Pediatric drug development: existing challenges and recommendations

Despite the international regulatory initiatives of the last 20 years, many challenges persist in the development and evaluation of drugs for paediatric use. Indeed, drug research and development remains essentially oriented towards adult indications, thus excluding many sick children, limiting the feasibility of pediatric trials and favoring competitive developments. This limits the feasibility of pediatric trials and favors competitive developments. Prescribing of medicines outside the scope of marketing authorization (hors-AMM) persists, and the development of galenic forms adapted to children's age remains limited. In this context, the members of the round table recommend the creation of multi-partner thematic exchange forums to focus research and development of new drugs on the real, unmet needs of sick children and adolescents, and in relation to drug mechanisms of action. Multi-partner scientific interactions are also needed to define reference evaluation methodologies for each medical field. Exchanges can be organized via existing national and European pediatric research structures/networks. The involvement of these structures dedicated to pediatric research will also facilitate trial set-up and recruitment. In addition, the creation of specific calls for projects and private-public partnerships remain necessary to support studies to reposition drugs in pediatric indications and pharmacokinetic studies to determine appropriate dosages. The development of new galenic formulations better suited to pediatrics, and the concrete valorization of the resulting innovations, will boost future investments. Initiatives to collect observational data on safety and efficacy after use on derogatory access, off-MA and/or post-MA should also be encouraged to compensate for the loss of information observed in these contexts. Finally, the creation of a Committee for the Protection of Persons (CPP) with a "mother-child" specificity seems desirable in order to apply the current regulations (Jarde law) by taking into account the specificities of pediatrics in drug trials.