bluuebunny/crossref_metadata_2025_split huggingface dataset:
PP14 Value-Based Pricing For Advanced Therapy Medicinal Products: Emerging Affordability Solutions
Authors: Gonçalves Elisabete 
Published: December 2022

IntroductionThe emergence of advanced therapy medicinal products (ATMPs), a disruptive class of health technologies, is generating important challenges in terms of value assessment, and their high prices introduce critical access and affordability concerns.MethodsThe aim of this oral presentation is to expose the challenges of traditional value assessment and pricing and reimbursement methods in the evaluation of ATMPs, and to characterize the current and prospective financing solutions that may ensure patient access to and affordability for these health technologies.ResultsStandard health technology assessment (HTA) is not designed for assessing ATMPs and may delay access to these therapies; thus, a broader concept of value is required. As a result, value-based pricing methodologies have been gaining prominence as a way to cope with the specific challenges of ATMPs. The pricing and reimbursement framework should ensure a balance between encouraging innovation and maximizing value for money for payers through the attribution of a fair price to new health technologies. The provision of early scientific advice to developers by regulatory and HTA bodies is key, as it will help diminish the perspective gap between developers, regulators, and payers.ConclusionsThe high efficacy and high price dynamic of many ATMPs necessitates novel financing models, both in the European Union and in the USA. Managed entry agreements, where financing is conditional upon the submission of additional evidence, linked with leased payments may offer effective strategies to address the uncertainties caused by the evidence gap associated with ATMPs, ensuring affordable and sustained access to these therapies.
PP17 Is the PriTec Tool Useful In The Identification Of Disruptive Healthcare Technologies?
Authors: Puñal-Riobóo Janet , Maceira-Rozas Maria del Carmen , Faraldo-Vallés Maria José , Varela-Lema Leonor 
Published: December 2022

IntroductionA disruptive technology has been defined as an innovation that completely changes the way things are done. Early identification of potential disruptive health technologies has become a key point in the agenda of decision makers and health technology assessment (HTA) bodies. The PriTec tool is an automatically executable web application that was developed in 2009 by the Galician HTA Agency to facilitate decision-making regarding the selection of technologies for post-launch observation. The tool has been updated to allow scoring and ranking of technologies before their introduction into the healthcare portfolio. The aim of this work was to propose a framework for assessing the usefulness of the PriTec tool in relation to identifying possible disruptive innovations.MethodsTo evaluate the applicability of the PriTec tool for distinguishing disruptive from non-disruptive innovations, we selected a few examples from prior acknowledged disruptive and non-disruptive innovations. These technologies were scored against the predefined criteria and the results were compared and analyzed globally and by type of domain. The PriTec tool assesses six domains of technologies: clinical condition, comparative effectiveness and safety, economic impact, implementation consequences, and dissemination speed.ResultsDisruptive technologies (e.g., transcatheter aortic valve implantation or point-of-care tests) had higher weighted global scores than non-disruptive technologies. In the domain analysis, the scores for implementation consequences were higher for disruptive than for non-disruptive technologies. Both types of technologies had similar scores in the other domains.ConclusionsThe PriTec tool seems to be useful for identifying potential disruptive technologies through its implementation domain. Further validation strategies are required to confirm the tool’s applicability and to improve its accuracy in the field of health disruption. The tool could be used by governments, horizon scanning organizations, and HTA organizations to promote the evidence-based detection of disruptive technologies in clinical practice. However, it is advisable that the tool be further tested and validated in other contexts.
PP21 Efficacy And Safety Of Aromatherapy: An Overview Of Systematic Reviews
Authors: Duarte-Díaz Andrea , Rivero-Santana Amado , Perestelo-Pérez Lilisbeth , Álvarez-Pérez Yolanda , Ramos-García Vanesa , Torres-Castaño Alezandra , Abt-Sacks Analía , Toledo-Chávarri Ana , Padilla-Ruiz María , Rodríguez-Rodríguez Leticia , González-Rodríguez Carlos , Serrano-Aguilar Pedro 
Published: December 2022

IntroductionAromatherapy is the field of herbal medicine that uses essential oils distilled from flowers, roots, and herbs and other plant compounds to promote physical and psychological well-being. Essential oils are absorbed into the body in different ways, with the inhaled and topical routes being the most widely used. The aim of this review was to critically evaluate and synthesize the available scientific evidence on the efficacy and safety of aromatherapy for the management of any therapeutic indication. This report was requested by the Spanish Ministries of Health and Science and Innovation.MethodsAn overview of systematic reviews (SRs) was performed. The MEDLINE, Embase, CINAHL, and PsycINFO databases were searched for literature published from January 2006 to August 2021. SRs reporting the efficacy and safety of aromatherapy were included. We applied no restrictions in terms of administration route or essential oil used. Two reviewers independently performed screening and selection, data extraction, and quality assessment.ResultsWe included 74 SRs covering a wide variety of populations and settings. The most reported outcome was anxiety, followed by pain, and the most commonly used essential oil was lavender. Fifteen SRs reported mild adverse events with aromatherapy. Only 11 SRs assessed the certainty of evidence using the GRADE approach. Aromatherapy reduced heart rate and likely reduces anxiety and breathing rate in patients with cardiovascular diseases. Aromatherapy probably also reduces pain in women with primary dysmenorrhea. Additionally, it may reduce blood pressure, acute pain, subjective stress, and the need for antiemetic drugs after surgical procedures. However, the evidence was very uncertain regarding the effect of essential oils on anxiety, pain, and quality of life in patients with cancer, anxiety and pain after a caesarean section, and dental anxiety.ConclusionsAromatherapy may be useful for managing psychological and physical symptoms in different settings. However, the conclusions of this review are not definitive because of the moderate to high risk of bias in many of the primary studies included in the SRs.
PP23 Lost In Translation? The Differences In The Use Of Real-World Evidence Across Key Markets
Authors: Crossman-Barnes Christina-Jane , Xu Weiwei , Kaur Ishneet 
Published: December 2022

IntroductionHealth Technology Assessment (HTA) agencies have recognized the importance of real-world evidence (RWE) to inform access decision-making and different HTA agencies establish distinct requirements for their local jurisdictions. The objective of this study is to understand the differences of RWE included in HTA reports and HTA agencies’ perception of RWE.MethodsHTA reports from agencies in France, Germany, Spain, Italy, United Kingdom (UK), Canada, Australia and South Korea from January 2011 to November 2021, including original submissions, resubmissions, extensions of original indications and renewals were analyzed.ResultsAcross the eight countries, RWE has been used in nineteen percent of all HTA reports (N=2,960/15,561), with an exponential increase observed between 2019 and 2021. RWE on clinical effectiveness was mostly used in HTA submissions in the UK (twenty-two percent), with twenty-six percent perceived with full acceptance. In contrast, RWE on safety and epidemiology was reported widely in HTA reports in France and Germany (83% and 87%), respectively. Ninety-three percent of RWE received full acceptance in France, followed by forty-four percent in Germany. A mixed picture of the types of RWE included in HTA reports was observed in the other countries, with high variance of acceptance (between 5 to 37%).ConclusionsFrance, Germany, and the UK are the top three countries with a large proportion of HTA reports where RWE was mentioned. The type of RWE used is related to a large extent to the local evidence requirements. For example, RWE around epidemiology was included widely in Germany due to the needs of providing local data for budget impact analyses required by the Federal Joint Committee (G-BA); RWE on tolerability as reported in periodic safety update reports (PSURs) needs to be included in French HTA submissions. RWE on clinical effectiveness has been evaluated the most by the UK HTA bodies.
PP32 Assessment Of Preferences For Treatment: A Discrete Choice Experiment Among Italian Patients With Prostate Cancer
Authors: Di Brino Eugenio , Di Bidino Rossella , Basile Michele , Rumi Filippo , Beccaglia Patrizia , Vetrini Damir , Cicchetti Americo 
Published: December 2022

IntroductionThe integrated patient-centered, evidence-based approach to care recognizes the role of patient preferences. A discrete choice experiment (DCE) was developed with the aim of identifying the preferences of men with prostate cancer in Italy regarding the different risk-benefit factors of various treatment options.MethodsThe DCE was developed with the support of prostate cancer patients and oncologists and was based on a targeted scoping review. The final DCE included 26 choice sets divided into two blocks. The first block focused on all prostate cancer patients (both metastatic and non-metastatic), while the second block aimed to assess preferences for patients with metastatic hormone-sensitive prostate cancer (mHSPC). Patients were asked to choose from ten attributes in the first block and six in the second block. The aim was to identify attributes and levels with a statistically significant impact on patient preferences. Preference estimates were calculated using a conditional logit regression model and the results were stratified by cancer stage (metastatic or non-metastatic) in the first block.ResultsA total of 202 patients (mean age 72 years) completed the DCE. In the first block, the most important attribute was quality of life (QoL), particularly for patients with metastatic cancer. The other three attributes found to be significant, in order of relevance to patients, were the risks of experiencing cognitive impairment, hematologic complications, and fatigue. For patients with mHSPC, QoL was the strongest determinant of preference. The risk of experiencing fatigue was also a relevant attribute, followed by skin irritation.ConclusionsThis study shows that the effect of treatment on QoL was the most important attribute for patients diagnosed with prostate cancer. Specific risk factors play a different role in the choice of treatment depending on cancer type, with the risk of experiencing fatigue being valued by all groups. Identifying and understanding patients’ preferences related to treatments for prostate cancer will help physicians identify the best treatment strategy.
PP37 A Systematic Review of Machine Learning and Statistical Models For Predicting Coronary Heart Disease In Diabetic Patients
Authors: Jiu Li , Vreman Rick , Wang Junfeng , Mantel-Teeuwisse Aukje , Goettsch Wim 
Published: December 2022

IntroductionRisk prediction models, using either machine learning or statistical algorithms, can act as inputs of a cost-effectiveness model when predicting costs and effectiveness of an intervention. This systematic review has two objectives: to evaluate methodological quality of the published models to predict diabetic coronary heart disease (CHD) risk; to evaluate whether the models were sufficiently reported to judge their applicability to the cost-effectiveness modelling.MethodsA targeted review of journal articles published in English, Dutch, Chinese, or Spanish was undertaken in PubMed, Embase, Scopus, Web of Science, and IEEE Explore from 1 January, 2016 to 31 May, 2021. To assess the methodological quality and reporting of the models, we used PROBAST (Prediction model Risk Of Bias Assessment Tool), CHARMS (a Checklist for critical Appraisal and data extraction for systematic Reviews of prediction Modelling Studies), and a checklist (Betts 2019) summarizing the application of cardiovascular risk prediction models to health technology assessment.ResultsOur search retrieved 6,579 hits, of which 18 models were eligible for inclusion. Among them, four studies developed machine learning models (2 recurrent neural networks, 1 random forest models, and 1 multi-task learning model) while 14 studies developed statistical models (8 Cox models, 5 logistic models, and 1 microsimulation model). More than 70 percent of models were of high methodological quality in aspects of participants (89%), predictors (72%), and outcomes (72%), while only five models (28%) in aspects of statistical analysis. For the reporting, only two models provided sufficient evidence in all aspects (i.e., participants, predictors, and outcomes) for judging their applicability to the cost-effectiveness modelling. Most models were reported sufficiently regarding participants (78%) and outcomes (72%), but only three models regarding predictors (17%).ConclusionsTo apply the CHD risk prediction models to cost-effectiveness modelling, concerns remain regarding the potential risk of bias due to inappropriate use of analysis methods, and regarding insufficient reporting on how to measure and assess the predictors.
PP47 Modelling Non-small Cell Lung Cancer Treatment: Predicted and Observed Impact Of Immunotherapy In The Netherlands
Authors: Mfumbilwa Zakile A , Wilschut Janneke A , Simons Martijn J.H.G , Ramaekers Bram , Joore Manuela , Retel Valesca , Cramer-van der Welle Christine M , Schramel Franz M.N.H , van de Garde Ewoudt M.W , Coupé Veerle M.H 
Published: December 2022

IntroductionPatients treated with immunotherapy are divided into two subgroups: (i) long-term survivors (LTS) and (ii) moderate survivors. Nevertheless, clinical trials (RCTs) report only average treatment effects such as hazard rate (HRs). Health economic-models often only input average treatment effects, even though it has been shown that accounting for the LTS subgroup is crucial for accurate projection of long-term survival under immunotherapy. We investigated the incorporation of a statistical mixture cure model (MCM) in a health-economic model for lung cancer as a way to account for LTS while incorporating reported average RCT-based treatment effects.MethodsWe developed a microsimulation model describing disease progression under three treatment lines in advanced lung cancer using Dutch real-world data of chemotherapies treated patients. Here we focus on first-line treatment, for which we used gompertz distribution to simulate time-to-progression. To simulate the impact of immunotherapy, we adjusted base-model assuming MCM for first-line treatment, where the LTS subgroup was not at risk to progress, but instead die from background mortality. The subgroup of moderate survivors on the other hand are at risk to progress with adjusted progression-free HR (PF-HR). We simulated the model with size of LTS (prop_LTS) ranging from 14-34 percent (keynote-001 five-year overall survival [OS], 95% confidence interval) while fixing average RCT PF-HR at 0.5. Model predictions under the different prop_LTS were compared to real-world Dutch OS as well as the long-term RCT five-year OS.ResultsWith respect to observed short-term survival outcomes, model predictions were insensitive to assumptions regarding the size of the LTS subgroup. However, to match the five-year RCT OS rate reported (32%), the prop_LTS had to be equal to 34 percent. Under this latter setting for the prop_LTS, the progression HR in the subgroup of moderate survivors was calibrated to be 1.1.ConclusionsThe use of a mixture cure model improves long-term model-based projections with the implicit assumption that moderate survivors have little or no treatment benefit.
PP48 A Micro-Costing Study For Circulating Tumor DNA testing In Oncology
Authors: Kramer Astrid , Schuuring Ed , Vessies Daan , van der Leest Paul , Geerlings Maartje , Rozendal Pim , Lanfermeijer Mirthe , Linders Theodora , van Kempen Léon , Fijneman Remond , Ligtenberg Marjolijn , Meijer Gerrit , van den Broek Daan , Retèl Valesca , Coupé Veerle 
Published: December 2022

IntroductionCirculating tumor DNA (ctDNA) is a promising new biomarker with multiple potential applications in cancer care. As part of the “ctDNA on the way to implementation in the Netherlands (COIN)” project, an early, comprehensive Health Technology Assessment (HTA) is ongoing. Information about the costs of ctDNA testing is essential for implementation. Estimating the total cost associated with ctDNA-testing is challenging due to variation in the workflow, wide range in purchase and operational costs of the platforms, and the highly dynamic field. As a first step in the HTA, the aim of this study was to develop a flexible micro-costing framework and open-access model for consistent cost calculation of ctDNA-testing.MethodsFirst, the complete diagnostic workflow of ctDNA-testing was mapped based on expert discussions. This step-wise workflow was used as the foundation of the framework. Second, the activity-based costing method was used and included costs for personnel, materials, equipment, overhead, housing, and test failures. Third, the framework was validated by experts and by applying the cost calculation model to six case studies.ResultsThe diagnostic workflow was mapped from blood sample collection to reporting the diagnostic findings. The framework was developed from a Dutch perspective and takes into account the testing volume. The total cost per sample for the case studies with different workflows and testing volumes ranged from EUR 168 to EUR 7,638.ConclusionsThe developed micro-costing framework can be used to calculate the costs for ctDNA-testing for different workflows. The results from the case studies show the wide range of costs for ctDNA-testing and that the costs are determined by the choice of platform, setting, and testing volume. The open access model allows users to adapt and specify parameters in the diagnostic workflow matching their setting and can be used to support investment decisions and future cost-effectiveness studies.
PP49 Financing The Line Of Care In The First Biochemical Relapse Of Prostate Cancer After [68Ga] PSMA PET- CT
Authors: Pontes Paiva Marcia Maria Diniz , Pozzo Lorena 
Published: December 2022

IntroductionIt is estimated that prostate cancer will reach 66 thousand by the triennium 2020-2022 according to the National Cancer Institute (INCA). After initial diagnosis and staging the patient may undergo radical prostatectomy and/or curative radiotherapy. In patients with biochemical relapse (PSA >0.2 ng/ml) initially treated with radical prostatectomy, salvage external radiotherapy is indicated. The [68Ga] Prostate Specific Membrane Antigen Positron Emission Tomography-Computed Tomography (PSMA PET-CT) scan is mainly used for localization of prostate cancer in the setting of first biochemical recurrence and can significantly influence the clinical management of the patient.MethodsThe overall objective of this work is to perform a treatment cost analysis for patients in first biochemical recurrence of prostate cancer after curative radical prostatectomy and after performing [68Ga] PSMA PET-CT from the perspective of the Brazilian Health System (SUS). A decision tree was constructed through consultation with experts to outline the patient’s entire treatment. Values per modelled therapeutic procedure were surveyed in two different scenarios, with and without [68Ga] PSMA PET-CT. The average treatment in scenario 1 was stereotaxic radiation therapy (SBRT), and rescue radiotherapy and androgen deprivation therapy (ADT). In scenario 2, it was salvage radiotherapy and ADT. The reimbursement table was prepared from data collected by SUS system. Variations were analyzed using a sensitivity study. Total average values included: individual procedure, according to medical management (up to 3 years) and population percentage with and without [68Ga] PSMA PET-CT.ResultsValues were calculated in Brazilian currency (BRL) for each procedure. The total amount calculated for scenario 1 was BRL 264,965,465.00 (USD 55,642,747.65) and for scenario 2 was BRL 123,585,612.72 (USD 26,162,978.67).ConclusionsThe reimbursement of line of care adopted after [68Ga] PSMA PET-CT is an important information to expand access to the Brazilian population. It shows an increased cost with [68Ga] PSMA PET-CT adoption. A prospective study should be considered with high follow up.
PP53 Applying The VALIDATE Approach To Frame The Assessment Of Integrated Care Management In Aortic Valve Stenosis
Authors: Valls-Comamala Victòria , Fernandez-Barceló Carla , Trilla Marc , Vidal Barbara , Sitges Marta , Sampietro-Colom Laura 
Published: December 2022

IntroductionThe assessment of current technologies needs a more holistic approach to obtain accurate recommendations for decision-making. The VALues In Doing Assessments of health TEchnologies (VALIDATE) methodology considers that facts and values from all stakeholders need to be included in the scoping of an assessment to gather the comprehensive information needed for unbiased decision-making. This report aimed to explore how to properly assess the integrated care of patients with aortic valve stenosis (AVS) using the VALIDATE approach.MethodsA literature review was conducted, and 11 semi-structured interviews were performed with various hospital-based healthcare professionals (cardiac surgeon, clinical cardiologist, interventional cardiologist, anesthetist, process coordinator nurse, and others) and patients. Content analysis was used for data analysis and integration.ResultsThe literature review showed that the cardiology and cardiac surgery perspectives were dominant in 90 percent of the articles and present in the remaining ten percent. The perspectives of other specialties (anesthesiology, primary care, and psychology) were included in three percent of the articles and patient perspectives were included in nine percent. Interviewing and considering the perspectives of the different stakeholders involved in the care pathway identified the following indicators that should be included in the assessment care for patients with AVS: difficulties associated with late diagnosis of AVS; the need to incorporate a multidisciplinary approach in patient risk assessment; the importance of geriatric evaluations; considering patient (and family and caregiver) preferences for type of treatment; the importance of following up pharmaceutical treatment and palliative care; use of telemonitoring; and digital exclusion of patients with respect to the use of apps for prehabilitation and rehabilitation.ConclusionsThe stakeholders interviewed were involved in different steps of the care pathway and had differing needs, some of which were not found in the literature. The indicators suggested for inclusion in the assessment differed according to type of stakeholder and their involvement in the care pathway. Therefore, this case study exemplifies the VALIDATE method and endorses the need for multistakeholder involvement in eliciting values when scoping the assessment of a complex technology.