Module 9 2021

22/03/2021

Preclinical testing of biologics

High level definition of requirements in several guidelines…

And….all local National Authorities

But these need to be converted into the best toxicology package for the individual molecule

Preclinical testing of Biologics – March 22 nd 2021

The Organisation for Professionals in Regulatory Affairs

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Preclinical testing of biologics - Design of toxicology package

The aim of any toxicology package is essentially to : • Predict potential for toxicity in humans • Identify potential organs involved and whether any effects are permanent or reversible • Identify pharmacodynamic biomarkers for use in the clinic • Special considerations for the intended population – Children? Elderly? Women? • Special considerations for the indication – Life-threatening? Severe? • Assess whether the new drug can be used safely in clinical trials and at what dose • Support selection of a safe starting dose and maximum dose for Phase I First-In- Human clinical studies, and doses for longer term clinical use • Identify safety parameters/biomarkers for clinical monitoring • e.g. altered blood pressure, liver enzymes or intended changes such as BMD gains

Preclinical testing of Biologics – March 22 nd 2021

The Organisation for Professionals in Regulatory Affairs

8

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