Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research
-
Informant-reported outcome measures, usually completed by parents, are often administered in pediatric clinical trials with the intention of collecting data to support claims in a medical product label. Recently, there has been an emphasis on limiting these measures to observable content, as recommended in the US Food and Drug Administration guidance on patient-reported outcomes. This qualitative study explores the concept of observability using the example of childhood attention deficit/hyperactivity disorder (ADHD). ⋯ Findings illustrate the substantial functional impairment associated with childhood ADHD while highlighting the challenges of developing informant-reported outcome measures limited to observable content. Because ADHD has an impact on children's functioning in a wide range of contexts, a parent-report measure that includes only observable content may fail to capture important aspects of functional impairment. Approaches for addressing this observability challenge are discussed.
-
To examine treatment patterns, dosing, health care resource utilization, and cost of tumor necrosis factor inhibitors (TNFi), adalimumab (ADA) and infliximab (IFX), among patients enrolled in US Humana insurance plans who have been diagnosed with ulcerative colitis (UC). ⋯ Approximately half of ADA and IFX users discontinued, with approximately half of discontinuers not restarting/switching therapies. Further investigation of treatment patterns and outcomes after TNFi discontinuation is required.