bluuebunny/crossref_metadata_2025_split huggingface dataset:
PD67 Real-World Evidence To Inform Reflexive Practice And Create Value In Lung Cancer Care
Authors: Boily Gino , Guédon Aude-Christine , Abiala Godwill , Qureshi Samia , Golo Kossi Thomas , Roussafi Ferdaous , Lehuédé Camille , Lanthier Julie , Soltana Kahina , Kuzminski Frédéric , Strumpf Erin , Boulanger Jim , Pagé Élisabeth , Truchon Catherine 
Published: January 2025

IntroductionHealth technology assessment (HTA) agencies assess the value of innovative therapies and publish recommendations for practice. However, is publishing HTA products sufficient to generate value in the real world? The objectives of our work were to: (i) determine whether innovative therapies for lung cancer produce the expected results in the real-world setting; and (ii) assess whether recommendations are followed in real-world practice.MethodsClinical administrative data were used in this two-phase project. In the first phase, a descriptive portrait of the use of epidermal growth factor receptor tyrosine kinase inhibitors (EGFR-TKIs) for treating lung cancer was produced. Their value was assessed by comparing overall survival of treated patients observed in the province of Québec to the published literature. The second phase focused on the initial evaluation of patients diagnosed with lung cancer and treated first by surgery. The delay between first evidence of cancer and surgery was assessed, and the utilization of 27 healthcare services was analyzed and assessed according to our recommendations (algorithms) for lung cancer management.ResultsFrom the date the first EGFR-TKI was listed, it took about five years before these drugs were fully integrated into clinical practice. The median overall survival of patients in Québec who used an EGFR-TKI (three indications) was similar to that in most published studies, supporting previous reimbursement decisions. The median delay between first evidence of cancer and surgery was longer than the 60-day consensus target. Utilization of most healthcare services was heterogeneous between regions. Bronchoscopy on its own seemed overused in many regions, whereas non-surgical approaches as a first method for invasive mediastinal evaluation should have been more systematically applied.ConclusionsAt a relatively low cost, real-world evidence can serve as a powerful tool to validate reimbursement decisions and measure the state of clinical practice. By sharing results with stakeholders, it will enable clinical teams to reflect upon their practice and implement local improvement strategies.
PD72 Are Patient Experience Data Currently Embedded In Reimbursement Decision-Making? An Analysis Of Belgian Reimbursement Documents
Authors: Vanneste Alice , Verbeke Charlotte , Deschepper Katrien , Donvil Danique , Barbier Liese , Janssens Rosanne , Huys Isabelle 
Published: January 2025

IntroductionStakeholders in the drug life cycle recognize the importance of integrating patient experience data (PED) into healthcare decision-making. PED includes patient input, patient reported outcomes (PROs), patient reported experiences, and patient preference information. However, it remains unclear if, how, and to what extent PED are used in reimbursement decisions in Belgium.MethodsA document analysis was performed to evaluate and compare the reporting of PED in Belgian reimbursement documents for COVID-19, oncology, and cardiology. Documents of medicinal products that received a European marketing authorization between 1 January 2015 and 30 September 2023 and were reimbursed in Belgium were included. Data were analyzed descriptively and qualitatively.Results
Preliminary results showed that PED was generally either not reported or not explicitly reported in Belgian reimbursement documents. In some documents, patient reported outcome measures (PROMs) were mentioned. Additionally, few documents stated PROs such as quality of life. PROMs, PROs, and quality of life outcomes appeared in the reimbursement dossier section describing the therapeutic value of the product. From the preliminary findings it seems that PED were more often present in oncology documents. The reimbursement dossiers did not report whether or how PED were used to inform a particular reimbursement decision.ConclusionsPreliminary findings suggest that PED are not systematically included in Belgian reimbursement documents. To improve transparent submission and reporting of PED in reimbursement dossiers, guidance from reimbursement agencies as well as checklists and evaluation templates are needed.
PD80 Exploring Key Factors Influencing Patients’ Antidepressant Preferences: Insights From A Multicenter Best-Worst Scaling Survey
Authors: Tao Ying , Ren Yanfeng , Liu Shimeng , Chen Yingyao 
Published: January 2025

IntroductionDepression is associated with serious disease burden. Despite the multitude of antidepressant options available, the adherence rate is often low. Accounting for patient preferences can potentially boost adherence to antidepressant medication and elevate patient satisfaction. However, limited evidence exists regarding patient preferences for antidepressant selection. This study aims to elicit patient preferences regarding the benefits, risks, and cost attributes of antidepressants in China.MethodsA best-worst scaling profile case experiment was conducted using a face-to-face survey administered to patients diagnosed with depression. Patients were recruited from general and psychiatric hospitals. We utilized a multiphase approach that integrated literature review, expert consultation, and best-worst scaling to develop attributes within choice sets. The attributes with each varying across two or three levels encompassed remission rate, sleep disorders, risk of headache or dizziness, risk of gastrointestinal adverse events, risk of liver or kidney injury, and monthly out-of-pocket costs. Each respondent answered seven choice tasks, including a dominant task. Data were analyzed using conditional logit, mixed logit, and generalized multinomial logit models. Subgroup analyses were conducted to explore preference heterogeneity.ResultsA total of 331 participants completed the survey and met the inclusion criteria. Almost all attribute levels were statistically significant. Overall, the most desirable characteristics of antidepressant medications were higher remission rates (80% and 55% rates; p<0.05), lower risk of liver or kidney injury (1% rate; p<0.05), and fewer monthly out-of-pocket costs (CNY100 [USD13.93, EUR12.75]; p<0.05). Risks of gastrointestinal adverse events (60% and 35% rates) and insomnia were the least preferred features. Regarding attributes, efficacy, the risk of gastrointestinal adverse events, and sleep disorders were relatively important factors influencing patient choice. Preferences differed slightly by age, degree of education, personal annual income, and treatments currently received.ConclusionsOur study suggests that efficacy, gastrointestinal adverse effects, sleep disorders, and treatment costs are critical drivers behind medication choices among patients with depression. Preference heterogeneity also exists regarding individual and therapeutic characteristics, which need more samples and further analyses to identify. These discoveries hold the potential to enrich the shared decision-making process between physicians and patients within healthcare settings.
PD92 Health Technology Assessment Of Genetic And Genomic Applications: A Proposal For A Standardized Approach
Authors: Sciurti Antonio , Migliara Giuseppe , Baccolini Valentina , Pitini Erika , Kaminska Anna Ewa , Soccodato Valentina , Mussetto Ilaria , Marzuillo Carolina , Villari Paolo 
Published: January 2025

IntroductionFrameworks based on the ACCE model, which have a limited health technology assessment (HTA) approach, have long been used in the assessment of genetic and genomic applications (GGAs). While ACCE frameworks are mainly focused on technical aspects, HTA assessments include economic and organizational aspects. The aim of this study was to develop a framework able to address a comprehensive assessment of GGAs.MethodsWe analyzed the most currently used HTA model in Europe, the EUnetHTA HTA Core Model®, to investigate its suitability as an HTA framework for GGAs and to identify any possible shortcomings in its evaluation. The Sapienza evaluation framework, which is based on a previous systematic review of assessment frameworks for GGAs, was used as the comparison. The HTA Core Model applications were then used as a basis for an assessment framework for GGAs, classified according to “GGA function/use setting” pairs.ResultsThe domains included in the Sapienza framework fully corresponded with the HTA Core Model. In addition, the “Screening Technology” application of the HTA Core Model was found to be suitable for diagnostic, pre-symptomatic, predictive, and carrier GGAs when used in screening settings. The “Diagnostic Technologies” application was deemed appropriate for the pairs “Diagnostic/Diagnosis”, “Pre-symptomatic/Diagnosis”, “Prognostic/Staging”, and “Pharmacogenetic/Staging”, although it may require additional considerations such as the need for screening in the case of germline mutations, the use of appropriate accuracy measures for prognostic GGAs, and the evaluation of the clinical utility of pharmacogenetic or prognostic GGAs.ConclusionsThe proposed approach is a flexible tool that allows assessment of GGAs using a shared and validated methodology that considers the technical aspect, clinical utility, economic value, and delivery models. We found that the HTA Core Model fits the needs of GGA assessment, although some GGA peculiarities should be further explored in the assessment process.
PD97 Comparison Of Clinical Outcomes Of Self-Expandable Versus Balloon-Expandable Valves For Transcatheter Aortic Valve Replacement: A Population-Based Cohort Study
Authors: Chung Chinghu , Moore Matt , Hung Ya-Ping , Hsu Ju-Yi 
Published: January 2025

IntroductionTranscatheter aortic valve replacement (TAVR) has become a leading treatment for patients with severe aortic stenosis (AS). Recent studies comparing TAVR outcomes with balloon-expandable valves (BEVs) and self-expandable valves (SEVs) show generally similar results, although BEVs have lower rates of moderate-to-severe aortic regurgitation and pacemaker implantation. This study aimed to compare the clinical outcomes of SEVs and BEVs in Taiwan.MethodsThe Taiwan National Health Insurance Research Database (NHIRD) is a representative claims database capturing 99.9 percent of residents. We identified patients who underwent TAVR with either a SEV or BEV in 2021 using the NHIRD. The outcomes were six-month survival, length of hospital stay (LOS) and intensive care unit (ICU) stay, postoperative complications, and healthcare expenditure. We used inverse probability of treatment weighting (IPTW) based on age, gender, and Charlson Comorbidity Index score to identify the effect of TAVR valve type on LOS and ICU stay, postoperative complications, and healthcare expenditure. Differences between SEVs and BEVs for IPTW-weighted Kaplan-Meier curves of overall survival were measured with the log rank test.ResultsAmong the patients identified who underwent TAVR, 366 received a SEV and 132 received a BEV. The mean ages were 82.70 (standard deviation [SD] 8.08) years and 82.25 (SD 7.53) years, respectively. The hazard ratio for six-month mortality rate for SEVs compared with BEVs was 2.78 (95% confidence interval 1.52, 5.09). The six-month mortality rate was also significantly higher for SEVs than for BEVs (13.11% versus 4.55%). For clinical outcomes, the mean total LOS (14.78 [SD 12.19] versus 14.45 [SD 12.96] days), mean ICU stay (5.91 [SD 9.78] versus 6.23 [SD 8.04] days), rate of complications (<3%) and in-hospital healthcare costs (USD43,285 [SD 11,993] versus USD42,920 [SD 13,931]) were similar for both groups. The results were also similar after weighting.ConclusionsPatients in Taiwan who underwent TAVR with BEVs had better survival outcomes than those who received SEVs, while other clinical and cost outcomes were comparable between the valve types.
PD103 Pediatric Exoskeleton For The Treatment Of Spinal Cord Atrophy And Other Neuromuscular Diseases
Authors: Loeches Ana Alcalde , Arribas Blanca Novella , Navarro Gustavo Mora , Belinchon Pilar Loeches , Salvanés Francisco Rodriguez 
Published: January 2025

IntroductionSpinal cord atrophy (SMA) affects one in every 1,000 live births in Spain. Cerebral palsy (CP) is the most common cause of chronic motor disability in children, affecting two to three in every 1,000 live births. The Atlas 2030 exoskeleton is the first portable robotic pediatric exoskeleton aimed at facilitating walking in these children. It is used as a complement to other therapies.MethodsWe aimed to compare the efficacy and safety of the ATLAS 2030 exoskeleton with conventional physiotherapy in children aged between four and 12 years with SMA or CP. We conducted a literature search to identify clinical trials and systematic reviews in the PubMed, Embase, Web of Science, and Cochrane Library databases, finding 201 original articles published between 2017 and 2022. No controlled studies were found on the ATLAS 2030 or any other portable device, even after expanding the searches to any robot-assisted gait devices.ResultsAlthough systematic reviews evaluating the efficacy and safety of robotic orthoses in general were found, there were no specific controlled studies on the ATLAS 2030. The five studies found (two on CP and three on SMA) were of low quality and did not show conclusive results in terms of efficacy and safety. However, some of them suggested that this technology could provide significant improvements in acceptability, range of motion, and spasticity. With respect to safety there appeared to be no evidence of serious adverse effects from its use, reinforcing the developer’s initial hypothesis that the technology is safe.ConclusionsAlthough the available evidence is very limited, the ATLAS 2030 appears to show efficacy in some gait-related outcomes without serious adverse effects. To demonstrate safety and efficacy, randomized controlled trials are required that compare the ATLAS 2030 with conventional physiotherapy, with more participants and a longer duration, and are conducted by independent expert groups without conflicts of interest.
PD108 Digital Transformation Of An Organization Supporting Informal Caregivers During Pandemic: Lessons Learned And Future Direction For A Sustainable Solution
Authors: Khan Amir J , Szczepura Ala , Wild Deidre , Nelson Sara , Collinson Mark , Greenwood Marc , Woodhouse Sonja 
Published: January 2025

IntroductionThe COVID-19 pandemic has catalyzed a move from face-to-face to online delivery of services by hospitals and primary care providers, but little is known about the impact of digital transformation in organizations supporting unpaid caregivers. The value of care provided by informal caregivers since the start of the COVID-19 pandemic has been estimated at EUR111 billion in England.MethodsThe study assessed the impact of digital transformation in an English caregivers’ support organization covering a population of 0.98 million. A retrospective mixed method study was conducted of digital and non-digital support service utilization among caregivers in city and rural geographical areas from January 2019 to June 2021. Organizational performance and service quality indicators were compared for two financial years: 2019-2020 and 2020-2021. A survey of users was conducted to evaluate barriers to and facilitators of digital service uptake, computer proficiency among caregivers, and preferences for future digital service provision.ResultsThe number of caregivers registered with the organization rose by 36 percent to 20,237 in 2021. Monthly contacts rose by 225 percent to 6,500, with remote contacts rising from 65 to 85 percent. Observed behavior patterns differed between city and rural caregivers. Overall, one-to-one contacts increased by 89 percent and caregiver assessments by 21 percent, with no expansion in staffing. User-reported service quality improved in five out of eight indicators (p<0.05). The demographic characteristics of survey respondents (152 caregivers) were similar to all registered caregivers. The mean short form Computer Proficiency Questionnaire score of 25.61 indicated a relatively high computer proficiency. Qualitative analysis confirmed a preference for face-to-face and online options. The most highly rated online services were peer support groups and wellbeing assessment and support needs checks.ConclusionsConsidering the economic importance of unpaid caregivers, more attention should be paid to the organizations supporting them and the potential for technology to enhance caregivers’ access to and benefit from such services. This initial assessment of digital transformation in one such organization demonstrates the potential for cost-effective service transition. Further research is required to inform sustainable future solutions.
PD109 Health Vulnerability: Rethinking Intervention Strategies In A Health Maintenance Organization In Brazil
Authors: del-Valle Matheus , Franco Braga Paulo Estevao , Borin Marcus Carvalho , Bruschi Kelles Silvana Marcia , Biscione Fernando Martin 
Published: January 2025

IntroductionThis study identified health susceptibility using socioeconomic and environmental indicators, focusing on their impact on population health, by using a health vulnerability index (HVI). The aim was to map vulnerabilities using the HVI among beneficiaries of a health maintenance organization (HMO) in the metropolitan region of Belo Horizonte (MRBH), to enhance access, efficiency, equity, and quality in health services.Methods
Established in 2012 by Belo Horizonte’s municipality using 2010 census data, the HVI incorporates eight socioeconomic and sanitation indicators. This methodology was extended to all HMO beneficiaries in the MRBH using georeferencing to assess their vulnerability levels (low, medium, high, or very high) and proximity to health facilities.ResultsThe findings revealed that 5.44 percent of the HMO’s clients reside in areas categorized as high or very high risk, which corresponds to more than 70,000 individuals. Notably, 91.8 percent of these high-risk beneficiaries are situated in the suburban and peripheral areas of the region and predominantly utilize health facilities located on the outskirts of Belo Horizonte or within the metropolitan area. This distribution underscores a notable disparity in healthcare accessibility and service utilization patterns, with a marked inclination toward the use of emergency services among these populations.ConclusionsThe study underscores a strong correlation between vulnerability and the type of healthcare service utilized, with vulnerable groups often resorting to emergency services, which leads to fragmented care. It highlights the need to improve service processes, particularly for those reliant on public transport, and advocates for an integrated approach to health interventions that promotes equitable healthcare access in complex socioeconomic landscapes.
PD116 Development Of A Value Framework For The Appropriate Prescription Of High-Cost Cancer Drugs In A Cancer Center
Authors: Quirland Camila , Carreño-Leiton Diego , Lahoz-Quintanilla Daniela , Muñoz-Montecinos Carlos , Maza Felipe , González-Browne Catalina 
Published: January 2025

IntroductionEffectiveness, efficiency, and consistency with patient preferences are requirements for appropriate healthcare. The Complex Treatment Evaluation Committee (CTEC) at the Arturo López Pérez Foundation is a multidisciplinary committee that assesses the appropriateness of high-cost cancer drug prescriptions (HCCDP) and authorizes their use accordingly. Our study aimed to develop a value framework to assess the appropriateness of HCCDP at the Foundation.MethodsWe conducted a literature review to identify appropriateness criteria for oncology prescriptions and the judgments used by the Chilean healthcare system for clinical practice guideline recommendations and reimbursement decisions for these medications. The results were discussed by the CTEC to establish a final value framework through consensus and to define a methodology to assess the appropriateness of HCCDP weekly. Annual indicators were designed to improve the agreed methods and the adequacy of prescriptions.ResultsCriteria for the value framework were grouped into three categories: magnitude of clinical benefit, efficiency, and sustainability. Every criterion should be met to consider an HCCDP as appropriate. Adequacy was evaluated by assessing prescription evidence identified from electronic databases, evidence-based clinical practice guidelines, regulatory agency reports, and health technology assessment reports. From 2019 to 2022, 1,626 cases have been evaluated. Although potentially inappropriate CTEC authorizations have decreased over time, there was a growing mismatch between these decisions and the prescribing behavior of clinicians.ConclusionsBy involving clinicians, managers, and health economists we developed a value framework for the timely assessment of the appropriateness of HCCDP in a hospital setting. Further research on the underlying reasons for the differences observed is needed, along with additional appropriateness criteria such as consistency with the preferences and ethical principles of patients.
PD118 Company-Led Submissions For Cancer Medicines: The Singapore Experience
Authors: Ong Jiamin , Loke Lydia , Lin Liang , Ng Kwong 
Published: January 2025

IntroductionThe Agency for Care Effectiveness (ACE) conducts health technology assessments (HTAs) to inform funding decisions by the Ministry of Health (MOH) Drug Advisory Committee (DAC) in Singapore. In 2021 ACE introduced the company-led submission (CLS) process for cancer medicines, which allows pharmaceutical companies to request evaluations alongside regulatory reviews. This review reports key findings from the first year of its implementation.MethodsA total of 10 CLS topics from the first year of implementation were included. We reviewed the status and outcomes of the DAC recommendations. We also used descriptive statistical methods to evaluate the time from HTA submission to first HTA recommendation and from regulatory approval to first HTA recommendation. The timelines were further analyzed by whether submissions were parallel submissions (i.e., HTA submission in tandem with regulatory review) or sequential submissions (i.e., HTA submission after regulatory approval). These statistics were compared with overseas reference jurisdictions (Australia, Canada, and the UK).ResultsAt the time of review, three topics were pending discussion. Of the remaining seven topics, three (43%) received positive recommendations for inclusion on the MOH Cancer Drug List and three (43%) received negative recommendations. The DAC was unable to make a recommendation on one topic. The median time from HTA submission or regulatory approval to first HTA recommendation was 172 days (range 169 to 263 days) and 279 days (range 53 to 374 days), respectively. Notably, parallel submissions (75 days; n=2) had considerably shorter timelines from regulatory approval to first HTA recommendation than sequential submissions (328 days; n=4). These timelines were within the range of the overseas reference countries.ConclusionsParallel CLS allows HTA processes to be conducted in tandem with regulatory reviews, moving HTA recommendations upstream and expediting patient access to clinically effective and cost-effective medicines. Efforts will be made to further evolve the CLS process to achieve timely reimbursement reviews from regulatory approval and to expand this process to noncancer medicines.