We sought to examine patients’ perceptions of disease burden and treatment impact, and what patients value over the course of the asthma experience.

Patient focus groups were conducted in three countries, the USA (n = 8 patients), the United Kingdom (n = 7 patients), and Germany (n = 7 patients), to examine aspects of disease burden and patient experience (physical, emotional, clinical, economic, and social). Cause and effect were also explored. Global Initiative for Asthma guidelines were used to screen patients by severity, based on age, sex, time since diagnosis, number of attacks, oral corticosteroid use, and number of therapies needed to control symptoms. Patients classified and ranked aspects of disease burden, including whether it was continuous or episodic in nature, and discussed the interventions used to manage their asthma.

The overall burden of illness was driven by the combined effect of the disease and treatment burdens. All patients highlighted the negative impact of oral corticosteroids. Patients believed that they were the key actors in their asthma management (not healthcare professionals), and reported the physical and emotional burdens as being the most dominant. Understanding of the terms “attack” or “exacerbation” differed significantly between patients, and did not necessarily match the clinical definitions. Patients considered asthma to be an individualized condition that drives lifestyle changes; disease management drives burden and vice versa. Patients perceived that burden was continuous over time, with specific phases of variable duration—before, during, and after an attack—whereas other stakeholders had a more episodic focus. Patients expected more holistic and personalized approaches for managing their asthma.

The research indicated a misalignment between what patients value and what clinicians, payers, and regulators consider in their assessments and decisions. Greater alignment among the different stakeholders, and more inclusion of patients’ values in decision making, will improve outcomes.

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.

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.

There is a general consensus on the need to involve patients and the public in Health Technology Assessment (HTA) but questions remain about the best strategies for involving them into HTA structures and activities. The aim of this study was to update a systematic review (published in 2011) on patient and public engagement in HTA.

We searched papers published between January 2009 (end of the initial search) and November 2016 in eight databases and HTA journals using specific search strategies. We identified other publications through citation tracking, Internet search engines, HTA agencies websites, and discussion with experts in the field. Studies in English or French were included if they met the following criteria: (i) qualitative, quantitative or mixed-methods study; (ii) describing patients or public involvement; and (iii) in the HTA field. We extracted information using a pre-established grid including: characteristics of studies, type of activities for involving patients or public, effects on decisions, and factors facilitating or limiting involvement.

We identified a total of 4,762 new publications from the main search strategy. Among them, twenty-eight articles (reporting on twenty-three studies) met the inclusion criteria, whereas seventeen articles were included in the previous systematic review. Research designs are qualitative (18/23), quantitative (3/23) or mixed (2/23). Two main strategies for involving patients and public are generally described. The first is when public representatives participate directly in decision-making processes (participation) and the second is when patient or public input is sought to inform decisions (consultation or indirect participation).

The number of studies on patient and public involvement in HTA has increased in recent years. Findings from this update are mainly consistent with those of the previous systematic review. However, studies are still needed to assess the effectiveness of different strategies for involving patients and the public in HTA.

Consideration of ethical, legal, and social issues plus patient values (ELSI+) in health technology assessment (HTA) is challenging because of a lack of conceptual clarity and the multi-disciplinary nature of ELSI+. We used concept mapping to identify key concepts and inter-relationships in the ELSI+ domain and provide a conceptual framework for consideration of ELSI+ in HTA.

We conducted a scoping review (Medline and EMBASE, 2000–2016) to identify ELSI+ issues in the HTA literature. Items from the scoping review and an expert brainstorming session were consolidated into eighty ELSI+-related statements, which were entered into Concept Systems® Global MAX™ software. Participants (N = 38; 36 percent worked as researchers, 21 percent as academics; 42 percent self-identified as HTA experts) sorted the statements into thematic groups, and rated them on importance in making decisions about adopting technologies in Canada, from 1 (not at all important) to 5 (extremely important). We used Concept Systems® Global MAX™ software to create and analyze concept maps with four to sixteen clusters.

Our final ELSI+ map consisted of five clusters, with each cluster representing a different concept and the statements within each cluster representing the same concept. Based on the concepts, we named these clusters: patient preferences/experiences, patient quality of life/function, patient burden/harm, fairness, and organizational. The highest mean importance ratings were for the statements in the patient burden/harm (3.82) and organizational (3.92) clusters.

This study suggests an alternative approach to ELSI+, based on conceptual coherence rather than academic disciplines. This will provide a foundation for incorporating ELSI+ into HTA.

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.

In 2020, Canada spent 12.9 percent of its GDP on healthcare, of which 3 percent was on medical devices. Early adoption of innovative surgical devices is mostly driven by physicians and delaying adoption can deprive patients of important medical treatments. This study aimed to identify the criteria in Canada used to decide on the adoption of a surgical device and identify challenges and opportunities.

This scoping review was guided by the Joanna Briggs Institute Manual for Evidence Synthesis and PRISMA-ScR reporting guidelines. The search strategy included Canada’s provinces, different surgical fields, and adoption. Embase, Medline, and provincial databases were searched. Grey literature was also searched. Data were analyzed by reporting the criteria that were used for technology adoption. Finally, a thematic analysis by subthematic categorization was conducted to arrange the criteria found.

Overall, 155 studies were found. Seven were hospital-specific studies and 148 studies were from four provinces with publicly available Web sites for technology assessment committees (Alberta, British Columbia, Ontario, and Quebec). Seven main themes of criteria were identified: economic, hospital-specific, technology-specific, patients/public, clinical outcomes, policies and procedures, and physician specific. However, standardization and specific weighted criteria for decision making in the early adoption stage of novel technologies are lacking in Canada.

Specific criteria for decision making in the early adoption stage of novel surgical technologies are lacking. These criteria need to be identified, standardized, and applied in order to provide innovative, and the most effective healthcare to Canadians.

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.