Clinical Trials

Question 1

Phase 1

Answer 1

Safety, tolerability, PK
Drug safe? (in healthy people)
What doses are tolerated
What is the disposition of drug? PK/ ADME
- small sample
- Subjects minimise inter-subject variability
- healthy volunteers
- highest risk (first human dosing)
- short duration
- moderate fail rate

Question 2

Phase 2

Answer 2

Safety, efficacy, PK in patients
Drug safe? (in patients)
Tolerated doses how effective?
Disposition of drugs in patients?
- Small sample
- moderate inter-subject variability (patients screened)
- moderate risk (better characterised adverse effect profile, dose dependent toxicities identified)
- moderate duration
- highest failure rate (efficacy related)

Question 3

Phase 3

Answer 3

Safety, efficacy, cost-effectiveness
Drug safe? (in larger population, more rarer side effects?)
Is drug better than current therapies?
- large sample
- high subject variability (closely mimic patients)
- moderate risk (characterised rarer side effects with long exposure of AE profiles)
- long duration
- moderate fail rate (due to cost effectiveness)
- most expensive