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Executive Summary – Jul. 11, 2013

Fourth Symposium on Comparative Effectiveness Research Methods: From Efficacy to Effectiveness

In June 2012, AHRQ’s Effective Health Care Program, through the DEcIDE Network, sponsored its fourth symposium on research methods for comparative effectiveness research and patient-centered outcomes research studies. The symposium examined the methodological work that serves to illuminate the mechanisms contributing to differences that might be observed between results from randomized clinical trials of treatments (efficacy) and observational studies of treatments outside of controlled research environments (effectiveness).

Presenters at the symposium submitted manuscripts for independent peer review and publication in a journal supplement of the Journal of Clinical Epidemiology.  Below are links to each article. A printed copy of the supplement can be ordered from the AHRQ Publications Clearinghouse by calling toll free 800-358-9295 or emailing AHRQpubs@ahrq.hhs.gov. Please refer to AHRQ publication number OM13-0049, when ordering.

For information about ordering supplements from prior symposia, please visit the bottom of this page.

Table of Contents

Methods for Comparative Effectiveness Research/Patient-Centered Outcomes Research

Methods for comparative effectiveness research/patient-centered outcomes research: from efficacy to effectiveness. Exit Disclaimer
Schneeweiss S, Seeger JD, Jackson JW, et al. Methods for comparative effectiveness research/patient-centered outcomes research: from efficacy to effectiveness. J Clin Epidemiol. 2013 Aug;66(8 Suppl):S1-4. DOI: 10.1016/j.jclinepi.2013.05.012

Comparative Effectiveness Research/Patient-Centered Outcomes Research Implementations

Implementing randomized effectiveness trials in large insurance systems. Exit Disclaimer
Choudhry NK, Shrank WH. Implementing randomized effectiveness trials in large insurance systems. J Clin Epidemiol. 2013 Aug;66(8 Suppl):S5-11. DOI: 10.1016/j.jclinepi.2013.03.022.

Measure once, cut twice—adding patient-reported outcome measures to the electronic health record for comparative effectiveness research. Exit Disclaimer
Wu AW, Kharrazi H, Boulware LE, et al. Measure once, cut twice—adding patient-reported outcome measures to the electronic health record for comparative effectiveness research. J Clin Epidemiol. 2013 Aug;66(8 Suppl):S12-20. DOI: 10.1016/j.jclinepi.2013.04.005.

Single-patient (n-of-1) trials: a pragmatic clinical decision methodology for patient-centered comparative effectiveness research. Exit Disclaimer
Duan N, Kravitz RL, Schmid CH. Single-patient (n-of-1) trials: a pragmatic clinical decision methodology for patient-centered comparative effectiveness research. J Clin Epidemiol. 2013 Aug;66(8 Suppl):S21-8. DOI: 10.1016/j.jclinepi.2013.04.006.

Comparative Effectiveness Research Challenges in Mental Health

The use of clinical trials in comparative effectiveness research on mental health. Exit Disclaimer
Blanco C, Rafful C, Olfson M. The use of clinical trials in comparative effectiveness research on mental health. J Clin Epidemiol. 2013 Aug;66(8 Suppl):S29-36. DOI: 10.1016/j.jclinepi.2013.02.013.

Assessing the comparative effectiveness of long-acting injectable vs. oral antipsychotic medications in the prevention of relapse provides a case study in comparative effectiveness research in psychiatry. Exit Disclaimer
Kane JM, Kishimoto T, Correll CU. Assessing the comparative effectiveness of long-acting injectable vs. oral antipsychotic medications in the prevention of relapse provides a case study in comparative effectiveness research in psychiatry. J Clin Epidemiol. 2013 Aug;66(8 Suppl):S37-41. DOI: 10.1016/j.jclinepi.2013.01.012.

Studying the Effectiveness of Health Care Delivery System Changes

Different analyses estimate different parameters of the effect of erythropoietin stimulating agents on survival in end stage renal disease: a comparison of payment policy analysis, instrumental variables, and multiple imputation of potential outcomes. Exit Disclaimer
Dore DD, Swaminathan S, Gutman R, et al. Different analyses estimate different parameters of the effect of erythropoietin stimulating agents on survival in end stage renal disease: a comparison of payment policy analysis, instrumental variables, and multiple imputation of potential outcomes. J Clin Epidemiol. 2013 Aug;66(8 Suppl):S42-50. DOI: 10.1016/j.jclinepi.2013.02.014.

Approaches to inverse-probability-of-treatment–weighted estimation with concurrent treatments. Exit Disclaimer
Ellis AR, Brookhart MA. Approaches to inverse-probability-of-treatment–weighted estimation with concurrent treatments. J Clin Epidemiol. 2013 Aug;66(8 Suppl):S51-6. DOI: 10.1016/j.jclinepi.2013.03.020.

Simulating changes to emergency care resources to compare system effectiveness. Exit Disclaimer
Branas CC, Wolff CS, Williams J, et al. Simulating changes to emergency care resources to compare system effectiveness. J Clin Epidemiol. 2013 Aug;66(8 Suppl):S57-64. DOI: 10.1016/j.jclinepi.2013.03.021.

Analytic Issues in Comparative Effectiveness Research

Matching on provider is risky. Exit Disclaimer
Walker AM. Matching on provider is risky. J Clin Epidemiol. 2013 Aug;66(8 Suppl):S65-8. DOI: 10.1016/j.jclinepi.2013.02.012.

What is the effect of area size when using local area practice style as an instrument? Exit Disclaimer
Brooks JM, Tang Y, Chapman CG, et al. What is the effect of area size when using local area practice style as an instrument? J Clin Epidemiol. 2013 Aug;66(8 Suppl):S69-83. DOI: 10.1016/j.jclinepi.2013.04.008.

Prognostic score–based balance measures can be a useful diagnostic for propensity score methods in comparative effectiveness research. Exit Disclaimer
Stuart EA, Lee BK, Leacy FP. Prognostic score–based balance measures can be a useful diagnostic for propensity score methods in comparative effectiveness research. J Clin Epidemiol. 2013 Aug;66(8 Suppl):S84-90. DOI: 10.1016/j.jclinepi.2013.01.013.

Targeted maximum likelihood estimation in safety analysis. Exit Disclaimer
Lendle SD, Fireman B, van der Laan MJ. Targeted maximum likelihood estimation in safety analysis. J Clin Epidemiol. 2013 Aug;66(8 Suppl):S91-8. DOI: 10.1016/j.jclinepi.2013.02.017.

Super learning to hedge against incorrect inference from arbitrary parametric assumptions in marginal structural modeling. Exit Disclaimer
Neugebauer R, Fireman B, Roy JA, et al. Super learning to hedge against incorrect inference from arbitrary parametric assumptions in marginal structural modeling. J Clin Epidemiol. 2013 Aug;66(8 Suppl):S99-109. DOI: 10.1016/j.jclinepi.2013.01.016.

Validation sampling can reduce bias in health care database studies: an illustration using influenza vaccination effectiveness. Exit Disclaimer
Nelson JC, Marsh T, Lumley T,  et al; Vaccine Safety Datalink Team. Validation sampling can reduce bias in health care database studies: an illustration using influenza vaccination effectiveness. J Clin Epidemiol. 2013 Aug;66(8 Suppl):S110-21. DOI: 10.1016/j.jclinepi.2013.01.015.

Design Issues in Comparative Effectiveness Research

A model for incorporating patient and stakeholder voices in a learning health care network: Washington State’s Comparative Effectiveness Research Translation Network. Exit Disclaimer
Devine EB, Alfonso-Cristancho R, Devlin A, et al; CERTAIN Collaborative. A model for incorporating patient and stakeholder voices in a learning health care network: Washington State’s Comparative Effectiveness Research Translation Network. J Clin Epidemiol. 2013 Aug;66(8 Suppl):S122-29. DOI: 10.1016/j.jclinepi.2013.04.007.

Bayesian adaptive trials offer advantages in comparative effectiveness trials: an example in status epilepticus. Exit Disclaimer
Connor JT, Elm JJ, Broglio KR; ESETT and ADAPT-IT Investigators. Bayesian adaptive trials offer advantages in comparative effectiveness trials: an example in status epilepticus. J Clin Epidemiol. 2013 Aug;66(8 Suppl):S130-7. DOI: 10.1016/j.jclinepi.2013.02.015.

Impact of immortal person-time and time scale in comparative effectiveness research for medical devices: a case for implantable cardioverter-defibrillators. Exit Disclaimer
Mi X, Hammill BG, Curtis LH, et al. Impact of immortal person-time and time scale in comparative effectiveness research for medical devices: a case for implantable cardioverter-defibrillators. J Clin Epidemiol. 2013 Aug;66(8 Suppl):S138-44. DOI: 10.1016/j.jclinepi.2013.01.014.

 Proceedings from Past Symposia

Proceedings from past symposia on research methods for comparative effectiveness and patient-centered outcomes research can be accessed on the right side of this web page and downloaded for free. A printed copy of the supplement(s) can be ordered from the AHRQ Publications Clearinghouse by calling toll free 800-358-9295 or emailing AHRQpubs@ahrq.hhs.gov. Please refer to AHRQ publication number when ordering. 

  • 2007 Medical Care journal supplement on Emerging Methods in Comparative Effectiveness & Safety, AHRQ Publication No. OM07-0085.
  • 2010 Medical Care supplement on Research Methods for Clinical and Comparative Effectiveness Studies, AHRQ Publication No. OM10-0067.
  • 2012 Pharmacoepidemiology and Drug Safety supplement on Research Methods for Patient-Centered Outcomes Research, AHRQ Publication No. OM12-0043.
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