These are the most extensive revisions to date, and aim to "better balance the need to support development of medicines in children with the goal of avoiding exposing children to unnecessary studies".
PDCO vice chair Dr. Koenraad Norga and Dr. Jordi Llinares, head of product development scientific support at EMA, said that revising the class waiver list would "stimulat[e] research into the use of medicines in children and increase[e] medicines authorised for use in children.
"It is extremely clear, if we are revoking or revising waivers, we are telling the sponsors they have to" develop their products for children."
The revisions follow the PDCO’s review of the experience with class waivers since the Paediatric Regulation came into force in 2007. In its review, it noted that most of the class waivers refer only to medicines targeting specific diseases. This could mean that the potential for the use of the medicines in children more generally is not explored. The PDCO concluded that the current class waiver list resulted in insufficient opportunities for the Committee to consider the potential benefits of some new medicines for children.
As a result, the PDCO has assessed, for all class waivers, the available information on the disease area, the characteristics of the medicines and any available evidence on their possible use in children. It has revoked eight class waivers, updated 15 class waivers and confirmed nine class waivers in the current class waiver list.
Companies developing medicines, which are not covered by the revised list of class waivers, will need to submit a request for a PIP or a product-specific waiver, for scientific review and agreement by the PDCO. This means that, in future, more medicines will be reviewed by the PDCO for potential development for use in children.
The PDCO will continue to revise the class waiver list as more information on medicines and diseases becomes available.
Since the Paediatric Regulation came into force, companies applying for marketing authorisation for new medicines need to submit early in the development of the medicine a plan (known as a PIP) that describes how the medicines should be studied in children.
The PDCO assesses and agrees the content of the PIP to ensure that the necessary data are obtained through studies in children, when it is safe to do so, to support the authorisation of medicines for children. Companies can only apply for a marketing authorisation for their medicine if they can demonstrate that they have conducted studies in accordance with the plan, unless they have agreed with PDCO to defer these studies or the obligations have been waived.