A confirmatory trial is an adequately

controlled trial A randomized controlled trial (or randomized control trial; RCT) is a form of scientific experiment used to control factors not under direct experimental control. Examples of RCTs are clinical trials that compare the effects of drugs, surgical ...

where

hypotheses A hypothesis (plural hypotheses) is a proposed explanation for a phenomenon. For a hypothesis to be a scientific hypothesis, the scientific method requires that one can test it. Scientists generally base scientific hypotheses on previous obser ...

are stated in advance and evaluated according to a

protocol Protocol may refer to: Sociology and politics * Protocol (politics), a formal agreement between nation states * Protocol (diplomacy), the etiquette of diplomacy and affairs of state * Etiquette, a code of personal behavior Science and technology ...

. This type of trial may be implemented when it is necessary to provide additional or firm evidence of efficacy or safety. The mechanism of the trial implements a key hypothesis of interest which is rigorously tested at the end of the confirmatory trial and directly follows the predefined primary objective of the trial. Of importance in a confirmatory trial is the process of estimating with due precision potential effects attributable to the treatment, the quantity of effects and relating these effects to their clinical significance. According to the

International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) is an initiative that brings together regulatory authorities and pharmaceutical industry to discuss scientific and technical aspects of pha ...

: "Confirmatory Trials are intended to provide firm evidence in support of claims and hence adherence to

protocols Protocol may refer to: Sociology and politics * Protocol (politics), a formal agreement between nation states * Protocol (diplomacy), the etiquette of diplomacy and affairs of state * Etiquette, a code of personal behavior Science and technology ...

and

standard operating procedures A standard operating procedure (SOP) is a set of step-by-step instructions compiled by an organization to help workers carry out routine operations. SOPs aim to achieve efficiency, quality output, and uniformity of performance, while reducing misc ...

is particularly important; unavoidable changes should be explained and documented, and their effect examined." Basic outline of the process of a confirmatory trial # A design justification and statistical aspects such as the principal features of the planned analysis, is established as part of the initial protocol. # The confirmatory trial should be concise in addressing only a specific number of questions. # The confirmatory trial should clarify key clinical questions relevant to efficacy and/or safety clearly and definitively. # The target patient population segmented for the trial is clearly outlined, understood and defined as this may influence the test sites and scientists (practitioners, specialists, etc.) involved.

References

Further reading