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Use of patient-reported outcomes in randomized, double-blind, placebo-controlled clinical trials.

Publication ,  Journal Article
Dinan, MA; Compton, KL; Dhillon, JK; Hammill, BG; Dewitt, EM; Weinfurt, KP; Schulman, KA
Published in: Med Care
April 2011

BACKGROUND: To optimize the use of patient-reported outcomes (PROs) in clinical research, it is first necessary to review the current use of these outcomes in clinical trials to determine under what circumstances they are most useful, and to reveal current limitations. PURPOSE: To investigate current patterns of use of PROs in clinical trials. RESEARCH DESIGN: We conducted a systematic literature review of all double-blind, placebo-controlled, randomized clinical trials using one or more PROs as a study outcome from 2004 to 2006. Data were abstracted and analyzed with descriptive statistics and logistic regression to characterize the use of PROs in clinical trials. RESULTS: The 180 clinical trials that met the study inclusion criteria used 173 unique instruments to measure a total of 466 PROs. Most PRO measurements were obtained using relatively few PRO instruments, with one-third of PRO instruments applied in more than 1 trial. In multivariable analysis, tests of statistical significance were more often reported for PROs used as primary trial outcomes. Statistically significant PRO outcomes (P<0.05) were more likely among disease-specific PROs compared with general PROs, PROs with a discussion of minimally important difference, and larger trials. CONCLUSIONS: PRO instruments may be improved through efforts to provide centralized electronic administration, cross-validation, and standardized interpretation of clinically relevant outcomes. The majority of PROs used in current clinical trials come from relatively few, commonly used disease-specific PRO instruments within major therapeutic areas.

Duke Scholars

Published In

Med Care

DOI

EISSN

1537-1948

Publication Date

April 2011

Volume

49

Issue

4

Start / End Page

415 / 419

Location

United States

Related Subject Headings

  • Research Subjects
  • Randomized Controlled Trials as Topic
  • Quality of Life
  • Quality Indicators, Health Care
  • Placebos
  • Outcome Assessment, Health Care
  • Humans
  • Health Policy & Services
  • Double-Blind Method
  • Data Interpretation, Statistical
 

Citation

APA
Chicago
ICMJE
MLA
NLM
Dinan, M. A., Compton, K. L., Dhillon, J. K., Hammill, B. G., Dewitt, E. M., Weinfurt, K. P., & Schulman, K. A. (2011). Use of patient-reported outcomes in randomized, double-blind, placebo-controlled clinical trials. Med Care, 49(4), 415–419. https://doi.org/10.1097/MLR.0b013e3182064aa2
Dinan, Michaela A., Kate L. Compton, Jatinder K. Dhillon, Bradley G. Hammill, Esi Morgan Dewitt, Kevin P. Weinfurt, and Kevin A. Schulman. “Use of patient-reported outcomes in randomized, double-blind, placebo-controlled clinical trials.Med Care 49, no. 4 (April 2011): 415–19. https://doi.org/10.1097/MLR.0b013e3182064aa2.
Dinan MA, Compton KL, Dhillon JK, Hammill BG, Dewitt EM, Weinfurt KP, et al. Use of patient-reported outcomes in randomized, double-blind, placebo-controlled clinical trials. Med Care. 2011 Apr;49(4):415–9.
Dinan, Michaela A., et al. “Use of patient-reported outcomes in randomized, double-blind, placebo-controlled clinical trials.Med Care, vol. 49, no. 4, Apr. 2011, pp. 415–19. Pubmed, doi:10.1097/MLR.0b013e3182064aa2.
Dinan MA, Compton KL, Dhillon JK, Hammill BG, Dewitt EM, Weinfurt KP, Schulman KA. Use of patient-reported outcomes in randomized, double-blind, placebo-controlled clinical trials. Med Care. 2011 Apr;49(4):415–419.

Published In

Med Care

DOI

EISSN

1537-1948

Publication Date

April 2011

Volume

49

Issue

4

Start / End Page

415 / 419

Location

United States

Related Subject Headings

  • Research Subjects
  • Randomized Controlled Trials as Topic
  • Quality of Life
  • Quality Indicators, Health Care
  • Placebos
  • Outcome Assessment, Health Care
  • Humans
  • Health Policy & Services
  • Double-Blind Method
  • Data Interpretation, Statistical