Introduction to Drug Development

Question 1

How long does the drug discovery pipeline take?

Answer 1

It takes 10 - 15 years.

Question 2

what is the target of drug development?

Answer 2

produce the drug in a marketable form and gain regulatory permission to use it for target indication as quickly as possible.

Question 3

What phases are involved with the discovery pipeline?

Answer 3

Discovery Phase
Development Phase

Question 4

What are the stages within the discovery phase?

Answer 4

Target Selection
Target Validation
Lead Generation

Question 5

What are the stages within the development phase?

Answer 5

Lead Optimisation and Evaluation
Clinical Trials

Question 6

What does Target Selection involve?

Answer 6

Literature Search.
Looking for pharmacological targets for a disease.

Question 7

What does Target Validation involve?

Answer 7

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

Question 8

What does Lead Generation involve?

Answer 8

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

Question 9

What does Lead Optimasation and Evaluation involve?

Answer 9

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

Question 10

What does Clinical Trials involve?

Answer 10

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

Question 11

Where does ideas for drug discovery come from?

Answer 11

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

Question 12

What is the main aim of preclinical research?

Answer 12

Is it safe for humans?
Estimate dose in humans.

Question 13

what are the stages and decison points of pre-clinical trials?

Answer 13

Question 14

What are the 2 types of pre - clinical research?

Answer 14

In - Vitro = Outside a living organism.
In - Vivo = Inside a living organism.

Question 15

What is harmonisation of standards?

Answer 15

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

Question 16

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

Answer 16

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

Question 17

What occurs in regulatory approval?

Answer 17

All data is reviewed from pre - clinical to phase 3.
This allows proposed labelling and patient information sheets to be filled in.

Question 18

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

Answer 18

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 19

What are some challenges with drug development?

Answer 19

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

Question 20

What are the partnerships in drug development?

Answer 20

See hand drawn diagram

Question 21

List some reasons for attrition.

Answer 21

Lack of efficacy
Safety
Pharmacokinetics
Formulation
Bioavailability
Commercial
Cost of goods

Question 22

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

Answer 22

Toxicology.

Question 23

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

Answer 23

Safety.

Question 24

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

Answer 24

Efficacy.

Question 25

what is the benefit of Pharmacovigilance?

Answer 25

want to find the problems as early as possible, to save money