Clinical Trials

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❘❙❚■ GlossaryHazard ratioIn survival analysis, the hazard (rate) represents an instantaneous event rate(incidence rate) at a certain time for an individual who has not experienced anevent at that time. A hazard ratio compares two hazards of having an eventbetween two groups. If the hazard ratio is 2.0, then the hazard of having an eventin one group is twice the hazard of having an event in the other group. Thecomputation of the hazard ratio assumes that the ratio is consistent over time(proportional hazards assumption).Hypothesis testing or significance testingA statistical procedure for assessing whether an observed treatment differencewas due to random error (chance) by calculating a P-value using the observedsample statistics such as mean, standard deviation, etc. The P-value is theprobability that the observed data or more extreme data would have occurredif the null hypothesis (ie, no true difference) were true. If the calculated P-valueis a small value (eg,
<strong>Clinical</strong> <strong>Trials</strong>: A Practical Guide ■❚❙❘Meta-analysisThe systematic review and evaluation of the evidence from two or moreindependent studies asking the same clinical question to yield an overall answerto the question.Normal distributionA bell-shaped symmetric distribution for a continuous variable with the highestfrequency at a mean value and the lowest frequency further away from this meanvalue. A normal distribution can be completely described by two parameters:mean (μ) and variance (σ 2 ). In the special case of μ = 0 and σ 2 = 1, it is called thestandard normal distribution.Number needed to treat (NNT)This term is often used to describe how many patients would need to be given atreatment to prevent one event. It is determined from the absolute differencebetween one treatment and another. For example, in a randomized study, a groupreceiving treatment A had a death rate of 12.5% and a group on treatment Bhad a death rate of 15.0% in groups matched for size and length of follow-up.Comparing the two treatments there was an absolute risk reduction of15% – 12.5% = 2.5% for treatment A. From this we can derive that the NNT(= 1/0.025) is 40. This means 40 patients need to be given treatment A rather thantreatment B to prevent one additional death.Odds ratio (OR) and risk ratio (RR)These terms compare the probability of having an event between two groupsexposed to a risk factor or treatment. The risk ratio (RR) is the ratio of theprobability of occurrence of an event between two groups. The odds ratio (OR) isthe ratio of patients with and without an event in each group. For example, if thenumber of deaths in the treatment and control arms (both of sample size 100) ofa randomized study are 50 and 25 respectively, the RR = (50/100) / (25/100) = 2.The treatment group has a 2-fold relative risk of dying compared with the controlgroup. The OR = (50/50) / (25/75) = 3, indicating that the odds of death in thetreatment arm is 3-fold that of the control arm.457
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❘❙❚■ ContributorsStephen L
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Clinical Trials: A Practical Guide
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■■❚❙❘Chapter 1RandomizedC
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■■❚❙❘Chapter 4EndpointsAm
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■■❚❙❘Part IIAlternativeTr
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■■❚❙❘Part IIIBasics ofSta
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■■❚❙❘Chapter 17Types of D
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■■❚❙❘Chapter 19Comparison
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■■❚❙❘Chapter 20Comparison
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■■❚❙❘Part IVSpecial Trial
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■■❚❙❘Chapter 22Intention-
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■■❚❙❘Chapter 24Regression
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■■❚❙❘Chapter 25Adjustment
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■■❚❙❘Chapter 32Overview o
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■■❚❙❘Chapter 33Trial Prof
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■■❚❙❘Chapter 35Use of Tab
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