bluuebunny/crossref_metadata_2025 huggingface dataset:
PD66 Scalability Of Integral, Transversal, And Multidisciplinary Management Of Aortic Stenosis In Catalan Hospitals
Authors: Scheepen Franka Van , Fernandez-Barcelo Carla , Hagemeijer Bàrbara Vidal , Abbas Ismail , Carreño Marta Sitges , Sampietro-Colom Laura
Published: January 2025

Introduction

A program for integral, transversal, and multidisciplinary management of aortic stenosis (MITMEVA) was implemented at the Clinic Barcelona University Hospital to provide adequate and personalized treatment for patients with aortic stenosis (AS). MITMEVA includes 11 actions in the AS care pathway. This study aimed to test the scalability of MITMEVA by gathering the perspectives of relevant stakeholders in two other Catalan hospitals.

Methods

A mixed method study design was used to collect and analyze the views of relevant stakeholders on the scalability of the MITMEVA project. Qualitative and quantitative methods were used to answer the research question. All participants were interviewed individually using a semi-structured interview guide. The interviews were transcribed and analyzed by performing a content analysis with Atlas.ti software. Quantitative data were gathered and analyzed by adjusting the Intervention Scalability Assessment Tool (ISAT).

Results

Nine participants were interviewed from two tertiary hospitals in Catalonia and eight ISAT questionnaires were completed. From the content analysis, 11 of the 15 themes identified related to actions implemented in the MITMEVA program. The results pointed toward a positive view of implementation of the MITMEVA program in general, showing a good scalability for all the ISAT domains. On a scale of zero to three, most domains were scored two or above.

Conclusions

The qualitative and quantitative results indicated that the MITMEVA program has potential for scaling up to other Catalan hospitals to improve the management of AS. Generally, stakeholders from the two participating hospitals were positive about the project. However, small adjustments will be needed to account for the culture and organizational characteristics of the hospitals when scaling up the MITMEVA program.
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

Introduction

Health 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.

Methods

Clinical 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.

Results

From 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.

Conclusions

At 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

Introduction

Stakeholders 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.

Methods

A 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.

Conclusions

Preliminary 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

Introduction

Depression 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.

Methods

A 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.

Results

A 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.

Conclusions

Our 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

Introduction

Frameworks 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.

Methods

We 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.

Results

The 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.

Conclusions

The 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

Introduction

Transcatheter 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.

Methods

The 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.

Results

Among 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.

Conclusions

Patients 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

Introduction

Spinal 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.

Methods

We 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.

Results

Although 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.

Conclusions

Although 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

Introduction

The 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.

Methods

The 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.

Results

The 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.

Conclusions

Considering 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

Introduction

This 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.

Results

The 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.

Conclusions

The 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

Introduction

Effectiveness, 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.

Methods

We 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.

Results

Criteria 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.

Conclusions

By 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

Introduction

The 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.

Methods

A 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).

Results

At 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.

Conclusions

Parallel 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.
PD128 Evaluating The Implementation Of An Abdominal Aortic Aneurysm Screening Program In Spain
Authors: Pla Anna Godo , Soto Gonzalo Bravo , Sisó-Almirall Antoni , de Céniga Melina Vega , Bertran Marta Trapero , Vargas Martínez Ana Magdalena , Estrada Sabadell Maria Dolors , Vivanco-Hidalgo Rosa Maria
Published: January 2025

Introduction

Abdominal aortic aneurysm (AAA) is a progressive and silent enlargement of the aorta, but aortic rupture is associated with a high mortality rate. The main objectives of this study were to assess evidence on the clinical safety, efficacy, and cost effectiveness of an AAA screening program using abdominal ultrasound. An economic evaluation was also conducted to assess the introduction of AAA screening into the Spanish Healthcare System.

Methods

A systematic literature search was conducted according to PRISMA recommendations. Randomized controlled trials were selected to assess clinical safety and efficacy. Systematic reviews were selected to identify prognostic factors for AAA and full economic evaluations were selected for the cost-effectiveness analysis. Data extraction and assessment of evidence certainty were performed using the GRADE methodology. A Markov model was developed to perform an economic evaluation and budget impact analysis of an AAA screening program in the Spanish context.

Results

Screening had a beneficial effect on AAA detection and rates of all-cause and AAA-related mortality in men older than 65 years. It had no effect on rates of AAA rupture or emergency surgery and may increase rates of elective surgery. The evidence was very uncertain for women older than 65 years. Sex and family history of AAA were the most important prognostic factors. Economic evaluation of a program screening men older than 65 years in primary care centers resulted in 0.33 life-years gained, 0.18 quality-adjusted life-years (QALYs) gained, and an incremental cost-effectiveness ratio of EUR81.98 per QALY. The program’s implementation would cost an additional EUR15.68 to EUR28.40 per patient.

Conclusions

The implementation of an AAA screening program in men older than 65 years in Spain is considered effective and could be implemented using the infrastructure, materials, and human resources already present in the Spanish health system. Relevant indicators and dynamic monitoring of the program should be established to allow for continuous and flexible evaluation of results over time.
PD132 A Target Trial Emulation Application Assessing The Survival Of Erythropoiesis-Stimulating Agents In Patients With Lower Risk Myelodysplastic Syndrome
Authors: Bennett Alastair , Manca Andrea
Published: January 2025

Introduction

Patients with myelodysplastic syndromes (MDS) can be treated with erythropoiesis-stimulating agents (ESAs) to alleviate anemia-related symptoms and delay the need for expensive transfusions. However, clinicians disagree on prescribing ESAs because evidence on the effectiveness of ESAs is limited. This study aimed to reliably estimate the survival of a dynamic ESA treatment regimen using a novel causal inference approach.

Methods

The European MDS Registry collects data on patients with MDS every six months. We followed a two-step framework to develop a hypothetical and emulated trial protocol. The eligible population consisted of patients with intermediate-1 to low-risk MDS who were treatment-naïve, had a hemoglobin concentration of less than 10 grams per deciliter, and did not have a chromosome 5q deletion (non-del(5q) MDS). Red blood cell transfusion was allowed before the date of diagnosis. Patients were cloned and assigned to both treatment groups, thereby eliminating immortal time bias, and were censored as soon as they stopped following the assigned treatment strategy. This artificial censoring introduced selection bias, which was adjusted for by using inverse probability of censoring weighting. The weights model adjusted for time varying confounders.

Results

Of the 611 patients qualifying for the study, 282 started ESAs within the six-month grace period and 329 did not take ESAs. The median follow-up was 2.4 years (interquartile range 1.3 to 4.2). A naïve analysis of our cohort suggested that no ESA was significantly more beneficial than taking ESAs (hazard ratio [HR] at year four: 1.24, 95% confidence interval [CI]: 1.03, 1.50). However, after correcting for biases the adjusted Kaplan-Meier curves showed that ESAs were beneficial over the first two years (HR at year one: 0.75, 95% CI: 0.41, 1.39), compared with no ESAs. Thereafter there was no difference between treatment groups (HR at four years: 1.01, 95% CI: 0.80, 1.27).

Conclusions

We found that early—within six months of becoming eligible—initiation of ESAs as first-line therapy for treatment-naïve patients with non-del(5q) low-risk MDS and hemoglobin levels of less than 10 grams per deciliter improves survival over for the first two years. Using target trial emulation to make accurate survival estimates can improve decision-making in health technology assessment.
PD134 Projecting The 10-Year Cost Of Care Burden For Depression Until 2032 In Hong Kong: A Real-World Evidence Based Markov Model
Authors: Yi Chan Vivien Kin , Mallory Leung Man Yee , Man Chan Sandra Sau , Yang Deliang , Knapp Martin , Luo Hao , Craig Dawn , Chen Yingyao , Bishai David Makram , Yan Wong Gloria Hoi , Lum Terry , Yin Chan Esther Wai , Kei Wong Ian Chi , Li Xue
Published: January 2025

Introduction

We developed a real-world evidence (RWE) based Markov model to project the 10-year cost of care for patients with depression from the public payer’s perspective to inform early policy and resource planning in Hong Kong.

Methods

The model considered treatment-resistant depression (TRD) and development of comorbidities along the disease course. The outcomes included costs for all-cause and psychiatric care. From our territory-wide electronic medical records, we identified 25,190 patients with newly diagnosed depression during the period from 2014 to 2016, with follow-up until December 2020 for real-world time-to-event patterns. Costs and time varying transition inputs were derived using negative binomial and parametric survival modeling. The model is available as a closed cohort, which studies a fixed cohort of incident patients, or an open cohort that introduces new patients every year. Utilities values and the number of incident cases per year were derived from published sources.

Results

There were 9,217 new patients with depression in 2023. Our closed cohort model projected that the cumulative cost of all-cause and psychiatric care for these patients would reach USD309 million and USD58 million by 2032, respectively. In our open cohort model, 55,849 to 57,896 active prevalent cases would cost more than USD322 million and USD61 million annually in all-cause and psychiatric care, respectively. Although less than 20 percent of patients would develop TRD or its associated comorbidities, they contribute 31 to 54 percent of the costs. The key cost drivers were the number of annual incident cases and the probability of developing TRD and associated comorbidities and of becoming a low-intensity service user. These factors are relevant to early disease stages.

Conclusions

A small proportion of patients with depression develop TRD, but they contribute to a high proportion of the care costs. Our projection also demonstrates the application of RWE to model the long-term costs of care, which can aid policymakers in anticipating foreseeable burden and undertaking budget planning to prepare for future care needs.
PD139 Investigation Of The Current Status Of New Health Technology Assessment In Korea And The Factors Influencing Assessment Results
Authors: Kang Jihyun , Kim Jinho , Shin Chae Min , Park Boyoung
Published: January 2025

Introduction

This study aimed to evaluate factors influencing assessment results in the new health technology assessment (nHTA) system in Korea.

Methods

Publicly available HTA reports obtained from the nHTA website were selected as a data source. A total of 258 nHTA reports including 305 technologies were included in the analysis. The detailed information in the reports was classified into three major categories: technical characteristics, evaluation methods, and publication types. A chi-squared test was used to investigate differences in the levels of evidence (high, medium, or low) and assessment results (pass or fail) according to the three categories. Univariate and multivariate logistic regression analyses were performed to identify factors associated with the levels of evidence and assessment results.

Results

nHTA reports that performed a meta-analysis and included randomized controlled trials for evidence synthesis were associated with higher levels of evidence. The corresponding odds ratios were 5.008 (95% confidence interval [CI]: 1.265, 18.826) and 27.052 (95% CI: 7.802, 103.330), respectively. The analysis showed that as the level of evidence increased, the likelihood of the assessment passing was significantly higher (odds ratio 2.789, 95% CI: 1.284, 6.057). However, univariate analysis indicated that performing a meta-analysis or including randomized controlled trials, both of which affect evidence level, did not have a statistically significant association with assessment results.

Conclusions

This study is the first systematic analysis of the factors influencing the results of nHTA reports in Korea. While higher evidence levels were associated with positive assessment outcomes, factors affecting the evidence level itself did not directly influence assessment results. More efforts are needed to integrate high levels of evidence into assessments.
PD142 Implantable Neurostimulation Devices For The Treatment Of Drug-Resistant Pediatric Epilepsy
Authors: García Vanesa Ramos , Álvarez-Pérez Yolanda , Sacks Analía Abt , Rivero-Santana Amado , Duarte-Díaz Andrea , Santos-Álvarez Anthea , González-Hernández Yadira , Rivas-Ruiz Francisco , Perestelo-Pérez Lilisbeth
Published: January 2025

Introduction

Epilepsy affects approximately 10.5 million individuals under the age of 15 years worldwide. In Spain, 3.7 per 1,000 inhabitants aged 6 to 14 years have epilepsy, making it the third most common neurological emergency. Drug resistance is observed in eight to 33 percent of cases. Responsive neurostimulation (RNS) systems could improve seizure control in pediatric patients who are not eligible for brain surgery.

Methods

We systematically searched for articles published up to September 2022 in the following bibliographic databases: MEDLINE, Embase, Web of Science, and CINAHL. We included primary experimental and observational studies as well as case series studies addressing the safety, efficacy, and cost effectiveness of RNS in the treatment of drug-resistant pediatric epilepsy.

Results

Two systematic reviews of prospective and retrospective case series studies and four primary experimental studies were identified. The case series studies found that a large proportion of pediatric patients responded to RNS, with a reduction of between 50 and 75 percent in the frequency of seizures. The intensity and duration of seizures also decreased after using RNS. Adverse effects of the RNS implantation process were related to infections, erythema, and hematomas. Only one study (n=17) reported moderate adverse effects related to stimulation (dysesthetic pain in the upper and lower right limb), but there were no serious reactions leading to RNS discontinuation.

Conclusions

Randomized controlled trials in pediatric drug-resistant populations ineligible for brain surgery with adequate sample sizes are needed to determine the effectiveness of RNS in terms of seizure frequency, duration, and intensity. No cost-effectiveness studies have been conducted on RNS in this cohort.
PD147 Overview Of Multiplex Antigen Near-Patient Tests For Acute Respiratory Infections
Authors: Lynch Tom , McDowell Cillian , Burke Eimear , McLoughlin Carol , O’Neill Michelle , Spillane Susan , Ryan Máirín
Published: January 2025

Introduction

Multiplex antigen near-patient tests (NPTs) can detect multiple virus-specific antigens during acute infection. This project aimed to provide an overview of evidence on the effectiveness, advantages and disadvantages, and feasibility of multiplex antigen NPTs to identify common respiratory pathogens (including SARS-CoV-2 and one or both of the influenza and respiratory syncytial viruses) in residential and primary care settings.

Methods

A non-systematic literature search was conducted on the 28 July 2023 in the MEDLINE, Embase, Cochrane Library, and ClinicalTrials.gov databases to identify relevant literature. National and international agency websites were searched for guidance or recommendations relating to multiplex antigen NPTs. Relevant citations were screened and extracted by one reviewer and cross-checked by a second reviewer. Health technology assessments, systematic reviews, observational studies, and randomized and non-randomized controlled trials were considered eligible for inclusion in the overview. No formal quality appraisal of included documents was conducted. Due to the variation in study types, study findings were narratively assessed.

Results

Ten documents were identified in total. One complete prospective evaluation and seven incomplete clinical trials were identified. No relevant primary studies were identified for effectiveness outcomes such as time to appropriate treatment with antibiotics or antivirals. An evaluation published by the Haute Autorité de Santé in France reported that there was insufficient performance data regarding multiplex antigen NPTs in clinical practice. In a joint statement on respiratory virus testing, the Public Health Laboratory Network and Communicable Diseases Network in Australia indicated that antigen NPTs were not recommended due to their poor ability to identify influenza A.

Conclusions

Evidence on the effectiveness, advantages, disadvantages, and feasibility of multiplex antigen NPTs to detect common respiratory pathogens in primary and residential care settings is sparse. Studies are required to assess the diagnostic performance (relative to reverse transcription polymerase chain reaction tests) and clinical utility of multiplex antigen NPTs in residential and primary care settings.
PD153 Horizon Scanning Analysis Of The Obesity Medicines Pipeline
Authors: Akinbolade Sola , Nesworthy Jane , Fairbairn Ross , O’Connor Nicole , Hussain Amy , Harris Bethan , Craig Dawn
Published: January 2025

Introduction

Horizon scanning provides timely intelligence about innovative health technologies in clinical development by commercial and non-commercial organizations. The horizon scanning for obesity medicines, carried out by the National Institute for Health and Care Research Innovation Observatory (IO), aimed to identify emerging obesity medicines to inform decision-making by national stakeholders and to shape future research.

Methods

In July 2023, the IO utilized horizon scanning methodology to identify medicines for preventing and treating obesity either primarily or as a comorbidity. The scans included medicines in preclinical and clinical development (phase I, I/II, II, II/III, III, or IV) sponsored by industry and non-industry for all population groups. Trial locations included Australia, Canada, the European Union, the UK, and the USA. Data were collected from the IO’s internal database (the Medicines Innovation Database), ClinicalTrials.gov, the European Union Drug Regulating Authorities Clinical Trials Database, the World Health Organization International Clinical Trials Registry Platform, and the Citeline Pharmaprojects database. The data were systematically screened and analyzed.

Results

A total of 405 clinical trials were identified that evaluated 177 unique medicinal interventions. Among these, 47 unique preclinical interventions were identified from preclinical studies. A total of 256 (63%) trials were sponsored by industry, 139 (34%) by non-industry, and 10 (3%) by industry and non-industry jointly. The top five drug classes included anorectic or anti-obesity medicines (n=75; 42%), antihyperglycemics (n=24; 14%), anti-inflammatories (n=8; 5%), hepatoprotectants (n=7; 4%), and antihyperlipidemics (n=4; 2%). At the time of scanning, 48 (27%) medicines were unlicensed in the UK and 129 (73%) were not. Among the licensed medicines, 37 (77%) were off patent and 11 (23%) were on patent.

Conclusions

The IO’s horizon scanning process can identify and deliver timely intelligence to support decision-making and facilitate adoption of new medicines to target areas of unmet clinical need. The obesity medicines scan identified medicinal interventions in preclinical and clinical development and provides valuable insights into the trends and research gaps in preventing and treating obesity.
PD165 To What Extent Do Health Technology Assessment Bodies Cross-Reference Each Other In Their Reports?
Authors: Wagner Peter , Szawara Paula , Panda Sattwik Kumar , Kanthi Vinay M
Published: January 2025

Introduction

Due to different timing of drug launches across countries, published health technology assessment (HTA) findings from one country may impact HTA outcomes in other countries. The aim of our work was to identify the most influential HTA bodies by analyzing to what extent HTA bodies cross-reference each other in their HTA reports.

Methods

We analyzed the HTA reports on single drug assessments (SDA) published by 46 HTA bodies from 28 countries (and cross-country collaborations) with decision dates between January 2011 and November 2023. We searched the identified HTA reports by using natural language processing and a predefined set of keywords to identify whether, and to what extent, HTA bodies reference each other in their HTA reports. Additionally, we assessed if there is a trend over time in the cross-referencing, and whether any clusters could be identified.

Results

Based on the analysis of 24,793 SDAs, the National Institute for Health and Care Excellence (NICE) was referenced the most (in 4,198 HTA reports across 39 HTA bodies), followed by the Canadian Agency for Drugs and Technologies in Health (in 2,034 reports across 35 HTA bodies), and the Scottish Medicines Consortium (SMC) (in 1,960 reports across 31 HTA bodies). The HTA bodies that most often referenced other HTAs were the Agency for Health Technology Assessment and Tariff System, the Haute Autorité de santé, and NICE. Seven HTA bodies were not referenced in any HTA report, while four did not reference any other HTA body.

Conclusions

Our research shows that most of the analyzed HTA agencies not only referenced other HTA bodies in their HTA reports but were also referenced by other HTA bodies. The most often referenced HTA agencies were mostly from English-speaking countries, were well recognized, and had well defined methodologies.
PD170 Modification Of The All Wales Medicines Strategy Group Appraisal Process Provides Faster Access To Children’s Medicines In Wales
Authors: Wood Kelly , Varnava Alice , Lang Ruth , Williams Anthony , Coulson Professor James , Champion Andrew , Ganderton Claire
Published: January 2025

Introduction

Medicines routinely funded for use in Wales undergo health technology appraisal by the All Wales Medicines Strategy Group (AWMSG) or the National Institute for Health and Care Excellence (NICE). This includes pediatric license extensions (PLE) notwithstanding any existing advice in adults. A review of the PLE process was conducted with the aim of providing faster access to children’s medicines in Wales.

Methods

Data were collected for PLE appraisals of medicines previously approved for adults by the AWMSG or NICE that subsequently went through the original PLE process between January 2010 and December 2020, or a simplified PLE process between January 2021 and March 2023. Data were analyzed using descriptive statistics and a two-tailed t-test (unequal variance) to test the null hypothesis that the difference between the two means was zero. An alpha of less than 0.05 was considered significant. Feedback was obtained from relevant stakeholders including the Association of the British Pharmaceutical Industry (Wales) and the Royal College of Paediatrics and Child Health.

Results

The AWMSG issued positive recommendations for all PLE appraisals included in the data collected, and these were endorsed by the Welsh Government. Appraisals that went through the original PLE process (n=56) took a mean 229.8 days (standard deviation 55.6), whereas those that went through the simplified PLE process (n=15) took a mean 102.6 days (standard deviation 48.1; p < 0.0001). The rapid access to children’s medicines was welcomed by the Association of the British Pharmaceutical Industry and the Royal College of Paediatrics and Child Health.

Conclusions

Review of the 2020 and 2023 PLE processes facilitated faster access to clinically effective and cost-effective medicines for children in Wales. In March 2023, the AWMSG and the Welsh Government reviewed these results and agreed that because all PLE medicines were approved for use within Wales irrespective of the process used, the AWMSG would no longer be required to routinely appraise PLEs.