CHARACTERIZING AGE-RELATED PATTERNS IN HEALTHCARE COSTS AND UTILIZATION AMONG PEOPLE LIVING WITH ANGELMAN SYNDROME IN THE UNITED STATES
Author(s)
Farah Pathan, PhD1, Lynne Bird, MD2, Xinshuo Ma, MS1, Xiyuan Wu, MS3, Kyle Householder, PhD4, Montserrat Vera-Llonch, MS4, Steven Kymes, MHA, PhD4.
1Komodo Health, New York, NY, USA, 2Rady Children’s Hospital, San Diego, CA, USA, 3Komodo Health, San Francisco, CA, USA, 4Ionis Pharmaceuticals, Inc, Carlsbad, CA, USA.
1Komodo Health, New York, NY, USA, 2Rady Children’s Hospital, San Diego, CA, USA, 3Komodo Health, San Francisco, CA, USA, 4Ionis Pharmaceuticals, Inc, Carlsbad, CA, USA.
OBJECTIVES: Angelman Syndrome (AS) is a rare genetic neurodevelopmental disorder with lifelong medical needs. Our objective was to assess healthcare resources utilization (HCRU) and costs in pediatric and adult patients with AS across calendar years (CY) 2021-2024.
METHODS: This retrospective, observational study was conducted using the Komodo Research Dataset. Insured beneficiaries in the United States with AS were identified using ≥2 medical claims, at least 30 days apart between January 1, 2021, and December 31, 2024. Age at index defined pediatric (<18 years) and adult (≥18 years) cohorts. Costs (adjusted to 2024 US dollars) and events were summarized descriptively by year and age group.
RESULTS: CY2021-2024, the annual pediatric cohort ranged from 419-554 patients and adult cohorts 459-548; mean age was 10 years for pediatrics and 29 years for adults. During the years of 2021-2024, in the pediatric cohort the annual prevalence of seizures ranged from 74% to 78%, gastrointestinal issues from 40% to 45%, and abnormal movement or mobility issues in 31% to 38%. Adults found a similar annual prevalence of seizures (73% to 80%), and gastrointestinal issues (36% to 49%), but a lower reported prevalence of abnormal movement or mobility issues (18% to 23%). During the four-year period, the mean total annual costs were similar between pediatric (ranging from $33,072-$40,508) and adult ($30,034-$43,726) patients. The primary cost driver for pediatric patients was outpatient services, ranging from $15,385-$19,009/year during the period. For adults it was home health services/durable medical equipment, ranging from $11,756-$19,781.
CONCLUSIONS: The study highlights the growing economic burden of AS. Age-stratified analyses describe similar overall costs in children and adults, despite nominal differences by age in outpatient and home health services use. Further research is needed to determine if this trend is robust with the aging of people living with AS.
METHODS: This retrospective, observational study was conducted using the Komodo Research Dataset. Insured beneficiaries in the United States with AS were identified using ≥2 medical claims, at least 30 days apart between January 1, 2021, and December 31, 2024. Age at index defined pediatric (<18 years) and adult (≥18 years) cohorts. Costs (adjusted to 2024 US dollars) and events were summarized descriptively by year and age group.
RESULTS: CY2021-2024, the annual pediatric cohort ranged from 419-554 patients and adult cohorts 459-548; mean age was 10 years for pediatrics and 29 years for adults. During the years of 2021-2024, in the pediatric cohort the annual prevalence of seizures ranged from 74% to 78%, gastrointestinal issues from 40% to 45%, and abnormal movement or mobility issues in 31% to 38%. Adults found a similar annual prevalence of seizures (73% to 80%), and gastrointestinal issues (36% to 49%), but a lower reported prevalence of abnormal movement or mobility issues (18% to 23%). During the four-year period, the mean total annual costs were similar between pediatric (ranging from $33,072-$40,508) and adult ($30,034-$43,726) patients. The primary cost driver for pediatric patients was outpatient services, ranging from $15,385-$19,009/year during the period. For adults it was home health services/durable medical equipment, ranging from $11,756-$19,781.
CONCLUSIONS: The study highlights the growing economic burden of AS. Age-stratified analyses describe similar overall costs in children and adults, despite nominal differences by age in outpatient and home health services use. Further research is needed to determine if this trend is robust with the aging of people living with AS.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
EE135
Topic
Economic Evaluation
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
SDC: Neurological Disorders