Italian National Node

>> WebsiteCNR is the largest public research institution in Italy, the only one under the Research Ministry performing multidisciplinary activities. Founded as legal entity on 18 November 1923, CNR’s mission is to perform research in its own Institutes, to promote innovation and competitiveness of the national industrial system, to promote the internationalization of the national research system, to provide technologies and solutions to emerging public and private needs, to advice Government and other public bodies, and to contribute to the qualification of human resources. In the CNR’s research world, the main resource is the available knowledge, which means people, with their skills, commitment and ideas. This capital comprises more than 8.000 employees, of whom more than half are researchers and technologists. Some 4.000 young researchers are engaged in postgraduate studies and research training at CNR within the organization’s top-priority areas of interest. A significant contribution also comes from research associates: researchers, from Universities or private firms, who take part in CNR’s research activities.

CNR participates in EPTRI-IT with 13 Institutes. Read more about them.

>> WebsiteFounded in 1088, UNIBO is recognized as the oldest university of the Western world. Nowadays, UNIBO is the second largest university in Italy and one of the most important institutions of higher education across Europe. UNIBO in numbers: 5 operating sites and a permanent headquarter in Buenos Aires, 637 M€ turnover, 32 Departments, 5 Schools, 87.758 students (among which, 6.484 International students), 5.733 permanent staff, 2nd Italian University in QS World University Ranking 2019 for Life Science & Medicine, 30 research outputs per day and 11.000 research outputs per year. Research and Innovation are a priority of UNIBO. Regarding its fundraising ability, UNIBO is very active at both national and the European level in all the research areas. Among its assets, UNIBO encompasses Bologna University Hospital Authority St. Orsola-Malpighi, which is home to the School of Medicine and Surgery. The University Hospital is an internationally acclaimed institution for the study and treatment of diseases, and each year organizes medical conferences and conventions attended by professionals of international fame. In Horizon 2020, UNIBO is involved in 306 funded projects (81 as coordinator) with more than 128 M€ of funding. In the framework of the Societal Challenge 1 – Health, Demographic Change & Wellbeing and the Innovative Medicine Initiative and related funding frameworks, UNIBO is participating in 30 projects, including 5 under IMI-2 JTI and 2 projects selected for funding under the SC1-PHE-CORONAVIRUS-2020-2 call, with a total EU contribution of over 14 M€. Moreover, the University of Bologna has advanced skills in management of large amount of digital data. The interdisciplinary research focuses on healthy ageing, cognitive impairment and musculoskeletal conditions, among others. Research activities spans from the application of virtual/augmented reality (VR/AR) and digital diagnostics and care, to the use of health informatics and mobile technologies.

Health Research
The main research activities developed in UNIBO are referred to twelve main fields:

1. Ambient Assisted living
2. Big Data and Omics medicine
3. Brain Ageing And neurodegeneration
4. Cancer
5. Digital Medicine and e-health
6. Epidemiology, biobanking and Cohorts
7. Multimorbidity and age-related Diseases
8. Pharmaceutical biotech
9. Rare Diseases and Undiagnosed disorder
10. Regenerative medicine and tissue Engineering
11. Skeletal Health and Diseases
12. Systems and Personalised medicine

With four specific paediatrics research domains:

• Neonatology
• Emergency pediatrics
• Pediatric oncology
• Pediatric neurology

>> WebsiteIt is a non-profit organisation whose mission is to perform research and education in the pharmaceutical field integrating regulatory, clinical, epidemiological, economical and statistical expertise. It is a consortium between Università degli Studi di Bari ‘Aldo Moro’ (UniBA), Fondazione per la Ricerca Farmacologica Gianni Benzi Onlus (FGB), TEDDY – European Network of Excellence for Paediatric Reserach (TEDDY) and Mediterranea – Associazione per lo sviluppo locale (MEDITERRANEA). The main fields of interest are life sciences and biotechnologies, drug development for small populations (paediatric and rare diseases), research management and methodology, monitoring, statistics, regulatory, ethics and pharmacovigilance. CVBF can count on the expertise and collaboration of researchers, scientists, health experts and research centers working together to identify the most appropriate research tools and procedures that reflect the specificity of the paediatric patient population. CVBF has a significant expertise in the project coordination, conducting research activities mainly in the context of national and EU funded projects and being Coordinator of the first paediatric network of excellence under the FP6 (TEDDY), 2 FP7 projects (DEEP, GAPP) and Partner in many projects funded by the European Commission (DG-Research and DG-SANCO).

>> Websiteis a Scientific Research Organisation, registered as a private, not-for-profit Foundation in 2007 to promote research in biological, medical and pharmaceutical areas, with a special attention to populations with specific therapeutic needs, including children and patients affected by rare diseases. During the past 20 years, initially through its founders and then as an organisation, FGB has been an active part of the European common efforts to improve the conditions for the development of innovative and safe medicines in Europe, including the setting up of a large partnership working on novelties and good practices for the European Pharmaceutical System which led to the establishment of the TASK-FORCE IN EUROPE FOR DRUG DEVELOPMENT FOR THE YOUNG – TEDDY, the first European Network of Excellence funded to promote the availability of safe and effective medicines for children in Europe, and its successors GRiP – Global Research in Paediatrics Network of Excellence and c4c – Collaborative Network for European Clinical Trials for Children. Key achievements include the 13 yearly editions of the Foresight Training Course, international courses aimed at training high-specialised professionals in the different fields of regulatory sciences and representing a relevant occasion to disseminate good practices and to promote pharmacological and health science research and innovation among the main stakeholders.

FGB staff members have gained skills and knowledge in the fields of Regulatory Science and Ethics related to paediatrics and rare diseases as well as HTA, they contribute to international research activities by designing Paediatric Investigation Plans (PIPs) and preparing the related dossiers, by preparing guiding documents and templates on paediatric consent and assent forms, by collecting, managing, analysing, sharing, and reusing health data and by producing health technology assessment reports. FGB experts are or have been members of the Paediatric Committee (PDCO) and other scientific committees and working groups at the European Medicines Agency (EMA), the Italian Medicines Agency (AIFA), the Italian National Agency for Regional Health Services (AGENAS) and the Regional Agency for Health and Social Care of Puglia, Italy (AReSS). FGB regularly contributes to public consultations on ethics, paediatrics, rare diseases, clinical trials, preclinical studies, patients’ empowerment in drug development, etc. launched by the EMA and the European Commission.

Within its IT-Lab, researchers and data scientists are engaged in designing, developing and managing platforms, databases and other IT-tools devised to support, manage and monitor research activities (e.g., patients registries, surveys and other data collection tools, e-learning environments, Project Management and Performance Monitoring Systems, etc.).

In the European Paediatric Translational Research Infrastructure – ID-EPTRI project, FGB has been member of the Project Steering Committee and substantially contributed to the Conceptual Design Report (CDR) and the technical design of the future Research Infrastructure as well as in other activities such as the context analysis and the ethical review of the infrastructure.

Fondazione Penta ONLUS – Child Health Research was established in 1991 as an independent collaboration among paediatric HIV centres across Europe aiming to address questions about antiretroviral therapy (ART) in HIV infected children. The Penta Foundation was subsequently set up in 2004 as the legal body representing the Penta network. In 2011 Penta evolved to Penta-ID (Infectious Diseases, integrating the expertise acquired in more than 20 years of successful activity in the area of HIV to other paediatric infectious diseases and investigate and implement the best ways to prevent, diagnose and treat diseases in children. To date, Penta sponsored 20 trials in paediatric HIV. The Penta network benefits from international recognition, wide geographical representativeness with over 130 centers in 42 countries, very high-quality research, an outstanding track record and diversified public and private funding. Penta-ID has been recognized as a Level-1 Network for paediatric infectious disease in Europe by EnprEMA. Penta is also member of a WHO Network (Global Accelerator for Paediatric Formulations (GAP-f)) aimed at enhancing the coordination and accelerating the prioritization, investigation, development, introduction, and safe rollout of high-quality medicines in optimal formulations for children in need. Current activities beyond clinical trials and epidemiologic studies, include training/educational programmes, capacity building activities, and development of HIV treatment guidelines.

>> WebsiteIt is dedicated to research, treatment and training in the field of neurological and neuropsychic pathologies. The researchers work on neonatology, neonatal and paediatric intensive care, psychiatry and psychology, otolaryngology, neurology, ophthalmology, musculoskeletal diseases, pneumology and respiratory diseases. The Neurobiology Laboratory studies the paediatric development mechanism in neurology (e.g., movement disorders, infant brain atlas) and in rehabilitation. They also develop new model platforms using fly models. They perform developmental molecular and experimental pharmacology using in-vivo and ex-vivo model to test drugs. Moreover, the researchers use drosophila model to study motor neuron diseases, in particular hereditary spastic paraplegia genes. They use also the imaging technology to study brain and muscle. Furthermore, they work to identify the defect in autophagy process in hereditary spastic paraparesis type 15. At the Bioengineering Laboratory, the researchers study new conventional and small active pharmaceutical ingredients, and develop oral, buccal, sublingual, solid monolithic, solid powder, solid multiparticulates, liquid, and semisolid solutions. They perform also study in medical devices field, prototyping and validating devices and software dedicated to improving quality of life, rehabilitate and promote social inclusion of patients with chronic diseases.

>> WebsiteIt is a no profit consortium composed by the main Italian children’s hospitals, the largest departments of paediatrics as well as national and international paediatric therapeutic networks coordinated by Italian Institutions. It is coordinated by Ospedale Pediatrico Bambino Gesù and counts 23 Partners. Its primary mission is to foster high-quality research on drugs in children, but also improve opportunities for performing paediatric clinical trials, provide training and create competences, harmonize joint national operational models, create guidelines and templates to help serve research, involve children and young people and their families in paediatric research.

Founded in 1939, the Italian Society of Pharmacology (SIF) was recognized in 1996 as a non-profit scientific association by the Ministry of University and Scientific and Technological Research. SIF is a member of IUPHAR (International Union of Pharmacology) and EPHAR (Federation of European Pharmacological Societies). The current membership consists of approximately 1,200 Members. Recently within the Society, a Working Group on Pediatric Pharmacology (http://www.sifweb.org/la_societ%C3%A0#Gruppi_di_lavoro) has been established, joined by 44 pharmacologists. To respond to the increasing interest of individual researchers, scientific societies and pharmaceutical companies, the Pediatric Pharmacology Working Group aims to address the many specificities characterizing clinical pediatric research. Among these: (i) the need to develop protocols designed for specific age groups, as protocols used for the adult population may be inadequate for children, as well as those for older children may be inadequate for infants and newborns; (ii) the consolidation of competences for conducting early stage pediatric clinical trials (phase I / II, pharmacokinetic / pharmacodynamic studies) and the development of pharmacokinetic models suitable for children; (iii) the identification of biomarkers and new non-invasive parameters for drug efficacy assessment; (iv) the development of formulations suitable for children of various ages; (v) the application of innovative biostatistics methodologies; (vi) new preclinical protocols for the development of pediatric medicines and ATMP and (vii) the design of studies to evaluate long-term efficacy and safety. These activities are also implemented within SIF via the involvement of other existing SIF working groups, such as that SIF4RARE (Rare disease and Orphan Drugs) and Neuropsycopharmacology for specific projects on pediatric diseases.

TEDDY European Network of Excellence for Paediatric Research is a scientific organization arisen from the Network of Excellence named “Task-force in Europe for Drug Development for the Young”, created in 2005 within the TEDDY project (LSHB-CT-2005-005216) funded under the Sixth Framework Programme of the European Commission with the aim to favour the integration of the paediatric research activities, a social awareness on the importance of the paediatric medicines across and the improvement of the relevant ethical, legal and regulatory frameworks. The main objective of the TEDDY Network is to promote children tailored medicine, guarantying children rights and well-being. It addresses methodological, ethical, legal and social issues of research (from the preclinical phase to clinical evaluations and medicines utilization, access policies and education) and implements good practices and tools to plan, design and perform clinical studies at national and international level. TEDDY is a category 1 network member of Enpr-EMA, the European Network of Paediatric Research at the European Medicines Agency and registered at ENCePP (the European Network of Centres for Pharmacoepidemiology and Pharmacovigilance). TEDDY can count on the multidisciplinary expertise and collaboration of researchers, scientists, health experts and paediatric clinical research centres working together to identify the most appropriate research tools and procedures that reflect the specificity of the paediatric patient population in compliance with children rights. It collaborates with existing paediatric networks, and research organizations with the goal to promote and foster scientific and technological excellence in an ethically sound paediatric research in Europe. Moreover, TEDDY has developed a European Paediatric Medicines Database (EPMD) including information on medicines approved for children by EMA (indication, dosages, by age populations, studies and trials). TEDDY has also developed a long-standing experience and expertise focused on innovative methodologies and ethical, legal, and societal issues relevant for paediatric research also developed within EU funded projects and in collaboration with European institutions such as the European Medicine Agency, the European Commission, and the Council of Europe.  TEDDY is particularly engaged in increasing awareness on the topics of paediatric research and encouraging the participation of children in research. TEDDY provides support, education and information to patients and families regarding their participation in clinical trials. To this aim, different tools and educational resources with different format (e.g., serious games, comics, videos on the development of clinical trials) have been developed. Regarding the patients’ empowerment and the involvement in the paediatric clinical research, TEDDY has promoted the creation of several Young Persons Advisory Group (YPAG) in Italy, Albania, Greece, Romania and Czech Republic. TEDDY is also strongly involved in the initiative of the Council of Europe on strengthening children’s participation in the decision-making process on matters regarding their health.Thanks to the international recognition and the wide geographical representativeness, TEDDY is able to provide the paediatric community with: advice on Paediatric Medicine Development Issues; advice on relevant Ethical, Legal and Societal Issues (ELSI) in a children/fundamental rights perspective (e.g. data protection, right to health protection…); advice on children empowerment and engagement in paediatric studies; easy dissemination of relevant information; documents and recommendations in the sector of paediatric study methodology and ELSI issues.

>> WebsiteIt is a multidisciplinary university, which offers a wide range of academic programs in different disciplinary fields. At the Department of Pharmacy and Pharmaceutical Sciences, the researchers perform educational and research activities on drug design, parallel synthesis of focused molecular libraries, in vitro target validation, in silico predictive toxicology, pre-formulation and formulation studies in paediatric, and prospective repurposing development process. They develop palatable oral liquid formulations of insoluble drugs using complexing excipients such as cyclodextrins, which improve both aqueous solubility and stability of interacting drugs. The unit has all the facilities to produce microparticulate-based formulations by prilling and spry-drying technologies, useful to mask the unfavourable taste of active pharmaceutical principles, increasing the acceptability of the paediatric patients. Innovative tailored formulation based on nanotechnology and 3D printing are also explored in order to generate patient centric dosage forms. Moreover, this department offers a wide expertise and technical equipments for the production and optimisation of different pharmaceutical formulations, and a computational laboratory for molecular modelling of drug and excipients.

>> WebsiteIt is a multidisciplinary university, which offers a wide range of academic programs in different disciplinary fields. The research areas include life science, physical sciences and engineering, social sciences and humanities. The researchers work in cardiology and vascular diseases, neurology, hepatology, haematology, pneumology, respiratory diseases, oncology, rheumatology, infectious diseases, immunology and genetics. The researchers study paediatric development mechanisms in oncology (e.g., neuroblastoma, epigenetics, glioma), but also new treatment based on immunotherapy (e.g., natural kill cell mediated) and chemotherapy. They perform in vitro and in vivo studies using animal models (e.g., rat), proteomics to study TRIP8b protein in brain. Moreover, they use genetics approaches to identify shared polygenic risks and causal associations in amyotrophic lateral sclerosis. They also investigate glioma microenvironment using molecular and imaging biomarkers to overcome treatment resistance. They study the effects of different type of molecules (e.g., crizotinib) in patients with anaplastic lymphoma kinase positive refractory to chemotherapy. They also perform stem cell and regenerative medicine with tissue engineering approaches. Recently, they study the infective mechanism, the effects in children, adult and older of COVID-19, and compare and analyse data using genetics approaches.