Industry and physicians want to use European regulatory framework

 

The new European directive on children's pharmaceuticals, which became effective on January 1, 2007, must be rated as a great success for the pharmaceutical safety of children and adolescents. This is the unanimous sentiment of the German Society for Pediatric and Adolescent Medicine (DGKJ) and the German Association of Research-based Pharmaceutical Companies (VFA). This directive will help children and adolescents obtain more specially approved pharmaceuticals.

Today, both associations appealed to politicians to implement the new directive in Germany systematically and speedily and to create effective and transparent structures for this purpose.

At the workshop "EC Directive on Children's Pharmaceuticals - Prerequisites for a Successful Implementation in Germany," which was organized by both organizations, pediatricians and industry representatives held discussions with experts from the German health ministry, the ethics commissions, self-help groups and parents' organizations as well as politicians and other institutions and initiatives concerned with pharmaceutical studies.

Based on the directive, it will be necessary to conduct considerably more pharmaceutical studies with minors than before. The protection of the little patients, their dignity and benefits must always be ensured in these studies: "During these studies, medical care is always provided based on the latest medical findings and is often more comprehensive than normal therapy can be when it comes to medical history and the diagnostics that accompany therapy. Furthermore, there is an opportunity to obtain a novel and potentially more effective therapy earlier than others," said Prof. Dr. Fred Zepp, spokesperson of PAED-Net, the expert network for the planning and implementation of multi-center studies in pediatrics with six affiliated university clinics. The safety of minors has top priority: If possible, children and adolescents will only receive products that have been successfully tested on adults, but these drugs are naturally only administered in an age-appropriate optimized dose and preparation. The physicians have an obligation to keep the burden for the study participants as low as possible.

The difficult situation of the parents in view of studies involving their children was also clearly addressed in the workshop. Transparent handling of study projects and results is an indispensable trust-building measure to increase the parents' general acceptance and readiness to participate, according to Hermine Nock of the German Association of Children with Cardiac Diseases: "When it comes to studies, parents want to be informed reliably of applications and exclusion criteria as well as negative findings." To give priority to urgently needed pharmaceuticals but also to avoid unnecessary studies, an independent register would have to be established first.

Prof. Dr. Hannsjörg W. Seyberth, chairman of the Commission for Pharmaceutical Safety for Children (KASK) of the DGKJ and founding member of the European Network for Drug Investigation in Children (ENDIC) of the ESDP (European Society for Developmental Pharmacology) viewed the European directive as the long wished-for and indefatigably pursued breakthrough for pharmaceuticals geared more closely to the needs of children. Seyberth stressed that a successful implementation is only possible based on collaboration: "Everybody participating in the development, prescription and application of pediatric pharmaceuticals is now asked to implement this European directive toward children as responsibly as possible. This also includes the ongoing professional training for pediatricians and nursing staff with regard to conducting pharmaceutical studies according to the very comprehensive and elaborate EU guidelines and directives, the development of child-appropriate testing methods with as small a burden as possible and the establishment of study networks with as many pediatric clinics and practices as possible. In addition, we must set up several centers recording undesirable drug effects in children and establish registers that record the entire progress up to adulthood for children, who receive novel pharmaceutical therapy with an unclear long-term risk."

Based on the European directive, all pharmaceutical companies will be obligated from 2007 onward to test all drugs undergoing development also for children and adolescents and to submit them for marketing authorization, if they can be administered to minors. In return, they receive six months of extended protection from copycat products. There is no obligation for already authorized products but incentives to also test them for children and adolescents and submit an authorization extension. "The number of child-appropriate pharmaceuticals must be increased quickly, and we are happy that the European directive creates the right framework in this respect," Prof. Dr. Torsten Strohmeyer, head of research and medicine at GlaxoSmithKline in Munich, said on behalf of the research-based pharmaceutical companies. These companies already conducted pediatric studies in the past to be able to launch about 20 drugs for children each year. However, in the future the extent of these activities will likely increase by more than 100 additional studies in Europe per year. "Many research-based pharmaceutical companies are interested in involving as many German study centers as possible in their multinational pediatric studies," Strohmeyer added. While the company is happy about the pediatricians' willingness to cooperate, capacity problems are already emerging.

"In German academic medicine and clinical research, there are still serious structural and financial problems preventing the university pediatric clinics, which are now particularly challenged, from being provided with the necessary valences for professional clinical pharmaceutical trials," Seyberth confirmed. "New ways must be found to finance the additional work and additional expense that is shouldered by the hospital stations and practices for non-commercial studies not supported by industry. Among others, this is a challenge for hospital sponsors and other sponsoring institutions that should have a strong interest in ensuring high quality in patient-appropriate pharmaceutical care for their patients or members. However, naturally, the medical divisions are also asked to acknowledge the academic achievement associated with the highly responsible work of the medical study director of a clinical trial during the development of a pharmaceutical as well as the basic research commitment at the molecular biology lab."

Together, Seyberth and Strohmeyer appealed to politicians to support the development of the pediatric study infrastructure in Germany. With PAED-Net, Germany has a high-quality basis in this respect. However, this base must be broadened considerably, if Germany is to keep up with other European countries during the impending boom in pediatric research.

The German Society for Pediatric and Adolescent Medicine (DGKJ) is the professional scientific organization for the entire pediatric sector in Germany: www.dgkj.de

Stand: 09.11.2006