Expedited research and approval of medicines for drug-resistant bacterial infections in children.

Abstract

Multidrug-resistant pathogens cause severe infections with substantial morbidity and mortality in neonates, infants and children, as they do in adults. However, the approval of therapies for multidrug-resistant infections in paediatric patients often lags years behind approval for adults, which creates an untenable situation for people who care for affected children. Many issues contribute to the current unacceptably long period between adult and paediatric new drug approval, including the requirement for sequential age group enrolment in clinical trials, the types of regulatory studies required and a lack of alignment between regulatory authorities. To streamline paediatric drug development, more efficient approaches to investigational and approval processes that meet acceptable safety and efficacy metrics are required. For products that address an urgent unmet need in children, approval should be based primarily on matching drug exposure to levels in adults and on extrapolating safety and efficacy data from adults, where appropriate, rather than on requiring clinical trials in all paediatric age groups for each approved indication in adults. In addition, real-world and post-approval data could be used to support early, limited drug approval for paediatric patients. However, there are economic challenges in developing anti-infective drugs for paediatric populations and increased funding is needed to ensure that neonates, infants and children globally have timely access to safe, effective and life-saving therapies.