We are happy to announce that even this year and with an ongoing pandemic, the youths of KIDS Bari have kicked off their activities[ML1] ! On November 27, KIDS BARI went back to work to promote the involvement of young persons and patients in the research and clinical...
The European Joint Programme on Rare Diseases (EJP RD) has launched the EJP RD Joint Transnational Call (JTC) 2021 and will fund multilateral research projects on rare diseases under the EJP-COFUND action. EJP RD has successfully implemented two Joint Transnational...
During the COVID-19 pandemic, the European Medicines Agency (EMA) is implementing exceptional measures to maximise the transparency of its regulatory activities on treatments and vaccines for COVID-19 that are approved or are under evaluation. EMA is achieving this by...
The European Medicines Agency (EMA) organized a forum on 15 December 2020 to address the following objectives: - inform on implementation of the HMA-EMA Big Data Task Force; priority recommendations; - understand stakeholders’ perspectives; - discuss opportunities for...
“The Innovative Medicines Initiative is making a difference to children living with paediatric diseases. Children represent 20% of the EU population and yet many of them live with significant unmet medical need. Less than half of all medicines used by children today...
Ivacaftor (Kalydeco, Vertex Pharmaceuticals) is already FDA approved to treat cystic fibrosis in patients aged 6 months and older. The new approval was based on data from ARRIVAL, a phase 3, open-label, safety cohort study of six children with cystic fibrosis aged 4...
In October 2020 the European Medicines Agency (EMA) expressed positive opinion on the adoption and extension of indication for a number of treatments addressed to the paediatric population. This is the case of Dupixent (dupilumab), for which on 15 October 2020, the...
The European Paediatric Formulation Initiative (EuPFI) works to improve the preparation of better and safer medicines for children. One area they are interested in is the “gadgets” or devices (e.g. measuring spoons, dosing cups, inhalers) that are used to help...
The first General Assembly of the EPTRI (European Paediatric Translational Research Infrastructure) Italian Node (EPTRI-IT) was held online on October 28th, 2020. The 3 hours meeting was attended by more than 60 representatives coming from the 24 Italian Institutions...
On 31 August 2020, the Enpr-EMA (European Network of Paediatric Research at the European Medicines Agency) working group on trial preparedness published recommendations on preparedness of medicines’ clinical trials in paediatrics. Enpr-EMA held a two-month public...