Draft guidance has been released by the US Food and Drug Administration (FDA) which could help to accelerate the development of drugs for treating paediatric rare diseases. The draft guidance could eradicate the need for certain clinical studies and, when paediatric clinical studies are required, could decrease the total number of patients who would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 Read more “FDA releases draft guidance to facilitate drug development in paediatric rare diseases”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.