10/11 October 2023
The European Medicines Agency has published the final Guideline on pharmaceutical development of medicines for paediatric use. The date for coming into effect is 15 February 2014.
On the 26th of January 2007, the "Paediatric Regulation" entered into force (Regulation EC No 1901/2006) with the aim to facilitate the development and accessibility of age-appropriate paediatric medicines. Two things need to be considered achieving this aim:
The new guideline now "intends to strike a balance between predictable and consistent regulatory assessments of paediatric medicines (either generic, innovative, existing or new), the speed of development, industrial feasibility and the need to develop medicinal products that are more appropriate for use in children, rather than continuing the practice of unapproved, pharmacy compounded medicines and off-label use". Principles of the guideline should be considered during the pharmaceutical development of paediatric medicines and for the purpose of paediatric investigation plan (PIP) applications.
The new guideline also links to ICH Q8, saying that the quality target product profile (QTPP) should be established taking into consideration the specific needs of the paediatric population and that critical product quality attributes (CQAs) should be based on that.
Further it defines the expectations regarding the characteristics of the active substance but also those of the excipients. It is recommended that "the selection of the form of the active substance (…) takes into consideration the properties affecting development of paediatric medicinal products".
When choosing the route of administration and dosage form, advantages and disadvantages of a particular paediatric dosage form and a particular route of administration should be discussed and justified. Quite a few different possibilities are described in the document, including modified release preparations.