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Flashcards in Clinical Trial Design Deck (14):

Why are clinical trials important?

-provide evidence
-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
-£100 million


What is involved in pre clinical development?

-animal pharmacology (dose, adverse effects)
-animal toxicology (teratogenicity, fertility, mutagenicity)
-tissue culture


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?

-double blind
-single blind
-placebo controlled
-comparison with other therapy
-cross over


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


What does p<0.05 mean?

Usually represents significant results