COST OF CARE ANALYSIS FOR U.S. PATIENTS WITH PRADER-WILLI SYNDROME (PWS)

Author(s)

Butler MG1, Manzardo A1, Strong TV2, Li JW3, Yin D3, Meng Q3, Silber A3, Francis K3, Hadker N3, Czado S4, Yeh M5, Miller JL6
1University of Kansas Medical Center, Kansas City, KS, USA, 2The Foundation for Prader-Willi Research, Walnut, CA, USA, 3Trinity Life Sciences, Waltham, MA, USA, 4Millendo Therapeutics, Westford, MA, USA, 5Millendo Therapeutics, Ann Arbor, MI, USA, 6University of Florida College of Medicine, Gainesville, FL, USA

Presentation Documents

OBJECTIVES: This study analyzed direct medical costs of PWS patients compared to non-PWS patients; cost variation between those receiving growth hormone (GH) and those not was also evaluated.

METHODS: Costs for commercially-insured PWS and non-PWS patients (<65 years of age) were identified via ICD diagnosis codes in IQVIA™ Health Plan Claims Data (1/2006 – 11/2018). Patients were required to have ≥12 months of continuous enrollment with observation limited to the first 12 months. Controls were matched based on age, sex, length of continuous enrollment, and payer type.

RESULTS: Observable medical expenditures were captured for 1,621 PWS and 8,105 non-PWS patients. The direct medical costs for PWS were 10 times higher than non-PWS overall. These differences peaked in infancy and adolescence ranging from 12.1 to 14.7 times higher for PWS patients <18 years of age compared to non-PWS. Across ages, the primary cost driver was inpatient care (~48% of PWS patient costs), followed by outpatient-based care and prescription costs (~21% and ~31% of costs, respectively). The mean annualized costs per PWS patient per year overall were $35,197 and $3,529 for PWS and non-PWS, respectively; ranges by age group were $7,960 - $97,673 and $1,589 - $8,724, respectively. Costs were higher among PWS patients taking GH (driven primarily by the cost of GH itself).

CONCLUSIONS: PWS patients require life-long care, reflected in higher costs relative to non-PWS patients. Because infancy and adolescence appear to be a critical period for intervention, preventative care should be a priority. Quantitation of direct medical costs may stoke awareness of PWS and heighten calls to investigate effective disease management, particularly for patients <18 years old. Future research should seek to elucidate long-term quality of life implications of access to select services (including GH).

Conference/Value in Health Info

2020-05, ISPOR 2020, Orlando, FL, USA

Value in Health, Volume 23, Issue 5, S1 (May 2020)

Code

PRO22

Topic

Economic Evaluation, Health Service Delivery & Process of Care

Topic Subcategory

Disease Management, Treatment Patterns and Guidelines

Disease

Diabetes/Endocrine/Metabolic Disorders, Pediatrics, Rare and Orphan Diseases, Systemic Disorders/Conditions

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