bluuebunny/crossref_metadata_2025_split huggingface dataset:
OP70 Treating Patients With Hormone-Sensitive Cancer On Endocrine Therapy With Denosumab (Prolia®): A Systematic Review And Network Meta-Analysis
Authors: Nicolopoulos Konstance , Moshi Magdalena Ruth , Stringer Danielle , Ma Ning , Jenal Mathias , Vreugdenburg Thomas 
Published: December 2023

IntroductionPatients receiving endocrine therapy for hormone-sensitive cancers, such as men with prostate cancer (MPC) on hormone ablation therapy (HAT) and women with breast cancer (WBC) on adjuvant aromatase inhibitor therapy (AAIT), have an increased risk of developing osteoporosis. The aim of this study was to compare the safety and effectiveness of denosumab (Prolia®) with selective estrogen receptor modulators (SERMs) (raloxifene and bazedoxifene), bisphosphonates (zoledronate, ibandronate, alendronate, and risedronate), and placebo for the treatment of osteoporosis in patients receiving endocrine therapy for hormone-sensitive cancer.MethodsSystematic literature searches were conducted in three biomedical databases (PubMed, the Cochrane Library, and Embase) to identify randomized controlled trials (RCTs). Only RCTs that investigated MPC on HAT or WBC on AAIT allocated to denosumab, SERMs, bisphosphonates, or placebo were included. RCTs were appraised using the Cochrane Risk of Bias 2.0 tool. Frequentist network and pairwise meta-analyses were performed on predetermined outcomes of vertebral or nonvertebral fractures, treatment-related adverse events (AEs), bone mineral density (BMD), mortality, withdrawal due to treatment-related AEs, and serious AEs.ResultsA total of 14 RCTs (15 publications, 6,463 participants) were included. Relative to placebo, denosumab was found to prevent vertebral fractures in cancer patients receiving endocrine therapy. Moreover, denosumab, alendronate, and zoledronate increased femoral neck (FN) and lumbar spine (LS) BMD in MPC on HAT, compared with placebo, whereas denosumab, risedronate, and ibandronate improved LS and total hip BMD in WBC on AAIT. Similarly, denosumab and risedronate increased trochanteric BMD in WBC on AAIT, compared with placebo. In WBC on AAIT, only denosumab increased FN BMD relative to placebo.ConclusionsDenosumab was more effective than placebo in preventing vertebral fractures and improving BMD at the LS and FN in MPC on HAT, and in preventing vertebral fractures and improving FN, trochanteric, total hip, and LS BMD in WBC on AAIT. From a policy perspective, the continued reimbursement of denosumab needs to be reviewed.
OP79 Gene Expression Profiling In The Diagnosis Of Aggressive Large B Cell Lymphoma: An Early Exploratory Economic Evaluation
Authors: Bouttell Janet , Hawkins Neil , Masson Rachel , Goodlad John 
Published: December 2023

IntroductionThe addition of gene expression profiles (GEP) to the current clinicopathological diagnosis of aggressive large B cell lymphomas may lead to the reclassification of patients, treatment changes and improved outcomes. A GEP test is in development using TempoSeq technology to distinguish Burkitt Lymphoma (BL) and Primary Mediastinal Large B Cell lymphoma (PMBCL) from Diffuse Large B Cell Lymphoma (DLBCL). This study aims to inform developers about the potential impact of the test on costs and health outcomes, and pricing and evidence generation strategies.MethodsDecision models compared current diagnosis with current plus GEP signatures over a lifetime horizon using a UK health and social care perspective. Inputs were taken from the literature and based on assumptions. Threshold estimates were made of the maximum price of the test and impact of incorrect disease classification using a threshold of GDP30,000 (USD37,155) per Quality Adjusted Life year (QALY). One way sensitivity analysis was conducted.ResultsAt base case values the BL signature delivers incremental QALYs of 0.0249 at an additional cost per patient of GBP508 (USD629). This results in a net monetary benefit (NMB) of GBP239 (USD296). The PMBCL signature delivers 0.0011 QALYs, a cost saving of GBP202 (USD250) and an NMB of GBP236 (USD292). The maximum threshold price for a combined test to be cost effective is GBP776 (USD961) (base case GBP400 (USD495)). Results are sensitive to cost differences in first line treatments and impact of false diagnoses.ConclusionsA combined test could be cost-effective in a UK context at a price around GBP750 (USD929). The developers can use this estimate to inform return on investment calculations. The number of patients who were reclassified as a result of the addition of GEP in our model was taken from small retrospective studies and the impact of false diagnoses was based on limited evidence. If the developers choose to proceed with the development, these aspects should be incorporated in evidence generation strategies.
OP82 Positron Emission Tomography Combined With Computed Tomography Using 18F-Sodium Fluoride
Authors: Aimanova Tansolpan , Akhmetova Makpal , Okesh Makhabbat , Avdeyev Andrey , Shanazarov Nasrulla , Albayev Rustam , Saduakassova Aigul , Lyugay Yekaterina , Tleulessova Indira , Turar Olzas , Bariyeva Gulzada 
Published: December 2023

IntroductionPositron emission tomography combined with computed tomography (PET/CT) using 18F-sodium fluoride (18F-NaF) is used for functional imaging in diseases to detect abnormally altered osteogenic activity, such as benign and malignant bone diseases and inflammatory or traumatic changes in skeletal bones.MethodsA systematic search of literature using keywords in the MEDLINE database was conducted to identify literature on the clinical and cost effectiveness of using PET/CT with 18F-NaF-based radiopharmaceuticals in the diagnosis of bone and cartilage cancer. The search retrieved 323 publications. The analysis included 11 publications that met the selection criteria, including one meta-analysis and ten literature reviews.ResultsThe pooled sensitivity, specificity, diagnostic odds ratio (DOR) and area under the receiver operating characteristic curve of 18F-NaF-based PET/CT for the detection of bone metastases were 0.98 (95% confidence interval [CI]: 0.95, 0.99), 0.90 (95% CI: 0.86, 0.93), 123.2, and 0.97, respectively. 18F-NaF-based PET/CT was highly effective in detecting bone metastases during staging and restaging of patients with high-risk prostate cancer. The effectiveness of 18F-NaF-based PET/CT was superior to bone scintigraphy with technetium-99m and single-photon emission computed tomography (SPECT) and was comparable to diffusion-weighted imaging.ConclusionsPET/CT with 18F-NaF is a more accurate method of localizing and characterizing malignant bone lesions than SPECT. This method has improved clinical accuracy and provides greater convenience for patients and referring physicians. PET/CT with 18F-NaF in malignant neoplasms is a more specific, sensitive study than 18F-fluorodeoxyglucose PET/CT. These results were submitted to the Ministry of Health for a decision on the inclusion of 18F-NaF-based PET/CT in the state reimbursement system.
OP90 Multiple Myeloma: Developing A Benchmark Patient Experience Index In Australia And New Zealand
Authors: Fifer Simon , West Bronwyn , Godsell Jenni , Combe Nella , Stephenson Todd 
Published: December 2023

IntroductionWithin Australia and New Zealand (ANZ) there is limited evidence regarding the experience and satisfaction across the healthcare system of people living with multiple myeloma (MM). We aimed to quantify the patient experience across the healthcare system to help identify potential areas of the healthcare pathway that could be targeted for improvement to maximize patient satisfaction.MethodsA 30-
to 40-minute online survey was completed by adults in ANZ diagnosed with MM. Anchored best-worst scaling (ABWS) is a technique that takes advantage of an individual’s ability to reliably identify extremes (‘best’ and ‘worst’) in sets of items, eliciting discriminating rankings free of scale bias. This study implemented a novel anchoring process to rescale importance and satisfaction best-worst scores for factors across the MM healthcare pathway, which could be compared and combined to form a patient experience index (PEI). There were 15 factors or ‘moments that matter’ (MTM), each describing a different aspect of the patient journey, such as time to diagnosis, treatment logistics, and side effects. The MTM were derived from qualitative research with patients as well as a workshop with key opinion leaders. Additional survey questions were included to help identify potential ways to improve patient satisfaction.ResultsThe results were based on 62 patients with MM. The overall median PEI score was 63.1. The top three MTM that were most important to patients, but they were least satisfied with (calculated by combining the top four of each most important/least satisfied factor for each participant), were side effects of medication, effectiveness of medication, and medication access.ConclusionsThe findings from this research contribute to the understanding of patient experiences of treatment and care for MM. The results can inform healthcare decisions for prioritizing interventions that align with patient experiences. In the future, the study could be executed longitudinally to assess shifts in satisfaction within the MM healthcare journey, which would be especially worthwhile if new programs are implemented to improve patient satisfaction.
OP93 The Cost Effectiveness Of Antiretroviral Therapy Adherence Interventions In HIV/AIDS Patients: A Systematic Review Of Decision Analytic Models
Authors: Ahmed Ali , Dujaili Juman , Le Long , Khanal Saval , Chuah Lay Hong 
Published: December 2023

IntroductionPeople living with HIV/AIDS (PLWHA) frequently struggle to maintain optimal adherence to antiretrovirals (ARVs). Different adherence-improving interventions have been developed and examined through decision analytic model-based health technology assessments. Therefore, we aimed to conduct a systematic review of all decision analytic models developed to improve adherence in PLWHA.MethodsThe review protocol was registered on Prospero (CRD42022270039) and relevant studies published from inception to 23 October 2022 were identified through searches of the following databases: PubMed, Embase, the National Health Service Economic Evaluation Database, PsycINFO, the Health Economic Evaluations Database, and EconLit. Studies were included if they were modeling works of full economic evaluations, including cost-effectiveness analyses (CEA), cost-utility analyses, cost-benefit analyses, and cost-consequence analyses. The primary outcome was the cost effectiveness of adherence interventions reported as the incremental cost per additional quality-adjusted life-year (QALY). Study quality was assessed with the Quality of Health Economics Studies instrument. Due to the heterogeneity of the data, a permutation matrix was used for quantitative data synthesis rather than a meta-analysis.ResultsThe 15 studies identified were conducted in North America (8/15), Africa (4/15), and Europe (3/15). The time horizon was one year in one study, ten years in one study, 20 years in three studies, and a lifetime horizon in ten studies. The types of interventions were smartphone-based (5/15), nurse involved (2/15), directly observed therapy (2/15), case manager involved (1/15), simplification of regimens (1/15), Link4Health (1/15), and others (3/15) that involved multicomponent intervention. The interventions gained higher QALYs with cost savings in all 15 studies and gained QALYs at a higher cost at an acceptable incremental cost-effectiveness ratio in 80 percent (12/15) of studies. The studies were of fair (13%) to high quality (87%).ConclusionsThis study is the first systematic review of decision analytic model-based CEAs of adherence interventions in the management of PLWHA. Most of the identified studies recently published good quality cost-effectiveness analyses with an adequate timeframe.
OP94 Economic Evidence On Hemodialysis Access Creation Procedures In Patients With End-Stage Kidney Disease: A Systematic Literature Review
Authors: Gupta Ritu , Gaba Upasna , Delaney Christopher , Papadopoulos George 
Published: December 2023

IntroductionIt is important to create and maintain durable hemodialysis (HD) access in health systems to reduce morbidity and maintain overall cost control in patients with end-stage kidney disease (ESKD). To evaluate the choice of HD vascular access creation procedures and their related economic costs, we aimed to identify economic evaluations on vascular access (VA) creation procedures in patients with ESKD.MethodsA systematic literature review was conducted using the Cochrane methodology to identify cost-effectiveness analyses (CEAs), budget impact analyses, and cost analyses of various HD access creation procedures. Eligible publications published from 2012 onwards were retrieved by searching PubMed, Embase, and the Cochrane Library. The Consolidated Health Economic Evaluation Reporting Standards 2022 checklist and ISPOR Task Force guidelines were used to appraise the quality of the economic evaluations and budget impact analyses, respectively. Costs were adjusted for inflation and purchasing power parity and standardized to US dollars.ResultsA total of 40 economic evaluations met the inclusion criteria, including 28 cost analyses, three budget impact analyses, and nine CEAs. Widely evaluated procedures in the published literature were endovascular and surgical arteriovenous fistula (AVF), arteriovenous graft (AVG), and central venous catheterization (CVC). The results indicated that AVF was the most cost-effective strategy, followed by AVG, and CVC. Three studies showed that endovascular AVF was cost effective, compared with surgical AVF, and resulted in overall cost savings of about USD53 million dollars over a five-year period. Results of the quality assessment showed that budget impact analyses scored 63 percent, while the average score for economic evaluations was 58 percent.ConclusionsIt was challenging to identify a single effective method of managing vascular access due to the substantial heterogeneity among VA creation techniques. However, most of the included economic evaluations showed that AVF was a cost-effective method of VA creation relative to other identified techniques for patients with ESKD on HD.
OP100 Patient Perspectives In Value Assessment Frameworks: The Asia Pacific Perspective
Authors: Best Alex , Tsai I-Ching , Tan Jin Yu , Wu David bin-chia , Yu DaeYoung , Keetley Alison , Wong-Rieger Durhane , Jain Ritu 
Published: December 2023

IntroductionThe importance of patient centricity in healthcare decision making has been recognized and advocated for decades. However, approaches for including the patient perspective are diverse, and progress varies among countries. Some reimbursement bodies acknowledge the importance of patient preferences in health technology assessment (HTA) and funding decision processes. However, patients’ perspectives are not yet systematically and transparently included in value assessment frameworks globally, and even less so in the Asia-Pacific (APAC) region. This systematic review aimed to investigate how patients’ perspectives are used to inform pricing and reimbursement decisions in the APAC region.MethodsA systematic review is ongoing that utilized a search of 12 databases, including MEDLINE and Embase, to identify publications on the consideration of patient perspectives in health policy decision-making published to November 2022. Conference abstracts published in the last five years from ISPOR and Health Technology Assessment International (HTAi) were screened, along with gray literature and government websites from Australia, China, Japan, Malaysia, New Zealand, the Philippines, Singapore, South Korea, Taiwan, and Thailand. Publications were included if the impact of either one or more of the following on HTA decision-making was assessed: active participation of patients or patient advocacy groups; type, extent, and evolution of patient-reported outcomes; health-related quality of life or quality of life tools; and themes where the impact of patients’ perspectives on value assessment was the primary outcome. Countries were characterized into archetypes based on similarities or differences in the weight and value assigned to patient perspectives in decision-making.ResultsA total of 6,438 retrieved citations will undergo the systematic review process. Additionally, 758 conference abstracts from ISPOR, 1,312 from HTAi conferences and 73 records from gray literature will be screened.The results of the systematic review will be consolidated into country archetypes, examples, and learnings. Gaps and opportunities will also be identified.ConclusionsThe research will provide recommendations to increase shared decision-making and support the development of decision-making frameworks that systematically incorporate patients’ perspectives in value assessment across APAC countries.
OP115 Scoping Meta-Review On Methods Used To Assess Artificial Intelligence-Based Medical Devices For Heart Failure
Authors: Di Bidino Rossella , Piaggio Davide , Iadanza Ernesto , Pecchia Leandro , Andellini Martina , Zeeshan Raza , Merino-Barbancho Beatriz , Pérez Laura López , Fico Giuseppe , Ni Melody , Borsci Simone , Zhu Tianhui 
Published: December 2023

IntroductionThe International Federation of Medical and Biological Engineering created a multidisciplinary working group to discuss assessments of artificial intelligence and machine learning (AI/ML) applications in health care. Engineers, clinicians, and economists identified evidence generation as a critical topic. Heart failure (HF) was selected to investigate the available evidence on the clinical effectiveness and safety of AI/ML applications. Attention was paid to transparency of AI/ML methods and their data sources.MethodsA scoping review was conducted on AI/ML algorithms developed for the management of HF. A search for systematic reviews, scoping reviews, and meta-analyses published from 1976 to October 2022 was conducted in Embase, MEDLINE, and Scopus.ResultsOf 456 relevant publications, 21 papers were included in the final analysis. Most papers (10 systematic reviews, five meta-analyses, and six non-systematic or scoping reviews) included studies conducted in North America. No study was conducted in Africa. The healthcare setting was not clearly stated in approximately half of the studies. A lack of agreement was noticed regarding the quality assessment tools used among the reviews. The most common data source for AI/ML algorithms was electronic health records, but in some cases data sources were not reported. While deep learning emerged as the most common adopted methodology, covariates were not always included in the algorithm development. The review demonstrated that comparative assessment of algorithms requires further investigation, given the high variability in the comparator used (e.g., clinical gold standard, other AI/ML algorithms, or other statistical methods). The main investigated endpoints were the incidence of HF and the number of hospital admissions.ConclusionsWhen assessing innovative health technologies such as AI/ML applications in health care, evidence is among the main challenges. Our scoping review, focusing on algorithms developed to manage HF, showed that the biggest challenges relate to the quality of the studies, the adoption of a comparative approach, and transparency of methods.
OP128 Uncertainties In The Cost-Effectiveness Analysis Of Onasemnogene Abeparvovec For Spinal Muscular Atrophy Type 1
Authors: Borges Stefani , Fernandes Brígida , Rodrigues Fernanda , Cirilo Hérica Núbia , Krug Bárbara , Probst Livia , Zimmermann Ivan 
Published: December 2023

IntroductionNusinersen and risdiplam are available in the Brazilian Unified Health System (SUS) for the treatment of spinal muscular atrophy (SMA) type 1. Onasemnogene abeparvovec, a promising gene therapy, was approved in 2020 in Brazil. Given the high cost of this therapy and its promise of a lifetime effect, the objective of this study was to evaluate the cost effectiveness of onasemnogene abeparvovec, compared with nusinersen and risdiplam, in the treatment of SMA type 1 from the perspective of SUS in different scenarios.MethodsA Markov model was adapted from one originally developed for the USA that considers five states of health. Short-term data were obtained from pivotal clinical trials and long-term survival curves were extracted from published reports from the USA. Maintenance of motor function milestones achieved at the end of follow up in clinical trials was considered until death. Costs and quality-adjusted life-years (QALYs) were discounted at five percent per year over a baseline lifetime time horizon. Alternative scenarios were evaluated for horizons of five and ten years, with and without a discount.ResultsOnasemnogene abeparvovec resulted in an incremental cost of BRL742,890 (USD297,156) per QALY and an increase of 3.32 QALYs in relation to the alternatives over a lifetime time horizon. In the same time horizon, but without the discount, onasemnogene abeparvovec resulted in an incremental cost-effectiveness ratio (ICER) of BRL166,539 (USD66,615) per QALY. In a five-year time horizon, considering the discount rate, the therapy resulted in an ICER of BRL12,527,667 (USD5,011,066); in ten years the ICER was BRL3,384,793 (USD1,353,917).ConclusionsSince the benefits of onasemnogene abeparvovec mainly occur in the long term, decision makers need to consider the uncertainty of assumptions of sustained effectiveness in view of the high initial cost of the technology.
OP149 How To Improve The Impact Of Health Technology Assessment: Stakeholders’ Perspectives In Spain
Authors: Triñanes Yolanda , Gómez Patricia , Faraldo-Vallés María J , Pérez-Ríos Mónica , Ruano-Raviña Alberto , lema Leonor Varela 
Published: December 2023

IntroductionHealth technology assessment (HTA) agencies in Spain have an important role in informing decisions about the introduction and use of health technologies in the Spanish National Health System. However, although different approaches have been taken to measure and improve their impact, no study to date has explored the perceived impact of HTA products at the national level. The aim of this study was to explore the perspectives of macro-, meso-, and micro-level decision makers on how to improve the impact of HTA.MethodsThree online focus groups were conducted with policy makers, healthcare managers, clinicians, and patients. The transcripts were evaluated using a deductive thematic analysis based on a multidimensional framework to explore mechanisms of impact.ResultsFour key themes were identified:(i)Timeliness and use of HTA assessments: Although the quality of the reports was recognized, the time taken for the elaboration and extension of reports negatively affected their use. Participants considered that reports should be tailored to the needs of end users (e.g., briefer versions available for meso-and micro-level use);(ii)Effective engagement and external communications: The engagement of multiple stakeholders (policy makers, manufacturers, clinicians, and patients) in the elaboration process was considered crucial to improve HTA impact and ensure adequate communication of results;(iii)Good institutional reputation and fit within the healthcare and policy making system: Stakeholders agreed on the need to strengthen collaboration at the national level and increase public understanding of the value of HTA and its use in healthcare decision-making; and(iv)Effective implementation of policy change regarding health technologies: Stakeholders were very receptive to the results and recommendations of HTA reports when new technologies are demanded, but the identification and selection process should be improved to guarantee that these reports are available on time.ConclusionsThis study has identified different proposals and mechanisms that could improve the impact of HTA in Spain.