Estimating Healthcare Resource Use Associated With the Treatment of Phenylketonuria in the United States
Author(s)
Karly S. Louie, MSc, PhD1, Bharath Kumar Vendatham, MBA2, Rohit Marwah, MS2, Erin Muller, MS, MBA3, Kristin Lindstrom, MD3, Paul Okhuoya, MSc1.
1BioMarin Europe Ltd., London, United Kingdom, 2Definitive Healthcare, Framingham, MA, USA, 3BioMarin Pharmaceutical Inc, Novato, CA, USA.
1BioMarin Europe Ltd., London, United Kingdom, 2Definitive Healthcare, Framingham, MA, USA, 3BioMarin Pharmaceutical Inc, Novato, CA, USA.
OBJECTIVES: To estimate the healthcare resource utilization (HRU) associated with treatment of phenylketonuria (PKU).
METHODS: A retrospective study was conducted using deidentified HealthVerity claims data in the United States from 24 May 2018 to 31 March 2023. Adults (≥18 years) and adolescents (12-17 years) with a record of PKU (i.e., diagnosis and/or treatment with sapropterin dihydrochloride or pegvaliase) and with at least 12 months of continuous insurance enrolment post index date were eligible. The index date was assigned based on the first date of treatment recorded or first medical record for those with no record of treatment (i.e., proxy for patients prescribed medical nutrition therapy [MNT] alone, which is not uniformly covered by insurance). The analysis included 1721 adults with PKU (299 pegvaliase, 311 sapropterin dihydrochloride, 266 MNT only, and 845 with no record of treatment) and 405 adolescents (8 pegvaliase, 180 sapropterin dihydrochloride, 95 MNT only and 122 with no record of treatment). Annualized average HRU by treatment was calculated, and differences between treatment groups were tested using 95% confidence intervals (CI) of mean differences (MD).
RESULTS: Significantly lower HRU was observed in adults with PKU treated with pegvaliase vs. MNT only: total visits (MD: -40.0 [CI: -53.2, -26.8]), medical visits (MD: -47.8 [CI: -61.1, -34.5]), inpatient visits (MD: -1.0 [CI: -1.8, -0.2]), length of inpatient stay (days) (MD: -47.7 [CI: -71.2, -24.2]), other visits (MD: -36.5 [CI: -50.2, -22.8]). No significant differences were observed in number of outpatient, emergency room, or pharmacy visits. Both adults and adolescents with PKU on pharmacological therapy (pegvaliase or sapropterin dihydrochloride) had lower HRU compared with those treated with MNT only or with no record of treatment.
CONCLUSIONS: Adults and adolescents with PKU treated with a Phe-restricted diet and MNT only incur a substantial health economic burden compared with those receiving pharmacological treatment.
METHODS: A retrospective study was conducted using deidentified HealthVerity claims data in the United States from 24 May 2018 to 31 March 2023. Adults (≥18 years) and adolescents (12-17 years) with a record of PKU (i.e., diagnosis and/or treatment with sapropterin dihydrochloride or pegvaliase) and with at least 12 months of continuous insurance enrolment post index date were eligible. The index date was assigned based on the first date of treatment recorded or first medical record for those with no record of treatment (i.e., proxy for patients prescribed medical nutrition therapy [MNT] alone, which is not uniformly covered by insurance). The analysis included 1721 adults with PKU (299 pegvaliase, 311 sapropterin dihydrochloride, 266 MNT only, and 845 with no record of treatment) and 405 adolescents (8 pegvaliase, 180 sapropterin dihydrochloride, 95 MNT only and 122 with no record of treatment). Annualized average HRU by treatment was calculated, and differences between treatment groups were tested using 95% confidence intervals (CI) of mean differences (MD).
RESULTS: Significantly lower HRU was observed in adults with PKU treated with pegvaliase vs. MNT only: total visits (MD: -40.0 [CI: -53.2, -26.8]), medical visits (MD: -47.8 [CI: -61.1, -34.5]), inpatient visits (MD: -1.0 [CI: -1.8, -0.2]), length of inpatient stay (days) (MD: -47.7 [CI: -71.2, -24.2]), other visits (MD: -36.5 [CI: -50.2, -22.8]). No significant differences were observed in number of outpatient, emergency room, or pharmacy visits. Both adults and adolescents with PKU on pharmacological therapy (pegvaliase or sapropterin dihydrochloride) had lower HRU compared with those treated with MNT only or with no record of treatment.
CONCLUSIONS: Adults and adolescents with PKU treated with a Phe-restricted diet and MNT only incur a substantial health economic burden compared with those receiving pharmacological treatment.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE420
Topic
Economic Evaluation
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Rare & Orphan Diseases