PoD - Clinical Trials

Question 1

why are clinical trials important?

Answer 1

• provide evidence to determine best medical practice for treatment

Question 2

what does a clinical trial need to determine?

Answer 2

• does it work?
• what dose is therapeutic?
• what dose is toxic?
• is it safe?
• is it necessary?

Question 3

what are the stages in drug development?

Answer 3

• drug discovery
• pre-clinical development (animal studies)
• phase 1 - clinical development (healthy volunteers - pharmacokinetic, metabolic and pharmacodynamic data)
• phase 2 - drug to patients (if volunteers are healthy after drug, drug given to patient. provides some evidence of efficacy and determines dose range)
• phase 3 - formal therapeutic trial (efficacy established, evidence of safety - can now apply for license)
• phase 4 - post-marketing surveillance (long term safety)

Question 4

what is a pilot study?

Answer 4

• not monitoring drug but testing study design

Question 5

what is a double blind trial?

Answer 5

• patient AND doctor don’t know who is in what group

- BEST kind of study as no-one can predict outcome

Question 6

what is a single blind study?

Answer 6

• only the patient is blinded

Question 7

what is a placebo controlled study?

Answer 7

• half of patients receive active drug, half receive placebo
• compare outcome in two groups - is the drug actually effective?

Question 8

what is a cross-over design study?

Answer 8

• 100 patients - 50 study drug, 50 comparative therapy
• cross over/wash out period
• groups swap over so those getting study drug now get other therapy
• less people required to test, but more difficult to control

Question 9

what is randomised control clinical trial?

Answer 9

• gold standard

- patients assigned at random to either treatment or control

Question 10

what are the disadvantages of a randomised control clinical trial?

Answer 10

• not generalisable results (subjects may not represent general patient population)
• double the number of patients recruited
• acceptability of randomisation process (some patients will refuse only wanting treatment)

Question 11

what is a superiority design?

Answer 11

• show that the new treatment is better than the control or standard

Question 12

what is a non-inferiority design?

Answer 12

• show that the new treatment isn’t worse than the standard significantly
• show the new treatment would have beaten a placebo