Feasibility of effectiveness trials in pediatric anesthesia

Effectiveness studies aim to investigate whether an intervention does more good than harm in normal clinical practice.

Historically, research in pediatric anesthesia has appropriately concentrated on efficacy studies which even if of excellent design, tell us little about how generalizable or applicable the findings are to routine clinical practice. Simultaneously effectiveness and cost-effectiveness research has been limited due to systematic and cultural factors. To understand the challenges of effectiveness research in children, it is necessary to examine the overlapping relationship between efficacy, effectiveness, and cost-effectiveness studies. This can be illuminated with Cochrane´s hierarchy of investigation and the disease, illness, and sickness modelfrom the philosophy of science. Effectiveness (illness perspective) studies form the middle rung of Cochrane´s research hierarchy, between efficacy (disease perspective) and cost-effectiveness (sickness perspective) research. Effectiveness studies aim to be generalizable and determine whether interventions work in routine clinical practice. Since outcomes that reflect patient priorities offer the most generalizability these are encouraged in effectiveness studies. Due to developing consciousness and communication in young children, identifying and measuring patient-centered outcomes has challenges. These challenges are not insurmountable and with appropriate communication and research techniques the subjective nature of the experience of illness in the young may be elucidated and should direct research goals where possible. Sickness perspective research, in terms of cost-effectiveness, remains underdeveloped in pediatric anesthesia.

Pediatric anesthesia has a strong base in efficacy research, but the need to expand research to include effectiveness and eventually cost-effectiveness studies should not be ignored.