OBJECTIVE: Treat-to-target (T2T) recommendations suggest that rheumatoid arthritis (RA) patients should strive for remission or low disease activity (LDA). However, it is unclear whether patients experiencing a good response to biologic agents might experience further improvement in patient-reported outcomes (PROs) if they subsequently achieve a lower disease activity state, particularly the T2T goals of LDA or remission. METHODS: Using the Consortium of Rheumatology Researchers of North America database, we identified RA patients initiating biologic agents. We restricted the analysis to patients with improvement (Clinical Disease Activity Index [CDAI] improvement of >/=10 units) at 3-6 months (baseline visit; n = 1,368) with a followup visit approximately 9 months later (n = 984). Patients in CDAI remission or with a worsened disease activity category were excluded, leaving 562 eligible patients. PROs (global assessment, pain, and fatigue by 0-10 visual analog scales and disability by the modified Health Assessment Questionnaire [M-HAQ]) were examined at these 2 visits. Mean change in PROs compared achievement of a lower disease activity category versus staying in the same disease activity category, adjusting for potential confounders. RESULTS: Patients who achieved a lower disease activity category (40% of the eligible cohort, 86% of these achieving LDA or remission) had significantly better improvement in patient pain (-14.9; 95% confidence interval [95% CI] -18.4, -11.6), patient global (-17.5; 95% CI -20.8, -14.3), fatigue (-8.5; 95% CI -15.8, -1.3), and M-HAQ score (-0.13; 95% CI -0.18, -0.08) compared to patients who stayed in the same disease activity category. However, even for patients improving, fewer than half exceeded the minimum clinically important difference for each PRO. CONCLUSION: Achievement of a lower disease activity disease state, especially T2T goals, was associated with further improvement in PROs, albeit modest in magnitude.

BACKGROUND: Longer survival of patients with cystic fibrosis (CF) has been linked to initiation of national newborn screening, new therapies that prevent and treat pulmonary exacerbations, and closer monitoring of health outcomes. However, few studies have examined the economic impact of these medical advances on costs, and none have examined these costs longitudinally. METHODS: We used a nationwide database of the healthcare claims of privately insured individuals with CF between 2001 and 2007. Study subjects had at least two claims with diagnoses of CF (ICD-277.xx). We extracted inpatient admissions, outpatient visits, prescribed therapies, and screening procedures and then calculated all-cause medical utilization and annual medical costs, adjusted for inflation. We adjusted for comorbidity burden and tested longitudinal time trends using regression models. RESULTS: We identified 3,273 individuals with CF. Overall, the costs of prescription drugs, outpatient visits, and durable medical equipment increased by 59% during the 7-year period ($18,715 in 2001 vs. $29,718 in 2007, P < 0.001). The proportion of individuals hospitalized increased from 24.0% to 38.9%, P < 0.001. Annual testing of pulmonary function increased 53% (49.9% in 2001 to 76.3% in 2007, P < 0.001) and respiratory cultures more than doubled (27.9-67.5%, P < 0.001). Use of CF-related therapies also significantly increased (dornase alfa, 32.1-52.4%, P < 0.001; oral antibiotics, 54.1-71.8%, P = 0.007). Analyses by age showed the largest increases in total medical care costs occurred for the oldest CF patients (aged >30; $20,536 in 2001 to $56,116 in 2007, P < 0.001) and the youngest (aged <11; $3,060 in 2001 to $31,723 in 2007, P < 0.001). CONCLUSIONS: Although improvements in diagnosis and treatment have yielded substantial benefits, they have come at considerable cost, both in terms of treatment burden and healthcare dollars.