Week 2

Question 1

What is a ‘fair test’ and how is it achieved? (8)

Answer 1

• ‘Perfect experiment’
• Only one variable is changed

Can be achieved by:

• Randomisation
• Double blinding
• Controlled
• Pre specificy data analysis
• Analysing data before unblinding

Question 2

What are the different phases in clinical trials? (8)

Answer 2

Phase 1: 50-200 healthy volunteers
- Safety, PK and PD

Phase 2: 100-400 patients with the target disease
- Safety, PK and PD, proof of concept

Phase 3: 100- 5000 patients with the target disease
- Efficacy, proof of concept, less PK and PD

Phase 4: 1000- 1M- Post marketing

• Long term safety and efficacy in clinical practice
• Comparison to existing treatment

Question 3

What do the FINER and PICOT acronyms stand for and what do they mean? (2)

Answer 3

FINER: for a good research question
Feasible
Interesting
Novel
Ethical
Relevant

PICOT: to refine a research question
Population
Intervention
Control
Outcome
Time

Question 4

What is the difference between a parallel and cross over study? (3)

Answer 4

Parallel:

• Independent groups take different drugs
• Unpaired

Cross over study:

• Participants take the same treatments in a different sequence, may involve a washout period
• Paired
• Own control group

Question 5

What do GCP, IB, IMPD and SmPC mean? (4)

Answer 5

GCP:
- Set of internationally recognised ethical and scientific quality requirements for clinical trials

IB:

• Summary info of all preclinical and clinical data
• PK/ PD
• Toxicology
• Safety

IMP dossier:
- Info about quality, manufacture and control of IMP

SmPC:

• Legal document after licensing that contains all information on the drug
• Mirrors IB

Question 6

What are clinical trial registers and what are examples? (2)

Answer 6

• Practice of documenting clinical trials before they are performed in a clinical trial registry
• Combat publication bias and selective reporting

Examples:
EudraCT (EU)
ISRCTN register (international standard randomised controlled trial number) (UK)
Clinicaltrials.gov (USA)

Question 7

What is a sponsor? (1)

Answer 7

• Individual, companty, institution, organisation or group of organisations that take on responsibility for:
  Initiation, management, financing

Legal requirement

Question 8

What is the difference between a chief investigator and principal investigator? (2)

Answer 8

Chief investigator:
- Overall responsibility for the conduct of the whole project in the UK

Principal investigator:
- Conduct of research at a research site