Scientific advice in EU for products for treatment of rare diseases

Abstract

The national scientific advice procedure can vary from country to country, with some national regulatory agencies not giving advice at all. Usually it is a face-toface meeting, for which there are no pre-specified dates. It can be requested at any time during the development process, also while concerned studies are in progress. The national procedure timelines are flexible and depend upon a local procedure, and can last from two to four months. The procedure is coordinated by a group of national staff, which often includes CHMP and SAWP members of the Agency, but can involve external experts as well. Essentially, the national scientific advice is a national opinion around issues concerning pre-clinical development, clinical development (trial design, statistical methods, RMP, etc), and quality aspects. Any deviation from the advice has to be justified. The advantages of asking a national scientific advice revolve around the flexibility of the procedure, with the possibility of having face-to-face meetings and ask general questions, as well as addressing specific national issues. On the other hand, timelines for the procedures are very variable, in particular when external experts are required. In addition, the national agency may ask the sponsor to go to CHMP for scientific advice. Finally it is important to consider that the national advice is the opinion from a single country and may diverge from the EU-wide position. In fact, the national advice is perceived as non-binding by some EU regulators.