A project was run within the French paediatric clinical research network, c4c national hub Pedstart (Kids France, French NH PPI leader) in collaboration with the French network on rare diseases (Fimatho) to assess the knowledge of children, young patients and their parents, about involvement in pediatric clinical research. The goal of this project is to develop with children, young patients and their parents their priorities for being involved as a partner in paediatric research.

A questionnaire was sent to children aged 8 to 12 years old, young patients aged 13 to 18 years old and parents through Pedstart, Fimatho and patients organizations related to the network.

24 children (mean age 10.5 +/-1.4 years), 11 young patients (mean age 14.2 +/-1.5 years) and 107 parents/carers responded to the questionnaire.

24 (100%) children, 10 (91%) young patient and 98 (91%) parents declared having poor to low knowledge about clinical research.

Responders were asked about which areas of research they would like to be involved in. Children, young patients and parents all strongly expressed that they would like to be involved in defining priorities for paediatric clinical research (71%, 64% and 62 % respectively). Half of children and young patients indicated that they would like to be involved in helping in medicines formulation. Ensuring support to families during their participation in research was also considered a good way to be involved. Children who responded to the survey had low interest in reviewing patients’ documentation, whereas young patients and parents wanted to be involved. Participating to the dissemination of study results was relevant to more than half of parents.

Below are some ideas from responders to the survey about how patients and their families could be involved in research:

This first survey shows us that there is still a need to better communicate about what it means to be involved in clinical research, where patients and their families can be involved and how. Ideas from children, young patients and parents should be listened to when defining and communicating about involvement in pediatric clinical research.

These results are preliminary and will allow us and researchers to better take into account the ideas and experiences of children, young patients and parents when designing activities to involve them, in the field of paediatric rare diseases. This questionnaire could be sent widely, at a European level, through the c4c Patient and Public Involvement (PPI) team. Next step, in France, is to train children, young patients and parents and start potential consulting groups depending on the needs of patients and families and projects developed. This is going to be performed through Pedstart and Fimatho networks.

Pr Frédéric Gottrand (Fimatho rare diseases network), Pr Régis Hankard (Pedstart, c4c NH) and Mrs Segolene Gaillard (Kids France, Pedstart, c4c PPI team)

More information: segolene.gaillard@chu-lyon.fr


Project coordinated by co-coordinator of Pedstart (Pr Frédéric Gottrand) and PPI lead at Pedstart (Segolene Gaillard, KidsFrance).