Big Data in Head and Neck Cancer.

OPINION STATEMENT: Head and neck cancers can be used as a paradigm for exploring "big data" applications in oncology. Computational strategies derived from big data science hold the promise of shedding new light on the molecular mechanisms driving head and neck cancer pathogenesis, identifying new prognostic and predictive factors, and discovering potential therapeutics against this highly complex disease. Big data strategies integrate robust data input, from radiomics, genomics, and clinical-epidemiological data to deeply describe head and neck cancer characteristics. Thus, big data may advance research generating new knowledge and improve head and neck cancer prognosis supporting clinical decision-making and development of treatment recommendations.

The cost-effectiveness of the Argus II retinal prosthesis in Retinitis Pigmentosa patients.

BACKGROUND: Retinitis Pigmentosa (RP) is a hereditary genetic disease causing bilateral retinal degeneration. RP is a leading cause of blindness resulting in incurable visual impairment and drastic reduction in the Quality of life of the patients. Second Sight Medical Products Inc. developed Argus II, a retinal prosthesis system for treating RP. Argus II is the world's first ever-commercial implant intended to restore some vision in the blind patients. The objective of this study was to assess the cost-effectiveness of the Argus® II Retinal Prosthesis System (Argus II) in Retinitis Pigmentosa (RP) patients. METHOD: A multi -state transition Markov model was developed to determine the cost-effectiveness of Argus II versus usual care in RP from the perspective of healthcare payer. A hypothetical cohort of 1000 RP patients aged 46 years followed up over a (lifetime) 25-year time horizon. Health outcomes were expressed as quality adjusted life years (QALYs) and direct healthcare costs expressed in 2012 €. Results are reported as incremental cost per ratios (ICERs) with outcomes and costs discounted at an annual rate of 3.5%. RESULTS: The ICER for Argus II was €14,603/QALY. Taking into account the uncertainty in model inputs the ICER was €14,482/QALY in the probabilistic analysis. In the scenarios of an assumption of no reduction on cost across model visual acuity states or a model time horizon as short as 10 years the ICER increased to €31,890/QALY and €49,769/QALY respectively. CONCLUSION: This economic evaluation shows that Argus II is a cost-effective intervention compared to usual care of the RP patients. The lifetime analysis ICER for Argus II falls below the published societal willingness to pay of EuroZone countries.

High-cost drugs for rare diseases: their expenditure and value based on a regional area-based study.

Background: in the field of rare diseases (RDs) most of the European studies on budget impact analysis of drugs that have been conducted often lay on theoretical assumptions and focus only on Orphan drugs (ODs). Objectives: we aimed to estimate the budget impact of specific drugs for non-oncological RDs, both ODs and non-ODs, using real-world data about patients residing in Veneto Region (Italy) and to describe its expenditure structure and dynamics. Methods: a population-based multi-source observational study was conducted using data from Regional administrative databases; an ad-hoc drugs' list specific for RDs including both ODs and non-ODs and classifying them by ATC codes has been created. Results: In 2019, the total expenditure for drugs specific for RDs was EUR 97.2 million (6.6% of the total Regional budget). The RD drug list included 58 ATC codes, of which 15 ATC had an annual budget impact over EUR 1 million ("blockbuster drugs"). The most expensive treatment was a non-OD drug (Coagulation factor VIII). The two most represented therapeutical areas were the metabolic and the hematological ones. Conclusions: Cost analyses on RD high-cost drugs expenditure should consider any specific RD drug, not only ODs. Expenditure dynamics for RD drugs are peculiar showing "blockbuster drugs". Some therapeutical areas seem to be lacking in the drug research field.

Sustaining universal health coverage: the interaction of social, political, and economic sustainability.

The sustainability of health care systems, particularly those supporting universal health care, is a matter of current discussion among policymakers and scholars. In this article, we summarize the controversies around the economic sustainability of health care. We attempt to extend the debate by including a more comprehensive conceptualization of sustainability in relation to health care systems and by examining the dimensions of social and political sustainability. In conclusion, we argue that policymakers when taking decisions around universal health care should carefully consider issues of social, political, and economic sustainability, their interaction, and often their inherent trade-offs.

Examining the antecedents of engaged nurses in Italy: perceived organisational support (POS); satisfaction with training and development; discretionary power.

AIM: This paper used Social Exchange Theory to empirically examine whether perceived organisational support, satisfaction with training and development and perception of discretionary power are antecedents of engagement for registered nurses working in Italian public and private hospitals (n = 827). BACKGROUND: According to Social Exchange Theory, effective workplace relationships support employees and encourage nurses to use training to enhance their workplace outcomes. METHOD: This research used a cross-sectional design. Data were collected from registered nurses working in six Italian hospitals using a survey-based, self-report strategy. RESULT: Regression analysis found that the variance of process-oriented supervision accounted for 6.9% of Italian registered nurse's perception of engagement, training and development accounted for 26.8% and discretionary power accounted for 2.1%. CONCLUSION: Workplace relationships enhance autonomy and engagement. Effective workplace relationship impacts positively on nurses' outcome. IMPLICATIONS FOR NURSING MANAGEMENT: This paper confirms the relevance of training to enhance engagement of nurses. It also confirms the importance of workplace relationships in enhancing autonomy and engagement. Previous research has identified the importance of nurses' autonomy in an environment where there are shortages of nurses. This study confirms a similar situation for Italian nurses. The findings underline the relevance of investments in continuous professional development to enhance nurses' engagement in private and public health-care settings.

The evolution of personalized healthcare and the pivotal role of European regions in its implementation.

Personalized medicine (PM) moves at the same pace of data and technology and calls for important changes in healthcare. New players are participating, providing impulse to PM. We review the conceptual foundations for PM and personalized healthcare and their evolution through scientific publications where a clear definition and the features of the different formulations are identifiable. We then examined PM policy documents of the International Consortium for Personalised Medicine and related initiatives to understand how PM stakeholders have been changing. Regional authorities and stakeholders have joined the race to deliver personalized care and are driving toward what could be termed as the next personalized healthcare. Their role as a key stakeholder in PM is expected to be pivotal.

Cost-Utility of Intermediate Obstetric Critical Care in a Resource-Limited Setting: A Value-Based Analysis.

Background: Sierra Leone faces among the highest maternal mortality rates worldwide. Despite this burden, the role of life-saving critical care interventions in low-resource settings remains scarcely explored. A value-based approach may be used to question whether it is sustainable and useful to start and run an obstetric intermediate critical care facility in a resource-poor referral hospital. We also aimed to investigate whether patient outcomes in terms of quality of life justified the allocated resources. Objective: To explore the value-based dimension performing a cost-utility analysis with regard to the implementation and one-year operation of the HDU. The primary endopoint was the quality-adjusted life-years (QALYs) of patients admitted to the HDU, against direct and indirect costs. Secondary endpoints included key procedures or treatments performed during the HDU stay. Methods: The study was conducted from October 2, 2017 to October 1, 2018 in the obstetric high dependency unit (HDU) of Princess Christian Maternity Hospital (PCMH) in Freetown, Sierra Leone. Findings: 523 patients (median age 25 years, IQR 21-30) were admitted to HDU. The total 1 year investment and operation costs for the HDU amounted to €120,082 - resulting in €230 of extra cost per admitted patient. The overall cost per QALY gained was of €10; this value is much lower than the WHO threshold defining high cost effectiveness of an intervention, i.e. three times the current Sierra Leone annual per capita GDP of €1416. Conclusion: With an additional cost per QALY of only €10.0, the implementation and one-year running of the case studied obstetric HDU can be considered a highly cost-effective frugal innovation in limited resource contexts. The evidences provided by this study allow a precise and novel insight to policy makers and clinicians useful to prioritize interventions in critical care and thus address maternal mortality in a high burden scenario.

Getting health reforms right: what lessons from an Italian case?

Over the past few years (2001-2007), the Italian National Health Service has been undergoing an important process of decentralization with a significant transfer of powers and responsibilities from the central government to the Regions. In this context, a particularly innovative example is represented by the case of Marche Region that extensively reformed its health-care system. For the analysis of this case, we used a theoretical framework that describes a health-care system in terms of components and goals. Policy-makers can act on the structural components of the system influencing the behaviour of individuals and organizations in order to lead the system towards the achievement of key performance goals. Marche regional Government was able to improve the system in crucial areas (clinical appropriateness and economic equilibrium) by intervening on institutional arrangements (particularly on the number and dimension of health-care delivery organizations) and on key control knobs (particularly planning and control mechanisms and the information system). Some conditions have allowed Marche policy-maker to successfully carry out this extensive process of reforms, particularly: (1) a shared sense of urgency; (2) agreement on the identification of problems and on the relative reform strategies to adopt; (3) coordination of all change strategies through a precise and clear long-term plan aimed at developing an integrated health-care delivery network.

Economic aspects in prolonged life sustainable treatments.

In a context of limited resources and continuous increase of healthcare expenditures, policy makers need to carefully evaluate the economic impact of their decisions. In the last decade economists have been particularly productive in offering to the decision makers a set of tools able to compare costs and benefits of each single medical procedure. The underlying assumption is that it is not acceptable the investment of considerable amounts of resources in medical procedures able to guarantee only limited improvements in health outcomes. In this particular type of context, an open issue is represented by the delicate topic of the provision of prolonged life sustaining treatments (PLST) to patients in vegetative state. The author, sustains that the provision of this type of treatments can be justified under a mere economic perspective. The traditional economic evaluations focus their attention only to the concept of incremental absorption of resources failing to capture other relevant dimensions: total costs and budget impact. The overall cost related to PLST due to the relative low unit cost and the low prevalence of patients in need of these treatments, represents a negligible part of the total health care budget. Hence, the emphasis on costs, in this circumstance, is misleading, especially considering that the current health care systems in the developed countries are characterized by wastes and inefficiencies that unduly increase costs. In addition, PLST provide a high return in terms of new knowledge, giving valuable insight on the clinical management of other severe health conditions. Long term therapies can also be considered a highly effective learning environment for health professionals. Finally, prolonged life therapies and treatments can also be considered an environment for experimenting "creative" solutions and approaches.

A model for Southern Mediterranean research institute self-assessment: a SWOT analysis-based approach to promote capacity building at Theodor Bilharz Research Institute in Cairo (Egypt).

BACKGROUND AND STUDY AIMS: THEBERA is a project funded by the European Union (EU), as an ERA-WIDE FP7 project, aiming to strengthen the Theodor Bilharz Research Institute (TBRI) capacities. MATERIALS AND METHODS: A SWOT (strength/weakness/opportunities/threats) analysis of human, structural and organisational existing resources was performed in light of an extensive analysis of liver disease research and clinical management in Egypt, for a full understanding of TBRI needs. RESULTS: Strength and weakness features were identified and analysed, so were actions to be implemented and targets to be accomplished, to develop a business plan gathering the required critical mass (political, scientific, industrial, social) to select investment priorities, to sacrifice non-strategic areas of research, to promote national and international connections and industrial innovations, to update diagnostics and research device technologies and clinical management processes at European levels, to implement fundraising activities, to organise and properly assess training activities for young researchers, physicians, nurses, and technicians. CONCLUSIONS: Research institute self assessment is a priority need for sustainable capacity building and for future build-up of a competent health care research institute. Sustainable capacity building strategies must be designed on needs assessment, involving salient requirements: clear strategy, leverage of administrative capacities, industrial support and connections, systematised training programmes and enhancement of mobility of health care staff implemented within ill-defined boundaries and continuously re-evaluated with multiple feedback loops in order to build a complex, adaptable and reliable system based on value.

O gerenciamento na unidade sanitária local italiana / The management of the Italian local health system

Considera que os sistemas sanitários públicos têm problemas mais complexos e diversos do que os enfrentados pelas empresas. Discute e reflete sobre estas dificuldades (AMSB)