The project aims to create a transparent ecosystem to foster decision-making in paediatric and orphan drugs development by integrating innovative methodologies, such as modelling and simulation (M&S) methods and related data types, within regulatory procedures. With three pillars: a data repository, the development of high-quality standards for data and analytical methods, and an Artificial Intelligence-based platform, it aims to establish the credibility of M&S methods and related results as sources of evidence within regulatory procedures.

ROLE OF THE FOUNDATION

The Foundation is involved in constructing the integrated model for the application of innovative methodologies in paediatric and orphan drug development, while also assessing their regulatory acceptability. Furthermore, it is involved in applying the model to three specific case studies of paediatric and rare diseases. Lastly, the Foundation collaborates to the preparation and application of the qualification procedure to the European Medicines Agency (EMA) to validate the project methodology tools.

www.erametproject.eu

The project “DANTE: Digital Solutions for a Healthy, Active and Smart Life”, coordinated by Cluster Tecnologico Nazionale SMILE “Smart Living Technologies” (dante@clustersmile.it; www.clustersmile.it; Via Monteroni A 3, Lecce 73100, Italy) focuses on the application of Artificial Intelligence, Cyber Security and High-Performance Computing to the Smart Living and Health and Active Life fields. The identified catchment area will be at national level, covering different regions, in a networked structure that aims to interact with many stakeholders, public and private entities,  operating in the different sectors: Smart Home, Smart Living Environments, Social Housing, Telemedicine, Remote Monitoring & Assistance, Tele-Care, Mobile Health, Internet of Things, Service-Assistive and Rehabilitation Robots, Big Data for Health, Sensors and Integrated Systems, Wearable Devices.

ROLE OF THE FOUNDATION
The Foundation is leader of the WP3 “DANTE DIH go-live, operation & improvement” and member of the coordination team.

https://dante-edih.clustersmile.it/

Funded by the European Union under grant agreement no 101093913. Views and opinions expressed are however those of the author(s) only and do not necessarily reflect those of the European Union. The European Union cannot be held responsible for them.

The project "Integration of the pharmacokinetics-dynamics models to the multiple-criteria decision analysis (MCDA) for the perspective risk-benefit assessment of the iron oral chelators in paediatric patients with transfusion dependent haemoglobinopathies" aims to prove the usefulness and benefits of the modelling approach, multiple criteria decision analysis (MCDA), integrated with pharmacokinetics-dynamics models (PK/PD) for the risk-benefit assessment of medicines for rare diseases. To this end, a case study on the use of Deferiprone (DFP), an oral chelator, is carried out. The analysis is based on efficacy (ferritin) and safety (neutropenia and other adverse events) endpoints. It aims to predict the efficacy and safety profile of DFP. The model will be validated with literature data and with an elicitation process involving international clinical and regulatory experts.

ROLE OF THE FOUNDATION
The Foundation deals with the methodological and regulatory aspects of the research. It is involved in the literature search to collect existing data on the DFP efficacy and safety. Furthermore, the Foundation cooperates with the project Scientific Coordinator in the MCDA integration with a PK/PD model to support the relationship between neutropenia and ferritin levels in the DFP case study. The Foundation arranges regular meetings with the experts and it is in charge of the administrative task of the project.

The project is aimed to renew and implement the EPIICAL 1 mission of designing and conducting studies in paediatric HIV populations both in Europe and Africa, by recruiting new patients and following up the existing ones.

ROLE OF THE FOUNDATION
The Foundation is involved in Working Group 4 “Data Analysis, Mathematical modelling, Communication, Regulatory, Management” where coordinates the activities related to data protection and confidentiality and to the ethics and regulatory aspects of the clinical studies.

www.epiical.org

BIOMIS - "Costituzione della biobanca del microbiota intestinale e salivare umano: dalla disbiosi alla simbiosi" is a collaborative project aimed at establishing the first Italian biobank of intestinal, salivary and vaginal microbiota. Microorganisms will be characterized using high-throughput meta-omic approaches. In vivo and in vitro studies will be performed, with animal models matching patients’ clinical characteristics. Therapeutic approaches will be explored, studying the efficacy of faecal microbiota transplantation (FMT) in patients with different diseases, to restore the healthy microbiome in the diseased colon, leading to symptoms resolution. Innovative capsules and faecal suspensions will be produced in order to implement new administration strategies for the FMT. The outcomes of BIOMIS will enable healthcare professionals to custom therapeutic applications by modulating patient microbiota and specific host – microbe interactions. Top-leading research and clinical centres will collaborate with private companies to streamline project objectives and open new therapeutic perspectives for several human diseases.

www.ponricerca.gov.it

ROLE OF THE FOUNDATION
The Foundation will ensure the compliance of the project activities with the regulatory and ethical standards and will support the participation of the new BIOMIS biobank in the European research infrastructure BBMRI-ERI. Moreover, the Foundation will define the quality procedure to ensure the technical and scientific consistency of the activities.

The EJP RD brings over 130 institutions from 35 countries to create a comprehensive, sustainable ecosystem allowing a virtuous circle between research, care and medical innovation. The EJP RD has two main objectives: on one hand, to improve the integration, the efficacy, the production and the social impact of research on rare diseases through the development, demonstration and promotion of Europe/world-wide sharing of research and clinical data, materials, processes, knowledge and know-how; on the other hand, this project aims at implementing and further developing an efficient model of financial support for all types of research on rare diseases (fundamental, clinical, epidemiological, social, economic, health service) coupled with accelerated exploitation of research results for benefit of patients.

ROLE OF THE FOUNDATION
The Foundation leads the activities necessary to provide the proper strategy to address ethical, regulatory and legal issues, ensuring that relevant rules are complied with within the course of the project. This will allow EJP RD to perform not only ethically-sound but also regulatory-compliant multi-national research.

http://ejprarediseases.org/

The ARISE consortium will establish an inter-agency and multidisciplinary staff exchange programme between researchers and other staff members among partners. This programme will foster sharing of best practice in Newborn Screening, diagnosis and treatment of Sickle Cell Disease (SCD) leading to improvement in overall disease outcome. A large secondments plan will enable collaborative research and training initiatives, covering the following items: analysis of SCD prevalence in consortium member countries and identification of specific haemoglobin genotypes and phenotypic presentations; establishment of laboratory diagnosis and quality assurance systems for population screening; setting up of SCD national prevention programmes; newborn and infant screening for SCD early diagnosis; treatment protocols for the management of common SCD complications and transition from paediatric to adult care, health promotion strategies and nutrition.

ROLE OF THE FOUNDATION
Gianni Benzi Foundation will be responsible for the ARISE Scientific Coordination and Project Management, in collaboration with Guy's & St. Thomas' Hospital NHS Foundation Trust (UK). The Foundation will lead the WP2 activities aimed at implementing eHealth technologies to support a Newborn screening programme in Kenya, Nigeria and Lebanon and will oversee all the project ethical requirements (WP8).

https://www.ariseinitiative.org/

The project is setting up a large collaborative paediatric network that will facilitate the development of new drugs and other therapies for the entire paediatric population in Europe. the consortium works to build capacity for the implementation of multinational paediatric clinical trials whilst ensuring the needs of babies, children, young people and their families are met. c4c strives to bring innovative processes to all stages of clinical development by generating a new model of organization and of the clinical development process. By emphasizing inclusiveness and collaboration across geographical, specialty, sectoral, cultural and societal backgrounds, it will set up a new infrastructure to support all evaluations of medicines in children.

ROLE OF THE FOUNDATION
The Foundation is partner of the project co-leading the activities related to “data coordinating centre and data quality standards”, specifically contributing to implementing performance metrics in execution of Paediatric Clinical Trials. Other activities are carried out for preparing training course and material and dissemination campaignes to increase awareness on paediatric research.

http://conect4children.org/ - c4c (CORDIS)

The main objective of this project is to design the framework for the European Paediatric Translational Research Infrastructure (EPTRI), a new Research Infrastructure (RI) aimed to enhance technology-driven paediatric research in discovery and early development phases to be translated into clinical research and paediatric use of medicines.

ROLE OF THE FOUNDATION
The Foundation is a third party of CVBF. Its main responsibility is designing the organisational and technical model of the infrastructure, providing the setting-up and implementation of IT platform and IT technologies to coordinate the information flow (communication, collaboration, and information sharing) among the participating units and services within the research infrastructure and with the external environment (member states, industry, patients and other research groups).

https://eptri.eu - EPTRI (CORDIS)

The project is aimed to create a paediatric research infrastructure starting from ECRIN infrastructure and including 14 partners involved in paediatric clinical research. PedCRIN represents a valid implementation step covering the issue of managing good paediatric trials.

ROLE OF THE FOUNDATION
The Foundation provides resources to CVBF with the aim to make available expertise to develop and adapt tools to be used for multinational paediatric clinical trials. Also, it provides for the training and support of partners on the use of these tools.

www.ecrin.org/projects/pedcrin - PedCRIN (CORDIS)

The project is aimed to establish predictive in vitro and in vivo platform to select promising HIV therapeutic vaccine candidates and to evaluate a therapeutic vaccine strategy to improve the management of perinatal HIV-Infected children.

ROLE OF THE FOUNDATION
The Foundation provides resources to CVBF with the aim to make available expertise to coordinate all ethical and regulatory activities related with clinical studies and health data collection and sharing.

www.epiical.org

The twinning project is aimed to raise the Children’s Memorial Health Institute (IPCZD, Warsaw) staff’s research profile and increase capacity in providing innovative paediatric research methods such as PK-PD data Modelling&Simulation (M&S) data extrapolation, and suitable dosage forms identification.

ROLE OF THE FOUNDATION
The Foundation provides resources to CVBF with the aim to make available expertise to coordinate the training program including face-to-face, e-learning and staff-exchanges to provide competences and skills in the 4 research profiles according to students preferences and plan of activities.

http://www.smart-h2020.eu - SMART (CORDIS)

Conduction of a multicentre clinical trial of clonidine in children until the age of 18. Indeed, despite its widespread use in Pediatric Intensive Care Units (PICU), this drug still lacks authorisation for paediatric use. A parenteral age-appropriate formulation of clonidine for sedation in PICU will be developed and tested in order to obtain a Pediatric Use Marketing Authorisation (PUMA).

ROLE OF THE FOUNDATION
The Foundation is partner of the project as involved in regulatory and ethical issues and coordinates the dissemination activities. It also supports the project management.

www.closed-fp7.eu - CloSED (CORDIS)

The project is aimed to study and expand the availability of drugs for children in chronic pain through the development of a gabapentin formulation appropriate for them.

ROLE OF THE FOUNDATION
The Foundation supports the ethical and regulatory activities related to the clinical studies and coordinates the project Ethics Boards.

www.pediatricpain.eu - GAPP (CORDIS)

The project aims to increase the current knowledge on iNMDs (Inherited NeuroMetabolic Diseases), speed up the timely and precise identification of patients, who thus may benefit from the available (experimental and marketed) treatments, and also favour biomedical research. The Platform also acts to connect the Network nodes, to stimulate collaborative working and to promote the networking activities.

ROLE OF THE FOUNDATION
As a project partner, the Foundation was involved in the project management and in the development of the IT platform used to collect, share and store the information on Inherited NeuroMetabolic Diseases (iNMDs) and in the preparation of ethical and methodological recommendations to favor biomedical research.

The project is aimed to develop small biologically active molecules for the diagnosis and the treatment of neuroblastoma.

ROLE OF THE FOUNDATION
The Foundation was the project partner in charge of the identification of the existing educational and training programmes and in the preparation of the educational material of the Module of the Master related to paediatric clinical trials methodology, including privacy and data protection.

GRIP, the Global Network of Excellence is aimed to implement an infrastructure to stimulate and facilitate the development and the safe use of medicines for the paediatric population. Its main outcome is the creation of the Master of Science in Paediatric Medicines Development and Evaluation.

ROLE OF THE FOUNDATION
The Foundation provides resources to CVBF with the aim to make available regulatory, ethical and pharmacoeconomic expertise. The Foundation was also involved in the project management and training activities.

www.grip-network.org/index.php/cms/en/home - GRIP (CORDIS)

The project is aimed to study the efficacy and safety of deferiprone in children affected by β-thalassaemia major and other transfusion-dependent haemoglobinopathies.

ROLE OF THE FOUNDATION
The Foundation supported of the ethical and regulatory activities.

www.deepproject.eu/ - DEEP (CORDIS)