Newborn Screening in the DMV Area

The vast majority of children in the United States undergo newborn screening shortly after birth, making it a remarkably successful public health initiative. Though a Recommended Uniform Screening Panel (RUSP) has been developed and continues to be updated, comprised of both a core conditions panel and a secondary targets panel, its implementation is not required. Each jurisdiction ultimately decides which conditions to include on its newborn screen (NBS), leading to considerable variation across the country. Here, we assessed the panels in Maryland, the District of Columbia (DC) and Virginia. We considered the differences between the three and compared each panel to the RUSP. Importantly, we also investigated why these differences exist. We found that though all three included a significant proportion of the core conditions panel on their NBS, none included the entire panel. When the secondary targets were considered, we found that both Maryland and DC again included many of the conditions, though not all. Virginia did not include any secondary target conditions. Maryland and DC also included conditions that were absent from both the core conditions and secondary targets panels to their NBS. We identified technical constraints and testing optimization as the main factors contributing to the size of Virginia\u27s NBS as well as to the group of conditions it includes. Continued research is needed to identify the contributing factors in both Maryland and DC. Additionally, the reasons for including conditions not currently on the RUSP in both these jurisdictions remain to be determined

A Rare Disease, But a Costly One: The Case of Myasthenia Gravis

Myasthenia gravis (MG) is a chronic, autoimmune neuromuscular disorder affecting fewer than 200,000 individuals in the United States. Disease management is primarily outpatient, but disease exacerbations leading to hospital admission occur in upwards of a third of patients. We sought to determine the inpatient cost of care and assess changes over a 10 year epoch. Our cohort was identified from the Nationwide Inpatient Sample database for the years 2003 through 2013 using the ICD-9-CM codes. We compared MG to a more common chronic neurological disorder, multiple sclerosis (MS), in which patients also require episodic hospitalizations and to the total US discharges. Total costs of MG inpatient care costs rose by 13 fold during the study period to $546,834,101. In contrast, MS and all inpatient costs doubled. The increase was largely accounted for by an increase in patient discharges for MG from 870 to 5,353, while MS discharges were close to flat and total discharges dropped. Per discharge costs rose from$48,024 to $98,795 for MG,$16,989 to $32,767 for MS, and$19,666 to 39,462 for all discharges. Length of stay for MG patients increased from 7.4 to 8.0 days while staying flat in the other groups. More discharges were appreciate in the 0-17 year and 85+ year age groups for MG patients in 2013. For MG patients, the percentage with private insurance decreased and number of uninsured MG patients increased in 2013 compared to 2003. Regional variations in cost were appreciated with greater rises in mean charges in the Midwest and South for MG out of proportion to MS and all hospital discharges. A dramatic rise in hospital discharges for MG has occurred with considerable increase in cost to the health care system. This was largely driven by growth in patient discharges. Our data set does not allow us to identify reasons for this but may reflect an increase in the prevalence of MG, improved identification of patients, or an underlying change in practice patterns

Shortage of Neurological Therapeutics: An Escalating Threat to Patient Care

Background: Drug shortages are a well-recognized and growing threat to patient care, and can lead to the potential for substitutions of medications that are less effective and may delay critical treatments. Shortages have not been assessed for therapies for neurological conditions. Objective: We assess longitudinal trends in the shortages of generic drugs used for neurological conditions over a fifteen-year period in the United States. Methods: Drug shortage data from the University of Utah Drug Information Service (UUDIS) from 2001 to 2015 were analyzed. Medications were defined as those likely to be prescribed by a neurologist to treat a primary neurological condition or critical for care of a patient with a neurological condition. Trends in shortage length were assessed using standard descriptive statistics. Results: A total of 2,081 drug shortages were reported by UUDIS and 311 (15%) involved medications for neurological conditions. After excluding discontinued products, 291 shortages were analyzed. The median number of neurological drugs in shortage was 21 per month (median 7.4 per month). From 2001 to 2009, the number of neurological drugs in shortage never exceeded 25 in any month. However, beginning in 2009, shortages rose steadily, reaching a high of 50 in December 2012 and 50 again in December 2014. By the end of the study period, 30 neurological drugs remained in shortage. In over half the shortages, manufacturers did not provide a reason for the shortage. When reported, manufacturing delays, followed by supply/demand issues, raw material shortages, regulatory issues and business decisions were cited. Conclusion: Caring for patients with neurological conditions is becoming increasingly compromised by U.S. drug shortages. Manufacturers, together with professional organizations, patient advocacy groups, and the government needs to continue to address this issue, which will escalate with a growing burden of neurological disease