Planning Your First-In-Human Trial

A first-in-human (FIH) clinical trial is a significant milestone in the development of a potential new drug in that it will be the first opportunity for a drug development sponsor to evaluate the impact of their new chemical entity (NCE) or biologic in humans. Typically, FIH trials with compounds intended for treatment of diseases other than cancers or certain rare non-malignant diseases are conducted using normal healthy volunteers (NHVs), unless there is an ethical concern (such as known toxicity) in administering the investigational drug to an otherwise healthy population.

During a FIH study, objectives may include:

• Evaluating safety and tolerance
• Determining pharmacokinetics (exposure and dose proportionality)
• Identifying early pharmacological activity relative to exposure level, either based on measured physiologic responses, or on biomarkers of response identified during preclinical testing
• Assessing observed effects on subsets of participants based on age, gender, or ethnicity
• Evaluating the therapeutic outcomes in a small group of patients suffering from the targeted disease

At this stage of the investigational product’s (IP) lifecycle, a drug may fail simply because the human participant does not respond to the drug in the same manner that was suggested by preclinical testing. Mitigate risks with this stepwise approach, and by partnering with an experienced and skilled CRO/CDMO.*