These guidelines outline the scenarios and relevant procedures for applicants wishing to submit a UK Paediatric Investigation Plan (PIP), request for modification and/or waiver. There is also information on how to submit completed studies for processing and assessment. Read more “Latest guidance on conducting UK paediatric studies after Brexit”
The European Commission (EC) has published results from an evaluation of the EU orphan and paediatric regulations. Read more “The impact of the orphan and paediatric regulations”
The new guideline outlines the required format and content of applications for agreement or modification of a Paediatric Investigation Plan (PIP) and requests for waivers or deferrals. Read more “The MHRA have published new guidance on Paediatric Investigation Plans (PIP), requests for waivers, deferrals and compliance in the event of a no-deal Brexit”
The European Network of Paediatric Research at the European Medicines Agency (Enpr-EMA) has invited relevant stakeholders, and anyone involved in the preparation and conduct of paediatric clinical trials, to send comments on the draft framework released about paediatric clinical trial preparedness. Read more “Did you know that the Enpr-EMA has published a draft framework regarding paediatric clinical trial preparedness?”
Draft guidance has been released by the US Food and Drug Administration (FDA) which could help to accelerate the development of drugs for treating paediatric rare diseases. The draft guidance could eradicate the need for certain clinical studies and, when paediatric clinical studies are required, could decrease the total number of patients who would receive a placebo rather than a potentially beneficial drug. Read more “FDA releases draft guidance to facilitate drug development in paediatric rare diseases”
The European Medicines Agency (EMA) has decided to give children and adolescents a voice in some activities of scientific committees and working parties.
On 3rd July 2017 in a joint proposal, the European Medicines Agency (EMA) and US Food and Drugs Administration (FDA) launched a new strategy aimed at encouraging innovative approaches in the development of medicines for rare diseases in children.