Topic 1: Basic Information Flashcards
When do you measure baseline characteristics
Prior to randomisation
When do you compare outcomes from
Point of randomisation
Why do you take baseline characteristics
To compare the characteristics of patients in your trial to the characteristics of the population
What does baseline data tell you
Who you can generalise the results to
What is a subgroup effect
The effect of certain characteristics that can be used to predict outcomes, given a prognostic model and can therefore be predictors of the size of treatment effect
What is often looked at in exploratory analysis
Subgroup effects
Common exclusions of patients
Learning difficulties, elderly, frail, pregnant
How many primary research questions are there usually
One
What does PICOTC stand for
Population, Intervention, Control, Outcome, Timing and Context
Is the design and cost of the trial dictated by the primary or secondary research question
Primary
Secondary Questions are regarded as exploratory, true or false
True
What is the PICOTC framework used for
Designing Research questions
Examples of Secondary Objectives
Adverse effects, withdrawals, quality of life, subgroup effects, what baseline factors predict a reduction in score etc.
What happens in Phase 1 Trial
Look at if drug is harmful to humans, uses small number of participants. Might look at what the maximum tolerated dose is
What happens in Phase 2 Trials
Does it have the potential to be beneficial to patients. Make a stop-go decision.
What happens in Phase 3 Trials
A randomised, large, comparative trial. Confirmatory. Confirms effectiveness within a population.
What is efficacy
Treatment effect produced under ideal conditions
What is effectiveness
Treatment effect that would be produced in real clinical practice
When is an explanatory approach typically taken
Earlier stage trials
What is the sample like in an explanatory trial
Carefully defined patients in a research clinic
What is the sample like in a pragmatic trial
Reflects variation between treatments that occurs in real clinical practice in order to reduce the chance of over-estimating treatment effect,
What are the outcomes in an explanatory trial usually
Biological
What are the outcomes in a pragmatic trial usually
Broad health effects
What is the aim of an explanatory trial
Further scientific knowledge
What is the aim of a pragmatic trial
Inform choice of treatment for a patient
Is per-protocol explanatory or pragmatic
Explanatory
Is intention to treat explanatory or pragmatic
Pragmatic
What does the intention to treatment analysis population consist of
All randomised patients in analysis regardless of anything that happens after randomisation.
