Single-patient (N-of-1) trials: A pragmatic clinical decision methodology for patient-centered comparative effectiveness research

Naihua Duan, Ph.D.
Columbia University

ABSTRACT:
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.  I will give 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;
• Interpretation of the SPT as human subjects research vs. quality improvement;
• Pros and cons for blinding and washout as design features for SPTs;
• Statistical methods for the combination of individual SPT data and aggregate data from similar SPTs (borrowing from strength) to enhance treatment decisions for the specific patient;
• Information technology infrastructure for the implementation of SPTs; and
• Methodological developments that will enhance the utility of SPTs.