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Title: | Using an interim analysis based exclusively on an early outcome in a randomized clinical trial with a long-term clinical endpoint | Authors: | Barrado, Leandro Garcia BURZYKOWSKI, Tomasz LEGRAND, Catherine BUYSE, Marc |
Issue Date: | 2022 | Publisher: | WILEY | Source: | PHARMACEUTICAL STATISTICS, 21 (1), p. 209-219 | Abstract: | In RCTs with an interest in a long-term efficacy endpoint, the follow-up time necessary to observe the endpoint may be substantial. In order to reduce the expected duration of such trials, early-outcome data may be collected to enrich an interim analysis aimed at stopping the trial early for efficacy. We propose to extend such a design with an additional interim analysis using solely early-outcome data in order to expedite the evaluation of treatment's efficacy. We evaluate the potential gain in operating characteristics (power, expected trial duration, and expected sample size) when introducing such an early interim analysis, in function of the properties of the early outcome as a surrogate for the long-term endpoint. In the context of a longitudinal age-related macular degeneration (ARMD) ophthalmology trial, results show potentially substantial gains in both the expected trial duration and the expected sample size. A prerequisite, though, is that the treatment effect on the early outcome has to be strongly correlated with the treatment effect on the long-term endpoint, that is, that the early outcome is a validated surrogate for the long-term endpoint. | Notes: | Barrado, LG (corresponding author), Int Drug Dev Inst IDDI, Ave Prov 30, B-1340 Louvain, Belgium. leandro.garciabarrado@iddi.be |
Keywords: | efficacy stopping;group-sequential design;interim analysis;randomized clinical trial;surrogate endpoint | Document URI: | http://hdl.handle.net/1942/36193 | ISSN: | 1539-1604 | e-ISSN: | 1539-1612 | DOI: | 10.1002/pst.2165 | ISI #: | WOS:000694628500001 | Rights: | 2021 John Wiley & Sons Ltd.1 | Category: | A1 | Type: | Journal Contribution | Validations: | ecoom 2022 |
Appears in Collections: | Research publications |
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Pharmaceutical Statistics - 2021 - Garcia Barrado - Using an interim analysis based exclusively on an early outcome in a.pdf Restricted Access | Published version | 1.03 MB | Adobe PDF | View/Open Request a copy |
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