Flashcards in Clinical Trial Design Deck (14):
Why are clinical trials important?
-to test efficacy compared to placebo and other drugs
-to test safety
Why are clinical trials conducted?
What works in theory might not be best practice
What is the cost of a clinical trial from chemical structure to licensed drug?
-more than 10 years
What is involved in pre clinical development?
-animal pharmacology (dose, adverse effects)
-animal toxicology (teratogenicity, fertility, mutagenicity)
What does phase 1 in clinical development involve?
-Volunteer studies= clinical pharmacology in normal volunteers generating pharmacokinetic, metabolic and pharmacodynamics data
What does phase 2 in clinical development involve?
-clinical investigations to confirm kinetics and dynamics in patients
-provides some evidence of efficacy and identifies a likely dosage range
What does phase 3 in clinical development involve?
-formal therapeutic trials where efficacy will be established and evidence of safety obtained
-at completion, all data is submitted as an application to the regulatory authority for a license to sell the drug
What does phase 4 of clinical development involve?
-post-marketing surveillance to produce evidence of long term safety
What does a pilot study do?
Tests study design
What kind of trials are there?
-comparison with other therapy
What are the disadvantages of randomised control trials?
-Subjects may not represent general patient population
-twice as many new patients needed for the study
-some physicians will refuse (PFO closure)
-some patients will refuse (want treatment)
-complexity of randomisation methods
Superiority vs Non-inferiority trials
-superiority design show that the new treatment is better than the control or standard
-non-inferiority show that the new treatments is not worse that the standard by more than a margin or would have beaten placebo if a placebo arm had been included
What can act as end points to a trial?
-no of hospital admissions
-lowering of bp
-comparison of pain or mood