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Medicines for Children

The Regulation sets up a system of requirements, rewards and incentives together with horizontal measures to ensure that medicines are researched, developed and authorised to meet the therapeutic needs of children.

The key objectives of the Regulation are:

• To ensure high-quality research into the development of medicines for children.
• To ensure, over time, that the majority of medicines used by children are specifically authorised for such use.
• To ensure the availability of high-quality information about medicines used by children.

The Regulation also establishes the Paediatric Committee, which is responsible for providing opinions on the development of medicines for use on children.

Why was an EU regulation on medicines for children necessary?

Before any medicine is authorised for use on adults, the product must undergo extensive testing, including pre-clinical tests and clinical trials to ensure that it is safe, of high quality and effective.

The same procedure may not always be applied to medicines used to treat children, however. Studies have shown that over 50% of the medicines used in children may not have been tested for use in this specific age group. The absence of suitable authorised medicinal products to treat conditions in children results from the fact that pharmaceutical companies frequently do not perform the necessary research and development to adapt medicinal products to the needs of the paediatric population. This leaves health care professionals with no alternative other than to use products "off-label" and to use unauthorised products with the associated risks of inefficacy and/or adverse reactions.

In response to this situation and in order to safeguard the health of children, on 29 September 2004 the European Commission adopted a proposal for a Regulation of the Council and the European Parliament on medicinal products for use in children. Following the co-decision procedure at the European Parliament and Council of Ministers, the Regulation entered into force on 26 January 2007.

What are the key measures?

The key measures included in the EU Regulation are:

• the establishment of an expert paediatric committee within the EMEA;
• a requirement at the time of marketing authorisation applications for new medicines and line-extensions for existing patent-protected medicines for data on the use of the medicine in children resulting from an agreed paediatric investigation plan;
• a system of waivers from the requirement for medicines unlikely to benefit children and a system of deferrals of the timing of the requirement to ensure medicines are tested in children only when it is safe to do so and to prevent the requirements delaying the authorisation of medicines for adults;
• a reward for compliance with the requirement in the form of a six-month extension to the supplementary protection certificate - SPC (in effect, a six-month patent extension on the active moiety);
• for orphan medicines, a reward for compliance with the requirement in the form of an additional two-years of market exclusivity added to the existing ten-years awarded under the EU's Orphan Regulation;
• a new type of marketing authorisation, the PUMA, which allows ten years of data protection for innovation (new studies) on off-patent products;
• measures to increase the robustness of pharmacovigilance and to maximise the impact of existing studies on medicines for children;
• an EU inventory of the therapeutic needs of children to focus the research, development and authorisation of medicines;
• an EU network of investigators and trial centres to conduct the research and development required;
• a system of free scientific advice for the industry, provided by the EMEA;
• a public database of paediatric studies;
• a provision on EU funding into research leading to the development and authorisation of off-patent medicines for children.