bluuebunny/crossref_metadata_2025_split huggingface dataset:
OP90 Optimizing Health Technology Assessment And Appraisal For Orphan Drug Reimbursement: Experiences And Tools For Improvement
Authors: Blonda Alessandra , Denier Yvonne , Huys Isabelle , Simoens Steven 
Published: December 2022

IntroductionThe very high costs of orphan drugs, together with the uncertainties regarding their (cost-)effectiveness raise questions regarding the efficiency and legitimacy of their health technology assessment (HTA) and appraisal process. The aim of the present, qualitative study was to investigate how experts on the reimbursement of these treatments perceive the HTA and appraisal process in their country. Moreover, it aimed to provide specific conditions and practical recommendations for their improvement.MethodsTwenty-two European experts from 19 different countries were included in a qualitative survey and semi-structured interviews. Transcripts were analyzed using the qualitative data analysis software Nvivo. A grounded theory approach was adopted to develop a set of well-defined concepts from the cyclic analysis of the empirical data.ResultsFirst, analysis of the expert interviews yielded five good practices for an efficient HTA and appraisal of orphan drugs: a high level of transparency, patient involvement, a clear decision-making structure with room for flexibility, mechanisms to minimize bias and an explicit consideration of the opportunity cost. Meanwhile, participants highlighted several barriers to the overall process, such as a lack of trust between the different stakeholders and imbalances in negotiation power. In addition, the results allowed to identify a number of ‘contextual’ determinants that may undermine the legitimacy of the final decision, such as bias and the perverse effects of the orphan drug legislation. Drawing from the experts’ experiences, a toolkit was developed that includes an extensive number of specific recommendations (and conditions) for decision-makers to improve the legitimacy and efficiency of their HTA and appraisal of orphan drugs.ConclusionsOverall, the results showed that decision-makers should focus on limiting the impact of the contextual determinants rather than improving the methods included in the HTA. This will contribute to further legitimize reimbursement choices for orphan drugs towards the wider public.
OP95 Examining The Feasibility And Acceptability Of Valuing The Lebanese Version Of The SF-6D Using Standard Gamble
Authors: Kharroubi Samer , Beyh Yara , El Harake Marwa , Dawoud Dalia , Rowen Donna , Brazier John 
Published: December 2022

IntroductionThe SF-6D is a preference-based measure of health, derived from the SF-36 for economic evaluation. No value set exists for the SF-6D in Lebanon and other Arabic speaking countries in the Middle East. The aim of this study was to examine the feasibility and acceptability of using the standard gamble (SG) technique to generate preference-based values for the Arabic version of SF-6D in a Lebanese population.MethodsThe SF-6D was translated into Arabic using forward and backward translations. Forty-nine states defined by the SF-6D were selected using an orthogonal design and grouped into seven sets. A gender-occupation stratified sample of 126 Lebanese adults from the American University of Beirut were recruited to value seven states and the pits (worst) SF-6D health states using SG. Mean and individual level multivariate regression models were fitted to estimate preference weights for all SF-6D states. The quality of data and the predictive power of the models were compared with results from the United Kingdom (UK).ResultsAll respondents completed the interviews with 25 percent reporting that the SG task was difficult and 21 percent felt some degree of irritation or boredom. A total of 992 (98% out of 1,008 observations) SG valuations were useable for econometric modeling. There was no significant change in the test–retest values of 21 subjects. The mean absolute errors in the mean and individual level models were 0.036 and 0.050, respectively, both of which were lower than the UK results. The random effects model adequately predicts the SG values, with the worst state having a value of 0.322 compared to 0.271 in the UK.ConclusionsThis study confirmed that it was feasible and acceptable to generate preference values with the SG method for the Arabic SF-6D in a Lebanese population. This would be the first step towards developing SF-6D value set for Lebanon to be used in economic evaluation studies and to support resources allocation decisions.
OP96 Patient Reported Outcome Measure-Select App
Authors: Quik Elise 
Published: December 2022

IntroductionA patient/person-reported outcome measure (PROM) is a questionnaire that measures patient’s health self-assessment (PROs), such as pain/wellbeing. HTx is a Horizon 2020 project supported by the European Union lasting for 5 years from January 2019. The main aim of HTx is to create a framework for the Next Generation Health Technology Assessment (HTA) to support patient-centred, societally oriented, real-time decision-making on access to and reimbursement for health technologies (HTs) throughout Europe. Individual values, needs, and preferences should be taken into account when appraising HTs and in clinical decision-making. Better guidance is needed about how to develop and select generic and/or more specific outcome measures that are relevant to patients and fit-for-purpose. Our goal is to provide that guidance via the PROM toolbox and enable users to select PROMs by using this. The HTx project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement N° 825162.MethodsA framework for the assessment of PROMs was discussed with experts, tested for a subset of PROMs (Linnean menu) and resulted in the PROM-cycle. Multiple literature studies were performed on PROMs, their properties, and characteristics. The general population was studied as well as cancer, diabetes, multiple sclerosis, myelodysplastic syndrome, and (long-lasting) COVID-19. This resulted in an Excel database (PROM-overview) made available via a user-friendly web-application helping users to select PROMs: the PROM select app via https://www.prom-select.eu/proms.ResultsMultiple tools were developed and are available in the PROM toolbox starting with the PROM-guide, Literature review on the use of PROMs, PROM-cycle accompanied by the Linnean menu. In addition, PROM-links, The making of the PROM-overview & PROM-select app, PROM-overview, PROM-select app, its usability test report and PROM toolbox presentation can be found on https://www.zorginzicht.nl/ondersteuning/prom-toolbox-summary-in-english#what-is-the-prom-cycle.ConclusionsThe PROM toolbox guides users and helps them to select PROMs and trustworthy information and finding relevant scientific articles.
OP99 Participatory Approaches Used In Research And Development Of Medical Devices: A Comparison Of Focus groups, Interviews And Surveys
Authors: Woudstra Kas , Tummers Marcia , Klijn Catharina , Sondag Lotte , Schreuder Floris , Reuzel Rob , Rovers Maroeska 
Published: December 2022

IntroductionMulti-stakeholder engagement in the development and evaluation of medical devices is crucial for aligning devices with stakeholders’ views, needs, and values. Focus groups, interviews, and surveys are often used to involve stakeholders, but these methods have rarely been compared to analyse their relative merits. Therefore, we systematically compared these three methods in terms of themes, interaction, and feasibility.MethodsThe methods were compared in a case-study on surgery with a new endoscopic device for patients with intracerebral haemorrhage. We asked patients, relatives, healthcare professionals and decision-makers about their perspective on this device, and about their perceived quality of hospital care. We conducted the focus groups and interviews in one explorative and one interactive round. The comparison was made in terms of number and content of themes, who and how participants interact, and in terms of hours that needed to be worked by researchers to apply a method.ResultsWe enrolled 18 participants in the focus groups, 17 in the interviews and 43 in the survey. Focus groups generated 31 and 19 themes, and interviews 58 and 40 themes in the explorative and interactive round. Surveys generated 42 themes. Interviews produced various themes about the device that did not occur in the other methods. In the two rounds of the focus group, 13 and 42 percent of the interactions were directly between participants. In interview round one, 98 percent of the interactions were between the interviewer and participant, whereas 80 percent of the interactions in round two were discussions of other participants’ opinions. In focus groups participants were inclined to emphasise agreement, whereas the interviews generated more in-depth discussions. Interviews took three times as many hours as the focus groups and survey.ConclusionsMethods for multi-stakeholder involvement in device development vary considerably. These methodological differences should be taken into account when selecting a method for engaging stakeholders in the early stages of the lifecycle of a medical device.
OP103 Enhancing Legitimacy And Coherent Value Appraisal Across Interventions In Healthcare And Social Services: Strategy Of The Québec Agency
Authors: Goetghebeur Mireille , Wagner Monika , Ganache Isabelle , Demers-Payette Olivier , De Guise Michèle 
Published: December 2022

IntroductionThe Institut national d’excellence en santé et en services sociaux (INESSS) makes recommendations regarding introduction, coverage, optimal use or withdrawal of interventions in physical and mental health and social services to support fair and reasonable decisions. The objectives of this work were to develop a statement of principles and ethical foundations for a common appraisal framework across diverse interventions, to enhance legitimacy and coherence of evaluation practices, and develop reflective approaches throughout the institution to operationalize the principles.MethodsTo develop this statement, INESSS reviewed its practices across different units, surveyed the literature on innovative practices and the evolution of HTA, and undertook an extensive internal and external consultative process. The principles are used to develop reflective activities as part of a continuous improvement strategy.ResultsThe adopted approach to value appraisal considers the contributions of interventions to the Triple Aim of health and social services systems as well as their organizational and sociocultural feasibility and impacts (clinical, populational, economic, organizational, sociocultural dimensions). This approach is articulated around five principles including: (i) evaluating the most relevant objects and adapting evaluation modalities; (ii) mobilizing and integrating diverse types of knowledge; (iii) supporting multidimensional deliberation including diverse perspectives; (iv) developing fair and reasonable recommendations; (v) promoting value creation by supporting the implementation of recommendations and re-evaluation. Although all principles contribute to the legitimacy and credibility of the recommendations, which we aim to implement and consolidate through a set of activities, deliberation is an important part of the process that we are striving to improve. A first set of reflective activities are planned to support its operationalization, including: materials to promote a common understanding of the diverse aspects of the deliberation, reflective workshops on selected past projects, and sharing emerging reflections across INESSS units to further continuous improvement in operationalizing the principles.ConclusionsMoving forward, INESSS’s strategic intention is to mobilize its staff and collaborators to facilitate the rigorous, agile and coherent application of these principles.
OP105 Gaps, Challenges And Priorities For Neurodegenerative Disease Research And Health Technology Assessment
Authors: Salih Fatima , Bradshaw Angela , Killin Lewis , Coll Nina , Díaz Carlos , Diaz-Ponce Ana , Gove Dianne , Dawoud Dalia 
Published: December 2022

IntroductionThere are many challenges encountered in the health technology assessment (HTA) of treatments for the management of neurodegenerative diseases like Alzheimer’s disease. A recent example where such challenges were visible was the assessment of aducanumab, a medication developed for Alzheimer’s disease. As part of the Innovative Medicines Initiative (IMI) Neuronet project, a series of workshops were held to understand the gaps in neurodegenerative disease research and identify the key research priorities, from the perspective of different stakeholders, that can inform decision-making and enable timely access to effective and safe treatments.MethodsThree workshops were organised by Neuronet to consult with HTA and regulatory experts, people with dementia and their supporters, and academic and industry researchers. The first consultation was with the European Working Group of People with Dementia and their supporters, and their feedback was fed into the following two workshops which were conducted with HTA and regulatory experts, and with researchers. Discussions during the workshops were used to develop a summary of the gaps in research, and priorities from the perspective of each stakeholder group to be submitted to IMI to inform future research funding allocation.ResultsKey priorities that were highlighted from an HTA and regulatory perspective were the collection of longitudinal data to understand the natural history of the disease, the generation of data on long-term clinical outcomes and the use of trial endpoints that are relevant to clinical practice. There is also a need for disease-modifying treatments that is yet to be met in this disease area. Furthermore, the involvement of the public, particularly people with dementia and relevant organisations, in setting research priorities and informing study designs was highlighted as a priority by all stakeholders.ConclusionsThere are key priorities for future neurodegenerative research from an HTA and regulatory perspective to address current gaps and challenges. It is important to involve different stakeholders in priority setting and decision-making to produce relevant and satisfactory research outputs.
PP03 Early Assessment Of Video Consultations In Rehabilitation After Hand Injury
Authors: Støme Linn Nathalie , Vaksvik Tone , Wilhelmsen Christian Ringnes , Kværner Kari Jorunn 
Published: December 2022

IntroductionWith the aim of reducing patient travel and related costs, physiotherapists and occupational therapists at the Oslo University Hospital began offering video consultations to patients with hand and arm injuries in March 2020. A feasibility study was initiated to describe the first year of using video consultations in the rehabilitation of upper extremity injuries in children and adults, and to assess the acceptability of the service from the perspective of hand therapists. The secondary objective of the study was to investigate the economic effects of using video consultation for this patient group.MethodsThe therapists documented the individual consultations in an Excel spreadsheet. Utility and acceptability were analyzed based on the content of each video consultation. The therapists also registered the patient’s municipality in order to calculate costs related to travel, accommodation, and other costs related to in-person consultation. Utility was analyzed using an early economic model based on scenario analysis to compare the costs of video consultations with in-person consultations.ResultsBased on the content analysis from 89 consultations, video consultations were deemed acceptable by the therapists. The total travel distance from patients’ homes to the hospital was 20,190 km, as hand rehabilitation is a national service. The video consultations that replaced the consultations at the hospital were potentially more time efficient, reduced patients’ travel time and absence from home and work, and saved costs for the Oslo University Hospital and society.ConclusionsBased on early decision support provided by this study, adaptions were made to the delivery of video consultations to improve the cost effectiveness of the service. The findings from this study provided an indication of the potential value of the new service, which may be used for benchmarking purposes to ensure that it meets the needs of users, the health service, and society.
PP04 Supporting The Social Integration Of Persons With Brain Injury Using Psychoactive Substances: A Health Technology And Intervention Assessment
Authors: Maltais Christine , Djouini Akram , Messier Frédéric , Bélanger Karine , Lespérance Nadia , Deguise Élaine 
Published: December 2022

IntroductionPeople with a traumatic brain injury and who present with an unsafe or problematic substance use of psychoactive substances (PAS) face specific challenges in social integration resulting from the negative impacts of substance use on the benefits of rehabilitation services and treatments.MethodsA systematic review of grey and scientific literature was conducted. The selection and coding of the included interventions (according to their characteristics), as well as descriptive analyses, resulted in findings which describe the interventions under study. Grades of evidence strength were attributed to the findings based on a range of factors including the methodological characteristics of the studies. Findings resulting from the systematic review were contextualized with information collected from administrative databases, clinical program documentation, and experiential data obtained from rehabilitation professionals. Recommendations were elaborated based on the aggregated data sources and informal deliberations by a committee involving researchers and clinicians.ResultsOut of 5014 selected references, 14 studies examining 13 interventions which targeted social integration of the management of PAS were included. Interventions with the strongest evidence base are user-centered (i.e., support individual choices and personal autonomy), individualized (i.e., considered individual needs and characteristics), and educational (i.e., support the acquisition of strategies and abilities). The evidence base was considered “emerging” for interventions which are systemic in nature (i.e., relate to familial, social, cultural, educational or professional environments), interdisciplinary (i.e., involve clinicians with distinct training, competences and expertise), and ecological (i.e., which involve interventions in the client’s living settings). While additional research is necessary to better support their efficacy, these interventions exhibited promising effects on various outcomes.ConclusionsThe analysis and synthesis of three streams of information resulted in five clinical practice recommendations relating to integration to school and productive activities, psychological adaptation, integration in living settings and independence, social life and sense of belonging, and finally, use of PAS. Recommendations should help support clinicians in rehabilitation settings.
PP06 Clinical Effectiveness Of Fluticasone Furoate Nasal Spray For Perennial Allergic Rhinitis In Children: A Systematic Review And Meta-Analysis
Authors: Rivera Paola Andrea 
Published: December 2022

IntroductionAlthough previous studies suggest that fluticasone furoate nasal spray (FFNS) is superior to placebo in reducing symptoms in adolescents and adults with allergic rhinitis (AR), there is still uncertainty about its clinical effectiveness in the pediatric population. The aim of this study was to assess the clinical effectiveness of FFNS, compared with placebo, in reducing nasal symptoms in children with perennial AR.MethodsA systematic review was conducted of studies identified from the MEDLINE and Embase databases that were published up to January 2021. The population of interest was patients aged 2 to 12 years with perennial AR. Included studies were limited to randomized controlled trials (RCTs) comparing FFNS (110 μg once daily) with placebo. The outcomes of interest included the reflective Total Nasal Symptom Score (rTNSS) and safety. Meta-analyses were performed using RevMan 5.4. The Cohen’s guideline was used to assess the minimum clinically important difference for rTNSS; that is, if the pooled standardized mean difference (SMD) and the lower limit of the 95 percent confidence interval (CI) exceed -0.5, the treatment effect was considered clinically significant.ResultsThree RCTs (959 pediatric patients) were included. One study evaluated the short-term use of FFNS, one evaluated the long-term use of FFNS, and the third evaluated both the short- and long-term use of FFNS. FFNS produced a statistically significant reduction in rTNSS (SMD -0.35, 95% CI -0.63, -0.08; p<0.001) relative to placebo in the long-term treatment studies, but not in the short-term studies. However, since the mean reduction did not reach the minimum clinically important difference (SMD -0.5), these results were not considered clinically relevant. The safety outcomes for FFNS were similar to placebo.ConclusionsThe currently available evidence suggests that FFNS does not produce a meaningful clinical effect on nasal symptoms in children with perennial AR, compared with placebo. In the past decade, however, some guidelines have unequivocally endorsed this treatment.
PP07 Improving The Use Of Real-World Evidence In The Development Of NICE Guidance
Authors: Kent Seamus , Duffield Stephen , Rowark Shaun , Kincaid Lynne , Gupta Vandana , Sharma Manuj , Jonsson Pall 
Published: December 2022

IntroductionThe National Institute for Health and Care Excellence (NICE) is determined to make better use of health and social care data in the development of its guidance. Real-world data (RWD) has the potential to significantly improve our understanding of the value of new and existing health and social care interventions. RWD is already widely used to characterize populations, interventions, and outcomes and to populate economic models, but its use in estimating the effects of interventions remains limited, especially for medicines. Key barriers to its greater use in this context include limited transparency around studies, sometimes a lack of confidence in their integrity, and methodological concerns around how studies have addressed major sources of potential bias.MethodsThis abstract focuses on the real-world evidence (RWE) framework developed by NICE to support its ambitions. The framework was developed in an iterative way based on: reviews of best practice approaches to the conduct and assessment of real-world evidence studies; case studies; and workshops with key external stakeholders. The initial version of this living framework focuses on de novo RWE studies using individual patient data.ResultsThe RWE framework consists of an overarching research governance framework which describes expectations around the planning, conduct, and reporting of RWE studies across uses of real-world data. Uses are categorized by risk according to their importance to decision-making, the impact of decisions on patient and system outcomes, and their complexity as proxied by risk of bias. Studies of the effects of interventions on patient health and system outcomes are considered the highest risk. The research governance framework is supported by a tool to aid assessment of data suitability for its intended application, and detailed guidance on the conduct and reporting of comparative effect studies using RWD, following the target trial approach.ConclusionsThe RWE Framework underpins NICE’s ambitions to make better use of RWD in its guidance and is intended to improve the quality and utility of RWE studies submitted to NICE enabling more consistent and appropriate evaluation.