Drug Discovery

Question 1

Target Selection

Answer 1

Me Too Drug
- Make their own version of a drug

First in Class
- First kind of the drug

More emphasis on First-in-Class now

Question 2

Drug Discovery Phase

Answer 2

Target Selection based on knowledge of mechanisms
- Altered Signaling Pathways
- Genomic Data
- Proteomic Data

Once target is picked needed to develop an assay to measure if drug candidate modifies the target

Question 3

Preclinical Development

Answer 3

• Safety Pharmacology
• Preliminary Toxicology Testing
• Pharmacokinetic/Pharmacodynamic Testing
• Chemical/Pharmaceutical Development
  –> Can the drug realistically be safe and efficient

Question 4

Phase 1

Answer 4

Small group of healthy individuals (20-80)
- Assess safety and pharmacokinetics of drug

Question 5

Phase 2

Answer 5

Small group of patients (100-300)
- Determine if compound has clinically beneficial pharmacodynamic effects for the intended patient population

Question 6

Phase 3

Answer 6

1000s of Patients
- Double Blind Randomized Placebo Controlled Trials
–> Assess efficacy of drug verses placebo or standard of care

Question 7

Phase 4

Answer 7

Post Marketing Surveillance Study
- Detect long-term or rare adverse effects