Most drug development has traditionally been focused on adults with little or no clinical evaluation undertaken in children; hence licences for paediatric use are not common and therefore present a large unmet medical need. Although many medicines are administered to children, the majority are not licensed for use in children and are not in an age-appropriate format.
In 2007, the European Regulators initiated a number of developments aimed at providing incentives to companies to license existing off-patent authorised products for use in children. The most significant of these was the PUMA (Paediatric Use Marketing Authorisation) licensing process. Proveca worked with the European Medicine Agency’s Paediatric Committee to gain a PUMA in September 2016 for the symptomatic treatment of severe sialorrhoea in children.
This type of marketing authorisation covers the indication and appropriate formulation for the paediatric population. The development requirements of the medicine for use in children are set out in an agreed PIP (Paediatric Investigational Plan).
Proveca’s paediatric drug pipeline is developed through a structured identification and appraisal process with each candidate being evaluated against a number of key factors to help identify the most appropriate ones for progression through development.
The process includes candidate identification and evaluation (technical, regulatory and market assessment). Appropriate candidates are then taken through the PIP/PUMA process in order to gain European paediatric licenses.
Reviewed November 2021