Introduction

Question 1

Drug Repurposing

Answer 1

It refers to the use of existing, approved drugs for new indications (treating different diseaes for what it was intended)

Question 2

Drug Repositioning

Answer 2

It involves the development of an existing, previously evaluated but unapproved active pharamaceutical ingredient for the treatent of a different disease.

Question 3

Example of Drug Repurposing

Answer 3

-Asprin repurposed for colorectal cancer
-Metaformin repurposed from diabetic drug to anticancer

Question 4

Who is involved in drug discovery

Answer 4

-Biology experts
-Chemistry experts
-ADME experts (what drug does to body)
-Toxicology experts
-Pharmaceutical experts

Question 5

Target identification

Answer 5

Find the cause of a specific disease so that drugs can be developed to inhibt/enhance it

Question 6

What are the 3 main phases of discovery

Answer 6

-Target identification
-Lead molecule identification
-Lead optimisation

Question 7

Lead identification

Answer 7

Creation and identification of a molecule that attaches to the cause of a disease and produces an effect

Question 8

Lead optimisation

Answer 8

Molecules are optimised to improve their safety and efficacy in this phase whilst also reducing off-target effects

Question 9

Pre-clinical test

Answer 9

Evaluate the best 1-3 compounds on animals and work out their therapeutic index

Question 10

Therapeutic index

Answer 10

Drug concentration associated with safety/Drug concentration associated with toxicity

Question 11

IND application

Answer 11

-Application to test drugs on humans
-FDA must approve prior to First-In-Human (FIH) clinical trial
-contains:
-Acute toxicity of substance in at least 2 animal species
-short-term toxicity studies
-Pharmacological profile of drug substance

Question 12

Phase 1 Clinical trials

Answer 12

Human Pharacology
-FIH dosing to healthy volunteers
-Single escalating doses are given to small number of subjects
-10-15 volunteers
-Just checks if drug has any immediate negative effect
-MTD: Maximum tolerated does is determined

Question 13

Phase 2 Clinical Trial

Answer 13

Therapeutic exploration
-First dose to patients
-Fewer dosages than phase 1
-20-100 subjects
-Asses safety profile and proof-of-concept
-Consists of two stages: Therapeutic dose set and overall efficacy

Question 14

Phase 3 Clinical trial

Answer 14

Therapeutic Confirmation
-Most important phase
-Test effectiveness of drug and common effects in larger population
-finalize prescribing label
Over 1000 subjects

Question 15

Phase 4 Clinical Trials

Answer 15

Therapeutic Use
-Physicians record any side effects
-Post-market approval studies
-Additional information about effectiveness and safety is added which leads to alteration in labels
-300-3000 volunteers