Spring Intro 2023

Mike Kelly

Phase II Studies Patient Studies

 Looking in small number of patients ● Safety and efficacy

● Evaluation of Pharmacokinetics in patient population ● Measurement of pharmacodynamics in patient population – Proof of concept/principle (POC/POP) ● Dose range finding study  Provide information to enable exploration of exposure/dose – response (PK/PD) relationships  To ensure robust dose selection for larger Phase III patient trials

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Phase III Studies

 Large patient studies primarily to assess safety and efficacy

 Pharmacokinetics ● Compliance assessment ● Population pharmacokinetics (PopPK) to identify subgroups ● Therapeutic drug monitoring ● Fewer observations in more patients – Sparse PK data modeling  Pharmacodynamics ● Usually ‘macro - scale’ ● Based on therapeutic outcome

 PK/PD modelling within clinical trial design can £m ● Reduction in treatment arms ● Reduction in patient numbers

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