CMB2000/L06 Clinical Trials II

Question 1

Define ethics.

Answer 1

Moral principles that govern a person’s behaviour or the conducting of an activity

Question 2

What is informed consent?

Answer 2

An intent that human participants enter research voluntarily with full information about what it means for them to take part and that they give consent before they enter the research

Question 3

What is bias?

Answer 3

Systematic errors that encourage one outcome over others
Investigators may come to the wrong conclusions about beneficial and harmful effects of interventions

Question 4

Define a control group.

Answer 4

The standard to which comparisons are made in an experiment
A group of participants who do not receive experimental treatment

Question 5

How are control groups allocated?

Answer 5

Clinician choice
Patient choice
Different consultants
Different hospitals
Consenting and non-consenting groups
ALL can lead to bias

Question 6

Define randomisation.

Answer 6

The process by which treatments are assigned to participants by chance rather than choice
To avoid bias

Question 7

Give 3 types of randomisation.

Answer 7

Simple
Block
Stratified randomisation

Question 8

What is simple randomisation?

Answer 8

Based on a single sequence of random assignments

Question 9

Give an advantage and a disadvantage to simple randomisation.

Answer 9

+ simple
+ easy to implement
- could result in unequal number amongst groups

Question 10

What is block randomisation?

Answer 10

Designed to randomise subjects into groups that result in equal sample sizes

Question 11

Give an advantage and a disadvantage of block randomisation.

Answer 11

+ balance in sample size
- can predict blocks
- groups may not be comparable in terms of covariates

Question 12

What is stratified randomisation?

Answer 12

Addresses the need to control and balance under influence of covariates
Achieved by generating a separate block for each combination of covariates

Question 13

Give an advantage and a disadvantage of stratified randomisation.

Answer 13

+ simple for small trials
+ takes covariates into consideration
- complicated if many covariates
- subjects have to identified before assignment

Question 14

Give 2 practical considerations of randomisation.

Answer 14

Central telephone or web randomisation is ideal
Sealed opaque envelopes

Question 15

What is blinding?

Answer 15

Studies designed to prevent members of research teams and study participants from influencing results

Question 16

Who knows which treatment is assigned in single-blinded studies?

Answer 16

Only the researcher

Question 17

Who knows which treatment is assigned in double-blinded studies?

Answer 17

No-one until the trial is over

Question 18

What is the process of hypothesis testing?

Answer 18

Test if the alternative hypothesis (H1) is more probable than the null hypothesis (H0) using statistics

Question 19

Give 3 considerations when setting up and running a clinical trial.

Answer 19

Blinding
Consent
Controls
Randomisation
Sample size

Question 20

What is the true endpoint of a clinical trial?

Answer 20

A clinically meaningful endpoint that directly measures patients

Question 21

What is a surrogate endpoint of a clinical trial?

Answer 21

A measurement of a specific outcome used in place of another as a predictor to tell if a treatment works
Usually occurs before a true endpoint and yields conclusions about effect of treatment on true endpoint

Question 22

What is dissemination of results?

Answer 22

Getting the findings of your research to the people who can make use of them to maximise benefit of the research

Question 23

Give 3 principles of good dissemination.

Answer 23

Stakeholder engagement
Format
Utilise opportunities
Context
Timing

Question 24

Give 3 things to consider when disseminating results.

Answer 24

Objective
Audience
Timeline
Resources
Strategy