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CHIPTS Cross-Core Meeting Keynote Address
Single-patient (n-of-1) trial:
A pragmatic clinical decision methodology for patient-centered comparative effectiveness research
Naihua Duan, Ph.D.
Division of Biostatistics, Department of Psychiatry, Columbia University
Monday, January 13 3:00-4:00pm
Single-patient trials (SPTs, a.k.a. n-of-1 trials) are multiple-period crossover trials conducted within individual patients to evaluate the comparative effectiveness of two or more treatments for each specific patient (Duan, Kravitz, Schmid, 2013, J. of Clinical Epidemiology). SPT has the potential to serve a rather unique dual role, both to inform individual treatment decisions in clinical care, and to support pragmatic patient-centered comparative effectiveness research. Dr. Duan gives a brief overview of the core methodology for SPT, and discuss key implementation issues, including:
• Indications and contraindications for clinical conditions and treatments suitable for evaluation with the SPT;
• Pros and cons for blinding and washout as design features for SPTs; and
• Statistical and information technology infrastructure for the implementation of SPTs.
Dr. Duan also discuss the interpretation of the SPT as human subjects research vs. quality improvement, and the broader issues involved in the production and consumption of generalizable knowledge in human subjects research vs. local knowledge that is often needed in quality improvement investigations.
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.
Kravitz RL, Duan N, eds, and the DEcIDE Methods Center N-of-1 Guidance Panel (Duan N, Eslick I, Gabler NB, Kaplan HC, Kravitz RL, Larson EB, Pace WD, Schmid CH, Sim I, Vohra S). Design and Implementation of N-of-1 Trials: A User’s Guide. AHRQ Publication No. 13-EHC122-EF. Rockville, MD: Agency for Healthcare Research and Quality; in press, www.effectivehealthcare.ahrq.gov/reports/final.cfm.
Cheung K, Duan N. Design of implementation studies for quality improvement programs: an effectiveness-cost-effectiveness framework. Am J Public Health. 2014 Jan;104(1):e23-30. Epub 2013 Nov 14.
Mohr DC, Cheung K, Schueller SM, Hendricks Brown C, Duan N. Continuous evaluation of evolving behavioral intervention technologies. Am J Prev Med. 2013 Oct;45(4):517-23.