Paediatric Research Network

In March 2011 the European Medicines Agency (EMA) and The Organisation for Professionals in Regulatory Affairs (TOPRA) held a workshop with the newly established European Network for Paediatric Research (Enpr-EMA) and industry. The establishment of this initiative dedicated to paediatric medicine was the result of several years' work at European level.

For years, the requirements for paediatric medicines globally have not been addressed by the standard development of medicines for adults. Apart from treatments focused on a specific paediatric population, most have had to manage with cut down adult formulations. The neonates and the very young have been left with more than 90 per cent of their treatments in neonatal units as 'make-do' treatments based on the adult dose, with little paediatric data to support them.

A few years ago, the European Commission made this situation a critical issue for the EU and in December 2006 the Paediatric Regulation (EC) No 1901/2006, came into law. This meant that the EMA had to move quickly, as then the industry was required to provide Paediatric Investigation Plans (PIP) against aggressive timelines. The effect on the EMA was colossal as at times almost everyone at the agency had to provide support for PIPs in their spare time, trying to keep pace with the many applications being submitted.

Before the Paediatric Committee (PDCO) was in place, the EMA had the foresight to establish a working party looking at Paediatric Medicines, led by Daniel Brasseur, then the Head of Committee for Human Medicines, and now the chair of the PDCO. The energy of these paediatric experts and the staff at the EMA has made the Paediatric Regulation a success. More than 1,000 applications have been reviewed since July 2007. The pharmaceutical industry should also be acknowledged as it has invested many hours of work in the generation of the PIPs and, most importantly, has started to advance the development of treatments for children.

From 2008, the PDCO identified that there was something missing in this work, given all the effort that was being invested in paediatrics: the numbers of clinical trials in children were increasing exponentially, but there was no way to determine the priorities and practicalities of the studies. There was a need to share experiences across the EU and the practical aspects of running paediatric studies had to be more clearly understood. The PDCO had experienced paediatricians who could help but there was an even better answer: to join together a network of highly trained, experienced paediatricians who were able to run the studies demanded by the Regulation, within and across the Member States.

Established networks
Studies undertaken across the EU determined that in some countries there were either well established networks or centres of excellence around which a network could be formed. So the PDCO and EMA began working in the background to build on this to establish the Paediatric Research Network. A critical component of this work is the role of the patient organisation, often a key repository of databases of patients and providing awareness of the day-to-day needs of families. It is vital that the involvement of patient groups is expanded.

As a patient representative myself, I know that training families is imperative to ensure that clinical trials are successful. My association, the Jennifer Trust for Spinal Muscular Atrophy, has contributed to the activities of the representative body European Organisation for Rare Disorders (EURORDIS), which has made education of families a high priority for years. In order to be successful in the EU and around the world, the new network needs to work with the US Food and Drug Administration and the World Health Organisation, coordinating effort, sharing experience and providing a natural forum where issues on running paediatric studies can be addressed.

Network launched
In the recent meeting at the EMA, the membership was announced and the criteria for being in the network were outlined. This is a growing group, with over 30 networks and centres having completed self-assessment reports so far, with the expectation that it will reach out to paediatricians across the EU.

the organisations need to fulfil exacting criteria for membership based on the following:

1. Research experience and ability
2. Network organisation and processes
3. Scientific competencies and ability to provide expert advice
4. Quality management
5. Training and educational capacity to build competences
6. Public involvement.

The organisations that join are assessed and join at levels 1-3. A Category 1 member meets all the criteria, Category 2 includes the networks and centres undergoing clarification before membership and those in Category 3 aspire to be able to organise and conduct paediatric clinical trials in line with Good Clinical Practice requirements.

The March meeting at the EMA also established that the new network should be used to help determine if studies being proposed as part of PIP applications are both practical and have adequate numbers of patients available. In addition, the point was made that the network should consider such questions as whether it is critical to have five studies in children running for medicines with the same mechanism of action, for example. In order to be successful, the Network must also interact with the pharmaceutical industry, to help in clinical trial design, to assist patient enrolment and act as an interlocutor between industry, the PDCO and EMA who are required to meet the demands of the Paediatric Regulation.

By drawing together the R&D might of the pharmaceutical industry, the expertise of patient organisations and the clinical experience of the paediatric network with the drive and effort of the PDCO and EMA, the premature baby in its incubator is more likely to receive medication in a manner that has been pharmaceutically designed and tested for use in that child. It is also hoped that the new effort that this brings will help answer the need of many hospitals in obtaining paediatric formulations for the many off-patent medicines that still need to be supported.

The Author
John Irwin is a member of the Jennifer Trust for Spina Muscular Atrophy (part of the EURORDIS Network) and a Fellow of TOPRA.

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