Since 2008, we in the Servier Group have a paediatric team dedicated to medicines intended for children.
Only a decade ago, such products could be summed up as scaled-down formulations and dosages of adult medicines, without clinical trials specific to the paediatric population. However, given that children are not scaled-down versions of adults, this approach left many medical needs unmet.
Our paediatric team endeavours to provide quality responses to the specific needs of this population by mobilising the various specialist areas of R&D around a shared focus: paediatric clinical research.
For each molecule, indication and new formulation, we set up Paediatric Investigation Plans (PIPs) before each Marketing Authorisation (MA) application, whether for a new MA or to modify an existing one. The purpose of these plans is to demonstrate a product’s safety and potential benefits in children.
As part of these PIPs, we conduct paediatric clinical trials in areas of major unmet medical need. Trials are already ongoing in the domains of paediatric cardiology, neuropsychiatry and haematology and we expect to extend these to include paediatric oncology and central nervous system diseases, in line with our developments in adult patients.
Specific ethical and scientific considerations govern paediatric clinical trials which, before given the green light, require:
To best meet the needs of children, their families and their caregivers, we have been working increasingly closely with paediatric patients’ associations and international clinical networks. This approach makes it possible to benefit from their feedback and promote the harmonisation of practices.
Acute lymphoblastic leukaemia (ALL) primarily strikes children. It is a malignant disease of the bone marrow that can be treated effectively in 85 – 90 % of cases. For relapsed/refractory ALL, we at Servier are developing, in partnership with Pfizer and Cellectis, a particularly innovative form of cell therapy. Derived from immuno-oncology research, the principle of this innovation is to use CAR-T cells (T-lymphocytes, playing a major role in cell-mediated immunity) to stimulate the patient’s immune system to fight the tumour. To best tailor this treatment to the needs of children, our R&D teams have been conducting since 2016 a paediatric clinical trial whose initial findings show promise.