CRED Understanding Clinical Development 2024

08/10/2024

Considerations for the endpoint selection (Primary and Secondary) ⚫ Is the primary endpoint (PE) a hard, subjective or surrogate endpoint?

⚫ Statistical assumptions, study numbers, cost

⚫ Single, co-primary endpoint, composite endpoint for Phase 3? ⚫ Are they accepted and validated - regulatory guidelines?

⚫ Scientific Advice (EMA, FDA, National – Local HA in EU) ⚫ Novel/new endpoint?

⚫ Scientific Advice/Qualification Advice (EMA) ⚫ Precedence – PE/SE’s

⚫ Market products/SOC - What has been done before?

⚫ Improvements? ⚫ Identify potential key SE ⚫ Exploratory endpoints – biomarker identification

The Organisation for Professionals in Regulatory Affairs

ASPIRATIONAL LABEL CLAIMS

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Dose Response Relationship Pharmacokinetic/Pharmacodynamic evaluation (PK/PD)

⚫ Dose optimization (risk/benefit)

⚫ Important to be able to distinguish between doses (dose response)

⚫ Ideally a minimum of 3 doses should be evaluated

⚫ Need to be able to distinguish safety and efficacy between doses (doubling of doses)

⚫ PK profile and variability in patients

⚫ Start developing a Population PK model (developing a pharmacokinetic model for future extrapolations)

⚫ PD – safety and efficacy

What type of measurement?

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⚫ How can the data be modelled?

The Organisation for Professionals in Regulatory Affairs

SELECTING THE DOSE/S FOR Ph 3

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