Patient-reported outcomes (PROs) have been successfully developed for a variety of chronic respiratory diseases, such as asthma and cystic fibrosis (CF). They have recently been used to evaluate the efficacy of new medications and assess current patient functioning. Although regulatory bodies have favored PROs that measures symptoms, other domains of functioning, such as treatment burden, should be considered. This review examines current guidelines for the development and application of PROs in clinical trials, describes methods for selecting appropriate measures for paediatric populations, and presents a model incorporating PROs into clinical practice. Guidance on interpretation of these measures and graphic presentation of results are illustrated. PROs can serve as the link between the health care provider and patient to foster collaborative and personalized medicine. This model promotes greater patient responsibility, facilitates communication with providers, encourages shared decision-making, and enhances adherence.
Keywords: Collaborative Medicine; EMA; FDA; Health-Related Quality of Life; Patient Reported Outcomes; cystic fibrosis.
Published by Elsevier Ltd.