Spring Intro 2023

16/02/2023

Parallel group design

Proof of concept: Does the drug have an effect?

Typical characteristics:

• Small size (e.g. n=30 per group) • Carefully selected population • Short duration (e.g. 2-4 weeks) • Easily measured primary endpoint (often a marker) • Many secondary endpoints

Test drug (high but safe dose)

Placebo

• Is there an efficacy signal? • First hint of safety profile in patients • First opportunity to assess benefit-risk profile • Is it worth investing in larger, more expensive studies? What you will learn

The Organisation for Professionals in Regulatory Affairs PMS

Ph I

Ph IIa

Ph IIb

Ph III

Ph IIIb/IV

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Parallel group design

Dose optimisation: Right dose, right frequency

Typical characteristics:

• Significant size (e.g. n=100 per group) • Carefully selected population • Primary endpoint ideally as planned for study in phase III • Duration as required by endpoint • Many secondary endpoints

Dose 4 Placebo Dose 3 Dose 2 Dose 1

Response

Dose

The Organisation for Professionals in Regulatory Affairs PMS

Ph I

Ph IIa

Ph IIb

Ph III

Ph IIIb/IV

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