The methods of economic evaluation and HTA should be based on best practices and standards, tailored to unique country contexts that can be systematically applied to inform decisions. This paper outlines standards for the conduct of economic evaluations for HTA in Ghana.

A five-step process was followed to develop the HTA reference case as a methodological and reporting benchmark. These include (a) a review of literature and evidence synthesis, (b) a review of country policies, (c) a review and adaption of international frameworks, (d) expert/stakeholder consultations, and (e) the development of a methodological framework. A series of stakeholder consultations were done to refine, finalize, and validate the outcomes of the processes to generate a finalized reference case.

The Ghana reference case is made up of 14 components comprising: evidence synthesis, evaluation type, perspectives on cost, perspectives of outcomes, choice of comparator, data sources, outcome measures, discount rate, uncertainty, equity considerations, time horizon, heterogeneity, transparency, and budget impact. These provide methodological considerations and reporting requirements for economic evaluations for HTA. It provides a framework to ensure the best research methods are adopted to harmonize the evidence-generation process with the expectations of policy and decision-makers and ensure that policy decisions are based on uniform evidence.

Recommendations set out in this reference case when followed can provide context-specific evidence to support a rigorous and transparent system for evaluating healthcare interventions and technologies. It will support decision-making, ultimately improving the quality and efficiency of healthcare delivery in the country.

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.

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.

Left ventricular assist devices (LVAD) are a therapeutic option in patients with advanced heart failure (HF) not a candidate for cardiac transplant as destination therapy (DT). However, important uncertainties remain regarding the use of LVAD in the long-term in real practice settings. When planning registries, it is important to choose the appropriate outcomes that ensure comparability and reduce the possibility of bias.

The purpose of this study was to establish a minimum dataset (MDS) that should be collected in all LVAD for DT registries to meet the needs and demands of Health Technology Assessment (HTA) doers and health professionals.

To design the MDS for LVAD, a preliminary list of outcome domains and data items were developed attending to the gaps and research needs derived from existing evidence coming from HTA carried out at the European Network of Health Technology Assessment (EUnetHTA) level. The list of data items and domains was agreed upon by all involved HTA organizations and a three-round Delphi was conducted among an experienced panel of cardiologists to rate the importance of the items for measuring uncertainty gaps.

After the three-round Delphi process, the expert panel reached a consensus regarding eighteen outcomes and forty-seven variables divided into seven main domains (safety, effectiveness, patient acceptability, satisfaction, healthcare system impact, pharmaceutical management, and technique-related factors).

The MDS of outcomes and measures, developed based on research gaps and needs, can allow for standardizing data collection and improving the quality of data for decision making and practice.

Advances in mobile apps, remote sensing, and big data have enabled remote monitoring of mental health conditions, but the cost-effectiveness is unknown. This study proposed a systematic framework integrating computational tools and decision-analytic modeling to assess cost-effectiveness and guide emerging monitoring technologies development.

Using a novel decision-analytic Markov-cohort model, we simulated chronic depression patients’ disease progression over 2 years, allowing treatment modifications at follow-up visits. The cost-effectiveness, from a payer’s viewpoint, of five monitoring strategies was evaluated for patients in low-, medium-, and high-risk groups: (i) remote monitoring technology scheduling follow-up visits upon detecting treatment change necessity; (ii) rule-based follow-up strategy assigning the next follow-up based on the patient’s current health state; and (iii–v) fixed frequency follow-up at two-month, four-month, and six-month intervals. Health outcomes (effects) were measured in quality-adjusted life-years (QALYs).

Base case results showed that remote monitoring technology is cost-effective in the three risk groups under a willingness-to-pay (WTP) threshold of U.S. GDP per capita in year 2023. Full scenario analyses showed that, compared to rule-based follow-up, remote technology is 74 percent, 67 percent, and 74 percent cost-effective in the high-risk, medium-risk, and low-risk groups, respectively, and it is cost-effective especially if the treatment is effective and if remote monitoring is highly sensitive and specific.

Remote monitoring for chronic depression proves cost-effective and potentially cost-saving in the majority of simulated scenarios. This framework can assess emerging remote monitoring technologies and identify requirements for the technologies to be cost-effective in psychiatric and chronic care delivery.

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.

It is vital that horizon scanning organizations can capture and disseminate intelligence on new and repurposed medicines in clinical development. To our knowledge, there are no standardized classification systems to capture this intelligence. This study aims to create a novel classification system to allow new and repurposed medicines horizon scanning intelligence to be disseminated to healthcare organizations.

A multidisciplinary working group undertook literature searching and an iterative, three-stage piloting process to build consensus on a classification system. Supplementary data collection was carried out to facilitate the implementation and validation of the system on the National Institute of Health and Care Research (NIHR) Innovation Observatory (IO)‘s horizon scanning database, the Medicines Innovation Database (MInD).

Our piloting process highlighted important issues such as the patency and regulatory approval status of individual medicines and how combination therapies interact with these characteristics. We created a classification system with six values (New Technology, Repurposed Technology (Off-patent/Generic), Repurposed Technology (On-patent/Branded), Repurposed Technology (Never commercialised), New + Repurposed Technology (Combinations-only), Repurposed Technology (Combinations-only)) that account for these characteristics to provide novel horizon scanning insights. We validated our system through application to over 20,000 technology records on the MInD.

Our system provides the opportunity to deliver concise yet informative intelligence to healthcare organizations and those studying the clinical development landscape of medicines. Inbuilt flexibility and the use of publicly available data sources ensure that it can be utilized by all, regardless of location or resource availability.

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.

The primary objectives of this umbrella review were to (a) quantify the relative importance, of “severity” and “rarity” criteria in health resource allocation; and (b) analyze the contextual factors influencing the relative importance. The secondary objective was to examine how “severity” and “rarity” criteria are defined.

Searches were carried out in PubMed and Embase to identify eligible systematic reviews. Quality appraisal of systematic reviews was undertaken. From identified systematic reviews, primary studies were extracted and further screened for eligibility. The inclusion of severity and rarity criteria and their respective weights in primary studies were examined. Descriptive and regression analyses were performed.

Twenty-nine systematic reviews were screened, of which nine met the inclusion criteria. Primary studies included in these systematic reviews were retrieved and screened, resulting in forty articles included in the final analysis. Disease severity was more frequently considered (n = 29/40) than disease rarity (n = 23/40) as an evaluation criterion. Out of all cases where both were included as evaluation criteria, disease severity was assigned higher weights 84 percent of the time (n = 21/25).

Our review found consistent evidence that disease severity is more relevant and preferred to rarity as a priority-setting criterion albeit constraints in statistical analysis imposed by limited sample size and data availability. Where funding for rare diseases is concerned, we advocate that decision-makers be explicit in clarifying the significance of disease severity and/or rarity as a value driver behind decisions. Our findings also reinforce the relevance of disease severity as a criterion in priority setting.

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.

The costs of low back pain (LBP) are complex and difficult to estimate. This study aims to adapt the Cost for Patients Questionnaire (CoPaQ) for use in LBP populations.

In a cross-sectional qualitative study, we conducted cognitive interviews to assess the CoPaQ’s suitability for addressing costs related to LBP. Three groups of participants were included (n = 5 each): (i) persons with a history of LBP or primary caregiver, (ii) researchers with expertise in LBP, and (iii) primary care providers specialized in treating LBP. The interpretation, analysis, and summary of results used Knafl et al.’s qualitative content analysis method.

Persons with a history of LBP (n = 5), had a median age of 60 years (Interquartile Range (IQR): 26–71.5), and varying durations of LBP, the median duration of LBP 7 years (IQR: 4–32.5). Researchers (n = 5) had a median age of 33 years (IQR: 29–45). Primary care providers (n = 5) had a median age of 40 years (IQR: 37.5–65), and a background in chiropractic care (n = 3) and physiotherapy (n = 2). Content analysis of the interviews revealed sources of error with five pre-determined themes (clarity/comprehension, relevance, inadequate response definition, reference point, perspective modifiers) and one developed theme (organization). We modified the questionnaire for LBP populations based on the feedback.

Our study evaluated the content validity of a questionnaire that assesses the direct and indirect costs associated with LBP. Future studies should pilot this questionnaire with persons of varying LBP severity and compare it with cost diaries.

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 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.

The management of rare diseases is rarely addressed among policymakers and public communities. It is hindered by the lack of information on its epidemiology and burden, especially from the perspective of patients and families with rare diseases. This study aims to understand the perceptions of rare disease patients and their families in the management of rare diseases in Malaysia.

A qualitative interview was used to explore the perceptions of patients and families regarding the management of rare diseases in Malaysia. In-depth interviews were conducted with the rare disease patients or their parents/guardians provided by three major rare disease advocacy groups, between 1 July and 15 September 2016. The interviews focused on two key areas: the challenges associated with rare disease and the issues related to accessing medication.

Out of the nineteen recruited participants, seventeen (89.5 percent) completed the interview sessions. The significance of awareness, knowledge, and support from others emerged as crucial for families and patients living with rare diseases. Despite facing delayed diagnosis and treatment, a majority of patients and parents expressed satisfaction with the advancements in rare disease management. Nevertheless, a prominent challenge revolves around access to enzyme replacement therapy for eligible patients.

This study emphasizes the importance of healthcare professionals understanding patient with rare diseases perceptions to tailor communication strategies, provide accurate information, and address concerns effectively. The message underscores the significance of collaboration between healthcare providers and patient support groups to deliver adequate health information, potentially enhancing patients’ understanding and their illness perceptions.

We draw from the Health Technology Assessment (HTA) literature to propose how hospitals and local health networks can prepare the key components of early economic evaluations to support the development and management of health service interventions.

Using the case example of a proposed intervention for older people in the Emergency Department (ED), a conceptual logic model of a new health service intervention is articulated to inform the structuring and population of a decision-analytic model using observed data on the existing care comparator and structured elicitation exercise of initial stakeholder expectations of intervention effects.

The elicited patient pathway probabilities and lengths of stay quantities profile which of the existing types of patients are expected to avoid the ED and how this impacts the lengths of stay across the system. The exercise also quantifies the stakeholders’ uncertainty and disagreement, with qualitative insights into why. The elicitation exercise participants draw upon the rationale for how the intervention is expected to affect a change within the local context, as captured within the logic model, together with the descriptive analyses of the characteristics and utilization of their target population. Feedback indicates the methods are acceptably robust yet pragmatic enough for healthcare delivery settings.

As proposed in this paper, HTA methods can be used to capture how key stakeholders initially expect a service intervention to affect a change within their local context. The example results can be used in a decision-analytic model to guide the development and management of an intervention.

Our objective was to explore procedures and methods used at health technology assessment (HTA) agencies for assessing medical devices and the underlying views of HTA practitioners about appropriate methodology to identify challenges in adopting new methodologies for assessing devices. We focused on the role of normative commitments of HTA practitioners in the adoption of new methods.

An online survey, including questions on procedures, scoping, and assessments of medical devices, was sent to members of the International Network of Agencies for Health Technology Assessment. Interviews were conducted with survey respondents and HTA practitioners involved in assessments of transcatheter aortic valve implantation to gain an in-depth understanding of choices made and views about assessing medical devices. Survey and interview questions were inspired by the “values in doing assessments of health technologies” approach towards HTA, which states that HTA addresses value-laden questions and information.

The current practice of assessing medical devices at HTA agencies is predominantly based on procedures, methods, and epistemological principles developed for assessments of drugs. Both practical factors (available time, demands of decision-makers, existing legal frameworks, and HTA guidelines), as well as commitments of HTA practitioners to principles of evidence-based medicine, make the adoption of a new methodology difficult.

There is a broad recognition that assessments of medical devices may need changes in HTA methodology. In order to realize this, the HTA community may require both a discussion on the role, responsibility, and goals of HTA, and resulting changes in institutional context to adopt new methodologies.

Computerized clinical decision support software (CDSS) are digital health technologies that have been traditionally categorized as medical devices. However, the evaluation frameworks for traditional medical devices are not well adapted to assess the value and safety of CDSS. In this study, we identified a range of challenges associated with CDSS evaluation as a medical device and investigated whether and how CDSS are evaluated in Australia.

Using a qualitative approach, we interviewed 11 professionals involved in the implementation and evaluation of digital health technologies at national and regional levels. Data were thematically analyzed using both data-driven (inductive) and theory-based (deductive) approaches.

Our results suggest that current CDSS evaluations have an overly narrow perspective on the risks and benefits of CDSS due to an inability to capture the impact of the technology on the sociotechnical environment. By adopting a static view of the CDSS, these evaluation frameworks are unable to discern how rapidly evolving technologies and a dynamic clinical environment can impact CDSS performance. After software upgrades, CDSS can transition from providing information to specifying diagnoses and treatments. Therefore, it is not clear how CDSS can be monitored continuously when changes in the software can directly affect patient safety.

Our findings emphasize the importance of taking a living health technology assessment approach to the evaluation of digital health technologies that evolve rapidly. There is a role for observational (real-world) evidence to understand the impact of changes to the technology and the sociotechnical environment on CDSS performance.

This study examined the application, feasibility, and validity of supervised learning models for text classification in appraisals for rare disease treatments (RDTs) in relation to uncertainty, and analyzed differences between appraisals based on the classification results.

We analyzed appraisals for RDTs (n = 94) published by the National Institute for Health and Care Excellence (NICE) between January 2011 and May 2023. We used Naïve Bayes, Lasso, and Support Vector Machine models in a binary text classification task (classifying paragraphs as either referencing uncertainty in the evidence base or not). To illustrate the results, we tested hypotheses in relation to the appraisal guidance, advanced therapy medicinal product (ATMP) status, disease area, and age group.

The best performing (Lasso) model achieved 83.6 percent classification accuracy (sensitivity = 74.4 percent, specificity = 92.6 percent). Paragraphs classified as referencing uncertainty were significantly more likely to arise in highly specialized technology (HST) appraisals compared to appraisals from the technology appraisal (TA) guidance (adjusted odds ratio = 1.44, 95 percent CI 1.09, 1.90, p = 0.004). There was no significant association between paragraphs classified as referencing uncertainty and appraisals for ATMPs, non-oncology RDTs, and RDTs indicated for children only or adults and children. These results were robust to the threshold value used for classifying paragraphs but were sensitive to the choice of classification model.

Using supervised learning models for text classification in NICE appraisals for RDTs is feasible, but the results of downstream analyses may be sensitive to the choice of classification model.