Case Unit 2 - CML and Imatinib

Question 1

What is rational drug design?

Answer 1

Using knowledge of a disease mechanism to identify drug targets
Creating a compound designed to interact with identified target

Question 2

What does PICO stand for?

Answer 2

Population of interest
Intervention
Control
Outcome

Question 3

What does PICO stand for?

Answer 3

Population of interest
Intervention
Control
Outcome

Question 4

How do you calculate QALYs?

Answer 4

Life expentancy (life years) x HR-QoL

Question 5

What are the 5 domains of health tested by the EQ-5D questionnaire?

Answer 5

Mobility
Self-care
Usual activities
Pain/discomfort
Anxiety/depression

Question 6

What does ICER stand for and how do we calculate it?

Answer 6

Incremental cost-effectiveness ratio
Difference in cost/difference in QALYs
Results in cost per QALY gained

Question 7

Name some ‘down the line’ costs that may affect a treatment options price

Answer 7

Administration of treatment
Primary, community and social care
Hospital care, outpatient consultations, tests, imaging
Emergency episodes
End of life palliation

Question 8

What happens in Phase 1 of a clinical trial?

Answer 8

First in man studies
50-100 healthy volunteers
Aim to find the maximum tolerated dose

Question 9

What happens in Phase 2 of a clinical trial?

Answer 9

First in patient studies

Aims to determine the dose for larger scale phase III studies

Question 10

What happens in Phase 3 of a clinical trial?

Answer 10

Multicentre trials

Confirms the efficacy and safety of the drug