ABSTRACT
This chapter provides the reader with a framework for thinking about the design of clinical trials testing the direct or indirect effect of interventions on dyspnea outcomes, with an emphasis on measurement using one or more patient-reported outcome (PRO) instruments. Various PRO instruments are available for measuring dyspnea symptoms and/or impact to inform sample selection or test outcomes. The importance of dyspnea assessment in diagnosis and disease management translates directly into the role dyspnea measurement plays as a primary or secondary end point in clinical trials evaluating new interventions to eliminate or reduce this symptom and its impact. Cardiopulmonary exercise testing is often used to standardize activity parameters in clinical trials to test the effect of treatment on dyspnea during known or controlled levels of exertion. Drug development is a systematic process that can take 4–10 or more years to complete, from discovery through clinical development, regulatory review and approval, to product launch.
