaches and how they will be met; and to identify differences in applying adaptive approaches to drugs versus medical devices. The Forum also explored the different implications of adaptive approaches for various stakeholders, including patients, regulators, HTA/coverage bodies, health systems, clinicians, and industry.
Conclusions: A key outcome of the meeting was a clearer understanding of the opportunities and challenges adaptive approaches present. Furthermore, the Forum brought to light the critical importance of recognizing and including a full range of stakeholders as contributors to a shared decision-making model implicit in adaptive pathways in future discussions on, and implementation of, adaptive approaches.
Background: Identifying treatments that offer value and value for money is becoming increasingly important, with interest in how health technology assessment (HTA) and decision makers can take appropriate account of what is of value to patients and to society, and in the relationship between innovation and assessments of value.
Methods: This study summarizes points from an Health Technology Assessment International (HTAi) Policy Forum discussion, drawing on presentations, discussions among attendees, and background papers.
Results and Conclusions: Various perspectives on value were considered; most place patient health at the core of value. Wider elements of value comprise other benefits for: patients; caregivers; the health and social care systems; and society. Most decision-making systems seek to take account of similar elements of value, although they are assessed and combined in different ways. Judgment in decisions remains important and cannot be replaced by mathematical approaches. There was discussion of the value of innovation and of the effects of value assessments on innovation. Discussion also included moving toward “progressive health system decision making,” an ongoing process whereby evidence-based decisions on use would be made at various stages in the technology lifecycle. Five actions are identified: (i) development of a general framework for the definition and assessment of value; development by HTA/coverage bodies and regulators of (ii) disease-specific guidance and (iii) further joint scientific advice for industry on demonstrating value; (iv) development of a framework for progressive licensing, usage, and reimbursement; and (v) promoting work to better adapt HTA, coverage, and procurement approaches to medical devices.
Background: Health systems face rising patient expectations and economic pressures; decision makers seek to enhance efficiency to improve access to appropriate care. There is international interest in the role of HTA to support decisions to optimize use of established technologies, particularly in “disinvesting” from low-benefit uses.
Methods: This study summarizes main points from an HTAi Policy Forum meeting on this topic, drawing on presentations, discussions among attendees, and an advance background paper.
Results and Conclusions: Optimization involves assessment or re-assessment of a technology, a decision on optimal use, and decision implementation. This may occur within a routine process to improve safety and quality and create “headroom” for new technologies, or ad hoc in response to financial constraints. The term “disinvestment” is not always helpful in describing these processes. HTA contributes to optimization, but there is scope to increase its role in many systems. Stakeholders may have strong views on access to technology, and stakeholder involvement is essential. Optimization faces challenges including loss aversion and entitlement, stakeholder inertia and entrenchment, heterogeneity in patient outcomes, and the need to demonstrate convincingly absence of benefit. While basic HTA principles remain applicable, methodological developments are needed better to support optimization. These include mechanisms for candidate technology identification and prioritization, enhanced collection and analysis of routine data, and clinician engagement. To maximize value to decision makers, HTA should consider implementation strategies and barriers. Improving optimization processes calls for a coordinated approach, and actions are identified for system leaders, HTA and other health organizations, and industry.
Background: The relationship between regulatory approval on the one hand and health technology assessment (HTA) and coverage on the other is receiving growing attention. Those responsible for regulatory approval, HTA, and coverage have different missions and their information requirements reflect these. There is nonetheless an increasingly popular view that improved communication and coordination between these functions could allow them all to be undertaken effectively with a lower overall burden of evidence requirements, thus speeding patient access to new products and reducing unnecessary barriers to innovation. This study summarizes the main points emerging from a recent discussion of this topic at the HTAi Policy Forum.
Results and Conclusions: After considering the roles of the various bodies, stakeholder perspectives and some current practical initiatives, those present at the Forum meeting discussed possible goals and challenges for improved interactions—in general and at specific stages of the product development life cycle. Opportunities for progress were seen in: continuing the dialogue to promote understanding and interaction between the different bodies and stakeholders; working to align scientific advice for manufacturers on the design and data requirements of pre- and post-marketing evaluation of products (specifically phase 2/3 and phase 4 trials for drugs); and extending the current dialogue to include discussion of product development to address unmet health needs.