Trials and stats Flashcards
(16 cards)
What is the aim of a phase 1 trial?
Seeking maximum tolerated dose (MTD) e.g. toxicity
What is the definition of dose limiting toxicity (DLT)?
unacceptable toxicity within cycle 1
what is the definition of maximum tolerated dose (MTD)?
where 20-30% have observed DLT
How is the recommended phase 2 dose (RP2D)?
Usually ≤ MTD
What is the aim of a phase 2 study?
Characterise safety and toxicity of drug therapy
What is the difference between Adverse event (AE) and Reaction (AR)
AE are not related to treatment but AR are
Who can determine casualty between AE and drug in a trial?
medical trained fellow and the principle investigator
Who is responsible for the OVERALL conduct
of a trial?
Sponsor
What does “fast track” relate to in facilitating faster drug approval?
Speeds up new drug review
What does “break through” relate to in facilitating faster drug approval?
shortens development time of promising new therapy which has preclinical data
What does “Priority review” relate to in facilitating faster drug approval?
review at 6m rather than 10m
What does “Accelerated review” relate to in facilitating faster drug approval?
uses surrogate endpoint in drugs with benefit in serious conditions. End points will be confirmed later.
What does random assignment do in RCT?
- removes bias
- produces comparable groups
- allows statistical test to have significance
Disadvantages of RCT?
Not always generalisable
Large recruitment needed
Acceptability of randomness
Administrative complexity
What type of biomarkers are there?
risk/predisposition screening diagnostic prognostic predictive Pharmacological
What is an example of a diagnostic biomarker?
Mammogram
