Use of Patient-Reported Outcomes Measurement Information System Pediatric Measures as Clinical Trial Endpoints: Experience from a Multicenter Pragmatic Trial in Children with Crohn's Disease.

OBJECTIVES: To evaluate whether Patient-Reported Outcomes Measurement Information System (PROMIS) pediatric patient-reported outcome (PRO) measures can serve as valid endpoints in a clinical trial of a chronic pediatric illness. STUDY DESIGN: We evaluated the responsiveness of PROMIS pediatric measures collected through the Clinical Outcomes of Methotrexate Binary Therapy in Practice (COMBINE) trial, a multicenter, randomized, double-blind, placebo-controlled, pragmatic clinical trial in pediatric patients with Crohn's disease (CD). We examined the relationships between changes in PROMIS pediatric measures and changes in disease activity by evaluating PRO score changes among patients who did and patients who did not experience improvement in disease activity. RESULTS: Participants included 266 children and adolescents with CD from a total of 35 institutions. Over the course of follow-up, participants showed improvement in most PRO domains, with the largest effect sizes observed for the clinically improved group. Patients who maintained steroid-free remission showed significantly lower PRO scores for the Pain Interference, Fatigue, and inflammatory bowel disease (IBD) Symptoms domains and higher scores for the Positive Affect domain. CONCLUSIONS: This study demonstrates the responsiveness of the PROMIS pediatric measures of Fatigue and Pain Interference as study endpoints in a large, multicenter pragmatic trial in pediatric CD, extending a growing body of research supporting the use of PROMIS pediatric measures as reliable PRO endpoints for clinical trials.

Differences in Reporting Child Health Needs and Status and Developmental Concerns between Mothers and Fathers: Findings from the National Survey of Children's Health.

Nationally representative data of 140 000 children in 2-parent households showed that fathers were more likely than mothers to report that a child was in good health and less likely to report the presence of a specific health condition, special health care needs, or unmet health service needs.

Creating Unidimensional Global Measures of Physician Practice Quality Based on Health Insurance Claims Data.

OBJECTIVE: To explore the extent to which commonly used claims-based process quality indicators can be used to create an internally valid global composite measure of physician practice quality. DATA SOURCES: Health insurance claims data (October 2007-May 2010) from 134 physician practices in Seattle, WA. STUDY DESIGN: We use confirmatory and exploratory factor analysis to develop theory- and empirically driven internally valid composite measures based on 19 quality indicators. DATA COLLECTION METHODS: Health insurance claims data from nine insurance companies and self-funded employers were collected and aggregated by third-party organization. PRINCIPAL FINDINGS: Our results did not support a single global measure using the entire set of quality indicators. We did identify an acceptable multidimensional model (RMSEA = 0.059; CFI = 0.934; TLI = 0.910). The four dimensions in our data were diabetes, depression, preventive care, and generic drug prescribing. CONCLUSIONS: Our study demonstrates that commonly used process indicators can be used to create a small set of useful composite measures. However, the lack of an internally valid single unidimensional global measure has important implications for policy approaches meant to improve quality by rewarding "high-quality physicians."