Module 9 2021

19/03/2021

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

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The Organisation for Professionals in Regulatory Affairs

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First-In-Human Study 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

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The Organisation for Professionals in Regulatory Affairs

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