bluuebunny/crossref_metadata_2025 huggingface dataset:
OP82 Patient Involvement In Health Technology Assessment
Authors: Kulembekova Lyazzat , Kosherbayeva Lyazzat , Hailey David , Tabarov Adlet , Imanbekova Anar
Published: December 2022

Introduction

Patients are increasingly involved in the decision-making process for health technology assessment (HTA), but the question of at what stage they can be involved is still controversial. In Kazakhstan, the HTA process began in 2010. Over the past 2 years, implementation of a project to develop a priority-setting tool based on evidence-informed deliberative processes has made it possible to discuss the participation of patients in HTA. We explored the possibilities of participation of patients or a patient-oriented group in the HTA process.

Methods

Structured interviews were held with eight people with interests in HTA. Two were representatives of universities, two from a patient-oriented group, two experts in HTA, and two physicians. Interviews were held online by mobile phone or Zoom for 25–30 minutes. Question structures were formed based on the report ‘Patient Involvement in Health Technology Assessment in Europe 2010’. Seven stages were considered.

Results

All participants partially or completely agreed with the involvement of patients at the HTA stages of identification and prioritization. One or two did not agree with their involvement at the HTA assessment, information production, internal and external review, and diffusion and dissemination stages. Challenges for patient involvement in HTA can be related to other commitments for patients and their carers, lack of financial affordability, conflict of interest, and lack of capacity of the HTA agency to involve them. Five participants agreed on challenges for patients to being meaningfully involved in decision-making on health technologies. These included understanding which institution makes the decisions, finding an interlocutor within the decision-making body, and understanding the decision-making process. Other issues were technical and language difficulties, lack of commitment from decision-makers and the legal or policy framework for patient involvement in HTA decision-making.

Conclusions

Patients can participate in HTA, but the HTA agency must first prepare and agree on the level of patient participation, and develop measures to reduce barriers such as language difficulties, and patient obligations.
OP84 Cost Consequence Analysis: A Potential Framework To Incorporate Patient Preferences Into Health Technology Assessment And Reimbursement Decisions
Authors: Whitty Jennifer , Marsh Kevin , Low Eric , Shah Koonal , Dzingina Mendwas
Published: December 2022

Introduction

Patient preferences (PPs) are an important source of evidence in health technology assessment (HTA). However, a methodological framework to achieve their integration in decision-making is lacking. We aim to investigate the potential role of evaluative frameworks to integrate PP evidence into HTA and decision-making.

Methods

We undertook a scoping review to identify potential methodological frameworks to consider PP evidence in HTA and evidence of the acceptability of these frameworks for decision-makers. We searched PubMed, Cochrane, and the grey literature to identify relevant studies, reports, or guidance documents. We restricted our search to the use of PP rather than patient experience data and excluded articles solely relating to deliberative approaches.

Results

Frameworks identified as having the potential to integrate PP evidence included cost-utility analysis, cost-consequence analysis (CCA), the efficiency-frontier approach, and multi-criteria decision analysis. All have been used in various HTA contexts, but not necessarily for inclusion of PP evidence. Distinct benefits and challenges of integrating PP data were identified for each framework. These included the theoretical basis of the frameworks, their ability to consider non-health as well as health outcomes, and their ability to separate outcomes based on PPs from outcomes based on population preferences. There is limited evidence and no consensus on the application of these frameworks to consider PPs in HTA or on their acceptability for decision-makers. However, CCA has the advantage that it is both based on economic decision theory and it leaves patient preferences disaggregated from population preferences in an HTA.

Conclusions

The frameworks identified in this review offer potential approaches to systematically and transparently integrate PPs into HTA and decision-making. Based on the review findings, we propose a research agenda to explore the potential of CCA in particular. We anticipate that our findings will augment the recommendations of the Innovative Medicines Initiative PREFER project, which are expected to report in 2022.
OP88 Drawing Lines In The Sand: How Do We Define The Scope Of Analysis In HTA And Economic Evaluation?
Authors: Richardson Marina , Miller Fiona , Daneman Nick , Sander Beate
Published: December 2022

Introduction

We explore how the scope of analysis is defined in health technology assessment (HTA) and economic evaluation and consider the potential implications of these decisions.

Methods

The scope of analysis, including decisions about which methods and domains of HTA to include in the assessment, which costs, and health outcomes are most meaningful, and which comparators are the most relevant are typically informed by the needs of the decision-maker. We undertook two systematic scoping reviews to assess: (i) to what extent systems thinking is considered in literature-based technology assessments; and (ii) how the scope of the analysis is defined in economic evaluation using Clostridioides difficile infection as an exemplar. We synthesized the findings from these reviews and offer three key observations for future research and exploration in the field of HTA.

Results

Our scoping reviews found that the scope of analysis in economic evaluations typically focus on single interventions, often ignoring upstream and downstream interventions. Similarly, published technology assessments have narrowly defined and inconsistent scopes of analysis, with limited consideration of indirect health and non-health impacts. Three key observations for the field of HTA include: (i) economic evaluations focus on the value of single heath interventions. A focus on a single health intervention may simplify the analysis; however, will this siloed decision-making lead to optimal health resource allocation? (ii) published assessments have inconsistently defined scopes of analysis. A decision problem that focuses on the needs of the decision-maker is practical; however, will inconsistencies in perspectives across assessments create unfair conceptualizations of value? (iii) HTA is technology-focused, not patient-focused. A technology-focused HTA system aligns with the technology diffusion process; however, does this move us away from the patient-centered mandate of HTA?

Conclusions

The dynamic nature of HTA leads to many conceptualizations of value. Considering the potential implications of narrowly defined, inconsistent, and technology-focused scopes of analyses may have consequences on achieving a patient-centered high-quality health system.
OP92 Impact Of Real-World Evidence On Health Technology Assessment And National Guidance For Interventional Procedures: A UK Perspective
Authors: Mandava Lakshmi , Powell John , Harris Kevin , Chalkidou Anastasia
Published: December 2022

Introduction

The National Institute for Health and Care Excellence (NICE) interventional procedures programme (IPP) issues guidance on the safety and efficacy of new interventional procedures (IPS). The IPP considers a range of evidence from randomized controlled trials (RCTs) to case series to make final recommendations. Real-world evidence (RWE) can provide additional information on long-term outcomes and patient population characteristics that are not easily captured by RCTs. This study explores the impact of RWE in complementing RCTs on long-term safety and efficacy for national guidance development.

Methods

We review the impact of RWE in IPS guidance (IPG) updates and change in guidance recommendations in the last 5 years. This is done by analyzing NICE guidance updates and supporting evidence. A range of RWE was considered in the supporting evidence, for example, registries and clinical audits.

Results

The addition of RWE evidence to IPP guidance update has led to significant changes in the recommendations. For example, in one recent IPG, standard arrangements recommendation based on short-term RCT evidence was changed to a research recommendation when RWE showed an increase in long-term adverse effects. In another recent IPG update, special arrangements recommendation based on short-term RCT evidence was changed to a research recommendation when RWE reported long-term inferior efficacy and safety for the new intervention compared to current standard of care. A complete overview of results of the last 5 years will also be presented at the meeting.

Conclusions

These findings indicate that increased availability of RWE in HTA has the potential to impact national guidance recommendations. In addition, it shows how RWE can fill the evidence base gaps created by RCTs. Such data can confirm or contradict the findings of RCTs, or generate questions needing further research, or support disinvestment in non-effective technologies.
PP05 Effect Modifiers In Indirect Treatment Comparisons: Guidance Is Needed To Ensure An Unbiased Identification In Decision-Making
Authors: Freitag Andreas , Gurskyte Laura , Rizzo Maria , Sarri Grammati
Published: December 2022

Introduction

Unadjusted effect modifiers (EM) in indirect treatment comparisons (ITC) can produce biased and uncertain results in health technology assessments (HTA). Even though research on advanced ITC methods to adjust for EMs has attracted much attention, less emphasis has been placed on how these EMs are identified and assessed in the first place. This is surprising given the evidence-based approach underpinning all analyses supporting HTAs. To address this gap, our aim was to identify what HTA guidance exists on the selection process for EMs, and how the selection of EMs has been justified in the context of published ITCs.

Methods

A pragmatic review of HTA guidance documents was conducted in 2021 to describe current requirements for the selection of EMs for ITCs. A supplementary Embase and Medline search was conducted to identify primary research on ITCs published between 2015-2021 presenting information on how EMs were selected to inform these analyses.

Results

Our review found that guidance on this topic focusses on developing and testing different methodologies to adjust for EMs. No detailed guidance was identified in any of the reviewed HTAs, although the National Institute for Health and Care Excellence (NICE) briefly mentioned that companies should identify EMs through a topical review or expert discussions. Similar findings were also revealed through the database search; few published ITCs included information on the EM selection process which was either based on evidence highlighted in the literature or findings from prior trial subgroup analyses. No reference to a systematic identification of EMs was found.

Conclusions

To fill the guidance gap identified in our review an extension of current ITC guidelines (including those from HTA bodies) is needed, including (i) indication on how EMs should be identified through systematic reviews, (ii) a quantitative assessment of the EM distribution and (iii) formal expert elicitation prior to the selection of ITC methods. Without these additional steps, ITC results may be biased, potentially negatively impacting decision-making and ultimately patient care.
PP10 Impact Of COVID-19 On The Management Of Breast Cancer In Italy: The Perspectives Of Patients And Coordinators
Authors: Di Brino Eugenio , Laurita Roberta , Cicchetti Americo
Published: December 2022

Introduction

Breast Units (BU) represent an opportunity to adequately manage and improve quality of care for patients with breast cancer. The presence of specific national guidelines should promote safe and good quality, integrated care. In fact, the presence of a multidisciplinary team of specialists whose workflows follow specific guidelines, set to the highest European standards, should ensure that patients in Breast Units receive appropriate care at diagnosis and throughout the course of the disease.

Methods

Two surveys were developed and administered to Breast Unit coordinators and patients throughout Italy. The surveys investigated the provision of healthcare services and the implementation of a new organizational model over two time periods: the first wave of the coronavirus disease 2019 (COVID-19) pandemic from March 2020 to August 2020 and the second wave from September 2020 to December 2020. The surveys aimed to assess the continuity of care during the pandemic from the clinician and patient perspective.

Results

Patients observed a reduction in the postponement of care between the first wave of COVID-19 and the second wave. Some services were delayed, particularly in the second wave where a large number of services were not rescheduled. Management of the COVID-19 pandemic resulted in many healthcare professionals being reallocated to other duties and hospital departments. The effect of this was particularly relevant for both patients and clinicians, with few healthcare professionals having the capacity to focus on conditions other than COVID-19. In the first wave of the pandemic 42 percent of professionals were redeployed, compared with 27 percent in the second wave.

Conclusions

This study demonstrates how COVID-19 affected the care of patients with breast cancer in Italy. Patients and coordinators from Breast Units across the country highlighted many important aspects that should be considered when assessing the effects of the COVID-19 pandemic on the entire healthcare system in order to be better prepared for future pandemics.
PP14 Value-Based Pricing For Advanced Therapy Medicinal Products: Emerging Affordability Solutions
Authors: Gonçalves Elisabete
Published: December 2022

Introduction

The 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.

Methods

The 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.

Results

Standard 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.

Conclusions

The 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

Introduction

A 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.

Methods

To 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.

Results

Disruptive 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.

Conclusions

The 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

Introduction

Aromatherapy 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.

Methods

An 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.

Results

We 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.

Conclusions

Aromatherapy 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

Introduction

Health 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.

Methods

HTA 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.

Results

Across 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%).

Conclusions

France, 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

Introduction

The 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.

Methods

The 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.

Results

A 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.

Conclusions

This 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

Introduction

Risk 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.

Methods

A 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.

Results

Our 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%).

Conclusions

To 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

Introduction

Patients 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.

Methods

We 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.

Results

With 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.

Conclusions

The 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

Introduction

Circulating 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.

Methods

First, 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.

Results

The 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.

Conclusions

The 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

Introduction

It 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.

Methods

The 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.

Results

Values 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).

Conclusions

The 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

Introduction

The 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.

Methods

A 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.

Results

The 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.

Conclusions

The 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.
PP61 Plugging the Gap of Fetoscopy in Congenital Diaphragmatic Hernia Pregnancies: Value for Money?
Authors: Janssen Jip , van Drongelen Joris , Daamen Willeke F , Grutters Janneke
Published: December 2022

Introduction

Fetoscopic endoluminal tracheal occlusion (FETO) for congenital diaphragmatic hernia (CDH) fetuses increases the neonatal survival rate. However, FETO also increases the number of preterm prelabour rupture of membranes (PPROM) and preterm deliveries (PTDs) as fetal membrane defects after fetoscopy do not spontaneously heal. To solve this issue, an advanced sealing plug is being developed. Through early-stage health economic modelling, we estimated the potential value of this innovative plug in terms of costs and effects and determined the properties for it to become cost-effective.

Methods

We applied early-stage health economic modelling to the case of performing FETO in singleton pregnant women whose fetus is prenatally diagnosed with CDH. We simulated a cohort of women using a state-transition model over a 45-year time horizon. In our best-case scenario analysis, we compared the current care strategy to a perfect plug strategy, which reduces PPROM and PTDs by 100 percent, to determine the maximum quality-adjusted life years (QALYs) gained and costs saved. Using threshold analysis, we determined the minimum percentage of reduction in PPROM and PTDs for the plug to be considered cost-effective. Model parameters’ impact on outcomes was investigated in a sensitivity analysis.

Results

Our model indicated that a perfect plug strategy would yield an additional 1.94 QALYs at a cost decrease of EUR 2,554 per patient per year. These values were strongly influenced by the percentage of very preterm deliveries. Threshold analysis showed that, for EUR 500 per plug, the plug strategy needs a minimum relative reduction of 1.83 percent in PPROM and PTDs (i.e., PPROM: 47.50 to 46.63 %, PTDs: 71.50 to 70.19 %) to be cost-effective.

Conclusions

Our model-based approach showed clear potential for the plug strategy when applied in the context of FETO for CDH fetuses, as only a small reduction in PPROM and PTDs is needed for the plug to be cost-effective. Its value is expected to be even higher when used in conditions suffering from more very preterm deliveries. Continuation of investment in the innovation’s research and development seems to provide value for money.
PP69 Supporting Decision Making: A Health Technology Assessment Training Proposal for Decision Makers
Authors: Faraldo-Valles Maria-Jose , Varela-Lema Leonor , Perez-Rios Monica
Published: December 2022

Introduction

Health technology assessment (HTA) reports are complex technical documents that address multiple aspects of the incorporation of a technology into the healthcare system applying complicated methodologies coming from different disciplines. The purpose of HTA is to support decision makers, who should have an adequate level of training to fully understand these assessments. However, most HTA education programs and courses are intended for HTA doers and there is a lack of practical guidance training aimed at preparing health managers or policy makers in HTA. The objective is to describe an HTA training program developed for decision makers of the three levels (health care administration, hospital management and clinical practice).

Methods

The education program has been designed through a collaboration between the Public Health Department of the Faculty of Medicine of the University of Santiago de Compostela (USC) and the Galician Health Technology Assessment Agency that belongs to the Spanish HTA Bodies Network. The duration of the course is 200 hours and the methodology will be distance learning, through the virtual classroom of the USC. The teaching collaborators come from the academic field and the HTA area.

Results

The course will cover the legal, clinical and organizational framework in which the HTA is developed in Spain and in Europe; and will approach the methodology used in HTA. The course is structured in six modules: (i) Research, development and regulation of health technologies; (ii) Role of HTA as a decision support tool; (iii) HTA Methodology; (iv) Health information systems (including use of real world data); (v) Incorporation of HTA into society (stakeholders); (vi) Future challenges (personalized medicine and e-health).

Conclusions

A specific training about HTA from a practical approach not theoretical could be of interest for different stakeholders involved in the decision-making process across the health systems. This type of educational program will allow decision makers to have a good understanding of the wide range of information they handle.
PP71 Lessons Learned During Analyses Of Appropriate Hearing Care: Building Bridges Between Patient Organizations, Health Professionals And Insurance Companies
Authors: Stam Mariska , Vreugdenhil Tjitske , Hermens Marleen , Maagdenberg Hedy
Published: December 2022

Introduction

As part of the cyclic Appropriate Care programme of the National Health Care Institute in the Netherlands, a systematic analysis of hearing health care is taking place. Parties in hearing health care are actively involved throughout the entire process. This abstract focuses on lessons learned from the cooperation as a HTA body with a diverse group of stakeholders.

Methods

We carried out an in-depth analysis for the patient journey of both children and adults with ear complaints or hearing impairment. Different kinds of information were included in the analyses, including claims data, quantitative and qualitative research, analyses of (international) guidelines and patient information. A range of strategies were used to co-operate and interact with patient organizations, hearing health care professionals, institutes/hospitals and insurance companies.

Results

Close collaboration between the project team and patient organizations turned out to be effective to comprehend patient’ perspectives. Data analyses were often found to be challenging in hearing health care, as the reimbursement data lacked sufficient information. In several cases, building bridges between parties, but also in relation to our HTA body was needed. Conclusions from the analyses were being shared and discussed with a panel of involved stakeholders, leading to support, but not always consensus on potential room for improvement. An internal review process turned out to be helpful in sharing experiences on effective multi-stakeholder management.

Conclusions

We believe that the process did influence the way stakeholders think about the appropriate use of the different available treatment options. Building bridges, and combining different perspectives from patient organizations, health-care professionals and insurance companies is necessary in a cyclic approach. The cyclic appropriate Care programme proved to be a constructive approach for collaboration with stakeholders.
PP72 SARS-CoV-2: A Rapid Review On The Effectiveness Of Face Coverings To Reduce Transmission
Authors: Winfield Thomas , Hopkin Gareth , Elston Lauren , Davis Claire , Washington Jenni , Jarrom David , McDermott Katie , Myles Susan
Published: December 2022

Introduction

At various stages of the COVID-19 pandemic, face coverings have been recommended and encouraged as one of the interventions to reduce transmission of the SARS-CoV-2 virus. However, in the earlier stages of the pandemic, decisions on face coverings relied primarily on evidence based on other viral respiratory infections. More direct evidence on the use of face coverings with COVID-19 developed in tandem with the pandemic.

Health Technology Wales undertook an ultra-rapid review to inform national guidelines, the work assessed the evidence on the effectiveness of face coverings to reduce SARS-CoV-2 transmission. We also reviewed evidence on the efficacy of different types of face coverings.

Methods

We conducted a systematic literature search for evidence to address (i) the effectiveness of face coverings to reduce the spread of COVID-19 in the community, and (ii) the efficacy of different types of face coverings designed for use in community settings. We identified a rapid review in 2021 by Public Health England that closely aligned with our review questions. This provided the main source for identifying relevant studies, supplemented by a search for publications following their search date.

Results

We identified two evidence reviews (including the Public Health England review) that examined the effectiveness of face coverings on reducing transmission of SARS-CoV-2; reporting on 31 and 39 studies, respectively. Two further primary studies were published after the two evidence review searches were included. Overall, the evidence suggested that face coverings may provide benefits in preventing SARS-CoV-2 transmission, although the higher-quality studies suggested that these benefits may be modest. Medical masks appeared to have higher efficacy than fabric masks, although the evidence was mixed.

Conclusions

At the time of this review, evidence on the effectiveness of face coverings remains limited and conclusions rely on low-quality sources of evidence with high risk of bias, although higher-quality evidence points to some benefit. Face coverings may play a role in preventing transmission of SARS-CoV-2, particularly as part of a bundle of other preventative measures.