We are happy to announce that the manuscript “Target therapy for high-risk neuroblastoma treatment: integration of regulatory and scientific tools is needed” has just been published in the Frontiers in Medicine journal.

The article describes the developmental and regulatory status of active substances targeting neuroblastoma with the aim to underline the existing regulatory gaps in the medicinal product development and to discuss possible improvements.

Neuroblastoma is the commonest paediatric extracranial solid tumour. While local low-risk neuroblastoma can be controlled with a high rate of cure, there is an urgent need to develop new treatment options for the high-risk neuroblastoma still representing a leading cause of death from cancer in children.

Authors investigate the developmental and regulatory status of selected active substances by searching for related preclinical studies, clinical trials as well as regulatory procedures such as marketing authorisations, paediatric investigation plans and orphan designations.

Results confirm that it is necessary to identify a more efficient, less costly, and time-consuming “pediatric developmental model” integrating predictive preclinical study and innovative clinical study designs. Furthermore, stricter integration between scientific and regulatory efforts should be promoted.

Discover more by reading the full article here!