4 Your Kids Care: Reducing Non-Emergent Hospitalization In A Medicaid Pediatric Population Through Hands-On Training And Parent/Caregiver Education

Abstract

objeCtives: In 2012 United States Food and Drug Administration (FDA) created a new expedited pathway of 'Breakthrough Therapy Designation' (BTD) to enable early approval of therapies, which have shown substantial activity in early trials. The objective of this study was to understand the impact on BTD on patients and payers. Methods: The data for number of granted BTDs was obtained from FDA. gov. The data for publically disclosed BTDs was obtained from sponsor's press releases. For all products the information for their mechanism of action, type of molecule, trial design, clinical efficacy and safety, and pricing and time to approval (for approved products) were obtained from peer-reviewed publications, conference abstracts, FDA and sponsor websites. Results: Since the establishment of the BTD pathway, 37 products have been granted breakthrough therapy designations (2012-2013), of which, 28 have been publically disclosed by the manufacturers and 3 have been approved by the FDA. In terms of indications, 12 (43%) are for cancer, 5 (18%) are for genetic diseases and 4 (14%) are for Hepatitis C Genotype 1. The three approved drugs with BTD are Gazyva, Imbruvica and Sovaldi. The median time to approval for these three drug was ~5 years, significantly shorter than the 2012 median time to approval for priority review applications (6 years). However, the price premium was 30-50% compared to other drugs in the same category. Two of the drugs with BTD did not meet primary endpoint in their pivotal trial. While the BTD pathway promises to reduce development time, the high price is a major concern for payers and patients. ConClusions: BTD is a promising pathway to shorten development time and provides early access, however, high price could pose challenges for payers and patients.

Characteristics of non‐urgent neonatal presentations to an Australian metropolitan emergency department

“…There was no statistically significant difference in the urgency of presentations or admissions pre/post‐COVID‐19. This contrasts with previous studies, which found decreased presentation rates, decreased NUPs and increased admission rates 17,18,20,21 . It would be worthwhile to see whether there is any change in presentations/admission post‐Delta variant where the caseloads were much higher than had been seen the year prior.…”

“…19 Similarly, a study by Grant et al examining the effect of a short programme that aimed at providing education on how to deal with common childhood illnesses to parents and advising for review by primary care provider before seeking ED care showed a decrease in NUPs among the study group who received an education. 20 This study also found that neonates with younger mothers were more likely to have NUPs to the ED. This finding is consistent with previous studies identifying younger maternal age as a risk factor.…”

“…Similarly, a study by Grant et al . examining the effect of a short programme that aimed at providing education on how to deal with common childhood illnesses to parents and advising for review by primary care provider before seeking ED care showed a decrease in NUPs among the study group who received an education 20 …”

“…This contrasts with previous studies, which found decreased presentation rates, decreased NUPs and increased admission rates. 17,18,20,21 It would be worthwhile to see whether there is any change in presentations/admission post-Delta variant where the caseloads were much higher than had been seen the year prior. Further studies are warranted to elucidate this.…”