bluuebunny/crossref_metadata_2025_split huggingface dataset:
PP27 Evidence Quality and HTA outcomes in Reappraisals For Drugs For Rare Diseases In Germany
Authors: Wiedmann Lea , Cairns John , Nolte Ellen 
Published: January 2025

IntroductionEvidence on re-appraisals of health technologies in Germany is limited, and for rare disease treatments (RDTs) the Federal Joint Committee (GBA) uses different processes depending on whether the annual revenue threshold has been exceeded. We analyze RDTs for which an initial appraisal and a reappraisal were conducted to better understand processes used to determine the clinical benefit rating and their outcomes.MethodsWe review appraisal documents for 55 RDT indications published between January 2011 and September 2023. We extract information for the change in the maturity of survival data, the type of evidence, the risk of bias, and the availability of additional evidence as proxies for evidence quality. Specifically, we review the reasons for conducting reappraisals; examine how evidence quality and the clinical benefit rating differed between initial appraisals and re-appraisals; and explore the association between evidence quality and the clinical benefit rating following reappraisal.ResultsMost reappraisals were conducted because of exceeding the revenue threshold of EUR50 million (USD54 million) per year or reaching the review date when an initial decision was time limited. Almost all initial appraisals used the limited process, while the majority of reappraisals used the regular process. While nine out of 55 reappraisals achieved a higher benefit rating in reappraisals compared to initial appraisals, in 21 out of 55 reappraisals the benefit rating decreased. There was some evidence that reappraisals with accepted randomized controlled trial evidence were significantly more likely to achieve a higher clinical benefit rating.ConclusionsOur findings confirm that reasons and processes for completing reappraisals of RDTs in Germany differ. Moreover, the quality of the evidence submitted for both initial appraisals and reappraisals of RDTs was limited and achieving a high clinical benefit rating in reappraisals was rare.
PP31 Logical Model Articulating Pharmacy Commissions And Hospital-Based Health Technology Assessment Nuclei: Collaborative Strategies For The Drug Selection
Authors: Stopatto Denise Souza , Silva Elias Flávia Tavares 
Published: January 2025

IntroductionCollaborative strategies between the Pharmacy and Therapeutics Committee (Group 1) and hospital-based health technology assessment (HTA) nuclei (Group 2) using HTA tools can promote rational use and medicines access. The aim was to develop a logical model for the drug selection process in Brazilian university hospitals, considering the articulation between the Pharmacy and Therapeutics Committee and hospital-based HTA nuclei (NATS).MethodsA qualitative study created and validated a logical model. The data collection considered documental analysis and semistructured interviews with key informants from both groups about the processes adopted by eight federal university hospitals. The European Project on Hospital-Based Health Technology Assessment (AdHopHTA) was selected as a theoretical reference. The model was discussed in a focal group with the participation of five experts in medicine selection tasks and HTA. The final version of the model was submitted for consistency and vulnerability analysis by the authors. The ethical approval number is 4.784.861.ResultsThe logical model was conceived with dimensions of resources, actions, products, results, and impacts. It has allowed a structured and organized view of the 15 actions in the medicine selection. From these 15 actions, seven can be articulated between both groups. The high management support, training, and dissemination of the procedures and flows that organize HTA processes were solutions to minimizing collaborative barriers. The selection of medicines was considered a dynamic process. Some actions, such as the use of pharmacovigilance data, post-selection monitoring, and horizon technology scanning can bring results in terms of treatment innovations and compliance with clinical guidelines.ConclusionsCollaborative strategies were presented in a logical model with seven joint actions to guide the medicine selection process in hospitals involving tools, flow audits, governance, and human resources. The consistency and vulnerability model analysis defined indicators for HTA collaborative actions.
PP34 Impact Of The COVID-19 Pandemic On Depression Incidence And Healthcare Service Use Among Patients With Depression
Authors: Yi Chan Vivien Kin , Chai Yi , Man Chan Sandra Sau , Luo Hao , Jit Mark , Knapp Martin , Bishai David Makram , Ni Michael , Kei Wong Ian Chi , Li Shirley Xue 
Published: January 2025

IntroductionMost studies on the impact of the COVID-19 pandemic on depression burden focused on the earlier pandemic phase specific to lockdowns, but the longer-term impact of the pandemic is less well studied. In this population-based cohort study with quasi-experimental design, we examined both the short-term and long-term impacts of COVID-19 on depression incidence and healthcare service use among patients with depression.MethodsUsing the territory-wide electronic medical records in Hong Kong, we identified patients with new diagnoses of depression from 2014 to 2022. An interrupted time-series (ITS) analysis examined changes in incidence of depression before and during the pandemic. We then divided patients into nine cohorts based on year of incidence and studied their initial and ongoing service use until December 2022. Generalized linear modeling compared the rates of healthcare service use in the year of diagnosis between patients newly diagnosed before and during the pandemic. A separate ITS analysis explored the pandemic impact on the ongoing service use among preexisting patients.ResultsThere was an immediate increase in depression incidence (RR=1.21; 95% CI: 1.10, 1.33; p<0.001) in the population since the pandemic with a nonsignificant slope change, suggesting a sustained effect until the end of 2022. Subgroup analysis showed that increases in incidence were significant among adults and the older population, but not adolescents. Depression patients newly diagnosed during the pandemic used 11 percent fewer resources than the prepandemic patients in the first diagnosis year. Preexisting depression patients also had an immediate decrease of 16 percent in overall all-cause service use since the pandemic, with a positive slope change indicating a gradual rebound.ConclusionsDuring the COVID-19 pandemic, service provision for depression was suboptimal in the face of greater demand generated by the increasing depression incidence. Our findings indicate the need to improve mental health resource planning preparedness for future public health crises.
PP35 Keeping It Real Around The World: Comparing Real-World Evidence Guidance From Regulatory And Health Technology Assessment Bodies
Authors: Wee Yan Ran , Ng Alvin , Dinkar Eesha , Evans Jennifer Sara 
Published: January 2025

IntroductionThe growing use of real-world evidence (RWE) in pharmaceutical decision-making has prompted various guidelines, including REALISE (REAL World Data In ASia for HEalth Technology Assessment in Reimbursement). We compared RWE guidance from Asian, European, and North American health technology assessment (HTA) and regulatory bodies against the REALISE guidance.MethodsFollowing a previous search for China/Japan guidelines in 2022, websites of the U.S. Food and Drug Administration (FDA), the National Institute for Health and Care Excellence (NICE), the European Medicines Agency (EMA), and the Canadian Agency for Drugs and Technologies in Health (CADTH) were searched for RWE guidance in May 2023. Sections from each guidance were mapped onto REALISE and categorized as “agree,” “mixed,” “disagree,” or “missing” based on coverage/consistency.ResultsEleven guidelines were identified: four from Japan, three from China, and one each from FDA, NICE, EMA, and CADTH. No disagreements were found (all mapped sections were tagged “agree”/”mixed”); divergences were in coverage only. Most regulatory guidance had narrower scopes: EMA covered registry-based studies, the FDA’s framework mainly referenced other documents, while Japan had guidance for database and registry data. Conversely, HTA guidelines (CADTH, NICE) were more comprehensive and provided specific recommendations on preferred methods (e.g., transparent reporting) that were “missing” (45 to 46%) from REALISE’s more conceptual discussions. Chinese regulatory guidance was the exception, with similar coverage as REALISE (77% “agree”/“mixed”).ConclusionsGuidelines varied in scope, but there was overall concordance where recommendations could be mapped across documents. While regulatory bodies could focus on specific types of RWE, reflecting the specific role of RWE in regulatory evaluations (for example demonstrating safety), guidance for HTA was broader to account for different possible use cases in demonstrating comparative effectiveness and value.
PP41 Enhancing Health Technology Assessment Through Behavioral Economics: A Strategic Approach To Sustainable Healthcare Innovation
Authors: Swami Shilpi 
Published: January 2025

IntroductionBehavioral economics (BE) offers a spectrum of underutilized tools that could significantly enhance health technology assessment (HTA). This study proposes integration of BE into HTA, recognizing that understanding behavioral drivers can help in evaluating health technologies’ sustainability and adoption rates, thereby aligning HTA more closely with real-world healthcare dynamics and patient needs.MethodsThe study reviews existing key BE concepts and proposals for integrating BE concepts such as loss aversion, present bias, framing effects, and social norms influence into HTA framework. Loss aversion refers to the tendency of people to prefer avoiding losses rather than acquiring equivalent gains, while present bias is the tendency to prioritize immediate rewards over future benefits. Framing effect is the cognitive bias where people decide on options based on whether they are presented with positive or negative connotations.ResultsLoss aversion can increase patient adherence to chronic disease management technologies by emphasizing the avoidance of negative health outcomes. Present bias limits adopting preventive technologies, where immediate costs overshadow long-term benefits. Framing effects may determine how the presentation of technologies influences acceptance; for example, by framing a new surgical device as reducing the risk of postoperative complications (a positive frame), rather than not increasing the risk (a negative frame), it may be more readily accepted. Additionally, use of default options in electronic health records can improve data accuracy, and recognizing social norms can drive broader adoption and success of telehealth solutions.ConclusionsIntegrating BE with HTA methodology offers a pathway to more patient-centered and sustainable health technology implementation, with an emphasis on patient behavior patterns. This approach enables HTA to more effectively address human behavior intricacies, ensuring that health technology evaluations are both comprehensive and relevant to the evolving needs of healthcare systems and patients.
PP45 Core Outcome Sets For Research And Core Outcome Sets For Routine Care: Do They Overlap?
Authors: Kearney Anna , Gorst Sarah , Williamson Paula , Dodd Susanna 
Published: January 2025

IntroductionCore outcome sets (COS) are increasingly being developed for routine care, but it is unclear how they compare to COS for research for a given condition, particularly regarding recommended outcomes. If similar, embedding COS within clinical practice creates opportunities for improving data for real-world evidence. This work aims to compare outcomes in COS for different settings.MethodsCancer, neurology, cardiovascular, rheumatology, and orthopedic COS within the Core Outcomes Measures in Effectiveness Trials (COMET) database were reviewed to create matched sets of COS (COS that were developed for the same condition but different settings). Recommended outcomes were extracted along with information on COS scope (condition, population, and intervention), patient involvement, and year of publication. Specific outcome matches (e.g., cognition and executive capacity) and general outcome matches (e.g., mobility and physical function) were identified within each matched COS set to report the number and percent of distinct outcomes recommended for both settings.ResultsEighteen matched sets were identified. The median (IQR) number of distinct outcomes recommended for both settings was 6 (4, 8) and the median (IQR) percent of all distinct outcomes that were recommended for both settings, of those included across both settings, was 20 percent (12%, 33%), ranging from nine percent for stroke rehabilitation to 50 percent for psoriatic arthritis. Variation due to potential factors such as outcome granularity, number of matched COS per condition, and scope discrepancies will be discussed. Case studies will be presented for localized and advanced prostate cancer, which demonstrated high levels of overlap.Conclusions
Within a given condition, there appears to be a subset of outcomes relevant to both research and care, but the degree of overlap varies by disease area. Capturing these overlapping subsets of outcomes within routine data collection could provide a starting point to support generation of real-world evidence, thereby ensuring focus on outcomes of crucial importance to key stakeholders.
PP47 The Role Of Target Product Profiles Within Health Technology Assessment: A Narrative Review And Case Study
Authors: Hopkin Gareth , Chalkidou Anastasia , Ismail Farhan , Pelekanou Charlotte , Salmon Mark , Dawoud Dalia 
Published: January 2025

IntroductionTarget product profiles (TPPs) can be used to outline the minimal and desirable characteristics of a health technology to meet a particular health need. They have been used by industry, regulators, and international health agencies to guide product development and facilitate communication across stakeholders, but there appears to have been limited consideration of their utility within HTA.MethodsWe completed a narrative review of academic and grey literature on uses of TPPs across the product development, regulatory, and HTA pathway and considered areas within HTA where TPPs could have a role. We then developed a case study alongside an assessment of virtual ward platforms for acute respiratory infection. This case study aimed to explore whether it was possible to construct a TPP to support guidance based on HTA committee discussions and to consider associated opportunities and challenges. We also compared our TPP with other product specifications used within NHS England to support commissioning.ResultsThere has been discussion about how industry can build HTA perspectives into TPPs. However, we did not identify use of TPPs led by HTA agencies. Two use cases of TPPs were identified: (i) to support demand signaling for areas of unmet need and (ii) to provide a framework for class-based recommendations. HTA committee discussions supported the development of a TPP for virtual ward platforms with domains relating to patient population, technological specification, evidence requirements, and other factors (e.g., patient choice). Challenges around the time needed to consider both evidence and desired attributes and overlap with approaches used in commissioning were identified.ConclusionsTPPs may have a role to play within HTA to highlight areas of unmet need or to support innovative approaches to guidance. Digital health technologies may be particularly amenable to these approaches due to the larger range of similar products and ease of replicability. Further work should define how TPPs can be used to their full potential.
PP51 Pharmaceutical Innovativeness Index (PII): A New Framework For Assessing Health Technology Innovation Based On Therapeutic And Social Relevance
Authors: Gargano Ludmila , André Ariane , Freitas Isabela , de Assis Acurcio Francisco , Alvares-Teodoro Juliana , Guerra Augusto 
Published: January 2025

IntroductionThe value of a new pharmaceutical product should rely on its therapeutic benefits while also considering social health needs. Adapting health technology assessment (HTA) tools, we propose a transparent framework, with well-defined criteria and script, to determine the value of innovation (i.e., innovativeness) with clinical and methodological parameters, based on the social relevance and therapeutic value of new medicines.Methods
The study was developed by adapting HTA-based methods identified in the literature: the Italian Medicines Agency (AIFA) and the EValuation of pharmaceutical Innovations with regard to Therapeutic Advantage (EVITA) tool. A sample of oncology drugs approved by the U.S. Food and Drug Administration (FDA) between 2011 and 2021 were assessed by researchers trained in HTA tools using those methods. After assessing the sample, researchers discussed the results, difficulties, and issues experienced. Those issues were addressed, resulting in the creation of the new framework, which included the redefinition of domains, definition of classification criteria for each domain, and scores according to relevance.ResultsThe Pharmaceutical Innovativeness Index (PII) was proposed, with a script, domains, criteria, and an algorithm. The evaluation begins defining indication, outcomes, therapeutic alternatives, time perspective, and data sources. Four domains were considered: Therapeutic Need, which evaluates the existence and benefits of alternatives; Added Therapeutic Value, which talks about the incremental clinical benefit when compared to those alternatives (these first two domains were graded into five levels ranging from absent to maximum); Study Design and Methodological Quality, both classified into three levels. Classification criteria for each domain can be adapted according to the indication and relevant outcomes.ConclusionsThe PII framework considers clinical and social value weighted by methodological limitation of available evidence to determine the value of innovation of pharmaceutical products. It stands out as a transparent, adaptable, and reproducible tool that aims to reduce the subjectivity of analyses and assist with decision-making. PII has the potential to inform decision-making processes involving value-based pricing, reimbursement, and research and development investment.
PP57 Outcomes Model For Assessing Strategies Improving In Vitro Fertilization Birth Rates
Authors: Mongan Suzanna , Lyu Juntao , Kim Hansoo 
Published: January 2025

IntroductionInfertility affects one-sixth of women worldwide, with over seven million assisted reproductive cycles performed annually. Oral dydrogesterone is recommended alternatively for luteal phase support in in vitro fertilization (IVF), preventing miscarriages and improving live birth rates. This study aims to develop an outcomes model comparing oral dydrogesterone treatment with the standard of care in the IVF cycle over a 10-year period.MethodsA two-level Markov cohort model in Microsoft Excel includes six health states: IVF, pregnancy, miscarriage, live birth, perinatal death, and maternal death. Miscarriage, live birth, and perinatal death are sub-states of pregnancy. Transition probabilities are based on published rates with medical intervention limited to the first 12 weeks of gestation. A sensitivity analysis of treatment was performed. Data from a published meta-analysis of nine dydrogesterone studies for IVF luteal phase support were used. The baseline cohort is 10,000 Australian females undergoing IVF annually over a 10-year period.ResultsOver the 10-year time horizon, compared to standard care, the group treated with dydrogesterone was estimated to increase the number of live births by 3.5 percent (range: 3.4 to 3.7%), reduce the number of miscarriages by 69.4 percent (range: 66.2 to 72.7%), reduce the perinatal death by 10.9 percent (range: 10.4 to 11.4%), reduce the IVF cycles by 11.56 percent (range: 11.0 to 12.11%), and reduce the death of the mothers by 10.9 percent (range: 10.4 to 11.4%).ConclusionsThe outcomes model projected that treatment with oral dydrogesterone significantly reduced the number of miscarriages and improved the number of live births compared to the standard of care used for IVF patients in Australia.
PP60 Enhancing Patient Empowerment: European Capacity Building Initiative And Curriculum Development In Health Technology Assessment Training
Authors: Muehler Milena , Siebert Silke , Schnell-Inderst Petra , Roßmann Sonja , Puntscher Sibylle , Planinschitz Tanja , Hallsson Lára R. , Houÿez François , Delaye Julien , Strammiello Valentina , Siebert Uwe 
Published: January 2025

IntroductionThe new Regulation (EU) 2021/2282 on health technology assessment (HTAR) highlights the increasing importance of patient perspectives, emphasizing their crucial role in HTA. The European Capacity Building for Patients (EUCAPA) project aims to empower patients for active involvement in HTA. EUCAPA focuses on equipping patients with capacities through the development of HTA training, including introductory, fast-track, and extended training programs.MethodsWithin the EU-funded EU4Health program, the EUCAPA consortium was tasked with capacity building for patients and patient representatives. Training programs were developed using a coproduction approach, integrating patient representatives’ perspectives into both curriculum development and the design of training sessions. This innovative approach involved close collaboration between patient representatives and HTA scientists, jointly shaping the program content. The curriculum was designed to incorporate the heterogeneous medical and cultural backgrounds and varying levels of prior knowledge among participants, recognizing diverse experiences that individuals bring to the training. To enhance accessibility, a blended learning approach with offline, virtual, and in-person parts was adopted.ResultsThe training comprised introductory (two hours), fast-track (eight hours), and extended (four days) training sessions. Training modules include competencies such as a profound understanding of HTA processes and key principles, domains, impact of HTA, outcome measurements, and study designs to raise awareness, promote, and enhance patient engagement as foreseen in the HTAR. The training modules focus on empowering patients to advocate for their rights and communities and to contribute meaningfully to comprehensive treatment evaluation. Specifically, modules on communication and personal skills, patient involvement, and engagement at both national and European levels underscore promotion of patient empowerment. Training sessions can be accessed online (EUCAPA.eu).ConclusionsThe training development within the EUCAPA project addresses the evolving landscape of joint HTA by emphasizing the significance of patient perspectives, fostering core competencies, and responding to the increasing demand for active patient involvement and advocacy at both national and supranational levels.