Defined terms in the registry — page 2
195 documents of type defined term.
Documents
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The largest difference, specified before the trial, that would still be regarded as acceptable in a non-inferiority design. A non-inferiority conclusion is only as…
30 Jul 2026 -
A design in which participants who have responded during an open-label lead-in are randomised to continue or to discontinue. Establishes whether an effect requires…
30 Jul 2026 -
A design in which each participant receives more than one treatment in sequence, separated by a washout. Efficient for stable conditions and unsuitable where the…
30 Jul 2026 -
A period between treatments in a crossover design intended to allow the effect of the first to dissipate. A washout shorter than approximately five half-lives leaves…
30 Jul 2026 -
A trial that continues until a pre-specified number of endpoint events has accrued rather than for a fixed duration. Follow-up duration is therefore a consequence of the…
30 Jul 2026 -
An event-driven trial with a composite cardiovascular endpoint, conducted for several agents in this class as a regulatory requirement to exclude excess risk. A trial…
30 Jul 2026 -
A period before randomisation during which all participants receive the same treatment. A run-in that excludes non-responders or non-tolerators produces a randomised…
30 Jul 2026 -
The stepwise escalation of dose toward a target. Tolerability in this compound class depends on the titration schedule as much as on the compound, which the Institute…
30 Jul 2026 -
The dose reached after titration and administered for the remainder of the treatment period. The dose to which an effect estimate attaches.
30 Jul 2026 -
Loss of participants from a trial before its primary analysis timepoint. Differential attrition between arms threatens the comparability that randomisation established.
30 Jul 2026 -
Analysis of participants in the group to which they were randomised, regardless of the treatment they received. Preserves the comparison randomisation created and…
30 Jul 2026 -
Analysis restricted to participants who adhered to the protocol. Breaks the randomised comparison and is reported by the Institute only as a secondary analysis alongside…
30 Jul 2026 -
A precise statement of the quantity a trial is estimating, including how intercurrent events such as discontinuation or rescue therapy are handled. Two trials reporting…
30 Jul 2026 -
An event occurring after randomisation that affects the interpretation of the outcome, such as treatment discontinuation, rescue medication or death. The strategy for…
30 Jul 2026 -
Independent classification of endpoint events by a committee blinded to assignment. Reduces the risk that ascertainment differs between arms for events requiring…
30 Jul 2026 -
An independent group with access to unblinded accumulating data, empowered to recommend modification or termination. Its recommendation to stop for efficacy is a source…
30 Jul 2026 -
Termination of a trial before its planned completion, most often for efficacy at an interim analysis. Trials stopped early for efficacy systematically overestimate…
30 Jul 2026 -
Randomisation within strata defined by prognostic factors, to ensure balance on those factors. Stratification factors should be included in the primary analysis model.
30 Jul 2026 -
A trial designed to estimate effectiveness under ordinary conditions rather than efficacy under ideal ones. Typically has broader eligibility, less intensive follow-up…
30 Jul 2026 -
First administration to humans, evaluating safety, tolerability and pharmacokinetics, usually in small numbers and often in healthy volunteers. Efficacy endpoints in a…
30 Jul 2026 -
Exploratory clinical development establishing dose and preliminary efficacy. Phase 2 estimates in selected populations are systematically larger than the confirmatory…
30 Jul 2026 -
Confirmatory clinical development, adequately powered for a pre-specified primary endpoint in the population for which authorisation is sought.
30 Jul 2026 -
A study comparing several doses to characterise the dose–response relationship, usually conducted in phase 2 and usually not powered for a definitive efficacy conclusion…
30 Jul 2026 -
A period following the randomised phase in which all participants receive active treatment. Reports persistence and long-term tolerability; cannot re-establish…
30 Jul 2026 -
A range constructed so that, over repeated experiments, the stated proportion of such ranges would contain the true value. The Institute reports the interval alongside…
30 Jul 2026 -
The single value that best summarises the observed effect. Never reported by the Institute without its interval.
30 Jul 2026 -
The ratio of the instantaneous event rates in two arms, assumed constant over follow-up. A hazard ratio without an absolute risk difference does not convey how much…
30 Jul 2026 -
The assumption that the ratio of hazards between arms is constant over time. Where the assumption fails, a single hazard ratio summarises a time-varying effect and can…
30 Jul 2026 -
The difference in event probability between arms. Depends on baseline risk and is the measure from which a number needed to treat is derived.
30 Jul 2026 -
The reciprocal of the absolute risk difference: the number of people who must be treated for the stated duration for one additional person to benefit. Rises sharply as…
30 Jul 2026 -
The ratio of the odds of an event in two arms. Approximates the risk ratio only when the event is uncommon, and departs from it materially for common outcomes.
30 Jul 2026 -
The ratio of event probabilities in two arms. More directly interpretable than an odds ratio for common outcomes.
30 Jul 2026 -
Variation in effect estimates across studies beyond that expected from chance. Quantified imperfectly by the I-squared statistic and assessed by inspection of the…
30 Jul 2026 -
The proportion of observed variation across studies attributable to heterogeneity rather than to chance. Does not indicate whether the heterogeneity matters, and takes…
30 Jul 2026 -
A pooling model assuming that true effects vary across studies and estimating their distribution. Produces wider intervals than a fixed-effect model and gives relatively…
30 Jul 2026 -
A pooling model assuming a single true effect common to all contributing studies. Appropriate only where that assumption is defensible.
30 Jul 2026 -
A figure showing each contributing estimate with its interval against a line of no effect, with the pooled estimate where one is presented. The standard figure of the…
30 Jul 2026 -
A scatter of effect estimate against study precision, inspected for asymmetry as an indication of small-study effects. Uninformative below approximately ten contributing…
30 Jul 2026 -
The number of participants or events a body of evidence would require to be as informative as an adequately powered single trial. Used by the Institute in assessing…
30 Jul 2026 -
A property of a result relative to a threshold, not a measure of effect size or of importance. The Institute reports effect sizes alongside any statement of significance…
30 Jul 2026
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