Obesity, defined as a body mass index (BMI) greater than 30kg/m2, is a multifactorial disease with severe health and economic consequences. As obesity associated events impact long-term survival, health economic (HE) modelling is commonly used. However, the current set of modelling approaches are very diverse and lack external model validation. The aim of our research was to compare the event simulation and the HE outcomes of different structural approaches.

We performed an external validation of three main structural modelling approaches for estimating obesity-associated events: (i) continuous BMI-related risks; (ii) general risk equations; and (iii) categorical BMI-related risks. The Swedish Obese Subjects (SOS) intervention study was used for validation. Outcomes compared were mortality, cardiovascular events (CVE), and type 2 diabetes (T2D), over time using the long-term data from the SOS study, looking at both the surgery arm and the control arm. Concordance between modelling results and the external validation study was measured by visual examination of the best fitting linear regression lines, R2 coefficients, the root mean squared errors, and F-tests. Furthermore, we compared the HE modelling results, comparing surgery versus control, expressed as cost per quality-adjusted life-year (QALY) gained based on 1,000 Monte Carlo simulation samples.

Mortality was overestimated by all approaches irrespective of the study arm. For CVE an overestimation by all structural approaches was observed for the control arm. The CVE surgery arm was overestimated by the categorical BMI approach and slightly underestimated by the others. For T2D an underestimation was observed for the continuous and the categorial BMI approaches, whereas there was an overestimation by the risk equation approach. Considering different confidence interval limits, the cost per QALY gained are comparable between all structural approaches.

None of the structural approaches provided perfect external event validation results although the risk equation approach showed the smallest deviations compared to the external validation study. The cost per QALY gained resulting from the three approaches were fairly comparable.

With the disease spectrum changing in China, type 2 diabetes mellitus (T2DM) has become the main chronic disease which affects people’s health severely, bring patients serious economic burden of disease. For T2DM patients, reliable quality of evidence in decision-making are significant, improving the efficiency of the adjustment of the National Reimbursement Drug List (NRDL). Based on the Consolidated Health Economic Evaluation Reporting Standards (CHEERS), we aimed to evaluate the quality of all published pharmacoeconomic evaluations on T2DM drugs in 2020 NRDL.

Because the 2020 NRDL came into effect on 1 March 2021, we searched all published pharmacoeconomic evaluations about T2DM drugs in 2020 NRDL before March 2021 in China National Knowledge Infrastructure (CNKI), Wan fang Data, China Science and Technology Journal Database (VIP), PubMed, and Web of Science. According to the criterion of inclusion and exclusion, all documents were screened and then relevant basic information of targeted documents was extracted. The quality was evaluated by calculating the final scores based on CHEERS. Two reviewers assessed each publication’s quality using the CHEERS instrument and summarized study quality.

A total of 910 papers were searched, and 24 papers were included. These involved six T2DM drugs, specifically IDegAsp, exenatide, liraglutide, lixisenatide, dapagliflozin and empagliflozin. The average score was 18.31, the standard deviation was 3.67, and the average scoring rate was 77.41 percent. Among all items, “characterizing heterogeneity” scored 0.04, least satisfied with requirements. “Setting and location”, “choice of health outcomes” and “assumptions” scored one, most satisfied with requirements. Among the average scores of all parts, “results” scored lowest at 0.55, and “methods” scored highest at 0.85. The Wilcoxon sum-rank tests showed that score rate which represented reporting quality of economic evaluation (EE) was significantly related to “journal type”, “EEs type”, “model choice” and “study perspective”.

The methodological quality of pharmacoeconomic evaluations about T2DM drugs in 2020 NRDL needs to be improved. Improving the quality of literature is the basic guarantee of scientific decision-making in national medical insurance negotiation.

The interactions between regulators, health technology assessment (HTA), and companies play a significant role in the process of getting medicine to patients. These have evolved at a product level as well as at a policy and cross-jurisdictional level; however, it is important these activities are adding value for stakeholders involved. A survey conducted in March 2021 assessed the current interactions from multi-stakeholders, and their perceptions on the added value these interactions bring to better decision-making.

Three separate questionnaires containing nine questions were developed to assess the perceptions from pharmaceutical companies, regulators, and HTA agencies. The three questionnaires contained analogous questions where appropriate. The company questionnaire was sent to senior management at 19 international pharmaceutical companies, the agency survey was sent to 32 agencies (17 regulatory agencies and 15 HTA agencies) in Australia, Canada, Europe, and Asia.

Seven regulators, seven HTA agencies, and nine companies responded to the survey. All regulators and HTAs indicated they have interactions with their peer agencies, as well as between regulators and HTA. The top areas of interactions for regulators were formal work-sharing between regulators during review (86% response) and regulatory strengthening (86%), whilst for HTAs, interactions between HTA on methodology/framework (83%) and HTA capacity building (67%). Regulatory-HTA interactions were seen to have fewer practical benefits, which may suggest areas for improvement. Both companies and agencies believed an effective engagement model should support evidence generation; agencies also viewed an aligned process and improved decision-making as important. Respondents believed that an ideal ecosystem for interactions should facilitate separate remits for stakeholders, converged requirements, aligned process and increased transparency and trust.

This survey provided a snapshot of the current landscape interactions between stakeholders during the life cycle of new medicines, identified the areas where value is added and improvement are needed. Suggested building blocks to improve future interactions included early scientific advice, alignment of evidence requirements, and a collaborative approach among all stakeholders.

There is a range of activities that health technology assessment (HTA) doers have started to improve the process of generation of required evidence for new technologies, and the alignment of regulatory and reimbursement processes that retard the access to patients to them. Different organizations call those processes early advice, early dialogue, or scientific advice to those activities.

We performed a systematic review of the activities named scientific advice (SA), early advice (EA) and early dialogue (ED). Major databases and HTA organizations were explored. The protocol and search strategy were published in PROSPERO. The selection of final articles and documents was done in pairs, and when discrepancies were found a third person resolved with the consensus of the others. A matrix was used to define the commonalities and differences of the described processes.

We initially retrieved 949 documents, after the analysis of duplications and the full text reading of the selected ones, we finally selected 39 documents and described: the type of technologies, the process, the stakeholders, the duration, the costs, and the impact. Big HTA agencies such as the Canadian Agency for Drugs and Technologies in Health (CADTH) or the National Institute for Health and Care Excellence (NICE) included EA or SA among their portfolio of activities as well as networks (European Network for HTA (EUnetHTA) or smaller agencies such as HTA Wales or Basque Office for HTA (Osteba) among others. The type of activity, the process, duration, purpose and costs differ among HTA doers.

There is a need to define what we meant when we are talking about SA, ED, and EA. In fact, regulators used the same processes with different purposes. Our systematic review and the lessons learnt from the European-funded SAFENMEDTECH project will propose a detailed framework that can be useful to better understanding the needs of each of the involved parties and how to make the processes involved more efficient.

The Washington Health Technology Assessment (HTA) program has been guiding coverage decisions in the state since 2007. The Center for Evidence-based Policy works with the program to develop evidence-based HTA reports. In 2020, we presented an HTA on cell-free DNA prenatal screening for chromosomal aneuploidies. In the committee’s discussion, questions around access to screening tests and to prenatal care more generally were raised. We present a case study of how health equities were considered in the development of coverage criteria.

We conducted an HTA using standard systematic review methodologies. Outcomes focused on test accuracy and the impact of screening. We did not look for evidence on access to prenatal screening tests or people’s experience of prenatal screening. We reviewed the meeting transcript to identify issues of health equity and how they influenced the final decision.

During the discussion of the evidence, the committee raised concerns around equitable access to cell-free DNA tests and prenatal screening, including: direct costs to the person; access to the full range of prenatal screening, including ultrasound; and, uptake of prenatal screening. Based on the findings from the evidence report, expert testimony, and public comment, the committee voted to cover cell-free DNA prenatal screening for chromosomal aneuploidies unconditionally.

Health equity is increasingly important in healthcare decision-making. Decision makers should consider how a decision may reduce health inequities and how it may inadvertently increase existing health inequities. Decision makers also need to understand the context within which the decision will be implemented. Consideration of health equity can be addressed in a number of ways, for example through systematic review of equity issues or patient experience or through the use of contextual knowledge from expert and public testimony. Regardless of the method, decision makers should remain transparent in how health equity considerations influenced their final determination.

Coverage of childhood cancer treatment under the Ghanaian National Health Insurance Scheme (NHIS) has been a policy discussion recently. To improve priority setting, Ghana introduced and used health technology assessment (HTA) processes to guide the resource allocation. To understand the role of stakeholders throughout the HTA lifecycle and for this decision, a stakeholder mapping and engagement was undertaken. We share our results of this mapping and analysis for improving management and financing of childhood cancers in Ghana.

We used two main approaches; first, we undertook a systematic policy documents and literature review of stakeholders relevant in childhood cancer management and financing in Ghana. This was followed by a stakeholder engagement workshop of key stakeholders from the Ministry of Health, Ghana Health Service (GHS), National Health Insurance Authority (NHIA), academia, non-governmental organizations (NGOs), private sector, teaching hospitals, patient groups, and civil society organizations. Participation was done in-person and virtual. Questions were moderated using a focus group discussion approach where responses were recorded. Data were analysed using synthesis and development of themes. Mapping of stakeholders was done using Mendelow’s power-interest grid.

The mapping identified eight key stakeholders in different roles: policy makers (Ministry of Health), payer (NHIA), healthcare providers (teaching hospitals, GHS, private hospitals), pharmaceutical companies, patient group (Ghana Parents Association for Childhood Cancers), and advocacy group (NGOs). Analysis showed that power and interest are concentrated with Ministry of Health and NHIA primarily because of control over resources. Also, while healthcare providers, patient groups, and advocacy groups have high interest, their power ranges from low to moderate. Further analysis of data from the workshop revealed that inability to pay for high cost of treatment leads to treatment abandonment. Payment for treatment was mainly out-of-pocket and by donation from philanthropist.

There was a strong will from stakeholders to extend coverage of the NHIS to childhood cancers in Ghana. Stakeholder engagement is a powerful tool and should be an integral part of every HTA process.

Blood collection in a pediatric population is a time-consuming activity and an unpleasant experience. Moreover, many laboratory tests require only small amounts of blood while larger quantity of blood is usually drawn, generating excess waste that must be properly disposed of. To solve patient concerns and workflow inefficiencies biomedical companies developed Pain-Free Blood Draw (PFBD) devices. The aim of this health technology assessment (HTA) study is to compare the performances of PFBD devices with the standard venipuncture to evaluate the potential benefits of introducing PFBD devices into clinical practice.

PFBD devices use microneedles that breach the stratum corneum, significantly reducing the pain perception due to the superficial skin penetration. Decision-oriented HTA method, was applied to conduct the HTA process. It is an analytical instrument that integrates the EunetHTA CoreModel with the analytic hierarchy process, to choose the best technology solution by identifying the main evaluation criteria and defining the weightsof system and performance values. Eight professionals have been involved to define the evaluation criteria and to measure the two technologies’ performance. As the method requires, a literature review was conducted to define the evaluation scheme represented by a multilevel decision tree composed of evaluation areas (domains) and key performance indicators (KPI).

Five evaluation domains were included in the analysis (clinical effectiveness, safety, costs, organizational aspects, and technical characteristics), described by 35 KPIs. Preliminary clinical effectiveness results showed diagnostic concordance between blood samples obtained with PFBD and venipuncture. Even if the additional costs of PFBD, these devices seem to improve the safety by reducing the biological risks for operators. Moreover, considering pediatric patients, organizational aspects would benefit by the use of PFBD in terms of ease of use, compliance of patients, and time reduction for blood collection.

Results showed that PFBD not only have great repercussions in terms of clinical benefits, especially for pediatric patients, but also a significant impact in terms of organizational aspects.

SINTESIS-new technologies is the early warning system for new and emerging technologies of the Agencia de Evaluación de Tecnologías Sanitarias, Instituto de Salud Carlos III. SINTESIS is part of the Action Plan for the Early Detection of New and Emerging Technologies of the Spanish Network of Health Technology Assessment Agencies (RedETS). In RedETS, four agencies are responsible for the identification of innovative technologies. These agencies have been collaborating since 2016 according to the early awareness methods contained in the EuroScan Methods Toolkit. SINTESIS focuses on secondary information sources (i.e., experts and literature). This study describes the experience of SINTESIS in identifying and filtering new technologies in recent years.

Retrospective analysis of all new and emerging technologies notified by SINTESIS to RedETS since 2018. Technologies were analyzed on a year-by-year basis for their source of information, the clinical specialties involved, and whether technologies found in the identification phase were selected for further assessment.

Between 2018–2020, SINTESIS identified 69 emerging and new technologies. Most of the information came from medical press news (35%), and medical web news (22%); other sources included experts (15%), licensing news search (12%), general press (12%), and scientific websites (6%). Almost 37 technologies (54%) were selected for further analysis. Reasons for exclusion included too early identification of technologies/prototypes without enough evidence (52%), technologies already implemented (28%), overlapping technologies between agencies (17%), and not being medical technologies (3%).

Experience suggests that news sections of general and medical journals, websites, and expert consultation are useful sources to identify new and emerging health technologies. The main limitation is that the technologies identified are often at too early a stage of development for further assessment. SINTESIS contributes, within a national horizon scanning system with other agencies, to broaden the information sources and provide useful data on early awareness of innovative technologies. Further studies are needed to assess the impact of emerging technologies detection on healthcare delivery.

While various criteria exist to define or categorize innovative medicines as new or repurposed, to our knowledge there are no standardized systems that sufficiently capture the range of pipeline products. The National Institute for Health and Care Research Innovation Observatory (NIHR IO) undertakes routine horizon scanning to support health technology assessment (HTA) in England and maintains a comprehensive Medicines Innovation Database (MInD). The aim of this project is to develop a ‘technology type’ (new versus repurposed) classification system for application within the MInD and to provide a high-level analysis of the emergent data.

We reviewed gray literature, regulatory websites, and drug repositories to identify existing ‘technology type’ classification criteria. Preliminary definitions and classifications for use on the MInD were discussed, refined, and agreed by consensus. Innovative medicines on the MInD were classified as either new or repurposed based on their regulatory approval status (Marketing Authorization) using data from the electronic medicines compendium. For repurposed medicines, further classification was undertaken using abbreviated new drug application (ANDA) data from the FDA Orange Book to identify generic medicines (patency and exclusivity status). We combined a range of semi-automated and manually derived data during this process.

Six technology types were identified and applied to the MInD: (i) new technology; (ii) repurposed technology (on-patent/branded); (iii) repurposed drug (off-patent/generic); (iv) repurposed technology (never commercialized); (v) new and repurposed technology (combinations); and (vi) repurposed technology (combinations). Preliminary analysis of a subset of MInD records identified in July 2021 (n = 113) found mainly 52 percent new technologies, 27 percent new and repurposed technologies (combinations) and 14 percent repurposed technology (never commercialized). Further analysis of approximately 7000 MInD records are ongoing and will report temporal trends, regulatory status, and key challenges.

Our novel evidence-based approach to developing classifications for technology types of innovative medicines resulted in six mutually exclusive states that can be applied to a larger dataset. We believe this offers HTA stakeholders a mechanism to gain valuable insights into the innovation trends, gaps, and areas of unmet need.

The National Institute for Health and Care Research Innovation Observatory (IO) is a horizon scanning centre based at Newcastle University, United Kingdom. The IO provides horizon scanning intelligence on new and innovative medicinal products to the National Institute for Health and Care Excellence (NICE) as technology briefing notifications (TBNs). We present an analysis of how TBNs produced between April 2017 and October 2021 feed into the NICE HTA process and used to inform their Technology Appraisal (TA) programme.

TBNs were mapped to relevant published NICE TA guidance and time from horizon scanning identification to NICE recommendation was studied. For mapping technologies undergoing appraisal, provisional guidance-in-development (GID) identification numbers (IDs) were used. For technologies that had not reached the NICE scoping stage yet, the NICE Topic Selection decision and ID was used.

Six hundred and ninety-three TBNs were submitted to NICE between April 2017 and October 2021; 653 were prioritised for TA. Of those, eleven percent mapped to a published NICE TA guidance; forty-three percent to a GID, twenty-two percent were undergoing consultation, and three percent were not traced. Further twenty-one percent mapped to a suspended or terminated TA. Reasons for this included HTA timeliness, regulatory issues or companies unwilling to submit evidence to NICE. Time from technology identification to TA guidance publication ranged from twenty-two to 115 months. The average time from TBN submission to NICE recommendation was thirty months.

Timely notification is key in achieving TA recommendation aligned with market authorization but not the only influencing factor. After issuing a TBN, the NICE appraisal process might be terminated, suspended or withdrawn due to unforeseen factors. Horizon scanning plays a key role triggering the NICE TA process; understanding factors that influence the successful TA completion would streamline processes and find efficiencies.

Inappropriate prescribing of antibiotics is a significant driver of antimicrobial resistance (AMR) which is a global health challenge. Technological innovations present an opportunity to reduce demand for antimicrobials through infection prevention, detection, and management. The National Institute for Health and Care Research (NIHR) Innovation Observatory (IO) has developed horizon scanning methods to identify promising innovations (devices/diagnostics/digital) and anticipate technological trends. Together these insights build a comprehensive landscape and presents a significant opportunity for decision-makers and HTAs to consider the clinical, financial, infrastructural, and logistical provisions to improve preparedness for the potential adoption of these future innovations.

The IO developed a detailed dataset of technologies by formulating search strategies for AMR, based on a comprehensive list of terms and input from expert panels. Primary and secondary sources were systematically scanned using a combination of traditional scanning methods, automated and novel artificial intelligence (AI)/machine learning techniques. Sources included clinical trial registries, MedTech news, academic sources, funding agencies, commercial sites, and regulatory authorities.

Our global dataset identified over 3000 innovative preventative, detection, and monitoring technologies mapped across AMR clinical pathways (including sepsis, respiratory tract infections). Development activity largely concentrated in the United States of America and United Kingdom. Emerging trends included the application of novel materials to prevent infections (e.g., catheter coatings) and novel analytical techniques (e.g., biosensors, microfluidics, breath analysis) to support optimal patient treatment. Data analysis revealed a high proportion of technologies were diagnostic innovations addressing unmet needs such as rapid and accurate detection (including drug-resistant infections).

The rapid development and application of technological interventions presents an opportunity to strengthen national AMR strategies worldwide, through the adoption of new innovations. Improvements in exiting technologies, along with technological advancements have the potential to support appropriate prescribing of antimicrobials and thus address the rise in AMR.

As a new milestone in health technology assessment (HTA) implementation in Tunisia, L’Instance Nationale de l’Evaluation et de l’Accréditation en Santé (INEAS)—the Tunisian HTA body—published a set of methodological guidelines to support HTA dossier submission by the pharmaceutical industry. Including, ‘guide for submitting clinical data for an HTA at INEAS’, ‘methodological choices guide for pharmacoeconomic analysis at INEAS’, and ‘methodological choices guide for budget impact analysis at INEAS’. We aim to report the major methodological recommendations of the pharmacoeconomic analysis guideline.

The ‘methodological choices for pharmacoeconomic analysis at INEAS’ guideline was reviewed and the major recommendations were retrieved and reported.

The reference analysis required by INEAS is the cost-utility analysis systematically combined with a cost-effectiveness analysis (cost per life-year gained) from the public payers’ perspective. The choice of any other type of analysis must be duly justified. Comparators should include alternative treatments which are considered to be ‘the standard of care’ (i.e., interventions routinely used in Tunisia for the same indication) and in which public resources are invested. The time horizon should be sufficiently long to reflect all differences in costs and outcomes. Additionally, a discount rate of 5 percent per year is recommended. The best available evidence for efficacy, safety and quality of life is required. An indirect measure of patient preference, through a validated measurement instrument is preferred for utility calculation. Cost inputs should be identified from Tunisian sources. Health resource utilization should reflect the care pathway in Tunisia. INEAS favors the use of a recognized model. Uncertainty and impact of the input parameters on the results should be assessed and reported through probabilistic and deterministic sensitivity analyses. Model validation tests to assess face validity and internal validity should be performed, and a discussion of the methods used provided. Demonstration of external validity is required. Results should be presented in incremental cost-utility and cost-effectiveness ratios.

The recommendations of ‘methodological choices for pharmacoeconomic evaluation at INEAS’ is an important step to facilitate and harmonize pharmaceutical companies’ submissions and to enhance the use of these analyses in decision-making.

The French National Authority for Health (HAS) “defines and issues guidelines and medico-economic opinions on prevention, healthcare, prescription, and best care strategies, and contributes to their comparison or ranking to support public health and optimize health insurance spending.” Based on a decade of producing cost-effectiveness evaluations, the Economic Evaluation and Public Health Committee (CEESP) issued two documents to frame its activity related to the economic evaluation of health products: (i) the new guidance highlights the expectations of the CEESP regarding cost-effectiveness evaluations; (ii) the doctrine elucidates the grading of methodological reservations expressed during the technical appraisal of manufacturers’ submissions, the CEESP’s statements regarding its findings, and the key messages it wishes to convey to public decision-makers, especially to negotiate healthcare product prices.

We aim at sharing the content of these documents and describing the willingness of the CEESP to support decision-makers in implementing evidence-based pricing policies.

The new guidance provided an opportunity for HAS to stress the importance of interpreting the evaluations, which are often perceived as highly technical. In this perspective, several guidelines call for more reasoned reflection on the objectives of the evaluation upon its conception, along with a constant effort to justify the methodological choices made and an extensive interpretation of the results produced.

The doctrine highlights two steps taken by the CEESP, mainly built on analyzing the cost-effectiveness evaluation’s uncertainty. First, the ability to characterize the level of the ICER in a context where no thresholds for willingness-to-pay exist in France; second, the suggestion of specific regulation schemes to increase the cost-effectiveness of the products.

The CEESP developed the new guidance and its doctrine as conditions to ensure the usefulness of the economic evaluation for decision-making.

The Brazilian health technology assessment (HTA) process includes calls for public consultations, in which society can give its opinion on reports emitted by the National Committee for Health Technology Incorporation (CONITEC). Open and closed queries for public consultation are performed by official formularies and can be accessed online at CONITEC webpage. Queries are divided into two categories of reports: clinical protocols and guidelines, and incorporation/exclusion demands. Incorporation/exclusion queries are subdivided in two additional categories: opinion and experience, or technical. In this study we analyze the weight of patients’ participation in opinion and experience queries and their opinion (pro or con) on inclusion/exclusion of health technologies.

Formularies concerning concluded public consultations on health technology incorporation/exclusion reports were extracted from CONITEC website from 1 January to 26 November 2021. Entries on the opinion and experience formularies included amongst others, a close-ended question about the opinion of participants on health technology incorporation/exclusion reports (“favorable”/“against”/“neither”). In this study, we analyzed patients’ opinion contained within concluded public consultations on incorporation/exclusion of health technologies.

A total of 63 health technology incorporation/exclusion queries were performed in the analyzed period, of which there were only four exclusions. A total of 32,209 contributions were registered. “Patients”, “Health professionals”, “Family or caregivers”, “Interest on the theme”, accounted for 99.4 percent (13.5, 16.7, 32.3, 36.7%, respectively). Patient participation accounted for 4,367 (13.5%) entries. The total number of opinions in favor of the presented documents by the “Patients” was 4,268 (97.7%), 59 (1.4%) disagreed and 40 (0.9%) had no opinion.

Public consultation of official HTA reports is a very useful tool to legitimize decisions through social participation. Although patient participation is not numerically the most important category to contribute on public consultation queries, patients are, if not the most influential stakeholder, the main recipient of decisions concerning health technologies incorporations. Further analyses shall investigate experience narratives included in public consultation queries.

The Scottish Medicines Consortium (SMC) conducts early health technology assessment (HTA) of new medicines, the primary output of which is a document referred to as the Detailed Advice Document (DAD). This comprises an overview of all data considered on the clinical and cost-effectiveness of the medicine, as well as the input from patient groups (PGs), patients, and carers. In 2020, SMC commenced a stakeholder evaluation of the DAD including a workshop with PGs and public partners (members of the public who volunteer with SMC) to explore the potential for using the DAD more widely.

PGs and public partners, all having significant experience of engaging with SMC, participated in the workshop. Feedback was gathered using virtual post-it notes, collated and analysed for key themes. We also gathered oral feedback from participants. Sample DADs were distributed for two medicines recently appraised, one of which included a Patient and Clinician Engagement (PACE) meeting. These were chosen because they reflect different aspects of public and patient involvement at SMC, including how this is presented in the DAD.

Overall, the workshop participants (n=7) recognised the DAD was a useful document for the clinicians who are its primary audience. Its language was perceived to be challenging, including complex information that is not accessible to a wide audience and may only be fully understood by those with a good understanding and knowledge of HTA. This was a key barrier to using the DAD more widely, in particular the health economics information. Suggestions for broadening the audience of the DAD included summaries of key points and an introductory section clarifying the purpose of the DAD and its intended audience, along with signposting to the plain language summary produced by SMC. These will be implemented where possible.

Improving how SMC communicates decisions to patients and the public, by working in partnership with these stakeholders, will help strengthen public involvement throughout the HTA process.

Involving and engaging the public is an essential step to engender trust and confidence in HTA organizations. In 2021 the National Institute for Health and Care Excellence (NICE) launched NICE Listens, a new programme of deliberative public engagement seeking to address topics that have complex social, moral, or ethical dimensions. Health inequalities (HI), defined as unfair and avoidable differences in health across populations, was the first topic. The aim was to understand how the public would like NICE to act in regard to HI. Despite repeated attempts to tackle HI in England, the gaps in life expectancy between the most and least deprived continue to widen. NICE has committed to addressing HI in its five-year strategy and NICE Listens forms part of a comprehensive engagement strategy to understand how best to do this.

Twenty-eight (28) members of the public from across England took part in four iterative two-hour online workshops, held fortnightly in late 2021. They consisted of both plenary and breakout sessions and incorporated a range of stimuli including trade-off exercises and interview clips with HI experts.

The findings show clear public support for HI being a high priority for NICE, albeit with limits on how and when HI should be addressed. Actions towards reducing HI should focus on supporting a preventative and systemic response. Importantly, there is a need for a transparent process for incorporating HI within NICE guidance as well as rigorous staff training in understanding and addressing HI. Recommending technologies that benefit the majority even when not accessible for all is acceptable if there are clear plans to manage access gaps.

Reducing health inequalities should be a high priority for NICE and other HTA organizations. Organizations should seek to have clear processes for embedding HI in decision-making. Priority should be given to actions that focus on prevention of ill health and those that have wider system impacts.

Stakeholder involvement in medical device development draws much attention. To make well-considered methodological choices while involving stakeholders, it is essential to know what approaches are available and what challenges they bring in practice. Therefore, the aim of this review was to study which participatory approaches are used in the early stages of the lifecycle of medical device development, and to describe the most important characteristics of these approaches.

We conducted a scoping review and searched PubMed, Embase and Web of Science for articles published between July 2014 – July 2019. Papers were included if they presented original research featuring any form of stakeholder participation in the development of medical devices. We used The Spectrum of Public Participation to categorise the approach of each paper. We describe four characteristics of each approach: the stakeholders involved, data-collection methods, topics addressed, and the challenges associated with the approaches as perceived by the researchers.

From the 14,838 papers from the initial search, 278 were included. All papers could be categorized into three levels of participation: collaboration, involvement, and consultation. The results show that patients and healthcare professionals are most frequently engaged in all approaches, besides stakeholders like citizens, relatives, and experts. The most often used data-collection methods are workshops in the collaboration approach, and interviews in the involvement and consultation approach. Topics addressed in all approaches are: the initial problem, requirements of devices, design choices, testing of devices, and procedural aspects of the involvement. Challenges in the approaches are related to sampling, analysis, social dynamics, feasibility, and closure.

This review shows that despite the abundance of methods mentioned in literature, there are three main approaches to involving stakeholders in device development: collaboration, involvement, and consultation. These mainly differ in the degree of power that is granted to stakeholders, but are comparable in terms of data-collection methods, stakeholders, topics, and challenges.

A recent proliferation of value frameworks, as well as the emergence of innovative approaches to treating disease (e.g., cell/gene therapies) have been accompanied by an increased focus on nontraditional elements of value. We sought to understand whether and how health technology assessment (HTA) agencies consider novel aspects of treatment in value assessments.

We defined treatment novelty as follows: (i) a new mechanism of action or administration; (ii) addresses an unmet need; or (iii) confers a distinct benefit that transforms clinical practice or that is difficult to quantify. We reviewed technical guidance and peer-reviewed literature to investigate how organizations in eight countries (Australia, Canada, England, France, Norway, the Netherlands, Sweden, and the United States) consider aspects of this definition.

All (n = 8) organizations give special consideration to interventions that address an unmet need, particularly in cancer, rare diseases, and other severe conditions. Nearly all (n = 5) organizations consider whether an intervention produces benefits that may not be adequately quantified. Organizations in England, Norway, and France sometimes recommend drugs with less favorable cost-effectiveness estimates than traditionally considered if the drug addresses rare or severe conditions, or if its quality-of-life benefit is thought to be inadequately quantified. The Institute for Clinical and Economic Review in the United States models cost-effectiveness in rare diseases using both a modified societal and health care system perspective. Importantly, the benefits of novel treatments are frequently considered uncertain, particularly treatments with a new mechanism of action. When uncertainty is high, organizations in Canada, England, France, the Netherlands, and Sweden sometimes issue conditional recommendations until additional evidence is submitted. England and Australia have used risk sharing agreements for drugs determined to be novel but uncertain.

The most widely considered aspects of treatment novelty in HTA are unmet needs and potential benefits that are not easily measured. The willingness to pay for novel treatments is often greater, despite inherent uncertainties about benefit and cost-effectiveness.

Research suggests a different willingness to pay for more effective interventions than willingness to accept (WTA) for less effective interventions which has prompted debate as to whether the threshold in the southwest (SW) quadrant should be kinked to reflect this disparity. Acceptance of less costly, less effective interventions with incremental cost-effectiveness ratios (ICERs) in the SW-quadrant presents an opportunity for resource-constrained healthcare systems by releasing resources for other purposes, which is of particular importance during a pandemic. The National Institute for Health and Care Excellence (NICE)’s methods guide suggests the threshold for decision-making for SW-quadrant interventions be the same as for more expensive, more effective interventions. To assess NICE’s WTA less effective treatments, the objective was to review the outcomes and decision drivers for interventions presenting SW-quadrant ICERs.

A review of NICE health technology appraisals (HTAs) containing SW-quadrant ICERs identified from 2015-2021 was conducted. Appraisal details were extracted and analyzed to identify trends in the WTA and decision drivers.

The HTA review identified twenty-one submissions containing SW-quadrant ICERs in the base-case/scenario analysis. Eighty-one percent received a positive recommendation, with ICERs ranging from GBP 30,000-GBP 4.2m (EUR 35,264-EUR 5m) compared to a range of GBP 789-GBP 50,905 (EUR 927-EUR 59,837) for negative recommendations. The HTAs covered a wide range of therapeutic areas including psoriasis, multiple sclerosis and multiple oncology indications. Decision drivers identified that may have had a positive influence on final outcomes included a high net monetary benefit, a small QALY difference, clinical unmet need, poor tolerability of existing treatments, and oral administration route.

The analysis suggests that there is a high rate of acceptance of interventions with ICERs in the SW-quadrant, however, the threshold for acceptance is unclear. The high frequency of HTAs with SW-quadrant ICERs identified in this review indicates the need for further guidance on such interventions in the NICE reference case.

Clinical Decision Support Software (CDSS) can improve the quality and safety of care by providing patient-specific diagnostic and treatment recommendations. However, robust evaluation is required to ensure that the recommendations provided are clinically valid, up-to-date, and relevant to a specific clinical context. Most evaluation studies assess CDSS performance from the perspective of end-user requirements. But only occasionally is CDSS subject to stringent pre- and post-market evaluation, making it difficult to determine the safety and quality in practice. This study aimed to assess CDSS evaluation in Australia to identify gaps in evaluation approaches.

We conducted 11 semi-structured interviews with different policymakers from committees involved in digital health activities in Australia. Data were thematically analyzed using both theory-based (deductive) and data-driven (inductive) approaches.

Our findings indicated that evaluating CDSS as a purely technical intervention has overly narrowed the assessment of benefits and risks by inadequately capturing the sociotechnical environment. Existing evaluation methods, adopting a static view of the implemented system, cannot discern the impact of the dynamic clinical environment and rapidly evolving technology on CDSS performance. The timeframe of evaluation studies are also incongruent with fast software upgrade cycles, with clinical practices and software potentially changing by the time evaluation is complete. The regulation of software as a medical device depends on the intended use. CDSS are exempt from regulation because they only ‘produce advice’; however, this ignores the fact that they can transition to specifying a diagnosis and treatment after a software update. There is no framework for continuous post-market monitoring, and this is especially important when a CDSS algorithm can change and impact on patient management.

The sociotechnical environment is a significant factor influencing the impact of CDSS on clinical practice, therefore evaluation approaches must acknowledge the dynamic nature of clinical and organizational contexts. Pragmatic and data-driven methodologies are required for CDSS evaluation that acknowledge the evolving landscape of clinical practice and its relationship to technology.