N Of 1 Clinical Trial
- A clinical-trial design that tests one patient to determine the effect of a treatment for that individual.
- Useful for tailoring treatment recommendations and collecting data for rare or unusual conditions.
- Results are specific to the patient studied and may not generalize to others.
Definition
Section titled “Definition”A N of 1 clinical trial, also called a single case study or individualized clinical trial, is a type of clinical trial that focuses on the effects of a specific treatment on an individual patient. It is conducted on a single patient rather than on a larger group of patients as in traditional clinical trials.
Explanation
Section titled “Explanation”In a N of 1 clinical trial the patient receives a treatment and their progress is closely monitored over a defined period. Outcomes observed during the treatment are compared to the patient’s baseline condition prior to the intervention. Trials may also compare multiple treatments given to the same patient one at a time to identify which is most effective for that individual. These trials offer more personalized evidence about treatment effectiveness and safety for the patient being studied, and can generate useful data for rare or poorly understood conditions that are often excluded from larger trials.
Examples
Section titled “Examples”Rare genetic condition
Section titled “Rare genetic condition”A patient with a rare genetic condition that has no known cure is given an experimental treatment. The patient’s progress is closely monitored over a set period and the results are compared to their baseline condition before treatment.
Chronic disease management
Section titled “Chronic disease management”A patient with a chronic disease, such as asthma or diabetes, is given several different treatments sequentially. The effects of each treatment are compared to determine which treatment is most effective for that specific patient.
Use cases
Section titled “Use cases”- Personalizing and tailoring treatment recommendations for an individual patient.
- Generating effectiveness and safety data for new treatments in rare or unusual conditions that are difficult to study in larger trials.
- Evaluating the best treatment option for a specific patient when multiple options exist.
Notes or pitfalls
Section titled “Notes or pitfalls”- Results from a N of 1 clinical trial may not be generalizable to other patients because the trial involves only a single individual.
- These trials can be more time-consuming and expensive than traditional clinical trials due to the need for individualized attention and close monitoring.
Related terms
Section titled “Related terms”- single case study
- individualized clinical trial
- traditional clinical trials