Effects of working from home on lifestyle behaviors and mental health during the COVID-19 pandemic: A survey study.

The COVID-19 outbreak has led to relevant changes in everyday life worldwide. One of these changes has been a rapid transition to and an increasing implementation of working from home (WH) modality. This study aimed to evaluate the impact of mandatory WH during the COVID-19 pandemic on lifestyle behaviors, Mediterranean diet adherence, body weight, and depression. An online cross-sectional survey was conducted in the early 2022 at the National Research Council of Italy using ad hoc questions and validated scales collecting information on physical activity, sedentary behavior, hobbies/pastimes, dietary habits including adherence to the Mediterranean diet, body weight, and depression during WH compared with before WH. 748 respondents were included in the study. An increased sedentary lifetime was reported by 48% of respondents; however, the subsample of workers who previously performed moderate physical activity intensified this activity. Body weight gain during WH was self-reported in 39.9% of respondents. Mediterranean diet adherence increased (p≪0.001) during WH compared with before WH. The average level of mental health did not record an overall variation; however, the proportion of subjects with mild and moderate depression increased (p = 0.006), while workers who reported values indicative of depression before the transition declared an improvement. These findings highlight health-related impact of WH during the COVID-19 pandemic that may inform future strategies and policies to improve employees' health and well-being.

Association Between Physical Activity and the Risk of Burnout in Health Care Workers: Systematic Review.

BACKGROUND: Burnout is a multidimensional psychological syndrome that arises from chronic workplace stress. Health care workers (HCWs), who operate in physically and emotionally exhausting work contexts, constitute a vulnerable group. This, coupled with its subsequent impact on patients and public economic resources, makes burnout a significant public health concern. Various self-care practices have been suggested to have a positive effect on burnout among HCWs. Of these, physical activity stands out for its ability to combine psychological, physiological, and biochemical mechanisms. In fact, it promotes psychological detachment from work and increases self-efficacy by inhibiting neurotransmitters and neuromodulators, increasing endorphin levels, enhancing mitochondrial function, and attenuating the hypothalamic pituitary-adrenal axis response to stress. OBJECTIVE: Our objective was to conduct a systematic review of the evidence on the association between physical activity and burnout among HCWs. METHODS: We considered HCWs, physical activity, and burnout, framing them as population, exposure, and outcome, respectively. We searched APA PsycArticles, MEDLINE, and Scopus until July 2022. We extracted relevant data on study design, methods to measure exposure and outcome, and statistical approaches. RESULTS: Our analysis encompassed 21 independent studies. Although 10% (2/21) of the studies explicitly focused on physical activity, the remaining investigations were exploratory in nature and examined various predictors, including physical activity. The most commonly used questionnaire was the Maslach Burnout Inventory. Owing to the heterogeneity in definitions and cutoffs used, the reported prevalence of burnout varied widely, ranging from 7% to 83%. Heterogeneity was also observed in the measurement tools used to assess physical activity, with objective measures rarely used. In total, 14% (3/21) of the studies used structured questionnaires to assess different types of exercise, whereas most studies (18/21, 86%) only recorded the attainment of a benchmark or reported the frequency, intensity, or duration of exercise. The reported prevalence of physically active HCWs ranged from 44% to 87%. The analyses, through a variety of inferential approaches, indicated that physical activity is often associated with a reduced risk of burnout, particularly in the domains of emotional exhaustion and depersonalization. Furthermore, we compiled and classified a list of factors associated with burnout. CONCLUSIONS: Our comprehensive overview of studies investigating the association between physical activity and burnout in HCWs revealed significant heterogeneity in definitions, measurements, and analyses adopted in the literature. To address this issue, it is crucial to adopt a clear definition of physical activity and make thoughtful choices regarding measurement tools and methodologies for data analysis. Our considerations regarding the measurement of burnout and the comprehensive list of associated factors have the potential to improve future studies aimed at informing decision-makers, thus laying the foundation for more effective management measures to address burnout.

Corrigendum: The medium-term perceived impact of work from home on life and work domains of knowledge workers during COVID-19 pandemic: a survey at the National Research Council of Italy.

[This corrects the article DOI: 10.3389/fpubh.2023.1151009.].

miRNAs as biomarkers of musculoskeletal pain in long-distance runners: the MiMuS study protocol.

miRNAs are involved in the generation and progression of musculoskeletal pain, a condition that causes significant clinical, economic and social burden. In runners, the presence of musculoskeletal pain related to an inflammatory state or ongoing underlying tissue damage may result in poor training ability and performance. This study aims to evaluate the association between circulating and salivary miRNAs and pain in runners with and without musculoskeletal pain and to observe whether dysregulated miRNAs can distinguish between responders and nonresponders to a kinesiological intervention. The possible correlation between these miRNAs and inflammatory molecules, stress parameters and individual or behavioral characteristics will be evaluated. Finally, in silico analysis will be used to characterize miRNAs function. Ethics approval was obtained.

The medium-term perceived impact of work from home on life and work domains of knowledge workers during COVID-19 pandemic: A survey at the National Research Council of Italy.

Objective: The study aimed to investigate perceptions and determinants of the overall impact on life and work domains among a community of knowledge workers after 18 months of forced work from home due to the pandemic. Methods: A cross-sectional study with a retrospective assessment was conducted early in 2022 at the National Research Council of Italy. Five single-item questions explored the perceived impact on life domain while a 7-item scale the impact on the work domain. Bivariate analyses and multivariate regressions were used to evaluate the associations between impacts and some key factors defined by 29 ad hoc closed questions. Results: More than 95% of the 748 respondents reported a perceived change in at least one item of the life domain. For each of these items, although a large group of subjects has reported that working from home had no impact (from 27 to 55%), in the rest of the sample the positive evaluation (from 30 to 60%) clearly prevailed over the negative one. Overall, most of the subjects (64%) rated the impact on the work experience positively. Relationship with colleagues and participation in the work context were the items where the greatest number of negative rates was concentrated (27 and 25%, respectively). On the other hand, positive perceptions prevailed over both negative perceptions and lack of impact perceptions on the subjects of organizational flexibility and quality of work. The frequency of work-room sharing, home-work commute time and changes in sedentary lifestyle, have been identified as common explanatory factors of perceived impacts on both domains. Conclusion: Overall, respondents reported positive rather than negative perceived impacts of forced work from home in both their lives and work. The obtained results suggest that policies to promote the physical and mental health of employees, strengthen inclusion and maintain a sense of community are necessary to improve workers' health and prevent the effects of perceived isolation on research activities.

Economic Evaluations of Magnetic Resonance Image-Guided Radiotherapy (MRIgRT): A Systematic Review.

OBJECTIVES: This review systematically summarizes the evidence on the economic impact of magnetic resonance image-guided RT (MRIgRT). METHODS: We systematically searched INAHTA, MEDLINE, and Scopus up to March 2022 to retrieve health economic studies. Relevant data were extracted on study type, model inputs, modeling methods and economic results. RESULTS: Five studies were included. Two studies performed a full economic assessment to compare the cost-effectiveness of MRIgRT with other forms of image-guided radiation therapy. One study performed a cost minimization analysis and two studies performed an activity-based costing, all comparing MRIgRT with X-ray computed tomography image-guided radiation therapy (CTIgRT). Prostate cancer was the target condition in four studies and hepatocellular carcinoma in one. Considering the studies with a full economic assessment, MR-guided stereotactic body radiation therapy was found to be cost effective with respect to CTIgRT or conventional or moderate hypofractionated RT, even with a low reduction in toxicity. Conversely, a greater reduction in toxicity is required to compete with extreme hypofractionated RT without MR guidance. CONCLUSIONS: This review highlights the great potential of MRIgRT but also the need for further evidence, especially for late toxicity, whose reduction is expected to be the real added value of this technology.

Expression and Biological Functions of miRNAs in Chronic Pain: A Review on Human Studies.

Chronic pain is a major public health problem and an economic burden worldwide. However, its underlying pathological mechanisms remain unclear. MicroRNAs (miRNAs) are a class of small noncoding RNAs that post-transcriptionally regulate gene expression and serve key roles in physiological and pathological processes. This review aims to synthesize the human studies examining miRNA expression in the pathogenesis of chronic primary pain and chronic secondary pain. Additionally, to understand the potential pathophysiological impact of miRNAs in these conditions, an in silico analysis was performed to reveal the target genes and pathways involved in primary and secondary pain and their differential regulation in the different types of chronic pain. The findings, methodological issues and challenges of miRNA research in the pathophysiology of chronic pain are discussed. The available evidence suggests the potential role of miRNA in disease pathogenesis and possibly the pain process, eventually enabling this role to be exploited for pain monitoring and management.

Health Technology Assessment for In Silico Medicine: Social, Ethical and Legal Aspects.

The application of in silico medicine is constantly growing in the prevention, diagnosis, and treatment of diseases. These technologies allow us to support medical decisions and self-management and reduce, refine, and partially replace real studies of medical technologies. In silico medicine may challenge some key principles: transparency and fairness of data usage; data privacy and protection across platforms and systems; data availability and quality; data integration and interoperability; intellectual property; data sharing; equal accessibility for persons and populations. Several social, ethical, and legal issues may consequently arise from its adoption. In this work, we provide an overview of these issues along with some practical suggestions for their assessment from a health technology assessment perspective. We performed a narrative review with a search on MEDLINE/Pubmed, ISI Web of Knowledge, Scopus, and Google Scholar. The following key aspects emerge as general reflections with an impact on the operational level: cultural resistance, level of expertise of users, degree of patient involvement, infrastructural requirements, risks for health, respect of several patients' rights, potential discriminations for access and use of the technology, and intellectual property of innovations. Our analysis shows that several challenges still need to be debated to allow in silico medicine to express all its potential in healthcare processes.

The expression of microRNAs and exposure to environmental contaminants related to human health: a review.

Environmental contaminants exposure may lead to detrimental changes to the microRNAs (miRNAs) expression resulting in several health effects. miRNAs, small non-coding RNAs that regulate gene expression, have multiple transcript targets and thereby regulate several signalling molecules. Even a minor alteration in the abundance of one miRNA can have deep effects on global gene expression. Altered patterns of miRNAs can be responsible for changes linked to various health outcomes, suggesting that specific miRNAs are activated in pathophysiological processes. In this review, we provide an overview of studies investigating the impact of air pollution, organic chemicals, and heavy metals on miRNA expression and the potential biologic effects on humans.Abbreviations: AHRR, aryl-hydrocarbon receptor repressor; AHR, aryl-hydrocarbon receptor; As, arsenic; BCL2, B-cell lymphoma 2; BCL2L11, B-cell lymphoma 2 like 11; BCL6, B-cell lymphoma 6; BPA, bisphenol A; CVD, cardiovascular diseases; CD40, cluster of differentiation 40; CCND1, Cyclin D1; CDKN1A, cyclin-dependent kinase inhibitor 1A; Cr, chromium; CTBP1, C-terminal binding protein 1; CXCL12, C-X-C motif chemokine ligand 12; DAZAP1, deleted in azoospermia associated protein 1; DEP, diesel exhaust particles; EGFR, epidermal growth factor receptor; eNOS, endothelial nitric oxide synthase; EVs, extracellular vesicles; FAK, focal adhesion kinase; FAS, fas cell surface death receptor; FOXO, forkhead box O; HbA1c, glycated hemoglobin; Hg, mercury; HLA-A, human leukocyte antigen A; HMGB, high-mobility group protein B; IFNAR2, interferon alpha receptor subunit 2; IL-6, interleukin-6; IRAK1, interleukin 1 receptor associated kinase 1; JAK/STAT, janus kinase/signal transducers and activators of transcription; MAPK, mitogen-activated protein kinase; miRNAs, microRNAs; MVs, microvesicles; NCDs, noncommunicable diseases; NFAT, nuclear factor of activated T cells; NFkB, nuclear factor kappa B; NRF2, nuclear factor, erythroid-derived 2; NRG3, neuregulin 3; O3, ozone; OP, organophosphorus pesticides; PAHs, polycyclic aromatic hydrocarbons; Pb, lead; PCBs, polychlorinated biphenyls; PDCD4, programmed cell death 4; PDGFB, platelet derived growth factor subunit beta; PDGFR, platelet-derived growth factor receptor; PI3K/Akt, phosphoinositide-3-kinase/protein kinase B; PKA, protein kinase A; PM, particulate matter; PRKCQ, protein kinase C theta; PTEN, phosphatase and tensin homolog; SORT1, sortilin 1; TGFß, transforming growth factor-ß; TLR, toll-like receptor; TNF, tumor necrosis factors; TRAF1, tumor necrosis factors-receptor associated factors 1; TRAP, traffic-related air pollution; TREM1, triggering receptor expressed on myeloid cells 1; TRIAP1, TP53 regulated inhibitor of apoptosis 1; VCAM-1, vascular cell adhesion molecule 1; VEGFA, vascular endothelial growth factor A; XRCC2, X-ray repair cross complementing 2; YBX2, Y-box-binding protein 2; ZEB1, zinc finger E-box-binding homeobox 1; ZEB2, zinc finger E-box-binding homeobox 2; 8-OH-dG, 8-hydroxy-guanine.

Burnout Among Healthcare Workers in the COVID 19 Era: A Review of the Existing Literature.

In the current period of global public health crisis due to the COVID-19, healthcare workers are more exposed to physical and mental exhaustion - burnout - for the torment of difficult decisions, the pain of losing patients and colleagues, and the risk of infection, for themselves and their families. The very high number of cases and deaths, and the probable future "waves" raise awareness of these challenging working conditions and the need to address burnout by identifying possible solutions. Measures have been suggested to prevent or reduce burnout at individual level (physical activity, balanced diet, good sleep hygiene, family support, meaningful relationships, reflective practices and small group discussions), organizational level (blame-free environments for sharing experiences and advices, broad involvement in management decisions, multi-disciplinary psychosocial support teams, safe areas to withdraw quickly from stressful situations, adequate time planning, social support), and cultural level (involvement of healthcare workers in the development, implementation, testing, and evaluation of measures against burnout). Although some progress has been made in removing the barrier to psychological support to cope with work-related stress, a cultural change is still needed for the stigma associated with mental illness. The key recommendation is to address the challenges that the emergency poses and to aggregate health, well-being and behavioral science expertise through long term researches with rigorous planning and reporting to drive the necessary cultural change and the improvement of public health systems.

MiRNA expression profiling in HIV pathogenesis, disease progression and response to treatment: a systematic review.

Aim: A systematic review was conducted to identify the association of miRNA expression with HIV pathogenesis, progression and treatment. Methods: A search of articles was conducted in MEDLINE®, Cochrane Central Register of Controlled Trials and Global Health. Results: 35 articles were included. Due to the heterogeneity of HIV phenotypes, a harmonization based on key progression parameters was proposed. The hsa-miR-29 family, hsa-miR-146b-5p and hsa-miR-150-5p, are the most frequently differentially expressed in HIV. Direct comparison of studies was not possible due to heterogeneity in biological samples and miRNA analysis techniques. Conclusion: This is the first attempt to systematically identify miRNA's different expression in well-defined patient phenotypes and could represent a helpful way to increase general knowledge in this field.

Lay abstract miRNAs play important role in the regulation of gene expression and are involved in various physiological processes. Dysregulation of their function can lead to human diseases including cancer, cardiovascular and metabolic diseases, liver conditions and immune dysfunction. The aim of this work is to systematically analyze the current scientific literature to identify miRNAs linked to the mechanism, development and treatment of HIV. A total of 35 articles were included and the miRNAs that were found with significantly different levels in compared groups of subjects (e.g., subjects with HIV vs healthy persons, persons able to limit the disease progression without therapy vs those whose immune system is already compromised by HIV) were highlighted. The most frequently reported miRNAs were: the hsa-miR-29 family, hsa-miR-146b-5p and hsa-miR-150-5p. To our knowledge, this is the first attempt to systematically identify the miRNAs associated with HIV and could be a useful contribution to general knowledge in this field.

Healthcare and Economic Impact of Lung Perfusion Scintigraphy in Patients Affected by Acute Pulmonary Embolism.

Acute pulmonary embolism (APE) is a cardiovascular emergency, representing the main cause of mortality, morbidity, and hospitalisation in Europe. We aim to evaluate the economic and healthcare impact of lung perfusion scintigraphy (LPS) used in patients with suspected APE, in the event of non-conclusive or contraindicated computed tomography pulmonary angiography (CTPA). We considered two alternative healthcare processes for APE diagnosis, with and without LPS. We performed a cost analysis with the aim of evaluating the average direct healthcare costs for diagnosis, risk assessment, and treatment of APE. We used data from a monocentric trial. Our economic model showed that the strategy with LPS was preferable in terms of costs. The average per-patient costs for the diagnosis and treatment of the acute phase of PE in low-risk patients with a non-conclusive or not-executable CTPA, with and without LPS, are EUR 2145.25 and EUR 4912.45, respectively. LPS is a simple, quick, and economic examination, useful in this setting of patients not only for an early diagnosis but also to exclude APE, demonstrating an advantage in terms of healthcare resources. To the best of our knowledge, this study is the first to analyse the economic and healthcare impact of the use of LPS in the diagnostic pathway of suspected APE.

Discrimination capability of pretest probability of stable coronary artery disease: a systematic review and meta-analysis suggesting how to improve validation procedures.

OBJECTIVE: Externally validated pretest probability models for risk stratification of subjects with chest pain and suspected stable coronary artery disease (CAD), determined through invasive coronary angiography or coronary CT angiography, are analysed to characterise the best validation procedures in terms of discriminatory ability, predictive variables and method completeness. DESIGN: Systematic review and meta-analysis. DATA SOURCES: Global Health (Ovid), Healthstar (Ovid) and MEDLINE (Ovid) searched on 22 April 2020. ELIGIBILITY CRITERIA: We included studies validating pretest models for the first-line assessment of patients with chest pain and suspected stable CAD. Reasons for exclusion: acute coronary syndrome, unstable chest pain, a history of myocardial infarction or previous revascularisation; models referring to diagnostic procedures different from the usual practices of the first-line assessment; univariable models; lack of quantitative discrimination capability. METHODS: Eligibility screening and review were performed independently by all the authors. Disagreements were resolved by consensus among all the authors. The quality assessment of studies conforms to the Quality Assessment of Diagnostic Accuracy Studies (QUADAS-2). A random effects meta-analysis of area under the receiver operating characteristic curve (AUC) values for each validated model was performed. RESULTS: 27 studies were included for a total of 15 models. Besides age, sex and symptom typicality, other risk factors are smoking, hypertension, diabetes mellitus and dyslipidaemia. Only one model considers genetic profile. AUC values range from 0.51 to 0.81. Significant heterogeneity (p<0.003) was found in all but two cases (p>0.12). Values of I2 >90% for most analyses and not significant meta-regression results undermined relevant interpretations. A detailed discussion of individual results was then carried out. CONCLUSIONS: We recommend a clearer statement of endpoints, their consistent measurement both in the derivation and validation phases, more comprehensive validation analyses and the enhancement of threshold validations to assess the effects of pretest models on clinical management. PROSPERO REGISTRATION NUMBER: CRD42019139388.

Simulated versus physical bench tests: The economic evaluation of the InSilc platform for designing, developing, and assessing vascular scaffolds.

BACKGROUND: In silico medicine allows for pre-clinical and clinical simulated assessment of medical technologies and the building of patient-specific models to support medical decisions and forecast personal health status. While there is increasing trust in the potential central role of in silico medicine, there is a need to recognize its degree of reliability and evaluate its economic impact. An in silico platform has been developed within a Horizon 2020-funded project (In-Silc) for simulations functional to designing, developing, and assessing drug-eluting bioresorbable vascular scaffolds.The main purpose of this study was to compare the costs of 2 alternative strategies: the adoption of In-Silc platform versus the performance of only physical bench tests. METHODS: A case study was provided by a medical device company. The values of the model parameters were principally set by the project partners, with use of interviews and semi-structured questionnaires, and, when not available, through literature searches or derived by statistical techniques. An economic model was built to represent the 2 scenarios. RESULTS: The InSilc strategy is superior to the adoption of physical bench tests only. Ceteris paribus, the costs are 424,355€ for the former versus 857,811€ for the latter. CONCLUSIONS: In silico medicine tools can decrease the cost of the research and development of medical devices such as bioresorbable vascular scaffolds. Further studies are needed to explore the impact of such solutions on the innovation capacity of companies and the consequent potential advantages for target patients and the healthcare system.

The effect of a postural exercise program on muscle power in Italian high school students.

[Purpose] The present study aimed to investigate the effect of an innovative postural program (the Canali Postural Method, CPM) on muscle power in Italian high school students. It is note that deficits in posture control may, in long term, generate posture weakness as early as childhood and adolescence. Postural programs based on stretching and strengthening exercises can remove these deficits and can be framed in general physical or sport activities. [Participants and Methods] Thirty-four students completed a 8-week postural program. The intervention, consisting of stretching and muscle activation exercises, was integrated in physical education lessons. For the evaluation of the effect of CPM program, we have used the countermovement jump (CMJ), a simple and versatile test that measures muscle power. [Results] The CPM program resulted in significantly increased vertical jump height of the students. The average difference between Initial and Final CMJ was 2.1 cm. [Conclusion] This finding indicates the benefic effect of this new postural program on physical performance in the youth. Further randomized control trials should be conducted to evaluate CPM long-term implications in the prevention of posture weaknesses and its inclusion in the regular school curriculum.

Obstructive sleep apnoea: Improving healthcare services by combining process modelling and population analysis.

CONTEXT: Disease management broke through in the early 1990s to counterbalance hyper-specialization with a more comprehensive approach. Its role became immediately relevant in chronic conditions and, consequently, in Obstructive Sleep Apnoea (OSA). This is a common chronic condition for which is important to organise services at the local level, taking into account organisational factors and the characteristics of the assisted population. OBJECTIVES: The aim of this work is to propose and apply, coherently with a disease management approach, a combination of healthcare process modelling and population analysis as a way to identify critical issues and explore shared solutions. METHODS: A multidisciplinary working group was created with scholars who are skilled in process analysis, statistics and medicine. Through semi-structured interviews and on-site meetings, healthcare processes were represented with a standard graphical language: Unified Modeling Language™. Population analysis was based on statistical analysis performed on a 5-year retrospective cohort assisted by a Community Pulmonary Service. RESULTS: A shared graphic presentation of the current healthcare process and the results of the statistical analyses constituted the knowledge base to identify critical issues and recommend corresponding solutions, which include: a) refine the local patient database with additional details on comorbidities and risk factors; b) support a greater involvement of "gate-keepers" in the screening phase; c) provide practical tools for the definition of strategies to increment the adherence to therapy; d) include recommendations for physical exercise and interdisciplinary cooperation; and e) define process indicators for measuring the quality of the screening and therapeutic phases. CONCLUSION: The concomitant analyses of formalised processes and critical risk factors represent a useful approach for systematically identifying areas of improvement in healthcare processes and allow us to discuss solutions. Moreover, the specific adoption of UML® for graphical modelling and representation of patient care processes allows us to formalise them by adopting a standard language that can be taken as the basis for implementing web services to support the execution of the modelled processes.

Standardized languages and notations for graphical modelling of patient care processes: a systematic review.

PURPOSE: The importance of working toward quality improvement in healthcare implies an increasing interest in analysing, understanding and optimizing process logic and sequences of activities embedded in healthcare processes. Their graphical representation promotes faster learning, higher retention and better compliance. The study identifies standardized graphical languages and notations applied to patient care processes and investigates their usefulness in the healthcare setting. DATA SOURCES: Peer-reviewed literature up to 19 May 2016. Information complemented by a questionnaire sent to the authors of selected studies. STUDY SELECTION: Systematic review conducted in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses statement. DATA EXTRACTION: Five authors extracted results of selected studies. RESULTS OF DATA SYNTHESIS: Ten articles met the inclusion criteria. One notation and language for healthcare process modelling were identified with an application to patient care processes: Business Process Model and Notation and Unified Modeling Language™. One of the authors of every selected study completed the questionnaire. Users' comprehensibility and facilitation of inter-professional analysis of processes have been recognized, in the filled in questionnaires, as major strengths for process modelling in healthcare. CONCLUSION: Both the notation and the language could increase the clarity of presentation thanks to their visual properties, the capacity of easily managing macro and micro scenarios, the possibility of clearly and precisely representing the process logic. Both could increase guidelines/pathways applicability by representing complex scenarios through charts and algorithms hence contributing to reduce unjustified practice variations which negatively impact on quality of care and patient safety.

Reimbursed Price of Orphan Drugs: Current Strategies and Potential Improvements.

The pricing and reimbursement policies for pharmaceuticals are relevant to balance timely and equitable access for all patients, financial sustainability, and reward for valuable innovation. The proliferation of high-cost specialty medicines is particularly true in rare diseases (RDs) where the pricing mechanism is characterised by a lack of transparency. This work provides an overall picture of current strategies for the definition of the reimbursed prices of orphan drugs (ODs) and highlights some potential improvements. Current strategies and suggestions are presented along 4 dimensions: (1) comprehensive value assessment, (2) early dialogs among relevant stakeholders, (3) innovative reimbursement approaches, and (4) societal participation in producing ODs. Comprehensive value assessment could be achieved by clarifying the approach of distributive justice to adopt, ensuring a representative participation of stakeholders, and with a broad consideration of value-bearing factors. With respect to early dialogs, cross-border cooperation can be determinant to companies and agencies. The cost-benefit ratio of early dialogs needs to be demonstrated and the "regulatory capture" effect should be monitored. Innovative reimbursement approaches were developed to balance the need for evidence-based decisions with the timely access to innovative drugs. The societal participation in producing ODs needs to be recognised in a collaborating framework where adaptive agreements can be developed with mutual satisfaction. Such agreements could also impact on coverage and reimbursement decisions as additional elements for the determination of a comprehensive societal value of ODs. Further research is needed to investigate the highlighted open challenges so that RDs will not remain, in practical terms, orphan diseases.

A conceptual framework for rationalized and standardized Universal Newborn Hearing Screening (UNHS) programs.

Congenital hearing loss is the most frequent birth defect. The American Academy of Pediatrics and the Joint Committee on Infant Hearing established quality of care process indicators for Universal Newborn Hearing Screening starting from 1999. In a previous systematic review of Universal Newborn Hearing Screening studies we highlighted substantial variability in program design and in reported performance data. In order to overcome these heterogeneous findings we think it is necessary to optimize the implementation of Universal Newborn Hearing Screening programs with an appropriate application of the planning, executing, and monitoring, verifications and reporting phases. For this reason we propose a conceptual framework that logically integrates these three phases and, consequently, a tool (a check-list) for their rationalization and standardization.Our paper intends to stimulate debate on how to ameliorate the routine application of high quality Universal Newborn Hearing Screening programs. The conceptual framework is proposed to optimize, rationalise and standardise their implementation. The checklist is intended to allow an inter-program comparison by removing heterogeneity in processes description and assessment.

Evaluating reporting and process quality of publications on UNHS: a systematic review of programmes.

BACKGROUND: Congenital hearing loss is one of the most frequent birth defects, and Early Detection and Intervention has been found to improve language outcomes. The American Academy of Pediatrics (AAP) and the Joint Committee on Infant Hearing (JCIH) established quality of care process indicators and benchmarks for Universal Newborn Hearing Screening (UNHS). We have aggregated some of these indicators/benchmarks according to the three pillars of universality, timely detection and overreferral. When dealing with inter-comparison, relying on complete and standardised literature data becomes crucial. The purpose of this paper is to verify whether literature data on UNHS programmes have included sufficient information to allow inter-programme comparisons according to the indicators considered. METHODS: We performed a systematic search identifying UNHS studies and assessing the quality of programmes. RESULTS: The identified 12 studies demonstrated heterogeneity in criteria for referring to further examinations during the screening phase and in identifying high-risk neonates, protocols, tests, staff, and testing environments. Our systematic review also highlighted substantial variability in reported performance data. In order to optimise the reporting of screening protocols and process performance, we propose a checklist. Another result is the difficulty in guaranteeing full respect for the criteria of universality, timely detection and overreferral. CONCLUSIONS: Standardisation in reporting UNHS experiences may also have a positive impact on inter-program comparisons, hence favouring the emergence of recognised best practices.