Abstract 196

The N-of-1 trial is a randomized experiment with a single participant. The patient is given two treatments, an active therapy and a placebo or alternate therapy; the order is determined by random allocation. Both the patient and treating clinician may be blinded as to which particular treatment is being used at a particular time. In Pediatrics, the practice of N-of-1 technique is being used in cases where there is no standard of care or where there are insufficient data to prove a treatment's safety and efficacy.

A variety of arguments have been used to support the use of N-of-1 trials. These include intervention designed to enhance patient wellbeing, a reasonable expectation of success, or as a last resort. These arguments do not hold true for all N-of-1 trials. Many N-of-1 trials are designed to test the efficacy of a specific therapy. This is the purpose of experimental research and raises ethical concerns that must be addressed. First, respect for patients, expressed by informed consent must be paramount. Second, beneficence should be ensured by balancing expected risks and benefits. Third, the assignment of patients enrolled must reflect justice. To avoid the pitfalls of not taking these considerations into account, physicians should adhere to the usual guidelines for research with human subjects, including review by the institutional review board (IRB).

Nonetheless, the urgency of clinical practice may be frustrated by the delay in the approval of these trials that often accompanies IRB approval. Submitting possible N-of-1 protocols in advance of actual trials for those physicians with an already known population may be a solution to this problem.