Economic Burden of Prader-Willi Syndrome Among Pediatric Patients in the United States
Author(s)
Nerissa C. Kreher, MD1, Deborah Hoffman, PhD2, Jan Tuzil, PhD3, Thibaud J. Prawitz, MSc3;
1Mountainview Clinical Development Consulting, LLC, Zillah, WA, USA, 2Acadia Pharmaceuticals, San Diego, CA, USA, 3Maple Health Group, New York, NY, USA
1Mountainview Clinical Development Consulting, LLC, Zillah, WA, USA, 2Acadia Pharmaceuticals, San Diego, CA, USA, 3Maple Health Group, New York, NY, USA
OBJECTIVES: Prader Willi syndrome (PWS) is a rare, neurobehavioral, life-long genetic disorder affecting 8,000-10,000 people in the United States (US). PWS evolves in several phases. In the first year, infants are hypotonic and feed poorly. Hyperphagia and lack of satiety develop between the age of 4.5 and adulthood and are associated with serious, life-threatening, health problems. Our study aims to estimate the economic burden of PWS among US pediatric patients.
METHODS: The all-payer claims dataset (APCD) was used to estimate the costs of PWS in patients 0 to 17 years old insured between 2014 and 2024. A comparative control group of randomly selected members was built through 1:1 propensity score-matching on sex, age, race/ethnicity, region, and payer type. A sensitivity analysis was conducted in which the matching included the van Walraven comorbidity score. Economic burden was quantified with cost ratios per person-year vs. controls among patients with at least one year of continuous enrollment.
RESULTS: The base-case matching provided two balanced cohorts of 2,578 patients in each group. Mean age across both cohorts was 6.3 years, female patients constituted 47.5% of the sample. Most (51.8%) were Medicaid-insured, 43.3% were commercially insured, 1.5% were dual-insured, and 3.4% had other insurance plans. Patients with PWS accrued 4.9 times greater total costs, 4.4 times greater medical costs, and 12.5 times greater pharmacy drug costs per year compared to patients without PWS. Most pharmacy costs were associated with growth hormone and most medical costs were accrued in the inpatient setting. The difference in annual costs was generally higher among patients aged < 2 years and among commercially-insured patients (respective cost ratios of 6.0 and 5.4).
CONCLUSIONS: Our analysis highlights the significant costs incurred by PWS patients relative to matched controls and the need for effective interventions to control PWS symptoms and prevent the use of costly healthcare resources.
METHODS: The all-payer claims dataset (APCD) was used to estimate the costs of PWS in patients 0 to 17 years old insured between 2014 and 2024. A comparative control group of randomly selected members was built through 1:1 propensity score-matching on sex, age, race/ethnicity, region, and payer type. A sensitivity analysis was conducted in which the matching included the van Walraven comorbidity score. Economic burden was quantified with cost ratios per person-year vs. controls among patients with at least one year of continuous enrollment.
RESULTS: The base-case matching provided two balanced cohorts of 2,578 patients in each group. Mean age across both cohorts was 6.3 years, female patients constituted 47.5% of the sample. Most (51.8%) were Medicaid-insured, 43.3% were commercially insured, 1.5% were dual-insured, and 3.4% had other insurance plans. Patients with PWS accrued 4.9 times greater total costs, 4.4 times greater medical costs, and 12.5 times greater pharmacy drug costs per year compared to patients without PWS. Most pharmacy costs were associated with growth hormone and most medical costs were accrued in the inpatient setting. The difference in annual costs was generally higher among patients aged < 2 years and among commercially-insured patients (respective cost ratios of 6.0 and 5.4).
CONCLUSIONS: Our analysis highlights the significant costs incurred by PWS patients relative to matched controls and the need for effective interventions to control PWS symptoms and prevent the use of costly healthcare resources.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
EE341
Topic
Economic Evaluation
Topic Subcategory
Cost/Cost of Illness/Resource Use Studies
Disease
SDC: Neurological Disorders, SDC: Rare & Orphan Diseases