Module 9 2024

03/09/2024

First-In-Human Studies – Risk Mitigation

• Factors to be considered in ascertaining the balance between the degree of uncertainty and potential risks • Study population • Trial site(s) • Number of subjects per cohort • Sequence and interval between dosing of subjects in each cohort

• Transition to the next cohort/part of study • Stopping rules/monitoring of adverse events • Route and rate of dosing • Dose escalation

The Organisation for Professionals in Regulatory Affairs

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First-In-Human Studies - Population

• Studies in Healthy Volunteers requires justification • Objective of FIH studies is more focused on assessing safety and tolerability than efficacy • IMP target may respond differently in healthy subjects compared to patients with the disease of interest • PK can be impacted • Dose-response may be different • Immune status needs to be considered • Safety can be underestimated

The Organisation for Professionals in Regulatory Affairs

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