Patient-important outcomes in registered diabetes trials

Gunjan Y. Gandhi, Mohammad H Murad, Akira Fujiyoshi, Rebecca J. Mullan, David N. Flynn, Mohamed B. Elamin, Brian A. Swiglo, William L. Isley, Gordon H. Guyatt, Victor Manuel Montori

Research output: Contribution to journalArticle

142 Citations (Scopus)

Abstract

Context: Concerns about the safety and efficacy of diabetes interventions persist, in part because randomized clinical trials (RCTs) have not measured their effect on patient-important outcomes, ie, death and quality of life (morbidity, pain, function). Objective: To systematically determine the extent to which ongoing and future RCTs in diabetes will ascertain patient-important outcomes. Data Sources: On November 10, 2007, we searched primary RCT registries ClinicalTrials.gov (http://www.clinicaltrials.gov), International Standard Randomized Controlled Trial Number Register (http://isrctn.org), and Australian New Zealand Clinical Trials Registry (http://www.anzctr.org.au). Study Selection: We identified phase 2 through 4 RCTs enrolling patients with diabetes. Of 2019 RCTs, 1054 proved eligible. We randomly sampled 50% of the eligible RCTs (527 of 1054) and selected 436 registered since registration became mandatory (2004). Data Extraction: Pairs of reviewers working independently collected study characteristics and determined the outcomes measured and their type (physiological outcomes, surrogate outcomes thought to reflect an increased risk for patient-important outcomes, and patient-important outcomes). Results: Of the 436 registered RCTs included in this analysis, 24 (6%) had not started enrollment, 109 (25%) were actively enrolling, and 303 (69%) had completed enrollment. Primary outcomes were patient-important outcomes in only 78 of 436 RCTs (18%; 95% confidence interval [CI], 14%-22%), physiological and laboratory outcomes in 69 of 436 (16%; 95% CI, 13%-20%), and surrogate outcomes in 268 of 436 (61%; 95% CI, 57%-66%). Patient-important outcomes were reported as primary or secondary outcomes in 201 of 436 (46%; 95% CI, 41%-51%). In multivariate analysis, large trials (odds ratio [OR], 1.10; 95% CI, 1.02-1.19 for every additional 100 patients) and trials of longer duration (OR, 1.03; 95% CI, 1.01-1.06 for every additional 30 days) were more likely while parallel design RCTs (OR, 0.15; 95% CI, 0.05-0.44) and type 2 diabetes trials (OR, 0.23; 95% CI, 0.09-0.61) were less likely to assess patient-important outcomes as a primary outcome. Conclusion: In this sample of registered ongoing RCTs in diabetes, only 18% included patient-important outcomes as primary outcomes.

Original languageEnglish (US)
Pages (from-to)2543-2549
Number of pages7
JournalJAMA - Journal of the American Medical Association
Volume299
Issue number21
DOIs
StatePublished - Jun 4 2008

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Randomized Controlled Trials
Confidence Intervals
Odds Ratio
Registries
Information Storage and Retrieval
New Zealand
Type 2 Diabetes Mellitus
Multivariate Analysis
Quality of Life
Clinical Trials
Morbidity
Safety
Pain

ASJC Scopus subject areas

  • Medicine(all)

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Patient-important outcomes in registered diabetes trials. / Gandhi, Gunjan Y.; Murad, Mohammad H; Fujiyoshi, Akira; Mullan, Rebecca J.; Flynn, David N.; Elamin, Mohamed B.; Swiglo, Brian A.; Isley, William L.; Guyatt, Gordon H.; Montori, Victor Manuel.

In: JAMA - Journal of the American Medical Association, Vol. 299, No. 21, 04.06.2008, p. 2543-2549.

Research output: Contribution to journalArticle

Gandhi, GY, Murad, MH, Fujiyoshi, A, Mullan, RJ, Flynn, DN, Elamin, MB, Swiglo, BA, Isley, WL, Guyatt, GH & Montori, VM 2008, 'Patient-important outcomes in registered diabetes trials', JAMA - Journal of the American Medical Association, vol. 299, no. 21, pp. 2543-2549. https://doi.org/10.1001/jama.299.21.2543
Gandhi, Gunjan Y. ; Murad, Mohammad H ; Fujiyoshi, Akira ; Mullan, Rebecca J. ; Flynn, David N. ; Elamin, Mohamed B. ; Swiglo, Brian A. ; Isley, William L. ; Guyatt, Gordon H. ; Montori, Victor Manuel. / Patient-important outcomes in registered diabetes trials. In: JAMA - Journal of the American Medical Association. 2008 ; Vol. 299, No. 21. pp. 2543-2549.
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abstract = "Context: Concerns about the safety and efficacy of diabetes interventions persist, in part because randomized clinical trials (RCTs) have not measured their effect on patient-important outcomes, ie, death and quality of life (morbidity, pain, function). Objective: To systematically determine the extent to which ongoing and future RCTs in diabetes will ascertain patient-important outcomes. Data Sources: On November 10, 2007, we searched primary RCT registries ClinicalTrials.gov (http://www.clinicaltrials.gov), International Standard Randomized Controlled Trial Number Register (http://isrctn.org), and Australian New Zealand Clinical Trials Registry (http://www.anzctr.org.au). Study Selection: We identified phase 2 through 4 RCTs enrolling patients with diabetes. Of 2019 RCTs, 1054 proved eligible. We randomly sampled 50{\%} of the eligible RCTs (527 of 1054) and selected 436 registered since registration became mandatory (2004). Data Extraction: Pairs of reviewers working independently collected study characteristics and determined the outcomes measured and their type (physiological outcomes, surrogate outcomes thought to reflect an increased risk for patient-important outcomes, and patient-important outcomes). Results: Of the 436 registered RCTs included in this analysis, 24 (6{\%}) had not started enrollment, 109 (25{\%}) were actively enrolling, and 303 (69{\%}) had completed enrollment. Primary outcomes were patient-important outcomes in only 78 of 436 RCTs (18{\%}; 95{\%} confidence interval [CI], 14{\%}-22{\%}), physiological and laboratory outcomes in 69 of 436 (16{\%}; 95{\%} CI, 13{\%}-20{\%}), and surrogate outcomes in 268 of 436 (61{\%}; 95{\%} CI, 57{\%}-66{\%}). Patient-important outcomes were reported as primary or secondary outcomes in 201 of 436 (46{\%}; 95{\%} CI, 41{\%}-51{\%}). In multivariate analysis, large trials (odds ratio [OR], 1.10; 95{\%} CI, 1.02-1.19 for every additional 100 patients) and trials of longer duration (OR, 1.03; 95{\%} CI, 1.01-1.06 for every additional 30 days) were more likely while parallel design RCTs (OR, 0.15; 95{\%} CI, 0.05-0.44) and type 2 diabetes trials (OR, 0.23; 95{\%} CI, 0.09-0.61) were less likely to assess patient-important outcomes as a primary outcome. Conclusion: In this sample of registered ongoing RCTs in diabetes, only 18{\%} included patient-important outcomes as primary outcomes.",
author = "Gandhi, {Gunjan Y.} and Murad, {Mohammad H} and Akira Fujiyoshi and Mullan, {Rebecca J.} and Flynn, {David N.} and Elamin, {Mohamed B.} and Swiglo, {Brian A.} and Isley, {William L.} and Guyatt, {Gordon H.} and Montori, {Victor Manuel}",
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AU - Gandhi, Gunjan Y.

AU - Murad, Mohammad H

AU - Fujiyoshi, Akira

AU - Mullan, Rebecca J.

AU - Flynn, David N.

AU - Elamin, Mohamed B.

AU - Swiglo, Brian A.

AU - Isley, William L.

AU - Guyatt, Gordon H.

AU - Montori, Victor Manuel

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N2 - Context: Concerns about the safety and efficacy of diabetes interventions persist, in part because randomized clinical trials (RCTs) have not measured their effect on patient-important outcomes, ie, death and quality of life (morbidity, pain, function). Objective: To systematically determine the extent to which ongoing and future RCTs in diabetes will ascertain patient-important outcomes. Data Sources: On November 10, 2007, we searched primary RCT registries ClinicalTrials.gov (http://www.clinicaltrials.gov), International Standard Randomized Controlled Trial Number Register (http://isrctn.org), and Australian New Zealand Clinical Trials Registry (http://www.anzctr.org.au). Study Selection: We identified phase 2 through 4 RCTs enrolling patients with diabetes. Of 2019 RCTs, 1054 proved eligible. We randomly sampled 50% of the eligible RCTs (527 of 1054) and selected 436 registered since registration became mandatory (2004). Data Extraction: Pairs of reviewers working independently collected study characteristics and determined the outcomes measured and their type (physiological outcomes, surrogate outcomes thought to reflect an increased risk for patient-important outcomes, and patient-important outcomes). Results: Of the 436 registered RCTs included in this analysis, 24 (6%) had not started enrollment, 109 (25%) were actively enrolling, and 303 (69%) had completed enrollment. Primary outcomes were patient-important outcomes in only 78 of 436 RCTs (18%; 95% confidence interval [CI], 14%-22%), physiological and laboratory outcomes in 69 of 436 (16%; 95% CI, 13%-20%), and surrogate outcomes in 268 of 436 (61%; 95% CI, 57%-66%). Patient-important outcomes were reported as primary or secondary outcomes in 201 of 436 (46%; 95% CI, 41%-51%). In multivariate analysis, large trials (odds ratio [OR], 1.10; 95% CI, 1.02-1.19 for every additional 100 patients) and trials of longer duration (OR, 1.03; 95% CI, 1.01-1.06 for every additional 30 days) were more likely while parallel design RCTs (OR, 0.15; 95% CI, 0.05-0.44) and type 2 diabetes trials (OR, 0.23; 95% CI, 0.09-0.61) were less likely to assess patient-important outcomes as a primary outcome. Conclusion: In this sample of registered ongoing RCTs in diabetes, only 18% included patient-important outcomes as primary outcomes.

AB - Context: Concerns about the safety and efficacy of diabetes interventions persist, in part because randomized clinical trials (RCTs) have not measured their effect on patient-important outcomes, ie, death and quality of life (morbidity, pain, function). Objective: To systematically determine the extent to which ongoing and future RCTs in diabetes will ascertain patient-important outcomes. Data Sources: On November 10, 2007, we searched primary RCT registries ClinicalTrials.gov (http://www.clinicaltrials.gov), International Standard Randomized Controlled Trial Number Register (http://isrctn.org), and Australian New Zealand Clinical Trials Registry (http://www.anzctr.org.au). Study Selection: We identified phase 2 through 4 RCTs enrolling patients with diabetes. Of 2019 RCTs, 1054 proved eligible. We randomly sampled 50% of the eligible RCTs (527 of 1054) and selected 436 registered since registration became mandatory (2004). Data Extraction: Pairs of reviewers working independently collected study characteristics and determined the outcomes measured and their type (physiological outcomes, surrogate outcomes thought to reflect an increased risk for patient-important outcomes, and patient-important outcomes). Results: Of the 436 registered RCTs included in this analysis, 24 (6%) had not started enrollment, 109 (25%) were actively enrolling, and 303 (69%) had completed enrollment. Primary outcomes were patient-important outcomes in only 78 of 436 RCTs (18%; 95% confidence interval [CI], 14%-22%), physiological and laboratory outcomes in 69 of 436 (16%; 95% CI, 13%-20%), and surrogate outcomes in 268 of 436 (61%; 95% CI, 57%-66%). Patient-important outcomes were reported as primary or secondary outcomes in 201 of 436 (46%; 95% CI, 41%-51%). In multivariate analysis, large trials (odds ratio [OR], 1.10; 95% CI, 1.02-1.19 for every additional 100 patients) and trials of longer duration (OR, 1.03; 95% CI, 1.01-1.06 for every additional 30 days) were more likely while parallel design RCTs (OR, 0.15; 95% CI, 0.05-0.44) and type 2 diabetes trials (OR, 0.23; 95% CI, 0.09-0.61) were less likely to assess patient-important outcomes as a primary outcome. Conclusion: In this sample of registered ongoing RCTs in diabetes, only 18% included patient-important outcomes as primary outcomes.

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