Due to the progress of the situation related to the new coronavirus infection and the declaration of the pandemic status by the WHO, the management team analysed the practicability to organise the EPTRI Open Meeting on the 2nd and 3rd of April 2020 in Brussels. To guarantee the safely participation of everybody and the contribution to this crucial meeting, the EPTRI Open Meeting will be perfomed remotely. Details for the connection will be shared soon. The event will be the occasion to sum up the activities and the goals achieved as well as to discuss the challenges of the future EPTRI’s work. The first part of the meeting will be focused on an overview of the activities carried out so far in the project, together with a description of the future EPTRI architecture in terms of governance model, IT structure, services and the design of the future platforms. An open meeting will take place in the second day with the worthwhile presentations of the abstracts selected through the call for abstracts launched on the 5th of February, on several topics related to the four thematic research areas of EPTRI. All the posters selected by the EPTRI Scientific Committee wil be presented as teaser videos of 3 minutes and will be published on the EPTRI website. All the oral presentations will be available on the website as videos and some of them will be also presented during the Open Meeting morning session. A dedicated session on the advocacy and the importance of the networking and outreach is foreseen in the afternoon of the second day. Moreover, the special issue “Scientific Highlights in the First European Paediatric Translational Research Infrastructure Open Meeting” has been created on Pharmaceutics, an Open Access Journal with a 4.7 impact factor. All the selected abstracts and the EPTRI partners have been invited to submit papers [...]
SAVE THE DATE. EUCROF Webinar: Traceability in Clinical Trials: Challenges of the new EU TMF/eTMF Guideline
The EUCROF Events&Training Working Group, which Chair is our CEO Dr Donato Bonifazi, presents the webinar “Traceability in Clinical Trials: Challenges of the new EU TMF/eTMF Guideline” that will be held on 28 April 2020 (from 10:00 to 11:30 CET). The webinar offers an overview of the EU Guideline on electronic Trial Master File (eTMF) and familiarizes the audience with the important topic of traceability in clinical trials. The webinar encompasses the building of the TMF in the right structure, making it available when necessary and archiving it in such a way that inspectors will be satisfied. It will be presented by Dr Dagmar Chase, GCP Consultant and Trainer at Clinrex, Munich (Germany). It is possible to register until 27 April 2020 at the following link The list of all EUCROF webinars is available here.
Since the terrible stress upon hospitals and ICUs is one of the greatest challenges the disease created, and for now possibly the most crucial point for saving life, once a person gets ill, The State of Israel is establishing a central data center, where medical electronic records (ER) of the COVID-19 patients, and particularly the Intensive Care Units (ICU) ER, shall be gathered. The final aim is to accumulate large amounts of ICU-ER data, enough to allow a selected group of the best researchers, both medical and mathematics/computer science specialists, to find ways to improve and optimize the medical treatment given to COVID-19 patients. For this reason, all the medical centers involved in the fight against COVID-19 are invited to fill in dataset file (that you can download here) and send it to Prof. Pierre Singer ([email protected]), the Director of the ICU of Beilinson Hospital. Prof. Pierre Singer, is a senior member of one of the research groups and can serve as the professional point of contact for additional research interaction.
A pneumonia of unknown cause detected in Wuhan, China was first reported to the World Health Organisation Country Office in China on 31 December 2019. After just three months WHO characterised COVID-19 as pandemic, currently affecting 157 countries and territories around the world. Besides the impact on international financial market and individual social life, COVID-19 emergency is affecting also the field of clinical research and drug development process. Challenges may arise, for example, from quarantines, site closures, travel limitations, interruptions to the supply chain for the investigational product, or other considerations if site personnel or trial subjects become infected with SARS-CoV-2, the virus that causes COVID-19. These challenges may lead to difficulties in conducting the clinical trials and get worse the already challenging conditions of paediatric clinical trials often affected by issues such as lack of patient recruitment and retention. For this reason, on 18 March 2020 FDA released a guidance for industry, investigators and institutional review boards conducting clinical trials during the COVID-19 pandemic. FDA outlines considerations to assist sponsors in assuring the safety of trial participants, maintaining compliance with good clinical practice and minimizing risks to trial integrity. Considerations recommended include, among others, sponsors evaluating alternative methods for assessments, like phone contacts or virtual visits and offering additional safety monitoring for those trial participants who may no longer have access to investigational product or the investigational site. From the European front, EMA and its partners in the European medicines regulatory network are closely monitoring the potential impact of the outbreak of the novel coronavirus disease (COVID-19) for example on pharmaceutical supply chains into the European Union (EU). By the way, no reports of current shortages or supply disruptions of medicines marketed in the [...]
On March 2020, the European Medicines Agency released its annual overview of the involvement of patients and healthcare professionals in the work of the Agency. The report underlined a high percentage (90%) of cases, in which coordinators of scientific advice procedures considered patient involvement as a key action in their decision-making process. Several events have been organised involving the patients and patient organisation such as the “Regulatory Science to 2025: multi-stakeholder workshop” and the “Methotrexate containing medicines- stakeholder meeting”. In particular, this last meeting involved mainly patients representing rheumatoid arthritis, psoriasis and Crohn’s disease groups, healthcare professional specialists and medication error experts and was aimed to discuss new measures to prevent serious and potentially fatal errors with the dosing of methotrexate. Despite the higher number of initiatives increasing awareness of importance of patient engagement, the actions foreseeing the involvement of paediatric patients and children are still rare. Nevertheless, these initiatives could be very useful in overcoming challenges and issues of paediatric clinical trials as lack of patients’ recruitment and retention.
The Italian Ministry of Health presented the new website www.malattierare.gov.it dedicated to rare diseases to collect all the reference points on the Italian territory for rare patients. It is estimated that over one million rare patients live in Italy, while around 325 thousand patients are registered in the National Register of Rare Diseases, which reports the pathologies registered by the LEA (Essential Levels of Assistance), coming from about 90% of the Regions. The new interinstitutional portal on rare diseases is dedicated to patients, their families and all operators of the national health system involved in the world of these pathologies. It is the result of a collaboration between the Ministry of Health and the National Center of Rare Diseases (CNMR) at the Istituto Superiore di Sanità (ISS).
HEPATOMICS: CVBF will contribute to the new project selected by the Cypriot Research Promotion Foundation
The Cypriot Research Promotion Foundation (RPF) selected for funding the project “HEPATOMICS”, aimed to identify novel peripheral blood biomarkers for liver damage in b-thalassaemia through the use of “omics” technologies. β-thalassaemia is a hereditary haemoglobinopathy caused by reduced or absent expression of β-globin. In its most serious form, it leads to severe anaemia and related complications in major organs. Unless treated, it results in childhood death. In the overwhelming majority of patients, treatment is symptomatic rather than curative and involves regular blood transfusions and iron chelation therapy. This project, started in 2019, aims primarily to identify potential non-invasive, novel serum diagnostic biomarkers for different stages of liver disease (fibrosis and cirrhosis) in β-thalassaemia patients using combined data from proteomics, metabolomics and transcriptomics. Apart from CVBF, the project, lasting two years, foresees the involvement of The Cyprus Institute of Neurology and Genetics (CING), coordinator of the project, the Cypriot Ministry of Health and The Royal London Hospital (UK). CVBF will provide its contribution in the investigation of regulatory issues on the management of ‘omics’ data.
SAIL is an observational study to assess the effectiveness, safety profile and real-life prescribing and utilization patterns of a monoclonal antibody in patients with moderate to severe plaque psoriasis in routine clinical practice. Psoriasis affects an estimated 7.8 million adults in Europe and approximately 125 million individuals worldwide, according to the release. Plaque psoriasis appears as red, raised areas of skin covered with flaky white scales that can be itchy and painful. CVBF is the CRO appointed for submission and monitoring activities for 8 Italian clinical sites that will be authorized in the next months.
A new project for CVBF: SINERGY – Toward extracellular vesicle-based therapeutics for the treatment of bronchopulmonary dysplasia
Fondazione Città della Speranza-Onlus awarded the project “SYNERGY - toward extracellular vesicle-based therapeutics for the treatment of bronchopulmonary dysplasia”. The project, started in 2019, aims to fill in the pre-clinical gaps required to allow the clinical application of extracellular vescicles (EVs) for the treatment of Bronchopulmonary dysplasia (BPD). In particular, the project, that lasts two years, intends to focus on the identification of the cellular target(s) of EVs, the identification of the pathways mediating the anti-inflammatory effect of EVs as well as the in vivo validation of the therapeutic procedure. Moreover, the metabolomic analysis for the selection of biomarkers predicting the development of BPD will be performed, to allow an appropriate selection of patients requiring specific intervention. Finally, the preparation of an Investigational Medicinal Product Dossier (IMPD) for submission to the regulatory Authorities is foreseen, paving the way for a first-in-man study. CVBF will provide regulatory support and will lead the preparation of the IMPD. The clinical translation of the project is supported by the production of clinical-grade EVs in a GMP facility.
On 16th and 17th March, 2020 the new project Sharing Good practices for Brain Education in Europe (Share4Brain) was kicked off. In light of the COVID-19 epidemic, the kick-off meeting (KoM) was held by teleconference, in the context of the International Brain Awareness Week (BAW2020) and was aimed to discuss the future activities and the strategies to implement the two-years project. “Share4Brain” is an Erasmus+ project, involving organisations and institutions 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). It aims to follow the example of European countries by uniting patients, caregivers, brain researchers, scientists, clinicians at a national level and by providing them with the skills and knowledge necessary to promote brain research and raise awareness about the societal impact of brain disorders. Representatives of the above groups will meet on multiple occasions during the lifecycle of the project in order to share good practices, exchange perspectives on effective advocacy strategies and learn how to engage with decision-makers at national and EU levels. CVBF will be involved in the implementation of training activities and sharing of good practices through workshops and translational meetings. On October 2021, in occasion of the World Brain Day, CVBF will organise the final project meeting in Bari. A press release of the Kick-off meeting has been prepared and is available here. For more information visit the official Share4brain website.