A pivotal trial is typically a

Phase III clinical trial The phases of clinical research are the stages in which scientists conduct experiments with a health intervention to obtain sufficient evidence for a process considered effective as a medical treatment. For drug development, the clinical phases ...

in the multi-year process of

clinical research Clinical research is a branch of healthcare science that determines the safety and effectiveness ( efficacy) of medications, devices, diagnostic products and treatment regimens intended for human use. These may be used for prevention, treatm ...

intended to demonstrate and confirm the safety and efficacy of a treatment – such as a

drug candidate In the fields of medicine, biotechnology and pharmacology, drug discovery is the process by which new candidate pharmaceutical drug, medications are discovered. Historically, drugs were discovered by identifying the active ingredient from tradit ...

medical device A medical device is any device intended to be used for medical purposes. Significant potential for hazards are inherent when using a device for medical purposes and thus medical devices must be proved safe and effective with reasonable assura ...

or clinical diagnostic procedure – and to estimate the incidence of common

adverse effect An adverse effect is an undesired harmful effect resulting from a medication or other intervention, such as surgery. An adverse effect may be termed a "side effect", when judged to be secondary to a main or therapeutic effect. The term complica ...

s. A successful pivotal trial is required as evidence for drug marketing approval by the

United States Food and Drug Administration The United States Food and Drug Administration (FDA or US FDA) is a federal agency of the Department of Health and Human Services. The FDA is responsible for protecting and promoting public health through the control and supervision of food s ...

(FDA). In drug research, a pivotal Phase III trial may be referred to as a "therapeutic confirmatory study", and is conducted in a large number (hundreds to thousands) of subjects. Such pivotal trials are also designed to discover and estimate the prevalence of common adverse events, but based on their size only have the

statistical power In statistics, the power of a binary hypothesis test is the probability that the test correctly rejects the null hypothesis (H_0) when a specific alternative hypothesis (H_1) is true. It is commonly denoted by 1-\beta, and represents the chances ...

to establish an adverse effect rate of not less than 1 in 100 subjects. In an analysis of pivotal trials on medical devices conducted between 2006 and 2013, the

median In statistics and probability theory, the median is the value separating the higher half from the lower half of a data sample, a population, or a probability distribution. For a data set, it may be thought of as "the middle" value. The basic fe ...

duration was three years, with another two years needed for FDA review and approval for marketing. In 2017 in the United States, the median cost of a pivotal trial across all clinical indications was US$19 million. The cost of a pivotal trial increased when more subjects were added to clarify a treatment effect, when active drug comparators were used to improve understanding of the trial drug characteristics, or when specific

clinical endpoint Clinical endpoints or clinical outcomes are Outcome measure, outcome measures referring to occurrence of disease, symptom, Medical sign, sign or laboratory abnormality constituting a target outcome in clinical trial, clinical research trials. The t ...

s were measured rather than using surrogate outcomes.

References

{{reflist Clinical trials