Week 3

Question 1

What is the protocol and what is included in it? (11)

Answer 1

Set of instructions to carry out experimental procedure to address primary research question
Will need to be submitted for regulatory or ethics committee review to look at

Includes:

• Background
• Objectives and research question
• Design
• Intervention
• Follow up visits and schedules
• Safety assessment
• Data analysis
• End of study / termination policy
• Organisation

Question 2

What is the difference between superiority and non- inferiority trials? (4)

Answer 2

Superiority (Comparative trial):
To demonstrate if IMP is superior to standard treatment in terms of outcome measures
H0= 2 treatments have same effect

Equivalence (Non inferiority trial)
To demonstrate if X is no worse than standard treatment in terms of outcome measured
H0= 2 treatments are different

Question 3

When may a non- inferiority trial be used? (3)

Answer 3

• Bioequivalence trial in development of generic drugs
• New treatment has important advantages over standard treatment
• When testing new treatment against placebo is unethical

Question 4

What is a factorial design clinical trial? (1)

Answer 4

When participants are randomised to more than 2 treatments or there are many independent variables
(Eg: 2x2)

Question 5

How can the participant and investigator prevent missing data and maximise protocol adherence? (6)

Answer 5

Investigator:

• Simpler case report forms
• Reduce unnecessary collection of datapoints
• Training and re- training

Participant:

• Reminder to attend visits
• Reminder and training for trial assessments, such as adherence to treatment
• Set rules to exclude patients when non- adherent

Question 6

How might a clinical trial practitioner minimise variation? (4)

Answer 6

• Consistent endpoint measures
• Objective endpoint measured
  Such as same machine, technicians, imaging assessments, standardised trial procedures
• Adverse events reporting