Spring Intro 2023

16/02/2023

Parallel group design

What you will learn

• Understand the dose-response curve • Identify optimum dose and frequency for confirmatory testing at phase III

• Clinically relevant effect • Statistically significant effect • Acceptable safety profile

The Organisation for Professionals in Regulatory Affairs PMS

Ph I

Ph IIa

Ph IIb

Ph III

Ph IIIb/IV

21

Parallel group design

Confirmation of efficacy and safety: Are the phase II findings reliable?

Typical characteristics:

Design often agreed in advance with regulatory authorities

Test drug (proposed MA dose)

? Additional control arm (placebo or active) ? Test drug (alternative dose)

Control arm (active or placebo)

• Large studies • Very expensive !!

• Usually need at least two studies • Population similar to expected clinical use • Duration as required by endpoint • Fewer secondary endpoints than phase II

What you need

Primary endpoint success

• • • •

Clinically relevant effect; p<0.05 Secondary endpoint support

Acceptable safety profile

The Organisation for Professionals in Regulatory Affairs MS

Ph I

Ph IIa

Ph IIb

Ph III

Ph IIIb/IV

22