Medicinal products for paediatric use

KEY POINTS

Pharmaceutical companies must work with the European Medicines Agency — set up under Regulation (EC) No 726/2004 (see summary) — to draw up and agree on paediatric investigation plans whose aim is to collect clinical data on the use of a medicine in children. The results of the paediatric investigation plan have to be submitted when applying for authorisation to market the medicinal products. In exchange, and as a reward, a company receives a 6-month extension of its supplementary protection certificate — a form of intellectual property right. For orphan medicines, a manufacturer has an extra 2 years of market exclusivity.

An independent paediatric committee inside the European Medicines Agency, advises on questions raised regarding children’s medicines and is responsible for the scientific assessment and approval of the paediatric investigation plans.

The regulation introduces:

• an EU inventory of the therapeutic needs of children that focuses the research, development and authorisation of medicines;
• an EU network of investigators and trial centres that carries out research;
• a system of free scientific advice for the industry;
• a public database of paediatric studies.

Amendments to the regulation

Regulation (EU) 2019/5 amends Regulation (EC) No 1901/2006 in order to take into account that the specification of obligations that are subject to financial penalties are now laid down in Regulation (EC) No 726/2004 together with the powers that allow the Commission to lay down procedures for imposing such financial penalties.

Review and evaluation of the regulation

In 2017, the European Commission published a review of the 10 years of the regulation’s operation. It reports that:

• the regulation had an important impact on the development of paediatric medicines in the EU;
• considerable progress was seen in the availability of medicines for children in certain therapeutic fields, particularly in the fields of rheumatology and infectious diseases;
• from 2007-2016, over 260 new paediatric medicines were authorised and the number of agreed paediatric investigation plans increased significantly;
• in the area of research, there have been positive results, particularly in areas where the needs of adult and paediatric patients overlap, although in diseases that are rare and/or unique to children, and which, in many cases, are equally supported through Regulation (EC) No 141/2000 on orphan drugs (see summary), major therapeutic advances often failed to materialise.

An evaluation of the paediatric regulation, along with the orphan drugs regulation, took place in 2019 and 2020. The evaluation involved several steps, including the publication of a roadmap, studies on paediatric medicines and on medicines for orphan diseases, and a wide consultation of interested parties. Following the results of this evaluation, an impact assessment for a revision of these two regulations has been launched.