Final FDA guidance on clinical pharmacology studies in neonates

The US Food and Drug Administration (FDA) today announced the availability of a final guidance for industry entitled  General Clinical Pharmacology Considerations for Neonatal Studies for Drugs and Biological Products.

This guidance is intended to assist sponsors of investigational new drug applications (INDs) and applicants of new drug applications (NDAs), biologics license applications (BLAs), and supplements to such applications who are planning to conduct clinical studies in neonatal populations.

Effectiveness, safety, or dose-finding studies in neonates involve assessing clinical pharmacology information, such as information regarding a product’s pharmacokinetics (PK) and pharmacodynamics (PD) to inform dose selection and individualization. During in utero development, there are significant physiological changes in the fetus involving the maturation of organs and tissues, including enzyme systems, receptors, transporters, and neurotransmitters. The normal developmental trajectory of these systems is altered by preterm delivery. Postnatal development can also be adversely affected by concurrent illnesses, resulting in altered maturation of organs and tissues and affecting the systems responsible for product absorption (A), distribution (D), metabolism (M), and excretion (E), known collectively as ADME.