Control Clin Trials. 2002 Dec;23(6):607-25.

Statistical challenges in the evaluation of surrogate endpoints in randomized trials.

Molenberghs G, Buyse M, Geys H, Renard D, Burzykowski T, Alonso A.

Source

Limburgs Universitair Centrum, tUL, Center for Statistics, Biostatistics, Diepenbeek, Belgium. geert.molenberghs@luc.ac.be

Abstract

The validation of surrogate endpoints has been studied by Prentice, who presented a definition as well as a set of criteria that are equivalent if the surrogate and true endpoints are binary. Freedman et al. supplemented these criteria with the so-called proportion explained. Buyse and Molenberghs proposed to replace the proportion explained by two quantities: (1). the relative effect, linking the effect of treatment on both endpoints, and (2). the adjusted association, an individual-level measure of agreement between both endpoints. In a multiunit setting, these quantities can be generalized to a trial-level measure of surrogacy and an individual-level measure of surrogacy. In this paper, we argue that such a multiunit approach should be adopted because it overcomes difficulties that necessarily surround validation efforts based on a single trial. These difficulties are highlighted.

Comment in

Control Clin Trials. 2002 Dec;23(6):703-7.

PMID:: 12505240; [PubMed - indexed for MEDLINE]

Publication Types, MeSH Terms

Publication Types

MeSH Terms

LinkOut - more resources

Full Text Sources

Other Literature Sources

Libraries

LinkOut Holdings

Supplemental Content

Save items

Related citations in PubMed

Evaluation of surrogate endpoints in randomized experiments with mixed discrete and continuous outcomes. [Stat Med. 2001]
Evaluation of surrogate endpoints in randomized experiments with mixed discrete and continuous outcomes.
Molenberghs G, Geys H, Buyse M. Stat Med. 2001 Oct 30; 20(20):3023-38.
The validation of surrogate endpoints in meta-analyses of randomized experiments. [Biostatistics. 2000]
The validation of surrogate endpoints in meta-analyses of randomized experiments.
Buyse M, Molenberghs G, Burzykowski T, Renard D, Geys H. Biostatistics. 2000 Mar; 1(1):49-67.
Validation of surrogate markers in multiple randomized clinical trials with repeated measurements: canonical correlation approach. [Biometrics. 2004]
Validation of surrogate markers in multiple randomized clinical trials with repeated measurements: canonical correlation approach.
Alonso A, Geys H, Molenberghs G, Kenward MG, Vangeneugden T. Biometrics. 2004 Dec; 60(4):845-53.
Review A unified framework for the evaluation of surrogate endpoints in mental-health clinical trials. [Stat Methods Med Res. 2010]
Review A unified framework for the evaluation of surrogate endpoints in mental-health clinical trials.
Molenberghs G, Burzykowski T, Alonso A, Assam P, Tilahun A, Buyse M. Stat Methods Med Res. 2010 Jun; 19(3):205-36. Epub 2009 Jul 16.
Review Information-theory based surrogate marker evaluation from several randomized clinical trials with continuous true and binary surrogate endpoints. [Clin Trials. 2007]
Review Information-theory based surrogate marker evaluation from several randomized clinical trials with continuous true and binary surrogate endpoints.
Pryseley A, Tilahun A, Alonso A, Molenberghs G. Clin Trials. 2007; 4(6):587-97.

See reviews... See all...

Cited by 5 PubMed Central articles

ASSESSING SURROGATE ENDPOINTS IN VACCINE TRIALS WITH CASE-COHORT SAMPLING AND THE COX MODEL. [Ann Appl Stat. 2008]
ASSESSING SURROGATE ENDPOINTS IN VACCINE TRIALS WITH CASE-COHORT SAMPLING AND THE COX MODEL.
Qin L, Gilbert PB, Follmann D, Li D. Ann Appl Stat. 2008 Mar; 2(1):386-407.
On assessing surrogacy in a single trial setting using a semicompeting risks paradigm. [Biometrics. 2009]
On assessing surrogacy in a single trial setting using a semicompeting risks paradigm.
Ghosh D. Biometrics. 2009 Jun; 65(2):521-9.
Evaluating a surrogate endpoint at three levels, with application to vaccine development. [Stat Med. 2008]
Evaluating a surrogate endpoint at three levels, with application to vaccine development.
Gilbert PB, Qin L, Self SG. Stat Med. 2008 Oct 15; 27(23):4758-78.

See all...

Related information

Related Citations
Calculated set of PubMed citations closely related to the selected article(s) retrieved using a word weight algorithm. Related articles are displayed in ranked order from most to least relevant, with the “linked from” citation displayed first.
Cited in PMC
Full-text articles in the PubMed Central Database that cite the current articles.

Recent activity

Clear Turn Off Turn On

Statistical challenges in the evaluation of surrogate endpoints in randomized tr...
Statistical challenges in the evaluation of surrogate endpoints in randomized trials.
Control Clin Trials. 2002 Dec ;23(6):607-25.

PubMed
A linkage unit joining peptidoglycan to teichoic acid in Staphylococcus aureus H...
A linkage unit joining peptidoglycan to teichoic acid in Staphylococcus aureus H.
FEBS Lett. 1976 Jan 15 ;61(2):240-2.

PubMed
Incretin-based therapies for type 2 diabetes mellitus.
Incretin-based therapies for type 2 diabetes mellitus.
Nat Rev Endocrinol. 2009 May ;5(5):262-9.

PubMed
In vivo and in vitro derived Palo Alto lines of Plasmodium falciparum are geneti...
In vivo and in vitro derived Palo Alto lines of Plasmodium falciparum are genetically unrelated.
Mol Biochem Parasitol. 1991 Aug ;47(2):167-78.

PubMed
Diversity and complexity in DNA recognition by transcription factors.
Diversity and complexity in DNA recognition by transcription factors.
Science. 2009 Jun 26 ;324(5935):1720-3. Epub 2009 May 14 .

PubMed

Your browsing activity is empty.

Activity recording is turned off.

Turn recording back on

PubMed

Result Filters

Display Settings:

Send to: