Randomised Controlled Trials

Question 1

What are the 4 phases of drug development?

Answer 1

1) 20 to 100 people to test for safety
2) 100-500 to check efficacy and surrogate end points
3) 1000-5000 for effectiveness and safety evidence
4) real world studies

Question 2

What categories can define eligibility?

Answer 2

It’s important to define eligibility for participants like conditions, age, sex and severity of illness

Question 3

What should the outcomes of an assessment be defined as?

Answer 3

Death
Disease
Recurrence
Disability
Distress
Discomfort

Question 4

What is important to remember regarding ethical approval?

Answer 4

You must have informed consent
Explain purpose randomisation
Alternative treatments
And can withdraw at any time

Question 5

What are MAMS?

Answer 5

They are multi arm multi stage controlled trials which allow simultaneous assessment of a number of treatments

Question 6

What are adaptive trials?

Answer 6

They are ongoing where changes and decisions are made while the trial is ongoing

Question 7

What is a false positive?

Answer 7

Rejection of a true null hypothesis

Question 8

What is a false negative?

Answer 8

Non-rejection of a false null hypothesis

Question 9

What is a true positive?

Answer 9

When the machine learning model predicts the condition correctly

Question 10

What is a true negative?

Answer 10

When machine learning model correctly predicts the negative condition

Question 11

What are the problems and solutions regarding recruitment?

Answer 11

Problems - eligibility criteria too restrictive, eligible patients not identified or invited

Solutions - increase recruitment sites, ensure criteria is appropriate, accurately estimate numbers of patients, ensure eligible patients are identified

Question 12

Intention to treat analysis?

Answer 12

All individuals who were randomised should be included in the analysis and they’re analysed scored to allocated treatment and not treatment received
Avoids bias

Question 13

What is absolute and relative risk?

Answer 13

ARR = risk in control - risk in intervention
RRR = (risk in control - risk in intervention)/risk in control

Question 14

How to calculate number needed to treat?

Answer 14

NNT = 1/ARR

Question 15

What are the 5 domains of bias risk?

Answer 15

Bias arising from randomisation process
Bias due to deviations from intended interventions
Bias due to missing some data
Bias in measurement of the outcome
Bias in selection of the reported result

Question 16

What is internal and external validity?

Answer 16

Internal validity are strong protections against bias such as randomisation, blinding and intention to treat analysis

External validity is focused on generalisability like are subjects are typically of patients using same