Medicinal products for human use
Medicines for Children
New legislation governing the development and authorisation of medicines for paediatric use - Regulation (EC) N° 1901/2006 - entered into force in the European Union on 26 January 2007.
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.
What are the key measures ?
The key measures included in the EU Regulation are:
- 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 maximise the impact of existing studies on medicines for children, including a public database of paediatric studies;
- 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 EMA;
- a provision on EU funding into research leading to the development and authorisation of off-patent medicines for children.
Background: Why was an EU regulation on medicines for children necessary ?
The Regulation was seen as a response to the absence of sufficient numbers of suitable, authorised medicinal products to treat conditions in children in the European Union. Studies had shown that at that time over 50 % of the medicines used for children might not have been tested for use in this specific age group. The lack of suitable, authorised medicinal products to treat conditions in children can be explained by the fact that pharmaceutical companies frequently did not carry out the necessary research and development to adapt medicinal products to the needs of the paediatric population. This left healthcare professionals with no alternative but to use off-label products and unauthorised products with the associated risks of inefficacy or adverse reactions.
Reports on the application of the Paediatric Regulation
In June 2013 the Commission presented to the European Parliament and the Council a progress report on the Paediatric Regulation covering the five years of its application . In accordance with Article 50(2) of the Regulation this report includes an inventory of medicinal products authorised for paediatric use since its entry into force.
In preparation of this report the Commission released a public consultation document (67 KB). A summary of the replies ishere.
The Consultation builds on the ‘Five-year Report to the European Commission’ drafted by the European Medicines Agency with its Paediatric Committee.
Additionally, the Commission publishes on an annual basis a report on companies and products that have benefited from any of the rewards and incentives of the Regulation and companies that have failed to comply with obligations of the Regulation: