Pragmatic patient engagement in designing pragmatic oncology clinical trials.

Oncology trials are the cornerstone of effective and safe therapeutic discoveries. However, there is increasing demand for pragmatism and patient engagement in the design, implementation and dissemination of oncology trials. Many researchers are uncertain about making trials more practical and even less knowledgeable about how to meaningfully engage patients without compromising scientific rigor to meet regulatory requirements. The present work provides practical guidance for addressing both pragmaticism and meaningful patient engagement. Applying evidence-based approaches like PRECIS-2-tool and the 10-Step Engagement Framework offer practical guidance to make future trials in oncology truly pragmatic and patient-centered. Consequently, such patient-centered trials have improved participation, faster recruitment and greater retention, and uptake of innovative technologies in community-based care.

Economic burden of interstitial lung disease in a commercially insured population with Sjögren syndrome in the United States.

BACKGROUND: Patients with Sjögren syndrome (SjS) have substantial cost burden on the health care system; among these patients, those who develop interstitial lung disease (ILD) experience poorer quality of life and have a higher mortality risk. However, the economic burden of ILD has not been documented. OBJECTIVE: To estimate the direct health care costs associated with ILD among patients with SjS in a representative sample of the commercially insured population in the United States. METHODS: Individuals with a diagnosis of SjS between January 1, 2006, and September 30, 2015, with and without a diagnosis of ILD, were identified from the PharMetrics Plus for Academics database. The index date was defined as the later date of the first claim with a diagnosis of SjS or the first claim with a diagnosis of ILD for individuals with SjS and ILD (SjS-ILD), and the first claim with a diagnosis of SjS for SjS-only controls. All baseline variables were measured in the 180 days preindex period. A 5:1 propensity score matching was applied to controls for baseline demographic and geographic variables. The cost ratio and average marginal effect for total direct medical costs comparing SjS patients with and without ILD were estimated using a generalized linear model. Costs per health care resource utilization category were also reported. All costs were represented from a health plan payer perspective and inflated to 2020 US dollars. RESULTS: After applying the inclusion criteria, 815 SjS-ILD cases were identified and matched to 4,075 SjS-only controls based on the 5:1 propensity score matching procedure. The 180-day total cost of SjS-ILD cases was about 2 times higher compared with that of SjS-only controls (adjusted cost ratio = 1.95; 95% CI = 1.76-2.15). The average difference in total cost between patients with and without ILD was $8,814 (95% CI = $7,149-$10,479). Costs were mainly contributed from outpatient services other than physician office visit (such as radiological and pathological tests), inpatient services, and outpatient pharmacy cost components for both groups (39.4%, 38.8%, and 16.3% for SjS-ILD cases; 43.7%, 22.6%, and 22.9% for SjS-only controls, respectively). CONCLUSIONS: Total direct health care cost was substantially higher in patients with SjS and ILD compared with patients with SjS without ILD. Our findings provide the foundation for further economic evaluation for preventive strategies to reduce the clinical and economic burden imposed by ILD among patients with SjS.

Economic evaluation of 2014 cholera outbreak in Ghana: a household cost analysis.

INTRODUCTION: Ghana experienced its worst cholera outbreak in three decades in 2014. Evidence of cholera economic costs on affected households has been limited. This study aimed at determining economic costs on households affected by the  cholera outbreak in a Coastal Region of Ghana. METHODS: Two districts; High and Low Incidence Areas (HIA and LIA) were selected in comparative cost analysis and disease impact on affected households assessed based on scientifically documented economic indicators. A total of 418 (282 HIA and 136 LIA) households that experienced at least one case of cholera infection were interviewed. Direct and indirect costs were estimated. Correlates of household's cholera infection were estimated using Tobit Regression model in STATA 13. RESULTS: Average direct cost to households in HIA amounted to USD 106.88, almost 2 folds higher than LIA (USD 62.02). Potential cost saving of an episode of cholera is USD 99,201.28 in LIA and raises almost 8 folds in HIA (USD 782,611.60). Households in lowest income category had the highest incidence of cholera (0.073) compared to other categories plus other factors were significant in explaining cholera incidence. CONCLUSIONS: The study showed considerable differences in HIA and LIA costs with higher household economic impact of cholera on the lowest income category. Results underscore the need for pragmatic policy interventions  to avert recurrent outbreaks and emphasis huge potential  cost saving with reducing  cholera cases.