In the framework of the European Joint Programme on Rare Diseases (EJPRD), TEDDY European Network of Excellence for Paediatric Research is organising the first paediatric expert patients training workshops in collaboration with EURORDIS and the Sant Joan de Déu Research Foundation. The workshop is addressed to patients with a chronic rare disease, aged from 12 to 18, interested in the themes of health, biomedical research, healthcare, and children rights. 15 paediatric patients in Europe will be selected to participate in the workshop by a dedicated Project Committee. The workshop aims at preparing young patients to actively participate in scientific research and play an important role in improving research and healthcare services for all patients by contributing with their own specific experience. The workshop will take place online from November 2021 to February 2022 and will include monthly sessions of two and a half hours. Case studies, practical activities and discussions are foreseen in order to make easier the comprehension of the topics presented. Applications can be sent by October 30th filing in the registration form.
The iCAN groups KIDS Albania and KIDS Bari ,with the support of CVBF and TEDDY Network, have created a brand-new serious game: My Clinical Trial Center (My CTC) to share clinical research learnings. The game in fact aims at explaining to children in a fun way what clinical trials are, how they work and why they are so important to developing drugs suitable for children. It is extremely informative, as game-players will learn about clinical trials, study protocol, informed consent and assent in children, phases and procedures of clinical trials, data collection, and pharmacovigilance. The serious game was launched during the iCAN Summit held in July 2021 and offers the iCAN Seal of Approval as it is created by kids for kids. My Clinical Trial Center is available for download on Android and iOS systems.
Applied Genetic Technologies Corporation has run into safety problems after escalating the dose in its paediatric gene therapy trial. Sixty percent of subjects in the small high-dose cohort suffered suspected unexpected serious adverse reactions (SUSARs) after being treated for a rare eye disease. The study followed a dose escalation and age de-escalation model, giving progressively higher doses first to adults and then to children. AGTC gave the second highest dose to children without incident. After safely dosing adults with the highest dose, which was around threefold higher than the next rung down, AGTC enrolled five kids with total colour blindness to receive the gene therapy. Three of the five paediatric patients who received the highest dose developed severe inflammation around one month after dosing. Investigators saw inflammation in both segments of the eye and needed to perform a second procedure to diagnose or treat the condition with medications delivered to the jellylike fluid inside the eye. A fourth patient developed significant inflammation but didn’t meet the SUSAR definition. Read the entire article here. (Getty Images)
The Share4Brain Project meeting was held on 23-24 of September 2021 at Amarilla hotel in Athens, Greece. Greek policymakers such as MEP Stelios Kymporopoulos, the member of the Hellenic Parliament Mrs Marietta Gianakou, and the mayor of the city of Vouliagmeni Mr Grigoris Konstantellos endorsed the Athens meeting. Partners from Belgium (Belgian Brain Council), Greece (Greek Carers Network EPIONI and National and Kapodistrian University of Athens), Italy (CVBF), Serbia (Serbian Brain Council), and Spain (Spanish Brain Council) shared good practices on brain research and brain awareness. They exchanged perspectives on effective advocacy strategies and learned how to engage with decision-makers at a national and EU level. Share4Brain project will last until the end of May 2022. The organisers of the meeting in Athens chose to have joint sessions with partners from the European programs SESYCARE, linking Partners, and the Belgian Triathlon program implemented by institutions such as KU Leuven. This event of a total of 40 participants from different EU countries and Turkey resulted in the dissemination of the three projects and the creation of conditions for new synergies. The Share4Brain is an Erasmus+ project that unites patients, brain researchers, scientists, clinicians, and family carers at a national level. CVBF is involved in the implementation of training activities and sharing of good practices through workshops and translational meetings.
During the General Assembly meeting of the EOSC Association (EOSC-A) held on August 3rd, CVBF has been included as an Observer of the EOSC. The EOSC Association brings together key stakeholders in the European research environment to agree on strategies for the advancement of Open Science and to optimise the conditions for research outcomes, and ultimately, to make the European Open Science Cloud (EOSC) a reality. Its ambition is to provide European researchers, innovators, companies and citizens with a federated and open multi-disciplinary environment where they can publish, find and re-use data, tools and services for research, innovation and educational purposes. The EOSC is recognised by the Council of the European Union as the pilot action to deepen the new European Research Area (ERA) as well as the science, research and innovation data space which will be fully articulated with the other sectoral data spaces defined in the European strategy for data.
On June 22nd, 2021 CVBF has adopted the Family Audit certification to ensure a family-friendly work environment and cater better for the family-related needs of its employees. Family Audit is an important certification qualifying an organisation’s commitment to a favourable work-life balance of its employees and is a registered trademark owned by the autonomous Province of Trento which is the certifying body. The Family Audit certification is a management tool adopted on a voluntary basis by organisations, including private companies and public and non-profit bodies, that has the purpose of certifying a continuous commitment to a favourable work-life balance. By adopting the Family Audit certification, CVBF has started a cycle of continuous work-life conciliation measures with the introduction of organisational solutions, such as flexible work hours and smart working.
The 1st International Conference on Rare Diseases and Paediatric Research will be held for the first time virtually on 18-19 November 2021 in Athens, Greece! The Conference is organised by the “95” Rare Alliance Greece together with CVBF and ATHENARC - Athena Research &Innovation Information Technologies. The Conference is supported by the European Joint Programme on Rare Diseases (EJPRD), European Paediatric Translational Research Infrastructure (EPTRI), EURORDIS Rare Diseases Europe, European Patients’ Forum (EPF), EUPATI Greece (European Patients’ Academy on Therapeutic Innovation), Greek Patients’ Association and TEDDY (European Network of Excellence for Paediatric Research). The Conference will be an opportunity to be updated of all current developments, be an active participant and contribute to this European discussion. The following main topics will be covered: The future of the regulatory framework. Supporting tools and funding opportunities in Rare Diseases (RD) and Pediatric Research (PR). Rare and pediatrics diseases during COVID-19 pandemic. Innovative technologies and health data. RD and Paediatric Research: The role of Networks and Infrastructures. To register and have more information visit the dedicated website.
To increase the global scientific and technological capacity in modelling and simulation (M&S), CVBF is organising a workshop named “Modelling&Simulation: Research Methodologies for Small Populations in Rare Diseases”. The workshop aims at putting forward a cross-cutting topic while targeting a specific area of improvement, i.e. the application of innovative methodologies (use of PK-PD data for M&S and extrapolation principle application) for RD drug development. It is organised within the EJPRD ERN Research Workshop framework and will be held in Spring 2022. The workshop is addressed to ERN’s affiliated PhD students, post-doc researchers, senior scientists, young clinicians, investigators and academics. It will be an opportunity to outline and disseminate the benefits that M&S innovative research methodology can produce especially in specific and limited populations. The training methodology intended to provide concrete research skills will be based on lectures, seminars, and practical sessions organised in two days. More information will be available in the following months.
CVBF is Partner in many research networks at national and European level, aimed to promote the interaction and the active involvement of the main stakeholders facilitating the transfer of the achieved know-how at different levels, leading to a strengthening of the scientific excellence promoting national and international research. Research networks conceived within CVBF are the TEDDY Network, which was developed under the Sixth Framework Programme for Research and Technological Development with the goal of supporting and strengthening the scientific and technological excellence in the paediatric sector in Europe and the Inter-regional Network for Thalassemia. At the end of the funding period, the Task-force was further renewed and now TEDDY is an independent multidisciplinary and multinational Network, where researchers, scientists and health experts are still working together to identify the most appropriate common rules that reflect the specificity of this patient population. CVBF is partner in EUCROF (European CRO Federation), a non-profit organization founded in 2005 with the aim to promote Clinical Research by improving the knowledge, competences, expertise and skills of Contract Research Organisations (CROs) in Europe. CVBF and its Albanian branch actively participate in the EUCROF activities and in particular in the following Working Groups: Communication, Events and Training, Outsourcing, Paediatric, Patients. INCiPiT is a network coordinated by Ospedale Pediatrico Bambino Gesù IRCCS and composed by the main Italian Paediatric Hospitals and Scientific Institutes for Research, Hospitalization and Health Care (IRCCS) and other Research Organizations like CVBF, with the aim to promote and support the conduction of all types of clinical trials in the paediatric population by all kinds of sponsor (academic investigators, pharmaceutical industries, contract research organisations). CVBF is member also of AICRO – Associazione Italiana Contract Research Organisation, founded in 2004 with the aim to ensure quality [...]
We are happy to inform you that during the iCAN Summit in July 2021, iCAN launched a new learning module for clinical research, located on the iCAN website and freely accessible through this link. The new module was prepared with the support from the iCAN clinical research advisory team of community and industry leaders and the iCAN kids as they shared their voice to review materials and to produce a video series aimed at teaching kids how to better understand paediatric clinical trials. With this regard, KIDS Albania and KIDS Bari created, with the support of TEDDY and CVBF, a brand-new serious game: My Clinical Trial Center (My CTC) to further share clinical research learnings. The serious game was in fact launched during the iCAN Summit and offers the iCAN Seal of Approval as it is created by kids for kids. We hope you download this free game today which is available on both Android and iOS.