Introduction To Drug Development

Question 1

What are the 2 main phases in the drug discovery pipeline?

Answer 1

Discovery phase

Development phase

Question 2

What are the stages within the discovery phase?

Answer 2

Target selection
Target validation
Lead generation

Question 3

What are the stages within the development phase?

Answer 3

Lead optimisation and evaluation

Clinical trials

Question 4

What is the purpose of target selection?

Answer 4

Looking for pharmacological targets for a disease viable target, through literature searches

Question 5

What is the purpose of target validation?

Answer 5

To test the hypothesis and validate the target

Question 6

What is the purpose of lead generation?

Answer 6

To identify lead compounds and improve physiochemical properties.

Question 7

What is the purpose of lead optimisation and evaluation?

Answer 7

To optimise compounds and prepare for clinical trials. For example chemical properties, the efficacy, selectivity pharmacokinetics, toxicology

Question 8

What are the stages of clinical trials?

Answer 8

Phase 1: tolerability, safety, pharmacokinetics
Phase 2: proof of concept, dose finding, confirm efficacy in patients
Phase 3: large-scale studies for regulatory approval

Question 9

How was some drugs discovered?

Answer 9

New information on disease process/ pathophysiology and physiological mechanisms
Existing treatments with an unexpected effect, for example Viagra brackets sildenafil.
New technology, allows treatments to be more specific.

Question 10

What are the main aims of pre-clinical studies?

Answer 10

Is it safe for humans

Estimated dose in humans

Question 11

What are the two types of pre-clinical research?

Answer 11

In vitro = outside a living organism

In vivo = inside a living organism

Question 12

What are the three quality assurance programs that ensure high quality and integrity of research?

Answer 12

GMP - Good manufacturing practice
GLP - Good laboratory practice
GCP - Good clinical practice

Question 13

What takes place in regulatory approval?

Answer 13

All data is reviewed, from preclinical to phase 3

For proposed labelling and patient information sheets

Question 14

Give an examples of a pre-clinical trial study.

Answer 14

Genome wide associated studies (GWAS)

Question 15

What are the main causes of attrition?

Answer 15

Lack of efficacy
Safety
Pharmacokinetics
Cost of goods
Commercial

Question 16

What is the main reason for each attrition in pre-clinical studies?

Answer 16

Toxicology effects

Question 17

What is the main reason for attrition in the different clinical stages?

Answer 17

Phase 1 = safety

Phase 2 = efficacy, Intended effect

Question 18

Where are research findings reported? For scrutiny

Answer 18

Peer reviewed scientific journals (pubmed)

Oral presentations at large scientific conferences

Question 19

What is the reason for the rising cost of drug development?

Answer 19

Clinical trials design

Complex regulatory requirements

Question 20

What is the median cost for drug development from discovery phase to development phase?

Answer 20

£1 billion over 10 to 15 years

Question 21

What is the median cost of discovery phase?

Answer 21

£100 million on average

Question 22

What is the median cost of development phase?

Answer 22

£2 billion for research and development costs