For Patients

Conventional randomized trials compare the effect of treatments (or treatment versus control) across groups of patients. Most clinical trials are typically conducted by enrolling a large number of patients. Patients are assigned to one of two or more groups which compare different treatments or medication for the same condition. In the end, success is measured by comparing how the groups respond to the different treatments. The results can help clinicians and patients to decide together which treatments are worth trying.

For example, in this kind of trial

• Among a group of patients experiencing pain, half might be randomly assigned to take Tylenol while the other half takes Motrin for 2 weeks.
• Then, at the end of 2 weeks, the two groups would be compared to learn which group’s pain was reduced the most, the Tylenol group or the Motrin group
• The results can help doctors know which treatment works the best for people who are similar to the types of people enrolled in the clinical research trial

A different research approach is called Personalized Trials, also known as N-of-1 trials or patient-as-their-own-control or single case studies. This name came from statisticians because this trial focuses on one patient—the one patient who is trying to decide with their provider the best treatment for that one patient. In Personalized Trials, an individual patient decides on the treatments he/she would like to know about, and then is assigned to receive a sequence of treatments in a random order.

A patient in this kind of trial might receive Treatment A for 2 weeks, followed by Treatment B for 2 weeks. Often, neither the patient and nor the provider know which treatment that patient is receiving first and which one second. Throughout the trial, the patient is closely monitored and at the end of the trial, the treatment “code” is broken, the patient and the care provider jointly review the results to see if the patient did better with A or B, and they then decide which one would be best for that patient. In this type of trial, an individual patient is given the opportunity to test two or more treatments, often in a random order.

Blinding is when information about the treatment is masked (kept) from the participant and/or provider, to reduce or eliminate bias, until after a trial outcome is known. For example, patients may be more likely to think a brand name drug is more effective than a generic drug based on subliminal messaging, marketing or individual biases. Masking which drug you are taking might allow for a more scientific, objective assessment of treatment effects.

A washout period is a period of time during a trial when a participant is taken off of a drug or treatment in order to eliminate the effects of the treatment.

• A single patient who is experiencing pain might receive Tylenol for 2 weeks, followed by Motrin for 2 weeks, followed by Tylenol for another 2 weeks
• Throughout the trial, the patient monitors the effects of the treatment
• At the end of the trial, the patient and the doctor jointly review the results to see if the patient had less pain or side effects with Tylenol or Motrin
• With this information, the patient can then decide with their doctor on which treatment is best for them. These patients may learn that the treatment that is best for them is different than the treatment that is best for other patients

Personalized Trials can be conducted with varying levels of individual involvement. For example, in some Personalized Trials, you can select the types of treatments they wish to compare, the duration of the trial, and the outcomes you would like to use to compare the effects of treatments (e.g., side effects).

Patients can directly review the data obtained from the Personalized Trials and share in the decision-making with their clinician around choosing preferred treatments. Some patients prefer that their personal clinicians use the data from the Personalized Trials to arrive at treatment decisions with minimal patient input.