Overview of Research and Development

Question 1

What considerations go into making a new drug?

Answer 1

Strategic: should it be done?
Scientific and technical: can we do it?
Operational: can it be done?

Question 2

What are the steps in the drug life cycle?

Answer 2

Drug discovery, drug development, regulatory review and approval, commercialisation

Question 3

What is the difference between a drug substance and a drug product?

Answer 3

A drug substance = only the API. Drug product is the finished dosage form with the API and other active/inactive ingredients.

Question 4

What are the two types of drugs?

Answer 4

Small molecule drugs
Biologics

Question 5

What are the two approaches to drug discovery and how do they work?

Answer 5

Phenotypic based - watch what happens to cells when you add compounds/observations in groups of people
Target based - molecular approach

Question 6

What is an agonist and how is the potency measured?

Answer 6

Ligand that enhances cellular activity, potency measured by EC50 values

Question 7

What is an antagonist and how is the potency measured?

Answer 7

Ligand that reduces cellular activity, potency measured by IC50

Question 8

Why is understanding ADME and DMPK important?

Answer 8

Therapeutic dose needs to be maintained for efficacy of drug and avoidance of toxic levels building up. ADME and DMPK are key considerations in building a dosing regime.

Question 9

What are the steps in the drug discovery process?

Answer 9

Target identification, high-throughput screening to identify hits, modification to form leads in vitro, followed by in vivo optimisation to produce a drug candidate.