N-of-1 Trials

Medical research studies involving people are called clinical trials. Typically, a carefully selected group of patients undergoes the same procedure, and researchers measure its effects in comparison to those seen in others who receive the current standard of care. The aim of the trial is to find out if a new treatment or procedure is safe, has side effects, helps patients feel better and works better than current procedures. Not all clinical trials will result in new and better treatment. Some will discover that the treatment being tested does not work or that it has side effects that are much worse than with existing treatments.

 Presently healthcare is focused mainly on working out generalized solutions with which the biggest ratio of ill people can be treated. While pharmaceutical drugs are the product of expensive randomized clinical trials it is well accepted that the top ten highest-grossing drugs in the United States help between 1 in 25 and 1 in 4 of the people who take them. For some drugs, such as statins — routinely used to lower cholesterol — as few as 1 in 50 users benefit. Results from these randomized controlled trials, considered the “gold standard” of research evidence, may exclude many patients seen routinely by physicians.

In the absence of relevant research evidence clinicians rely on “trials of therapy.” This trial and error approach, over the course of several visits, hopefully finds something that works to alleviate symptoms. But this lack of relevant evidence limits a clinician’s ability to truly fulfill the mandate of evidence based medical decisions and usually does not get to the root of the problem. In the case of cancer treatment with chemotherapy, using the wrong treatment could mean the right therapy that might have worked may not work as a follow on therapy.

An N of 1 trial is a clinical trial in which a single patient is the entire trial – a single case study. Focusing on what treatment will work best for the majority of patients is the conventional medical model. The goal of a precision medicine N-of-1 trial is to do a deep-dive analysis and determine the optimal or best intervention for an individual patient using objective data-driven criteria. This information is then compared to the human reference genome and other reference data to gain in sights of how best to optimize a person’s health and wellness.

N-of-1 clinical trials are based on the growing awareness that grouping patients is limited in its ability to address the genetic variability seen in most chronic diseases. N-of-1 clinical trials aim to identify the specific genetic and molecular factors that are driving or may drive disease development in a single patient, are exploratory in nature and do not begin with a specific treatment in mind.

This type of study enables practitioners to achieve experimental progress without the overwhelming work of designing a group comparison study is to find an answer about treatment effectiveness for an individual patient; the individual patient’s health and well being are of primary interest. N-of-1 trials provide a structured, objective, and evidence-based framework for evaluating the treatment effect on an individual.

N-of-1 trials straddle the divide between randomized controlled trials and trials of therapy by providing scientific rigor and an individualized approach to patient care at the same time. They allow assessment of long-term therapy in chronic conditions to determine if treatment effectiveness is continuing to be achieved. They also establish comparative and additive treatment effectiveness for patients with various comorbid conditions using concurrent therapies and help promote patient safety by limiting therapies to those with demonstrated effectiveness.

N-of-1 trials form the foundation of precision medicine enabling the medical community to fulfill the age-old requirements of healing and doing no harm at the same time. This preventive and treatment approach considers individual variability in our genome, epigenome, microbiome, environment and lifestyle for each person.

Lifelong clinical studies at IRIS Wellness Labs will be run on individuals and their specific disease(s). Anyone who gets their whole genome sequenced through IRIS will have the option to be enrolled with their consent. Being part of our lifelong clinical studies means that you will be participating in a study where risks, trends, potential treatment(s) and/or cures may be discovered for specific diseases. We monitor many parameters to evaluate your overall health, energy level, cardio, and mental, muscular and molecular trends.

An N-of-1 trial provide patients with optimal clinical care – care that is personalized, precise and evidence-based – and reduces the potential for bias seen in routine clinical care.

Written by Bill Schaser, Director of Education