On September 29, the Commission announced that it had adopted its Proposal for a Regulation on Medicinal Products for Paediatric Use, which comes several years after the US launched its own initiative in this area.
However, the US effort has been criticised for giving pharmaceutical companies a route to gaining extra patent protection for their drugs - even if their use in children is questionable. The EC maintains that its initiative would avoid this while still rewarding companies for demonstrating safety and effectiveness in children.
The proposal, entitled Medicines for children: a new approach, is addressing the alarming situation at present in which over half of the medicines used for children in the European Union have never been tested or authorised for their use.
The European Commissioner responsible for Enterprise and the Information Society, Olli Rehn, said this means that children are denied innovative treatments and may be given ineffective or harmful drugs.
The reasons behind the paucity of drugs developed for paediatric use are complex - for example studies in children can be problematic to conduct - but what is clear is that the pharmaceutical industry has shown little inclination to pursue the development of drugs for children. Perhaps this is because the market is small compared to treatments for adults, he suggested.
Rehn said he believed the initiative achieves the right balance between promoting the availability of new and better medicines specifically designed for children and the need to strengthen the competitiveness of the pharmaceutical industry.
In essence, the new proposals - in development since 2000 - set out a hybrid regulatory environment that combines the situation in the US with the EU's existing legislation on 'orphan drugs', i.e. those developed for diseases that only affect a small number of patients.
The core objective of the initiative is to improve the health of European children by stimulating research into new medicines for children, promoting the development and authorisation of these drugs, and improving the information available on medicines used in children.
But this has to be achieved without requiring unnecessary studies in children and without delaying the authorisation of medicines for adults.
To achieve this, the Commission proposes the implementation of a series of key measures for patented and off-patent medicines, plus a range of other initiatives such as the setting-up of an expert paediatric committee at the European Medicines Agency (EMEA).
As in the US, the proposed system envisages a reward system for companies carrying out paediatric studies, but this will sit alongside an obligation in some cases.
The requirement is that, at the time of applications for marketing authorisations for new medicines, the results of studies in children should be conducted according to an agreed plan. This requirement will also apply to marketed medicines, which are still under patent when companies apply to extend their use by, for example, using them at a different strength.
However, not all medicines developed for adults will be useful for children. For example, some adult diseases do not occur in children and, in this situation, there will be a waiver from the paediatric studies requirement.
In some cases it may be safer to study some medicines in children only after trials have been conducted in adults, so there will be a provision to defer the timing of starting or finishing the paediatric studies. Deferrals will also ensure that this requirement does not delay the authorisation of medicines for adults, said Rehn.
In common with the US situation, the reward will be a six-month extension of the Supplementary Protection Certificate, thus extending the patent for this period.
The situation will be different for orphan medicines, said Commissioner Rehn, given that not all such products are patented and that they also already receive 10 years' market exclusivity. Their "reward, " therefore, will be two further years' market exclusivity in addition to the decade that they already have.
Generics get PUMA route
For off-patent products, the story is a little different, and much more difficult. These products have been on the market for many years and are usually made by multiple manufacturers, including generics companies and, while such products have no intellectual property rights, they may be of great therapeutic value to children, he said.
The Commission's proposed solution is the establishment of a new Paediatric Use Marketing Authorisation to cater for new, off-patent medicines that have been developed specifically for children. The PUMA will allow intellectual property rights to be awarded, thus providing an incentive to companies to undertake the necessary studies.
Newly developed but off-patent products to be granted 10 years' data protection on any newly conducted studies. The PUMA route will also allow the use of the existing brand name.
In terms of data requirements, PUMA will for the first time allow a generic-type application to be combined with the new paediatric studies, which should make it easier for industry to develop these medicines, according to Rehn.
Turning to the role of the new committee, Rehn said that it would be charged with scrutinising manufacturers' proposals for testing medicines in children, to ensure that research is of high quality and that it is conducted only when it will be of benefit. It will draw on a network of European experts in children's medicines.
Importantly for the industry and, particularly, for small and medium-sized enterprises, the EMA will provide manufacturers with scientific advice, free of charge, on issues such as how to conduct studies in children.
One of the first measures to be undertaken under the proposal is an inventory of the therapeutic needs of Europe's children to help identify where products are needed, as well as new product labelling requirements to ensure that children, parents and doctors can make informed decisions about treatment.
Another priority will be the establishment of a database of studies in children to avoid duplicating work. There will also be enhanced safety monitoring for marketed products to ensure newly developed medicines are being followed closely once on the market.
European pharmaceutical industry organisations broadly welcomed the Proposal, with the European Federation of Pharmaceutical Industries and Associations describing it as 'an opportunity to improve public health and strengthen Europe's science base'.
However, the Association of the British Pharmaceutical Industry said that the proposals do not go far enough.
"The European Commission is missing an opportunity to encourage clinical trial work in children to be carried out in Europe," said the ABPI in a statement.
Richard Tiner, director of medicine at the ABPI, expressed the group's disappointment that the proposed arrangements did not allow for immediate granting of incentives for paediatric clinical trial work.
This means that work on clinical trials for children is likely to wait until the proposals have completed their journey through the European system - probably about 18 months according to the ABPI - rather than being able to start immediately.