The Medicines Authority in collaboration with the European Medicines Agency (EMA) has an active role in ensuring that the quality, efficacy and safety of medicines used for children across Europe.
In the past medicines authorised for children were very few and adequate dosing was not studied causing great difficulty in prescribing and giving rise to dosing errors. The absence of suitable authorised medicinal products to treat conditions in children has been an issue of concern for some time. Hence, the European Union's Paediatric Regulation 1901/2006/EC came into force in January 2007 to allow the EMA to stimulate research into the use of medicines in children and thus improve the health of children in Europe without subjecting children to unnecessary clinical trials.
The regulation established the Paediatric Committee (PDCO) which coordinates all the paediatric work of the agency. The agency offers free scientific advice for paediatric development questions.
All applications for marketing authorisations for new medicines that were not authorised in the EU before 26 January 2007 have to include the results of studies carried out in children of different ages. The PDCO determines what these studies must be and describes these in paediatric investigation plans (PIPs).
This requirement also applies when a company wants to add a new indication, pharmaceutical form or route of administration for a medicine that is already authorised and patented. Once authorisation is obtained in all member states and the study results are included in the product information, even when negative, the medicine is eligible for six months' supplementary protection certificate (SPC) extension.
The PDCO grants deferrals for some medicines, allowing a company to delay development of the medicine in children until there is enough information to demonstrate its effectiveness and safety in adults. The PDCO also grants waivers when development of a medicine in children is not needed or is not appropriate, such as for diseases that only affect the elderly population.
A list of opinions and decisions on PIPs and waivers is available on the EMA website.
By being represented on the paediatric committee of the EMA, the medicines authority takes part as Rapporteur or Peer Reviewer in the Paediatric Investigation Plans which are required for all new medicines which apply for authorisation. The task involved is to ensure that medicines are developed appropriately for children by today's European standards by review of the medical literature.
Further by participation as Rapporteur in Paediatric Work sharing procedures (PDWS) (according to Article 45 of the Paediatric Regulation), the Medicines Authority reviews the medical literature available as well as any paediatric studies already completed (prior to 26 January 2008) to update the information for medicines used in children as required by today's standards.
Medicines that are developed specifically for children and are already authorised but are not patented can receive a paediatric-use marketing authorisation (PUMA). These medicines benefit from 10 years of data/market protection as an incentive as long as they containt the results from an agreed PIP.
The EMA has drawn up a list of off-patent medicines that are priorities for development as medicines for children. Funding from the EU is available for studies into these medicines.
The EMA maintains a database of studies carried out in children that were complete before the Paediatric Regulation came into force in 2007. For more information please visit this link.