The need for more local technical capacity in Health Technology Assessment (HTA) is a leading challenge to its use in low- and middle-income countries. Zambia has been considering using HTA to support its universal health coverage initiative, which includes health benefits package design and implementation. This study assesses the local HTA capacity for the steering committee tasked with supporting the design and implementation of the national health benefits package in Zambia.

The study applied a cross-sectional web-based survey design and the consensus-based Checklist for Reporting of Survey Studies. Data were collected from the steering committee of the benefits package working group, tasked with leading the design process of the health benefits package using the Instrument for the Assessment of Skills to Conduct a Health Technology Assessment tool.

The majority of respondents had not served on a selection and reimbursement committee. Clinical effectiveness skills in structuring a search strategy, handling missing data, conducting qualitative evidence synthesis, and grading the certainty of evidence were low. Skills for leadership, networking, conflict management, and project coordination, public and patient involvement were mid-level to low. Most of the respondents were aware of ethical issues with health technologies. Health economics skills in economic evaluations and decision analytic modeling, equity and health system efficiency measurement, budget impact analysis, and quality of life were identified for capacity strengthening.

Available technical capacities to revise and implement the national benefits package were lower in health economics, synthesis for clinical effectiveness evidence, ethics, patient and public involvement, and soft skills, in that order.

This study comprehensively reviewed reports on patient and public involvement and engagement (PPIE) in health technology assessment (HTA) overseas and identified the status and possible future measures, of PPIE in Japanese HTA.

The series of reviews targeted systematic reviews (SR#1), references in SR#1 (SR#2), and new articles after SR#1 (SR#3). The MEDLINE and Embase databases were searched through August 2024 using the terms “patient involvement/engagement,” “patient participation,” “community participation,” “public involvement/engagement,” and “health technology assessment.” The implementation details were extracted from information published on the websites of the HTA agencies.

Three review articles in SR#1, 12 references in SR#2, and 10 articles in SR#3 were selected. The opportunities for countries, including Japan, to participate in discussions on the HTA process did not differ significantly; however, information on PPIE in Japan was scarce and did not indicate their purpose and value.

Collected articles indicated that the value of PPIE in HTA includes relevance, equity, fairness, legitimacy, and capacity building. The participation of patient and public representatives in Japanese discussions since 2005 appeared to be very limited to consider PPIE in HTA. In countries that implement PPIE in HTA, the value of PPIE is explicit: the process guidelines are specific and provide an appropriate environment for input that includes education, training, and feedback. Future reforms of the Japanese system will require discussions on PPIE purpose and value, implementation, and creating an environment in which a diverse range of patients and the public can easily express their views.

Surrogate endpoints, used to substitute for and predict final clinical outcomes, are increasingly being used to support submissions to health technology assessment agencies. The increase in the use of surrogate endpoints has been accompanied by literature describing the frameworks and statistical methods to ensure their robust validation. The aim of this review was to assess how surrogate endpoints have recently been used in oncology technology appraisals by the National Institute for Health and Care Excellence (NICE) in England and Wales.

This article identifies technology appraisals in oncology published by NICE between February 2022 and May 2023. Data are extracted on the use and validation of surrogate endpoints including purpose, evidence base, and methods used.

Of the 47 technology appraisals in oncology available for review, 18 (38 percent) utilized surrogate endpoints, with 37 separate surrogate endpoints being discussed. However, the evidence supporting the validity of the surrogate relationship varied significantly across putative surrogate relationships with 11 providing randomized controlled trial evidence, 7 providing evidence from observational studies, 12 based on the clinical opinion, and 7 providing no evidence for the use of surrogate endpoints.

This review supports the assertion that surrogate endpoints are frequently used in oncology technology appraisals in England and Wales and despite the increasing availability of statistical methods and guidance on appropriate validation of surrogate endpoints, this review highlights that use and validation of surrogate endpoints can vary between technology appraisals, which can lead to uncertainty in decision making.

The EUnetHTA Core Model® is well-established in the HTA community. Some recommendations of corresponding guidance documents leave room for alternative methodological choices. Considering the new HTA regulation (HTAR), we aimed to identify needs for concretization (NCs) in EUnetHTA guidance and provide indicative methodological options.

We carried out a qualitative document analysis and structured group discussion. Twenty-two EUnetHTA documents were screened using transparent criteria. Identified NCs were classified into topics according to the PRISMA statement and presented to Austrian HTA practitioners (n = 11) during a structured group discussion. Participants rated NC’s importance. To identify potential solutions, selected key handbooks for generic (Cochrane) and HTA-specific (IQWIG/NICE) evidence synthesis were systematically reviewed and matching content was charted against the NCs.

Thirty-two topics with varying numbers of NCs were identified, twenty-six during the screening process, and six from the group discussion. Most of the topics related to evidence synthesis methods (nine topics), evidence eligibility criteria (nine topics), risk of bias (three topics), and certainty assessment (three topics). Other topics related to information sources, search strategy, data collection process, data items, effect measures, and reporting bias. One or more methodological approaches and recommendations could be identified for each identified topic from the included methodological handbooks.

Our analysis identified a need for concretization in some EUnetHTA guidelines. The structured overview of methodological options may support HTA doers in adapting and applying the guidelines to the national and local practical context.

The aim of this study is to propose and validate a value assessment framework for Health Technology Assessment (HTA) for rare diseases drugs in Brazil.

A scoping review was performed to identify criteria used by HTA agencies in countries with public healthcare systems when evaluating orphan drugs. Based on the findings, a criteria framework for rare disease drugs was proposed for Brazil. Content validity was conducted over three rounds using Delphi technique and content validity ratio (CVR) approach was employed to evaluate the ratings from the eighteen stakeholders (experts and patients).

Twenty-nine HTA criteria for rare disease drugs were identified to compose the Brazilian framework. After three Delphi rounds, the final value framework comprised fifteen criteria categorized into four domains: disease-related factors, treatment-related factors, social and political factors, and economic factors. Among the most well-rated criteria by the CVR, considering the relevance attribute, were “relevance of outcomes for a rare disease,” “impact on patient’s quality of life,” “price negotiation,” and “adjusted cost-effectiveness threshold.” On the other hand, “budget impact threshold,” “innovative nature of treatment,” and “willingness to accept greater uncertainty in clinical evidence” received negative evaluations and were excluded from the final framework.

A value assessment framework validated by key stakeholders of rare diseases in Brazil could contribute to improve HTA transparency, decision making, and efficiency of the healthcare system, and inspire the development of a local guidance for rare-disease HTA.

There are wide variations in the practices of patient involvement in health technology assessment (HTA) in Europe. The field is lacking a consensus on good practices, leading to divergent processes, methods, and evaluation of patient involvement. To identify potential good practice approaches and current gaps, a structured online survey was conducted among HTA stakeholders, including HTA practitioners, patient stakeholders, industry representatives, and others who had experienced patient involvement in HTA.

The questionnaire was co-created by HTA experts, patient stakeholders, and industry representatives and disseminated between 29 April and 14 September 2022.

Responses (n = 168) were submitted from thirty-two European countries by HTA practitioners (n = 33), patient stakeholders (n = 75), industry stakeholders (n = 42), providers (n = 5), academics (n = 7), and others (n = 6). The responses indicated that “allowing access to treatments that have demonstrated value”is the principle rationale for conducting HTA. In terms of the importance of patient involvement, there was consensus across stakeholder groups that “patients have insights and information [that] no other stakeholder has” and that patient involvement is important “to inform HTA which evidence is most patient-relevant”. Shortcomings were identified in the lack of systematic and transparent processes, an unsatisfactory level of information and guidance, and minimal communication and collaboration.

The diverse stakeholders who responded highlighted the need for improving specific aspects of patient involvement practices, including better guidance and information, a more consistent flow of communication between the HTA body and participating patient stakeholders, and the need to develop and implement a consensus on good practices.

This paper reports on the process used to embark on one of the core strategies of Abu Dhabi’s Department of Health, which was to develop a roadmap for HTA implementation and institutionalization, based on the aspirations and needs of local stakeholders and making use of the evidence-informed deliberative processes framework. The paper also highlights the main features of the road map that may be expected to address some of the current challenges.

A series of activities were undertaken that informed the subsequent development of the roadmap. They comprised a situation analysis using a combination of desk research and semistructured (group) interviews with 45 stakeholders. The findings were discussed in two workshops; face-to-face with nonindustry stakeholders from Abu Dhabi, and online with industry representatives.

Guided by the EDP framework, the roadmap provides instructions how to organize stakeholder involvement, how to identify and operationalize decision criteria, and how to ensure that the decision-making process is transparent. Specific guidance is given on establishing an HTA structure with an appropriate policy framework, the formulation of an HTA program, a communication strategy, as well as building and leveraging HTA expertise.

Broad stakeholder consultation has been instrumental toward the establishment of a comprehensive HTA framework in Abu Dhabi, and the development of a road map. The interest raised during stakeholder consultations and the commitments made hold promise for the adoption and establishment of EDP principles to support HTA in Abu Dhabi that have potential to contribute to a sustainable high-quality healthcare system.

Constrained resources under universal health coverage (UHC) necessitate a balance between medication costs and essential health system requirements. Policymakers practice priority-setting, as either implicit or explicit rationing, embedded in evidence-informed decision-making processes to guide funding decisions. Health technology assessment (HTA) is a method that may assist explicit evidence-informed priority setting. South Africa developed an official HTA methods guide in 2022, however before this, commissioning and performing economic evaluations was not standardized.

We conducted a descriptive collective case study to explore the impact of economic analyses on the selection of, and access to, essential medicines in South Africa. Four cases were purposefully selected, and both official information and secondary data, including media reports, were reviewed. Data elements were extracted and organized in a matrix. Cases were reported narratively with a positivist epistemological approach, presenting the authors’ reflections.

We found economic analyses that reflected methodologies described in the HTA guide: international reference pricing, cost-minimization, cost-effectiveness, cost-utility, and budget impact analyses. Economic analyses informing the ‘resource-use’ domain in the GRADE evidence-to-decision framework supported decision-making, influenced market-shaping with price reductions of interventions through benchmarking (fosfomycin, flucytosine), improved equitable access nationally (flucytosine), and prioritized a defined patient group in a justifiable and transparent manner (bortezomib).

A standardized HTA evaluation process guided by a nationally accepted framework is necessary for evidence-informed decision-making. Economic analyses (cost-effectiveness, affordability, and resource use) should be consistently included when making decisions on new interventions.

In the field of Laboratory Medicine, the evolution of knowledge and the innovation of technologies are the basis of analytical and diagnostic progress, leading to the development of new solutions based on innovative technologies. However, these advances must be accompanied by evidence of appropriateness, diagnostic effectiveness, and organizational efficiency, considering the test’s first impact on patient outcomes.

The Health Technology Assessment (HTA) is a valid management tool to support Laboratory Medicine professionals in assessing technologies and which is the most appropriate to adopt. This study is an illustrative case of the application of HTA, exploiting the EUnetHTA Core Model, on two analyzers able to determine the glycated hemoglobin (Hemoglobin A1c, HbA1c), the Capillarys 2 Flex piercing analyzer and the HLC-723G11 analyzer in the Laboratory Medicine Service of the IRCCS San Raffaele Hospital (Milan, IT). The main focus is related to potential differences in methods, organizational aspects, and clinical effectiveness of these approaches for measuring HbA1c.

The EUnetHTA Core Model has proven to be the optimal method for HTA in the field of Laboratory Medicine, as it allows to highlight both the peculiarities of the methods on which the analyzers are based and the clinical efficacy of the laboratory test on specific patient populations, considering individual variations in treatment responses, assessing the potential benefits for individual patients or small groups.

This granular analysis helps provide insights into the effectiveness and value of healthcare interventions at the patient level, contributing to evidence-based decision-making in clinical practice and healthcare policy.

The number of orphan drug (OD) approvals has increased sharply in Europe. In Germany, all ODs are initially subject to a limited assessment after market access. Their added benefit over the standard of care is accepted as established upon EU approval; a regular health technology assessment (HTA) is performed only in certain cases.

We assessed whether the increase in OD approvals has led to improvements in patient-relevant outcomes as supported by the evidence submitted by market authorization holders (MAHs) for HTA in Germany. We also examined the extent to which these ODs address unmet medical needs.

The results of limited assessments and regular HTAs of ODs in Germany (January 2011–September 2021, plus January–December 2023) were analyzed to determine their added benefit based on MAH-submitted dossiers. Added benefit was reported separately for each research question generated from the EU-approved therapeutic indications and any sub-indications (e.g., different subpopulations or control interventions) specified for HTA in Germany.

Eighty-nine ODs (limited assessments: sixty-nine; regular HTAs: twenty) were evaluated in 175 research questions (limited assessments: ninety-seven; regular HTAs: seventy-eight). The added benefit granted in limited assessments was non-quantifiable in nearly eighty percent of the ninety-seven questions. In regular HTAs, no proof of added benefit was shown in fifty-four percent of the seventy-eight questions, mainly due to insufficient comparative data with the standard of care. Established treatments were available for fifty-eight percent of the seventy-eight questions; more than half of which addressed oncology indications (although these account for only eight percent of rare diseases).

Due to evidence gaps in post-approval HTA, many ODs approved in the EU lack proof of added benefit in terms of improving patient-relevant outcomes. Moreover, most approved ODs are indicated for diseases with established treatments and oncology indications, while many unmet medical needs remain. Incentives are required to encourage research in areas of unmet medical need and to generate comparative data with the standard of care.

Artificial intelligence (AI)-based health technologies (AIHTs) have already been applied in clinical practice. However, there is currently no standardized framework for evaluating them based on the principles of health technology assessment (HTA).

A two-round Delphi survey was distributed to a panel of experts to determine the significance of incorporating topics outlined in the EUnetHTA Core Model and twenty additional ones identified through literature reviews. Each panelist assigned scores to each topic. Topics were categorized as critical to include (scores 7–9), important but not critical (scores 4–6), and not important (scores 1–3). A 70 percent cutoff was used to determine high agreement.

Our panel of 46 experts indicated that 48 out of the 65 proposed topics are critical and should be included in an HTA framework for AIHTs. Among the ten most crucial topics, the following emerged: accuracy of the AI model (97.78 percent), patient safety (95.65 percent), benefit–harm balance evaluated from an ethical standpoint (95.56 percent), and bias in data (91.30 percent). Importantly, our findings highlight that the Core Model is insufficient in capturing all relevant topics for AI-based technologies, as 14 out of the additional 20 topics were identified as crucial.

It is imperative to determine the level of agreement on AI-relevant HTA topics to establish a robust assessment framework. This framework will play a foundational role in evaluating AI tools for the early diagnosis of dementia, which is the focus of the European project AI-Mind currently being developed.