bluuebunny/crossref_metadata_2025_split huggingface dataset:
PP17 Navigating Health Technology Assessment (HTA) Requirements During Development Through Early HTA Scientific Advice: Insights From Companies’ Strategies, Challenges, And Priorities
Authors: Wang Tina , McAuslane Neil 
Published: January 2025

IntroductionPharmaceutical companies have been actively taking early scientific advice from health technology assessment (HTA) agencies during development, focusing on study design to understand the HTA evidentiary requirements. The evolving advice landscape, including multistakeholder and international collaborations, highlights proactive engagement’s importance. This opinion survey assessed international pharmaceutical companies’ current experiences in seeking early HTA and explored strategies for forward-looking actions and considerations.MethodsAn opinion survey was designed and conducted in 2023 as a cross-sectional questionnaire consisting of multiple-choice questions. The questionnaire provided a qualitative assessment of companies’ current strategies and experiences in taking early HTA advice as well as future considerations. Eligible survey participants were the senior management of Global HTA/market access departments at 22 top international pharmaceutical companies.ResultsResponses were received from 13 companies. The National Institute for Health and Care Excellence (NICE) or the Federal Joint Committee (Gemeinsamer Bundesausschuss, G-BA) were utilized mostly for early HTA advice (92% respondents). Past European Medicines Agency HTA experience exists (50%), but there is no current engagement in Joint Scientific Consultation (JSC). However, JSC is deemed a top priority (83%) given the Regulation (EU) 2021/2282 on health technology assessment. Challenges in seeking advice include agency availability and internal resource and timing constraint. Advice-seeking actions mostly occurred during phase II trials but were frequently limited by agencies’ availability (84% respondents). Divergences were identified regarding eligibility criteria to seek advice and internal practices. Five success indicators were identified with the top-rated being the impact on development plan.ConclusionsThis survey assessed companies’ practices in seeking early HTA advice, with most engaging national agencies like NICE and G-BA. The lack of current JSC participation, despite companies’ prioritization, highlights the agencies’ need to enhance capacity and resources. Survey results underscored the importance of companies’ adaptive strategies in the evolving environment, which can be supported by active measures of advice success.
PP26 Systematic Literature Review Of Funding Models For The Access To Drugs For Patients With Rare Diseases
Authors: Parada Constanza Vargas 
Published: January 2025

IntroductionThere is no comprehensive framework that considers the various features of current funding models on drug accessibility for rare diseases; such a framework would assist policymakers to more effectively meet the challenges of these patients. This article reviews the funding models implemented worldwide to facilitate this access.MethodsThe PRISMA guidelines were used to conduct a systematic literature review. The following databases were searched: Ovid (Embase/MEDLINE), Cochrane database, Web of Science, EconLit, the National Institute for Health and Care Research (NIHR), Centre for Review and Dissemination (CRD), and International Network of Agencies for Health Technology Assessment (INAHTA). Two independent reviewers screened all titles and abstracts, and one reviewer did the full-text review and data extraction. Data were collected on general study characteristics, general aspects of rare diseases, source of funding, allocation of resources, and pricing strategies.ResultsA total of 3,815 unique citations were screened, and 148 were included for data extraction. Each funding model was characterized based on its unique features specific to rare diseases, focusing on process, methods applied, and consideration of attributes. Sixty funding models were identified in 41 countries, categorized as separate processes (42%), exceptions to standard processes (32%), standard processes with no changes (23%), and alternative pathways (3%). More than one funding model was available for 29 percent of countries. Funding models varied in their approach to HTA, source of funding, consideration of uncertainty, and pricing strategies.ConclusionsThe diversity of funding models highlights the complexity of addressing access to treatments for rare diseases. Special considerations towards rare diseases generally targeted the greater uncertainty in the clinical evidence. Despite the existing platform that enables access for drugs for rare diseases, only 10 percent of rare diseases have an available treatment and fewer patients can access these technologies.
PP37 Guidance On Using Hospital-Based Real-World Evidence In Health Technology Assessments For Oncology
Authors: Al-khayat Zainab , Franzen Nora , Retèl Valesca P , van Harten Wim H 
Published: January 2025

IntroductionReal-world evidence (RWE) is increasingly used in healthcare research to address evidence gaps, reduce uncertainty about medical technology benefits, and provide real-world insights. Efforts to integrate RWE in regulatory and health technology assessment (HTA) processes are growing. However, variations among countries pose challenges. The objective is to analyze and compare various (inter)national RWE guidelines, focusing on real-world hospital data utilization.MethodsWe conducted a review to identify RWE guidance published from 2016 to 2023, with a focus on the EU5 nations (UK, France, Germany, Italy, and Spain) and the ONCOVALUE consortium affiliates (Finland, the Netherlands, Denmark, Italy, and Portugal). To ensure a comprehensive overview, we also investigated Canada, the European Medicines Agency (EMA), ISPOR, and the European Society for Medical Oncology (ESMO). We conducted in-depth interviews with HTA experts of all included countries, focusing on real-world hospital data within the European HTA context. The interviews underwent thematic analysis related to the utilization of RWE in HTA.ResultsWe identified nine guidance reports: six focused on HTA-RWE (Medicinrådet/Denmark, NICE/UK, AQuAS/Spain, HAS/France, IQWiG/Germany, CADTH/Canada), one from EMA, and two international (ISPOR, ESMO). Only NICE, IQWiG, and CADTH offered recommendations covering hospital data, emphasizing the data curation process. HAS addressed considerations in choosing secondary data sources, while IQWiG established robust criteria for registries to ensure data quality. Regarding patient-reported outcomes data, only HAS and NICE provided recommendations in their guidance. The HTA experts acknowledged the value of hospital data but expressed caution due to its unstructured nature, noting that the use of hospital-based RWE is more accepted in descriptive studies.ConclusionsGuidances prioritize the clinical domain, emphasizing transparency, fitness for purpose, reproducibility, robustness, bias minimization, and generalizability. Notably, there’s a lack of comprehensive source-specific guidance for real-world data sources, including registries, hospitals, claims, and wearables. Enhanced guidance on the total data generation process, (data mapping, data federation), cost data, quality of life, and cross-border data usage would strengthen hospital-based RWE assessments.
PP46 Economic Value Of Minimally Invasive Procedures: Examining Revolutionary And Evolutionary Devices In Aortic Stenosis And Benign Prostatic Hyperplasia
Authors: Lim Schezn , Swan Georgia , Evans Jennifer Sara , Lim Andrew , Fujita Natsumi , Lo Justin , Priest Virginia 
Published: January 2025

IntroductionMinimally invasive procedures (MIPs) shorten procedural, hospital, and patient recovery time, providing patient benefits and reducing healthcare resource use (HCRU). This study explored the economic impact of two MIPs as discussed in published literature: transcatheter aortic valve replacement (TAVI), a step-change in cardiac clinical practice (“revolutionary”); and water vapor thermal therapy (WVTT), a progressive innovation in benign prostatic hyperplasia (BPH) (“evolutionary”).MethodsTwo pragmatic literature reviews were conducted to identify studies reporting comparative HCRU for TAVI versus surgical aortic valve replacement (SAVR) in aortic stenosis (AS), and WVTT versus other procedures in BPH. Searches were conducted on 20 October 2023 in the MEDLINE and MEDLINE plus Embase databases for the AS and BPH reviews, respectively. Studies from Asia-Pacific (AS review) and all geographies (BPH review) were included. HCRU data reported in the literature were identified and extracted.ResultsForty eligible studies were included (14 AS, 26 BPH). Commonly reported outcomes were hospital length of stay (LOS) (AS, BPH studies), intensive care unit (ICU) LOS (AS studies), and procedure duration (BPH studies). In AS studies, hospital LOS was shorter for TAVI (8.00 to 19.96 days) than SAVR (13.09 to 29.50 days). ICU LOS was shorter with TAVI (0.00 to 6.40 days) than SAVR (1.00 to 8.13 days). In BPH, WVTT had shorter hospital LOS (0.00 to 1.10 days) than other procedures (1.00 to 3.00 days). Of five studies reporting procedure duration, four showed shorter procedure time with WVTT (4.00 to 30.00 mins) than other procedures (5.20 to 148.00 mins).ConclusionsMIPs, whether “revolutionary” or “evolutionary,” could offer notable time-savings (both in terms of procedure duration and length of stay) at the hospital level, which may also lead to cost savings. This highlights the importance of establishing mechanisms in payer-level economic evaluations or health technology assessments that can account for the time-saving advantages of MIPs at the hospital level.
PP48 Cost-Effectiveness Of Mepolizumab In Children And Adolescents With Refractory Severe Eosinophilic Asthma In Brazil
Authors: Zimmermann Ivan Ricardo , Elias Flávia Tavares , da Silva EricaTatiane 
Published: January 2025

IntroductionAsthma affects individuals of all ages and is the most common chronic disease among children. The Brazilian Unified Health System (SUS) reimburses the use of mepolizumab as an additional maintenance treatment for severe eosinophilic asthma in adults. This study aimed to evaluate the cost-effectiveness of expanding the reimbursement of mepolizumab to patients aged six years and older.MethodsThe model included patients aged six years and older refractory to inhaled corticosteroid (ICS) plus long-acting beta-agonist (LABA) treatment. Baseline characteristics and outcomes were based on clinical trials and national hospitalization data. A Markov model with monthly cycles considered the health states without exacerbation, with exacerbation (including the need for oral steroids, emergency room admission, or hospitalization), and death. Direct medical costs (rate: USD1=BRL4.93) associated with all treatments were estimated from public health reimbursement sources. Quality-adjusted life years (QALY) was the primary outcome. Deterministic (Tornado) and probabilistic (Monte Carlo simulations) sensitivity analyses were conducted using Microsoft Excel.ResultsConsidering the current mepolizumab price (USD964.49) reimbursed by the Ministry of Health (MOH), the incremental cost-effectiveness ratio (ICER) was USD137,384.92/QALY (95% confidence interval: USD92,625.64, USD208,542.44), exceeding the USD24,333.86 threshold proposed by the National Committee for Health Technology Incorporation (Conitec). Using the average price from local public purchases (USD417.74), the ICER was USD96,673.51/QALY. Considering the past proposed discount from the manufacturer (USD390,93) and dose fractionation in children up to 12 years, the ICER was estimated in USD50,574.06/QALY. The most impactful parameters were the utility without exacerbation, mepolizumab cost, and relative reduction in exacerbation rate.ConclusionsConsidering current reimbursement prices, mepolizumab use in children and adolescents with refractory severe eosinophilic asthma is not cost effective in Brazil. Alternative scenarios with price discounts and dose fractionation may alter these conclusions.
PP50 Facilitating Academic Life Science Innovation With Early Health Technology Assessment: A Survey Of Potential User Needs And Perceptions
Authors: Dragojlovic Nick , Nagase Fernanda , Kopac Nicola , Yi (Grace) Meng Fan , Akinsoji Toluwase , Lynd Larry D. 
Published: January 2025

IntroductionAcademic life scientists often struggle to develop and commercialize concrete medical products based on their discoveries. Early health technology assessment (eHTA) can help innovators to define target product profiles (TPPs) with strong value propositions. To understand how eHTA can best help facilitate the clinical translation of university-based inventions, we conducted a survey of stakeholders in the life science innovation ecosystem.MethodsOur 10-minute online survey includes questions on respondents’ location, organizational affiliations, experiences in health technology development, and awareness and perceptions of eHTA. eHTA is broadly defined as the use of tools from health economics, epidemiology, management, and related disciplines to assess the potential value of a medical product candidate for patients, payers, providers, manufacturers, and other stakeholders. The survey is being advertised using social media and email, and it will be followed up with semistructured interviews. Data on 51 complete responses were summarized using frequency tables and cross-tabulations, and the statistical significance of subgroup differences was evaluated using Fisher’s exact test.ResultsOf 51 respondents, a majority lived in Canada (38/51; 75%) and had an academic affiliation (39/51; 76%). A “lack of commercialization skills among academic life science teams” was identified as a barrier to clinical translation by 41 percent (21/51), though this varied by academic affiliation (33% vs 67%; p=0.051) and industry experience (65% vs 29%; p=0.033). While 31 percent (16/51) reported familiarity with eHTA, this also varied by academic affiliation (23% vs 58%; p=0.033). Only 20 percent (10/51) had previously used eHTA, but a majority expressed an interest in learning more (39/51; 76%) and in using eHTA in the future (31/51; 61%).ConclusionsMaking eHTA more accessible for academic life scientists who lack commercialization experience may mitigate an important barrier to clinical translation of university-developed health technologies. While awareness of eHTA is relatively low in this group, they are interested in learning more about and using eHTA, and efforts should be made to integrate eHTA with existing product development tools like the TPP.
PP55 Health Technology Assessment Scoping Definition For Health Regulation
Authors: Oliveira Margarete , Ramos Maira , Camargo Erika , Silva Erica , Elias Flávia Tavares 
Published: January 2025

IntroductionThe first phase of a health technology assessment (HTA) is to define the scope of the assessment and choose the appropriate type of studies. The HTA in Regulation has been produced in cooperation with the Oswaldo Cruz Foundation (Brasilia) and the Brazilian Health Regulatory Agency (Anvisa). This work reports the Brazilian experience with HTA priority-setting within the time period 2022 to 2023.MethodsFocusing on Anvisa’s 2022 to 2030 research themes, a workgroup developed a priority-setting cycle addressing the following four steps: (i) survey application to define problems and topics (August to October 2022); (ii) data compilation and exploratory searches by topic; (iii) workshops with technical areas (March to June 2023), aiming to refine themes, define research questions, and determine what types of studies will be conducted); and (iv) presentation of prioritized themes.ResultsForty-six topics at the intersection of HTA and health regulation were prioritized. Macro-agendas for medical devices and medicines were the most frequent. The studies (39 scoping reviews, nine rapid HTAs, and four technical reports) aimed at post-market surveillance, definition of regulatory problems, selection of the best regulatory options, and identification of the legal basis for the selected problem. These studies will be used to assess the impact of the proposed solutions on stakeholders and identify any potential risks associated with them. Lastly, the studies will provide recommendations for decision-making regarding health regulatory measures.ConclusionsBy establishing technical cooperation between stakeholders to gather the best available evidence for regulatory decision-making processes through HTA studies, scoping definition based on the real needs of decision-makers has taken place. In this collaboration, experts were mobilized to expand response capacity and technical-scientific knowledge to support decision-making in health regulation.
PP64 Impact Of The Cost-Effectiveness Threshold On Drug Funding Recommendations: The Case Of Rare Diseases In Brazil
Authors: Oliveira Eduardo , Xavier Luciana , Louly Priscila , Prado Clementina , Bonan Luciene 
Published: January 2025

IntroductionIn 2022, the Brazilian public health system has adopted an explicit cost-effectiveness threshold of USD24,232.91 per quality-adjusted life years (QALY) for evaluating technologies intended for rare diseases treatment. Although regarded as a strategy for increasing efficiency, the National Committee for Health Technology Incorporation (Conitec) has recommended that the threshold should not be used as a knockout parameter.MethodsA retrospective analysis of Conitec’s recommendations regarding technologies for rare diseases issued between January and October 2023 was conducted. The following data were extracted from Conitec’s reports: (i) disease (rare or ultra-rare); (ii) health technology evaluated; (iii) scientific evidence regarding efficacy, effectiveness, and safety of the technology; (iv) incremental cost-effectiveness ratio (ICER); (v) recommendation issued; and (vi) rationale for recommendation. Identified technologies will be divided into two groups according to the recommendation issued (positive or negative). The rationale for each recommendation, along with other information, will be reviewed to look for explicit threshold utilization and its impact on decision-making.ResultsTwelve technologies for nine rare diseases evaluated in 2023 were retrieved and eleven were included in the analysis. Six drugs have received a positive recommendation for funding. The ICERs estimated varied between ‒USD5,278,362.06 for emicizumab for hemophilia A and USD116,407.47 for elexacaftor/tezacaftor/ivacaftor for cystic fibrosis. Two of them have received a positive recommendation despite their associated ICER exceeding the explicit threshold. All negative recommendations were associated with ICERs higher than the threshold, which varied between USD52,456.32 and USD847,942.97. For two, the threshold was specifically mentioned as the rationale for the negative.ConclusionsAfter the adoption of an explicit cost-effectiveness threshold in 2023, Conitec has issued two positive funding recommendations for rare diseases technologies despite their associated ICER exceeding the threshold: agalsidase alpha for Fabry disease and elexacaftor/tezacaftor/ivacaftor for cystic fibrosis. For both recommendations, the drug’s favorable impact on the natural history of the disease was considered decisive.
PP65 How Does The Spanish Health Technology Assessment Network Assess The Value Of Robotic Surgical Platforms? A Review Of Challenges Faced
Authors: Vercher Beatriz Pellicer , Orozco María Álvarez , Morales Carlos Mansilla 
Published: January 2025

IntroductionThe value assessment of robotic surgical platforms poses several challenges on existing health technology assessment (HTA) frameworks of robotic-assisted surgery (RAS). Following widely RAS adoption, this work aimed to assess current RAS evaluation from the Spanish HTA network perspective taking into consideration, in the absence of local guidance, the principles published by Erskine J et al. in 2023.MethodsA review of the HTA reports comparing RAS versus open surgery (OS) and laparoscopic surgery (LPS) conducted by the Spanish HTA network and published between 2017 and 2023 was performed and compared against the consensus statement on RAS evaluation set out around seven key themes (i.e., evidence inclusion and exclusion, patient- and clinician-reported outcomes [PRO, CRO, respectively]), the learning curve, allocation of costs, appropriate time horizons, economic analysis methods, and robotic ecosystem/wider benefits].ResultsFour HTA reports, developed by AQuAS (Agència de Qualitat i Avaluació Sanitàries de Catalunya), assessing RAS versus OS and LPS were identified and reviewed in depth. Seven groups of indications and 57 outcomes were retrieved and classified according to the principles set out in the paper. Our findings show there is a strong lack of compliance with the consensus statement: real-world evidence studies are deprioritized or considered insufficient, and none of the reports provides either an economic evaluation or an assessment on the robotics ecosystem. Only PROs comply fully or partially, except for one report.ConclusionsSpanish HTA evaluation of robotic surgical platforms diverges significantly from the consensus. Discrepancies were observed across crucial domains, notably in evidence-selection criteria, economic evaluations, and holistic robotic ecosystem assessment, despite occasional compliance with PROs. Capturing specific, relevant outcomes to enable a comprehensive HTA beyond the context of therapy is key to informing decision-making and enhancing reliability of future evaluations.
PP70 Comparison Of Approval Dates Of New Substances Between The U.S. Food And Drug Administration And The European Medicines Agency
Authors: Batscheider Ariane , Shlaen Evgeniya , Weiß Katharina , Schnauffer Darya , Greiner Roger-Axel , Wagner Peter , Bonduelle Doreen 
Published: January 2025

IntroductionEarly access to innovative medications is a key factor for the quality of medical care. We investigated differences in approval dates of new active substances between the U.S. and Europe and a potential connection with company headquarter location.MethodsData of new active substances (no generics, biosimilars, and hybrids) approved by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) from 1 January 2018 to 29 November 2023 were retrieved from FDA and EMA websites. We calculated the time difference between the drug approval dates between the agencies. We also retrieved the location of the headquarters of pharmaceutical companies.ResultsSince 2018, 390 medicines were approved by FDA or EMA: 113 (29.0%) only by FDA versus 40 (10.3%) only by EMA, and 237 (60.8%) by both. Approval of 32.1 percent of medicines occurred within six months. Fifty-seven percent were approved more than six months earlier by FDA and 11.0 percent more than six months earlier by EMA. Overall, 45.6 percent of the headquarters are in Europe and 44.1 percent are in North America. For medicines approved by FDA only, 63.7 percent of headquarters are in North America and 26.5 percent are in Europe. This is reversed for medicines approved by EMA only. For substances approved by both, 50.6 percent of headquarters are in Europe and 39.2 percent are in North America.ConclusionsAlmost 30 percent of new medicines approved by FDA within the last five years are not yet approved in Europe whereas only 10 percent are approved by EMA only. There is a tendency for companies with headquarters in North America to seek approval from FDA first. For medicines with approval from both authorities, most companies are primarily located in Europe.