In the last two decades of the 21st century, many pediatricians and all scientific associations related to pediatrics, started a campaign to make regulatory authorities aware that most drugs that had marketing authorization had no clinical trials in children. Because of this lack of scientific evidence, all pediatricians were obliged to prescribe drugs to pediatric patients based only on their personal experience. This pressure was successful and all regulatory authorities, first of all, the FDA and the EMA, made it mandatory to submit, at the time of the NDA application, the results of clinical trials performed both in adults and in children.

‘Children’ is a broad term, as the pediatric population is divided into 5 different groups:

  • Pre-term newborn infants;
  • Term newborn infants (from birth to 30 days);
  • Infants (from 1 to 24 months);
  • Children (from 2 to 11 years);
  • Adolescents (from 12 to 18 years).

The first 3 categories of children represent the most fragile population: hence the FDA’s issuance of a new guideline for the planning and implementation of clinical trials.

“Detailed planning of neonatal studies should include input from a multidisciplinary team involved in neonatal care, including parents and NICU nurses, in the early stages of study design to provide their perspectives of study feasibility and the potential impact of study participation on neonates and their families. This guidance provides recommendations regarding approaches and design features for neonatal clinical pharmacology studies, including ethical considerations and coupling prior knowledge and data obtained from adult, preclinical, and other pediatric studies with innovative quantitative approaches, to help predict neonatal doses and optimize clinical trial design.”1

You can read the full guidance here.

References

  1. Center for Drug Evaluation and Research. (2022, July 26). General Clinical Pharmacology Considerations for Neonatal Studies for Drugs and Biological Products Guidance for Industry. U.S. Food and Drug Administration.

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  • Any personal opinions expressed are those of only the author(s) and are not intended to represent the position of any organization(s)
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