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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.
Background: To ensure rapid access to new potentially beneficial health technologies, obtain best value for money, and ensure affordability, healthcare payers are adopting a range of innovative reimbursement approaches that may be called Managed Entry Agreements (MEAs).
Methods: The Health Technology Assessment International (HTAi) Policy Forum sought to identify why MEAs might be used, issues associated with implementation and develop principles for their use. A 2-day deliberative workshop discussed key papers, members' experiences, and collectively addressed four policy questions that resulted in this study.
Results: MEAs are used to give access to new technologies where traditional reimbursement is deemed inappropriate. Three different forms of MEAs have been identified: management of budget impact, management of uncertainty relating to clinical and/or cost-effectiveness, and management of utilization to optimize performance. The rationale for using these approaches and their advantages and disadvantages differ. However, all forms of MEA should take the form of a formal written agreement among stakeholders, clearly identifying the rationale for the agreement, aspects to be assessed, methods of data collection and review, and the criteria for ending the agreement.
Conclusions: MEAs should only be used when HTA identifies issues or concerns about key outcomes and/or costs and/or organizational/budget impacts that are material to a reimbursement decision. They provide patient access and can be useful to manage technology diffusion and optimize use. However, they are administratively complex and may be difficult to negotiate and their effectiveness has yet to be evaluated.
Background: The way in which a health technology is used in any particular health system depends on the decisions and actions of a variety of stakeholders, the local culture, and context. In 2009, the HTAi Policy Forum considered how health technology assessment (HTA) could be improved to optimize the use of technologies (in terms of uptake, change in use, or disinvestment) in such complex systems.
Methods: In scoping, it was agreed to focus on initiatives to implement evidence-based guidance and monitoring activities. A review identified systematic reviews of implementation initiatives and monitoring activities. A two-day deliberative workshop was held to discuss key papers, members’ experiences, and collectively address key questions. This consensus paper was developed by email and finalized at a postworkshop meeting.
Results: Evidence suggests that the impact and use of HTA could be increased by ensuring timely delivery of relevant reports to clearly determined policy receptor (decision-making) points. To achieve this, the breadth of assessment, implementation initiatives such as incentives and targeted, intelligent dissemination of HTA result, needs to be considered. HTA stakeholders undertake a variety of monitoring activities, which could inform optimal use of a technology. However, the quality of these data varies and is often not submitted to an HTA.
Conclusions: Monitoring data should be sufficiently robust so that they can be used in HTA to inform optimal use of technology. Evidence-based implementation initiatives should be developed for HTA, to better inform decision makers at all levels in a health system about the optimal use of technology.
As more countries use HTA to inform decisions on the reimbursement of health technologies, harmonization of evidence requirements between jurisdictions has been proposed, mainly on the grounds of improved efficiency. Harmonization has the potential to avoid duplication of effort for both manufacturers and HTA bodies involved in preparing and reviewing HTA submissions for innovative technologies. However, it also carries risks of loss of local control over decisions, the application of general data standards which are not universally accepted and slowing the rate of development of innovation in the analytical disciplines supporting HTA. This study reviews the issues associated with harmonization taking into account the perspectives of the multiple stakeholders. This study draws on experiences from recent initiatives intended to promote the harmonization of HTA and experience from related fields, particularly regulatory approval of new medical technologies.
The application of conditionality to coverage decisions for healthcare technologies is increasing. Coverage with Evidence Development (CED) represents a specific approach to coverage for promising technologies for which the evidence remains uncertain. CED demands that additional evidence is generated to address the sources of uncertainty and secure ongoing coverage. This study explores the conceptual and policy issues relating to CED and discusses issues involved in operationalizing CED in practice, including presenting criteria for which technologies may be most suitable for CED. This study is intended to further the debate on the use of CED as well as highlight areas that warrant further research.