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.

The roles and potential value of patient preference (PP) data in health technology assessment (HTA) remain to be fully realized despite an expanding literature and various efforts to establish their utility. This article reports lessons learned through a series of collaborative workshops with HTA representatives, organized by the Health Technology Assessment International’s Patient Preferences Project Subcommittee.

Five online workshops were conducted between June 2022 and June 2023, seeking to facilitate collaborative learning and reflection on ways that PP data can be integrated into HTA. Participants included nine HTA representatives from the United States, Canada, Australia, England, and the Netherlands. Workshops were recorded, transcribed, and thematically analyzed.

Despite appreciating the value of PP data, participants were ambivalent about their use in HTA. Some felt that they were already getting the information they needed from the cost-effectiveness analysis or existing patient involvement processes. Others thought that PP data would be very helpful at the initial and final stage of the decision-making process and, particularly, in the following cases: (a) when technology has important non-health benefits; (b) when the clinical and/or cost-effectiveness evidence is marginal; and (c) when treatment is indicated for a large and heterogeneous population. Issues related to the validity and reliability of PP studies were frequently raised, with preference heterogeneity at the core of these concerns.

Collaborating with HTA representatives in the “co-creation” of PP research can help address their concerns and facilitate mutual learning about how PP data can be used in HTA.

International development agendas increasingly push for access to healthcare for all through universal healthcare coverage. Health economic evaluations and health technology assessment (HTA) could provide evidence to support this but do not routinely incorporate consideration of equitable access.

We undertook an international scoping review of health economic evaluation and HTA guidelines to examine how well issues of healthcare access and equity are represented, evidence recommendations, and gaps in current guidance to support evidence generation in this area. Guidelines were sourced from guideline repositories and websites of international agencies and organizations providing best practice methods guidance. Articles providing methods guidance for the conduct of HTA, or health economic evaluation, were included, except where they were not available in English and a suitable translation could not be obtained.

The search yielded forty-seven national, four international, and nine independent guidelines, along with eighty-six articles providing specific methods guidance. The inclusion of equity and access considerations in current guidance is extremely limited. Where they do feature, detail on specific methods for providing evidence on these issues is sparse.

Economic evaluation could be a valuable tool to provide evidence for the best healthcare strategies that not only maximize health but also ensure equitable access to care for all. Such evidence would be invaluable in supporting progress towards universal healthcare coverage. Clear guidance is required to ensure evaluations provide evidence on the best strategies to support equitable access to healthcare, but such guidance rarely exists in current best practice and guidance documents.

Health technology assessment (HTA) organizations generate guidelines to inform healthcare practices toward improved health outcomes. This review sought to identify and classify outcomes of guidelines from HTA organizations within published research.

We performed a systematic mixed studies review of empirical studies that (a) referred to a published guideline from an HTA organization and (b) reported an outcome resulting from a guideline. We searched the published literature in English or French within seven databases. Outcome types were classified within five dimensions of an existing framework for online health information (e.g., relevance, cognitive/affective impact, and use). Subdimensions were inductively developed. A two-phase sequential data synthesis was performed. Phase 1: a hybrid deductive–inductive thematic analysis identified the types of outcomes and displayed their relationships on a concept map. Phase 2: descriptive statistics were tabulated by the type of outcome.

A total of 6,719 records were retrieved through searches on 6 February 2023. After screening, we included 120 observational studies (twenty-one qualitative, ninety-four quantitative, and five mixed methods). Phase 1 identified twenty-nine types of outcomes. The most frequently reported outcomes were within the organizational dimension (reported in ninety-four studies). The most common subdimensions were “Referrals” (thirty-eight occurrences), the “Quality of Prescriptions” (fifteen occurrences), and the “Quality of Diagnosis” (eight occurrences). For Phase 2, we could only generate descriptive statistics on seventeen outcomes. These were almost equally distributed among positive, neutral, and negative effects. Our results contribute to knowledge about the outcomes of HTA guidelines and options for documenting and measuring them in future evaluations.

Health Technology Assessment (HTA) practitioners recognize the significance of qualitative methodologies that focus on how a technology is feasible, meaningfulness, acceptable, and equitable. This mapping aimed to delineate the frameworks employed to synthesize qualitative evidence and assess the quality of synthesis in HTA .

Mapping was conducted using Medline, LILACS, CINAHL, Embase, Web of Science, Scopus, PsycINFO, Cochrane Library, JBI, and ScienceDirect databases. Gray literature searches included PROQUEST, Open Grey, Canadian Agency for Drugs and Technologies in Health’s Grey Matters, Google Scholar, and HTA agency websites. The inclusion criteria were centered on global qualitative evidence synthesis frameworks. The data are presented in the tables.

Of the 2054 articles, 31 were included, mostly from Europe. Guide was the type of document more cited, and most authors are from HTA agencies and universities. Incorporating both patient and family perspectives is the most cited reason for include qualitative evidence. Regardless of the framework or tool, SPICE was the main acronym, and RETREAT was preferred for approach selection. Thematic synthesis dominated analytic methods, and CASP was the primary quality appraisal tool. GRADE-CERQual graded evidence synthesis, with ENTREQ as the top reporting guidance. The GRADE evidence-to-decision framework was mentioned for recommendations.

This mapping highlights the movement incorporate qualitative evidence in HTA employing specific frameworks. Despite the similarities among documents, most of them describe part of the process to synthesize qualitative evidence. Standardizing procedures to incorporate qualitative evidence into HTA can enhance decision-making. These findings offer essential considerations for HTA practice.

Evaluating the impact of patient involvement in health technology assessments (HTA) may help improve practices and avoid ineffective activities. Evaluation, however, continues to be infrequent, inconsistent, and often only relates to process quantity or quality. The Patient and Citizen Involvement in HTA Interest Group (PCIG) within Health Technology Assessment International set out to contextualize this impact to support evaluation.

Given the lack of established methodology to measure impact, the team performed a qualitative analysis of first-hand accounts about perceived changes in HTA due to involvement of patient stakeholders. A questionnaire was developed, piloted, and rolled out to collect personal perspectives from stakeholders with relevant experience. The stories were analyzed in the aggregate to identify themes in the data.

From January 2019 to September 2021, twenty-four responses were collected through PCIG’s network. Responses (including one joint industry-HTA body submission) came from patient representatives (12), HTA bodies (11), and industry representatives (2) from North America (5), South America (3), Europe (13), and Asia Pacific (3). Based on themes commonly reported, a three-domain framework for evaluating impact is proposed: impact on basis of HTA result or recommendation, impact on HTA body, and impact on patient participants. The framework includes components under each domain to support reporting.

Using the Three-Domain Impact Framework may be useful in identifying, evaluating, and communicating the value of patient involvement in HTA. Enhancing and increasing reporting practices may improve transparency and facilitate process improvements for meaningful integration of patient stakeholders into HTA appraisals across jurisdictions.

A conceptual framework, called Innovation of Health Technology Assessment Methods (IHTAM), has been developed to facilitate the understanding of how to innovate methods of health technology assessment (HTA). However, the framework applicability has not been evaluated in practice. Hence, we aimed to explore framework applicability in three cases of method innovation that are part of the HTx project and to develop a roadmap to improve framework applicability.

The IHTAM framework was applied to three cases of innovating HTA methods. We collected feedback from case study leaders and consortium members after a training session, an approximately 1-year follow-up of periodic case study meetings, and a general assembly meeting where innovation progresses of the three cases were reported through surveys and interviews. Feedback was then summarized using an open-coding technique.

According to feedback, the framework provided a structured way of deliberation and helped to improve collaboration among HTA stakeholders. However, framework applicability could be improved if it was complemented by a roadmap with a loop structure to provide tailored guidance for different cases, and with items to elaborate actions to be taken by stakeholders. Accordingly, a 48-item roadmap was developed.

The IHTAM framework was generally applicable to the three case studies. A roadmap, with loop structure and actionable items, could complement the framework, and may provide HTA stakeholders with tailored guidance on developing new methods. To further examine the framework applicability, we recommend stakeholders to apply the IHTAM framework and its roadmap in future practice.

The aim of this study is to understand the path for establishing digital health technologies-health technology assessment (DHT-HTA) in India.

A rapid review of HTA and DHT frameworks on PubMed (MEDLINE) and Google Scholar was conducted to identify DHT-HTA guidelines, and HTA processes in India. MS-Excel template was created with key domains for assessing DHT in resource-constrained settings based on studies and reports identified. Responses received from seventeen experts with varying expertise in DHT, HTA, clinical, and research were contacted using an online form. Following the principles of qualitative research rooted on grounded theory approach, themes and domains were derived for a framework which was again circulated through participants. Weightage for each theme was assigned based on the frequency of responses and qualifiers were used to interpret results. Inductively derived themes from these responses were clubbed together to identify macro-level systems requirements, and finally pre-requisites for setting up DHT-HTA framework was synthesized.

HT are commonly perceived by experts (64.7 percent participants) as a technology strictly connected to health information. Real-world data (i.e., electronic health data) are recognized as a relevant tool in support of decision-making for clinical and managerial levels. Experts identified some pre-requisites for the establishment of DHT-HTA in the country in terms of infrastructure, contextual factors, training, finance, data security, and scale-up.

Our research not only identified the pre-requisites for the adoption of a DHT-HTA framework for India, but confirmed the need to address DHT-HTA’s acceptability among. Hospitals and health insurance providers.

The sandbox approach, developed in the financial technologies sector, creates an environment to collaboratively develop and test innovative new products, methods and regulatory approaches, separated from business as usual. It has been used in health care to encourage innovation in response to emerging challenges, but, until recently, has not been used in health technology assessment (HTA). This article summarizes our learnings from using the sandbox approach to address three challenges facing HTA organizations and to identify implications for the use of this approach in HTA.

We identified three challenging contemporary HTA-related topics to explore in a sandbox environment, away from the pressures and interests of “live” assessments. We convened a pool of 120 stakeholders and experts to participate in various sandbox activities and ultimately co-develop solutions to help HTA organizations respond to the identified challenges.

Important general learnings about the potential benefits and implementation of a sandbox approach in HTA were identified. Consequently, we developed recommendations to guide its use, including how to implement an HTA sandbox in an effective way and the types of challenges for which it may be best suited.

For many HTA organizations, it is difficult to carefully consider emerging challenges and innovate their processes due to risks associated with decision errors and resource limitations. The sandbox approach could reduce these barriers. The potential benefits of addressing HTA challenges in a collaborative “safe space” are considerable.

To assess the health technology assessment (HTA) process in Greece from its establishment in 2018 until 2023 in terms of timeliness and productivity.

Data were collected from the HTA Committee’s database and other publicly available sources. The overall study timeframe was divided into three periods: (i) July 2018–January 2020, (ii) January 2020–July 2021, and (iii) July 2021–February 2023.

During the study period, a total of 1,157 applications for medicinal products (MPs) (including 219 new active substances (NAS) and orphans) were submitted to the HTA Committee. The number of HTA recommendations increased from 60 (first period) to 641 (third period), while the backlog of MPs pending HTA and price negotiations decreased from 89 and 106 (January 2020) to 8 and 44 (February 2023), respectively. The median time intervals for all application types decreased significantly over time. In February 2023, the median time for clinical data assessment of NAS (excluding orphans) almost halved from 207 days in the first period to 114 days; median times for NAS and orphans from regulatory approval to HTA application were 420 and 457 days, and from HTA application to reimbursement 228 and 417 days, respectively.

The performance of the HTA process in Greece improved significantly over time, with increased MP appraisals, backlog reduction, and decreased timelines. Delays in reimbursement of NAS were mainly caused by the long gap between regulatory approval and HTA application. Overall, HTA review times in Greece are now on par with that of well-established European HTA systems.