Classical clinical trials harvest a handful of measurements from thousands of people.  Precision medicine requires different ways of testing interventions.  Researchers need to probe the myriad factors — genetic and environmental, among others — that promote an individual’s health, their susceptibility to disease, and their response to a particular treatment.

Dr. Schork is Professor and Director, Human Biology, at the J. Craig Venter Institute (JCVI), Professor at the Translational Genomics Research Institute (TGen) in Phoenix and an Adjunct Professor at the University of California, San Diego. Prior to his position at the JCVI he was a Professor, Molecular and Experimental Medicine, at The Scripps Research Institute (TSRI) and also Director of Bioinformatics and Biostatistics for the Scripps Translational Science Institute (STSI). Dr. Schork has also held faculty appointments at Case Western Reserve University and Harvard University. He has published over 500 articles in the area of the genetic dissection of complex phenotypes. These articles include both methodological and applied studies. He also has a long history of collaborative and consortium-related research in which he has contributed analysis methodology and applied data analysis expertise. Dr. Schork is currently the Director of the Tanner Project, a study investigating the utility of early cancer detection biomarkers among individuals highly susceptible to cancer and an investigator associated with the Stand Up to Cancer Melanoma Dream Team clinical trial investigating the utility of tumor genomic profiling in making treatment decisions.

Personalized medicine: Time for one-person trials

Precision medicine requires a different type of clinical trial that focuses on individual, not average, responses to therapy, says Nicholas J. Schork.

The n-of-1 clinical trial: the ultimate strategy for individualizing medicine?

N-of-1 or single subject clinical trials consider an individual patient as the sole unit of observation in a study investigating the efficacy or side-effect profiles of different interventions. The ultimate goal of an n-of-1 trial is to determine the optimal or best intervention for an individual patient using objective data-driven criteria. Such trials can leverage study design and statistical techniques associated with standard population-based clinical trials, including randomization, washout and crossover periods, as well as placebo controls. Despite their obvious appeal and wide use in educational settings, n-of-1 trials have been used sparingly in medical and general clinical settings. We briefly review the history, motivation and design of n-of-1 trials and emphasize the great utility of modern wireless medical monitoring devices in their execution. We ultimately argue that n-of-1 trials demand serious attention among the health research and clinical care communities given the contemporary focus on individualized medicine.

Links to publications discussed in the webinar

A genome sequencing program for novel undiagnosed diseases.
Genet Med, 2015

Using N-of-1 Trials to Improve Patient Management and Save Costs
Journal of General Internal Medicine, 2010

Longitudinal Screening Algorithm That Incorporates Change Over Time in CA125 Levels Identifies Ovarian Cancer Earlier Than a Single-Threshold Rule
American Society of Clinical Oncology, 2015