Clinical and economic impacts of large volume delayed sampling and pathogen reduction technology platelet processing strategies in the United States.

BACKGROUND: Large volume delayed sampling (LVDS) and pathogen reduction technology (PRT) are strategies for platelet processing to minimize transfusion of contaminated platelet components (PCs). This study holistically compares the economic and clinical impact of LVDS and PRT in the United States. STUDY DESIGN AND METHODS: A decision model was constructed to simulate collection, processing, and use of PCs and to compare processing strategies: PRT with 5-day shelf life, LVDS with 7-day shelf life (LVDS7), and LVDS with 5-day shelf life extended to 7 days with secondary testing (LVDS5/2). Target population was adults requiring two or more transfusions. Collection, processing, storage, and distribution data were obtained from the National Blood Collection and Utilization Survey and published literature. Patient outcomes associated with transfusions were obtained from AABB guidelines, meta-analyses, and other published clinical studies. Costs were obtained from reimbursement schedules and other published sources. RESULTS: Given 10,000 donated units, 9512, 9511, and 9651 units of PRT, LVDS5/2, and LVDS7 PCs were available for transfusion, respectively. With these units, 1502, 2172, and 2329 transfusions can be performed with similar levels of adverse events. Assuming 30 transfusions a day, a hospital would require 69,325, 47,940, and 45,383 units of PRT, LVDS5/2, and LVDS7 platelets to perform these transfusions. The mean costs to perform transfusions were significantly higher with PRT units. CONCLUSIONS: Compared with PRT, LVDS strategies were associated with lower costs and higher PC availability while patients experienced similar levels of adverse events. Increased utilization of LVDS has the potential to improve efficiency, expand patient access to platelets, and reduce health care costs.

Mild traumatic brain injury in the United States: demographics, brain imaging procedures, health-care utilization and costs.

Objective: To characterize mild traumatic brain injury (mTBI) patients in the USA, describing location of diagnosis, timing, and modality of imaging procedures, health-care resource utilization (HRU) and costs in the 12-month period post-diagnosis. Research Design: Retrospective claims analysis Methods: Anonymized data from the OptumHealth Care Solutions claims database (2006-2016). The index date was the first date with an mTBI diagnosis. HRU and costs (2016 USD) were assessed in the 12-month post-index period. Results: A total of 80,004 patients with mTBI were included: 60% were under 26 years and 54% were male. Mild TBI was most frequently diagnosed in an emergency department (ED) for all age groups, except patients aged 11-17 years, for whom the outpatient setting was the most frequent place of diagnosis. Almost half (47%) received brain imaging on the index date, with 98% of which receiving computed tomography. Mean follow-up health-care costs were $13,564 (SD = $41,071), primarily from inpatient ($4,675, SD = $29,982) and non-ED outpatient/physician office visits ($4,207, SD = $12,697). Older patients had greater HRU and higher health-care costs. Conclusions: The findings of this claims-based study show substantial HRU and costs associated with mTBI diagnosis during a 12-month follow-up period.

Economic and clinical benefits of early identification of acute kidney injury using a urinary biomarker.

Purpose: To evaluate the budget impact of adding a diagnostic test of tissue inhibitor of metalloproteinase 2 and insulin-like growth factor binding protein 7 ([TIMP-2]·[IGFBP7]), which identifies patients at risk of moderate-to-severe acute kidney injury (AKI), to the current standard of care (SOC) in a hospital setting.Materials and methods: A budget impact model (2017 USD) was developed from the perspective of a hypothetical US hospital system serving 10,000 inpatients annually. The model estimated the impact of assessing the risk of AKI using SOC vs a combination of SOC and the US Food and Drug Administration-approved assay [TIMP-2]·[IGFBP7] over a 1-year period. Potential cost implications were assessed using estimates for payer mix among patients, diagnostic efficacy, and patient healthcare resource utilization. The model also considered provider adoption rates and the estimated costs of [TIMP-2]·[IGFBP7].Results: Compared to SOC alone, adding [TIMP-2]·[IGFBP7] to SOC was associated with a $1,855 reduction in uncompensated care per patient tested, which, after accounting for the additional costs of the test ($277), resulted in net savings of $1,578 per patient tested. The findings were robust to input parameter variations, as demonstrated by deterministic and probabilistic sensitivity analyses. In the probabilistic sensitivity analyses, net cost savings to the hospital ranged from $50,308-$3,971,514, or $101-$7,943 per tested patient (mean = $1,710; 95% confidence interval = $1,691-$1,729).Conclusions: The introduction of [TIMP-2]·[IGFBP7] as a novel tool in the identification of AKI risk may result in considerable cost savings from a hospital perspective under this model's base-case assumptions. Further prospective studies are needed to confirm these findings in a real-world setting.Key points for decision makersAn economic model was constructed to determine the budget impact of adding a diagnostic test ([TIMP-2]·[IGFBP7]), which identifies patients at risk of moderate-to-severe acute kidney injury, to the current standard of care (SOC) in a hospital setting.According to the present model, the use of [TIMP-2]·[IGFBP7] to identify acute kidney injury risk may reduce costs for hospitals by ∼$1,578 per patient tested.

Psychometric evaluation of a caregiver diary for the assessment of symptoms of respiratory syncytial virus.

BACKGROUND: There are no clinical outcome assessment (COA) tools developed in accordance with Food and Drug Administration (FDA) guidance suitable for the evaluation of symptoms associated with respiratory syncytial virus (RSV) infection among infants. The Gilead RSV Caregiver Diary (GRCD) is being developed to fulfill this need; the present research evaluates the GRCD and documents its reliability, validity, and responsiveness among children < 24 months of age with acute RSV infection. METHODS: A prospective, observational study was conducted in the United States during the 2014-2015 northern hemisphere winter season. Subjects were < 24-month, full-term, previously healthy infants with confirmed RSV infection and ≤5 days of symptoms. The GRCD was completed twice daily for 14 days by caregivers. Additional data were collected during the initial visit, subsequent visits, and end-of-study interview. Test-retest reliability (kappa and intraclass correlation coefficients [ICCs]), construct validity (correlations and factor analyses), discriminating ability (analyses of variance and chi-square), and responsiveness (effect sizes and standardized response means) were evaluated. RESULTS: A total of 103 subjects were enrolled (mean age 7.4 ± 5.3 months). GRCD items were grouped into different subscales according to question content, which, with the exception of the behavior impact domain (ICC = 0.43), demonstrated internal consistency (alphas = 0.78-0.94) and test-retest reliability (ICCs = 0.77-0.94). Hypothesized correlations with parent global ratings of RSV severity ranged from 0.45 to 0.70 and provided support for construct validity. Support for discriminating ability was limited. Effect sizes ranged from - 1.48 to - 4.40, indicating the GRCD was responsive to change. CONCLUSIONS: These psychometric analyses support the validity, reliability, and responsiveness of the GRCD for assessing RSV symptoms in children < 24 months of age.

Development of a novel observer-reported outcome measure for the assessment of Respiratory Syncytial Virus (RSV) infection symptoms in pediatric clinical trials.

BACKGROUND: Respiratory syncytial virus (RSV) is a seasonal infection affecting most children by 2 years of age and the leading cause of lower respiratory tract infection requiring hospitalization in infants. Novel antiviral medications are in development to improve the clinical outcomes of RSV; however, no clinical outcome assessments (COAs) for RSV have been developed in alignment with the United States Food and Drug Administration patient-reported outcome guidance to assist in the evaluation of new therapies. To address this need, an observer-reported outcome (ObsRO) measure designed to assess observable RSV symptoms was created. METHODS: The literature was reviewed to evaluate existing COAs and identify constructs of interest. Individual caregiver interviews elicited concepts that informed item development, and candidate items were subsequently evaluated in two rounds of cognitive testing. Separate cohorts of caregivers of RSV-infected nonhospitalized and hospitalized infants participated. Therapeutic-area experts provided input throughout the instrument development process. RESULTS: Caregivers of 39 children < 24 months old with RSV (31 nonhospitalized, 8 hospitalized) participated in in-depth, individual interviews during concept elicitation and cognitive debriefing, resulting in 21 concepts identified as potentially observable and relevant to young children with RSV. The item pool was reduced to 12 cardinal symptoms and behavior impacts reported to be directly observable by caregivers, with 10 daytime and 9 nighttime symptoms to capture diurnal variation in severity. CONCLUSIONS: The RSV Caregiver Diary assesses RSV symptom severity and change from the parent or caregiver perspective in a standardized manner to measure treatment benefit. Following psychometric evaluation and refinement, this tool is expected to be suitable for assisting in the clinical development of RSV therapeutics.

Improved timing of availability and administration of influenza vaccine through the US Vaccines for Children Program from 2007 to 2011.

The Vaccines for Children (VFC) program distributes one half of all vaccines administered to US children. Compared with commercial vaccine distribution, VFC distribution is more complex. This prospective observational study compares the delivery and administration of VFC versus non-VFC influenza vaccine in US pediatricians' offices across 3 influenza seasons and its apparent impact on 2-dose compliance rates. Because of earlier shipping (mean = 29-42 days) of non-VFC vaccines, administration of VFC vaccines was delayed relative to non-VFC vaccines by approximately 1 month in 2007-2008 and 2008-2009 and 2 weeks in 2010-2011. Two-dose compliance rates for the VFC and non-VFC populations were 38.5% and 47.5% (P < .001) in 2007-2008, 45.9% and 55.1% (P < .001) in 2008-2009, and 50.0% and 52.9% (P < .001) in 2010-2011, respectively. Despite delays, earlier VFC shipment in 2010-2011 enabled greater equity in 2-dose compliance.

A multi-season national estimate of adult influenza vaccination by U.S. office-based pediatricians, 2006-2011.

There is no national estimate of adult influenza vaccination by U.S. office-based pediatricians. De-identified patient-level data from an electronic healthcare claims database submitted to private and public insurers were analyzed for pediatric offices from the 2006-2007 through 2010-2011 seasons. An average of 321,000 (range: 225,000-434,000) influenza vaccinations per year were estimated to be administered to adults; 52%, 22%, and 26% were given to adults 19-49, 50-64, and ≥65 years of age, respectively. Consistent with the 2010 changes to national guidelines, recommending influenza vaccination of all individuals 6 months of age and older, pediatricians appear to be providing an increasing proportion of adult vaccinations against influenza to adults 19-49 years of age (probably parents of their pediatric patients).

Trends in U.S. pediatric influenza vaccination from 2006 to 2010 among children with private insurance.

In the United States, recommendations for the annual influenza vaccination of children have expanded significantly in recent years. Additionally, to facilitate influenza vaccination delivery by providers, recent recommendations have encouraged vaccination as soon as vaccine is available and throughout the influenza season. However, until now, there have been limited data published describing pediatric providers' responses to these recent recommendations. De-identified, patient-level data from an electronic health care reimbursement claims database that contains more than 60% of all medical claims from outpatient settings in the US were analyzed. Only claims from privately insured children were available; administration of federally purchased vaccine (i.e., via the Vaccines for Children program) and vaccinations administered in settings where claims data are not generated were not captured. Weekly counts of influenza vaccinations administered to children 6 months through 18 years of age between August 1 and March 31 for the 2006-2007 through 2009-2010 seasons were projected to yield national estimates. Seasonal vaccination peaked in November for the 2006-2007 and 2007-2008 seasons, October for the 2008-2009 season, and September for the 2009-2010 season. The proportion of vaccinations administered before November 1 increased each season from 2006-2007 through 2009-2010. In all seasons, vaccination dramatically declined in December and continued at a steadily declining rate through the end of the season. Vaccine delivery to children 6-23 months of age was more dispersed over the vaccination season relative to older age groups. Among children 6-23 months and 2-18 years of age, use of preservative-free inactivated vaccine and live attenuated vaccine, respectively, increased significantly over the study period. While pediatric influenza vaccination occurred earlier each year, vaccination in later months has not increased in recent seasons, despite efforts to extend the vaccination season.