Module 9 2021

19/03/2021

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

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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

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The Organisation for Professionals in Regulatory Affairs

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