Please use this identifier to cite or link to this item:
http://hdl.handle.net/1942/36193
Full metadata record
DC Field | Value | Language |
---|---|---|
dc.contributor.author | Barrado, Leandro Garcia | - |
dc.contributor.author | BURZYKOWSKI, Tomasz | - |
dc.contributor.author | LEGRAND, Catherine | - |
dc.contributor.author | BUYSE, Marc | - |
dc.date.accessioned | 2021-12-14T19:08:59Z | - |
dc.date.available | 2021-12-14T19:08:59Z | - |
dc.date.issued | 2022 | - |
dc.date.submitted | 2021-12-11T21:06:32Z | - |
dc.identifier.citation | PHARMACEUTICAL STATISTICS, 21 (1), p. 209-219 | - |
dc.identifier.issn | 1539-1604 | - |
dc.identifier.uri | http://hdl.handle.net/1942/36193 | - |
dc.description.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. | - |
dc.description.sponsorship | Service Public de Wallonie, Grant/AwardNumber: First Entreprice Docteurnr. 8162, jointly awarded to InternationalDrug Development Institute (IDDI) SAand the Université Catholique de Louvain(UCLouvain). The research has been partly funded by the Service Public de Wallonie, under the research grant First Entreprice Doct-eur nr. 8162, jointly awarded to International Drug Development Institute (IDDI) SA and the Université Catholique deLouvain (UCLouvain). | - |
dc.language.iso | en | - |
dc.publisher | WILEY | - |
dc.rights | 2021 John Wiley & Sons Ltd.1 | - |
dc.subject.other | efficacy stopping | - |
dc.subject.other | group-sequential design | - |
dc.subject.other | interim analysis | - |
dc.subject.other | randomized clinical trial | - |
dc.subject.other | surrogate endpoint | - |
dc.title | Using an interim analysis based exclusively on an early outcome in a randomized clinical trial with a long-term clinical endpoint | - |
dc.type | Journal Contribution | - |
dc.identifier.epage | 219 | - |
dc.identifier.issue | 1 | - |
dc.identifier.spage | 209 | - |
dc.identifier.volume | 21 | - |
local.format.pages | 11 | - |
local.bibliographicCitation.jcat | A1 | - |
dc.description.notes | Barrado, LG (corresponding author), Int Drug Dev Inst IDDI, Ave Prov 30, B-1340 Louvain, Belgium. | - |
dc.description.notes | leandro.garciabarrado@iddi.be | - |
local.publisher.place | 111 RIVER ST, HOBOKEN 07030-5774, NJ USA | - |
local.type.refereed | Refereed | - |
local.type.specified | Article | - |
dc.identifier.doi | 10.1002/pst.2165 | - |
dc.identifier.pmid | 34505395 | - |
dc.identifier.isi | WOS:000694628500001 | - |
dc.identifier.eissn | 1539-1612 | - |
local.provider.type | wosris | - |
local.uhasselt.uhpub | yes | - |
local.description.affiliation | [Barrado, Leandro Garcia; Burzykowski, Tomasz; Buyse, Marc] Int Drug Dev Inst IDDI, Ave Prov 30, B-1340 Louvain, Belgium. | - |
local.description.affiliation | [Barrado, Leandro Garcia; Legrand, Catherine] Louvain Inst Data Anal & Modeling, Inst Stat Biostat & Actuarial Sci ISBA, Louvain, Belgium. | - |
local.description.affiliation | [Burzykowski, Tomasz; Buyse, Marc] Hasselt Univ, Data Sci Inst, I BioStat, Hasselt, Belgium. | - |
local.uhasselt.international | no | - |
item.fulltext | With Fulltext | - |
item.contributor | Barrado, Leandro Garcia | - |
item.contributor | BURZYKOWSKI, Tomasz | - |
item.contributor | LEGRAND, Catherine | - |
item.contributor | BUYSE, Marc | - |
item.accessRights | Restricted Access | - |
item.validation | ecoom 2022 | - |
item.fullcitation | Barrado, Leandro Garcia; BURZYKOWSKI, Tomasz; LEGRAND, Catherine & BUYSE, Marc (2022) Using an interim analysis based exclusively on an early outcome in a randomized clinical trial with a long-term clinical endpoint. In: PHARMACEUTICAL STATISTICS, 21 (1), p. 209-219. | - |
crisitem.journal.issn | 1539-1604 | - |
crisitem.journal.eissn | 1539-1612 | - |
Appears in Collections: | Research publications |
Files in This Item:
File | Description | Size | Format | |
---|---|---|---|---|
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 |
WEB OF SCIENCETM
Citations
1
checked on Apr 26, 2024
Page view(s)
34
checked on Sep 7, 2022
Download(s)
6
checked on Sep 7, 2022
Google ScholarTM
Check
Altmetric
Items in DSpace are protected by copyright, with all rights reserved, unless otherwise indicated.