Spring Intro 2023

16/02/2023

See ICH E10

Types of control in clinical trials

Placebo control

Active comparator control

No control group

• When benefit over placebo already proven

• Gold standard • Response to placebo can be high • Not always ethical • Very important to FDA

• Very common in studies designed to collect safety data…

• When placebo not ethical

…otherwise not often used

• To assess relative benefits and risks vs other drugs

• Often needed in Europe

The Organisation for Professionals in Regulatory Affairs

7

Primary endpoint

Selection of clinical endpoints

• Endpoints must be prospectively defined

• Usually (and ideally) just one primary endpoint • May be related to efficacy or safety • Often a single parameter (e.g. survival)… • …but could be a composite (e.g. deaths plus hospitalisations) • Often a “surrogate” • BP in hypertension • PFS in cancer

The Organisation for Professionals in Regulatory Affairs

8