Module232025

07/05/2025

Pregnancy and Lactation studies – timing in the development program Increasing calls for the earlier inclusion of pregnant and lactating women in clinical development at an earlier stage. Currently, a common development process waits until Ph II data (mostly from males) are available. Reasons: Confirms confidence in rationale (the drug works). Identifies dose / dose range for confirmatory trials – disaggregation of efficacy and safety by sex can be assessed from Ph III database. Allows preliminary cost of goods calculation – is the drug viable at the expected dose? Enable discussions with EU and US regulators regarding future requirements. Expenditure on expensive toxicology (DART, carcinogenicity) deferred until drug is a “GO”. 60–70% of Phase II trials are unsuccessful 13 Developmental and Reproductive Toxicology (DART) and carcinogenicity normally takes ~2 years Note that 21 CFR §46.204 (Research involving pregnant women or fetuses) stipulates that “Where scientifically appropriate, preclinical studies, including studies on pregnant animals, and clinical studies, including studies on nonpregnant women, have been conducted and provide data for assessing potential risks to pregnant women and fetuses” So why delay Ph II by 2 years and incur substantial spend before the answers to the four questions above are known?

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Neonates and Premature Babies

The Organisation for Professionals in Regulatory Affairs

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