Since the enforcement of the Paediatric Regulation in 2007, there have been several developments in this area. Overall, the preliminary assessment introduced various improvements, like the following statement shows: “better and safer research, more medicines for children on the EU market and more information for parents and health professionals”.

Before the enforcement of the Paediatric Regulation, many products for children were not specifically authorised: more than 50% of medicines for adults were just prescribed in different dosages, without previously being specifically tested.

Key objectives of the Paediatric Regulation (EC) 1901/2006:

  • High-quality research into the development of medicines for children;
  • Over time, the majority of medicines used by children must be specifically authorised for such use with appropriate forms and formulations;
  • The availability of high-quality information about medicines used by children.

Key measures included in the Paediatric Regulation:

  • Establishment of a Paediatric Committee (PDCO): an expert committee, which also includes representatives of the civil society, which represent associations and health professionals. PDCO Members are appointed for a renewable period of three years.
  • A new type of marketing authorisation: Paediatric Use Marketing Authorisation (PUMA), to attract new paediatric indications for off-patent products
  • An EU-inventory of the therapeutic needs of children to focus the research, development and authorisation of medicines
  • A system of free scientific advice for the industry, provided by EMA
  • A public database of paediatric studies
  • A provision on EU funding for research to stimulate the development and authorisation of off-patent medicines for children

To address the problem of paediatric medicines, a system of obligation, rewards and incentives was introduced to support the research and development of medicinal products for paediatric use. Annual reports on the outcomes of this system are prepared by the EMA and then published by the Commission. Thus the Paediatric Regulation has a direct impact on companies’ R&D expenditure: due to the obligations introduced by the Regulation, companies have the duty to screen every new (adult) product for its potential paediatric use.

- Better and safer research

In addition to the Paediatric Committee, the European Network for Paediatric Research at the EMA (Enpr-EMA) was established in 2009 to bring together national and European networks with specific expertise in designing and conducting high quality studies in children. An increase in paediatric trials as such – which was generally seen as a consequence of the Paediatric Regulation – has not yet been observed in the figures of the EudraCT-database. This fact is generally accepted, as the aim should be achieved without subjecting children to unnecessary clinical trials. The report shows that:

  • pharmaceutical companies prepare Paediatric Investigation Plans (PIPs) when developing a new product. By 2012 EMA agreed on 600 PIPs.
  • 33 of these 600 approved PIPs were completed by the end of 2012.

- Additional information on medicines used in children

To address the lack of adequate information, the Regulation requires that companies submit their data on safety of products authorised for use by children. Since 2008, 18.000 study reports on roughly 2200 medicinal products have been submitted, revealing the large amount of existing paediatric information available at company level. The EU-budget covered a part of the operational costs of the Paediatric Regulation. For the period 2007-2012 the EU-budget contribution amounted to more than 39 million Euros.

- More medicines available for children

Since the Regulation came into force 31 out of 152 new medicines were authorized for use by children. By the end of 2011, 72 new paediatirc indications were aproved, and 26 new pharmaceutical forms were authorized for paediatric use.

- Conclusions & Outlook

Despite more than 5 years of experience, the true impact of the Paediatric Regulation will only become apparent over time, as experience is accumulated in the longer term. Benefits and deficits have become apparent and have been monitored, and the Commission intends to fine-tune the current implementation together with the EMA. However, a fundamental change of culture has taken place: undertakings now consider paediatric development to be an integral part of the overall development of a product, and a number of new products with paediatric indication and age-appropriate pharmaceutical forms have been authorised and made available to patients.


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Last modified on July 12 2013.