In March 2019, the Committee for Medicinal Product for Human Use (CHMP) on the basis of the draft Committee for Advanced Therapies opinion, adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Zynteglo, intended for the treatment of transfusion-dependent b-thalassemia (TDT). Since b-thalassaemia is a rare disease, Zynteglo was
In April 2019, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Xromi. It is a medicine used in adults, adolescents and children over two years of age who have sickle cell disease, a genetic disease where the red blood
Even this year TEDDY Network supports the Rare Disease Day, the thirteenth edition of the international initiative promoting awareness-raising activities on Rare Disease and that will take place on 29 February 2020. The first Rare Disease Day was celebrated in 2008 on 29 February, a ‘rare’ date that happens only once every four years. Ever
The Events&Training Working Group of EUCROF (European CRO Federation) organised the Webinar “The Patient Centricity Effect - How it benefits Patients, Sites and Sponsors – Practical Cases from Paediatric Studies” that was held on 25 February 2020. Patient centricity has become something of a buzz phrase over the last few years. However, behind the name
In the framework of the European Joint Programme on Rare Diseases (EJP RD) the following call for proposal has been launched: “Demonstration projects on existing statistical methodologies to improve RD clinical trials”. The call aims to show the usability and capability of the innovative statistical methodologies for clinical trials in rare diseases, which have not
The upcoming Clinical Trial Regulation requires the development and dissemination of Lay Summaries of clinical study results from commercial and non-commercial sponsors. However, there is no guidance for all types of sponsors on how best to handle the development process of Lay Summaries and how best to ensure reliable dissemination. To this aim, over 60
On December 2020, the Food and Drug Administration released a draft guidance for industry entitled “FDARA Implementation Guidance for Paediatric Studies of Molecularly Targeted Oncology Drugs”. This draft guidance addresses early planning for paediatric evaluation of certain molecularly targeted oncology drugs, including biological products, for which original new drug applications (NDAs) and biologics license applications
EPTRI (European Paediatric Translational Research Infrastructure) has announced a Call for Abstracts for the EPTRI Open Meeting that will be held on April 2-3 April 2020. EPTRI is an EU funded project coordinated by CVBF and aimed to design the framework for a new infrastructure dedicated to paediatric research that will work to accelerate a
It is possible to capture and communicate the essence of International Children's Advisory Network (iCAN) through the art? Can a drawing explain the importance of the role of iCAN organisation? This was asked to Emanuela Ruscitto, one of the members of KIDS Bari, who succeeded to create a drawing and a video representing the strength,
DIA (Drug Information Association) bring together its members to collaborate on and discuss global and local challenges facing the life sciences field in an increasingly more complex and interconnected world. DIA Europe 2020 is the largest and most forward-looking neutral healthcare conference in Europe. This annual healthcare meeting reflects DIA’s strategic initiatives across several Thought