bluuebunny/crossref_metadata_2025 huggingface dataset:
OP17 Robotic Versus Conventional Surgery: An Overview Of Systematic Reviews For Clinical Effectiveness With Quality Assessment Of Current Evidence
Authors: Lai Tzujung , Bouttell Janet , Boyd Kathleen
Published: December 2022

Introduction

Robot-assisted surgery (RAS) is being adopted rapidly internationally across a wide range of surgical procedures. Although a great deal of evidence of the clinical effectiveness of RAS has been generated, it is possible that the evidence base is not complete or persuasive in some areas where adoption is being considered. This review seeks to summarize systematic reviews (SRs) undertaken to date to illustrate the weight of evidence across specialties. We then take an in depth look at the quality of evidence across several indications where the adoption of RAS is currently underway.

Methods

A comprehensive literature search was conducted using Ovid Medline, Embase, and Cochrane Central Register of Systematic Reviews from January 2017 to April 2021 for SRs describing clinical effectiveness outcomes. The body of evidence was mapped across all specialties. For a selected number of indications currently under consideration in Scotland, results were comparatively summarized, and the quality of the reviews was evaluated with the AMSTAR-2 tool.

Results

A total of 451 SRs were found. Most were in urology (n = 130) where RAS is well established, followed by colorectal (n = 63), hepatology (n = 58), and gynecology (n = 41). From within these latter three specialties, we selected six indications in which RAS is currently being considered for adoption in Scotland for in depth review (colorectal cancer surgery, hysterectomy, gastrointestinal oncological resection, hepatic, pancreatic and biliary surgery). Evidence for the clinical effectiveness of RAS versus conventional laparoscopic surgery is mixed across indications and outcomes. In colorectal cancer surgery, for example, evidence was positive for conversion rate and neutral for length of hospital stays, blood loss and postoperative complication and negative for operative time. For hysterectomy, evidence was positive for the length of hospital stays and neutral for operative time, blood loss, conversion rate and postoperative complication. The quality of the included reviews was judged to be critically low.

Conclusions

The currently available evidence of clinical effectiveness is mixed across indications and of low quality.
OP18 Clinical Effectiveness And Safety Of Implantable Bulking Agents For Fecal Incontinence: A Systematic Review
Authors: Gassner Lucia , Wild Claudia , Walter Melanie
Published: December 2022

Introduction

The purpose of this systematic review is to evaluate whether implantable versus injectable bulking agents (second-line therapies) are equal/superior in terms of effectiveness (severity, quality of life [QoL], sustainability) and safety (adverse events) for fecal incontinence (FI).

Methods

A systematic review was conducted and five databases were searched (Medline via Ovid, Embase, Cochrane Library, University of York Centre for Reviews and Dissemination, and International Network of Agencies for Health Technology Assessment database). In-/exclusion criteria were predefined according to the PICOS scheme. The Institute of Health Economics risk of bias (RoB) tool assessed studies’ internal validity. According to the Grading of Recommendations, Assessment, Development and Evaluation (GRADE) approach, the strength of evidence for safety outcomes was rated. A qualitative synthesis of the evidence was used to analyse the data.

Results

Six prospective uncontrolled trials (143 patients) were included. The evidence consists of six prospective single-arm, before-after studies fulfilling the inclusion criteria for assessing clinical effectiveness and safety for implantable bulking agents. FI severity (Cleveland Clinic FI Score) statistically significantly improved to three months (p<0.01) and six months (p<0.05) follow-up (five studies). Improvements in severity sustainability were reported after 12, 14 (p<0.01), and 36 (p<0.0001) months postoperatively. Improved disease-related QoL (FI QoL Score) was found (p<0.05) 12 months after surgery, and statistically significant improvements in QoL’s sustainability after 12 months (one study).

Procedure-related adverse events (n=3) occurred, where prostheses extruded during surgery, and anal discomfort/pain was felt (n=11). Device-related adverse events, i.e., prostheses’ dislodgement (n=31) and removed/extruded prostheses (n=3), occurred. Studies were judged with moderate/high RoB. The strength of evidence for safety was judged to be very low.

Conclusions

Implantable bulking agents might be an effective and safe minimally invasive option in FI treatment if conservative therapies fail. FI severity significantly improved, but not QoL, which needs to be explored in further studies. Due to the uncontrolled nature of the case series, comparative studies need to be awaited.
OP21 A Critical Review Of Existing Health Inequality And Health Inequity Frameworks In Evidence Synthesis
Authors: Kunonga Patience , Hanratty Barbara , Bower Pete , Craig Dawn
Published: December 2022

Introduction

In recent years, there has been a growing recognition that health equity and health inequalities should be a consideration in all aspects of research. Since the Commission on Social Determinants of Health by the World Health Organization was established in 2005, there has been a growing interest in tackling systemic differences in health outcomes, including expanding the scope to health research including evidence synthesis and health technology assessments (HTA). This analysis aims to identify health inequality and health inequity frameworks that exist to help structure and plan research methods in evidence synthesis.

Methods

A critical analysis of the existing frameworks used in evidence synthesis to address health inequality and/or inequity was undertaken. Comprehensive, systematic searching of seven social science electronic databases and grey literature was undertaken based on the Behavior/phenomenon of interest, Health context and Model/Theory (BeHEMoTh) model, from 1990 to May 2022 to identify all relevant studies. A narrative synthesis approach was used to critically appraise the existing frameworks.

Results

Sixty-two reviews published between 2008 and 2022 reporting on using a framework to stratify health opportunities and outcomes met the inclusion criteria. Frameworks identified included the PROGRESS (place of residence, race or ethnicity, occupation, gender, religion, educational level, socioeconomic status, and social capital), PROGRESS-Plus (plus age, disability and sexual orientation) and Preferred Reporting Items for Systematic Reviews and Mata Analysis (PRISMA) – Equity checklist.

Conclusions

Currently, there does not seem to be consensus in how evidence of inequality or inequity in evidence synthesis or HTA are reported. As research interests in health inequality and inequity continue to grow, there is a need to develop a framework that provides an in-depth understanding of how inequalities in health and inequities in health should be considered within evidence synthesis and HTA. This will allow researchers to analyze not just the effects of interventions, but also how healthcare outcomes are impacted by inequalities or inequities.
OP30

M

odel for

AS

sessing The Value Of

AI

In Medical Imaging (MAS-AI)
Authors: Fasterholdt Iben , Kjølhede Tue , Naghavi-Behzad Mohammad , Schmidt Thomas , Rautalammi Quinnie , Kidholm Kristian , Hildebrandt Malene , Gerdes Anne , Barkler Astrid , Rasmussen Benjamin
Published: December 2022

Introduction

Artificial intelligence (AI) is seen as one of the major disrupting forces in the future healthcare system. However, assessment of the value of these new technologies is still unclear and no agreed international HTA-based guideline exists. Therefore, a Model for ASsessing the value of AI (MAS-AI) in medical imaging was developed by a multidisciplinary group of experts and patient representatives.

Methods

The MAS-AI guideline is based on four steps. First a literature review of existing guides, evaluations, and assessments of the value of AI in the field of medical imaging (5,890 studies were assessed with 86 studies included in the scoping review). Next, interviews with leading researchers in AI in Denmark. The third step was two workshops where decision-makers, patient organizations and researchers discussed crucial topics when evaluating AI. Between workshops, the multidisciplinary team revised the model according to comments from workshop-participants. Last step is a validation workshop in Canada.

Results

The MAS-AI guideline has three parts. There are two steps covering nine domains and then advises for the evaluation process. Step 1 contains a description of patients, how the AI-model was developed, and initial ethical and legal considerations. Finishing the four domains in Step 1 is a prerequisite for moving to step 2. In step 2, a multidisciplinary assessment of outcomes of the AI-application is done for the five remaining domains: safety, clinical aspects, economics, organizational aspects and patient aspects. The last part, is five advices to facilitate a good evaluation process.

Conclusions

We have developed an HTA based framework to support the prospective phase while introducing novel AI technologies into healthcare in medical imaging. MAS-AI can assist HTA organizations (and companies) in selecting the relevant domains and outcome measures in the assessment of AI applications. It is important to ensure uniform and valid decisions regarding the adoption of AI technology with a structured process and tool. MAS-AI can help support these decisions and provide greater transparency for all parties involved.
OP31 Assessment Of AI Supported Health Technologies - How To Move Forward?
Authors: Daugbjerg Signe , Di Bidino Rossella , Cicchetti Americo
Published: December 2022

Introduction

Artificial intelligence (AI)-supported technologies are rapidly developing and have the potential to improve healthcare quality at reduced cost. However, few examples exist of successfully deployed AI-technologies in a real-world context that have been adequately assessed. Therefore, the objective of this research is to: (i) identify existing health technology assessment (HTA) methods developed or adapted to assess AI-supported health technologies, (ii) identify new assessment topics or domains relevant for AI-technology uptake, and (iii) take the first step in developing a framework applicable for new challenges that emerge with the introduction of AI.

Methods

A systematic literature review of studies describing methods or frameworks to assess AI-supported health technologies was performed on PubMed from January 2010 until February 2021. Furthermore, a web page search of international HTA agencies and international organizations such as the World Health Organization, Organziation for Economic and Co-ordination and Development, and the European Commission was performed to identify important aspects to consider when implementing and assessing AI technologies.

Results

No assessment frameworks for AI technologies were identified from the systematic literature review or web page searches of international HTA agencies. Reports from international organizations highlight limitations or inability of most AI technologies to ‘explain’ their decision-making process (black box issue), leading to lack of trust in the technology that affects its adoption. It is recommended to put more emphasis on assessing transparency and ‘explainability’ of the AI solution as well as aspects of safety, ethical, legal, and social issues related to implementation and the development/training phase of the AI technology.

Conclusions

The results from this study uncover key gaps in frameworks posed for performing a systematic and holistic assessment of AI in a real-world context of health care. However, valuable information on relevant assessment aspects for AI-supported technologies have been identified.

The results will form the basis for the development of a framework to assist decision-makers in assessing AI-supported technologies in a holistic manner for a responsible deployment – the HTA AI Framework.
OP36 A Lifecycle Approach In Evaluating Medical Technologies: Insights From The National Institute For Health And Care Excellence Guidance Review Process
Authors: Maslyankov Ivan , Wang Ying-Ying , Bowie Dionne , Heaney Joanne , Chalkidou Anastasia
Published: December 2022

Introduction

Health Technology Reassessment (HTR) is emerging, as the focus of health technology assessment agencies shifts from traditional methods of technology adoption to managing technologies throughout their lifecycle. The National Institute for Health and Care Excellence (NICE) evaluates devices, digital and diagnostic technologies by producing medical technologies guidance, which could recommend for adoption, no adoption, or further research. The desire to move to a lifecycle approach in the evaluation of medical technologies is reflected in the guidance review process, which involves review of the technology every three years or upon notification of significant new evidence. The outcomes of the guidance review can be to amend, update, withdraw, or leave the guidance unchanged.

Methods

Information on all technologies which have undergone guidance review since the commencement of the process was collected, including the recommendation before and after review and the basis for this recommendation. The proportion of guidances which were not changed, amended, updated, and withdrawn was calculated and the trends, including the bases for recommendation change were analyzed.

Results

In total, 34 medical technology guidance reviews have been performed. During the process, 15 (44%) were amended to reflect minor changes in the economic or clinical evidence, which did not change the recommendation. Ten (29%) were not changed, while three (9%) were updated respectively. Three (9%) were withdrawn. Another three (9%) represent special cases, which entered guidance review, but were paused due to external reasons. Among the guidances that progressed to update, two out of three had a cost increase, whereas one was broadened to reflect evidence for a larger population.

Conclusions

HTR is an important mechanism to improve patient care and system efficiency. In NICE’s evaluation of medical technologies, changes in the recommendation stemmed from changes in the technology’s (or standard care’s) cost, the evidence for clinical effectiveness, or the safety profile.
OP41 Facilitating Dialogue Of Real-World Evidence Use In Health Technology Assessment: Taxonomy Of Question/Data Source Pairings To Support A Registry Of Studies
Authors: Akehurst Ron L , Murphy Linda A , Mestre-Ferrandiz Jorge , Solà-Morales Oriol , de Pouvourville Gérard , Cunningham David , Corry Sorcha , Franklin Matthew , Chapman Ann-Marie
Published: December 2022

Introduction

This paper reports the results of the collaboration within the European initiative of new Reimbursement and aCCess Approaches (EUreccA) which is concerned with the use of real-world evidence (RWE) in health technology assessment (HTA) decision-making. The work grew from the observation of a large, very experienced group of HTA practitioners which found that the use of RWE varied depending on the type of question asked and the particulars of the data source(s) used. We set out to examine how RWE is used in HTA decision-making and to make proposals on its facilitation.

Methods

Literature reviews covering earlier reviews of RWE use, academic papers, and HTA agency websites were combined with case studies involving interviews with decision-makers in four countries (England, France, Italy, Sweden) to identify the circumstances of breakdown of RWE use and to build a categorization of the uses of RWE and associated difficulties. This evidence supported the creation of a taxonomy of pairings of data sources and the questions they were used to address. The face validity of the approach was tested at an advisory board of senior HTA practitioners.

Results

In total, 27 questions were identified and 10 types of data source, giving 270 pairings. These pairings were linked to relevant methods guidance and to examples of their use, itemizing HTA issues and decisions made. Reports are being prepared for publication, covering the detail of the methods of the literature searches; methods of the country case studies; a description of the taxonomy; and guidance on governance.

Conclusions

When using RWE in HTA decision-making, the detail of the particular data sources and question addressed matter. Recently, both the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) and the Real-World Transparency Initiative have argued for a registry of the uses of RWE. The work described here offers a starting classification of the material that should be held in such a registry, and which in itself could be developed by the stakeholders, both agencies and companies, that use it, furthering trust and confidence.
OP50 Health Equity Considerations In HTA: A Case Example Of Prenatal Screening
Authors: Shaw Beth , King Valerie , Morse Josiah , Robalino Shannon , Harrod Curtis
Published: December 2022

Introduction

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.

Methods

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.

Results

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.

Conclusions

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.
OP57 The Identification Of Technological Innovations To Address The Challenge Of Antimicrobial Resistance Using Horizon Scanning Approaches
Authors: Barrass Lucy , Roberts Abigail , Arisa Oluwatomi , Robertson Emily , Oliver Aoife , Sharma Oshin , Kinnersley Janet , Craig Dawn , Oyewole Anne
Published: December 2022

Introduction

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.

Methods

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.

Results

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).

Conclusions

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.
OP59 The Tunisian Guidelines For Pharmacoeconomic Analysis: What We Need To Know
Authors: Harzallah Nabil , Chemli Jaafar , Grati Hela , Jebali Marie , Jameleddine Mouna , Hamouda Chokri
Published: December 2022

Introduction

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.

Methods

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

Results

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.

Conclusions

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.
OP76 “Thunderbirds Are Go!” Rapid Response HTA Outputs For COVID-19
Authors: Hyseni Lirije , Islam Elizabeth , Bowie Dionne , Dimmock Paul
Published: December 2022

Introduction

The COVID-19 pandemic has highlighted the need for rapid assessment of potential health technologies that can improve health outcomes in COVID-19 patients, as well as helping pressurized health service provision. Medical technologies play a key role in the COVID-19 pandemic, especially diagnostic tests and respiratory technologies. This study evaluates the rapid response work that the medical technology evaluation programme (MTEP) at the National Institute for Health and Care Excellence (NICE) has done in response to the COVID-19 pandemic.

Methods

Companies routinely submit medical technologies for evaluation by NICE through HealthTech Connect, which is an online portal for devices, diagnostics and digital technologies intended for use in the NHS or wider United Kingdom health and care system. During the COVID-19 pandemic, companies were able to use a designated email address if they perceived their technology may benefit the healthcare system regarding the COVID-19 pandemic. This new system bypassed the usual full registration and data submission. All technologies were reviewed that were submitted via HealthTech connect and email between March 2020 and June 2021.

Results

During this period, 20 technologies were submitted to MTEP. Most of these technologies were submitted via email. These technologies consisted of a mix of digital, diagnostic, and respiratory technologies. Seven technologies were selected for a rapid COVID-19 MedTech innovation briefing (MIB), with one specifically addressing issues around waiting lists because of knock-on effects of COVID-19 restricting normal clinical work. A further six technologies were not selected because of limited evidence, while one was not selected because it was not perceived as innovative. The other five technologies were progressed as normal MIBs as there was not enough evidence of potential benefits related to COVID-19 to expedite to a rapid COVID-19 MIB. In total, two technologies were selected for medical technology guidance (myCOPD and Anaconda) and are currently in development.

Conclusions

MTEP has responded to the COVID-19 pandemic by prioritising and producing rapid COVID-19 MIBs on technologies to improve health and social care.
OP89 The CE-Signal, A New Simplified Health Technology Assessment Method To Determine Whether Interventions Are Cost-Effective
Authors: Huisman Atse , Knies Saskia , Timmers Lonneke , Enzing Joost , Vreman Rick , Vrinzen Terry , Tran Ly
Published: December 2022

Introduction

Conducting a cost-effectiveness analysis (CEA) is resource consuming, and therefore the Dutch National Health Care Institute (ZIN) only performs those for interventions with a high budget impact. Sometimes, cost-effectiveness (CE) estimates are clearly below or far above reference values, which makes full cost-effectiveness assessments less vital. The objective of this study was to develop an efficient and simplified method to identify interventions that are clearly cost-(in)effective.

Methods

The method makes use of headroom analysis. Several HTA experts and other relevant stakeholders have been asked to provide feedback on a preliminary version of the CE signal.

Results

The method consists of five steps. In the first step (i) the relevant willingness-to-pay threshold is determined. Reference values are used by ZIN for the maximum willingness-to-pay per incremental quality-adjusted life year (QALY), depending on burden of disease.

In next step (ii) the health gain that can realistically be obtained with the new treatment is estimated. Hereby the effect of the intervention on the clinical outcomes, quality of life and gained life years is determined to estimate the number of QALYs gained, including uncertainty. Then (iii) the societal cost maximum (SCM) of the new treatment is calculated by multiplying step 2 with step 1. In step four (iv) the incremental treatment costs are estimated looking at both the costs and savings for both treatments options for the average patient. In the final step (v) the incremental treatment costs are compared to the SCM to determine if the intervention is probably cost effective, probably not cost-effective or if a conclusion cannot be drawn.

Conclusions

This method has proven to be feasible and could be a valuable addition to the current cost-effectiveness assessment toolbox. The CE-signal is being validated against performing a full cost-effectiveness analysis.
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

Introduction

The 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.

Methods

Twenty-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.

Results

First, 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.

Conclusions

Overall, 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

Introduction

The 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.

Methods

The 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).

Results

All 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.

Conclusions

This 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

Introduction

A 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.

Methods

A 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.

Results

Multiple 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.

Conclusions

The 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

Introduction

Multi-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.

Methods

The 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.

Results

We 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.

Conclusions

Methods 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

Introduction

The 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.

Methods

To 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.

Results

The 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.

Conclusions

Moving 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

Introduction

There 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.

Methods

Three 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.

Results

Key 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.

Conclusions

There 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

Introduction

With 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.

Methods

The 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.

Results

Based 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.

Conclusions

Based 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.