Clinical trials

Question 1

What is personalised or tailored medicine?

Answer 1

The genetic stratification of either patients or diseases in order to target the prescribing of medicine.

Question 2

List the type of trial designs

Answer 2

Standard RCT
Enriched RCT
A genotype guided RCT

Question 3

Standard RCT consists of what?

Answer 3

A standard RCT
What is the benefit of drug A over placebo?
Bias/variable, small sample size, lack of power, conclusions?

Question 4

State what an enriched RCT involves

Answer 4

What is the benefit of drug A over placebo for patients with a particular genetic profile?
Reduced risk, less non responders, more power

Question 5

State what a guided genotype RCT entails

Answer 5

A genotype guided RCT
Small/cheaper clinical trial, target population identification
Key role for accurate diagnostic testing

Particularly used in oncology drug testing, target driven drug development
‘Basket’ and ‘Umbrella’ trials

What is the cost-benefit ratio of genotype guided treatment over standard care?

Question 6

Define an umbrella trial

Answer 6

An ‘umbrella’ trial is a single trial that tests multiple targeted medicines based on the molecular mechanism of disease.

Question 7

What is a basket trial?

Answer 7

Recruits of patients with different types of cancer but a common mutation (BRAF/ALK/EGFr etc).