The European Medicines Agency plays an important role in the development of medicines for children. In the past, many medicines authorised in Europe were not studied adequately or authorised in children. This caused difficulties for prescribers and pharmacists treating children, as well as for their patients and carers.
To help to solve this problem, the European Union's Paediatric Regulation gave the Agency new responsibilities in 2007. These responsibilities allowed the Agency to stimulate research into the uses of medicines in children and to lead to their authorisation in all ages.
The Paediatric Regulation
The Paediatric Regulation's main aim is to improve the health of children in Europe without subjecting children to unnecessary trials, or delaying the authorisation of medicinal products for use in adults.
The Regulation came into force on 26 January 2007. Its main impact was the establishment of the Paediatric Committee (PDCO), which is responsible for coordinating the Agency's work on medicines for children. The Committee's main role is to determine the studies that companies must carry out on children as part of paediatric investigation plans (PIPs). The PDCO replaced the Agency's previous Paediatric Working Group.
In the European Union (EU), children are defined as people from birth up to 18 years of age.
The European Commission has addressed its general report on experience acquired as a result of the application of Regulation (EC) No 1901/2006 on medicinal products for paediatric use to the European Parliament and the Council. The report concludes that paediatric development has now become a more integral part of the overall development of medicinal products in the EU, with the Regulation working as a major catalyst to improve the situation for young patients. While some areas for improvement have been identified, such as the low uptake of the paediatric-use marketing authorisation (PUMA) by companies, the Agency, together with the Commission, intends to fine-tune the current implementation of the Regulation.
In this general report to the European Parliament and the Council, the European Commission presents an interim analysis of the experience gained using amongst other sources the results of a public consultation on the experience gained with the first five years of the Paediatric Regulation, building on the European Medicines Agency’s five-year report to the European Commission.
More information can also be found in successes of the Paediatric Regulation after five years, recently published by the Agency.
For more information, see:
- General report on experience acquired as a result of the application of Regulation (EC) No 1901/2006 on medicinal products for paediatric use
- Successes of the Paediatric Regulation after five years
- five-year report to the European Commission and public consultation
- History of the Paediatric Regulation
- Paediatric Regulation
Paediatric investigation plans
All applications for marketing authorisation 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.
- 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.
See the searchable list of opinions and decisions on PIPs and waivers.
The Agency checks that companies comply with the agreed studies and measures listed in each PIP. These checks are necessary before the company can apply for a marketing authorisation or a change to an existing marketing authorisation.
PIPs can be modified if new information becomes available during the medicine’s development. Companies need to apply to the PDCO for these modifications.
For more information on applying for a PIP or waiver, see paediatric medicine development.
Incentives for the development of paediatric medicines
Several incentives for the development of medicines for children are available in the EU and in Member States:
- medicines that have been authorised across the EU with the results of PIP studies included in the product information are eligible for an extension of their patent protection by six months. This is the case even when the studies' results are negative;
- for orphan medicines, the incentive is an additional two years of market exclusivity;
- scientific advice and protocol assistance at the Agency are free of charge for questions relating to the development of medicines for children;
- medicines developed specifically for children that are already authorised but are not protected by a patent or supplementary protection certificate, can apply for a paediatric-use marketing authorisation (PUMA). If a PUMA is granted, the product will benefit from 10 years of market protection as an incentive.
The Agency 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.
In 2008, a list of existing national incentives was published by the European Commission:
- Inventory of Community and Member State rewards and incentives to support research into, and the development and availability of, medicinal products for paediatric use
Updates to the national incentives are published in the yearly reports from the Agency to the European Commission:
- 2012 report to the European Commission on rewards and incentives under the Paediatric Regulation
- 2011 report to the European Commission on rewards and incentives under the Paediatric Regulation
- 2010 report to the European Commission on rewards and incentives under the Paediatric Regulation
- 2009 report to the European Commission on rewards and incentives under the Paediatric Regulation
Appropriate medicines for children
The Agency has published a paper summarising the types of medicines that are appropriate for children of different ages. The paper discusses how medicines should be given to children, what their inactive ingredients should be and how taste, smell and texture can have an impact on whether children will take medicines correctly.
For more information, see paediatric formulations.
Seeking the views of children and young people
In September 2012, the Agency launched a public consultation on its concept paper on the involvement of children and young people at the Paediatric Committee.
The paper describes the Agency's plans to develop a framework of interaction describing how the views of children and young people can be consulted as part of the Committee's work.
Working with international partners
The Agency works closely with its international partners on medicines for children:
- it has agreed to share information with the United States Food and Drug Administration (FDA) on scientific and ethical issues related to the development of medicines for children, to foster global development plans;
- it participated in the launch of the World Health Organization (WHO) initiative make medicines child size;
- it is a member of the Paediatric Medicines Regulators' Network (PmRN). This is a network of national medicines regulatory authorities set up by the WHO in 2010, which promotes the quality and availability of medicines for children and contributes to capacity-building.
European Network of Paediatric Research at the European Medicines Agency (Enpr-EMA)
The European Network of Paediatric Research at the European Medicines Agency (Enpr-EMA) is a network of research networks, investigators and centres with recognised expertise in performing clinical studies in children, which is coordinated by the Agency.
It aims to foster high-quality ethical research on quality, safety and efficacy of medicines to be used in children. It does this through networking and stakeholder collaboration with members from within and outside the EU.
Database of studies in children
The Agency maintains a database of studies carried out in children that were completed before the Paediatric Regulation came into force in 2007. The database contains the details of each study, including its title, its aims, the medicines studied and the ages of the patients included:
- Database of paediatric studies submitted in accordance with Article 45 of the Paediatric Regulation
- Submitting paediatric studies
Events and workshops
The Agency regularly hosts events and workshops on medicines for children.
The members of the Agency's paediatric team regularly present topics on the Paediatric Regulation on behalf of the Agency in meetings and workshops organised by third parties:
- Support offered by the European Medicines Agency for the development of paediatric medicines (European Forum for Good Clinical Practice / Drug Information Association (DIA) / European Medicines Agency, September 2011)
- Standards for approval of paediatric medicines (Europaediatrics, June 2011)
- After the PIP decision: Modification of the agreed PIP, validation, compliance check (DIA Euromeeting, March 2011)