PED1003/L18 Drug Development

Question 1

Give the 4 stages of drug development.

Answer 1

Pre-discovery
Drug discovery and preclinical studies
Clinical trials
Review and approval

Question 2

Describe phenotypic screening. (2)

Answer 2

Direct testing in cells
Works for unknown targets and complex disease mechanisms

Question 3

Describe target-based screening. (3)

Answer 3

Fast and efficient simultaneous check of large compound sets
Target molecule detects drug candidate with desired activity
Easy to implement

Question 4

Give 3 trial endpoint measures.

Answer 4

Clinical measures
PK measures
PD measures
Safety measures

Question 5

What is the aim of phase I trials? (3)

Answer 5

PK/PD properties in humans
Toxicological properties
Route and frequency of administration

Question 6

What is the aim of phase II trials? (3)

Answer 6

Non-clinical and human pharmacodynamics
First evidence of clinical effectiveness in humans
Pharmacodynamic dose-ranging
Additional safety information

Question 7

What is the aim of phase III trials?

Answer 7

Emphasis on efficacy

Question 8

What is the aim of phase IV trials? (3)

Answer 8

Investigate new indications for drug
In specific patient sub-groups
Investigate safety concerns