There are still many health conditions which only affect the paediatric population, this would be the case for 80% of rare diseases that lack approved therapeutic options. Clinical trials are essential for the development of new medicines. In the last years, in Europe the number of clinical trials for the paediatric population has increased. At
The Spanish Data Protection Authority published, on last February, the "Code of Conduct on the processing of personal data in the field of clinical trials and other clinical research as well as pharmacovigilance" (CoC). The CoC regulates how data protection rules impact the development of clinical trials and compliance with pharmacovigilance obligations. Regarding clinical trials,
The European post-authorisation study (EU PAS) register is a repository launched in 2010 by the European Medicines Agency. Multi-database studies (MDS) leveraging secondary data have become an important strategy to conduct post-authorization studies (PAS) in recent years. The “Overview of the European post-authorisation study registerpost-authorization studies performed in Europe fromSeptember 2010 to December 2018”, provides
Last February 28th we celebrated Rare Disease Day and Kids Bari, the first Young Persons Advisory Group (YPAG) in Italy, joined the #shareyourcolours campaign by making an amazing video showing their Special Colours. “These Kids and Teens are not defined by their disease, they are not numbers... they are amazing and stunning teens in
As part of the training activities proposed by EJP RD, an online training course on “Training on strategies to foster solutions of undiagnosed rare disease cases” is being organised by Istituto Superiore di Sanità (ISS) in close collaboration with EJP RD partners. The online event will take place over three days from on April 11th – 13th. Through the presentation of sample use cases that
The Therapies Scientific Committee (TSC) of the International Rare Diseases Research Consortium (IRDiRC) is establishing a Task Force to characterize specific commonalities amongst a large group of “disregarded” rare diseases, with the potential secondary aims to identify removable roadblocks that may foster future research and development. The TSC is specifically looking for members to populate the Task Force with the below expertise in one
IRDiRC's Therapies Scientific Committee (TSC) is establishing a Task Force on Drug Repurposing Guidebook aimed at helping developers identify specific tools and practices of relevance for repurposing projects, by focusing on repurposing approaches, following the same successful methodology used for the Orphan Drug Development Guidebook. Interested candidates can submit their application (CV, biosketch and letter of motivation, one paragraph each) to the Scientific Secretariat.
The International Rare Diseases Research Consortium (IRDiRC) is proud to announce the publication of a commentary on 10 years of progress and challenges of IRDiRC in the prestigious journal Nature Reviews Drug Discovery. The article summarizes IRDiRC’s vision and goals and highlights its achievements and prospects after its first decade (2011–2021). The commentary has been authored by Lucia Monaco (Consortium Assembly
The webinar ‘ADDRESSING THE CHALLENGES OF CONCOMITANT DEPLOYMENT OF COVID-19 AND INFLUENZA VACCINATION’ is organised by Active Citizenship Network(ACN) and will be held on 17th December 2021 at 11.00 CET. TEDDY Network will be represented by Prof. Carlo Giaquinto who will have a presentation during the roundtable with healthcare professionals. This multi-stakeholder event (civil society,
EPTRI is organizing the second webinar on medical devices: ‘Health technology designed for children with children’ that will be held on 16 December 2021 at 15.00 CET. Register here. Versatility is key to the development of health technology for children and young people so that it is adaptive to their social, physical and developmental needs. Co-design and development of
