Stimulating the development of medicines for children
EMA’s revision of class waiver list expected to boost exploration of medicines for children
The European Medicines Agency’s Paediatric Committee (PDCO) has revised the current list of class waivers for medicines that are not required to submit a paediatric investigation plan (PIP).
Said to be the most extensive revisions to date, their aim is to balance better the need to support development of paediatric medicines while avoiding exposing children to unnecessary studies. EMA hopes the revisions will encourage companies to develop more new medicines for childrens' use.
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 offered 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 and confirmed nine in the current class waiver list.
Companies developing medicines 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 have had 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.
As it is not always appropriate to develop certain medicines in children, the PDCO can waive the requirement to develop a specific medicine (product-specific waiver) or classes of medicines (class waiver) in children. Waivers aim to avoid unnecessary studies in children where the medicine is likely to be ineffective or unsafe, where the medicine does not represent a significant therapeutic benefit over existing treatments in children, or when the medicine is intended to treat a condition that only occurs in adults. When the intended use is covered by a class waiver, the developer is not required to submit an application for a medicine to the PDCO and so the PDCO cannot evaluate the potential of the medicine for children.
For further information see:
www.ema.europa.eu/docs/en_GB/document_library/Other/2015/07/WC500190384.pdf
www.ema.europa.eu/docs/en_GB/document_library/Other/2015/07/WC500190385.pdf
