Module 5 Presentations

16/07/2024

OTHER IMPORTANT GUIDANCE INCLUDES….

• Biotechnology products • ICH guideline Preclinical Safety Evaluation of Biotechnology-Derived Pharmaceuticals (ICH S6) • Pharmaceuticals for indications in life threatening or serious diseases without current effective therapy (e.g. Oncology) • In these cases, particular studies may be abbreviated, deferred or omitted (ICH S9) • Regulatory dialogue • Paediatric medicinal products • Innovative therapeutic approaches • Many others….Combinations, Orphan Drugs, Biosimilars

The Organisation for Professionals in Regulatory Affairs

Module 5 Lecture 3 – Nonclinical safety assessment

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SOME POINTS TO CONSIDER

There is NO…………… • single ’metric’ to make a quantitative risk assessment • multiple of human dose/exposure that can always be considered safe The non-clinical results………… • are usually a variety of changes with a range of severity & clinical importance • some may be readily ‘reversible’ or ‘adaptive’ • may contain endpoints that can be a monitored with clinical biomarkers • can guide choice of maximum doses/exposure for humans • may suggest a ‘special’ clinical (or non-clinical) study is required to advance

However, exposure (eg: AUC), at a toxicologic NOAEL, ten times greater the maximum intended for humans is often considered reassuring.

Remember………… • Use emerging clinical data & outcomes. Can you max adjust doses (up or down!)? • Do not become over reliant on animal data (numbers of animals is small) • Make an overall risk assessment to define clinical doses & progression Module 5 Lecture 3 – Nonclinical safety assessment

The Organisation for Professionals in Regulatory Affairs

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