bluuebunny/crossref_metadata_2025_split huggingface dataset:
OP64 NICE Listens: Engaging The Public On How To Address Health Inequalities In Health Technology Assessment And Guideline Development
Authors: Cresswell Katharine , Shah Koonal 
Published: December 2022

IntroductionInvolving 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.MethodsTwenty-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.ResultsThe 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.ConclusionsReducing 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.
OP65 An Overview Of Participatory Approaches, Stakeholders, Methods, Topics and Challenges In Medical Device Development: A Scoping Review
Authors: Woudstra Kas , Reuzel Rob , Rovers Maroeska , Tummers Marcia 
Published: December 2022

IntroductionStakeholder 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.MethodsWe 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.ResultsFrom 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.ConclusionsThis 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.
OP67 Considerations Of Treatment Novelty In Health Technology Assessment
Authors: Synnott Patricia , Enright Daniel , Voehler Dominic , Kowal Stacey , Ollendorf Daniel 
Published: December 2022

IntroductionA 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.MethodsWe 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.ResultsAll (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.ConclusionsThe 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.
OP78 Taking A Societal Perspective In Health Technology Assessment: Is Environmental Impact A Special Case?
Authors: Kenny Juliet , Shah Koonal 
Published: December 2022

IntroductionA source of debate among the health technology assessment (HTA) community is what perspective should be taken in health economic evaluations. Many stakeholders advocate that a societal perspective is taken in order to include a comprehensive range of costs and outcomes and (in theory) make societally optimal decisions. The Second Panel on Cost-Effectiveness in Health and Medicine recommended that a societal perspective be presented alongside a health sector one. The Second Panel included environment as one item on its impact inventory—alongside productivity, education, and others—intended to support the use of a societal perspective. However, many HTA agencies, including the National Institute for Health and Care Excellence (NICE), have continued to use health sector-specific evaluations to inform decision-making. The presentation seeks to examine whether consideration of the environmental impact of healthcare requires/implies the formal adoption of a societal perspective in health economic analyses.MethodsThe presentation will provide an overview of the societal perspective, explaining how it differs from a health sector perspective and describing its main strengths and weaknesses. We then present policy analysis undertaken by NICE’s Science Policy and Research team to identify reasons for measuring environmental impact in HTAs and examine whether these align with the broader arguments for or against adopting a societal perspective in economic analyses.ResultsThree reasons for considering environmental impact are identified: (i) to support parallel policies which demand healthcare system transformation against emissions targets; (ii) to ensure planetary and human health, in the future as well as the present; and (iii) to offset future healthcare resource use. We show that only the third reason aligns with arguments related to the choice of perspective for economic analyses. Moreover, this reason is arguably better aligned to maintaining a (potentially modified) health sector perspective. The implications of the results will be discussed with reference to updating reimbursement decision-making frameworks, such as those used by NICE, to account for the environmental consequences of healthcare.
OP81 Do Sustainable Healthcare Principles Inform Guidance Development? An Exemplary Case Study In Respiratory Care
Authors: Walpole Sarah , Khan Naba , Smith James , Rostkowska Olga , Miller Fiona 
Published: December 2022

IntroductionAt the United Nations Climate Change Conference (COP26) in 2021, over 40 countries made commitments to low carbon, sustainable health care. Respiratory care provides a case study to explore how existing evidenced-based guidance can inform progress towards more sustainable care pathways and technologies. Our aim is to identify whether environmental aspects of health technology assessment (HTA) are referenced in guidance and the extent to which the four principles of sustainable health care (prevention, self-care, streamlining, and low carbon technology) are promoted in guidance.MethodsInternet searches enabled identification of current national guidance on management of respiratory diseases in English, French or Polish. Guidances were reviewed to identify references to environmental aspects of HTA and recommendations that align with each of the four sustainable healthcare principles.ResultsGuidance on respiratory care is produced by varied stakeholders globally. Some principles of sustainable health care are frequently reflected in guidance to improve quality of care, but others are missed where environment sustainability is not considered. Reference to HTAs incorporating environmental impacts is lacking. There is limited engagement with the environmental impacts of inhalers in guidance. Guidance created by clinician groups (e.g., Greener Practice) and research networks (e.g., Centre for Sustainable Health Systems) has responded more quickly to the need to address sustainability concerns compared to guidance produced by national public bodies.ConclusionsHTA organizations may need to take a broader perspective, incorporating environmental impacts in assessments. This could have an influential role in enabling evidence-informed guidance and development of sustainable care pathways and technologies. Limitations of our study were lack of evaluation of local guidance due to limited capacity, language restrictions, and subjectivity in assessing whether each sustainable healthcare principle was addressed in guidance. There may be limited transferability of our results to other specialties or settings. Further research on the sustainability impacts and relative merits of different health technologies and care pathways is required to inform HTA and guidance.
OP83 Joining Efforts To Improve Patient Involvement In Health Technology Assessment: The Case Of The RedETS Patient Interest Group
Authors: Triñanes Yolanda , Reviriego-Rodrigo Eva , Prieto-Remón Lucía , Novellas-Arriba Blanca , Gallastegui Edurne , Molina-Linde Máximo , Palma-Ruiz Matilde , Toledo-Chavarri Ana 
Published: December 2022

IntroductionPatient involvement (PI) has become a key priority to the Spanish Network of Agencies for Assessing National Health System Technologies and Performance (RedETS). As part of the national strategy to promote PI, an interest group was created in 2017 to share knowledge, develop methodologies and standardize PI processes. The aim of this work is to analyze the main activities of the Patient Interest Group 5 years after its launch and to reflect on possible needs and challenges.MethodsNarrative description and an in-depth analysis of the main activities of the Interest Group from 2017 to the present.ResultsThe group is composed of HTA researchers from the 8 regional agencies in Spain and is supported by the Ministry of Health and the RedETS council. It currently has the participation of 26 researchers, organized into different working subgroups. The initial lines of work were the analysis of the situation, the development of procedures, and the initiation of training materials for patients on HTA. At present, the main projects are the development of metrics for evaluating the impact of patient participation, the development of procedural materials to promote methodological process standardizaton (e.g., a flowchart with the main process steps, checklists, templates), and the design and piloting of virtual training for patients in HTA. New lines include the analysis of the ethical challenges of PI and the feasibility of setting up an HTA patient registration system and a patient forum to facilitate participation. In addition, the interest group has promoted the exchange of relevant information for PI and the organization of capacity building activities.ConclusionsThe RedETS Patient Interest Group is encouraging the development of activities, reflection on collective experiences, and tools that facilitate PI in Spain. Among the main challenges are the need to ensure the quality and applicability of PI and to analyze the views of patients who have actively participated in HTAs.
OP94 Online Elicitation Of Personal Utility Functions (OPUF): A New Tool For Eliciting EQ-5D-5L Value Sets On The Societal-, Group-, Subgroup-, And Individual-Level
Authors: Schneider Paul , Brazier John , Devlin Nancy 
Published: December 2022

IntroductionThe ‘Online elicitation of Personal Utility Functions’ (OPUF) is a new method for valuing health states. It is based on compositional preference elicitation techniques. In contrast to established, decompositional techniques, such as time trade-off or discrete choice experiment (DCE), the OPUF approach does not require hundreds or thousands of respondents, but allows estimating utility functions for small (patient) groups and even on the individual level. The objective of this study was to generate and compare EQ-5D-5L value sets on the societal-, group-, subgroup-, and individual-level.MethodsA demonstration version of the EQ-5D-5L OPUF Tool is available at: https://eq5d5l.me. It broadly consists of three valuation steps: dimension weighting, level rating, and anchoring. Responses were combined on the individual level to construct personal utility functions. Every respondent also completed three conventional DCEs. Preferences were aggregated across individuals to estimate a societal and various group-level preference functions. We then assessed the heterogeneity of preferences between groups using descriptive statistics and k-means cluster analysis.ResultsA representative sample (n = 1,000) of the United Kingdom (UK) population was recruited through the prolific online platform. On average, it took participants about 7 minutes to complete the survey. Data of 874 respondents were included in the analysis. For each respondent, we constructed a personal EQ-5D-5L value set. The derived utility functions predicted respondents’ choices in DCE with an accuracy of 78 percent. On the societal level, the predicted values for the EQ-5D-5L health states ranged from -0.376 to 1. Health state preference varied greatly between groups. This was largely due to differences in the anchoring, while there was near consensus on the relative importance of the five EQ-5D dimensions between groups. Demographic characteristics explained only a small proportion of the variability.ConclusionsUsing the OPUF approach, we were not only able to estimate a new EQ-5D-5L value set for the UK, but also to examine the underlying individual preferences in an unprecedented level of detail.
OP97 Stakeholders’ Involvement In Brazilian Health Technology Assessment Incorporation In 2021: A Statistical Description Of Demands To The Brazilian National Committee For Health Technology Incorporation
Authors: Komoda Denis Satoshi , Silveira Correa Carlos Roberto , Visacri Marilia Berlofa , Santos Daniela 
Published: December 2022

IntroductionThe demands for incorporating or excluding health technologies within the Brazilian Public Health System (SUS) can be requested by different stakeholders, including public administration, pharmaceutical companies, specialists’ and patients’ organizations/associations. The Brazilian National Committee for Health Technology Incorporation (CONITEC), part of the Ministry of Health, is the responsible organ to evaluate these demands and emit recommendations. The aim of this study is to show an overview of stakeholders according to the type of technology under request.MethodsOn 26 November 2021, a search was performed at CONITEC website. Health Technology Assessment (HTA) incorporation reports from 1 January to 26 November 2021 were extracted. From these reports, data regarding the demanded technology (e.g., medications, diagnostic tests, etc.), demand (technology incorporation, exclusion, or alteration), pharmacological classification according with the Anatomical Therapeutic Chemical (ATC), and requester’ categorization (e.g., pharmaceutical companies, official administration organs, etc.).ResultsPreliminary results showed a total of 77 health technologies, in 63 HTA incorporation submissions, of which 87 percent (n = 67) were medications, and 9 percent (n = 6) were new medical procedures. Only one medical device, one vaccine and two diagnostic tests were requested. Technology incorporation accounted for 94.8 percent of the demands (73), and 4 demands of exclusion. Seventy-one percent of the requested medications were classified, according to first ATC coding level, within groups L (Antineoplastic and immunomodulating agents), N group (Nervous system), A group (Alimentary tract and metabolism), and R group (Respiratory system), accounting 28.3, 17.9, 8.9 and 8.9 percent, respectively. Regarding stakeholders, the Brazilian Ministry of Health and its associated departments were responsible for 57.1 percent of the demands, while pharmaceutical companies requested 37.6 percent of incorporations. Other requesters included the Federal Justice Department, patients’, and specialists’ organizations, summing up with only four demands.ConclusionsThese results present the numerical weight of stakeholders in the Brazilian HTA incorporation system, with special attention to Brazilian bureaucracy and pharmaceutical companies. Further analysis regarding association between demand and other variables such as budgetary impact, costs, and ICD-10, shall deepen our understanding of different stakeholders’ role in Brazilian HTAi.
PP22 A Lifecycle Approach To The Use Of Real-World Evidence In HTA Submissions And Resubmissions: A Decade’s Experience
Authors: Samaha Dima , Tavares Eliana , van Engen Anke , Szawara Paula 
Published: December 2022

IntroductionHealth technology assessment (HTA) bodies worldwide recognize the importance of real-world evidence (RWE) in addressing uncertainties around the effectiveness of new drugs at the time of launch and as part of resubmissions. We assessed the use and acceptability of RWE by analyzing HTA recommendations.MethodsWe analyzed 24,841 HTA reports, including original submissions, resubmissions, extensions of original indications, and renewals, published from January 2011 to October 2021 from more than 100 HTA agencies across 37 countries.ResultsOur analysis showed that 3,820 (15%) reports mentioned RWE. Between 2011 and 2021 there was an eight-fold increase in the use of RWE, from 4 percent in 2011 to 34 percent in 2021. RWE was most commonly included in HTAs in oncology (26%) and endocrine and metabolic diseases (13%). The main areas supported were effectiveness (40%), safety (38%), and epidemiology (35%). RWE supplemented evidence on survival and quality of life as well as resource utilization, proxy comparators, and utility. Based on an analysis of the 1,474 reports that mentioned RWE, effectiveness was mainly supported by cohort (22%) and observational studies (13%), safety was mainly derived from pharmacovigilance data (9%), and epidemiology data were collected from registries (23%). The top five HTA bodies mentioning RWE in their reports were from France, Germany, Poland, and the United Kingdom (n=2). RWE was most accepted when it supported safety and epidemiological considerations, and to a lesser extent when it was used for effectiveness aspects or understanding management pathways.ConclusionsThe inclusion and acceptability of RWE in HTA recommendations varies between HTAs according to their data requirements and assessment methods. While it is not always specified how RWE was considered, there is a clear tendency for its increased use and acceptability, albeit not in all areas. Greater use of and transparency around RWE are likely to continue as multiple RWE initiatives emerge globally.
PP24 Organizing Outpatient Parenteral Antibiotic Therapy: Lessons from Denmark
Authors: Loevschall Claus , Thomsen Anne Marie , Risoer Bettina , Groth Lotte , Lou Stina , Palmhoej Camilla 
Published: December 2022

IntroductionOutpatient Parenteral Antibiotic Therapy (OPAT) is a complex medical treatment used to treat patients with severe infections. OPAT is provisioned outside hospitals. There is wide variation in the use and organization of OPAT in Denmark. OPAT is increasingly used in Danish regions and municipalities, however, there is limited knowledge on the clinical, economic and organizational consequences of this technology. The purpose of the project was to establish an evidence base for decision-making prior to any further prioritization of OPAT as an alternative to intravenous antibiotic treatment in the hospital (IPAT). The HTA was produced at the request of the Health Directors in the Danish Regions to examine the consequences of using OPAT compared with IPAT.MethodsThe results were based on a systematic literature review and qualitative interviews with leaders (n=5), administrative employees (n=5) and health professionals (n=13) involved in the delivery of OPAT. Furthermore, a micro-costing analysis based on interviews with clinical experts was conducted.ResultsThe use of OPAT led to similar or better clinical results when compared with the use of IPAT. Current evidence supports OPAT as a safe model for intravenous antibiotic treatment. The organization of OPAT varied in Denmark as well as internationally. The selection of suitable patients for the different OPAT models was crucial for a successful treatment. Insight into patients’ understanding of the pros and cons of the technology indicated that most patients preferred treatment at home. In a Danish context the microeconomic analysis showed that different OPAT models generally led to a reduction in costs compared with IPAT.ConclusionsThe project contributes to practice and political decision making by identifying challenges and opportunities associated with OPAT. There is no one-size-fits-all solution. The choice of OPAT model must be based on careful clinical considerations. Coordination and communication across municipalities and hospitals is challenging. Reducing organizational complexity is necessary to achieve a more standardized practice.