Assistance with paediatric drug development

Legislation requiring pharmaceutical companies to consider the potential therapeutic benefit of their drugs in children has been introduced in both the US and EU.

The FDA asks for a paediatric plan at the stage of submission of an NDA or BLA for adult treatment, however not pursuing paediatric development will not be an obstacle for adult approval.

In contrast, EMA will not validate a submission for MAA without a PIP, a Paediatric Investigation Plan, having been accepted. Not submitting a PIP in time can thus become a road block for a drugs approval in Europe.

In Europe, the PIP should be submitted at the end of Phase 1, and it usually takes a year until its approval by the PDCO. Some diseases are exempt from paediatric development since they do not exist in children. A list of class waivers can be found on the EMA website.