The development of new medicines for paediatric patients, particularly in oncology and rare diseases, remains one of the greatest challenges in modern medicine. The growing importance of academic clinical research was highlighted during this year’s International Clinical Trials Day (ICTD).
The event, organised annually by the European Clinical Research Infrastructure Network (ECRIN), took place in Prague on 20 May and brought together experts from approximately 15 countries. This year’s edition was co-organised by the Czech research infrastructure CZECRIN as part of the CREATIC Centre of Excellence at the Faculty of Medicine, Masaryk University. The Czech Republic thus presented itself as a country that, in addition to providing high-quality healthcare, is playing an increasingly important role in the development, clinical evaluation, and accessibility of modern therapies within the European context.
“Cell and gene therapies currently represent the highest level of innovation in the treatment of rare diseases, particularly in paediatric patients. For these therapies to become part of routine clinical practice, clinical trials evaluating their safety and efficacy are essential. These studies also provide crucial data required for regulatory decision-making. In the Czech Republic, this responsibility lies with the State Institute for Drug Control,” said Associate Professor Regina Demlová, Director of the CREATIC Centre of Excellence and the national research infrastructure CZECRIN.
Experts in paediatric oncology have long pointed to the need for systematic support for the development of new medicines for children. “The pharmaceutical industry is naturally focused primarily on large patient populations. However, for most childhood cancers, commercial incentives alone are insufficient. Due to current legislation, both at the national and EU levels, children remain a long-neglected patient group. If we do not want children to continue waiting many years longer than adult patients for access to modern treatments, much greater involvement from governments, academia, and international institutions is needed,” said Professor Jaroslav Štěrba, Head of the Department of Paediatric Oncology at the Faculty of Medicine of Masaryk University and University Hospital Brno, leader of a research group within the CREATIC Centre of Excellence, and Scientific Director of the National Institute for Cancer Research.
According to Professor Štěrba, Czech paediatric oncology benefits from a highly developed professional background and is integrated into leading European research and treatment networks. However, to keep pace with the rapid advances of modern medicine, sustained and systematic support is needed for clinical research, data infrastructure, and innovative treatment approaches for small patient populations. Read more here.
