Treatment of pediatric diseases requires thoroughly investigated medicines
In HUS, pediatric clinical trials with medicinal products present increasingly more important part of the hospital’s high-quality, permanent activities, and that requires solid co-operation between professionals in various specialties.
For example, research provides crucial new information about the efficacy of medicines in the treatment of various diseases and about pediatric drug safety.
Docent Jaana Kallio and M.Sc. Pirkko Lepola are establishing pediatric clinical trial unit in the division of digital and innovation services at HUH Children and Adolescents. Some of the trials are researcher-initiated academic studies and some are funded by the pharmaceutical or biotech industry.
The EU regulation on medicinal products for pediatric use that came into force in 2007 increased the need for research. The regulation obliges pharmaceutical companies to study all new drugs for their use in children and adolescents as well, if the European Medicines Agency sees the products are necessary and needed for the pediatric population.
HUS directs resources to train research personnel, and all activities are guided by Good Clinical Practice guidelines.
To ensure high-quality research and applicability for children, expert teams evaluate and plan all studies.
Pediatric clinical trials can provide information about medicinal products such as their absorption and breakdown, elimination and excretion from the body (pharmacokinetic studies), efficacy and safety (clinical trials), and long-term effects (drug safety studies). In this way, it is possible to provide new innovative, safe and effective medicines also for children.
In the future, the implementation of clinical trials will include a separate youth panel where children and adolescents can express their opinions about, for example, the readability of trial information leaflets and about understanding the trial procedures involved in the study.
Importance of networking in pediatric clinical trials
HUS conducts pediatric clinical trials in collaboration with the Finnish investigators network for pediatric medicines (FinPedMed) together with similar Nordic (NordicPedMed) network, having a same aim to increase recruitment and participation in pediatric clinical trials in the future.
As required by the Paediatric Regulation, the pharmaceutical industry has already created over a thousand Paediatric Investigation Plans (PIP) approved by the Pediatric Committee of the European Medicines Agency.
– Only around 130 of these PIP trials have been carried out. The future trials would require the participation of over 200,000 children in Europe. This requires wide research networks, and we contribute to their development significantly, Jaana Kallio explains.
Common European c4c project
HUS researchers are participating in a project called c4c (Collaborative Network for European Clinical Trials for Children), which was launched in May 2018. It is funded by IMI2 (Innovative Medicines Initiative2).
– We are developing a Europe-wide network that is efficient and of high quality to act according to the ethical and regulatory guidelines, and fulfil all quality and safety requirements set for research. HUS acts as a national hub in the project. We are looking for methods that would allow a permanent and seamless network to develop. In this way, research would best serve the needs of pediatric drug treatment and would create new innovative, effective, and safe medicinal products for children at an accelerated pace, Kallio states.
Helsinki University Hospital, Children and Adolescents