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.

The 2021 Health Technology Assessment International (HTAi) Asia Policy Forum (APF) aimed to explore the challenges and opportunities presented by the global COVID-19 pandemic for health systems and industry in the Asia region, to discuss how HTA changed during the pandemic, and what its role may be moving forward into a postpandemic era.

Discussions during the virtual 2021 APF, informed by a premeeting survey of HTA agencies and industry attendees from the region describing their experience during the pandemic, form the basis of this paper.

During the pandemic, regulatory approval for COVID-related technologies was fast-tracked with fewer evidentiary requirements, and little or no HTA being conducted before these technologies were implemented in health systems in the region. “Living HTA” incorporating real-world evidence (RWE) as it was generated became part of the HTA landscape. In comparison, non-COVID technologies experienced regulatory approval and HTA delays. A major concern of APF members was future pandemic preparedness, and to ensure that lessons are learned from COVID-19. Governments need to continue to invest in innovation and allow early regulatory approvals with the increased use of RWE. Capacity building was identified as a key investment, including capacity in HTA, modeling, as well as local production of necessary supplies and equipment. Finally, collaboration at all levels of the health system was viewed as vital.

Post-COVID, different forms of HTA must be embraced as part of the new HTA landscape in addition to increased intra- and intercountry collaboration across all sectors of the health system, including regulatory bodies.

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.

This study evaluates the cost-effectiveness of tisagenlecleucel (a CAR T-cell therapy), versus blinatumomab, for the treatment of pediatric and young adult patients with relapsed/refractory acute lymphoblastic leukemia (R/R ALL) in the Irish healthcare setting. The value of conducting further research, to investigate the value of uncertainty associated with the decision problem, is assessed by means of expected value of perfect information (EVPI) and partial EVPI (EVPPI) analyses.

A three-state partitioned survival model was developed. A short-term decision tree partitioned patients in the tisagenlecleucel arm according to infusion status. Survival was extrapolated to 60 months; general population mortality with a standardized mortality ratio was then applied. Estimated EVPI and EVPPI were scaled up to population according to the incidence of the decision.

At list prices, the incremental cost-effectiveness ratio was EUR 73,086 per quality-adjusted life year (QALY) (incremental costs EUR 156,928; incremental QALYs 2.15). The probability of cost-effectiveness, at the willingness-to-pay threshold of EUR 45,000 per QALY, was 16 percent. At this threshold, population EVPI was EUR 314,455; population EVPPI was below EUR 100,000 for each parameter category.

Tisagenlecleucel is not cost effective, versus blinatumomab, for the treatment of pediatric and young adult patients with R/R ALL in Ireland (at list prices). Further research to decrease decision (parameter) uncertainty, at the defined willingness-to-pay threshold, may not be of value. However, there is a high degree of uncertainty underpinning the analysis, which may not be captured by EVPI analysis.

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.

Clinical practice is shifting toward an era of precision medicine. The use of comprehensive genomic profiling (CGP) in oncology has broad potential as a universal companion diagnostic for targeted therapies which may significantly improve health outcomes while using healthcare resources more efficiently. Given the nature of this technology, assessing the value of CGP presents unique challenges.

This paper draws on evidence from the academic and policy literature in oncology, as well as stakeholder interviews (health economists, payers, clinicians, and public policy officials) in countries using incremental cost-effectiveness ratios (ICER) as part of health technology assessment (HTA).

The degree to which CGP is subject to a value assessment varies significantly across healthcare systems. Current HTA processes focus on evaluating diagnostic testing through co-dependent assessment of diagnostic testing and associated therapeutic interventions. Diagnostic tests with multiple associated therapeutic interventions are rapidly evolving and poorly unsuited to current HTA approaches. Moreover, HTA approaches are limited in their ability to consider broader systemic benefits of the expanded diagnostic capabilities and enhanced opportunities for clinical trial participation offered by CGP.

The assessment of the overall value of CGP is limited by the current models of HTA. This paper suggests policy proposals for value assessment and funding reforms to help broaden patient access to CGP. These include investing in genomic testing infrastructure; decoupling the assessment of the value of CGP testing to identifying predetermined therapeutic interventions; tailoring evaluation methodology; and developing approaches to collecting evidence of clinical, healthcare system and societal benefit.

The reimbursement process for innovative health technologies in Hungary lacks any formalized assessment of clinical added benefit (CAB). The aim of this research is to present the development, retrospective testing, and implementation of a local assessment framework for determining the CAB of cancer treatments at the Department of Health Technology Assessment of the National Institute of Pharmacy and Nutrition in Hungary.

The assessment framework was drafted after screening existing methods and a retrospective comparison of local reimbursement dossiers to that of German and French methods. The Magnitude of Clinical Benefit Scale of the European Society for Medical Oncology was chosen to rate the extent of CAB in oncology, as part of a conclusion complemented by the assessment of endpoint relevance and the quality of evidence. Several rounds of retrospective assessments have been conducted involving all clinical assessors, iterated with semistructured discussions to consolidate divergence between assessors. External stakeholders were consulted to provide feedback on the framework.

Retrospective assessments resulted in average more than 75 percent concordance between assessors on each element of the conclusion. Input from ten stakeholders was also incorporated; stakeholders were generally supportive, and they mostly commented on the concept, the elements of the framework, and its implementation.

The procedure is suitable for routine use in the decision-making process to describe the CAB of antineoplastic technologies in Hungary. Further extension of the framework is required to cover more disease areas for structured and comparable conclusions on CAB of innovative health technologies.

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.

This study aimed to identify different criteria for priority setting of rare diseases to help policy makers in making evidence-informed decisions.

A scoping review was conducted to comprehensively examine the existing various methods and criteria for prioritizing orphan drugs and rare diseases. We performed searching in Scopus, PubMed, Embase, and websites of health technology assessment (HTA) agencies, 2000–21, and data were extracted.

From the 1,580 identified publications, eleven articles were included. Multicriteria decision analysis was the most frequent method (seven out of eleven studies) used for priority setting. The extracted criteria for priority setting of orphan products were analyzed based on six main categories as follows: health outcomes and clinical implications (six subsets which showed clinical implications), economic aspects (four subsets that indicated the economic effects of orphan drugs and rare diseases), disease and population characteristics (six subsets that included the characteristics of the rare diseases), therapeutic alternatives and uniqueness of orphan technologies (two subsets which discussed the alternatives and uniqueness of orphan technologies), evidence (three subsets which regarded the quality and availability of evidence), and other criteria (three subsets dealing with social and organizational criteria). Cost-effectiveness, budget impact, and disease severity were the most frequent criteria in the studies.

Because of the high price of orphan drugs and limitations of using HTA for reimbursement of them, it is critical to explore them by precise technical methods like multiple criteria decision making in priority setting.

To provide an update on the use of health technology assessment (HTA) in Asia and lessons for countries seeking to advance HTA.

Build upon the research by Chootipongchaivat and the World Health Organization identifying eighteen “factors conducive to the development of HTA in Asia.” These factors were used to create a balanced scorecard to assess the progress of HTA, measuring progress against each factor in China, India, Indonesia, Malaysia, Philippines, South Korea, Taiwan, Thailand, and Vietnam. A scoring system was used wherein: 1, No progress; 2, milestone at early stages, ad hoc HTA use; 3, progress on milestone but limited impact; 4, significant progress but limited remit; and 5, significant progress on milestone, routine HTA informs decisions. Total scores indicated progress of HTA while milestone scores provided contextual insights within countries. Literature reviews and expert interviews were used to complete scorecards.

South Korea and Thailand scored highest with seventy-three and seventy-one points, respectively, while Vietnam scored lowest at 28.5. Advanced HTA programs have independent HTA agencies with a broad remit, explicit process and methods, network of researchers, and routine use of HTA. Taiwan and Malaysia fall in a middle tier, with established HTA programs with limited remit. The final tier with China, India, Indonesia, Philippines, and Vietnam, emerging HTA processes.

Universal Health Coverage goals have catalyzed expansion of HTA. Political will, technical expertise, and sustained financing remain challenges for sustainable HTA programs. Legislation supporting HTA is helpful but political will is key. Recommendations for regional collaboration are provided.

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.