Module 9 2024

03/09/2024

Considerations for PK in Certain Populations

• Further studies may be required to address specific populations • Renal impairment • Particularly for mAbs where clearance could be impacted • Hepatic impairment • Not usually required due to low potential of interactions with CYP450 • Potential interactions with liver enzymes indirectly would lead to further investigation • Elderly • Usually required due to changes in PK profile with age, high probability of use with concomitant medication and other manifestations • Paediatrics • PK profile is different to adults, therefore dedicated studies would be required, these would be agreed via a PIP

The Organisation for Professionals in Regulatory Affairs

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Phase 2 Studies

• Therapeutic exploratory studies are governed by ICH*, CHMP general and specific-therapeutic area guidelines • Studies can be characterised into 2 subtypes • Phase 2a: designed to assess dosing • Phase 2b: designed to assess efficacy and optimise dosing (POC) • Potential to expedite the CDP by combining Phase 2a and 2b in an adaptive design • Possibility to submit a marketing authorisation based on a Phase 2b study • Discussion with regulators necessary to seek agreement (e.g. SA, PRIME) • Likely to be more successful for diseases with a high unmet need

The Organisation for Professionals in Regulatory Affairs

*ICH Guideline E8 (R1) on general considerations for clinical studies

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