bluuebunny/crossref_metadata_2025_split huggingface dataset:
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

IntroductionThis 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.MethodsThis 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.ResultsEmergency 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%).ConclusionsThis 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

IntroductionIn 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.ResultsThe 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.ConclusionsParticipants 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

IntroductionIf 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.MethodsLiterature 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.ConclusionsRegulations 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

IntroductionGlobally, 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.MethodsData 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.ResultsOrphan 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.ConclusionsThe 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

IntroductionRandomized 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.MethodsIndications 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.ResultsThe 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:•England: 29 of 46 (63%) indications assessed, (23/29 [79%] recommended);•Scotland: 28 of 46 (61%) indications assessed, (20/28 [71%] recommended);•France: 33 of 46 (72%) indications assessed, (7/33 [21%] received Amélioration du Service Médical Rendu level I to III);•Germany: 37 of 46 (80%) indications assessed, (2/37 [5%] achieved additional benefit);•Italy: 37 of 46 (80%) products assessed, (33/37 [89%] reimbursed); and•Spain: 34 of 46 (74%) products assessed, (23/34 [68%] reimbursed).

ConclusionsIndications 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

IntroductionBreast 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.MethodsA 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.ResultsIf 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.ConclusionsAs 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

IntroductionThe 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.MethodsPolicy 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.ResultsPolicy 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.ConclusionsThe 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.
PD225 Implementation Of Hospital-Based Health Technology Assessment For Innovative And Expensive Medical Devices In A French Teaching Hospital
Authors: Guerre Pascale , Huot Laure 
Published: January 2025

IntroductionTeaching hospitals are the first users of innovative, and often costly, technologies. In our institution, innovative medical devices are in demand in the early stage of market diffusion, but there is little data available to help decision-makers. Although decision criteria were identified, structured and contextualized work could help the process through formalized hospital-based health technology assessment (HB-HTA).MethodsA specialist team was set up to develop an HB-HTA process dedicated to innovative and expensive devices. The process is multidisciplinary and comprises methodologists, health economists, and pharmacists. The template developed by the Adopting Hospital Based Health Technology Assessment project was used to draft the report and to identify relevant elements adapted to the local context. Literature reviews were conducted. A list of contributors was drawn up, including the medical team and the various directors involved in generating decision support information (in particular, strategic and organizational impacts). Finally, workshops were held with patient representatives.ResultsThree applications have been processed, enabling the HB-HTA procedure to be improved. Coordination of the work by a dedicated independent team made it possible to establish the transparency and robustness required for an HTA process. The main difficulties were related to the multi-contributor aspect, which affected the timeline to produce the reports, introduced heterogeneity within the different items, and created some redundancy that had to be sorted out. Uncertainties were highlighted in relation to data on clinical benefits in our patient population and assumptions about activity and budget impact. The use of real-world studies to address these issues has been proposed.ConclusionsThe decision-maker appreciated being able to make decisions based on a single document resulting from multidisciplinary work. The experiences were positive and have led to the creation of an HB-HTA unit. Future reports should consider the weaknesses identified, and methodological developments are underway to improve the process.
PD226 Integration Of Patient And Clinician Insights Into The European Union Joint Scientific Consultations And Joint Clinical Assessments
Authors: Julian Elaine , Doeswijk Robin , Giuliani Rosa , Goettsch Wim , Brinkhuis Francine , Woermann Bernhard J. , Pavlovic Mira , Cardone Antonella , Bucher Heiner C. , Pérez-Valderrama Begoña , Bernardini Renato , Van Dyck Walter , Moelken Maureen Rutten-van , Gianfrate Fabrizio , Solà-Morales Oriol , Toumi Mondher , Widmer Daniel , Salmonson Tomas , Ruof Jörg 
Published: January 2025

IntroductionTo summarize insights generated during the preceding four conventions of the European Access Academy (EAA) regarding the interface of patient organizations and medical societies with the evolving European Union (EU) health technology assessment (HTA) process.MethodsIn 2022 and 2023 four EAA conventions were held on the EU HTA regulation, focusing on: (i) its relevance for beating cancer; (ii) stakeholder involvement; (iii) recommended preparatory steps to ensure its successful implementation; and (iv) the role of hematology and oncology as a pacemaker for the EU HTA process. Here we summarize insights generated at the four EAA conventions about the integration of patient and clinician insights in the evolving EU HTA process, including joint scientific consultations (JSC) and joint clinical assessments (JCA).ResultsThroughout the conventions it became clear that the interface of patient associations and clinical societies with the EU HTA process is key for successful implementation of the regulation. All involved stakeholders rely on the principles of evidence-based medicine (EBM), including best internal and external evidence, patient values and expectations, and clinical experience. It was agreed that patient and clinician perspectives on the assessments are needed to balance the technical analysis of best external evidence. While patient input is rather well defined, when and how input from clinical societies is best incorporated during the process remains unclear.ConclusionsAs stipulated by the EBM triad, systematic involvement of patients and clinicians throughout both JSC and JCA is key to ensuring best outcomes for patients and society as a whole, in line with the objectives of the EU HTA regulation.
PD230 The VALUE Project: Multistakeholder Assessment For A Value-Based Prevention of Vaccine-Preventable Lower Respiratory Tract Diseases
Authors: D’Ambrosio Floriana , Maida Ada , Pappalardo Ciro , Nisticò Anna , Scardigno Anna , Sezzatini Romina , Ricciardi Walter , Calabro’ Giovanna Elisa 
Published: January 2025

IntroductionLower respiratory tract diseases (LRTDs) pose a public health threat, particularly among vulnerable populations. Vaccination is crucial for disease control, promoting healthy aging, and reducing mortality. However, current immunization programs for adult and the elderly are suboptimal. This project engaged healthcare professionals and citizens to develop guidelines for immunization of adults and the elderly in Italy, with a specific focus on the currently available respiratory syncytial virus (RSV) vaccination.MethodsThe project will include the following three phases: (i) Exploratory phase: a scoping review—involving a search of MEDLINE and institutional websites—to investigate current vaccinations offered to adults and the elderly in Italy for vaccine preventable LRTDs; (ii) Assessment phase: a Delphi process to identify strengths and weaknesses in the current vaccinations offered to adults and the elderly and to evaluate the latest regional and national plans for immunizing the elderly against RSV; and (iii) Policy phase: a consultation with experts to formulate recommendations for the nationwide implementation of vaccination strategies for LRTDs among adults and the elderly, paying particular attention to RSV prevention.ResultsBy analyzing current vaccinations offered in Italy, the ongoing project released new evidence and data on vaccine preventable LRTDs to enhance existing vaccination policies and strategies. This aligned a value-based decision-making process with technological innovations in vaccination. Moreover, the involvement of different stakeholders was crucial for identifying health needs and recognizing potential barriers that may hinder adequate vaccination coverage among adult and elderly populations, thus defining priority areas.ConclusionsVaccination of adult and elderly populations must be a key component of strategies aimed at promoting the maintenance of good health in the long term. The disease burden of LRTDs and RSV infections is significant and likely underestimated among the elderly. Therefore, adequate prevention strategies are crucial to reducing the national and global burden of these respiratory diseases.