REAL-WORLD HEALTHCARE RESOURCE UTILIZATION AND ECONOMIC BURDEN OF CONGENITAL HYPERINSULINISM IN PEDIATRIC PATIENTS IN THE UNITED STATES
Author(s)
Bitten Kloster, MSc1, Shivani Pandya, MSc2, Rasmus Rogvi, MD1, Chi-Chang Chen, PhD3, Louise Fleron Herbild, PhD1.
1Zealand Pharma A/S, Soeborg, Denmark, 2IQVIA, New Brunswick, NJ, USA, 3IQVIA, Philadelphia, PA, USA.
1Zealand Pharma A/S, Soeborg, Denmark, 2IQVIA, New Brunswick, NJ, USA, 3IQVIA, Philadelphia, PA, USA.
OBJECTIVES: Congenital Hyperinsulinism (CHI) is an ultra-rare condition with neonatal onset characterized by dysregulated insulin secretion, recurrent hypoglycemia and high risk of neurological complications if uncontrolled. This study assessed healthcare resource utilization (HCRU) and costs among pediatric patients with CHI in the United States.
METHODS: Pediatric patients (≤18 years) with ≥1 diagnosis of CHI and ≥1 diazoxide prescription between April 2016 and June 2023 were identified from IQVIA’s Professional Fee (Dx) claims, Longitudinal Prescription (LRx) claims, and Hospital Charge Master databases. The index date was defined as earliest CHI diagnosis or first diazoxide prescription date. Claims activity in LRx and Dx databases was required for 12-month post-index (starting on index). Analyses focused on infants (0-<1 year) and evaluated all-cause HCRU and costs during the 12 months post-index period.
RESULTS: A total of 367 infants with CHI were included. Of these, 61.3% (n = 225) experienced ≥1 inpatient stay of any kind either as the initial qualifying CHI event or anytime in the 12 months post-index period, with mean of 1.8 admissions per patient and mean length of stay of 29.4 days. About half (51.2%; n = 188) had ≥1 ICU stay, with mean length of stay of 36.6 days. Mean total costs were $203,913 (Standard deviation [SD]: $418,477), primarily driven by inpatient care. Among those with ≥1 inpatient stay, mean inpatient costs were $285,142 (SD: $427,989). No patients with pancreatectomy or F-DOPA-PET-CT procedures were identified, reflecting limitations of claims capture and consequently underestimation of total costs.
CONCLUSIONS: Infants with CHI experience substantial clinical and economic burden, with over half requiring ≥1 inpatient stay during 12 months follow-up—often prolonged and involving ICU care—which is the primary driver of healthcare costs. These findings suggest a severe, difficult-to-manage population and highlight a significant unmet need for more effective therapies.
METHODS: Pediatric patients (≤18 years) with ≥1 diagnosis of CHI and ≥1 diazoxide prescription between April 2016 and June 2023 were identified from IQVIA’s Professional Fee (Dx) claims, Longitudinal Prescription (LRx) claims, and Hospital Charge Master databases. The index date was defined as earliest CHI diagnosis or first diazoxide prescription date. Claims activity in LRx and Dx databases was required for 12-month post-index (starting on index). Analyses focused on infants (0-<1 year) and evaluated all-cause HCRU and costs during the 12 months post-index period.
RESULTS: A total of 367 infants with CHI were included. Of these, 61.3% (n = 225) experienced ≥1 inpatient stay of any kind either as the initial qualifying CHI event or anytime in the 12 months post-index period, with mean of 1.8 admissions per patient and mean length of stay of 29.4 days. About half (51.2%; n = 188) had ≥1 ICU stay, with mean length of stay of 36.6 days. Mean total costs were $203,913 (Standard deviation [SD]: $418,477), primarily driven by inpatient care. Among those with ≥1 inpatient stay, mean inpatient costs were $285,142 (SD: $427,989). No patients with pancreatectomy or F-DOPA-PET-CT procedures were identified, reflecting limitations of claims capture and consequently underestimation of total costs.
CONCLUSIONS: Infants with CHI experience substantial clinical and economic burden, with over half requiring ≥1 inpatient stay during 12 months follow-up—often prolonged and involving ICU care—which is the primary driver of healthcare costs. These findings suggest a severe, difficult-to-manage population and highlight a significant unmet need for more effective therapies.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HSD92
Topic
Economic Evaluation, Health Service Delivery & Process of Care, Real World Data & Information Systems
Disease
Pediatrics, Rare & Orphan Diseases