Patient preferences for managing chronic breast cancer-related lymphedema in China: evidence from a discrete choice experiment.

The prevalence of diabetes in children has been increasing in recent decades [–]. Although type 1 diabetes (T1D) continues to be more common in children, type 2 diabetes (T2D) now accounts for approximately one in three new diabetes diagnoses []. Pediatric T2D tends to be more aggressive and resistant to treatment than T2D diagnosed in adulthood [, ]. Pediatric T2D is associated with substantial impairment in health-related quality of life, including impact on school, social, and emotional functioning [–]. Prior to 2019, metformin and insulin were the only approved treatments for pediatric T2D. Since then, the United States (US) Food and Drug Administration (FDA) has approved empagliflozin [], dapagliflozin [], liraglutide [], and dulaglutide [] as treatments for T2D in children. As new non-insulin treatments for pediatric T2D are developed and tested [, ], it is important to evaluate patients’ perspectives of their experiences with these novel treatments. Several questionnaires have been developed for use in pediatric T1D, such as the Diabetes Family Conflict Scale – Child Version (DFCS), the Problem Areas in Diabetes Survey – Pediatric Version (PAID-Peds), and the Pediatric Quality of Life Inventory (PedsQL) Diabetes Module [–]. However, these previously developed patient-reported outcome (PRO) measures may not be appropriate for assessing the impact of pediatric T2D and currently available treatments. Therefore, these instruments may not be sensitive to key outcomes and features associated with the newer medications for T2D, such as the potential for weight loss and more convenient treatment regimens. For example, although the PedsQL Diabetes Module has been evaluated in a sample of children with T2D [], it includes items that are specifically relevant for children treated with insulin (e.g., “It hurts to get insulin shots”). These items may not be appropriate for children with T2D treated with non-insulin medication, and the inclusion of these inappropriate items could undermine content validity in a pediatric T2D sample, introducing challenges to scoring and responsiveness in this population. Measuring treatment impact in pediatric T2D requires a clear understanding of the concepts that are most important to patients in this modern treatment era, such as the potential for weight loss and more convenient treatment regimens. The purpose of this study was to conduct qualitative research to support the development of a new PRO measure designed to assess the impact of pediatric T2D and its treatment. To generate content for the draft questionnaire, interviews were conducted with clinical experts, children with T2D, and their caregivers. The aim of this study was to create an instrument capturing the effects of T2D and its treatment that are important to children with T2D. Therefore, the items were drafted primarily based on the impact of T2D as reported by children and their caregivers, while also considering the clinician input and literature review. The newly developed draft PRO measure was then examined in cognitive interviews with children with T2D. This qualitative research study was conducted in accordance with FDA guidance documents and ISPOR task force recommendations for pediatric PRO instrument development [–].