Sudden cardiac arrest (SCA) is the most common cause of death in patients with coronary artery disease. Mostly, ventricular tachycardia (VT) and ventricular fibrillation (VF) are the underlying aetiology of SCA, which is claimed to be successfully treated by a novel therapy, a wearable cardioverter defibrillator (WCD, LifeVest®).

The assessment, performed within the European Network for HTA (EUnetHTA), aimed to provide valid data on clinical effectiveness and safety of the WCD. Furthermore, the project intended to elicit patients views on aspects regarding their cardiac disease and the WCD therapy as well as to identify neglected outcomes.

A synthesis of evidence on the basis of a systematic literature search in Medline via Ovid, Embase, the Cochrane Library, and the Centre for Reviews and Dissemination (CRD) databases was performed. The search was complemented by citation tracking and handsearch.

A face-to-face semi-structured focus group interview was performed with five cardiac disease patients in the scoping phase.

Since no prospective controlled trials were found, no assessment of effectiveness could be performed. With regard to safety, five prospective studies were included, but the quality of the body of evidence was very low. Adverse events (AEs) reported were skin rash/itching (6 percent), false alarms (14 percent), palpitations/lightheadedness/fainting (9 percent) and discontinuation due to comfort/lifestyle issues (16-22 percent). Serious adverse events (SAEs) were inappropriate shocks (0-2 percent) and unsuccessful shocks (0-.7 percent). Frequency of SAEs leading to death was 0-.3 percent.

Patients of the focus group reported that experiencing a sense of security was crucial to them. The WCD therapy was not considered an option for weeks or months, due to expected restrictions in living a ‘normal’ and secure life.

No statement can be made about the device effectiveness – further research is needed. More data and more adequate reporting of AEs and SAEs are needed in order to establish the device safety. In particular, more data is needed for risk stratification of high risk patients in order to further narrow down the wide range of indications for the WCD use.

Numerous studies have shown that arthroscopic partial meniscectomy (APM) is not (cost-) effective in patients with symptoms attributed to a degenerative meniscus tear. We aimed to assess the budget impact of reducing APM in routine clinical practice in this population.

A patient-level state transition model was developed to simulate patients recently diagnosed with a degenerative meniscus tear. Three strategies were compared: “current guideline” (i.e., postpone surgery to at least 3 months after diagnosis), “APM at any time” (i.e., APM available directly after diagnosis), and “nonsurgical” (i.e., APM no longer performed). Total societal costs over 5 years were calculated to determine the budget impact. Probabilistic and deterministic sensitivity analyses were conducted to address uncertainty.

The average cost per patient over 5 years were EUR 5,077, EUR 4,577, and EUR 4,218, for the “APM at any time,” “current guideline,” and “nonsurgical” strategy, respectively. Removing APM from the treatment mix (i.e., 30,000 patients per year) in the Netherlands, resulted in a reduction in health care expenditures of EUR 54 million (95 percent confidence interval [CI] EUR 38 million–EUR 70 million) compared to the “current guideline strategy” and EUR 129 million (95 percent CI EUR 102 million–EUR 156 million) compared to the “APM at any time” strategy. Sensitivity analyses showed that uncertainty did not alter our conclusions.

Substantial costs can be saved when APM is no longer performed to treat symptoms attributed to degenerative meniscus tears in the Netherlands. It is therefore recommended to further reduce the use of APM to treat degenerative meniscus tears.

To facilitate moving from research findings to conclusions when conducting systematic reviews (SRs) and health technology assessments (HTAs), evidence grading systems (EGSs) have been developed to assess the quality of bodies of evidence and communicate (un)certainty about the effects of evaluated technologies. Use of EGSs has become an essential step in conducting SRs and HTAs and those relying on review conclusions should be aware of EGSs’ potential limitations.

This study aims to identify EGSs used in SR and HTA practice, and summarize findings on their inter-rater reliability (IRR). Relevant sources were searched to identify EGSs used in recently published SRs and IRR studies of available EGSs. Members of the International Network of Agencies for Health Technology Assessment were surveyed regarding their current approaches.

Preliminary results indicate that only two conceptually similar EGSs are currently used by several organizations in SR and HTA practice: (i) the Grading of Recommendations Assessment, Development and Evaluation (GRADE) and (ii) the Agency for Healthcare Research and Quality Evidence-based Practice Center Program (AHRQ-EPC). Both approaches emphasize a structured and transparent method. However, results from published IRR studies suggest there is a risk for variability in their application due to researchers’ diverse levels of training and experience in using them, and the complexity and heterogeneity of evidence in SRs.

Validated EGSs can play a critical role in whether and how research findings are eventually translated into practice. However, our results indicate a low level of uptake of EGSs in HTA practice. Both currently used EGSs are susceptible to misuse that allows different researchers to grade the same body of evidence differently, and their performance has not been robustly explored in terms of IRR. If these results stand up to replication, one cannot rely on conclusions of published SRs, which has implications for the decisions they inform.

There is a lack of adoption and diffusion of health innovations needed to drive the implementation of important breakthroughs in value-based health care. To stimulate organizational changes, decision-makers need to see potential benefit at an early stage. The aim of the present study was to assess the potential effects of a conceptualized intention to provide digital home-based care and compare it to the current provision of such care. The new intervention aims to strengthen the municipality's care services by offering a digital communication platform to recipients of home-based health services and their dependents. The platform is designed to be implemented nationally and is in line with home service needs identified in several white papers.

An interdisciplinary team united to determine and quantify potential effects of the project. Effects of the digitalized service were distinguished in priced quantitative, unpriced quantitative and qualitative effects. A ten-year present value calculation with a calculation rate of four percent was used for the estimates. A risk analysis was also carried out.

The present value calculation resulted in estimated savings equal to EUR 25.8 million , with present value investments costs of EUR 5.5 million over ten years. This resulted in net present value per invested euro in the public sector equal to EUR 3.2. Overall assessment of uncertainty related to the intervention's socio-economic profitability was deemed average. Based on data quantified estimates from the conceptual phase, the project succeeded in the decision-making and funding needed to proceed into the next developmental phase of the project.

The present approach to early assessment may provide much desired decision support in an early innovation phase when data are still missing. Our experience is that early stakeholder involvement and the early assessment and quantification of value gains are of utmost importance to overcome the critical barriers to organization health innovations.

Health Technology Assessment (HTA) is a multidisciplinary activity that systematically examines different dimensions related to the direct and indirect consequences of health technologies when implemented in healthcare systems. HTA is developed by multidisciplinary teams in order to cover all the mentioned dimensions. However, the quality of the processes produced by HTA teams will depend upon the competencies that those teams will acquire and incorporate including knowledge, skills and attitudes (1). The aim of this research was to determine how well these dimensions and competencies are covered in HTA academic Masters degree courses and manuals.

We analyzed what had been done in terms of competencies definition in HTA: how it has been reflected; theoretically and according to the authors, and how competencies can be structured; know-how and values. We explored HTA manuals and HTA academic Masters degree courses. We searched in Google with specific terms: building capacities, HTA, programs, Masters, diplomas. We used the HTAi vortal and the information related to courses (for example Masters degrees) and HTA agencies and network webpages for programs. The inclusion criteria were formal programs that describe HTA capacity building and not partial teaching of certain aspects of HTA and we excluded non-recognized institutions, or where there was no description of the programs or lack of detail regarding objectives and competencies to be achieved.

We found 105 courses or programs and analyzed 8 reports and 3 manuals. The main challenges that we faced were: that information was difficult to retrieve, not similarly structured, difficulties to find key information in webpages, no program description at all in some cases and the need to contact institutions staff or register as a student to receive the information and finally, it was difficult to obtain contact details of key people. We structured the information on competencies in knowledge, skills and attitudes.

The analyzed Masters degree courses and manuals did not cover all of the dimensions of HTA analysis in an equal and standardized way. The ethical, legal, social and organizational aspects were lacking in some of the programs, while, on the contrary, clinical and economic aspects were substantially included. On the basis of the information retrieved it would be good to define core competencies for HTA.

The COVID-19 pandemic led to a significant surge in clinical research activities in the search for effective and safe treatments. Attempting to disseminate early findings from clinical trials in a bid to accelerate patient access to promising treatments, a rise in the use of preprint repositories was observed. In the UK, NIHR Innovation Observatory (NIHRIO) provided primary horizon-scanning intelligence on global trials to a multi-agency initiative on COVID-19 therapeutics. This intelligence included signals from preliminary results to support the selection, prioritisation and access to promising medicines.

A semi-automated text mining tool in Python3 used trial IDs (identifiers) of ongoing and completed studies selected from major clinical trial registries according to pre-determined criteria. Two sources, BioRxiv and MedRxiv are searched using the IDs as search criteria. Weekly, the tool automatically searches, de-duplicates, excludes reviews, and extracts title, authors, publication date, URL and DOI. The output produced is verified by two reviewers that manually screen and exclude studies that do not report results.

A total of 36,771 publications were uploaded to BioRxiv and MedRxiv between March 3 and November 9 2020. Approximately 20–30 COVID-19 preprints per week were pre-selected by the tool. After manual screening and selection, a total of 123 preprints reporting clinical trial preliminary results were included. Additionally, 50 preprints that presented results of other study types on new vaccines and repurposed medicines for COVID-19 were also reported.

Using text mining for identification of clinical trial preliminary results proved an efficient approach to deal with the great volume of information. Semi-automation of searching increased efficiency allowing the reviewers to focus on relevant papers. More consistency in reporting of trial IDs would support automation. A comparison of accuracy of the tool on screening titles/abstract or full papers may help to support further refinement and increase efficiency gains.

This project is funded by the NIHR [(HSRIC-2016-10009)/Innovation Observatory]. The views expressed are those of the author(s) and not necessarily those of the NIHR or the Department of Health and Social Care.

The rising costs of drugs have necessitated the exploration of innovative payment methods in healthcare systems. Risk-sharing agreements (RSAs) have been implemented in many countries as a value-based payment mechanism to manage the uncertainty associated with expensive technologies. This study aimed to investigate stakeholder perspectives on value-based payment in the Singaporean context, providing insights for future directions in health technology assessment and financing.

This descriptive qualitative inquiry involved participant interviews conducted between October 2021 and April 2022. Thematic analysis was conducted in two phases to analyze the interview transcripts.

Seventeen respondents participated in the study, and five key themes emerged from the analysis. Stakeholders viewed RSAs as moderately positive, despite limited experience with them. They emphasized the importance of clearly defining objectives and establishing transparent criteria for implementing these schemes. The current data infrastructure was identified as both a barrier and facilitator, as RSAs impose administrative burdens. To successfully implement these payment mechanisms, capacity building, and effective stakeholder engagement that fosters mutual trust and cocreation are crucial.

This study confirms previously identified barriers and facilitators to successful RSA implementation while contextualizing them within the Singaporean setting. The findings suggest that value-based payment has the potential to address uncertainty and improve access to healthcare technologies, but these barriers must be addressed for the schemes to be effective.

The National Institute For Health And Care Excellence (NICE) is widely acknowledged as a seminal health technology assessment (HTA) body, known for its transparent and accountable approach to decision-making. This research aimed to investigate the impact of NICE methodology and decisions on international HTA bodies. We sought to identify direct and indirect factors that may influence an international HTA body’s methods or outcomes. To the best of our knowledge, this is the first research to use a qualitative approach to understand the influence of NICE on other HTA bodies.

We conducted 13 semi-structured qualitative interviews with HTA and market access experts from industry and academia from nine countries (Brazil, Israel, Italy, Japan, Poland, Saudi Arabia, South Korea, Sweden, and the United Arab Emirates). The interview script was organized into three main sections: comparing NICE methods and processes with other HTA bodies; the impact of specific NICE decisions; and Likert scale questions (to allow for comparability of opinions).

Most interviewees believed their local HTA body would consider NICE’s decision when evaluating a medicine. However, the way and extent to which NICE influences HTA varied across countries. The most common means of considering a NICE decision was as background information or context for an HTA evaluation. Generally, interviewees suggested that negative NICE decisions had more impact on local decision-making than positive decisions. Nine of the 13 interviewees agreed or strongly agreed that their country’s HTA body considers the decisions of other HTA bodies in their decision-making process. Eleven of the 13 interviewees agreed or strongly agreed that the development of their country’s HTA body methods and processes was influenced by NICE.

NICE is perceived to be a seminal HTA body, with continued influence on HTA agencies in other countries. However, the mechanisms and extent of this influence varies considerably between countries. We suggest that implicit factors are likely to contribute more to NICE’s influence than individual decisions. Nevertheless, further research is needed to reveal these factors and increase efficiency in international HTA decision-making processes.

Malignant tumors have become a major public health problem and their treatment cost is increasing rapidly in China, but treatment aimed at healing diseases or extending patients’ life. There is little empirical research on utilization of healthcare resources of terminally ill cancer patients. In order to explore the optimal treatment decision for patients and provide information for relevant decision makers, this study analyzed the consumption status of medical resources in patients with cancer during the whole treatment period, and the current medical resource utilization efficiency in different levels of hospital for end-stage cancer patients.

This study was based on the clinical treatment and payment data of 2,536 cases of patients with lung cancer from the medical insurance database during the period of 2007 to 2014 in Hubei province. We retrospectively analyzed patients’ medical expenditure and utilization of different medical resources during their whole treatment period as well as at the end stage.

The per capita inpatient expenditures of patients under 50 years old was 193,000 CNY (27,451 USD), while that of the patients over 70 years were 80,000–90,000 CNY (11,379–12,802 USD). Secondly, the medical expenditures spent during the last 6 months of life accounted for 66.1 percent of the total expenditures. Lastly, the medical expenditure spent in tertiary hospitals accounted for 95.3 percent of the total expenditure, and the expenditure was 14,200 ± 17,030 CNY (2,019.82 ± 2,422.36 USD) per visit.

Population aging is not the only factor causing the rise of medical expenditure. The unclear objectives of treatment and the reverse of medical resource allocation are also important factors to boost the growth of medical expenditure. It is necessary to improve the healthcare insurance payment system, strengthen the capacity of primary medical institutions, and develop the palliative care system in China.

Technology assessment in hospital traditionally involves parameters of safety, effectiveness and costs. The prosperity of medical innovations in an era of scarce resources requires more precisely refined methodologies to measure ‘added value’. Our aim was to reveal the added values of technologies by asking professionals to prioritize their adoption into hospitals.

Twelve innovative technologies that were discussed for adoption over three years were controversial regarding their actual “added value”. Fifty-two managerial health professionals ranked these technologies on two scales: hierarchic importance (league scale) and comparative score rating (CSR), reflecting willingness-to-pay (WTP). The distribution of ranking indicates the internal agreement (IA) among the participants.

There was only partial correlation between the two scales. For example, glucose-monitoring was ranked ‘highly important’ on the hierarchic (league) scale with high CSR/WTP, but with low IA. This can be interpreted as “a valuable technology but with disagreement on comprehensive adoption in the entire hospital”. The surgical robot was ranked ‘highly important’ on the hierarchic scale with low CSR/WTP, but with high IA, meaning “a valuable technology but with consensus to delay adoption in the hospital”. Overall, the participants raised thirty-two “values” that can be assorted into five clusters of significance: clinical effect (6 values), social/public dimension (8 values), patient-physician interaction (9 values), technological aspect (5 values) and policy-regulatory perception (4 values).

We identified different ‘;patterns’ for defining the ‘value’ of various technologies. Revealing these aspects can create a “set of values” of relative weights that may explain the added value considerations in prioritization of decision making. Interestingly, there were technologies that were ranked low, but achieved a high rating. This can be explained by individual personal-oriented added value perspectives. Using this innovative tool to incorporate social value-based scores can assist in understanding the determinants, beyond the current traditional rationing mechanism, that guide professionals while prioritizing medical technologies.

Rapid reviews are of increasing importance within Health Technology Assessment (HTA) due to the need for timely evidence to underpin the assessment of new technologies as well as financial constraints. There are many rapid review methods available (1) although there is little guidance as to the most suitable methods (2). A recent paper outlines issues to consider when selecting rapid review methods (3). The aim of this presentation is to present key aspects to consider when selecting rapid review methods.

We searched the evidence base for guidance on the selection of rapid review methods. We also examined three recently completed systematic reviews to identify rapid review methods used, the reasons for selection and the strengths and weaknesses of each method. Finally we identified key aspects to consider when selecting rapid review methods.

The evidence on guidance identified for the selection of rapid review methods was very limited. The analysis of the three reviews found that each review had distinctly different challenges, such as large numbers of relevant trials and heterogeneity in terms of populations, interventions, comparators and outcomes. All reviews included at least ten randomized controlled trials and numerous outcome measures. Three different approaches to the rapid review of the evidence were used in the three reviews. Key themes to consider when selecting rapid review methods were identified. These include: the size and nature of the evidence base, the characteristics of included studies and the expectations of those commissioning the review.

Rapid review methods need to be chosen to fit the needs of the review, each of which may have different challenges. Collaboration between those producing rapid reviews and commissioners is crucial when choosing methods to ensure that the needs of commissioners are met and limitations associated with the chosen methods are understood.

Health state utilities measured by the generic multi-attribute utility instruments (MAUIs) differ. Empirical evidence suggests that some MAUIs are more sensitive than others in reflecting the quality of life (QoL) of patients in particular disease areas. Additionally, in order to estimate utilities based on cancer-specific health-related quality of life instruments (CSQoLs), a number of mapping functions have emerged. Although it is common practice to apply a CSQoL instead of a MAUI in clinical trials, CSQoL cannot be used to estimate utility values for economic evaluations. Mappings based on MAUIs that are not sensitive to changes in cancer patients’ QoL may result in misleading approximations of utilities that could affect allocation of resources. The study objective is to explore the validity and sensitivity of the major MAUIs to variation in the QoL measured by cancer-specific instruments. We aimed to investigate (i) the sensitivity of the general MAUIs scores to changes in the CSQoL, and (ii) whether particular dimensions of the general instrument are more sensitive.

A two stage systematic literature review is conducted. First, an update of the review done by McTaggart-Cowan et al. (2013) on the mapping methods used to determine utilities from cancer-specific instrument. Second, an analysis of studies that measure the relationship between CSQoLs and general MAUIs.

The literature suggests that differences exist between MAUIs in their capacity to capture the QoL dimensions of the CSQoLs. Additionally, the main challenge to build an appropriate mapping function for deriving utilities values from CSQoL is the definition of an appropriate methodology that (i) responds to the distribution of the selected sample and (ii) can successfully be validated in additional samples.

In the context of health technology assessment and cost effectiveness analysis, it is crucial to carefully select and report the CSQoL and MAUI involved in the estimation of the additional benefits. Policy makers need to be awarded of the sensitivity of the instruments to changes in QoL in relation to the CSQoL dimensions QoL.

The aim of this initiative was to examine collaboratively, in a multi-stakeholder team (health technology assessment (HTA) practitioners with patient involvement expertise, health technology industry, patient advocates, health policy experts, patient engagement experts), whether evidence generated through social media research (SMR) fills current information gaps relating to insights on specific aspects of patient experiences, preferences, or patient needs and delivers additional value to HTA.

The framing of the project was done in a co-creative, deliberative multi-stakeholder process. Challenge and refinement happened through discussions with 25 independent stakeholders from HTA bodies, industry, academia, and patient advocacy. For critical themes identified during the framing phase, scoping literature reviews were performed including the state of methods and examples for the use of SMR in HTA.

The framing and stakeholder discussions specified a set of expectations and requirements, and the scoping reviews revealed the current state of methods and usage of SMR in health-policy decision making.

The project concluded that SMR can contribute new, relevant evidence to HTA. It is however recommended to evolve the science through defining best practices when planning, conducting, and using SMR and to conduct multi-stakeholder pilot SMR projects to address questions relevant to current HTAs and to validate and improve the proposed practices.

Focal-onset-seizures (FOS) are commonly experienced by individuals with epilepsy and have a significant impact on quality of life (QoL). This study aimed to develop a mapping algorithm to predict the 6 dimension short form questionnaire (SF-6D) values in adults with FOS for use in economic evaluations of a new treatment, cenobamate.

An online survey, including questions on sociodemographic, disease history, the short form (SF) 36, and an epilepsy-specific measure (quality of life in epilepsy problems questionnaire, QOLIE-31-P) was administered to individuals with drug-resistant FOS in the top 5 EU countries (UK, Spain, Germany, Italy and France). A range of regression models were fitted to SF-6D scores including direct and response mapping approaches.

The analysis included 361 people. In the previous 28 days, the mean number of FOS experienced was three, (range: 0–43) and longest seizure-free period was 14 days (range: 1–28). Mean responses on all SF-36 dimensions were lower than general population norms. Mean SF-6D and QOLIE-31-P scores were 0.584 and 45.72, respectively. The best performing model was the ordinary least squares (OLS), with root mean squared error (RMSE) and mean absolute error (MAE) values of 0.0977 and 0.0742, respectively. Explanatory variables which best predicted SF-6D included seizure frequency, seizure severity, seizure freedom, and age.

People with drug-resistant FOS have poor QoL. The mapping algorithm enables the prediction of SF-6D values from clinical outcomes in individuals with drug-resistant FOS. It can be applied to outcome data from clinical trials to facilitate cost-utility analysis.

Lack of evidence regarding safety and effectiveness at market entry is driving the need to consider adopting a lifecycle approach to evaluating medical devices, but it is unclear what lifecycle evaluation means. This research sought to explore the tacit meanings of “lifecycle” and “lifecycle evaluation” as embodied within evaluation models/frameworks used for medical devices.

Drawing on qualitative evidence synthesis methods and using an inductive approach, novel methods were developed to identify, appraise, analyze, and synthesize lifecycle evaluation models used for medical devices. Data was extracted (including purpose; audience; characterization; outputs; timing; and type of model) from key texts for coding, categorization, and comparison, exploring embodied meaning across four broad perspectives.

Fifty-two models were included in the synthesis. They demonstrated significant heterogeneity of meaning, form, scope, timing, and purpose. The “lifecycle” may represent a single stage, a series of stages, a cycle of innovation, or a system. “Lifecycle evaluation” focuses on the overarching goal of the stakeholder group, and may use a single or repeated evaluation to inform decision-making regarding the adoption of health technologies (Healthcare), resource allocation (Policymaking), investment in new product development or marketing (Trade and Industry), or market regulation (Regulation). The adoption of a lifecycle approach by regulators has resulted in the deferral of evidence generation to the post-market phase.

Using a “lifecycle evaluation” approach to inform reimbursement decision-making must not be allowed to further jeopardize evidence generation and patient safety by accepting inadequate evidence of safety and effectiveness for reimbursement decisions.