bluuebunny/crossref_metadata_2025_split huggingface dataset:
OP11 Differences And Similarities In Past Health Technology Assessments In Beneluxa Initiative Countries
Authors: Vreman Rick , van Hoof Daan , Adams Roisin , van de Casteele Marc , Nachtnebel Anna , Timmers Lonneke 
Published: December 2022

IntroductionConducting joint health technology assessments (HTA) is one of the main goals of the Beneluxa Initiative. To strengthen this collaboration, this study aimed to assess similarities and differences between past assessments of Beneluxa Initiative member countries (Austria, Belgium, Ireland and the Netherlands).MethodsA retrospective comparative analysis was performed that investigated the similarities and differences in drug assessments in the period 2016 to 2020 in (i) the number and type of assessed indications; (ii) the conclusions within assessments performed by at least two member countries; and (iii) the main arguments leading to the conclusions through a qualitative analysis of selected cases, looking into the patient population, the intervention, comparator, outcome, timing, and included evidence.ResultsThe scope of HTA differs between the countries, with Belgium and Ireland assessing most, the Netherlands focusing on drugs above a budget impact threshold and Austria on outpatient drugs. Furthermore, indications might slightly differ between countries. Therefore, only 44 (10%) of the 444 included drug-indication combinations were assessed through a full HTA by all four countries. Between any pair of countries, the overlap was higher, from 63 (Austria-the Netherlands) to 188 (Belgium-Ireland). Added benefit conclusions matched exactly in 62 to 76 percent of the indications, depending on the compared countries. In the remaining cases, often a difference of one added benefit level was observed (e.g., higher versus equal relative effect). Contradictory outcomes were very rare. Differences were observed with regards to whether a cost-effectiveness analysis was performed. When assessing the underlying arguments within the reports for nine cases with different outcomes, it became clear that organizations agree on almost all aspects, and that differences are mostly attributable to slight differences in weighing of some aspects and uncertainties.ConclusionsOverall, which indications are assessed differs, but for those indications that are assessed by multiple member countries, considerations and assessment outcomes are similar.
OP16 Learning From Engagement With Human Immunodeficiency Virus Community Organizations In The Health Technology Assessment Lifecycle
Authors: Marsden Laura , Livingstone Heidi , Tonkinson Mandy 
Published: December 2022

IntroductionInvolving patients and community organizations in the health technology assessment (HTA) lifecycle is a core principle at the National Institute for Health and Care Excellence (NICE) and helps build public confidence in the healthcare decision-making. From 2019 to2021 NICE assessed a HIV-1 medicine for the first time. This presented new opportunities and challenges for engaging with Human Immunodeficiency Virus (HIV) community organizations who hadn’t participated in a NICE HTA before. To understand their experience, we collected feedback on the impact and experience of community involvement throughout the assessment.MethodsWe used a mixed-method approach using a survey and qualitative feedback from the committee lay member, community experts, and the community organizations. An impact survey was sent to key committee members, the NICE technical team and Associate Director. It included Likert scale questions and open text boxes to capture both quantitative and qualitative data. Additionally, qualitative feedback was gathered throughout the assessment’s lifecycle from the NICE team and the community stakeholders.ResultsResults from the impact survey showed that the community input: had significant impact on the evaluation (100% (n=5)); helped interpret the other evidence and information (80% (n=4)); provided new evidence (60% (n=3)); and, was consistent with the other evidence (40% (n=2)). Examples of impact included highlighting the population heterogeneity, stigma, side effects and the effects of frequent clinic attendance. The key feedback from the qualitative data from the community organizations and experts in terms of their experience were: early support and support throughout from the NICE team; NICE’s flexibility in involving them; and, meaningful inclusion of their evidence in the committee slides.ConclusionsBoth the NICE committee and the community stakeholders recognized that the community input was valued and had an impact on the decision-making. To ensure meaningful community engagement, support and flexibility from NICE were required throughout the assessment. This level of engagement will be adopted in future for community organizations new to medicines HTAs at NICE.
OP19 Comparative Effectiveness Of Common Treatment Options For Benign Prostatic Hyperplasia: A Systematic Review And Network Meta-Analysis
Authors: Rojanasarot Sirikan , Neeser Kurt , Fu Shuai , Bhattacharyya Samir , McVary Kevin 
Published: December 2022

IntroductionTreatment options for men with moderate-to-severe lower urinary tract symptoms due to benign prostatic hyperplasia (BPH) include medical therapy, minimally invasive surgical therapies (MISTs), and invasive surgical procedures. While these treatments are recommended by American Urological Association Guidelines, they have different clinical profiles impacting both efficacy and durability outcomes. Using an indirect comparison approach, this study assessed the clinical effects of combination therapy (CT) using alpha-blockers and 5-alpha reductase inhibitors, two emerging MISTs (prostatic urethral lift [PUL] and water vapor thermal therapy [WVTT]), and two invasive surgical procedures (photoselective vaporization of the prostate [PVP] and transurethral resection of the prostate [TURP]).MethodsA systematic search of Medline, Embase, Cochrane Library, and relevant health technology assessment (HTA) databases was conducted to identify randomized and non-randomized clinical trials of the five treatments published prior to December 2020. Trials were included if they reported changes in International Prostate Symptom Score (IPSS) and retreatment rates, without any country or language restrictions. A random-effects network meta-analysis (NMA) with an aggregate regression model was performed to account for the baseline BPH severity and characteristic differences among men from the different trials.ResultsA total of 237 of 3,104 retrieved abstracts were included for full-text review. Of these, 16 randomized and four non-randomized clinical trials were included in the NMA. The random-effects NMA showed among medical and minimally invasive therapies, WVTT had the greatest one-year IPSS improvement (-∆11.7), followed by PUL (-∆10.4) and CT (-∆10.3). The one-year IPSS improvement for TURP and PVP was comparable (-∆14.1 vs. -∆13.8, respectively; p-value=0.675). The one-year retreatment rates were lowest for WVTT (3.0%), followed by CT (3.6%), TURP (6.3%), PVP (7.8%), and PUL (8.0%).ConclusionsWVTT provided greater clinical and durability benefits compared to other less invasive treatment options for men with BPH. Given NMA is increasingly used in HTA processes, this study provided systematically synthesized evidence that could facilitate decision-makers in determining new technology coverage decisions globally.
OP22 Using Threshold Analysis To Guide Searches For Additional Sources Of Evidence
Authors: Anwer Sumayya , Dias Sofia , Simmonds Mark , South Emily , Wade Ros , Eastwood Alison 
Published: December 2022

IntroductionThreshold analysis is a novel statistical approach which can be used to investigate which direct comparisons in a network meta-analysis (NMA) have estimated relative effects that may not be robust to changes in the evidence, either due to possible bias, sampling variation, or relevance.MethodsIn a health technology assessment of the clinical effectiveness of ablative and non-invasive therapies for patients with early hepatocellular carcinoma (HCC), we conducted a threshold analysis to identify treatment comparisons that would be sensitive to changes in the randomized controlled trial (RCT) evidence used in the NMAs, potentially leading to a change in the recommended treatment. The results of the threshold analysis were used to guide a targeted systematic review of high-quality, non-randomized, prospective comparative studies that could strengthen the evidence for those comparisons identified as sensitive to change.ResultsWe conducted NMAs of RCT evidence for four outcomes: overall survival (16 RCTs), progression-free survival (6 RCTs), overall recurrence (7 RCTs), and local recurrence (10 RCTs). The results of the NMAs displayed a high level of uncertainty, attributable to the sparse nature of the network, characterised by interventions being mainly compared in small trials. A targeted systematic review was conducted on relevant interventions that were identified as being sensitive to changes in evidence by the threshold analysis. The studies identified in this review were incorporated into a second NMA to support the RCT evidence.ConclusionsThreshold analysis has been typically used as a tool to assess how robust comparisons in an NMA are to additional sources of evidence, but it can also be used to guide the search for additional non-randomized evidence when the available RCT evidence is sparse.
OP28 Health Technology Assessment: A Situation Analysis Of Zimbabwe
Authors: Dzingirai Blessing , Matyanga Celia , Manyau Pinky , Postma Maarten Jacubos , van Hulst Marinus , Mafirakureva Nyashadzaishe 
Published: December 2022

IntroductionWith ever increasing burden of disease and limited resources, health technology assessment (HTA) is required for efficient resources allocation and priority setting in healthcare. The objective of this study was to establish the baseline HTA evidence generation and use in Zimbabwe.MethodsIn 2019, we convened a stakeholder workshop on HTA at the University of Zimbabwe. Presentations on HTA processes, current healthcare reimbursement model, priority setting in the Ministry of Health and selection of medicines into the treatment guidelines, were done by the experts. We adapted the Health Intervention and Technology Assessment Program questionnaire for situational analysis of HTA introduction at national level and administered it among the workshop participants. We report the baseline information on HTA situation, the need, demand and supply of HTA in Zimbabwe obtained from the presentations and responses from workshop participants.ResultsA total of 33 participants attended the workshop. Participants indicated that there is no formal HTA agency or process in Zimbabwe. The selection of medicines into treatment guideline is determined by disease burden, safety, efficacy and cost data, and it is done by a group of experts. The Association of Healthcare Funders of Zimbabwe (AFHOZ) reported that private healthcare funders use resource-based relative value scale system to determine tariffs and reimbursement levels. The regulator requires safety, efficacy and product quality data for the registration of medicines. Transparency in decision-making, registration of heath technology and formulation of essential medicines and treatment guidelines were reported as the major needs of HTA. The major users of HTA outputs were reported as medicines regulator, AFHOZ and Ministry of Health. Key suppliers of HTA evidence are academic and clinical research institutions and healthcare workers. Lack of training in health economics was cited as the major challenge to supply of HTA evidence.ConclusionsThere is a need to institute a formal, systematic and transparent processes of determining value of health technologies.
OP29 Lifecycle Assessment Of Machine Learning-Derived Early Warning System. An Early Economic Evaluation Of An Intraoperative Hypotension Prediction Index
Authors: Piaggio Davide , Federici Carlo , Di Bidino Rossella , Bracale Umberto , Pecchia Leandro 
Published: December 2022

IntroductionAn iterative, life-cycle approach to the evaluation of healthcare technologies requires that clinical and economic evidence is collected since the initial stages of diffusion. Nevertheless, early cost-effectiveness models are challenging mainly due to the difficulties in estimating model parameters and faithfully characterizing parameter uncertainty. This is especially true with AI-based diagnostics, where attribution of effects on costs and patient-relevant outcomes is more challenging. Empirical applications of early-models are useful to identify the main challenges of iterative modelling and provide recommendations on best-practices. Here, we reported on a case study on a machine learning-derived hypotension predictive index (HPI), that predicts the onset of intraoperative hypotension and trigger corrective measures.MethodsA hybrid decision-tree/Markov model was developed comparing an HPI-based intervention protocol to standard-of-care intervention protocol during gynecological procedures. A short-term component of the model was populated using data from individual patients collected at one hospital in Italy. An historical control group was also defined using propensity score matching. Long-term costs and consequences of HPI were modelled using secondary data. A probabilistic version of the headroom approach was used to determine the maximum achievable price of HPI based on available evidence. Value of Information analysis was also conducted to identify the parameters that contribute the most to the overall uncertainty, and to identify optimal future study designs. Extensive deterministic and probabilistic sensitivity analyses were conducted to characterize the uncertainty over the cost-effectiveness of HPI.ResultsThe preliminary results of the analysis suggest that HPI has potential to improve patients’ outcomes and generate efficiency gains by reducing hypotension events and permanent complications, such as acute kidney injury. The link between reduction in hypotension and the rate of complications, or the long-term effects on healthcare costs and patients’ quality of life are the parameters that contribute the most to model uncertainty.ConclusionsEarly cost-effectiveness models are a valuable tool to inform further product development and evidence requirements, but characterization of uncertainty and transparency in modelling assumptions are key.
OP32 A Multistep Multistakeholder Priority Setting Exercise For Fecal Incontinence
Authors: O’connor Nicole , Thomson Katie , Dangova Kim , Gill Sean , Wallace Sheila , Jackson Sara , Pearson Fiona 
Published: December 2022

IntroductionFecal incontinence (FI) is the involuntary loss of feces and can affect up to 17 percent of community dwelling individuals, rising to 40 percent of older people in residential care homes. There is limited up-to-date evidence which formally set research priorities addressing FI. This project aimed to identify research topics of highest importance to key FI stakeholders.MethodsAn evidence gap map was produced incorporating three streams of evidence coded against predefined topic domains. The evidence streams included: emerging evidence identified through horizon scanning; existing evidence identified through systematic searches of bibliographic databases; and key FI stakeholder insights collected through an international survey. Findings were presented as a visual map to facilitate knowledge exchange during an online workshop with a purposeful sample of multidisciplinary stakeholders. The identified gaps in research were explored to see whether they were deemed representative of unmet needs, and as such, areas of priority to key FI stakeholders. Ideation techniques and group discussions were used to refine and rank priority areas.ResultsOverall, there was a mismatch between the existing and emerging evidence, and the priorities of key FI stakeholders. New pharmaceutical and medical technology innovations were limited. Eight percent of early-stage trials identified were concerned with the use of repurposed drugs. Within the existing evidence base, individual bowel management strategies and treatments were examined, however, key FI stakeholders desired interventions to improve patient education and the psychological aspects of living with FI. The five priority topics identified in order of importance are as follows: psychological support; lifestyle interventions; long-term effects; education; and constipation.ConclusionsThe robust methodology used to identify priority topics were successful in identifying broad and wide-ranging areas of importance to key stakeholders. The evidence gap map was a useful visual tool to facilitate knowledge exchange and highlight where research efforts have been focused historically, identifying a mismatch between the existing evidence base and what stakeholders consider important.
OP33 Expectations, Needs And Challenges Of Setting Up An International Collaboration On Horizon Scanning For Medical Devices
Authors: Michels Renee Else , de Graaff Bert , Abrishami Payam , Delnoij Diana Maria Johanna 
Published: December 2022

IntroductionInternational collaboration on horizon scanning for medical devices is seen as desirable, because the development of medical devices is not limited to the national level, and horizon scanning for medical devices on a country-level can be challenging due to scarcity and diversity of information. The International Horizon Scanning Initiative Medical Devices Working Group (IHSI MDWG) was set up in June 2021. The objective of this study was to research the expectations and experiences of the participants of this initiative, in order to reflect on the possibilities and challenges of governing innovative medical technologies.MethodsA questionnaire was sent out to 10 purposively selected representatives of the IHSI MDWG participating counties: Austria, Belgium, Canada, Denmark, Italy, Luxembourg, Netherlands, Norway, Portugal, and Sweden. The survey covered individual countries’ respective purposes for an international horizon scanning system as well as questions related to the desired scope and perceived challenges of such a system. The questionnaire was supplemented with online, semi-structured, in-depth interviews with the same representatives from each participating country. These interviews provided for diving deeper into the survey topics as well as discussing the relation between horizon scanning and health technology assessment, the relation to other international horizon scanning collaborations, and the relation between an international versus a national horizon scanning system. In addition, participant observations were conducted at the Dutch National Health Care Institute and during IHSI MDWG working group meetings.ResultsPreliminary results are discussed first with participants after which we will draw our final conclusions and recommendations for practice. Our analysis focuses on exploring participants’ expectations and experiences with international horizon scanning through triangulating the three sources of data in our analysis.ConclusionsThe study will report on the expectations, needs and challenges of setting up an international collaboration for horizon scanning of medical devices and reflect on the regulation and governance of innovative medical technologies across several countries in Europe and Canada.
OP35 Suitability Of Preference Methods Across The Medical Product Lifecycle: A Multicriteria Decision Analysis
Authors: Veldwijk Jorien , de Bekker-Grob Esther , Juhaeri  , van Overbeeke Eline , Tcherny-Lessenot Stephanie , Pinto Cathy Anne , DiSantostefano Rachael L. , Groothuis-Oudshoorn Catharina G.M. 
Published: December 2022

IntroductionTo understand the importance of the preference methods criteria to stakeholders at each decision point in the Medical Product Lifecycle (MPLC) and to determine the suitability of commonly applied preference methods (Discrete Choice Experiment [DCE], swing weighting [SW], probabilistic threshold technique [PTT], Best-Worst Scaling case 1 [BWS1], Best-Worst Scaling case 2 [BWS2]) for a given decision-point.MethodsNineteen preference methods criteria of an existing performance matrix were incorporated in an online survey of industry, regulatory, and health technology assessment (HTA) stakeholders. All methods criteria were given a relative weight based on the SW ranking and point allocation task in the survey. Based on this relative weight and the performance matrix values, an overall suitability score was calculated for each method per critical decision point along the MPLC. Several sensitivity analyses were conducted for which the performance matrix was adapted.ResultsIn total 59 industry, 29 regulatory, and 5 HTA representatives completed the survey. In general, ‘estimating trade-offs between characteristics’, and ‘estimating weights for treatment characteristics’ were important preference method criteria throughout all MPLC decision points, while other preference method criteria were most important only for specific MPLC stages. Both BWS1 and BWS2 seem equally suitable across decision points, DCEs seem most suitable during clinical development and regulatory launch, and SW and PTT seem most suitable throughout industry decision points. Sensitivity analysis showed substantial impact of slight changes in the performance matrix.ConclusionsWith rapid changes in preference research, performance matrices of preference methods should continue to be re-evaluated as more and more evidence accumulates. While DCE is the most applied preference elicitation method, other methods should also be considered to address the needs of MPLC stakeholders. Development of evidence-based guidance documents for designing, conducting, and analyzing such methods could enhance their use.
OP38 Managed (Early) Access In England: The ‘Ins And Outs’
Authors: Bee Charlotte , Hawksworth Claire , Wakefield Leanne , Groves Brad 
Published: December 2022

IntroductionManaged access approaches have been used by The National Institute for Health and Care Excellence (NICE) in partnership with NHS England since 2015. The Cancer Drugs Fund is an exemplar of this approach which enables earlier patient access to promising new treatments while further real-world data is collected to address evidential uncertainties. Increasingly, this approach is being applied to rare diseases and other conditions to address unmet clinical need with extensive involvement from patient organizations and clinicians.MethodsAll Managed Access Agreements (MAAs) in development or published in England were reviewed to present data on the number of technologies (i) entering, (ii) in active monitoring, and (iii) exiting managed access, for both cancer and other disease areas.ResultsAfter six years since the first MAA (at December 2021), over 73,000 patients have benefited from earlier access to promising new treatments for cancer, genetic and rare diseases, including cystic fibrosis, spinal muscular atrophy and sickle cell disease via managed access. Fifty-eight technologies were commissioned via managed access: thirty technologies in active data collection, eleven technologies being re-evaluated, and seventeen technologies have exited managed access. Patient and clinical engagement have been essential to the successful real-world data collections delivered and underway.ConclusionsManaged access is an approach for providing earlier patient access to promising new technologies that would not otherwise be recommended for use in England. The approach to managed access in England is maturing at the same time the volume of topics entering and exiting managed access in England is expected to grow throughout 2022 with the introduction of the Innovative Medicines Fund.