Pediatric asthma: “Real world” measures of effectiveness

In the treatment of pediatric asthma, measuring the effectiveness of an intervention in terms of cost or therapeutic value can be a challenging process. Parameters that should be considered in determining the cost effectiveness of an intervention vary considerably. In clinical therapeutic trials of pediatric asthma, subtle patient selection biases, difficulties in choosing and assessing treatment outcomes in young children, and the highly variable nature of asthma symptomatology can compromise valid measurements of the therapeutic effectiveness of an intervention. Ideally, outcome measurements in asthma should reflect “real world” conditions. They should be as broad‐based as possible and include both objective measures of pulmonary function and subjective measures of quality‐of‐life evaluated over the long term. Patient adherence is thus a critical variable that influences the ultimate effectiveness of treatment. The rate of patient adherence is influenced not only by drug characteristics such as route of administration and dosing frequency, but also by the impact of treatment on a patient's quality of life. Because asthma is a chronic disease with variable symptomatology, clinical trials investigating the cost and therapeutic effectiveness of asthma interventions must evaluate real‐world variables in long‐term trials. This article will provide information regarding proper assessment of an intervention's cost and therapeutic value. Pediatr Pulmonol. 2001; Supplement 21:31–37. © 2001 Wiley‐Liss, Inc.