bluuebunny/crossref_metadata_2025_split huggingface dataset:
PP68 Successfully Streamlining New Medicines Assessments In Scotland: A Stakeholder Evaluation Of The Scottish Medicines Consortium’s Abbreviated Submission Process
Authors: Henriquez Alex , McGuire Pauline , Hepburn Christine 
Published: January 2025

IntroductionThe Scottish Medicines Consortium (SMC) assesses new medicines for NHSScotland, based on company submissions. The abbreviated process previously applied to new formulations of medicines with anticipated low net budget impact. During the COVID-19 pandemic, SMC extended the abbreviated process to new medicines with alternatives within the same therapeutic class already approved in Scotland. This evaluation explored stakeholder perspectives on this processMethodsA mixed-method approach, with focus groups and online surveys, was utilized to collect data from stakeholders (n=56) including Patient Group Partners (n=14), Public Partners (n=2), pharmacists employed in local health boards and in national procurement (n=17), pharmaceutical industry representatives (n=10), SMC senior clinical assessors (n=6), and the SMC Executive Team (n=7). All participants were asked a series of open and semi-open questions about specific aspects of the process, its advantages, disadvantages, and potential areas for improvement. A thematic analysis, based on initial color coding and grouping of responses into themes, was employed to analyze the qualitative dataResultsOverall, the abbreviated process was commended for its time and resource efficiency, particularly benefiting the SMC assessment teams, decision-making committees, and the pharmaceutical industry. The decision-making committees experienced a decrease in workload and capacity gains, which allowed them to focus on more innovative new medicines. Overall, stakeholders welcomed rapid access to new medicines, and resource savings for pharmaceutical companies, patient groups, and the SMC assessment teams. Suggestions for improvement included strengthening patient group involvement in the process, improving transparency of the decision-making process, in particular the budget impact assessment, and providing further guidance on the definitions of “same therapeutic class.”ConclusionsRegulatory agencies are licensing more new medicines, in terms of volume and complexity. HTA bodies must consider innovative ways to undertake proportionate assessments. Extension of SMC’s abbreviated process was well commended by stakeholders, primarily due to resource-savings benefits and expedited access to new medicines. Evaluation of the change identified further areas for improvement.
PP74 Are Commonly Used Cost-Effectiveness Thresholds Too Low? Empirical Evidence From Economic Studies On The Value Of Life
Authors: Schlander Michael , Hernandez Diego , Schwarz Oliver , Schaefer Ramon 
Published: January 2025

IntroductionIn health technology assessment (HTA), “value for money” is frequently conceptualized as incremental cost-effectiveness (CE), with effectiveness measured as (quality-adjusted) life years gained (LYG). Commonly used CE thresholds have been subject to controversial debate. We reviewed and analyzed the worldwide economic literature on the value of a statistical life (VSL), which reported empirical estimates during the last 25 years.MethodsWe conducted an extended systematic literature search in the EconBiz and EconLit databases, spanning the period from January 1995 to December 2020. We transformed VSL data into the implied values of a statistical life year (VSLY) and analyzed the results by regional origin, elicitation method, and annual gross domestic product (GDP) per capita. Then, we performed a regression analysis using the ordinary least squares (OLS) model after log-transforming the VSLY estimates.ResultsWe identified 156 empirical economic studies with 169 unique VSL estimates. Overall, the median VSLY was EUR168,367 (mean, EUR256,701) or 6.3 times annual GDP/capita. The median VSLY (expressed as multiples of GDP/capita) differed by regional origin (North America: EUR288,994; 7.2; Europe: EUR168,367, 5.2; Asia: EUR45,260, 4.5) and by elicitation method (revealed preference/wage risk studies: EUR274,625, 9.1; stated preference/contingent valuation studies: EUR113,246, 4.4; stated preference/discrete choice experiments: EUR178,130, 5.2). Regression analyses confirmed that studies with data originating from North America resulted in significantly higher VSLY estimates. The difference remained statistically significant after adjusting for GDP/capita.ConclusionsThe empirical economic literature, while showing heterogeneity by elicitation method, suggests that the adoption of a preference-based or “demand-side” perspective leads to willingness-to-pay estimates for a LYG that exceed commonly used CE thresholds in the context of HTA.
PP76 “It Is Better For Me To Die Than To Be Disgraced”: Perceptions Of Worse-Than-Death Health States In Ghana
Authors: Essel Adwoa , Akweongo Patricia , Addo Rebecca , Nonvignon Justice , Affram Akosua Adjeiwa 
Published: January 2025

IntroductionMany cultures across the world have varying conceptions about death and dying. Perceptions about health states considered “worse than death” also vary based on sociocultural norms as well as health system capacity. We explore worse-than-death health states in Ghana as well as reasons for opting for death in those health states.MethodsWe interviewed 28 participants from three regions in Ghana to understand the contextual “value of life” in Ghana and factors influencing respondents’ decision to opt to die rather than live in a particular health state. Written consents were sought from all participants to partake in the study. Interviews were conducted in either Twi or English based on each participant’s preference and lasted for an average of 30 to 35 minutes. Interviews were transcribed verbatim and stored in NVivo software. Data were analyzed thematically.ResultsWe identified that health states perceived as worse than death were those associated with impairment in mobility, anxiety/depression, and pain/discomfort. Participants preferred death under these circumstances because they wanted to avoid the financial burden on themselves and family, time spent in caregiving by family, loss of personhood, and loss of social status. Decisions regarding health states worse than death hold considerable importance, particularly in a context where culture and societal norms play a role in shaping how quality of life is assessed.ConclusionsAn understanding of the value Ghanaians attach to health states perceived as worse than death provides useful information for patient-centered care. Findings from the study can provide evidence on healthcare resource allocation and aid policymakers and clinicians in making informed decisions on which treatments to prioritize, and how to maximize the overall health and well-being of individuals.
PP79 Challenges With Integrating Early-Stage Cancer Trial Endpoints Into Economic Models: Review Of Canadian And International HTA Recommendations
Authors: Beca Jaclyn , Chan Cherry , Ting Eon 
Published: January 2025

IntroductionTherapies for early-stage cancers demonstrate clinical benefit with early endpoints that measure delayed or avoided disease recurrence, and survival benefits take years to confirm. Economic evaluations for health technology assessment (HTA) require assumptions about long-term benefits to project lifetime disease trajectories. We examined economic modeling approaches used in HTA for adjuvant/neo-adjuvant therapies to understand challenges, explore patterns, and identify opportunities for methodological improvements.MethodsWe included drug indications with Canadian Agency for Drugs and Technologies in Health (CADTH) reimbursement recommendations as of November 2023 for adjuvant/neoadjuvant treatment of early-stage solid tumors. We collected recommendation outcomes and details of submitted clinical and economic evidence. Adapting prior work and focusing on threats to validity arising from equivocal methodological challenges, we classified issues raised in the economic review, particularly those related to surrogacy, treatment pathways, and long-term benefit assumptions (extrapolation, duration of benefit, and cure). We made comparisons with respect to the approaches to long-term benefit assumptions and treatment pathways for drug indications that also had appraisals by the National Institute for Health and Care Excellence (NICE, UK) and the Pharmaceutical Benefits Advisory Committee (PBAC, Australia).ResultsSeventeen drug indications for adjuvant/neoadjuvant treatment of solid tumors were included. Reimbursement was recommended in Canada for 83 percent of drug indications, all were reviewed and recommended in the UK, and 11/15 (73%) have been recommended in Australia. Assessments described overall survival (OS) as immature, but interim OS data appeared to be supportive. There was considerable variability in approaches to long-term benefit assumptions by both submitters and reviewers. Modifications to the assumptions were made in two-thirds of reviews before acceptance by HTA. There was notable inconsistency in approaches to handling treatment-waning, while cure assessment time greater than or equal to five years from initiation was consistently considered appropriate by reviewers.ConclusionsWhile all assessments recognized immature OS data, positive reimbursement recommendations were common. There was important variability in application of methods to estimate long-term treatment effectiveness, particularly determining appropriate, evidence-informed assumptions for duration of benefit and cure. This research can support guidance, methodological advancements, and consensus-building to appropriately capture benefits and assess uncertainties for treatments in early-stage cancers with more consistency.
PP83 Building Resilient Capacity For The Diagnosis Of Cardiac Pathologies Through Telemedicine: Pilot Study
Authors: Galvan Pedro , Ortellado José , Rivas Ronald , Grossling Benicio , Hilario Enrique 
Published: January 2025

IntroductionThe evolution of technology in medicine and information and communication technology (ICT) has been an important step to introduce innovative/resilient specialized health services. The emergence of telemedicine offers opportunities to enhance specialized diagnosis services. This study has evaluated the feasibility of using tele-Holter and tele-ABPM (ambulatory blood pressure monitoring) to build a resilient diagnosis network for cardiac pathologies in Paraguayan remote public hospitals.MethodsThis observational descriptive multicenter feasibility study is based on a telemedicine-driven approach for specialized cardiac diagnostic services using Holter and ABPM devices in remote and underserved public hospitals in Paraguay. A telemedicine platform was used to send records of Holter and ABPM devices from the remote hospitals to the cardiologist to screen cardiac pathologies.ResultsDuring the pilot study, 52 cardiac diagnostic tests were carried out using the tele-Holter and tele-ABPM approach in 10 regional hospitals countrywide. Cardiac diagnosis was performed in 24 patients using Holter and 28 patients using ABPM. The most frequent findings using tele-Holter were normal (91.6%), not sustained ventricular tachycardia (4.2%), and atrial fibrillation (4.2%). Regarding tele-ABPM, the diagnoses performed were arterial hypertension (50.0%), uncontrolled arterial hypertension (40.0%), and normal (10.0%). Overall, an average of 90.0 percent of diagnosed patients suffered high blood pressure and 8.4 percent suffered heart disorder.ConclusionsAccording to our results, the use of a telemedicine-driven approach to build a resilient diagnosis network for cardiac pathologies in remote underserved public hospitals in Paraguay is feasible. A widespread use-assessment should be analyzed before this tool is adopted.
PP88 Breaking Boundaries In Health Technology Assessment: Quantifying Non-Traditional Value Of Medical Technology Through ExpertLink Remote Connectivity Solutions
Authors: Wong Callix , Patel Dinesh , Cheng Michael , Khor Tze Wei , Manoharan Devindran , Sothilingam Selvalingam , Tan Hui Meng , Swan Georgia 
Published: January 2025

IntroductionA diverse range of services often supplements procedures that involve medical technologies and adds value along patient care pathways. However, these novel elements of value are often not captured in traditional assessment frameworks. ExpertLink is one such example. ExpertLink uses digital solutions to connect clinical experts worldwide, enabling remote training and collaboration, while maintaining the highest standard of patient care.MethodsRezum™ is a minimally invasive therapy for patients with symptomatic benign prostatic hyperplasia (BPH). It is a quick day procedure with proven safety, effectiveness, and durability in clinical outcomes. Leveraging ExpertLink, an expert in Sydney, Australia, remotely proctored 11 Rezum™ procedures in Malaysia in November 2022, supporting five urologists in five hospitals across five states within five hours. Efficient and straightforward procedures such as Rezum™ are well suited to remote proctorship. Through this case study, we quantify the sustainability, equity, and access benefits, illustrating the additional value ExpertLink brings across the healthcare continuum and beyond.ResultsFor a proctor traveling from Australia to Malaysia, over 6,500 kilometers and 17 hours travel time is saved, equating to an estimated 1,700-kilogram reduction in CO₂ emissions. Without ExpertLink, a proctor may be away from practice for up to a week. ExpertLink allows for continuity of practice, including consultations and procedures, during this time. For five doctors traveling from Malaysia to Australia for training, an estimated 7,400-kilogram reduction in CO₂ emissions and approximately 85 hours travel time is saved. ExpertLink provided 11 geographically dispersed patients with timely access to treatment and expedited the physician learning curve.ConclusionsThis case study illustrates the value for just one technology on one day. ExpertLink embodies novel elements of value that are not captured in traditional value assessment frameworks. Collaborative effort between stakeholders is needed to broaden the view of value in healthcare, incorporate additional elements of value in existing assessment frameworks, and appropriately recognize this often-uncounted value in decision-making.
PP94 Chronic Obstructive Respiratory Disease Patients’ Involvement In The Evaluation Of Transcutaneous Capnography In Primary Care
Authors: Rodríguez-Ortiz-de-Salazar Begoña , Cardaba-Arranz Mario , Palma-Ruiz Matilde , Hass D Nicole , Sanchez-Gomez Luis-Maria , González-Enríquez Jesús 
Published: January 2025

IntroductionPatient participation in health technology assessment (HTA) plays an increasingly relevant role due to increased recognition of its essential contribution to addressing uncertainties in evidence and its real-world application. The objective is to analyze and describe how patients with chronic obstructive pulmonary disease (COPD) participate in the evaluation of transcutaneous capnography (TC) in the primary care setting.MethodsThe Spanish Association of Patients with Chronic Obstructive Pulmonary Disease facilitated contact with three COPD expert patients. A face-to-face video interview was conducted with each patient to know about their knowledge of the technology, real-life experiences, and expectations. Patients were informed of the objective of the evaluation and signed confidentiality and conflict of interest forms. All interviews were conducted in April 2023 by two researchers. Expert patients with COPD were able to participate in the review of the protocol and in the final version of the report. Literature searches were also conducted on patient perceptions of TC compared to arterial blood CO2 measurement.Results
All patients were male, older than 60 years and were ex-smokers with greater than 30 years of tobacco consumption. Patients highlighted the relevance of early detection of COPD to facilitate the planning and organization of treatment as their clinical situation progresses. Likewise, they also emphasized the importance of the implementation of less invasive tests, and the proximity and accessibility of primary care. Two studies reported greater patient satisfaction and less pain with TC than with arterial blood gases.ConclusionsExpert patients show high expectations for the technology, as it is less invasive than arterial blood gases; they also acknowledged its proximity in primary care settings and the potential for detecting complications of oxygen therapy. Patient participation in HTA adds intangible value, as they provide “disease-specific knowledge” and real-life applicability of the technology.
PP100 A Study On Health-Related Quality Of Life Among Patients With Advanced Gastric Or Gastro-esophageal Junction Adenocarcinoma
Authors: Jin Doyeon , Ahn Jeonghoon , Lee Hyorim , Seo Hyerin 
Published: January 2025

IntroductionHealth-related quality of Life (HRQoL) is reported as a strong predictor of survival. However, little is known about the relationship between HRQoL and influencing factors of gastric cancer among Korean patients. This study aims to investigate the HRQoL of Korean patients with advanced gastric cancer and identify factors influencing their HRQoL.MethodsA total of 156 patients with advanced gastric cancer were 1:1 matched for demographics and clinical characteristics between a ramucirumab/paclitaxel combination group and a chemotherapy group. The objective response of the subjects was divided into four categories: partial response (PR), stable disease (SD), progressive disease (PD), and no complete response/no progressive disease (non-CR/non-PD). The six-month changes in HRQoL measured by EuroQol 5-dimension 5-level questionnaire (EQ-5D-5L) were estimated and further investigated for associations with demographic and clinical factors.ResultsThe average changes in HRQoL were highest in PR (0.086±0.057), followed by non-CR/non-PD (0.078±0.076), SD (0.035±0.097), and PD (no change). The HRQoL by age group was 0.176±0.088 in subjects 40 years or younger, 0.035±0.071 in the 50s, 0.067±0.072 in the 60s, and −0.003±0.029 in those who were 70 years or older. The HRQoL of females was 0.145±0.136, and for males it was 0.047±0.066. The HRQoL of the non-diabetic group was 0.072±0.095, and for the diabetic group was 0.044±0.057. The HRQoL of the non-hypertensive group was 0.097±0.097, and for the hypertension group was 0.020±0.048.ConclusionsFor advanced gastric cancer patients, the increase of HRQoL was diminished as cancer progresses, age increases, for males, and when comorbidity of diabetes or hypertension exists. The reported HRQoL data is valuable for health technology assessment of advanced gastric cancer agents such as ramucirumab, as this study is a publicly funded neutral study.
PD14 The Cost Effectiveness Of Robot-Assisted Gait Training For Patients In Japan With Subacute Hemiplegic Stroke
Authors: Morii Yasuhiro , Abiko Kagari , Ohashi Kazuki , Osanai Toshiya , Tani Yuji , Tanikawa Takumi , Takamiya Soichiro , Fujiwara Kensuke , Ogasawara Katsuhiko 
Published: January 2025

IntroductionAlthough it is reported that robot-assisted gait training (RAGT) with conventional physiotherapy for stroke patients increases the odds of walking independence at the end of the intervention, compared with conventional physiotherapy alone, it is not clear how long the benefit lasts or whether it is cost effective. This research aimed to clarify the duration of benefit of RAGT and its cost effectiveness.MethodsA cost-utility analysis was conducted that compared RAGT plus conventional physiotherapy with conventional physiotherapy alone from a public healthcare payer’s perspective. The population comprised patients with subacute hemiplegic stroke who had a modified Rankin Scale of three to five (severe) and were treated in Japanese hospitals. The time horizon was half a year after admission to a convalescent hospital, since no additional benefit has been proved beyond that point. A decision tree model was used for the analysis. The effect of RAGT on walking independence and the durability of the benefit were estimated based on a literature review of randomized controlled trials (RCTs) and a meta-analysis. Costs and utility values were estimated from the literature.ResultsThe literature review identified 14 RCTs. A meta-analysis of RCTs with more than two months’ follow-up after the intervention showed a significantly higher rate of independence in walking for the RAGT group at the end of follow-up (risk ratio 1.52, 95% confidence interval: 1.20, 1.93), whereas there was no significant difference between the groups more than three months after treatment. The incremental quality-adjusted life-years (QALYs) was 0.004 and the incremental cost was -USD287, indicating that RAGT with conventional physiotherapy was dominant. The incremental cost-effectiveness ratio was USD5,509 per QALY when it was assumed that the length of stay was not reduced by achieving early independence in walking.ConclusionsThe results showed excellent cost effectiveness for RAGT plus conventional physiotherapy in patients with subacute severe hemiplegic stroke in the Japanese setting when considering a reference value of USD34,000 (JPY5,000,000) per QALY in the cost-effectiveness evaluation. Although the incremental QALY gains were relatively small, cost savings could be expected from achieving early independence in walking.
PD18 Cytomegalovirus Prophylaxis With Valganciclovir For HIV-Immunosuppressed Patients: Cost Effectiveness And Budget Impact Analysis From The Brazilian Healthcare System Perspective
Authors: Gargano Ludmila , André Ariane , Freitas Isabela , de Assis Acurcio Francisco , Alvares-Teodoro Juliana , Guerra Augusto 
Published: January 2025

IntroductionCytomegalovirus (CMV) infections are usually mild but can lead to serious consequences in immunocompromised patients, such as those infected with human immunodeficiency virus (HIV). Valganciclovir, which is approved for treating CMV, may be a cost-effective prophylactic for CMV disease in immunocompromised patients. Cost-effectiveness and budget impact analyses were conducted to compare valganciclovir prophylaxis with no prophylaxis in immunosuppressed patients with HIV.MethodsImmunosuppressed patients with HIV who were seropositive for CMV were modeled in a one-year horizon decision tree. Outcomes were occurrence of CMV end-organ disease (EOD)–retinitis and gastroenteritis (85 and 15 percent of cases, respectively)–and survival probability after EOD. Direct medical costs related to prophylaxis (induction and maintenance phases) and treatment of EOD were considered. The budget impact analysis (BIA) used the clinical and cost parameters of the cost-effectiveness analysis to compare scenarios with valganciclovir prophylaxis and no prophylaxis in the public health system over a five-year time horizon. The annual market share adoption rate used was 10 percent. The number of eligible patients was calculated from HIV and acquired immunodeficiency syndrome cases reported in the country and epidemiological estimates.ResultsValganciclovir prophylaxis had an incremental cost of BRL31,781 (USD6,531) and added 0.69 percent in one-year survival, resulting in an incremental cost-effectiveness ratio (ICER) of BRL4,610,022 (USD947,414) per death avoided in one year. The annual budget impact ranged from BRL35,660,931 (USD7,328,743) in the first year to BRL457,707,796 (USD94,064,365) in the fifth year, totaling BRL1,230,400,576 (USD252,861,870) over five years. In sensitivity analyses, the lack of a maximum treatment time for the maintenance phase with valganciclovir (50 to 365 days per year) resulted in a wide variation in the ICER and BIA results.ConclusionsDespite being recommended for the treatment of CMV retinitis, the use of valganciclovir in immunocompromised patients with HIV who are seropositive for CMV but have not developed CMV disease has a higher ICER when compared with no prophylaxis. For these patients, appropriate use of standard antiretroviral therapy to improve the immune system may be more appropriate.