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Background: According to scientific literature, some 99% of patients affected by Alzheimer's disease (AD) suffer from behavioral and psychological symptoms of dementia (BPSD), also known as neuropsychiatric symptoms (NPSs). In particular, agitation is one of the most difficult disorders to treat. States of agitation represent a very serious problem as they make these subjects dangerous for themselves and others and worsen as the disease advances. To date, there are no specific solutions for treating agitation. The only authorized drug is risperidone (as well as brexpiprazole, approved by the FDA on 11 May 2023), which can be used for no longer than 6–12 weeks because it increases the risk of death—owing to cardiocerebrovascular accidents—by 1.6–1.7 times. Methods: In order to address the latter noteworthy unmet medical need, NanoBEO was produced. The aim of the present work is to generate the health technology assessment (HTA) of this nanotechnological device. The latter consists of a controlled release system, based on solid lipid nanoparticles loaded with bergamot essential oil (BEO). Results: The results of the present research assessed the current evidence in the field of non-pharmacological treatments for this condition, including relevant primary preclinical and clinical data studies supporting the use of this device and the production of the operative plan for its launch on the market. The findings offer recommendations for decision-making on its implementation in dementia. Conclusions: NanoBEO represents a public-worth innovation in this neglected area, marking a significant advancement in the history of dementia, moving from academic research to product development. [ABSTRACT FROM AUTHOR]

A health technology assessment (HTA) does not systematically account for the circumstances and needs of children and youth. To supplement HTA processes, we aimed to develop a child-tailored value assessment framework using a multicriteria decision analysis approach. We constructed a multicriteria-decision-analysis-based model in multiple phases to create the Comprehensive Assessment of Technologies for Child Health (CATCH) framework. Using a modified Delphi process with stakeholders having broad disciplinary and geographic variation (N = 23), we refined previously generated criteria and developed rank-based weights. We established a criterion-pertinent scoring rubric for assessing incremental benefits of new drugs. Three clinicians independently assessed comprehension by pilotscoring 9 drugs. We then validated CATCH for 2 childhood cancer therapies through structured deliberation with an expert panel (N = 10), obtaining individual scores, consensus scores, and verbal feedback. Analyses included descriptive statistics, thematic analysis, exploratory disagreement indices, and sensitivity analysis. The modified Delphi process yielded 10 criteria, based on absolute importance/relevance and agreed importance (median disagreement indices = 0.34): Effectiveness, Child-specific Health-related Quality of Life, Disease Severity, Unmet Need, Therapeutic Safety, Equity, Family Impacts, Life-course Development, Rarity, and Fair Share of Life. Pilot scoring resulted in adjusted criteria definitions and more precise score-scaling guidelines. Validation panelists endorsed the framework's key modifiers of value. Modes of their individual prescores aligned closely with deliberative consensus scores. We iteratively developed a value assessment framework that captures dimensions of child-specific health and nonhealth gains. CATCH could improve the richness and relevance of HTA decision making for children in Canada and comparable health systems. • Health technology assessment (HTA) methods for child health technologies are challenged by evidentiary limitations and incomplete consideration of the unique biological and social circumstances and needs of children and youth. Multicriteria decision analysis has emerged as an HTA tool for explicitly considering nonconventional values. • This article presents novel child-specific value assessment criteria based in societal preferences, which reflect the importance of family impacts and long-term flourishing of children beyond immediate health gains obtained through treatment. • A child-tailored multicriteria decision analysis approach facilitating HTA deliberations may broaden the normative lens through which child health technologies are evaluated, contributing to more comprehensive and relevant decision making. [ABSTRACT FROM AUTHOR]

To comprehensively identify and map an exhaustive list of value criteria for the assessment of next-generation sequencing/comprehensive genomic profiling (NGS/CGP), to be used as an aid in decision making. We conducted a systematic review to identify existing value frameworks (VFs) applicable to any type of healthcare technology. VFs and criteria were mapped to a previously published Latin American (LA) VF to harmonize definitions and identify additional criteria and or subcriteria. Based on this analysis, we extracted a comprehensive, evidence-based list of criteria and subcriteria to be considered in the design of a NGS/CGP VF. A total of 42 additional VFs were compared with the LA VF, 88% were developed in high-income countries, 30% targeted genomic testing, and 16% specifically targeted oncology. A total of 242 criteria and subcriteria were extracted; 227 (94%) were fully/partially included in the LA VF; and 15 (6%) were new. Clinical benefit and economic aspects were the most common criteria. VFs oriented to genomic testing showed significant overlap with other VFs. Considering all criteria and subcriteria, a total of 18 criteria and 36 individual subcriteria were identified. Our study provides an evidence-based set of criteria and subcriteria for healthcare decision making useful for NGS/CGP as well as other health technologies. The resulting list can be beneficial to inform decision making and will serve as a foundation to co-create a multistakeholder NGS/CGP VF that is aligned with the needs and values of health systems and could help to improve patient access to high-value technologies. • Value frameworks play a crucial role in enhancing transparency and facilitating informed decision making within the healthcare system. They serve as valuable guides, highlighting key dimensions that need evaluation when assessing various healthcare technologies. • Institute for Clinical Effectiveness and Health Policy recently developed a value framework for diagnostic technologies, based on a targeted systematic review and a participatory process with health technology assessment stakeholders in Latin America. • In this article, we report a systematic review to update the previous framework and develop a comprehensive evidence-based list of criteria and subcriteria that could be considered for creation of a next-generation sequencing/comprehensive genomic profiling-targeted value framework, as well as a starting point for value frameworks targeted to other health technologies. [ABSTRACT FROM AUTHOR]

The new EU Regulation on health technology assessment (HTAR) provides for joint clinical assessments (JCA) of health technologies at EU level. When Member States carry out health technology assessments (HTA) at the national level, they shall give due consideration to the results of a JCA and comply with other obligations of the Regulation. This article aims to clarify what these obligations mean for the Member States and whether JCA results have to be considered outside a national health technology assessment as well. In this context, the question of which processes qualify as 'national HTA' and which requirements need to be fulfilled to trigger the obligations under Article 13 HTAR are discussed in more detail in this paper. [ABSTRACT FROM AUTHOR]

Background: National cancer control plans (NCCPs) are complex public health programs that incorporate evidence‐based cancer control strategies to improve health outcomes for all individuals in a country. Given the scope of NCCPs, small and vulnerable populations, such as patients with childhood cancer, are often missed. To support planning efforts, a rapid, modifiable tool was developed that estimates a context‐specific national budget to fund pediatric cancer programs, provides 5‐year scale‐up scenarios, and calculates annual cost‐effectiveness. Methods: The tool was codeveloped by teams of policymakers, clinicians, and public health advocates in Zimbabwe, Zambia, and Uganda. The 11 costing categories included real‐world data, modeled data, and data from the literature. A base‐case and three 5‐year scale‐up scenarios were created using modifiable inputs. The cost‐effectiveness of the disability‐adjusted life years averted was calculated. Results were compared with each country's projected gross domestic product per capita for 2022 through 2026. Results: The number of patients/total budget for year 1 was 250/$1,109,366 for Zimbabwe, 280/$1,207,555 for Zambia, and 1000/$2,277,397 for Uganda. In year 5, these values were assumed to increase to 398/$5,545,445, 446/$4,926,150, and 1594/$9,059,331, respectively. Base‐case cost per disability‐adjusted life year averted/ratio to gross domestic product per capita for year 1, assuming 20% survival, was: $807/0.5 for Zimbabwe, $785/0.7 for Zambia, and $420/0.5 for Uganda. Conclusions: This costing tool provided a framework to forecast a budget for childhood‐specific cancer services. By leveraging minimal primary data collection with existing secondary data, local teams obtained rapid results, ensuring that childhood cancer budgeting is not neglected once in every 5 to 6 years of planning processes. The Childhood Cancers Budgeting Rapidly to Incorporate Disadvantaged Groups for Equity (CC‐BRIDGE) tool fills a current gap by providing a feasible, simplified toolkit for policymakers and childhood cancer stakeholders to calculate the costs required to develop a multiyear childhood cancer–specific budget, within existing National Cancer Control Plan (NCCP) activities. As governments continue to plan NCCPs, the CC‐BRIDGE tool can ensure that budgeting for vulnerable populations, such as childhood cancer patients, is not neglected once in every 5 to 6 years of planning processes. [ABSTRACT FROM AUTHOR]

Chronic RhinoSinusitis with Nasal Polyps (CRSwNP) represents a condition mainly caused by the type 2 inflammation presence and marked by the existence of polyps within the nasal and paranasal sinuses. The standard of care includes intranasal steroids, additional burst of systemic steroids, if needed, and surgery. However, recurrence is common, especially among patients with comorbid type 2 inflammatory diseases. Recently, biological drugs, addressing the underlying cause of the disease, have been approved in Italy (dupilumab, omalizumab, and mepolizumab). A Health Technology Assessment was conducted to define multidimensional impact, assuming Italian NHS perspective and a 12-month time horizon. The EUnetHTA Core Model was deployed, using the following methods to analyze the domains: (i) literature evidence; (ii) administration of semi-structured questionnaires to 17 healthcare professionals; (iii) health economics tools to define the economic sustainability for the system. Evidence from NMA and ITC showed a more favorable safety profile and better efficacy for dupilumab compared with alternative biologics. All the analyses, synthesizing cost and efficacy measures, showed that dupilumab is the preferable alternative. Specifically, the cost per responder analysis for dupilumab, exhibiting a 67.0% response rate at Week 52, is notably economical at 14,209EUR per responder. This presents a more economical profile compared with the cost per responder for omalizumab (36.2% response rate) at 24,999EUR and mepolizumab (28.5% response rate) at 31,863EUR. These results underscore dupilumab's potential, not merely in terms of clinical outcomes, but also in terms of economic rationality, thereby solidifying its status as a valid and preferrable alternative in the management of CRSwNP, in the context of the Italian NHS. [ABSTRACT FROM AUTHOR]

Background: There is limited evidence-informed guidance on TISP processes for countries where health technology assessment (HTA) is in a nascent phase. We aimed to explore the range of topic identification, selection and prioritization (TISP) processes and practices for HTA in selected countries and identify aspects relevant to emerging HTA systems. Methods: This mixed design study included a systematic literature review, an electronic survey, and individual interviews. We conducted a systematic literature review with criteria that were developed a priori to identify countries deemed to have a recently formalized HTA system. Based on the literature review, a twenty-three item online survey was shared with the identified countries, we completed follow-up interviews with ten participants who have experience with HTA. We analyzed documents, survey responses and interview transcripts thematically to identify lessons related to TISP processes and practices. Results: The literature review identified 29 nine candidate countries as having a "potential" recently formalized HTA system. Twenty-one survey responses were analyzed and supplemented with ten individual interviews. We found variation in countries' approaches to TISP — particularly between pharmaceutical and non-pharmaceutical interventions. Results indicate that TISP is heavily driven by policy makers, expert involvement, and to a lesser extent, relevant stakeholders. The use of horizon-scanning and early warning systems is uncommon. Interviewee participants provided further insight to the survey data, reporting that political awareness and an institutional framework were important to support TISP. TISP can be optimized by stronger national regulations and legislative structures, in addition to education and advocacy about HTA among politicians and decision-makers. In some settings regional networks have been useful, particularly in the development of TISP guidelines and methodologies. Additionally, the technical capacity to conduct TISP, and access to relevant local data were factors limiting TISP in national settings. Increased network collaboration and capacity building were reported as future needs. Conclusions: This study provides current insights into a topic where there is limited published peer reviewed literature. TISP is an important first step of HTA, and topics should be selected and prioritized based on local need and relevance. The limited capacity for TISP in settings where HTA is emerging may be supported by local and international collaboration to increase capacity and knowledge. To succeed, both TISP and HTA need to be embedded within national health care priority setting and decision-making. More in-depth understanding of where countries are situtated in formalizing the TISP process may help others to overcome factors that facilitate or hinder progress. [ABSTRACT FROM AUTHOR]

The Half-Transform Ansatz (HTA) is a proposed method to solve hyper-geometric equations in Quantum Phase Space by transforming a differential operator to an algebraic variable and including a specific exponential factor in the wave function, but the mechanism which provides this solution scheme is not known. Analysis of the HTA’s application to the Hydrogen atom suggests an underlying mechanism which the HTA is a part of. Observations of exponential factors that act on the wave function naturally suggest modeling quantum operator definitions as a point on a Riemannian manifold in the 4D Operator Space, a novel idea we call the Operator Space Manifold Theory. On this manifold, we explore the concepts of superposition, regions of unique energy eigenvalues, and translation operators. We also find the theoretical backing to derive the HTA and how Operator Space Manifold Theory can be used to describe and solve quantum systems by manipulating how a quantum state perceives position and momentum. [ABSTRACT FROM AUTHOR]

AlN epilayers were grown on magnetron-sputtered (MS) (11–22) AlN buffers on m-plane sapphire substrates at 1450 °C via hydride vapour phase epitaxy (HVPE). The MS buffers were annealed at high temperatures of 1400–1600 °C. All the samples were characterised using X-ray diffraction, atomic force microscopy, scanning electron microscope and Raman spectrometry. The crystal quality of epilayers regrown by HVPE was improved significantly compared to that of the MS counterpart. With an increasing annealing temperature, the crystal quality of both MS buffers and AlN epilayers measured along [11–23] and [1–100] improved first and then decreased, maybe due to the decomposition of MS buffers, while the corresponding anisotropy along the two directions decreased first and then increased. The optimum quality of the AlN epilayer was obtained at the annealing temperature of around 1500 °C. In addition, it was found that the anisotropy for the epilayers decreased significantly compared to that of annealed MS buffers when the annealing temperature was below 1500 °C. [ABSTRACT FROM AUTHOR]

Objective: To assess the profile of metabolic syndrome (MS) in a hypertensive population in Mali. Patients and methods: Prospective study from June 1, 2022 to June 31, 2022, in the cardiology department of the Mother-Child University Hospital in Bamako, Luxembourg. Our study sample consisted of adult hypertensive patients, aged 18 years and older, received as an outpatient. Patients were included in our study after informed consent. The SM was established based on International Diabetes Federation (IDF) definitions. Results: Out of 520 patients, 238 were included in our study. The prevalence of SM was 30.2% according to IDF criteria. There was a female predominance (66%). The mean age was 57 ± 5 years. Grade II hypertension was in the majority with 39%. Seventy-five percent of patients with metabolic syndrome had 3 risk factors. Ischemic stroke was the most common complication with a rate of 12%. Dual therapy was indicated in 59.23% of patients. Calcium channel blockers were the most prescribed with a rate of 74.5%. SM is a reality in Mali. Adequate preventive measures are necessary to limit its progression. [ABSTRACT FROM AUTHOR]

BACKGROUND: High-speed health trains are important tools and guarantees in major accidents, epidemic pandemics, disasters, and warfare health care, and the health trains currently developed for common train platforms have more functional defects. OBJECTIVE: The purpose of this study is to analyze the relationship between the medical transfer system and the medical system, and to obtain a better medical transfer train formation through the established model. METHODS: Based on the case study of medical transport tools, this paper analyzes the components and interrelationships of the medical transport system and the medical system, and then analyzes the medical transport task process of the health train by using the hierarchical task analysis (HTA) method. Combined with the Chinese standard EMU, a medical transport task model of the high-speed health train is established. Through this model, the functional compartment unit of the high-speed health train and the marshaling scheme of the high-speed health train are obtained. RESULTS: The expert system is used to evaluate the scheme. The results show that the train formation scheme formulated by the model in this paper is superior to other train formation schemes in three indicators, meeting the requirements of large medical transfer tasks. CONCLUSION: The results of this study can improve the ability of on-site treatment of patients, and can provide a basis for the research and development of a high-speed health train, which has a certain practical application value. [ABSTRACT FROM AUTHOR]

Since the groundwork published by Torres-Vega and Frederick, the Quantum Phase Space Representation (QPSR) has been explored as a method for solving a multitude of physical systems and describing phenomena. Most recently, Valentino A. Simpao has developed a method, the Heaviside Operational Ansatz, to solve the Time Dependent Schrodinger Equation (TDSE) in the QPSR, but there are still no general, direct methods to solve the Time Independent Schrodinger Equation in the QPSR. There is also no current formulation of quarkonium in phase space. In this paper, we describe the strong interactions of non-relativistic heavy quarks using the Cornell potential, and present a method, the Half-Transform Ansatz, to cast the Schrodinger Equation into a hyper-geometric form which can be solved for the phase space wave function and its energy eigenvalues using the Nikiforov-Uvarov method. This solution can be generalized for any two particle system with a scleronomic potential made up of polynomial and reciprocal terms. These results are compared to experimental results and other theoretical models. We also analyze the behavior of these wave functions, which suggest a correlation between radial momentum and the upper limit of existence in charm-anticharm mesons. [ABSTRACT FROM AUTHOR]

Objective. This study aims to investigate the risk factors associated with hypertension in pregnant women through a comprehensive literature review. Methodology. A retrospective bibliographic investigation was conducted by searching for relevant information in both Spanish and English languages using databases such as PubMed, Scielo, Medline, NIH, and Elsevier. Results. A total of 33 articles were included in this research, covering various aspects including definitions, etiology, classification, risk factors, and laboratory examinations related to hypertension during pregnancy. Conclusions. Hypertension during pregnancy is a pathological condition that not only affects the mother but also poses risks to the fetus. The mortality rate among pregnant women with hypertension is estimated at 35%. Failure to timely detect and diagnose hypertension can result in complications such as hemorrhages, early placental detachment, spontaneous abortions, or preterm births, which may lead to maternal and fetal mortality. Biochemical markers and regular blood chemistry or urine analysis tests can aid in the diagnosis of this condition. [ABSTRACT FROM AUTHOR]

Purpose: 2021 marks the tenth anniversary of the AMNOG process and brought with it a new German administration—two good reasons to take stock of where we stand today, what has been achieved so far, and how the path of early benefit assessments in Germany should continue. Results: From the perspective of manufacturers of cancer drugs, the AMNOG process, as a constantly evolving system, has for the most part proved itself—which does not mean that there is no longer room for improvement. Significant improvements have been achieved in the area of early consultation of medical societies regarding the selection of the appropriate comparator therapy as well as in the reimbursement of biomarker diagnostic tests in the outpatient sector. However, there is still a need for improvement, especially in the areas of patient-relevant outcomes accepted by the G-BA, the inclusion of real-world data in evidence assessments, or the transfer of evidence from certain patient groups to others. Conclusion: The current AMNOG structures were developed for the most part at a time when no one saw immuno-oncology or gene and cell therapies coming, when there were no multi-tumor drug approvals, and when few imagined that within a few years, the established tumor entities would be broken down into dozens of sub-entities on the basis of molecular genetic markers. Society wants these and other advances, and the HTA process must, therefore, take this into account in a healthcare system based on solidarity. [ABSTRACT FROM AUTHOR]

Introduction: Patient and public involvement (PPI) is an expectation when conducting research, including Health Technology Assessment (HTA), but practical guidance for those wishing to embed PPI into the grant application process is not always easily accessible. We wanted to ensure that PPI was central when preparing a proposal for an investigator‐led evidence synthesis HTA investigating nonsurgical interventions for pelvic organ prolapse (POP) in women. Here, we describe our methods. Methods: We recruited two patient co‐applicants separately through an open process to help ensure that patient voice was present within our proposal's management and direction. We invited co‐applicants to attend research team meetings and comment on the full proposal. We designed, recruited to and facilitated a scoping workshop, as well as undertook its subsequent evaluation. The insight shared within the workshop for patients with a lived experience of POP, including our patient co‐applicants, helped us develop the scope and rationale behind our HTA proposal. We particularly considered the interventions to include within the evidence synthesis. We also considered the outcome measures for both the evidence synthesis and economic evaluation. We elicited ideas about where and how results could be disseminated. Feedback suggested the workshop was as valuable for the attendees as it was for the researchers, making them feel valued and listened to. The time spent by researchers working on the activity was substantial and not directly funded but a necessary and valuable activity in developing our potential HTA. Our work was informed using the UK Standards for Public Involvement and the Authors and Consumers Together Impacting on eVidencE (ACTIVE) framework. Conclusions: PPI can be enormously valuable in both developing and strengthening research proposals. However, further guidance is needed to help researchers recognise the level and type of involvement to use at this early stage, particularly given the large time investment needed to embed meaningful PPI. Patient and Public Contribution: Women with a lived experience of POP were involved at every stage of the grant application process; their involvement is documented in full throughout this work. [ABSTRACT FROM AUTHOR]

Background: The present paper aims at evaluating the potential benefits of high-energy devices (HEDs) in the Italian surgical practice, defining the comparative efficacy and safety profiles, as well as the potential economic and organizational advantages for hospitals and patients, with respect to standard monopolar or bipolar devices. Methods: A Health Technology Assessment was conducted in 2021 assuming the hospital perspective, comparing HEDs and standard monopolar/bipolar devices, within eleven surgical settings: appendectomy, hepatic resections, colorectal resections, cholecystectomy, splenectomy, hemorrhoidectomy, thyroidectomy, esophago-gastrectomy, breast surgery, adrenalectomy, and pancreatectomy. The nine EUnetHTA Core Model dimensions were deployed considering a multi-methods approach. Both qualitative and quantitative methods were used: (1) a systematic literature review for the definition of the comparative efficacy and safety data; (2) administration of qualitative questionnaires, completed by 23 healthcare professionals (according to 7-item Likert scale, ranging from − 3 to + 3); and (3) health-economics tools, useful for the economic evaluation of the clinical pathway and budget impact analysis, and for the definition of the organizational and accessibility advantages, in terms of time or procedures' savings. Results: The literature declared a decrease in operating time and length of stay in using HEDs in most surgical settings. While HEDs would lead to a marginal investment for the conduction of 178,619 surgeries on annual basis, their routinely implementation would generate significant organizational savings. A decrease equal to − 5.25/−9.02% of operating room time and to − 5.03/−30.73% of length of stay emerged. An advantage in accessibility to surgery could be hypothesized in a 9% of increase, due to the gaining in operatory slots. Professionals' perceptions crystallized and confirmed literature evidence, declaring a better safety and effectiveness profile. An improvement in both patients and caregivers' quality-of-life emerged. Conclusions: The results have demonstrated the strategic relevance related to HEDs introduction, their economic sustainability, and feasibility, as well as the potentialities in process improvement. [ABSTRACT FROM AUTHOR]

New therapies in a publicly funded healthcare system are first appraised by health technology assessment agencies that provide funding recommendations to the payers. Treatment with Chimeric Antigen Receptor-T cell (CAR-T) therapy is revolutionizing the management of patients with relapsed/refractory aggressive B-cell lymphoma by providing an effective alternative to the standard of care. Yet, the implementation of CAR-T treatment has a substantial impact on the healthcare system due to its high cost, complex manufacturing process, and requirement for highly specialized services and expertise. CAR-T Cells, as a "living drug", are fundamentally different from usual medications, and their approvals and funding recommendations pose unique challenges to the health technology agency. In this paper, we explore the specific challenges that face the health technology agencies in reviewing reimbursement recommendations for CAR-T therapy. We take a Canadian perspective and use CAR-T treatment of relapse/refractory aggressive B-cell lymphoma as an example. [ABSTRACT FROM AUTHOR]

Background: Few antibiotics have entered the market in recent years despite the need for new treatment options. Some of the challenges of bringing new antibiotics to market are linked to the marketing authorization and health technology assessment (HTA) processes. Research shows great variation in geographic availability of new antibiotics, suggesting that market introduction of new antibiotics is unpredictable. We aimed to investigate regulatory authorities' and HTA agencies' role in developing non-financial incentives to stimulate antibiotic research and development (R&D). Methods: We conducted individual, semi-structured, stakeholder interviews. Participants were recruited from regulatory authorities (EMA and FDA) and HTA agencies in Europe. Participants had to be experienced with assessment of antibiotics. The data were analyzed using a deductive and inductive approach to develop codes and identify key themes. Data were analyzed using thematic analysis including the constant comparison method to define concepts, and rival thinking to identify alternative explanations. Results: We found that (1) interpretation of key concepts guiding the understanding of what type of antibiotics are needed vary (2) lack of a shared approach on how to deal with limited clinical data in the marketing authorization and HTA processes is causing barriers to getting new antibiotics to market (3) necessary adaptations to the marketing authorization process causes uncertainties that transmit to other key stakeholders involved in delivering antibiotics to patients. Conclusions: A shared understanding of limited clinical data and how to deal with this issue is needed amongst stakeholders involved in antibiotic R&D, marketing authorization, and market introduction to ensure antibiotics reach the market before resistance levels are out of control. Regulatory authorities and HTA agencies could play an active role in aligning the view of what constitutes an unmet medical need, and direct new economic models towards stimulating greater diversity in the antibiotic armamentarium. [ABSTRACT FROM AUTHOR]

Objectives: Dealing with uncertainty is one of the critical topics in health technology assessment. The greater decision uncertainty in appraisals, the less clear the clinical- and cost-effectiveness of the health technology. Although the development of targeted cancer therapies (TCTs) has improved patient health care, additional complexity has been introduced in drug appraisals due to targeting more specific populations. Real-world data (RWD) are expected to provide helpful information to fill the evidence gaps in appraisals. This study compared appraisals of TCTs with those of non-targeted cancer therapies (non-TCTs) regarding sources of uncertainty and reviewed how RWD have been used to supplement the information in these appraisals.Methods: This study reviews single technology appraisals (STAs) of oncology medicines performed by the National Institute for Health and Care Excellence (NICE) over 11 years up to December 2021. Three key sources of uncertainty were identified for comparison (generalisability of clinical trials, availability of direct treatment comparison, maturity of survival data in clinical trials). To measure the intensity of use of RWD in appraisals, three components were identified (overall survival, volume of treatment, and choice of comparators).Results: TCTs received more recommendations for provision through the Cancer Drugs Fund (27.7, 23.6% for non-TCT), whereas similar proportions were recommended for routine commissioning. With respect to sources of uncertainty, the external validity of clinical trials was greater in TCT appraisals (p = 0.026), whereas mature survival data were available in fewer TCT appraisals (p = 0.027). Both groups showed similar patterns of use of RWD. There was no clear evidence that RWD have been used more intensively in appraisals of TCT.Conclusions: Some differences in uncertainty were found between TCT and non-TCT appraisals. The appraisal of TCT is generally challenging, but these challenges are neither new nor distinctive. The same sources of uncertainty were often found in the non-TCT appraisals. The uncertainty when appraising TCT stems from insufficient data rather than the characteristics of the drugs. Although RWD might be expected to play a more active role in appraisals of TCT, the use of RWD has generally been limited. [ABSTRACT FROM AUTHOR]

High blood pressure, a relatively rare pathology diagnosed in pediatric age, is becoming more and more common in the diagnoses of children, thus becoming a real health problem, with the alarming increase in obesity in this age group. The prevalence of hypertension in children differs depending on age groups. In adolescents, the percentage reaches 10%, and in newborns at term 0.2%, therefore 3.5% of children are diagnosed with hypertension. Peripheral vascular resistance and cardiac output determine the force that blood flow exerts on the arterial wall and thus define blood pressure. Depending on the stage of hypertension and the clinician’s analysis, the management of hypertension is guided by two pillars: lifestyle along with non-pharmacological measures and drug therapy. A hypertensive child today is a future hypertensive adult tomorrow, for this reason we consider hypertension a real issue, especially in the context in which pediatric obesity is a global health problem. [ABSTRACT FROM AUTHOR]

Introduction: Drug reimbursement decisions are often made based on a price set by the manufacturer. In some cases, this price leads to public and scientific debates about whether its level can be justified in relation to its costs, including those related to research and development (R&D) and manufacturing. Such considerations could enter the decision process in collectively financed health care systems. This paper investigates whether manufacturers' costs in relation to drug prices, or profit margins, are explicitly mentioned and considered by health technology assessment (HTA) organisations. Method: An analysis of reimbursement reports for cancer drugs was performed. All relevant Dutch HTA-reports, published between 2017 and 2019, were selected and matched with HTA-reports from three other jurisdictions (England, Canada, Australia). Information was extracted. Additionally, reimbursement reports for three cases of expensive non-oncolytic orphan drugs prominent in pricing debates in the Netherlands were investigated in depth to examine consideration of profit margins. Results: A total of 66 HTA-reports concerning 15 cancer drugs were included. None of these reports contained information on manufacturer's costs or profit margins. Some reports contained general considerations of the HTA organisation which related prices to manufacturers' costs: six contained a statement on the lack of price setting transparency, one mentioned recouping R&D costs as a potential argument to justify a high price. For the case studies, 21 HTA-reports were selected. One contained a cost-based price justification provided by the manufacturer. None of the other reports contained information on manufacturer's costs or profit margins. Six reports contained a discussion about lack of transparency. Reports from two jurisdictions contained invitations to justify high prices by demonstrating high costs. Conclusion: Despite the attention given to manufacturers' costs in relation to price in public debates and in the literature, this issue does not seem to get explicit systematic consideration in the reimbursement reports of expensive drugs. [ABSTRACT FROM AUTHOR]

Introduction-- The goal of this work is to present i* framework as alternative to support design decisions in the Human Computer Interaction Area (HCI), mainly, in the process of task analysis. We show i* framework a modeling language suitable for an early phase of software system modeling to understand the problem domain, also, that provides methods to represent tasks, as well as support design decisions the development that satisfies the process requirements from the beginning until the end. To achieve our goal, approaches addressed to analyze notations aimed at task analysis and their representation. Finally, we present suggestions for improvement in the i* framework through a proposed extension for refining task model, instantiating examples and justifying our proposal through peer review. [ABSTRACT FROM AUTHOR]

The focus on health maximisation in a healthcare economic evaluation (HEE) – that is health gains are of equal value regardless of the recipient– has significant implications as health systems attempt to address persistent and growing health inequities. This study aimed to systematically compare and contrast the equity principles of different health technology assessment (HTA) agencies and how equity is addressed in HEE guidelines. HTA agencies were identified through the ISPOR, GEAR, iDSI, HTAi, INAHTA, HTAsiaLink, and RedETSA websites in June 2021 and updated in August 2023. Agencies websites were then searched to retrieve HEE guidelines. The guidelines were grouped into two categories: well-established and newly-developed agency guidelines, based on whether or not they published their first guidelines before 2009. Data extracted summarised the methodological details in the reference cases, including specifics on how equity featured and in what role. In those agencies where equity did not feature explicitly in the HEE guidelines, an additional search of the agency website was undertaken to understand if equity featured in those agencies' decision-making frameworks. The study included 46 guidelines from 51 countries. Only 30% of the guidelines were explicit about the equity assumptions. Health equity (using a broad definition) was mentioned in 29 guidelines and 14 included a specific definition while only seven recommended specific methods to incorporate inequalities. Addressing equity concerns was usually suggested as an additional analyses rather than a key part of the assessment. It was unclear how equity was incorporated into decision-making processes. In addition, equity was mentioned in other guidance – such as decision-making frameworks - provided by five agencies that did not mention it in the HEE guidelines, and 7 of 14 topic selection criteria that were identified. Equity is given less attention than efficiency in HEE guidelines. This indicates that HTA agencies while subscribing to an extra-welfarist approach have a narrow evaluative space – focusing on maximising health and not considering the opportunity cost of the equity constraint. The omission of equity and the lack of systematic approaches in guidelines poses a threat to the international endeavours to reduce inequities. It is timely for HTA agencies to reconsider their positions on equity explicitly. • Equity mentioned in 29 of 46 guidelines of HTA bodies, only 13 give methods to address inequalities. • Equity assumptions underlying economic evaluations is explicit in 14 guidelines. • The guidelines place a greater emphasis on efficiency rather than fairness. • Explicit consideration of equity needed to help deliver equitable health outcomes. [ABSTRACT FROM AUTHOR]

The traditional health economic analysis is limited to estimating the impact on the treated patient. As vaccines are usually aimed at preventing infectious diseases, they may be associated with additional values for the non-treated wider population. Although there are valid reasons for treating vaccines differently, and a wide support for a broader perspective in the literature (i.e., beyond the net costs and health gain related to the outcome for the vaccinated individual), it remains unclear to what extent the Health Technology Assessment (HTA) agencies accept and apply a broader perspective. The purpose of this study is to examine and discuss what type of consequences are relevant for a health economic analysis of vaccines and which consequences are considered by HTA agencies. The study includes a strategic review of literature and HTA decisions in Sweden and other countries, online round-table discussions with stakeholders in Sweden, and a basic estimation of the value of a COVID-19 vaccination in Sweden. The study shows that, other than herd effect, broader economic consequences for the population are generally not included in the economic evaluation of vaccines. Also, all economic consequences for the treated patient (production loss) and caregiver (health loss) are not always considered. The perspective chosen can have a major impact on the outcome of the analysis. A vaccine for COVID-19 is estimated to provide a value of €744–€956 per dose when using a societal perspective including broader consequences for the population. Providing a complete and appropriate picture of the value of vaccination is of importance to allocate resources efficiently, to provide incentives for vaccine development, and to show the cost of delaying decisions to implement a new vaccine. [ABSTRACT FROM AUTHOR]

There is an alarming delay in Europe for anticancer medicines becoming accessible for children. Following a paediatric European Union marketing authorisation, national Health Technology Assessment (HTA) agencies evaluate effectiveness, and safety of medicines to support decision on their cost and reimbursement. This study (a SIOPE Access to Medicines project) aimed to evaluate how these HTA evaluations take place for anticancer medicines indicated for paediatric use in Europe and to explore where the delays for market access originate. We obtained HTA reports from the public domain for nine European countries for blinatumomab, dinutuximab beta and tisagenlecleucel. We evaluated the time elapsed between marketing authorisation for a paediatric indication and a national HTA decision and the nature of the decision. Out of 23 HTA decisions (four countries without blinatumomab report), 18 were positive, two with restrictions, three negative. For blinatumomab, tisagenlecleucel and dinutuximab beta, the median time to an HTA decision after regulatory approval for paediatric use was 353 days (range 193–751), 141 days (range 77–517) and 515 days (range 0–780), respectively, with variability between countries. Dinutuximab beta and tisagenlecleucel were first introduced in children, but did not result in shorter time to HTA decision. For blinatumomab, marketing authorisation followed 1008 days after the indication in adults, with HTA applications submitted a median of 167 days later, and a recommendation after 145 days. This study reveals ample variability in HTA decision making in nine European Union countries. Collaboration and alignment of required evidence is needed to facilitate robust scientific HTA assessments, also considering methodological challenges in paediatric oncology. • Health technology assessments of paediatric oncology drugs vary greatly within Europe. • The timing of HTA evaluation adds to the delay of patient access. • Differences between adult and paediatric trial methodology should be considered. • Multistakeholder collaboration between EMA, academia, pharmaceutical companies, patients advocates and HTAs is needed. [ABSTRACT FROM AUTHOR]

Introduction: The incidence of foot fractures is increasing. These types of fractures represent the most expensive group of diagnoses in the emergency department. Next to this, the need for economic evaluation studies is growing fast. The aim of this article is to provide healthcare professionals with an introduction to economic evaluation studies in the field of foot fractures.Types Of Economic Evaluation Studies: Four types of economic evaluation studies exist: cost-minimization analysis (CMA), cost-benefit analysis (CBA), cost-effectiveness analysis (CEA), and cost-utility analysis (CUA). An economic evaluation study can be either trial- or model-based.Cost Assessment: When assessing costs in an economic evaluation study, three steps need to be made: identification of costs, measurement of costs, and valuation of costs.Perspective Of Analysis: Two main perspectives exist in economic evaluation studies. When using a healthcare perspective, only the potential costs and benefits of an intervention for the healthcare sector are included. The societal perspective includes all potential costs, including societal costs.Synthesis Of Costs and Effects and Uncertainty Analysis: The level of cost-effectiveness can be objectively expressed using the incremental cost-effectiveness ratio (ICER). This measure can be plotted in a cost-effectiveness plane and can be compared with existing regional ceiling ratios.Conclusion: Although this article is not a guideline for economic evaluations, we do want to present five tips to consider when performing an economic evaluation. Firstly, when measuring resource use, consult the Database of Instruments for Resource Use Measurements (DIRUM) to find an appropriate instrument. Secondly, when measuring utility values, use the EuroQol questionnaire if possible. Thirdly, when setting up an economic evaluation study, consult the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) database for the appropriate pharmacoeconomic guidelines. Fourthly, consult international guidelines to decide which study design is most appropriate. Finally, when performing an economic evaluation, consult a heath technology assessment (HTA) specialist from the start to ensure methodological quality. [ABSTRACT FROM AUTHOR]

Objective This analysis aimed to assess patient advocacy groups' (PAGs) perspectives on patients' and PAGs' potential role in Health Technology Assessment (HTA) decision-making in Greece. Methods In total, 22 representatives of PAGs participated in a consensus panel meeting. Participants' views were elicited via a consensus panel meeting, through a televoting procedure, based on a structured questionnaire with close-ended questions. Voting was anonymous, to avoid the influence of the dominant personality. After voting, the results were presented to the participants and televoting was repeated to achieve consensus. Key findings The majority of participants (94.8%) believe that valid information on the developments regarding HTA in Greece is not conveyed to them by the official national regulatory sources. Key sources of valid information on developments regarding HTA were: conferences (95.2%), international PAGs (85.7%) and HTA experts/scientists (76.2%). About 76.2% of participants evaluated PAGs' competence to participate in policy-making concerning HTA scheme formation as moderate or higher. Regarding patients' effective participation in HTA decision-making, greater importance (points 4 and 5 on a 5-point scale) was given to their education on treatment effects in terms of quality of life (100.0%), basic principles of health economics and pharmaceutical policy (95.5%) and ethical aspects of HTA (95.0%). About 55.0% strongly agree that patients should formally express their opinion on HTA issues, while 72.7% believe that patients' opinions on HTA issues should be expressed through participation in the process and right to vote. Conclusions The participation of patients in HTA decision-making is an issue not only of justice but also of essence. For a productive participation, PAGs training is essential. [ABSTRACT FROM AUTHOR]

Objectives: Immunization programs in low-income and middle-income countries (LMICs) are faced with an ever-growing number of vaccines of public health importance recommended by the World Health Organization, while also financing a greater proportion of the program through domestic resources. More than ever, national immunization programs must be equipped to contextualize global guidance and make choices that are best suited to their setting. The CAPACITI decision-support tool has been developed in collaboration with national immunization program decision makers in LMICs to structure and document an evidence-based, context-specific process for prioritizing or selecting among multiple vaccination products, services, or strategies.Methods: The CAPACITI decision-support tool is based on multi-criteria decision analysis, as a structured way to incorporate multiple sources of evidence and stakeholder perspectives. The tool has been developed iteratively in consultation with 12 countries across Africa, Asia, and the Americas.Results: The tool is flexible to existing country processes and can follow any type of multi-criteria decision analysis or a hybrid approach. It is structured into 5 sections: decision question, criteria for decision making, evidence assessment, appraisal, and recommendation. The Excel-based tool guides the user through the steps and document discussions in a transparent manner, with an emphasis on stakeholder engagement and country ownership.Conclusions: Pilot countries valued the CAPACITI decision-support tool as a means to consider multiple criteria and stakeholder perspectives and to evaluate trade-offs and the impact of data quality. With use, it is expected that LMICs will tailor steps to their context and streamline the tool for decision making. [ABSTRACT FROM AUTHOR]

Objectives: The availability of novel, more efficacious and expensive cancer therapies is increasing, resulting in significant treatment effect heterogeneity and complicated treatment and disease pathways. The aim of this study is to review the extent to which UK cancer technology appraisals (TAs) consider the impact of patient and treatment effect heterogeneity.Methods: A systematic search of National Institute for Health and Care Excellence TAs of colorectal, lung and ovarian cancer was undertaken for the period up to April 2020. For each TA, the pivotal clinical studies and economic evaluations were reviewed for considerations of patient and treatment effect heterogeneity. The study critically reviews the use of subgroup analysis and real-world translation in economic evaluations, alongside specific attributes of the economic modeling framework.Results: The search identified 49 TAs including 49 economic models. In total, 804 subgroup analyses were reported across 69 clinical studies. The most common stratification factors were age, gender, and Eastern Cooperative Oncology Group performance score, with 15% (119 of 804) of analyses demonstrating significantly different clinical outcomes to the main population; economic subgroup analyses were undertaken in only 17 TAs. All economic models were cohort-level with the majority described as partitioned survival models (39) or Markov/semi-Markov models. The impact of real-world heterogeneity on disease progression estimates was only explored in 2 models.Conclusion: The ability of current modeling approaches to capture patient and treatment effect heterogeneity is constrained by their limited flexibility and simplistic nature. This study highlights a need for the use of more sophisticated modeling methods that enable greater consideration of real-world heterogeneity. [ABSTRACT FROM AUTHOR]

Objectives: A recent debate in health economics and outcomes research community identified option value as one of the elements warranting consideration in the assessment of medical technologies. To conduct a scoping review of contributions on option value in the healthcare sector and identify relevant conceptual aspects and methods used to incorporate it in standard economic evaluations.Methods: A systematic search was conducted up to July 2020 to identify contributions from electronic bibliographic database and gray literature. Data on the proposed definitions of option value, theoretical implications of its use in economic evaluations, and methods used to estimate it were extracted and analyzed.Results: We found 57 eligible studies. Three different definitions emerged: insurance value, real option value, and option value of survival. Focusing on the latter (24 studies), we analyzed in depth 8 empirical applications across 7 therapeutic areas. The most relevant methodological challenges were on the perspective used in economic evaluations and how to robustly manage forecasting uncertainty, update cost-effectiveness thresholds, and avoid double-counting issues. For empirical studies assessing the total value of the technology, including option value, estimates ranged from +7% to +469% of its conventional value.Conclusions: This review synthesizes theoretical and empirical aspects on option value of healthcare technologies and proposes a terminology to distinguish 3 different concepts identified. Future work should focus primarily on agreeing on whether option value should be included in economic evaluations and, if so, on developing and validating reliable methods for its ex-ante estimation. [ABSTRACT FROM AUTHOR]

Standard outcome sets developed by the International Consortium for Health Outcomes Measurement (ICHOM) facilitate value-based health care in healthcare practice and have gained traction from regulators and Health Technology Assessment (HTA) agencies that regularly assess the value of new medicines. We aimed to assess the extent to which the outcomes used by regulators and HTA agencies are patientrelevant, by comparing these to ICHOM standard sets. We conducted a cross-sectional comparative analysis of ICHOM standard sets, and publicly available regulatory and HTA assessment guidelines. We focused on oncology due to many new medicines being developed, which are accompanied by substantial uncertainty regarding the relevance of these treatments for patients. A comparison of regulatory and HTA assessment guidelines, and ICHOM standard sets showed that both ICHOM and regulators stress the importance of disease-specific outcomes. On the other hand, HTA agencies have a stronger focus on generic outcomes in order to allow comparisons across disease areas. Overall, similar outcomes are relevant for market access, reimbursement, and in ICHOM standard sets. However, some differences are apparent, such as the acceptability of intermediate outcomes. These are recommended in ICHOM standard sets, but regulators are more likely to accept intermediate outcomes than HTA agencies. A greater level of alignment in outcomes accepted may enhance the efficiency of regulatory and HTA processes, and increase timely access to new medicines. ICHOM standard sets may help align these outcomes. However, some differences in outcomes used may remain due to the different purposes of regulatory and HTA decision-making. [ABSTRACT FROM AUTHOR]

Background: Advanced therapy medicinal products (ATMPs) represent an important cornerstone for innovation in healthcare. However, uncertainty on the value, the high average cost per patient and their one-shot nature has raised a debate on their assessment and appraisal process for pricing and reimbursement (P&R) purposes. This debate led experts providing for recommendations on this topic. Our primary objective is to investigate the ATMPs P&R process in the main five European countries and to understand if this process is consistent with published P&R expert recommendations. We also investigated the current ATMP pipelines to understand if future ATMPs will create challenges for their P&R process. Methods: P&R framework for ATMPs in the European Major five (EU5) countries was investigated through a literature search on PubMed, institutional websites of National Health Authorities and grey literature. The ATMPs pipeline database was populated from a clinical trial database (clinicaltrials.gov), relying on inclusion and exclusion criteria retrieved from the literature. Results: Reimbursement status of ATMPs is different across the EU5 countries, with the exception of CAR-Ts which are reimbursed in all countries. Standard P&R process in place for other medicinal products is extended to ATMPs, with the exception of some cases in Germany. List prices, where available, are high and, tend to be aligned across countries. Outcome-based Managed Entry Agreements (MEAs) have been extensively used for ATMPs. Extra-funds for hospitals managing ATMPs were provided only in Germany and, as additional fund per episode, in France. The accreditation process of hospitals for ATMPs management was in most countries managed by the national authorities. As far as ATMPs pipeline is concerned, ATMPs in development are mostly targeting non-rare diseases. Conclusions: Expert recommendations for ATMPs P&R were partially applied: the role of outcome-based MEAs has increased and the selection process of the centres authorized to use these treatments has been enhanced; additional funding for ATMPs management to accredited centres has not been completely considered and annuity payment and broader perspective in cost considerations are far from being put in place. These recommendations should be considered for future P&R negotiations to pursue rational resource allocation and deal with budget constraints. [ABSTRACT FROM AUTHOR]

La prise en charge de la prééclampsie est bien codifiée en phase aigüe, mais le devenir des patientes en post-partum l'est bien moins. Pourtant, la prééclampsie est associée à un excès de risque de morbimortalité cardiovasculaire au long cours. Un parcours de soin a été créé au Centre Hospitalier Universitaire de Nantes pour évaluer à distance de l'accouchement les facteurs de risque cardiovasculaires des patientes ayant présenté une prééclampsie. Notre étude en présente les premiers résultats. Nous avons inclus 134 patientes entre octobre 2016 et janvier 2019 qui ont présenté une prééclampsie dans la région nantaise et qui ont bénéficié de ce dépistage dans l'année suivant leur accouchement. L'hypertension artérielle était persistante chez 28 patientes (20,9 %). Trente-quatre patientes étaient obèses (25,3 %) et un diabète a été diagnostiqué chez une patiente. L'hypertension artérielle était à prédominance diastolique, légère et dans plus d'un tiers des cas masquée (35,7 % de pression artérielle normale lors de la consultation mais élevée sur les mesures à domicile). Une hypertension artérielle secondaire (cause hormonale ou rénale) a été identifiée dans un tiers des cas (32,1 %). L'hypertension survenait plus souvent chez les patientes plus âgées (OR : 2,26 ; IC 95 % : 1,25- 4,11, p = 0,072), originaire d'Afrique sub-saharienne (OR : 11,52 ; IC 95 % : 2,67- 49,86, p = 0,01) et multipares (OR : 7,82 ; IC 95 % : 1,15- 53,21, p = 0,035). Ce parcours de soins permet donc de dépister et de prendre en charge précocement les facteurs de risque cardiovasculaire de ces jeunes femmes. The medical treatment of preeclampsia is well structured in its acute phase but the required follow-up with patients in post-partum is discussed. However, preeclampsia is associated with an increased risk of cardiovascular morbi-mortality in the long term. In order to optimize the post-partum treatment, a care program has been developed for these patients in the city of Nantes, France. This includes a check-up of the cardiovascular risks at a day hospital. Our study presents the first results of this program. The study included 134 patients who were diagnosed with preeclampsia between October 2016 and January 2019 in the Nantes area, France, and took part in the program within the year following their childbirth. A descriptive analysis was first carried out and then a multivariate logistic regression model was used to investigate the risk factors for persistent high blood pressure after preeclampsia. The study detected 28 cases of persistent hypertension (20.9%), 34 cases of obesity (25.3%) and 1 case of diabetes. Hypertension was predominantly diastolic, mild and sometimes masked (35.7%). In a third of the cases (32.1%), the hypertension was secondary. High blood pressure was found to be more frequent in older patients (OR: 2.26; 95% CI: 1.25-4.11, p = 0.072), patients from sub-Saharan Africa (OR: 11.52; 95% CI: 2.67-49.86, p = 0.01) and multiparous patients (OR: 7.82; 95% CI: 1.15-53.21, p = 0.035). The study confirmed that this care program enables an earlier detection and therefore treatment of the cardiovascular risk factors of these young women. [ABSTRACT FROM AUTHOR]

The article presents the concept of the HB-HTA (Hospital-Based Health Technology Assessment) project implementation as one of the options for implementing HB-HTA in Poland. The model involves the creation of structures in selected hospitals dealing with the assessment of technologies that would be implemented in the units. The model is characterized by a small number of stakeholders and their interests do not conflict with one another. The basic condition for the success of the project is the reimbursement of the mentioned technologies, which would allow financing the described activities. The implementation of the project requires, above all, specialized staff (already present on the market), supported by appropriate technical and organizational solutions. The implementation of the project in the proposed version should bring benefits in the entire healthcare system - improved access to services, as well as improved quality of hospital management and improved financial results of the hospitals and the system itself. [ABSTRACT FROM AUTHOR]

Objective: The objective of this work was to study cardio-renal syndrome in patients hospitalized in the cardiology department of the University Hospital Center Yalgado OUEDRAOGO (CHUYO). Patients and Methods: This was a retrospective, descriptive study for a period of three years, from 1st January 2010 to 31st December 2012. The study incorporated patients with associated signs of Heart Failure (HF) and Renal Insufficiency (RI). Sociodemographic, clinical and paraclinical parameters were considered in the study. We evaluated the value of creatinine clearance at admission and its progress during the hospitalization until patients were discharged. Results: Sample group of 119 patients over the period; the prevalence of the syndrome cardio-renal (CRF) was 10.93%. The mean age of our patients was 52.6 ± 16.6 years. (extremes: 15-85 years). The sex ratio was 1.33. Our patients' medical histories were dominated by high blood pressure (58.8%) and hypertensive heart disease (33.6%). Dyspnea was the major functional sign in our study (84.9%). Left heart failure syndrome (LHFS) was the most frequently encountered clinical picture (91.3%). The mean value of the creatinine clearance at baseline was 41.5 ± 24.3 mL/min/1.73m2. The signs ECGs and Doppler echocardiograms were those of underlying heart disease: the hypertensive heart disease. An abdominal-pelvic ultrasound found a bilateral kidney failure in 65.7% cases. The average length of hospitalization was 17.7 ± 17.5 days. Death occurred in 19.5% cases. Conclusion: The prevalence of CRS was high in the CHU-YO. The prognosis was largely influenced by whether it was an acute or chronic kidney failure. Emphasis should be placed on primary prevention of CRS, early diagnosis and etiology of renal failure. [ABSTRACT FROM AUTHOR]

The use of incorrectly cleaned or disinfected hospital's equipment can also pose an infection risk and may contribute to the dissemination of microorganisms. Generally, stethoscopes are not considered a main infection risk, but the stethoscope is the most-used medical tool in the world. A new light-emitting diode (LED) disinfection option has recently been introduced by the LED industry. We wanted to determine where and when thi s new disinfection system would be useful in reducing contamination and whether any specific type of patient would benefit more from the use of this device. The evaluation was conducted using a multidisciplinary approach and has provided an analysis of the 7 domains recommended by EUnetHTA (Health problem and current use of the technology, technical characteristic, clinical effectiveness, safety, economic evaluation, organizational aspects, ethical aspects). We observe the use of sthethoscope while deliver ing routine care in three different intensity of care departments on at least one hour every week for three months. Between one patient and another, a disinfection or cleaning procedure is never performed by most operators. We collected 248 samples from different departments to evaluate the efficacy of a new device. We observed a reduction in the bacterial load in 70% of the cases after using the UV-C LED device. Discussion and Conclusion: This device could be useful in encouraging the adoption of good hy giene practices and could reduce the risks associated with the treatment of infections. The tool has proved to be particularly useful in the intensive care unit. [ABSTRACT FROM AUTHOR]

Résumé: Le dernier congrès de l'ISPOR Europe (International society for pharmacoeconomics and outcomes research) s'est tenu à Copenhague du 2 au 6 novembre 2019. Un groupe de pharmaciens, médecins et économistes en santé en synergie avec un groupe d'étudiants en master 2 de médico-économie présents à Copenhague, ont choisi de vous en restituer les messages forts. Cette synergie de groupe vise à développer « l'approche de la médico-économie sous toutes ses facettes ». Dans cet article, les auteurs évoquent les questions issues des différentes sessions et/ou posters, notamment l'évaluation des produits de santé : comment accélérer l'accès au marché des traitements tout en garantissant la pertinence clinique et la maîtrise des dépenses ? The last congress of ISPOR Europe (International society for pharmacoeconomics and outcomes research) was held in Copenhagen from 2 to 6 November 2019. A group of pharmacists, physicians and health economists in synergy with a group of students in Master 2 of medico-economics attending the conference have chosen to share some of the highlights. This group synergy aims to develop "the medico-economic approach in all its facets". In this paper, the authors discussed the following questions from the different sessions and/or posters, in particular the evaluation of health products: how to accelerate access to the treatment market while ensuring clinical relevance and control of spending? [ABSTRACT FROM AUTHOR]

The applications of Artificial Intelligence in the medical field has attracted, in recent years, both professionals and investors, the latter more than the former. However, the emphasis on both the potential of this technology and the observation of the first results, is not, to date, justified by a precise and scientific verification of the results (clinical, economic and organizational). The challenge to come for professionals who, on one hand, deal with HTA and, on the other, with the development of AI systems in the health field, will consist in defining the criteria with which health-related performance yield by these technologies have to be validated and assessed. [ABSTRACT FROM AUTHOR]

Résumé: Le pharmacien d'officine est reconnu comme un acteur de la prévention et de l'éducation thérapeutique. Un projet pédagogique mené dans les officines du Nord-Pas-de-Calais et coordonné par la faculté de pharmacie de Lille avec le réseau Néphronor nous a permis de collecter des données permettant d'évaluer l'intérêt d'une évaluation pharmaceutique officinale de la MRC. Nous rapportons ici le résultat de l'intervention réalisée dans 109 pharmacies d'officine. Les entretiens pharmaceutiques ont concerné 2 populations de patients. Le groupe A concerne des patients se connaissant porteurs d'une IRC (entretien pharmaceutique d'optimisation thérapeutique). Le groupe B regroupe des patients à risque de MRC pour lesquels est proposée une stratégie de dépistage. Ainsi, 354 patients ont été inclus dans le groupe A, majoritairement des femmes (51,2 %), en stade 3 d'IRC, d'âge moyen 73 ans, diabétiques (44 %). Environ 70 % des patients avaient déjà été vus par un néphrologue et 45 % d'entre eux présentaient une bonne adhésion thérapeutique selon le questionnaire de Morisky Green. Par ailleurs, 20 % des patients n'avaient pas de traitement néphroprotecteur en stades 3 et 4 de la MRC et plus de la moitié ne connaissaient pas les situations à risque d'insuffisance rénale aiguë. Le groupe B a inclus 532 patients (âge moyen 70 ans). L'intervention pharmaceutique a permis de dépister 10 % de patients avec un DFG < 60 mL/min/1,73 m2. L'entretien pharmaceutique en officine a permis d'optimiser la prise en charge thérapeutique et l'information des patients IRC, ainsi que le dépistage précoce chez certains patients à risque. Chronic kidney disease (CKD) is a major concern of public health. The pharmacist is known as a health practitioner involved in prevention and therapeutic education. Our study aimed at defining the impact of community pharmacists' interventions for preventing and screening CKD. In our observational prospective study of 5 months conducted in 109 community pharmacy, we included 2 groups of patients: A (therapeutic optimization): CKD patients and B (CKD screening): population at risk. In group A, we included 354 patients, mainly women (51.2%), in stage 3 of CKD, mean age 73 years old, with hypertension alone (40.6%) or associated with diabetes (44%). About 70% of the patients had a follow up by a nephrologist and 45% of them were good adherent according to the Morisky Green self-report. However, approximately 20% of patients did not have nephroprotective treatments in their regimen although they were on stage 3 or 4 CKD patients, and about half of them were not aware of medical situations at risk. Concerning group B, 532 patients were included. The pharmaceutical interventions screened 10 % of patients with a GFR < 60 mL/min/1.73m2. The community pharmacists' interventions helped to optimize the therapeutic management of CKD patients and in the early screening of patients at risk. More studies are needed to extrapolate our observations to a larger population. [ABSTRACT FROM AUTHOR]

Background: Decision-making in public health and health policy is complex and requires careful deliberation of many and sometimes conflicting normative and technical criteria. Several approaches and tools, such as multi-criteria decision analysis, health technology assessments and evidence-to-decision (EtD) frameworks, have been proposed to guide decision-makers in selecting the criteria most relevant and appropriate for a transparent decision-making process. This study forms part of the development of the WHO-INTEGRATE EtD framework, a framework rooted in global health norms and values as reflected in key documents of the World Health Organization and the United Nations system. The objective of this study was to provide a comprehensive overview of criteria used in or proposed for real-world decision-making processes, including guideline development, health technology assessment, resource allocation and others. Methods: We conducted an overview of systematic reviews through a combination of systematic literature searches and extensive reference searches. Systematic reviews reporting criteria used for real-world health decision-making by governmental or non-governmental organization on a supranational, national, or programme level were included and their quality assessed through a bespoke critical appraisal tool. The criteria reported in the reviews were extracted, de-duplicated and sorted into first-level (i.e. criteria), second-level (i.e. sub-criteria) and third-level (i.e. decision aspects) categories. First-level categories were developed a priori using a normative approach; second- and third-level categories were developed inductively. Results: We included 36 systematic reviews providing criteria, of which one met all and another eleven met at least five of the items of our critical appraisal tool. The criteria were subsumed into 8 criteria, 45 sub-criteria and 200 decision aspects. The first-level of the category system comprised the following seven substantive criteria: "Health-related balance of benefits and harms"; "Human and individual rights"; "Acceptability considerations"; "Societal considerations"; "Considerations of equity, equality and fairness"; "Cost and financial considerations"; and "Feasibility and health system considerations". In addition, we identified an eight criterion "Evidence". Conclusion: This overview of systematic reviews provides a comprehensive overview of criteria used or suggested for real-world health decision-making. It also discusses key challenges in the selection of the most appropriate criteria and in seeking to implement a fair decision-making process. [ABSTRACT FROM AUTHOR]

Objectives: Universal healthcare coverage in low- and middle-income countries requires challenging resource allocation decisions. Health technology assessment is one important tool to support such decision making. The International Decision Support Initiative worked with the Ghanaian Ministry of Health to strengthen health technology assessment capacity building, identifying hypertension as a priority topic area for a relevant case study.Methods: Based on guidance from a national technical working group of researchers and policy makers, an economic evaluation and budget impact analysis were undertaken for the main antihypertensive medicines used for uncomplicated, essential hypertension. The analysis aimed to address specific policy questions relevant to the National Health Insurance Scheme.Results: The evaluation found that first-line management of essential hypertension with diuretics has an incremental cost per disability-adjusted life-year avoided of GH¢ 276 ($179 in 2017, 4% of gross national income per capita) compared with no treatment. Calcium channel blockers were more effective than diuretics but at a higher incremental cost: GH¢ 11 061 per disability-adjusted life-year avoided ($7189 in 2017; 160% of gross national income per capita). Diuretics provide better health outcomes at a lower cost than angiotensin-converting enzyme inhibitors, angiotensin receptor blockers, or beta-blockers. Budget impact analysis highlighted the potential for cost saving through enhanced price negotiation and increased use of better-value drugs. We also illustrate how savings could be reinvested to improve population health.Conclusions: Economic evaluation enabled decision makers to assess hypertension medicines in a Ghanaian context and estimate the impact of using such evidence to change policy. This study contributes to addressing challenges associated with the drive for universal healthcare coverage in the context of constrained budgets. [ABSTRACT FROM AUTHOR]

Context: The National Institute of Excellence in Health and Social Services (INESSS), which functions as the Québec health technology assessment (HTA) agency, tested a new way to engage patients along with health‐care professionals in the co‐construction of recommendations regarding implantable cardioverter‐defibrillator replacement. Objective: The objective of this article was to describe the process of co‐construction of recommendations and to propose methods of building best practices for patient involvement (PI) in HTA. Design: Throughout the process, documents were collected and participant observations were made. Individual interviews were conducted with patients, health‐care professionals and the INESSS scientific team, from January to March 2018. Results: Three committees were established: an expert patient committee to reflect on patient experience literature; an expert health professional committee to reflect on medical literature; and a co‐construction committee through which both patients and health‐care professionals contributed to develop the recommendations. The expert patients validated and contextualized a literature review produced by the scientific team. This allowed the scientists to consider aspects related to the patient experience and to integrate the feedback from patients into HTA recommendations. The most important factor contributing to a positive PI experience was the structured methodology for selecting patient participants, and a key factor that inhibited the process was a lack of training in PI on the part of the scientific team. Conclusions: This experience demonstrates that it is possible to co‐construct recommendations, even for technically complex HTA subjects, through a more democratic process than usual which led to more patient‐focused guidance. [ABSTRACT FROM AUTHOR]

India is a diverse land with different cultures, social norms, castes, religions, faiths, languages, politics, and a complex healthcare system. As a step to enhance healthcare, the government of India announced a move toward universal health coverage to increase accessibility and affordability of health-related services. Recently, there has been an introduction of health technology assessment (HTA) in India to help inform evidence-based decision making in cases of limited resources and budgets. Nevertheless, there are challenges related to biased decision making, an unregulated healthcare framework, and the lack of data and capacity that will (directly or indirectly) affect the use of HTA in India. For HTA to be successful in India and in similar low- and middle-income countries, it is important that the decision makers acknowledge these challenges and embrace differences in ideologies, cultures, and politics instead of ignoring them. Drawing lessons from countries with well-developed HTA bodies may help, but these need to be modified for the country-specific context. Ensuring quality and transparency is key to building trust in medical decision making. Improved coordination at all levels of healthcare is vital to ensure the long-term success of HTA in India. This is challenging but achievable by spreading awareness among stakeholders and achieving moderate health-sector regulation that can combat corruption. HTA will prosper in India if it incorporates cultural and institutional diversity, alongside tackling socioeconomic inequalities. [ABSTRACT FROM AUTHOR]

Preparation of HTA reports requires access to the latest and most reliable knowledge in the field of health care. Systematic reviews are the basic tools used to obtain it. In order to find the literature necessary for a systematic review, search strategies are performed in databases. With their help it is possible to thoroughly and reliably search the resources of individual databases and find the required information. Properly executed search strategies also reduce the risk of missing valuable publications. During the preparation of the search strategy, one should use the tools and additional functions implemented in individual databases, thanks to which it is possible to strictly control the process and adapt it to your own needs. Performing a search strategy is a basic skill for anyone who prepares HTA reports. [ABSTRACT FROM AUTHOR]

The Agency for Health Technology Assessment and Tariff System is national institution supervised by Minister of Health and funded by government. Agency is an advisory body in the reimbursement process. Agency has an impact on content of benefit basket, not only by evaluating new technologies but also re-evaluating benefit basket. Main goal of Agency is to provide patients access to the most effective and safe technologies. The most important Agency statements concern medical technologies and its modifications, qualification, removal in reference to guaranteed medical services. The Minister of Health, taking decisions about which health technology should be financed by public found, takes into account: recommendations and opinions of President of Agency, opinions and statements of the Transparency Council and the Council for Tariffs Affairs. The Agency's task is also: setting tariffs for health care services; evaluating diagnostic and therapeutic guidelines prepared by medical societies; preparing opinions about national and local government health care programs. [ABSTRACT FROM AUTHOR]

Purpose: Centrally authorised medicinal products (CAMPs) in the European Union may offer added therapeutic value (ATV) but may be linked to high prices and limited efficiency. Health technology assessment (HTA) and managed entry schemes (MES) may facilitate the reimbursement decision by providing reliable estimates of the medicinal product's value and costs and by controlling the remaining uncertainty, respectively. We investigated the impact of HTA criteria and the initiation of a MES on the reimbursement decision of CAMPs in Belgium. Methods: We selected all reimbursement submissions for new centrally authorised medicinal products in the 2010–2015 period. We retrieved data relating to the reimbursement decision, the HTA outcome and the use of a managed entry scheme. Results: The decision of the Minister was available for 115 dossiers, covering 36 (31.3%) orphan medicinal products (OMPs) and 79 ATV products. A MES was used in 41 submissions. A positive reimbursement decision was obtained in 65% of cases. The significant factors affecting the reimbursement decision were the approval of ATV, the medical need if it was considered 'important or major' and the use of a managed entry scheme. Price, budget impact and efficiency had no significant impact. Conclusions: Added therapeutic value and high medical need increase the odds for a positive reimbursement decision. No impact could be demonstrated of the cost-related HTA criteria. Cost elements may be biased by the use of a confidential MES. Without a MES, only 53% of the centrally authorised medicinal products, including OMPs, are reimbursed in Belgium. [ABSTRACT FROM AUTHOR]

Background: Proportional hazards (PH) is an assumption often made by researchers, despite evidence of nonproportionality in a significant proportion of clinical trials. In the presence of non-PH, the interpretation of hazard ratios, medians, and landmark survival as summary measures of treatment effect can become problematic. Several recent studies have recommended restricted mean survival time (RMST) as an alternative metric for survival analysis, particularly where non-PH may apply.Objectives: To determine the current approaches of health technology assessment (HTA) agencies to value assessment in the presence of non-PH, and the extent to which RMST is accepted as an alternative measure of treatment benefit.Methods: Methodological guidelines published by 10 HTA agencies were reviewed to establish recommended approaches for presenting survival benefit from clinical trials. Published HTA reports for 23 oncology agents approved by the US Food and Drug Administration and the European Medicines Agency since 2014 were reviewed to determine how guidelines are implemented in practice and identify instances where the PH assumption was tested and RMST analyses reported.Results: Testing for non-PH is not widely incorporated into HTA except by the UK National Institute for Health and Care Excellence. RMST is used infrequently but has been used in a number of countries, particularly by agencies that focus on cost effectiveness.Conclusions: HTA agencies vary in their approaches to non-PH. Most do not routinely check the PH assumption. RMST has played a role in assessing clinical benefit within HTA, although not consistently within countries (across drugs) or across countries (for the same drug). [ABSTRACT FROM AUTHOR]

Hypertension is responsible for 8.5 million deaths from stroke, ischemic heart disease and other vascular and kidney diseases. In Algeria, hypertension (HTN) is the most common chronic disease with an estimated prevalence of 23,6%. The PACT II study is a national, observational, descriptive, cross-sectional, multicenter and non-interventional study of therapeutic strategy, conducted in Algeria on hypertensive patients treated for at least 3 months, followed by 100 specialists or general practitioners. The primary objective is to describe the hypertension management in Algeria through the distribution of patients treated for HTN through predefined levels of arterial pressure (AP) reached under treatment. 2000 eligible patients were recruited in the study. 63.5% and 36.4% of patients were female and male respectively. The average age was 62.4 years ± 10.7. The most affected age group (65.2%) was between 55 to 75 years old. Most of the patients had an elevated blood pressure (BP) with 49.30 % at level III AP (130 – 139 mmHg of SBP or 80 – 89 mmHg of DBP), with 49.6% for diabetic patients and 48.8% for non-diabetic patients, then followed by 20.35% at the level IV AP (140 – 159 mmHg of SBP or 90 – 99 mmHg of DBP) with 21.4% for diabetic and 18.3% for non-diabetic patients. Also, 47.32% were at AP level III in patients with Renal failure. Regarding the achievement of the BP target, it was 17.4% for the overall population with 18,3% for diabetic's patients and only 12.64% for Renal failure patients. Additionally, the risk of cardiovascular disease (CVD) was at very high level for most patients, corresponding to 38,4 % of the overall study population. At last, PACT II study data were collected on analysis of blood pressure levels in the overall population and in cardiovascular risk (CVR) population groups such as diabetes (65%), sedentary lifestyle (53%), dyslipidemia (50%), obesity (44%), and renal failure (11.3%). PACT II has made it possible to update national data relating to hypertension in Algeria. It confirmed the low level of achievement of the BP target, even if the average arterial pressure level obtained was acceptable in 76% patients with a BP level which was lower than 140 mmHg for SBP and less than 90 mmHg for DBP. Many efforts must be made, according to WHO recommendations, to improve the management of hypertensive patients and BP control in Algeria. [ABSTRACT FROM AUTHOR]