Children, Drugs and the EU Paediatric Initiative (article)

Did you know, however, that until now at least 50% of medicines used to treat children have never been tested in children but only in adults? Children (particularly those 0-28 days old i.e. neonates) respond to drugs in a very different way to adults. Typically, doses in children are calculated by weight and occasionally by body surface area with the dose never exceeding the maximum adult dose. This is only a guide as, for example, sometimes children require a higher dose per kilogram than adults because of their higher metabolic rates. To say that a child is half the size of an adult and therefore should receive half the adult dose is too simplistic and could be dangerous.

Many marketed medicines are used "unlicensed" or "off-label" for the treatment of children i.e. they have a licence for use in adults but children are not included within this licence. The lack of supporting data for the treatment of children has been a cause of concern throughout the EU for several years and is believed to have contributed to an unacceptable number of adverse reactions. As a result of this, and the overall need to improve on the information available, the European Paediatric Initiative came into being with a new EU Paediatric Regulation¹ entered into force on 26 January 2007. This new regulation has several implications with the overall purpose being to provide "better medicines for children"².

There are incentives for pharmaceutical companies to comply with this new regulation. If a new medicine is developed for use in both adults and children, the company is eligible for a 6-month extension of its patent protection. For those companies developing a medicine specifically for the treatment of children, they will benefit from 10 years of data protection. The latter is a new kind of licence called a PUMA (Paediatric-use Marketing Authorisation). In both cases, the drug development must follow an agreed Paediatric Investigation Plan (PIP).

For many companies the Paediatric Regulation is a completely new challenge and, up until recently, they will not have incorporated it into their business strategy. With ever decreasing headcount, it is becoming more and more difficult to keep up-to-speed with all the latest legislation and the implications of enforcement. Consultancies, such as Global Regulatory Services, can help companies by offering the services of senior regulatory professionals who already have working knowledge and experience of the Paediatric Regulation. With the pharmaceutical industry, the Health Authorities and regulatory professionals working together we can have a positive effect on the development, availability and safety of medicines for the treatment of children and improve the availability of information on the use of these medicines. This new legislation may be challenging and time consuming but, most importantly, will provide a safer future for our children.

¹ Regulation (EC) No 1901/2006 of the European Parliament and of the Council on medicinal products for paediatric use, amended by Regulation (EC) No 1902/2006.
² Source: EMEA

Author: Greer Deal, Director of Global Regulatory Services