PROVIDING METHODOLOGICAL SUPPORT TO THE BIOMEDICAL RESEARCH

ETHICS AND RESEARCH METHODOLOGY

Planning and performing observational and interventional studies and registries are some of our main activities. Our research methodological expertise is also actively involved in implementing innovative study’s designs and research methods, especially for rare and paediatric diseases.

We work on the Health Technology Assessment (HTA), to evaluate both real and potential effects of technology, as well as the consequences that the introduction or the exclusion of an intervention (for example a medicine or a device) has for the health system, the economy and society. The activity includes the conduct of studies and analysis aimed at providing summaries and evaluations of the available evidence, including Patient Reported Outcomes (PROs) and Quality of Life (QoL) assessment.

RESEARCH PROJECTS

We actively participate in many national and international collaborative projects providing scientific expertise as well as regulatory, ethical and methodological support.

Within these projects, we are involved in the ethical and methodological activities, study design, compliance with regulatory requirements, data collection and analysis. Moreover, we lead project management tasks and IT tools set up (platforms, databases, e-learning instruments, surveys), also providing contents to dissemination and educational activities.

INTEGRATION OF THE PK/PD MODELS TO THE MULTIPLE-CRITERIA DECISION ANALYSIS FOR THE RISK-BENEFIT ASSESSMENT OF THE IRON ORAL CHELATORS

Proving the usefulness and benefits of the modelling approach, multiple criteria decision analysis for the risk-benefit assessment of medicines for rare diseases

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.

FUNDED BY: Art.6 della Legge regionale del 14 Aprile 2018 n.15 - Interventi finanziari in favore della ricerca per la cura delle malattie rare
DURATION: Nov 2020 >
PARTNERS: 2
COUNTRIES INVOLVED Italy, United Kingdom

NOVEL STRATEGIES TO INDUCE LONG-TERM VIRAL REMISSION IN EARLY TREATED HIV INFECTED CHILDREN

To implement novel strategies to obtain long-term viral remission in Early Treated HIV infected Children

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

FUNDED BY: Fondazione PENTA Onlus​
DURATION: Feb 2020 > 
PARTNERS: 27
COUNTRIES INVOLVED Italy, UK, France, Spain, Portugal, USA, South Africa, The Netherlands, Thailand, Canada, Mozambique

BIOMIS

Establishing the first Italian biobank of intestinal, salivary and vaginal microbiota.

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.

FUNDED BY: PON “Ricerca e Innovazione” 2014-2020 e FSC
DURATION: May 2019 >
PARTNERS: 11
COUNTRIES INVOLVED
Italy

European Joint Programme Rare Diseases

Developing a sustainable ecosystem allowing a virtuous circle between rare diseases care, research and medical innovation

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/

FUNDED BY: H2020
DURATION: Jan2019 >
PARTNERS: 88
COUNTRIES INVOLVED
27 EU Member States (Austria, Belgium, Bulgaria, Czech Republic, Denmark, Estonia, Finland, France, Germany, Greece, Hungary, Croatia, Ireland, Italy, Netherlands, Latvia, Lithuania, Malta, Poland, Portugal, Romania, Spain, Sweden, Slovakia, Slovenia, United Kingdom), 7 associated (Armenia, Georgia, Israel, Norway, Serbia, Switzerland, Turkey) and Canada.

African Research and Innovative initiative for Sickle cell Education: Improving Research Capacity for Service Improvement

Sharing best practices in Newborn Screening, diagnosis and treatment of Sickle Cell Disease

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/

FUNDED BY: H2020
DURATION: Jan2019 >
PARTNERS:15
COUNTRIES INVOLVED
Italy, Cyprus, France, UK, Nigeria, Kenya, Lebanon, USA

c4c - COllaborative Network for European Clinical Trials For Children

Better medicines for babies, children and young people through a pan-European clinical trial network

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)

FUNDED BY: H2020
DURATION: may 2018 >
PARTNERS:43
COUNTRIES INVOLVED
Italy, UK, The Netherlands, Denmark, Switzerland, Portugal, Belgium, Estonia, Austria, Germany, Greece, Poland, Norway, Ireland, Sweden, Spain, Hungary, France, Czech Republic, USA

European Paediatric Translational Research Infrastructure

Translational infrastrucure to enhance paediatric research and foster innovation

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)

FUNDED BY: H2020
DURATION: Jan 2018 > Dec 2019
PARTNERS:26
COUNTRIES INVOLVED
Italy, The Netherlands, UK, Greece, Austria, Cyprus, France, Romania, Poland, Czech Republic, Portugal, Russia, Spain, Albania, Israel, Estonia, Germany, Switzerland, Sweden

Paediatric Clinical Research Infrastructure Network

Developing capacity for the management of multinational paediatric non-commercial clinical trials

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)

FUNDED BY: H2020
DURATION: Jan 2017 >
PARTNERS:15
COUNTRIES INVOLVED
France, UK, Italy, the Netherlands, Finland, Spain, Switzerland, Sweden, Norway, Estonia, Greece, Austria, Ireland

Early treated Perinatally HIV Infected individuals: Improving Children’s Actual Life

Predictive platform for the early identification of novel therapeutic strategies for HIV infected children

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

FUNDED BY: Fondazione PENTA Onlus
DURATION: Feb 2016 > Feb 2020
PARTNERS:27
COUNTRIES INVOLVED
Italy, UK, France, Spain, Sweden, USA, South Africa, The Netherlands, Thailand, Canada, Mozambique

Small Medicines Advanced Research Training

Improvement of the Children’s Memorial Health Institute staff’s research profile and increase capacity in providing innovative paediatric research methods

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)

FUNDED BY:H2020
DURATION: Jan 2016 > Dec 2018
PARTNERS:3
COUNTRIES INVOLVED
Poland, Italy, UK

Clonidine for Sedation of Paediatric Patients in the Intensive Care Unit

Studying clonidine for sedation in paediatric and neonatal intensive care units

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)

FUNDED BY:FP7
DURATION: Dec 2013 > Nov 2018
PARTNERS:10
COUNTRIES INVOLVED
Germany, UK, The Netherlands, Italy, Sweden, Czech Republic, Estonia, Spain

Gabapentin in paediatric Pain

Providing children with a safe and effective medication for chronic pain

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)

FUNDED BY:FP7
DURATION: Jul 2013 > Jun 2017
PARTNERS:15
COUNTRIES INVOLVED
Italy, The Netherlands, Estonia, UK, Germany, Albania, France, Spain, Greece

Inherited NeuRoMetabolic Diseases Information NETWORK

Information Network and IT Platform on Inherited NeuroMetabolic Diseases

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.

FUNDED BY:EU Health Programme
DURATION: Apr 2013 > May 2016
PARTNERS:5
COUNTRIES INVOLVED
Italy, Spain, Denmark, Croatia

Verso la Medicina Personalizzata: Sviluppo di nuove molecole selettive per la cura del Neuroblastoma

Small biologically active molecules for the diagnosis and the treatment of neuroblastoma

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.

FUNDED BY:Fondazione CON IL SUD
DURATION: Oct 2012 > Nov 2015
PARTNERS:5
COUNTRIES INVOLVED
Italy, Ireland

Global Research in Paediatrics

EU network of excellence to stimulate and facilitate the development and safe use of medicines in children

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)

FUNDED BY:FP7
DURATION: Jan 2011 > Jun 2017
PARTNERS:21
COUNTRIES INVOLVED
Italy, USA, The Netherlands, UK, France, Spain, Poland, Finland, Canada

Deferiprone Evaluation in Paediatrics

Providing children affected by thalassaemia and other transfusion-dependent haemoglobinopathies with a new formulation of deferiprone

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)

FUNDED BY:FP7
DURATION: Jan 2011 > Aug 2016
PARTNERS:16
COUNTRIES INVOLVED
Italy, the Netherlands, Greece, Albania, Cyprus, Egypt, Canada, Germany, Tunisia

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