Evidence development for medical devices is often focused on satisfying regulatory requirements with the result that health professional and payer expectations may not be met, despite considerable investment in clinical trials. Early engagement with payers and health professionals could allow companies to understand these expectations and reflect them in clinical study design, increasing chances of positive coverage determination and adoption into clinical practice.

An example of early engagement through the EXCITE International model using an early technology review (ETR) is described which includes engagement with payers and health professionals to better inform companies to develop data that meet their expectations. ETR is based on an early evidence review, a framework of expectations that guides the process and identified gaps in evidence. The first fourteen ETRs were reviewed for examples of advice to companies that provided additional information from payers and health professionals that was thought likely to impact on downstream outcomes or strategic direction. Given that limitations were imposed by confidentiality, examples were genericized.

Advice through early engagement can inform evidence development that coincides with expectations of payers and health professionals through a structured, objective, evidence-based approach. This could reduce the risk of business-related adverse outcomes such as failure to secure a positive coverage determination and/or acceptance by expert health professionals.

Early engagement with key stakeholders exemplified by the ETR approach offers an alternative to the current approach of focusing on regulatory expectations. This could reduce the time to reimbursement and clinical adoption and benefit patient outcomes and/or health system efficiencies.

In response to the Omicron surge in early 2022, the HTA Philippines evaluated the acceptability of Filipinos in using self-administered antigen tests (SAAgTs) as part of COVID-19 HTAs in the Philippines.

Scoping review from literature databases was initially conducted to identify preset codes in the use of SAAgT. Preset codes were used to establish the questions for focus group discussions (FGDs). Semi-structured questionnaires were created through Delphi technique. FGDs with four stakeholder groups (i.e., nine healthcare workers [HCWs], seven representatives of at-risk groups, six economic frontliners, and seven representatives of micro–small–medium-sized enterprises) were conducted.

Discomfort in being a target of stigma and being prescribed an “illness identity” when suspected or confirmed COVID-19-positive, along with lack of confidence to perform self-test, caused hesitancy in self-testing among participants. The need for subsidies for test kits from the government or employers was emphasized to increase its accessibility. Having a designated access point and reporting system for SAAgT was highlighted to avoid nepotism (padrino system attributed to debt of gratitude), inequitable distribution, and lapses in reporting. A participatory approach to education was perceived as crucial to reduce any misconceptions associated with the use of SAAgT.

All FGD groups expressed favorable reviews on the implementation of SAAgT because it can potentially reduce the burden of health facility-administered tests. These findings were considered by the HTA Council in the recommendation of SAAgT as part of the overarching national strategies for the diagnosis and screening of COVID-19.

The Health Technology Assessment (HTA) process aims to optimize health system funding of technologies. In recent years there has been an increase in what is known as Real-World Evidence (RWE) as a complement to clinical trials. The objective of Health Technology Assessment International’s Latin American Policy Forum 2022 was to explore the utility of incorporating RWE into HTA and decision-making processes in the region.

This article is based on a background document, survey, and the deliberative work of the country representatives who participated in the Forum.

There is a growing interest in the use of Real-World Data / Real-World Evidence in HTA processes in Latin America, although currently there are no specific local guidelines for RWE use by HTA agencies. At present, its use is limited to certain areas such as adding context to HTA reports, the evaluation of adverse events, or cost estimation.

Potential future uses of RWE were identified, including the creation of risk-sharing agreements, the assessment of technology performance in routine practice, providing information on outcomes that are not so easily evaluated in clinical trials (e.g., the identification of specific subpopulations or quality of life), and the estimation of input parameters for economic evaluations.

The participants agreed that there are several areas presenting significant potential to expand the application of RWD/RWE and that the development of normative frameworks for its use could be helpful.

Limited health budgets and continual advancement of health technologies require mechanisms for prioritization. Israel, with a publicly funded health service basket, has implemented and optimized such a health technology assessment process since 1999.

We describe the process of evaluating technologies according to the Israeli model, analyze its outputs and benefits over two decades of implementation, and compare its key features with international experience.

Retrospective data were collected between 1998 and 2023, including work processes, committee composition, number of applications submitted and approved by a clinical domain, and yearly cost of the basket. Features were evaluated within the evidence-informed deliberative process (EDP) framework.

This national model involves relevant stake holders in a participatory and transparent process, in a timely manner, and is accepted by the public, health professionals, and policy makers, facilitating early adoption of the newest medical technologies. Between 11 and 19 percent of applications are approved for reimbursement annually, mostly pharmaceuticals. On average 26 percent of approved technologies are added to the list without additional budget. Major domains of approved technologies were oncology, cardiology, and neurology.

Israel created a unique model for the expansion of the health service basket. Despite an increasing number of applications and rising costs, the mechanism enables a consensus to be reached on which technologies to fund, while remaining within budget constraints and facilitating immediate implementation. The process, which prioritizes transparency and stake holder involvement, allows just a resource allocation while maximizing the adoption of novel technologies, contributing to an outstanding national level of health despite relatively low health spending.

The aim of the 2022 Health Technology Assessment International (HTAi) Asia Policy Forum (APF) was to discuss experiences and challenges around health technology assessment (HTA) capacity building for both HTA agencies and companies in the Asia region and to identify possible solutions as part of a capacity building roadmap.

Discussions during the 2022 APF, informed by a pre-meeting survey of HTA agencies and industry attendees from the region, form the basis of this paper.

HTA is an essential element of priority-setting in healthcare; however, the scarcity of skilled technical HTA practitioners is a rate-limiting step in the conduct of HTA. The lack of investment in HTA and the political will to mandate the use of HTA in decision-making may be due to a lack of understanding of the value of the HTA process, and how HTA is interpreted and used in the healthcare decision-making process.

Increased demand for HTA is created when the value of HTA is recognized. HTA capacity-building challenges may be mitigated by educating stakeholders, particularly policymakers, on the value of, and the need to invest in, HTA as a transparent process to ensure equitable access to health care for all. Investigating a means of funding and implementing an HTA intern program between agencies, in partnership with industry, to facilitate a supportive environment to foster HTA skills and knowledge, build capacity or strengthen existing capacity should be a priority.

While patient participation in individual health technology assessments (HTAs) has been frequently described in the literature, patient and citizen participation at the organizational level is less described and may be less understood and practiced in HTA bodies. We aimed to better understand its use by describing current practice.

To elicit descriptive case studies and insights we conducted semi-structured interviews and open-ended questionnaires with HTA body staff and patients and citizens participating at the organizational level in Belgium, France, Quebec, Scotland, and Wales.

We identified examples of organizational participation in managerial aspects: governance, defining patient involvement processes, evaluation processes and methods, and capacity building. Mechanisms included consultation, collaboration, and membership of standing (permanent) groups. These were sometimes combined. Participants were usually from umbrella patient organizations and patient associations, as well as individual patients and citizens.

Although the concept, participation at the organizational level, is not well-established, we observed a trend toward growth in each jurisdiction. Some goals were shared for this participation, but HTA bodies focused more on instrumental goals, especially improving participation in HTAs, while patients and citizens were more likely to offer democratic and developmental goals beyond improving participation processes.

Our findings provide rationales for organizational-level participation from the perspectives of HTA bodies and patients. The case studies provide insights into how to involve participants and who may be seen as legitimate participants. These findings may be useful to HTA bodies, the patient sector, and communities when devising an organizational-level participation framework.

Lateral skull base procedures, such as translabyrinthine approach (TLA), are challenging. An autonomous surgical robot might be a solution to these challenges. Our aim is to explore in an early phase the economic consequences of an autonomous surgical robot compared with conventional TLA.

An early decision analytic model was constructed in order to perform a step-wise threshold analyses and a sensitivity analysis to analyze the impact of the several factors on the incremental costs.

Using surgical robot results in incremental costs – EUR 5,562 per procedure – compared to conventional TLA. These costs are most reduced by higher number of procedures, followed by lower price of the robot, saved operation time, and reduced risk of complication, respectively.

The incremental costs of using an autonomous surgical robot can be decreased by choosing applications with a high turnover rate, a long operation time, and a high complication rate.

Though there have been longstanding discussions on the value of ethics in health technology assessment (HTA), less awareness exists on ethics information retrieval methods. This study aimed to scope available evidence and determine current practices for ethics information retrieval in HTA.

Literature searches were conducted in Ovid MEDLINE, LISTA, Scopus, and Google Scholar. Once a list of relevant articles was determined, citation tracking was conducted via Scopus. HTA agency websites were searched for published guidance on ethics searching, and for reports which included ethical analyses. Methods sections of each report were analyzed to determine the databases, subject headings, and keywords used in search strategies. The team also reached out to information specialists for insight into current search practices.

Findings from this study indicate that there is still little published guidance from HTA agencies, few HTAs that contain substantial ethical analysis, and even less information on the methodology for ethics information retrieval. The researchers identified twenty-five relevant HTAs. Ten of these reports did not utilize subject-specific databases outside health sciences. Eight reports published ethics searches, with significant overlap in subject headings and text words.

This scoping study of current practice in HTA ethics information retrieval highlights findings of previous studies—while ethics analysis plays a crucial role in HTA, methods for literature searching remain relatively unclear. These findings provide insight into the current state of ethics searching, and will inform continued work on filter development, database selection, and grey literature searching.

Early access schemes (EASs) are approaches used by payers to balance and facilitate earlier patient access to innovative health technologies while evidence generation is ongoing. Schemes require investment from payers and are associated with significant risk since not all technologies will be routinely reimbursed. The purpose of this study was to gain the perspectives of policy experts about the key challenges for EASs and potential solutions for their optimal design and implementation.

Two virtual workshops were convened including (i) UK-based policy experts (England, Wales, and Scotland) and (ii) representatives from multiple healthcare systems (England, France, Sweden, Canada, Poland, and Norway). Participants were encouraged to share their experiences with EASs in their healthcare system and highlight key challenges for policy makers. Discussions were transcribed and analyzed using framework analysis.

Participants agreed that EASs have value when targeted toward innovative technologies with the potential for significant clinical benefit in an area of high unmet need. Participants discussed potential solutions to the challenges faced by payers implementing EASs, including defining eligibility criteria, supporting evidence generation, and approaches to reimbursement.

Participants agreed that EASs are one possible solution for their healthcare systems and have the potential to deliver significant clinical value to patients. However, widespread adoption of EASs is limited due to concerns about the risks for patients and healthcare budgets, further solutions are needed to deliver EASs for targeted therapies.

To summarize the key methodological challenges identified by health technology assessment (HTA) agencies assessing gene therapy (GT) and consideration of broad elements of value.

Economic evaluations (EEs) of voretigene neparvovec (VN) in RPE65-mediated inherited retinal disease (IRD) published in English were selected. HTA evaluations from Australia, Canada, Ireland, Scotland, England, and the United States were reviewed. An existing methodological framework was used to identify the challenges and considerations.

Eight unique EEs were identified of which six were evaluated by HTA agencies. Incremental cost-effectiveness ratios ranged from $68,951 to $643,813 per quality-adjusted life-years (QALY) gained (healthcare perspective) and dominant to $480,130 per QALY gained (societal perspective). The key challenges were the lack of validated surrogate outcome, utility values and indirect costs from IRD patients, and limited evidence of the long-term treatment effect. Two HTA agencies reviewed a range of novel broader elements of value and whether they were associated with VN while other agencies discussed some elements of broader value. Caregiver disutility was included in some, but not all, evaluations.

The methodological challenges were consistent with innovative interventions for rare diseases and managed using standard methods. Broader value was important to decision-makers but inconsistently applied across agencies. Possible reasons are limitations in the evidence available of the broader benefits that VN offers and how to incorporate these within an EE. A need exists for greater guidance and consistency across jurisdictions regarding the consideration of broader value that considers latest best practice.

To develop best-practice guidance for health technology assessment (HTA) agencies when appraising diagnostic tests for SARS-CoV-2 and treatments for COVID-19.

We used a policy sandbox approach to develop best-practice guidance for HTA agencies to approach known challenges associated with assessing tests and treatments for COVID-19. The guidance was developed by a multi-stakeholder workshop of twenty-one participants representing HTA agencies, clinical and patient experts, academia, industry, and a payer, from across Europe and North America. The workshop was supported by extensive background work to identify the key challenges, including: targeted reviews of existing COVID-related methods guidance for assessing interventions and clinical guidelines, engagement with clinical experts, a survey and workshop of HTA agencies, a systematic review of published economic evaluations, and a workshop of health economic modelers.

We suggest HTA agencies should consider using other types of evidence (e.g., real world) where high-quality randomized controlled trials may be lacking and healthcare systems would value timely HTA outputs. A “living” HTA approach may be useful, given the context of an evolving disease, scientific understanding and evidence base, allowing for decisions to be efficiently revisited in response to new information; particularly, if supported by a common “disease model” for COVID-19. Innovative ways of engaging with the public and clinicians, and early engagement with regulators and payers, are recommended.

HTA agencies should consider the elements of this guidance that are most suited to their existing processes to enable them to assess the effectiveness and value of interventions for COVID-19.

The objective of the present policy analysis was to understand how a disinvestment approach to the process of health technology assessment (HTA), applied to the field of medical devices, might help Italian policymakers to properly spend the resources in healthcare.

Previous international and national experiences in disinvestment for medical devices were reviewed. Precious insights for the rational expenditure of the resources were derived by assessing the evidence available.

The disinvestment of ineffective or inappropriate technologies or interventions with an inadequate value-for-money ratio has become a growing priority for National Health Systems. Different international disinvestment experiences of medical devices were identified and described through a rapid review. Although most of them have a strong theoretical framework, their practical application remains difficult. In Italy, there are no examples of large and complex HTA-based disinvestment practices, but their importance is becoming increasingly acknowledged, especially given the need to prioritize the funds provided by Recovery and Resilience Plan.

Anchoring decisions on health technologies without reassessing the current technological landscape through a robust HTA model might expose to the risk of not ensuring the best employment of the resources available. Thus, it is necessary to develop a strong HTA ecosystem in Italy through adequate consultation with stakeholders to enable a data-driven and evidence-based prioritization of resources toward choices characterized by high value for both patients and society as a whole.

The objective of Health Technology Assessment International’s 6th Latin America Policy Form, held in 2021, was to explore the implementation of deliberative processes in the framework of health technology assessment (HTA) and how agencies in the region could involve stakeholders in this process.

This paper is based on a preparatory survey, a background document, and the deliberative work of participants at the virtual Forum conducted in 2021. There were ninety-one participants in the open session and fifty-two in the closed sessions, representing twelve countries and diverse areas of the health sector.

While there are mechanisms in most countries in Latin America to consider stakeholder involvement to some degree, it remains reduced or limited to a consultative role, making true participative involvement rare. There are significant barriers and structural and contextual limitations that have impeded or slowed progress toward deliberative processes. Relatively low levels of institutionalization and knowledge about HTA, as well as the lack of trust among stakeholders are important challenges. This situation has impacted health systems by diminishing the legitimacy of decisions and the very structures and processes of HTA.

The Forum’s broad group of participants identified barriers, facilitators, and recommendations to improve the use of deliberative processes in Latin America to foster improved fairness and reasonableness in HTA and decision making.

The French health technology assessment (HTA) agency initiated a research between 2018 and 2019 with the aim of determining whether other HTA organizations (agencies, bodies, institutes, and expert networks) and researchers had developed an evaluation framework of organizational impacts (OIs).

Three types of investigation were carried out: (i) an analysis of documents published by selected HTA organizations, (ii) a rapid review on the OI issues, (iii) a questionnaire survey to experts of the International Network of Agencies for Health Technology Assessment.

The analyses highlight six key points: (i) there is no explicit conceptual definition of OIs; (ii) OIs are often not included in a specific dimension of the evaluation or in the same dimensions; (iii) three recurring categories emerge from the assessment of OIs: processes, structure, and culture; (iv) despite its limitations, the European Network for Health Technology Assessment framework (Core Model) is the most mature assessment model to date; (v) the question of the scope of OIs to be considered is unresolved (micro-meso-macro); and (vi) the delineation between OI assessment and economic assessment must be addressed.

Although the issue of considering OI in HTA has been raised for many years, it remains largely unresolved. Defining the concept of OI is a prerequisite for taking the next step toward an evaluation framework. As the question of the impact of innovation goes beyond the health sector, extensive research on how to define and take into account these OIs may be relevant.

The reimbursement process for innovative health technologies in Hungary lacks any formalized assessment of clinical added benefit (CAB). The aim of this research is to present the development, retrospective testing, and implementation of a local assessment framework for determining the CAB of cancer treatments at the Department of Health Technology Assessment of the National Institute of Pharmacy and Nutrition in Hungary.

The assessment framework was drafted after screening existing methods and a retrospective comparison of local reimbursement dossiers to that of German and French methods. The Magnitude of Clinical Benefit Scale of the European Society for Medical Oncology was chosen to rate the extent of CAB in oncology, as part of a conclusion complemented by the assessment of endpoint relevance and the quality of evidence. Several rounds of retrospective assessments have been conducted involving all clinical assessors, iterated with semistructured discussions to consolidate divergence between assessors. External stakeholders were consulted to provide feedback on the framework.

Retrospective assessments resulted in average more than 75 percent concordance between assessors on each element of the conclusion. Input from ten stakeholders was also incorporated; stakeholders were generally supportive, and they mostly commented on the concept, the elements of the framework, and its implementation.

The procedure is suitable for routine use in the decision-making process to describe the CAB of antineoplastic technologies in Hungary. Further extension of the framework is required to cover more disease areas for structured and comparable conclusions on CAB of innovative health technologies.

The 2021 Health Technology Assessment International (HTAi) Asia Policy Forum (APF) aimed to explore the challenges and opportunities presented by the global COVID-19 pandemic for health systems and industry in the Asia region, to discuss how HTA changed during the pandemic, and what its role may be moving forward into a postpandemic era.

Discussions during the virtual 2021 APF, informed by a premeeting survey of HTA agencies and industry attendees from the region describing their experience during the pandemic, form the basis of this paper.

During the pandemic, regulatory approval for COVID-related technologies was fast-tracked with fewer evidentiary requirements, and little or no HTA being conducted before these technologies were implemented in health systems in the region. “Living HTA” incorporating real-world evidence (RWE) as it was generated became part of the HTA landscape. In comparison, non-COVID technologies experienced regulatory approval and HTA delays. A major concern of APF members was future pandemic preparedness, and to ensure that lessons are learned from COVID-19. Governments need to continue to invest in innovation and allow early regulatory approvals with the increased use of RWE. Capacity building was identified as a key investment, including capacity in HTA, modeling, as well as local production of necessary supplies and equipment. Finally, collaboration at all levels of the health system was viewed as vital.

Post-COVID, different forms of HTA must be embraced as part of the new HTA landscape in addition to increased intra- and intercountry collaboration across all sectors of the health system, including regulatory bodies.