intro to clinical research Flashcards
what is efficacy?
does a treatment work under controlled conditions
what is effectiveness?
does a treatment work in the real-world
what is cost-effectiveness?
does a treatment deliver sufficient improvement to justify the cost incurred
what are the 5 stages of clinical trial
Preclinical: cell cultures/animal models. testing drug mechanism of action, pharmacodynamics and pharmacokinetics etc.
Phase I: 20-80 healthy volunteers testing safety
Phase II: several hundred patient participants. testing efficacy and safety (patients will have disease)
Phase III: hundreds or thousands of patient participants. testing efficacy against placebo and is randomised
Phase IV: hundreds or thousands of patient participants. testing long term clinical effectiveness.
what are 3 sub-types of randomisation
- stratified randomisation
- blocked randomisation
- cluster randomisation
what is stratified randomisation
when you group your population by strata i.e. age and then randomise people within each strata
what is block randomisation
where you allocate a certain number to treatment and a certain number to control in any time period
what is clustered randomisation?
when you randomise groups rather than individuals to prevent one individuals behaviour from affecting the behaviour of the others in your group
what does blinding participants reduce
performance bias
what does blinding clinicians reduce?
detection bias
what is the placebo effect
a perceived/measured improvement in a patient’s health even though they have taken an inert medication or a sham procedure
what is the benefit of adaptive study designs?
allows for much more rapid progeress and prevent the continuation of a study when the results are already clear.
