Peter Arlett from the European Commission proposing the new initiatives for children. BEN J. M. Verbeek/EFGCP

The European Union has taken one step closer to launching US-style regulations that encourage the development of drugs designed specifically for use in children.

Leading figures from industry, clinical practice and regulatory affairs met in Brussels on 25–26 January under the auspices of the European Forum for Good Clinical Practice. A final decision by Europe's lawmakers might be more than a year away, but to judge by the talk inside and outside the conference hall, it is not a question of whether the European Parliament and Council will adopt the proposals, but what happens after it does.

Under the proposals, explained Peter Arlett from the European Commission, companies seeking approval for new medicines will either have to go to a new European Paediatrics Committee of the European Medicines Agency with data from paediatric clinical trials, or ask the committee to grant a waiver or a deferral. In return for completing studies, the makers of patented medicines will gain a 6-month 'patent extension' in Europe, rising to an extra 2 years for drugs for rare, or orphan, diseases.

So far, so pretty much a copy of the regulations introduced into the US in 1997 and confirmed by Congress in the Best Pharmaceuticals for Children Act of 2002. Things get more complex with generic, or off-patent drugs, and the European Commission has had to be a bit more innovative.

The Commission has come up with a new vehicle for granting intellectual property, a Paediatric Use Marketing Authorisation, or PUMA. This will allow innovation on older drugs to be rewarded with the intellectual property right — data protection — which gives an element of market exclusivity.

Questions remain about how effective in practice the provisions for older medicines will be in encouraging drug companies to produce medicines for children. Experience in the US since 1997 looks encouraging, said Diane Murphy, Director of the FDA's Office of Pediatric Therapeutics. Up to December 2004 the authorization procedure had led to 17 product labels carrying new dosing recommendations, 21 with new children-specific label information, and 11 with declarations that effectiveness in children had not been established.

Expertise in interpreting data will be a big issue. According to some of the attendants, it is estimated that there are no more than 12 trained paediatric clinical pharmacologists in Europe. As this expertise is mainly limited to the academic/clinical sector, industry is going to have to rely on this sector for expertise — which seems to echo the experience in the US.

Another stumbling block will be informed consent. There are already guidelines for informed consent from both parents and from the child when the child is capable of understanding what is going on. But there are a huge number of tricky areas, including situations involving divorced parents and sperm-donor parents.

Despite a small, nagging worry that some of Europe's MEPs might baulk at the very idea of clinical trials involving babies and children, there's a lot of confidence that most can be won over. And there is general agreement that the proposals would be a good start for Europe even if they will still leave the continent's researchers and companies nearly 10 years behind the US.

That said, the proposal for regulation in Europe has the advantage of drawing on US experience and has been able to plug the gaps that some consider to exist in the US laws.