Economic evaluations of medical devices in paediatrics: a systematic review and a quality appraisal of the literature.

BACKGROUND: Although economic evaluations (EEs) have been increasingly applied to medical devices, little discussion has been conducted on how the different health realities of specific populations may impact the application of methods and the ensuing results. This is particularly relevant for pediatric populations, as most EEs on devices are conducted in adults, with specific aspects related to the uniqueness of child health often being overlooked. This study provides a review of the published EEs on devices used in paediatrics, assessing the quality of reporting, and summarising methodological challenges. METHODS: A systematic literature search was performed to identify peer-reviewed publications on the economic value of devices used in paediatrics in the form of full EEs (comparing both costs and consequences of two or more devices). After the removal of duplicates, article titles and abstracts were screened. The remaining full-text articles were retrieved and assessed for inclusion. In-vitro diagnostic devices were not considered in this review. Study descriptive and methodological characteristics were extracted using a structured template. The Consolidated Health Economic Evaluation Reporting Standards (CHEERS) 2022 checklist was used to assess the quality of reporting. A narrative synthesis of the results was conducted followed by a critical discussion on the main challenges found in the literature. RESULTS: 39 full EEs were eligible for review. Most studies were conducted in high-income countries (67%) and focused on high-risk therapeutic devices (72%). Studies comprised 25 cost-utility analyses, 13 cost-effectiveness analyses and 1 cost-benefit analysis. Most of the studies considered a lifetime horizon (41%) and a health system perspective (36%). Compliance with the CHEERS 2022 items varied among the studies. CONCLUSIONS: Despite the scant body of evidence on EEs focusing on devices in paediatrics results highlight the need to improve the quality of reporting and advance methods that can explicitly incorporate the multiple impacts related to the use of devices with distinct characteristics, as well as consider specific child health realities. The design of innovative participatory approaches and instruments for measuring outcomes meaningful to children and their families should be sought in future research.

Advancing hospital-based health technology assessment: evaluating genomic panel contracting strategies for blood tumors through a multimethodology.

INTRODUCTION: The adoption of genomic technologies in the context of hospital-based health technology assessment presents multiple practical and organizational challenges. OBJECTIVE: This study aimed to assist the Instituto Português de Oncologia de Lisboa Francisco Gentil (IPO Lisboa) decision makers in analyzing which acute myeloid leukemia (AML) genomic panel contracting strategies had the highest value-for-money. METHODS: A tailored, three-step approach was developed, which included: mapping clinical pathways of AML patients, building a multicriteria value model using the MACBETH approach to evaluate each genomic testing contracting strategy, and estimating the cost of each strategy through Monte Carlo simulation modeling. The value-for-money of three contracting strategies - "Standard of care (S1)," "FoundationOne Heme test (S2)," and "New diagnostic test infrastructure (S3)" - was then analyzed through strategy landscape and value-for-money graphs. RESULTS: Implementing a larger gene panel (S2) and investing in a new diagnostic test infrastructure (S3) were shown to generate extra value, but also to entail extra costs in comparison with the standard of care, with the extra value being explained by making available additional genetic information that enables more personalized treatment and patient monitoring (S2 and S3), access to a broader range of clinical trials (S2), and more complete databases to potentiate research (S3). CONCLUSION: The proposed multimethodology provided IPO Lisboa decision makers with comprehensive and insightful information regarding each strategy's value-for-money, enabling an informed discussion on whether to move from the current Strategy S1 to other competing strategies.

Which value aspects are relevant for the evaluation of medical devices? Exploring stakeholders' views through a Web-Delphi process.

BACKGROUND: Implementation and uptake of health technology assessment for evaluating medical devices require including aspects that different stakeholders consider relevant, beyond cost and effectiveness. However, the involvement of stakeholders in sharing their views still needs to be improved. OBJECTIVE: This article explores the relevance of distinct value aspects for evaluating different types of medical devices according to stakeholders' views. METHODS: Thirty-four value aspects collected through literature review and expert validation were the input for a 2-round Web-Delphi process. In the Web-Delphi, a panel of participants from five stakeholders' groups (healthcare professionals, buyers and policymakers, academics, industry, and patients and citizens) judged the relevance of each aspect, by assigning a relevance-level ('Critical', 'Fundamental', 'Complementary', or 'Irrelevant'), for two types of medical devices separately: 'Implantable' and 'In vitro tests based on biomarkers'. Opinions were analysed at the panel and group level, and similarities across devices were identified. RESULTS: One hundred thirty-four participants completed the process. No aspects were considered 'Irrelevant', neither for the panel nor for stakeholder groups, in both types of devices. The panel considered effectiveness and safety-related aspects 'Critical' (e.g., 'Adverse events for the patient'), and costs-related aspects 'Fundamental' (e.g., 'Cost of the medical device'). Several additional aspects not included in existing frameworks' literature, e.g., related to environmental impact and devices' usage by the healthcare professional, were deemed as relevant by the panel. A moderate to substantial agreement across and within groups was observed. CONCLUSION: Different stakeholders agree on the relevance of including multiple aspects in medical devices' evaluation. This study produces key information to inform the development of frameworks for valuing medical devices, and to guide evidence collection.

ANIPHI: An innovative pedagogical platform based on the Delphi method to support animal welfare teaching.

As a teaching subject, animal welfare is challenging for educators and learners, as was recently shown in a recent survey on the evolution of animal welfare teaching in Europe. Among several suggestions to overcome the current resistance to implementing animal welfare education, we highlight two. The first is that animal welfare education should be based on learner-centred approaches; the second is that it should encompass both animal welfare science and ethics and law. To the best of our knowledge, there are no learner-centred pedagogical approaches that can simultaneously explore scientific and ethical concepts. Furthermore, when exploring ethical concepts within the educational context, there is the additional challenge of being able to depart from discussion and debate to a systematic organization of knowledge. Our work simultaneously addresses these two challenges, presenting the design and implementation of a novel web-based learner-centred pedagogical platform for farm animal welfare teaching. The platform, named ANIPHI, uses the Delphi method's iterative nature as a learning process to generate both reflection and (online) debate among learners. ANIPHI can be used by educators in an online environment, in a classroom environment, or in a combination of the two environments. ANIPHI was developed within the ERASMUS+ ANICARE project and is an open web-based platform for all educators interested in teaching farm animal welfare. Given ANIPHI's flexible and user-friendly nature, the platform simultaneously exposes learners to ethical and scientific concepts in different educational realities, according to the educator's objectives. Furthermore, videos depicting different husbandry practices across different types of animal production and countries are embedded in the platform. These videos are commented on by the farmer himself and by animal scientists, which enriches the learner's experience. Educators across the ANICARE consortium have already successfully tested the ANIPHI platform for different farm animal welfare topics. We conclude this article by presenting one example of using ANIPHI in a real-life educational context, where we discuss some aspects of the design and use of our pedagogical platform.

Informing the Design of Data Visualization Tools to Monitor the COVID-19 Pandemic in Portugal: A Web-Delphi Participatory Approach.

Due to the large amount of data generated by new technologies and information systems in the health arena, health dashboards have become increasingly popular as data visualization tools which stimulate visual perception capabilities. Although the importance of involving users is recognized in dashboard design, a limited number of studies have combined participatory methods with visualization options. This study proposes a novel approach to inform the design of data visualization tools in the COVID-19 context. With the objective of understanding which visualization formats should be incorporated within dashboards for the COVID-19 pandemic, a specifically designed Web-Delphi process was developed to understand the preferences and views of the public in general regarding distinct data visualization formats. The design of the Delphi process aimed at considering not only the theory-based evidence regarding input data and visualization formats but also the perception of final users. The developed approach was implemented to select appropriate data visualization formats to present information commonly used in public web-based COVID-19 dashboards. Forty-seven individuals completed a two-round Web-Delphi process that was launched through a snowball approach. Most respondents were young and highly educated and expressed to prefer distinct visualisation formats for different types of indicators. The preferred visualization formats from the participants were used to build a redesigned version of the official DGS COVID-19 dashboard used in Portugal. This study provides insights into data visualization selection literature, as well as shows how a Delphi process can be implemented to assist the design of public health dashboards.

Setting Requirements for a Dashboard to Inform Portuguese Decision-Makers About Environment Health in an Urban Setting.

Dashboards are being increasingly used in the health field, and literature points out that accurate and efficient dashboards require not only dealing with data issues, but also ensuring that dashboards are user-friendly and that incorporate users' views and needs. The integration of evidence and data into decision aiding tools, such as dashboards, to assess and monitor environmental health (EH) in urban settings requires careful design. Departing from EH evidence and making use of the views of EH stakeholders and experts, this study aimed at defining requirements for a dashboard to help decision-makers analyzing and visualizing EH information in the Lisbon urban context. In order to set those requirements, it was combined a user-centered with a design card approach to engage EH potential end-users so as to collect their visualization preferences and gather information related to dashboard requirements. Specifically, three online group semi-structured interviews, involving 11 potential end-users from different organizations, were conducted; design cards with a set of visualization options regarding 17 indicators of built and natural environment determinants were used in the interviews to capture participants' preferences and their rationale; questions about other dashboard features were also asked; and the results from the interviews were synthesized into four separate, but interrelated features, and operationalized into 11 requirements for a dashboard to monitor EH in Lisbon. This study contributes to EH literature by producing knowledge to inform dashboard construction, by highlighting issues related with the usability, analysis, and visualization of data to inform EH decision-making in urban contexts, and by designing an approach that can be replicated to other EH dashboard contexts.

Selecting Indicators to Monitor and Assess Environmental Health in a Portuguese Urban Setting: A Participatory Approach.

Environmental health (EH) is influenced by complex interactions between health and the built and natural environments, there being little research on its specificities in urban settings. The use of suitable indicators to monitor and assess EH is fundamental in informing evidence-based interventions at the local level. A participatory approach to selecting indicators to inform the monitoring and assessment of EH in Lisbon is herein considered. Evidence derived from a systematic review of literature and data from Lisbon and Portuguese databases were analyzed by 12 Portuguese experts in individual semi-structured interviews. The interviews aimed at identifying relevant indicators and important emerging issues in the Lisbon urban setting. The outputs from the interviews were validated by a two-round Web-Delphi process in which panelists (22 experts) from different areas of expertise expressed their views regarding the relevance of the indicators for the analysis of EH in urban settings. Seventeen indicators were validated in the Web-Delphi process. High participation achieved along this process supports the view that this participatory approach was useful for validation. Results from the adopted participatory approach point out gaps in the collection of noise and mobility indicators data and raise emerging issues on housing indicators that require further research. The results also suggest the need for local action to improve indicators and tools in order to help the monitorization of EH in urban contexts. The adopted participatory approach can be replicated for other Portuguese and European urban settings.

Scenarios for population health inequalities in 2030 in Europe: the EURO-HEALTHY project experience.

BACKGROUND: Health inequalities have been consistently reported across and within European countries and continue to pose major challenges to policy-making. The development of scenarios regarding what could affect population health (PH) inequalities across Europe in the future is considered critical. Scenarios can help policy-makers prepare and better cope with fast evolving challenges. OBJECTIVE: This paper describes the three 2030 time-horizon scenarios developed under the EURO-HEALTHY project, depicting the key factors that may affect the evolution of PH inequalities across European regions. METHODS: A three-stage socio-technical approach was applied: i) identification of drivers (key factors expected to affect the evolution of PH inequalities across European regions until 2030) - this stage engaged in a Web-Delphi process a multidisciplinary panel of 51 experts and other stakeholders representing the different perspectives regarding PH inequalities; ii) generation of scenario structures - different drivers' configurations (i.e. their hypotheses for evolution) were organized into coherent scenario structures using the Extreme-World Method; and iii) validation of scenario structures and generation of scenario narratives. Stages ii) and iii) were conducted in two workshops with a strategic group of 13 experts with a wide view about PH inequalities. The scenario narratives were elaborated with the participants' insights from both the Web-Delphi process and the two workshops, together with the use of evidence (both current and future-oriented) on the different areas within the PH domain. RESULTS: Three scenarios were developed for the evolution of PH inequalities in Europe until 2030: 'Failing Europe' (worst-case but plausible picture of the future), 'Sustainable Prosperity' (best-case but plausible picture of the future), and an interim scenario 'Being Stuck' depicting a 'to the best of our knowledge' evolution. These scenarios show the extent to which a combination of Political, Economic, Social, Technological, Legal and Environmental drivers shape future health inequalities, providing information for European policy-makers to reflect upon whether and how to design robust policy solutions to tackle PH inequalities. CONCLUSIONS: The EURO-HEALTHY scenarios were designed to inform both policy design and appraisal. They broaden the scope, create awareness and generate insights regarding the evolution of PH inequalities across European regions.

Multi-criteria decision analysis for health technology assessment: addressing methodological challenges to improve the state of the art.

BACKGROUND: Multi-criteria decision analysis (MCDA) concepts, models and tools have been used increasingly in health technology assessment (HTA), with several studies pointing out practical and theoretical issues related to its use. This study provides a critical review of published studies on MCDA in the context of HTA by assessing their methodological quality and summarising methodological challenges. METHODS: A systematic review was conducted to identify studies discussing, developing or reviewing the use of MCDA in HTA using aggregation approaches. Studies were classified according to publication time and type, country of study, technology type and study type. The PROACTIVE-S approach was constructed and used to analyse methodological quality. Challenges and limitations reported in eligible studies were collected and summarised; this was followed by a critical discussion on research requirements to address the identified challenges. RESULTS: 129 journal articles were eligible for review, 56% of which were published in 2015-2017; 42% focused on pharmaceuticals; 36, 26 and 18% reported model applications, issues regarding MCDA implementation analyses, and proposing frameworks, respectively. Poor compliance with good methodological practice (< 25% complying studies) was found regarding behavioural analyses, discussion of model assumptions and uncertainties, modelling of value functions, and dealing with judgment inconsistencies. The five most reported challenges related to evidence and data synthesis; value system differences and participant selection issues; participant difficulties; methodological complexity and resource balance; and criteria and attributes modelling. A critical discussion on ways to address these challenges ensues. DISCUSSION: Results highlight the need for advancement in robust methodologies, procedures and tools to improve methodological quality of MCDA in HTA studies. Research pathways include developing new model features, good practice guidelines, technologies to enable participation and behavioural research.

Indicators for evaluating European population health: a Delphi selection process.

BACKGROUND: Indicators are essential instruments for monitoring and evaluating population health. The selection of a multidimensional set of indicators should not only reflect the scientific evidence on health outcomes and health determinants, but also the views of health experts and stakeholders. The aim of this study is to describe the Delphi selection process designed to promote agreement on indicators considered relevant to evaluate population health at the European regional level. METHODS: Indicators were selected in a Delphi survey conducted using a web-platform designed to implement and monitor participatory processes. It involved a panel of 51 experts and 30 stakeholders from different areas of knowledge and geographies. In three consecutive rounds the panel indicated their level of agreement or disagreement with indicator's relevance for evaluating population health in Europe. Inferential statistics were applied to draw conclusions on observed level of agreement (Scott's Pi interrater reliability coefficient) and opinion change (McNemar Chi-square test). Multivariate analysis of variance was conducted to check if the field of expertise influenced the panellist responses (Wilk's Lambda test). RESULTS: The panel participated extensively in the study (overall response rate: 80%). Eighty indicators reached group agreement for selection in the areas of: economic and social environment (12); demographic change (5); lifestyle and health behaviours (8); physical environment (6); built environment (12); healthcare services (11) and health outcomes (26). Higher convergence of group opinion towards agreement on the relevance of indicators was seen for lifestyle and health behaviours, healthcare services, and health outcomes. The panellists' field of expertise influenced responses: statistically significant differences were found for economic and social environment (p < 0.05 in round 1 and 2), physical environment (p < 0.01 in round 1) and health outcomes (p < 0.01 in round 3). CONCLUSIONS: The high levels of participation observed in this study, by involving experts and stakeholders and ascertaining their views, underpinned the added value of using a transparent Web-Delphi process to promote agreement on what indicators are relevant to appraise population health.

Detection of sweet potato virus C, sweet potato virus 2 and sweet potato feathery mottle virus in Portugal.

Field sweet potato plants showing virus-like symptoms, as stunting, leaf distortion, mosaic and chlorosis, were collected in southwest Portugal and tested for the presence of four potyviruses, sweet potato virus C (SPVC), sweet potato virus 2 (SPV2), sweet potato feathery mottle virus (SPFMV), sweet potato virus G (SPVG), and the crinivirus sweet potato chlorotic stunt virus (SPCSV). DsRNA fractions were extracted from symptomatic leaves and used as templates in single and multiplex RT-PCR assays using previously described specific primers for each analyzed virus. The amplified reaction products for SPVC, SPV2 and SPFMV were of expected size, and direct sequencing of PCR products revealed that they correspond to the coat protein gene (CP) and showed 98%, 99% and 99% identity, respectively, to those viruses. Comparison of the CP genomic and amino acid sequences of the Portuguese viral isolates recovered here with those of ten other sequences of isolates obtained in different countries retrieved from the GenBank showed very few differences. The application of the RT-PCR assays revealed for the first time the presence of SPVC and SPFMV in the sweet potato crop in Portugal, the absence of SPVG and SPCSV in tested plants, as well as the occurrence of triple virus infections under field conditions.

Using discrete event simulation to compare the performance of family health unit and primary health care centre organizational models in Portugal.

BACKGROUND: Recent reforms in Portugal aimed at strengthening the role of the primary care system, in order to improve the quality of the health care system. Since 2006 new policies aiming to change the organization, incentive structures and funding of the primary health care sector were designed, promoting the evolution of traditional primary health care centres (PHCCs) into a new type of organizational unit--family health units (FHUs). This study aimed to compare performances of PHCC and FHU organizational models and to assess the potential gains from converting PHCCs into FHUs. METHODS: Stochastic discrete event simulation models for the two types of organizational models were designed and implemented using Simul8 software. These models were applied to data from nineteen primary care units in three municipalities of the Greater Lisbon area. RESULTS: The conversion of PHCCs into FHUs seems to have the potential to generate substantial improvements in productivity and accessibility, while not having a significant impact on costs. This conversion might entail a 45% reduction in the average number of days required to obtain a medical appointment and a 7% and 9% increase in the average number of medical and nursing consultations, respectively. CONCLUSIONS: Reorganization of PHCC into FHUs might increase accessibility of patients to services and efficiency in the provision of primary care services.

Nondecision making and inertia in Portuguese health policy.

Despite there having been a positive context for initiating health care reforms in Portugal in the past fifteen years (accompanied by political consensus on the nature of the structural problems within the health care system), there has been a lack of reform initiatives. We use a process-based framework to show how institutional arrangements have influenced Portuguese health care reform. Evidence is presented to demonstrate inertia and nondecision making in three critical areas of Portuguese health policy: clarifying the public-private mix in coverage and provision, creating financial incentives and motivation for human resources, and introducing changes in the pharmaceutical market. Several factors seem to explain these processes, namely, problems in the balance of power within the political system, which have contributed to a lack of proper policy discussion; a lack of pluralism in the formation of health care policies (with low participation from citizens and high mobilization among structural interest groups); and the low priority of health care in public sector reforms. Portuguese politicians should be aware of the pitfalls of the current political system that constrain participatory arrangements and pluralism in policy making. In order to pursue health care reform, future governments will need to counterbalance the strong influence of structural interest groups.