Scientific advice in EU for products for treatment of rare diseases

Scientific advice constitutes an important part of the development strategy as it can increase the likelihood of regulatory success. For this reason, its timing has to be carefully planned.

There are essentially two routes which can be followed to get scientific advice in the EU:

a “national advice”, which is a decentralised procedure and involves meeting with one national authority;

a “scientific advice” at the CHMP level, which is centralised, called protocol assistance for designated orphan drugs. This is a pan-European advice, which is adopted by the CHMP based on the recommedations of the SAWP.