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Title: | Using an early outcome as the sole source of information of interim decisions regarding treatment effect on a long-term endpoint: The non-Gaussian case | Authors: | Barrado, Leandro Garcia BURZYKOWSKI, Tomasz |
Issue Date: | 2024 | Publisher: | WILEY | Source: | Pharmaceutical statistics, | Status: | Early view | Abstract: | In randomized clinical trials that use a long-term efficacy endpoint, the follow-up time necessary to observe the endpoint may be substantial. In such trials, an attractive option is to consider an interim analysis based solely on an early outcome that could be used to expedite the evaluation of treatment's efficacy. Garcia Barrado et al. (Pharm Stat. 2022; 21: 209-219) developed a methodology that allows introducing such an early interim analysis for the case when both the early outcome and the long-term endpoint are normally-distributed, continuous variables. We extend the methodology to any combination of the early-outcome and long-term-endpoint types. As an example, we consider the case of a binary outcome and a time-to-event endpoint. We further evaluate the potential gain in operating characteristics (power, expected trial duration, and expected sample size) of a trial with such an interim analysis in function of the properties of the early outcome as a surrogate for the long-term endpoint. | Notes: | Barrado, LG (corresponding author), Int Drug Dev Inst IDDI, Ave Provinciale 30, B-1340 Louvain La Neuve, Belgium.; Barrado, LG (corresponding author), Louvain Inst Data Anal & Modeling, Inst Stat Biostat & Actuarial Sci ISBA, Louvain La Neuve, Belgium. leandro.garciabarrado@iddi.com |
Keywords: | efficacy stopping;group-sequential design;interim analysis;randomized clinical trial;surrogate endpoint | Document URI: | http://hdl.handle.net/1942/43265 | ISSN: | 1539-1604 | e-ISSN: | 1539-1612 | DOI: | 10.1002/pst.2398 | ISI #: | 001238761300001 | Rights: | 2024 John Wiley & Sons Ltd. | Category: | A1 | Type: | Journal Contribution |
Appears in Collections: | Research publications |
Files in This Item:
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Pharmaceutical Statistics - 2024 - Garcia Barrado - Using an early outcome as the sole source of information of interim.pdf Restricted Access | Early view | 1.77 MB | Adobe PDF | View/Open Request a copy |
EUSEBio - Manuscript Revision_TB_clean_TB_clean.pdf Until 2024-12-05 | Peer-reviewed author version | 702.02 kB | Adobe PDF | View/Open Request a copy |
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