Document registry — page 6
Every document the Institute has published, 1,621 in total, with its identifier, type, certainty rating and review date.
Documents
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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 -
The magnitude of a difference, expressed on the scale of the outcome or standardised. A significant result with an effect size below the minimal clinically important…
30 Jul 2026 -
The smallest change in an outcome that a patient would identify as meaningful. Frequently contested and frequently unavailable, and the Institute names the source of any…
30 Jul 2026 -
The inflation of false-positive rates that follows from testing many hypotheses. Controlled by a pre-specified testing hierarchy, whose position each reported endpoint…
30 Jul 2026 -
A pre-specified order in which endpoints are tested, in which testing stops at the first failure. An endpoint below the point of failure is not confirmatory regardless…
30 Jul 2026 -
Observations not collected. The assumption under which they are handled is rarely testable and is the most common unstated assumption in a trial report.
30 Jul 2026 -
A method for handling missing data by generating several completed datasets under a stated model and combining the results. Depends entirely on the plausibility of the…
30 Jul 2026 -
The outcome on which a trial is powered and against which its success is judged. A trial has one primary endpoint, or a small number designated co-primary with an…
30 Jul 2026 -
An outcome measured alongside the primary endpoint. Confirmatory only where it occupies a place in a pre-specified testing hierarchy that was reached.
30 Jul 2026 -
An outcome analysed without multiplicity control and without pre-specified inferential status. Hypothesis-generating in every case, including where the result is…
30 Jul 2026 -
A measurement substituted for a clinical outcome on the assumption that a change in one predicts a change in the other. Validation of that assumption requires…
30 Jul 2026 -
An outcome a person would notice or care about directly: death, an event, a symptom, a function. Distinguished throughout the document set from a surrogate.
30 Jul 2026 -
An endpoint counting the first occurrence of any of several component events. Interpretable only when the components are of comparable importance and move in the same…
30 Jul 2026 -
Major adverse cardiovascular events. Conventionally a three-point composite of cardiovascular death, non-fatal myocardial infarction and non-fatal stroke; four- and…
30 Jul 2026 -
An outcome reported directly by the participant without interpretation by a clinician. At elevated risk of bias where the intervention produces a perceptible effect that…
30 Jul 2026 -
An analysis reporting the proportion of participants reaching a defined threshold. Reported by the Institute alongside the mean because a mean conceals the distribution…
30 Jul 2026 -
The outcome the Institute designates as primary for an indication across its whole document set, so that estimates for different compounds in that indication are…
30 Jul 2026 -
An effect expressed as the difference between the change in the active arm and the change in the placebo arm. The only form in which the Institute reports a within-arm…
30 Jul 2026 -
Any untoward occurrence in a participant, whether or not attributed to treatment. Ascertained by spontaneous report in most trials, which underestimates incidence…
30 Jul 2026
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