bluuebunny/crossref_metadata_2025 huggingface dataset:
PD122 Evaluating The Efficacy Of Cytokine Filtration In Cardiac Surgery For Endocarditis: A Comprehensive Study
Authors: Borin Marcus Carvalho , Martins Carina Rejane , dos Reis Daniel Pitchon , Coelho Ribeiro Geraldo Jose , Tupinambas Julia Teixeira , de Castro Zocrato Karina , de Almeida Carvalho Lelia Maria , de Freitas Marcela Pinto , da Gloria Cruvinel Horta Maria , Barbosa Mariana Michel , Torres Talim Mariza Cristina , Loureiro Bersan Sergio Adriano , Bruschi Kelles Silvana Marcia
Published: January 2025

Introduction

Despite medical advancements, endocarditis still results in high mortality rates. Surgery, while often essential, elevates the risk of hyperinflammation, sepsis, and cytokine release. The use of a cytokine filter to prevent this remains controversial. This study reviewed existing literature to assess the efficacy of cytokine filters and to support its integration into supplementary health services.

Methods

An exhaustive search of the MEDLINE, Cochrane Library, Embase, LILACS, and CytoSorbents Corporation databases was conducted to identify relevant meta-analyses and systematic reviews. The study focused on randomized controlled trials and case series studies assessing the efficacy of cytokine filtration. Key variables considered were the duration of antibiotic treatment, severity of endocarditis, and surgical treatment rationale. These factors were crucial for evaluating clinical outcomes and patient survival after surgery.

Results

The systematic reviews yielded mixed outcomes. Two found no benefits for hemoadsorption, while one found that it reduced mortality rates and intensive care unit stays based on observational studies. Randomized controlled trials, however, showed no significant impact for cytokine filters on mortality rates or postoperative hemodynamic parameters. In contrast, case series studies reported potential benefits, but these results were confounded by biases in patient allocation and failure to account for critical variables like antibiotic treatment duration, case severity, and surgical rationale. These discrepancies highlight the complexity of evaluating the effectiveness of cytokine filtration in surgical settings.

Conclusions

Randomized and non-randomized controlled trials on the role of cytokine filters in cardiac surgery for endocarditis reported contradictory findings. Only case series studies suggested benefits from cytokine filters, necessitating further high quality research before recommending their widespread use. Understanding the implications of these results is essential, underscoring the need for more rigorous studies to resolve these inconsistencies.
PD124 Genicular Artery Embolization For The Treatment Of Knee Osteoarthritis: A Systematic Review And Meta-analysis
Authors: González-Hernández Yadira , Hernández-Yumar Aránzazu , De Armas-Castellano Aythami , Valcárcel-Nazco Cristina , Carmona-Rodríguez Montse , Trujillo-Martín Mar , Pino-Sedeño Tasmania del
Published: January 2025

Introduction

The genicular artery embolization (GAE) procedure has been recently adopted for the management of pain secondary to inflammatory diseases of the locomotor apparatus. The number of studies assessing its use in patients with knee osteoarthritis (KO) has been increasing in recent years.

Methods

We included two randomized controlled trials (RCTs) evaluating the use of GAE in patients with chronic pain secondary to KO. A cost analysis was also conducted to compare the costs of GAE and standard treatment from the perspective of the Spanish National Health System over a time horizon of one year. The potential improvement in quality-adjusted life-years necessary to consider GEA as cost effective for this indication was estimated. We also ran extensive sensitivity analyses.

Results

Estimates for pain showed contradictory results, and no significant differences were observed between the two treatments with respect to overall function, health-related quality of life (HRQoL), and need for pain medication. No serious complications or major adverse events were observed. The quality of evidence was assessed by GRADE as moderate to low. The cost analysis showed that GAE results in an incremental cost of EUR3,432.37 per patient. Sensitivity analyses revealed a wide range within which the incremental cost can vary.

Conclusions

There are insufficient data to discern any differences between GAE and standard treatment for patients with KO in terms of pain, function, HRQoL, need for analgesics, and rates of adverse events and complications. Larger RCTs are required to evaluate the effect of GAE in patients with chronic pain secondary to KO and to determine whether its additional cost is warranted.
PD126 A Systematic Review Of Interval Cancer Rates In Colonoscopy Screening For Colorectal Cancer
Authors: Hamashima Chisato , Abe Koichiro , Terasawa Teruhiko , Hosono Satoyo , Katayama Takakafumi , Hoshi Keika , Tadano Toshihiro , Sasaki Seiju
Published: January 2025

Introduction

Colonoscopy screening has been suggested as a potential primary screening method for colorectal cancer (CRC). A 10-year screening interval has been recommended by some academic societies, but the scientific evidence for this needs to be more comprehensive. We performed a systematic review of interval cancer rates in colonoscopy screening in an asymptomatic population.

Methods

We searched the PubMed and Ichushi-Web bibliographic databases for papers published from inception to September 2022. The search terms were “colorectal cancer screening”, “interval cancer”, and “total colonoscopy”. Randomized controlled trials and observational studies were included. The target population was an asymptomatic average-risk population with no polyps or adenomas at the index colonoscopy. Advanced neoplasia (AN) was defined as CRC or an adenoma at least 10 mm in size with a villous component or high-grade dysplasia. The incidence rates of interval CRC and AN per 100,000 person-years (PYs) were estimated.

Results

Of the 694 potentially eligible articles, 15 were included. Rates of interval CRC and AN were reported in 15 and 11 studies, respectively. For the meta-analysis, 287,602 patients with negative colonoscopy results were included, with an average follow-up of 7.98 years. Negative colonoscopy results were defined as no adenoma present. The incidence rate per 100,000 PYs was 9.57 (95% confidence interval [CI]: 2.06, 29.94) for interval CRC and 311.5 (95%CI: 153.4, 550.7) for AN. Similar results were obtained even when a negative colonoscopy result was defined as no polyps present.

Conclusions

The selected studies were heterogeneous with respect to the target population, follow-up years, and patient characteristics. Although interval AN was reported in all studies, the interval cancer rate was low after a negative result at the index colonoscopy. While the screening interval might be defined as long term, a definitive screening interval could not be recommended because of insufficient evidence.
PD129 Extension Of An Influenza Vaccination Program To Include Those Aged 50 To 64 Years: A Rapid Health Technology Assessment Approach
Authors: Cardwell Karen , Mc Loughlin Carol , Swanton Dana , Harrington Patricia , Teljeur Conor , Ryan Máirín
Published: January 2025

Introduction

Influenza causes considerable morbidity and mortality. Vaccine effectiveness is variable due to the evolution of influenza viruses and antigenic mismatch. In Ireland, influenza vaccination is reimbursed for all individuals aged at least 65 years as well as those who are at increased risk of exposure or severe disease. However, it is unclear whether reimbursement should be extended from 2023 to 2024 to those aged 50 to 64 years in the general population.

Methods

A rapid health technology assessment (HTA) format was chosen due to time constraints, as the findings were to inform a decision for the upcoming influenza season only and would not result in a permanent change to the vaccination program. National sentinel surveillance data (for seasons 2010-2011 to 2022-2023) and hospitalization data for the publicly funded healthcare system (for years 2010-2022) were used. Central Statistics Office data indicated that the projected population for 2023 of those aged 50 to 64 years was 914,379. Given the one-year timeframe, the economic evaluation was limited to a costing analysis to estimate the potential cost associated with expanding the program.

Results

Surveillance data showed year-on-year variability in influenza incidence. Excluding the years not considered representative due to the influence of COVID-19, on average 12.9 percent (range 9 to 17%) of notified cases, 11.8 percent (range 9 to 17%) of influenza-related hospital admissions, 23.2 percent (range 12 to 34%) of influenza-related intensive care admissions, and 9.6 percent (range 0 to 44%) of influenza-related deaths per year were in people aged 50 to 64 years. Data were not disaggregated according to vaccination or risk status. The estimated mean incremental cost of extending eligibility for the 2023 to 2024 season (based on a 35% uptake rate) was EUR2.27 million (range EUR1.9 million to EUR2.65 million, depending on vaccine costs).

Conclusions

Given the relatively modest number of influenza-related hospitalizations in the 50-
to 64-year age group, and the substantial year-on-year variability in vaccine effectiveness, the potential for a reduction in demand for hospital care is likely to be small. While a rapid HTA approach facilitates timely decision support, it creates challenges for exploring the more complex facets of an intervention.
PD133 Exploring Real-World Data Based Financing Models For Lung Cancer Immunotherapy In The Czech Republic
Authors: Bártová Adéla , Říhová Barbora
Published: January 2025

Introduction

In alignment with recommendations from the Czech Society for Oncology, immunotherapy is gaining prominence in managing metastatic lung cancer. Public health insurance in the Czech Republic covers immunotherapy for defined categories of these malignancies. Our study aimed to evaluate the impact of introducing performance-based risk-sharing agreements (PBRSA) on budgetary considerations for immunotherapy treatment.

Methods

In collaboration with the Masaryk Memorial Cancer Institute in Brno, we conducted a retrospective analysis of 127 patients with lung cancer who were treated with immunotherapy (42 received nivolumab) between 2018 and 2022. We explored the reimbursed indications for pembrolizumab, nivolumab, atezolizumab, and durvalumab. Real-world progression-free survival (PFS) data from the medical records were compared with PFS data from randomized controlled trials. Patients were classified as either successfully or unsuccessfully treated according to the PFS threshold established in the comparator arm of the respective trials. Additionally, we explored a hypothetical scenario involving the potential implementation of PBRSA depending on the level of outcome achieved.

Results

In patients with advanced lung cancer who had received prior chemotherapy, nivolumab succeeded in 29 patients but failed to meet the defined success threshold in 13 patients. Unsuccessful cases incurred an average cost of EUR9,300 per patient over a median treatment period of 1.7 months. In contrast, the cost of successful treatment exceeded EUR29,700 per patient, which was sustained for a median treatment duration of 5.5 months. Manufacturers could cover up to 66 percent of the cost associated with unsuccessful treatment in the 13 patients, which would exceed EUR41,000. This approach might cover the expenses for one additional patient. The same calculation was performed for all other checkpoint inhibitors.

Conclusions

The analysis emphasizes the vital role of risk-sharing agreements in enhancing the affordability and sustainability of high-cost advanced therapies. Discrepancies between real-world clinical data and registration studies challenge full reimbursement sustainability. By redistributing financial responsibility, PBRSA alleviate costs for insurers and simplify market entry for manufacturers, contributing to a dynamic and inclusive healthcare landscape.
PD136 Re-evaluation Of Chest X-Ray Screening For Lung Cancer With Consideration Of Study Context
Authors: Hamashima Chisato , Terasawa Teruhiko , Kataoka Yuki , Anan Keisuke , Hosono Satoyo
Published: January 2025

Introduction

Lung cancer causes a heavy burden worldwide and an efficient screening program is needed. Although low dose computed tomography screening has become mainstream for lung cancer screening, chest X-ray (CXR) screening has continued in Japan. We re-evaluated the efficacy and effectiveness of CXR screening and reconsidered the context of the studies.

Methods

We performed a systematic review and meta-analysis of CXR screening for lung cancer. The study design included randomized controlled trials (RCTs), cohort studies, and case-control studies (CCSs) that evaluated the efficacy or effectiveness of CXR screening. Searches were conducted in the PubMed, Cochrane Library, Web of Science, and Ichushi-Web databases for literature published up to April 2022. We examined the settings of the selected studies.

Results

From about 4,000 candidate articles, six RCTs, one cohort study, and six CCSs were selected. Five RCTs were conducted in the 1960s and 1970s, except for the Prostate, Lung, Colorectal, and Ovarian Cancer (PLCO) Screening Trial. Six CCSs conducted in Japan reported reductions in mortality from lung cancer. A meta-analysis of the six CCS showed a 47 percent reduction in mortality from lung cancer (adjusted odds ratio 0.53, 95% confidence interval: 0.50, 0.63). In the PLCO trial, mortality was reduced by nine percent at the six-year follow up, but this result was not statistically significant. The histological distribution of lung cancer was similar between the PLCO trial and the Japanese CCSs.

Conclusions

The dilution effect might affect the PLCO trial results because of extended follow up beyond the lead time of CXR screening. Although evidence on CXR screening for lung cancer is limited, CXR screening might be adopted in Japan considering the histological changes in lung cancer that have occurred due to the decline in smoking rate.
PD137 Reviewing The Health Technology Assessment And Regulatory Policy Landscape On Acceptability Standards For Real-World Evidence - Initial Findings
Authors: Cresswell Katharine , Claire Ravinder , Avsar Tuba Saygin , Hudson Thomas , Purcell Rose , Peeters Rita , Jensen Lotte Groth , Chen Yun , Mogensen Stine Hasling , Bisordi Fabio , Crane Gracy , Dawoud Dalia , Colvin Heather
Published: January 2025

Introduction

There is an increasing number of policy and guidance documents on the use and acceptability of real-world evidence (RWE) to support regulatory and health technology assessment (HTA) decision-making. The Innovative Health Initiative Integration of Heterogeneous Data and Evidence towards Regulatory and HTA Acceptance (IDERHA) partnership is undertaking a global landscape review of these documents to understand where there is consensus and divergence, and where further policy development is needed.

Methods

A literature search of the MEDLINE and Embase databases was performed, in addition to handsearching the websites of specific HTA and regulatory organizations. All policies, standards, frameworks, and guidance documents on requirements for acceptable RWE data use published from 2017 were included. Two reviewers independently extracted data using a standard data extraction form that was pilot tested before use. Any discrepancies between the reviewers were resolved by consensus. Extracted data are currently being analyzed by researchers with regulatory or HTA expertise. A workshop held in October 2023 sought input from experts on analysis plans.

Results

The initial literature search yielded 3,184 results. After screening against the inclusion criteria, a total of 87 documents were selected for full-text review (21 HTA and 62 regulatory documents). Of these, 32 were identified as key documents and prioritized for initial review. Key themes in the documents, including transparency, data collection, study design, and data quality, were identified and validated in a workshop with five regulatory or HTA experts. Data extraction is ongoing for the remaining documents and any further themes identified will be added. Any gaps and areas of divergence will be identified, so they can be addressed by future IDERHA work.

Conclusions

This review assessed the increasingly complex global landscape of regulatory and HTA policies and guidance on the use of RWE. Through the exploration of similarities, differences, and gaps in these policies, this work will extend the current understanding of best practice and identify areas that need development of further guidance.
PD140 A Strengths, Weaknesses, Opportunities, And Threats Analysis Of Prehabilitation Before Elective Surgery For Frail Elderly Patients
Authors: Felgner Susanne , Fuchs Tamina , Pfab Carina , Sontag Anna Frederike , Eckhardt Helene , Quentin Wilm , Weber Zoe , Kiselev Jörn , Rombey Tanja
Published: January 2025

Introduction

As part of the prehabilitation of elderly frail or pre-frail patients prior to elective surgery (PRAEP-GO) randomized controlled trial (NCT04418271), we are investigating the approach of multimodal prehabilitation (e.g., physiotherapy) combined with frailty screening and a shared decision-making conference for frail elderly patients prior to elective surgery. The aim of this analysis was to identify strengths, weaknesses, opportunities, and risks of the PRAEP-GO intervention to inform its implementation within the German healthcare system.

Methods

This strengths, weaknesses, opportunities, and threats (SWOT) analysis was based on two expert interview studies and a realist review aiming to identify factors that might facilitate or hinder the implementation of prehabilitation from the perspectives of health service providers and patients. As part of the SWOT analysis, one author categorized these factors into external and internal factors, assessed their importance (scale of one [no/barely] to five [very high] points) and visualized it using radar plots, and assigned them to the categories of strengths, weaknesses, opportunities, or threats. Strategies were then developed to address the following combinations of the categories: strengths/opportunities, strengths/threats, weaknesses/opportunities, and weaknesses/threats.

Results

In the expert interviews and the realist review, 45 facilitating and 28 hindering factors focusing on health service provision, patients, and the implementation of prehabilitation for frail patients in general were identified. In the SWOT analysis these factors were assigned to 60 internal (resource analysis) and 13 external factors (environmental analysis), and were further sorted into eight and five categories, respectively; for example, “equipment” and “staff” (these two categories were assigned the highest importance [5 points]). Strategies for utilizing strengths and opportunities included, for example, promotion of interdisciplinary cooperation between service providers. The weaknesses and risks of PRAEP-GO might be mitigated, for example, by improving infrastructures.

Conclusions

A variety of factors in different categories may facilitate or hinder the implementation of PRAEP-GO. Based on preliminary results from this analysis, the identified strengths of prehabilitation outweigh its weaknesses, but when analyzing the external factors, the identified risks outweigh the benefits. Thus, the implementation of prehabilitation should be prepared by optimizing framework conditions (e.g., transportation for patients and allocation of personnel).
PD141 Health Technology Assessment Methodology Approach For Precision Personalized Medicine: An Innovative Public Procurement Case Study
Authors: Dobrzynska Agnieszka , Garcia-Sanz Patricia Julia , Rosario Lozano María Piedad , Aguilera-Cobos Lorena , Blasco-Amaro Juan Antonio
Published: January 2025

Introduction

Innovative public procurement (IPP) is a driver of innovation across sectors. IPP involves the strategic acquisition of cutting-edge technologies and solutions by public entities in collaboration with the private sector. This approach aims to leverage the potential of “omics” technologies, such as genomics and proteomics, within the public sector to advance healthcare solutions.

Methods

The IPP process comprises key phases such as needs identification, solicitation preparation, execution, evaluation and awarding, and impact and assessment. The methodology applied was based on the Rapid Assessment Tool for Omics Technologies developed by the Andalusian Agency for Health Technology Assessment, accompanied by clinical validity reports from the impact and evaluation phase of the Technical Office for IPP of Andalusia. The aim was to assess the clinical validation results of two omics technologies, that is, two diagnostic tests. A systematic review was performed to identify existing evidence. We also addressed the challenges associated with implementing the Rapid Assessment Tool in the IPP process.

Results

Systematic reviews identifying evidence on the clinical validity and utility of omics technologies provided a foundation for the subsequent evaluation of two technologies in development, once the clinical trials had finished. An analysis of scientific evidence, together with the compilation of information provided by industry, was conducted through a questionnaire and clinical data derived from the company’s studies. The analysis of clinical and diagnostic validity was not conclusive. We delivered the final assessment report to support decision-making in the public health system of Andalusia.

Conclusions

Both assessed technologies presented a high degree of innovation, but different challenges and issues were identified during the application of the Rapid Assessment Tool for Omics Technologies. Further improvement in IPP procedures for innovative technologies in Andalusia, including integration of our methodological approach at the start the IPP process, could facilitate the acquisition of cutting-edge technologies in collaboration with public entities.
PD149 Comparison Of The Draft European Union Health Technology Assessment Template With Germany’s AMNOG Template
Authors: Reuter Eva-Maria , Klein-Hessling Thomas , Rittmeyer Carolin , Schweitzer Maria Katharina , Werner Sebastian
Published: January 2025

Introduction

European Union Health Technology Assessment (EU HTA) aims to use resources more efficiently, ensure high quality assessments, and promote the widespread availability of medicinal products. We compared the draft EU HTA template (EUnetHTA21 submission dossier template) with Germany’s Arzneimittelmarkt-Neuordnungsgesetz (AMNOG) template to assess their conformity and to estimate whether the draft EU HTA template requires more or less effort than the AMNOG template.

Methods

Four experts (two statisticians and two medical writers) independently compared 39 categories of both templates following the four-eyes principle. The categories were defined based on the formal and methodological requirements as well as on the specific instructions regarding content, data presentation, and documentation in both templates. We assessed each category for conformity between templates (low, medium, high) and determined the EUnetHTA21 template’s additional effort (much less, less, equivalent, more, much more) relative to the AMNOG template. The comparison was carried out under the assumption that only a single research question on a population, intervention, comparator, and outcome (PICO) would be addressed in both submission procedures.

Results

We found that the draft EUnetHTA21 template and the AMNOG template had substantial conformity in most categories (21/39), indicating comparable or identical formal and methodological requirements. For 10 of the 39 categories the conformity between templates was rated as medium. Low conformity was found for eight of the 39 categories, including categories outside one template’s scope. For most categories (20/39) we expect an equivalent effort per PICO. More or much more effort is expected for 13 of the 39 categories. For only six of the 39 categories, less or much less effort is expected for the draft EUnetHTA21 template, compared with the AMNOG template.

Conclusions

The analysis highlights strong similarities between the templates but shows increased effort per PICO with the draft EUnetHTA21 template. This effort will be further increased with multiple PICO questions. This could challenge the feasibility of the EU HTA process, especially considering the short timeframe required. The template comparison will be updated once the final EU HTA template is available.
PD168 Developing And Implementing A Value Framework For Rapid Health Technology Assessments: Enhancing Evidence-Informed Coverage In Resource-Constrained Settings
Authors: Alcaraz Andrea , Argento Fernando , Alfie Verónica , Marti Sebastian Garcia , Bardach Ariel , Ciapponi Agustín , Augustovski Federico , Pichon-Riviere Andres
Published: January 2025

Introduction

Value frameworks play a crucial role in bridging the gap between evidence and decision-making in health care, particularly in settings with limited budgets. In this study, we present the results of an implemented value framework (VF) to provide coverage recommendations in rapid health technology assessment (HTA) reports.

Methods

A search was performed to identify existing HTA frameworks. Relevant criteria and methods of assessment were then selected. A color-coded system was applied to categorize each criterion. A Delphi panel was conducted to determine the overall recommendations and to weigh the criteria and correlate them with the recommendations. To assess the performance, we reviewed the results of rapid HTA reports from the last five years.

Results

The value framework had three domains. We adapted widely used methodologies for the quality of evidence and net benefit domains. The economic impact domain was the most complex to assess, so a customized method was developed. Analysis of 265 HTAs revealed the distribution of recommendations across various criteria and technology types. Most recommendations were for drugs (40.5%) or for therapeutic procedures (36%). Among the final recommendations, 0.8 percent were favorable, 19.7 percent were uncertain, and 44 percent were unfavorable.

Conclusions

The VF demonstrated its versatility and practicality in meeting the needs of rapid HTA requesters and facilitating evidence-informed decision-making. The VF serves as a valuable tool for conducting adaptive rapid HTAs and supports decision-making processes in Argentina and similar contexts.
PD172 Off-Label Rituximab As A First-Line Immunosuppressant Treatment For Generalized Myasthenia Gravis In Wales
Authors: Elliott Clare , Woodland Gail , Haines David , Coulson James
Published: January 2025

Introduction

Generalized myasthenia gravis (gMG) is a chronic autoimmune disorder that leads to muscle weakness and fatigue. Initial treatment is with pyridostigmine and corticosteroids, but if these are ineffective off-label immunosuppressants are used. There is some published evidence that rituximab, an anti-CD20 monoclonal antibody, may be effective in the treatment of gMG, although it is unlicensed for this condition.

Methods

Evidence for rituximab as a fourth-line or later immunosuppressant treatment for refractory gMG was assessed using the One Wales Medicines (OWM) process and made available in 2019. OWM provides an access route to medicines for specific patient cohorts where no licensed treatments are routinely available. A review in 2022 identified new evidence suggesting that lower dose rituximab could be an effective first-line immunosuppressant treatment for gMG. Clinicians confirmed an unmet need, so a reassessment by OWM was considered appropriate. Clinical and cost effectiveness were assessed through a literature search, budget impact analysis, and clinical expert opinion.

Results

The OWM Advisory Group assessed the evidence outlined in an evidence summary report. Clinical experts also provided the clinical context and current treatment options for patients with gMG. Overall, it was found that rituximab as a first-line treatment provided a potential improvement in patient outcomes and value for money, compared with current therapy. It was, therefore, supported for routine use in April 2023. Starting and stopping criteria for rituximab were developed in collaboration with clinical experts. All patients in Wales who meet the agreed starting criteria will now be given the option of routine treatment with rituximab.

Conclusions

The OWM process allows routine access to rituximab as a first-line immunosuppressant treatment for gMG. The OWM team are collaborating with clinicians across Wales to instigate a new systematic method for collecting and analyzing patient outcomes. These real-world data will be used to assess benefit to ensure continued access to the best treatments for Welsh patients.
PD175 Using Machine Learning To Optimize Systematic Literature Reviews
Authors: Chacko Akshay
Published: January 2025

Introduction

Screening and selecting publications are very time consuming when conducting systematic literature reviews. Currently, in the field of robotic-assisted surgery (RAS) there is an average of 12 to 15 studies published daily, making manual data management unsustainable. We aimed to investigate how machine learning (ML) can be used to optimize the manual processes of literature reviews.

Methods

New RAS publications in PubMed, Scopus, and Embase are routinely screened for relevancy and then tagged with metadata to aid future analysis. A curated library of approximately 40,000 tagged RAS publications served as our training dataset. To support manual screening and tagging efforts, multiple ML models were benchmarked, including logistic regression, decision trees, and gradient boosting. All model implementations came from the Python scikit-learn package. The evaluation metric for this study was the F1 score, and the fields of interest tagged were procedure type and surgical approach. Models were trained on publication abstracts and compared with a baseline keyword search to measure changes in performance.

Results

The findings demonstrated that ML models can classify key metadata with high levels of accuracy. The decision tree model correctly labeled the five most common procedures in the dataset, with an average F1 score of approximately 0.90. This same model predicted surgical approach with an average F1 score of 0.84. It is important to note that different models performed best in different scenarios. To compensate for this variability, all models were fed into a stacking classifier—an ensemble model that takes the output of other models as input training data.

Conclusions

It is evident that ML models can reduce the cognitive burden of clinical librarians and shift their role from hand-screening papers to validating ML predictions. Future work may involve comparing the performance of traditional ML models with large language models (LLMs) to further improve F1 scores and reduce class imbalances.
PD179 Navigating Health Crises In Emerging Economies: A Comprehensive Examination Of COVID-19’s Influence On Health Care Access And Resilience
Authors: Tibet Birol , Ozdemir Ekin Begum , Erkut Yaren , Okcun Selin , Kurnaz Mustafa , Kockaya Guvenc
Published: January 2025

Introduction

This research focuses on the impact of the COVID-19 pandemic on health care in emerging markets. It examines the pandemic’s effects on emergency surgeries, medical consultations, maternal care, primary health care services, and surgical interventions.

Methods

This study focused on assessing the effect of COVID-19 on healthcare services in emerging markets from 2019 to 2021. It involved a literature review of academic articles, health reports, and government data, targeting the pandemic’s effect on healthcare access. Data were collected from official health records in Brazil, China, Ethiopia, Egypt, Poland, Qatar, Sub-Saharan Africa, Thailand, and Türkiye. The analysis focused on emergency surgeries, outpatient visits, hospital admissions, primary health care, prenatal and maternal care, clinic visits for cardiac implantable electronic devices (CIED), and general surgeries, aiming to understand changes in health care access during and after the pandemic.

Results

Emergency surgeries in Ethiopia decreased by 77 percent, while in Egypt there was a 66.4 percent reduction in chest clinic visits. Outpatient and hospital admissions fell by 7 to 17 percent in Sub-Saharan Africa. In China, hospital, primary care, and inpatient visits declined by 33, 71, and 42 percent, respectively. In Qatar, physical healthcare visits fell by 36 percent, though virtual consultations increased notably. CIED visits in Poland fell by 26 percent in 2020. Thailand struggled with increased COVID-19 cases and deaths in 2021, whereas Brazil’s health care services were significantly reduced (42.6% reduction in screenings and 59.7% reduction in surgeries). In Türkiye, there was a 35 percent drop in hospital visits and a 15 percent drop in prescriptions, but with increased costs per visit (0.09%) and per prescription (42.3%).

Conclusions

This study highlights the profound impact of COVID-19 on health care in emerging markets, showing significant disruptions in services like surgeries and outpatient visits. The pandemic emphasized the necessity of robust, adaptable healthcare systems and accelerated the shift to digital health services. These findings urge the strengthening of healthcare in emerging markets to prepare them for future global health challenges.
PD187 Health Technology Assessment Training Program in Ukraine: An Example Of Collaborative Capacity Building
Authors: Kievit Wietske , Piniazhko Oresta , Sucu Rabia , Oortwijn Wija
Published: January 2025

Introduction

In 2017 health technology assessment (HTA) was included in the Ukrainian Health Law fundamentals, and its implementation has accelerated as it became mandatory by 2020. As part of the Safe, Affordable, and Effective Medicines for Ukrainians project, funded by the U.S. Agency for International Development (USAID), to support the Ministry of Health in Ukraine, we developed, conducted, and evaluated a need-based HTA training program for doers, users, and trainers from July 2022 to August 2023.

Methods

We developed the training program based on a review of current academic HTA master’s and advanced programs globally, as well as an assessment of the training needs of the intended participants. The program consisted of five modules comprising 30 online sessions of four hours, with a mixture of lectures and work sessions complemented with self-study assignments and a targeted train-the-trainers program. We asked participants to report on their level of confidence in each learning objective at the start and the end of each module. A questionnaire was administered at the end of the program to evaluate participants’ satisfaction with study load, course content, and organization. This research was funded by USAID, reference number RFP-2021-11-18, through a contract with Management Sciences for Health under AID-121-C-17-00004. The contents do not necessarily reflect the views of USAID or the U.S. Government.

Results

The five modules focused on the following: introduction to HTA; comparative effectiveness; health economics; qualitative evidence synthesis; patient and public involvement; and ethical, sociocultural, legal, and other relevant issues. Between 30 and 75 persons attended one or more of the training sessions. For each module, a learning effect was observed among participants. Among the 42 survey respondents, the average grade was excellent (9.5/10). The setup, organization, and content of the program and material provided, as well as the level of expertise provided by the teachers, were rated as very good.

Conclusions

Participants agreed that the program was relevant and contributed to their professional growth. Twelve academics and staff from the HTA body were trained to help sustain the program in Ukraine. The project resulted in HTA training for doers, users, and trainers in Ukraine, and can be an example for other countries wishing to increase sustainable HTA capacity.
PD198 A Review Of Regulatory Theory To Inform Discussion On Aligning Regulation And Health Technology Assessment Of Medical Devices
Authors: Harkin Kathleen , Sorensen Jan , Thomas Steve
Published: January 2025

Introduction

If regulation and health technology assessment (HTA) are to be aligned, we must understand regulation and regulatory policy goals. Furthermore, regulatory policy is most likely to achieve its goals if it is informed by theory and underpinned by evidence. However, regulatory theories are rarely explored by HTA bodies. Therefore, this study explored regulatory theories as they relate to medical device regulation.

Methods

Literature describing regulatory theories was reviewed and synthesized narratively to explain what is meant by regulation and why governments regulate. Competing theories were discussed, reflecting on their relevance to the regulation of medical devices.

Results

The theory of perfect competition suggests that, when key assumptions hold true, market forces will result in the most efficient allocation of resources. However, when they don’t, market failure results. Governments frequently regulate such markets. Stigler suggests that they do so to serve either public or private interests. Market failure is the norm in health care, so it requires regulation. If medical device regulation is intended to serve the public interest, it should be patient-centered, balance market power amongst many producers, reduce information asymmetry, and ensure that only safe and effective devices enter the market. HTA assumes a public interest perspective.

Conclusions

Regulations that maintain the confidentiality of clinical evidence underpinning medical devices and allow them to enter healthcare markets with insufficient evidence of efficacy do not serve the public interest. This raises the questions of whose interests does medical device regulation serve, and can HTA align with this?
PD201 Rare Disease Product Approvals: The Changing Regulatory And Health Technology Assessment Landscape Between 2013 And 2022
Authors: Wang Tina , Lara Juan , Magda Bujar , Sola Belen , McAuslane Neil
Published: January 2025

Introduction

Globally, 7,000 rare diseases affect 300 million people, which poses challenges for developing treatments in these discrete patient populations. Developing medicines for rare diseases requires innovation, but despite regulatory incentives challenges persist for health technology assessment (HTA) and payers. Understanding the current regulatory and HTA decision-making landscape for orphan products is essential for all stakeholders.

Methods

Data on new active substances (NAS) approvals (2013 to 2022) by the European Medicines Agency, the United States Food and Drug Administration (FDA), the Pharmaceuticals and Medical Devices Agency (PMDA), Swissmedic, and the Therapeutic Goods Administration were collected to analyze the timing, approval pathways, and global rollout trends for orphan and non-orphan products. Data were collected from HTA agencies in Australia, England, France, Germany, the Netherlands, Poland, Scotland, and Sweden to explore synchronization in decision timing and first HTA decision. Comparative analysis encompassed decision frameworks and funding mechanisms for orphan products among HTA agencies.

Results

Orphan drug approvals increased in the past decade, with the FDA having the highest designation rate (55% for 2018 to 2022). Flexible pathways, mostly used by the FDA (92%) and the PMDA (100%), expedited orphan drug reviews. However, international submissions for orphan drugs experienced longer gaps than non-orphan drugs across the jurisdictions. Divergence in rollout timing to HTA and recommendation resulted from varied submission strategies and review processes. Only the Scottish Medicines Consortium had a dedicated orphan pathway. In England, the National Institute for Health and Care Excellence patient access and managed access agreements (84% of orphan recommendations) and cancer drug fund (45% of orphan recommendations) facilitated patient access.

Conclusions

The study shows a decade long rise in global orphan drug approvals, underpinned by regulatory flexibility, particularly by the FDA and the PMDA. Identified divergences in decision frameworks among regulatory and HTA agencies, as well as HTA agencies themselves, call for increased stakeholder alignment. This necessitates synchronizing evidence generation during development and improving decision frameworks for streamlined review and reimbursement processes.
PD202 Reimbursement Success For Pharmaceutical Products With Noncomparative Data: Are Health Technology Assessment Bodies Well Suited To Embrace Innovations?
Authors: Kolukfaki Talya , Fineberg Reut , Leong Katherine , Macaulay Richard
Published: January 2025

Introduction

Randomized controlled trials are ideal for securing marketing authorization (MA) for pharmaceuticals. However, ethical and feasibility constraints have led to the use of noncomparative trials by regulatory bodies to balance evidence uncertainty and patient access, especially for medicines addressing rare diseases or unmet healthcare needs. This research focused on pharmaceuticals approved with noncomparative data and how these translate into reimbursement outcomes.

Methods

Indications approved by the European Medicines Agency (EMA) between January 2018 and October 2023 based on noncomparative data were identified. Generics and biosimilars were excluded. Approvals based on non-comparative trial data were identified from European public assessment reports and from “Procedural steps taken and scientific information after authorization” documents. The latter were also used to identify changes in MAs after submission of new data. Missing trial information was extracted from ClinicalTrials.gov. Health technology assessment (HTA) outcomes from the EU4Health programme and the UK were extracted from agency websites.

Results

The EMA approved 46 indications based on noncomparative data: 18 of the 46 (39%) received full MA, 23 (50%) received conditional MA (CMA), and five (11%) had a CMA converted to full MA with additional data. The results for HTAs conducted in various countries were as follows:

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England: 29 of 46 (63%) indications assessed, (23/29 [79%] recommended);

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Scotland: 28 of 46 (61%) indications assessed, (20/28 [71%] recommended);

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France: 33 of 46 (72%) indications assessed, (7/33 [21%] received Amélioration du Service Médical Rendu level I to III);

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Germany: 37 of 46 (80%) indications assessed, (2/37 [5%] achieved additional benefit);

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Italy: 37 of 46 (80%) products assessed, (33/37 [89%] reimbursed); and

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Spain: 34 of 46 (74%) products assessed, (23/34 [68%] reimbursed).

Conclusions

Indications approved by the EMA with noncomparative data achieved mixed HTA outcomes in the EU4Health programme and the UK. More negative outcomes were seen in markets with clinical-benefit payer archetypes (France and Germany). All conversions from CMA to full MA occurred within the last 24 months.
PD208 Optimal Scheme For Molecular Diagnosis Of BRCA1/2 Gene Mutations In Patients With Breast Cancer In Poland - Cost-Utility Analysis
Authors: Książek Paweł , Muzyka Maurycy , Nadzieja-Kozioł Agnieszka , Dziadek Karolina , Pruszko Cezary
Published: January 2025

Introduction

Breast cancer is the most frequently diagnosed cancer among women in Poland. Molecular diagnosis of cancer, including identification of mutations in the BReast CAncer 1 and 2 (BRCA1/2) genes, is publicly funded in Poland. However, the diagnostic process is hampered by various billing models that use different testing materials and methods, so the diagnostic scheme is not considered optimal by physicians or patients.

Methods

A cost-utility analysis was conducted to evaluate the cost effectiveness of modifying the molecular diagnosis protocol for breast cancer in Poland. The analysis compared the current diagnostic method with a new schema, which involves next-generation sequencing (NGS) for mutations in the BRCA1/2 genes for all patients with breast cancer. The model was developed using a comprehensive review of relevant literature, reimbursement data, and consultations with clinical experts.

Results

If all patients with breast cancer undergo molecular diagnosis, the number of BRCA1/2 gene mutation carriers detected will increase from 213 to 971 per year. Despite the increase in costs to the public payer, compared with the existing scenario, the incremental health effect of the new scenario generated an incremental cost-effectiveness ratio of EUR37,274 per quality-adjusted life-year. This finding effectively disproves the claim that it is not cost effective to perform tests using the NGS technique in all patients with breast cancer.

Conclusions

As a part of the cost-utility analysis it was shown that patients newly diagnosed with breast cancer should undergo molecular testing for mutations in the BRCA1/2 genes using NGS. This schema ensures patients’ access to appropriate targeted therapy. In addition, extended diagnosis of family members can prevent cancer in relatives who carry the mutation.
PD212 A Model-Based Study To Estimate Medicare Payment Methods With Chinese Characteristics – The Diagnosis Intervention Packet Policy
Authors: Fan Chengxin , Yin Wenqiang
Published: January 2025

Introduction

The diagnosis intervention packet (DIP) is a health insurance payment method with Chinese characteristics. Because the DIP is easy to understand and operable, it has become the main payment method promoted by China’s National Health Insurance Bureau. The development and reform of the DIP policy, which was introduced in 2020, is worth evaluating.

Methods

Policy texts were selected using conceptual sampling and expert interviews. The basic framework of policy evaluation was determined using text mining and statistical analysis methods. Word frequency analysis of the DIP payment policy content was undertaken using NVivo and Gephi software to compare the scope of concern before and after policy implementation. Quantitative evaluation of representative DIP health insurance payment policy content at the national level in China before and after policy implementation (2020 to 2022) was conducted by constructing a text mining and policy modeling consistency (TM-PMC) index model containing nine primary variables and 38 secondary variables.

Results

Policy content analysis using text mining tools revealed that DIP-related policy themes were relatively concentrated, primarily focusing on disease types, medical institutions, and directories. These themes continue to be consistently updated. The quantitative results of the TM-PMC index model showed that the overall design of the policies was reasonable, but there was a noticeable variation in differentiation between the policies. Out of the eight policies analyzed, five were rated as excellent, two as good, and one as acceptable.

Conclusions

The DIP policy is subject to continuous supplementation and optimization. The main factors that influence the value of the TM-PMC index for the policy include the following: policy objectives, policy objects (the groups for which the policy is implemented), policy tools, and policy perspectives. Therefore, various reforms related to collection and payment should be carried out in a locally adapted and standardized manner.