European Commission’s Proposal for a Regulation on Medicinal Products for Paediatric Use

No parent wants to see their children suffer from serious diseases for which there is no cure. Yet no parent wants to see their child being used as a “guinea pig” in uncontrolled biomedical research. Without research children will continue to suffer, yet research undertaken on children must respect and protect their vulnerability.

Parents of children with serious and life threatening diseases are uniquely able to perceive the needs of their offspring for treatment, and their views and voices have a unique authority in this debate. If parents have no confidence in the protection afforded to their children then consent will be withheld and research will become impossible.

The proposed European Paediatric Regulation

As CF-patients, affected by a chronic serious and life threatening disease, we welcome the proposal to introduce paediatric regulations for the development of medicines for children in Europe. We strongly endorse the intention to produce safe and effective medicines for children’s use.  When considering the proposed regulation we ask MEPs, the Commission and Member States’ governments for their support on the following 10 points:

1. Many childhood diseases are incurable or intractable. Research and the development of safe new medicines is urgently needed. Whilst safety is important, non-intervention in childhood diseases is not without cost.
   
    

2. Most new medicines are initially developed for adult forms of diseases that also affect children. The need for safe paediatric formulations should not slow access for sick adults to potentially life saving therapies. The needs of adults and children should not be seen as tradable.
    

3. Procedures for developing paediatric versions of adult medicines should not act as a disincentive to the development of therapies for conditions which only affect children (too complicated).
    

4. We strongly support the proposed scheme of incentives as well as obligations for pharmaceutical companies to encourage the development of safe medicines for children.
    

5. We urge that the tasks of the Paediatric Committee be clearly focused on the efficient and effective implementation of these new procedures to avoid causing administrative delays in bringing medicines to children.
    

6. The proposed list of therapeutic needs for children should already be drawn up by the Commission, EU and national experts, and not only within 3 years of the Regulation entering into force. The expertise of the Paediatric Committee is then better deployed identifying research priorities from that list.
    

7. We are concerned that the proposal does not sufficiently address incentives and funding for Clinical Trials in children for those working in non-commercial settings such as universities and hospitals.
    

8. We welcome the creation of European networks with specific expertise in Clinical Trials in the paediatric population and strongly urge that these be linked to and funded by the 6^th and 7^th Research Framework Programmes.
    

9. We regret that the paediatric study programme NICE is only mentioned as a commitment of intention and left largely undefined. We believe that a comprehensive plan to improve paediatric research in Europe must be drawn up now and not left to future legislation. 
    

10. Fortunately, many of the serious diseases that affect children in the Europe are individually rare. However there are thousands of these rare conditions, representing a huge toll on families and health care systems. Encouraging innovation and safe paediatric medicines through the development of an appropriate regulatory and incentive regime is a key challenge for the future health and well-being of millions of Europe’s children.

21 May 2005
Danish Cystic Fibrosis Association