What is a superiority trial?
A superiority trial is designed to detect a difference between treatments. The first step of the analysis is usually a test of statistical significance to evaluate whether the results of the trial are consistent with the assumption of there being no difference in the clinical effect of the two treatments.
What is the null hypothesis in a non-inferiority trial?
The null hypothesis in non-inferiority trials is that new treatment is inferior to standard treatment. The alternative hypothesis is the new treatment is non-inferior to standard treatment.
What is the difference between a non-inferiority trial and a superiority trial?
In comparison studies with a current therapy, non-inferiority is used to demonstrate that the new therapy provides at least the same benefit to the patient. Superiority trials are always used when comparisons are made to placebo or vehicle treatments.
Can non-inferiority trials show superiority?
In a non-inferiority trial, the focus is on the lower bound margin, what happens at the upper end is not of primary concern in this type of trial design. One can also declare superiority in a non-inferiority trial if the lower limit of CI of the new treatment is above the non-inferiority margin and above zero.
Why do a non-inferiority trial?
The non-inferiority trial is a useful alternative in such situations. As the name suggests, the aim is to show that the new treatment is not inferior to the existing one – that is, it is either equally effective or better.
What does a non-inferiority trial mean?
By definition, a non-inferiority trial aims to demonstrate that the test product is not worse than the comparator by more than a small pre-specified amount. This amount is known as the non-inferiority margin, or delta. Clinicians must know who has chosen the margin, and why.
How do you test for non-inferiority?
In noninferiority testing, a common practice is to set the value of δ to a fraction, f, of the lower limit of a confidence interval of the difference between the current therapy and the placebo obtained from a meta-analysis.
What is noninferiority margin?
A noninferiority margin (1.31%, M2) was defined for the point‐estimate method to preserve 50% of the pooled point estimate (2.62%, M1). This margin was exceeded by the upper limit of the CI form SPORTIF V (1.64%), therefore, noninferiority was not demonstrated.
How do you determine non-inferiority margin?
In order to demonstrate non-inferiority, the recommended approach is to pre-specify a margin of non- inferiority in the protocol. After study completion, a two-sided 95% confidence interval (or one-sided 97.5% interval) for the true difference between the two agents will be constructed.
What is a superiority margin?
There is a percentage above 70% at which the difference between the two treatments is no longer considered ignorable. The difference between these two percentages is called the margin of superiority.
How do you calculate sample size for non inferiority trials?
The sample size required for a non-inferiority clinical trial can be calculated using the formula in Figure 1 [5],[6]. Table 4 gives common Normal deviates for different percentiles. For example, for β = 0.1, we would have x = 0.1 and Z1-x = 1.282, while for α = 0.05, we would have x = 0.025 and Z1-x = 1.96.
What information is needed to calculate sample size for an equivalence study?
To determine the trial’s sample size, researchers must first define the null hypothesis that is appropriate for the equivalence study. πS and πT represent the effects of the standard treatment and of the new treatment, respectively.
What does Noninferiority mean?
By definition, a non-inferiority trial aims to demonstrate that the test product is not worse than the comparator by more than a small pre-specified amount. This amount is known as the non-inferiority margin, or delta.
What is intention to treat vs per protocol?
While an analysis according to the ITT principle aims to preserve the original randomization and to avoid potential bias due to exclusion of patients, the aim of a per-protocol (PP) analysis is to identify a treatment effect which would occur under optimal conditions; i.e. to answer the question: what is the effect if …
What is the intention to treat effect?
Intention-to-treat analysis is a method for analyzing results in a prospective randomized study where all participants who are randomized are included in the statistical analysis and analyzed according to the group they were originally assigned, regardless of what treatment (if any) they received.
What is full analysis set in clinical trial?
The Full Analysis Set (FAS) will be defined as all patients randomly assigned to a treatment group having at least one efficacy assessment after randomisation.
What is ICH e9?
This document provides guidance on the design, conduct, analysis and evaluation of clinical trials of an investigational product in the context of its overall clinical development. It presents a structured framework for clinical trial planning, conduct, data collection and interpretation of data analyses.
What is per protocol population?
What is a PP Population, or ‘Per Protocol’ Population in a Clinical Trial? The per protocol population, or PP population is usually defined as all patients completing the study without major protocol deviations – that is, those who followed the rules of the study.
What are topline results in clinical trials?
Top Line Data from a Clinical Trial shall mean the audited, quality-controlled tables, listings and figures in reasonable and customary form reflecting all results of the Clinical Trial.
What is efficacy analysis?
Efficacy Analysis In essence, it determines the efficacy of the new therapy under ideal circumstances, i.e., it tests the benefit of taking the therapy as opposed to the alternative.
What is Open label in clinical trials?
An open-label trial, or open trial, is a type of clinical trial in which information is not withheld from trial participants. In particular, both the researchers and participants know which treatment is being administered.
What is a Phase 3 clinical trial?
Phase III trials compare a new drug to the standard-of-care drug. These trials assess the side effects of each drug and which drug works better. Phase III trials enroll 100 or more patients. Often, these trials are randomized. This means that patients are put into a treatment group, called trial arms, by chance.
What is a washout period in clinical trial?
According to the U.S. National Library of Medicine, a washout period is defined as “a period of time during a clinical study when a participant is taken off a study drug or other medication in order to eliminate the effects of the treatment.” So basically, it means a time when a lung cancer patient is not being …