EMEA Sees Benefits for Children

The recent Pediatric Regulation created a new responsibility for the European Medicines Agency (EMEA) to establish a network of existing networks, centers, and investigators with competence in performing clinical research with children.

As required by law, the EMEA has agreed with stakeholders on a detailed strategy that was published in January 2008. To date, about 50 existing highly diverse networks have been identified. About seven are established at the national level. Many are established in therapeutic areas, with a multinational focus. Some perform research in a specific age group (i.e., neonates). Most are hospital based, which also means that community-based networks need to be developed.

Agnès Saint-Raymond

A first meeting of representatives took place in February 2009. There were generally high expectations from this network, as the need to collaborate is recognized by pediatricians, and in many cases already organized. It was agreed to delegate the initial work to two subgroups. One currently works on the organization and on the organization of the Coordinating Group defined in the Strategy, and the second on establishing the standards that will allow all these networks and centers to work together efficiently.

The main question raised by participants during the February meeting was: What is the added value of the network? This is a legitimate question considering that no funding for research will be available from this initiative.

What is the benefit?

In the view of the EMEA, the first added value is for the patients, as it will facilitate enrollment of children into trials because this brings about benefits to them. Research entails risks and these require prevention and/or minimization. However, research also produces benefits that should be shared by children, as expressed in the ethical principle of distributive justice.

Hopefully, children enrolled in research will reap these benefits directly as well as indirectly, with faster access to new medicines, and better use based on appropriate knowledge on efficacy, safety, and dose, whilst benefiting from better safety, due to increased monitoring in the trials on the one hand, and risk minimization postapproval, on the other. This will be better than receiving untested medicines.

It is also hoped that this pediatric initiative will continue to stimulate the creation of networks, as it already did in the last five years in several member states. This is necessary to include countries or areas where currently few pediatric trials are taking place, and where scientific research in this population is not high or not on the agenda. This will contribute to capacity building across Europe. These are the benefits for researchers and industry.

Sharing research protocols should increase their scientific quality, and trials more likely to answer the scientific question. Sharing competencies and in some cases pooling resources such as audits, should also reduce the cost of research and improve standards. Multinational trials of sufficient size combined with full transparency of these trials as mandated by the Regulation will decrease the number of small trials, which are often unable to reach statistical power, and avoid unnecessary duplication to protect this vulnerable population.

At this stage, this may sound like wishful thinking but the real life examples of successful pediatric networks demonstrate that the benefits are real and achievable in a reasonably short time frame. It only requires collaboration from all stakeholders.

Agnès Saint-Raymond Head of Sector Special Areas EMEA agnes.saint-raymond@emea.europa.eu