Introduction to Drug Development

Question 1

How long does the drug discovery pipeline take?

Answer 1

It takes 10 - 15 years.

Question 2

What phases are involved with the discovery pipeline?

Answer 2

Discovery Phase

Development Phase

Question 3

What are the stages within the discovery phase?

Answer 3

Target Selection
Target Validation
Lead Generation

Question 4

What are the stages within the development phase?

Answer 4

Lead Optimisation and Evaluation

Clinical Trials

Question 5

What does Target Selection involve?

Answer 5

Literature Search.

Looking for pharmacological targets for a disease.

Question 6

What does Target Validation involve?

Answer 6

Test hypothesis.
Validate the target.
= Double checks everything is working.

Question 7

What does Lead Generation involve?

Answer 7

Identify lead compounds (to allow a high throughput).
Improve physiochemical properties.
= Ensures the drug is soluble.

Question 8

What does Lead Optimasation and Evaluation involve?

Answer 8

Optimise compounds to prepare for clinical testing.
Efficacy / Selectivity / Pharmacokinetics / Toxicology
= Ensures there is no irritation in the skin.

Question 9

What does Clinical Trials involve?

Answer 9

3 major phases.
Phase 1 - Tolerability / Safety / Pharmacokinetics.
Phase 2 - Dosage / Efficacy in patients.
Phase 3 - Large scale pivotal studies.

Question 10

Where does ideas for drug discovery come from?

Answer 10

Accidental.
Existing treatment with unexpected effect.
New information on disease processes.
New technologies.

Question 11

What is the main aim of preclinical research?

Answer 11

Is it safe for humans?

Estimate dose in humans.

Question 12

What are the 2 types of pre - clinical research?

Answer 12

In - Vitro = Outside a living organism.

In - Vivo = Inside a living organism.

Question 13

What is harmonisation of standards?

Answer 13

This is when every result you get is standardised and therefore, can be applied to everyone too.

Question 14

What are the 3 features used in quality assurance to ensure high quality and integrity of research?

Answer 14

GLP = Good Laboratory Practice
GMP = Good Manufacturing Practice
GCP = Good Clinical Practice

Question 15

What occurs in regulatory approval?

Answer 15

All data is reviewed from pre - clinical to phase 3.

This allows proposed labelling and patient information sheets to be filled in.

Question 16

What is the life - cycle management in the post approval stage?

Answer 16

Maximising value of a drug through commercial and development life.
View any other competition from generic drugs.
The patency to sell lasts at least 20 years.

Question 17

What are some challenges with drug development?

Answer 17

Rising cost of drug development. 
- Clinical Trial Design
- Decline in Targets
- Complex regulatory requirement
Public perception of 'unmet needs'.

Question 18

What are the partnerships in drug development?

Answer 18

See hand drawn diagram

Question 19

List some reasons for attrition.

Answer 19

Lack of efficacy 
Safety
Pharmacokinetics
Formulation
Bioavailability
Commercial 
Cost of goods

Question 20

What is the most common reason for attrition in the pre - clinical phase?

Answer 20

Toxicology.

Question 21

What is the most common reason for attrition in the phase 1?

Answer 21

Safety.

Question 22

What is the most common reason for attrition in the phase 2/3?

Answer 22

Efficacy.