Real-world annualized healthcare utilization and expenditures among insured US patients with acute intermittent porphyria (AIP) treated with hemin

Abstract

Background and aims: Patients with acute intermittent porphyria (AIP) may suffer from acute non-specific attacks that often result in hospitalizations or emergency room (ER) visits. Prior to the recent approval of givosiran (November 2019), hemin was the only FDA-approved therapy for AIP attacks in the US. Our aim was to estimate the annual healthcare utilization and expenditures for AIP patients treated with hemin using real-world data.

Methods: Patients with ≥1 hemin claim and confirmed AIP diagnosis – 1 inpatient claim or 2 outpatient claims ≥30 d apart for AIP (2015–2017) or acute porphyria (prior to 2015) – were identified in MarketScan administrative claims dataset between 2007 and 2017. Continuous enrolment for ≥6 months from confirmed diagnosis was required. A secondary analysis (“active disease population”) limited the sample to adult patients with ≥3 attacks or 10 months of prophylactic use of hemin within a 12-month pre-index period. AIP-related care was defined by hemin use during an attack (daily glucose and/or hemin use) or prophylaxis (non-attack hemin use). Outcomes were annualized and expenditures were inflated to 2017.

Results: Across 10 years, patients with a confirmed AIP diagnosis (N = 8,877) and ≥1 hemin claim (N = 164) were restricted by ≥6 months continuous follow-up (N = 139). AIP patients were mostly female (N = 112; 81%), had median age of 40 and 3 years average follow-up. Annualized average total expenditures for AIP-related care were $113,477. Annualized average all-cause (any diagnosis) hospitalizations were statistically significantly lower for patients treated with hemin prophylaxis vs. acute treatment (1.0 vs. 2.1; p < .001). In the secondary analysis (N = 27), annualized average total expenditures for AIP-related care were higher ($187,480).

Conclusions: For AIP patients treated with hemin, patients treated for acute attacks may use a greater number of resources compared to patients treated prophylactically.

Keywords:

• Hemin
• panhematin
• acute intermittent porphyria
• AIP
• healthcare resource utilization
• costs

JEL CLASSIFICATION CODES:

• I19
• I00

Transparency

Declaration of funding

This study was funded by Recordati Rare Diseases.

Declaration of financial/other interests

PS is a paid employee at Recordati Rare Diseases. BB and JE are employees at Stratevi, which was retained for this work.

A peer reviewer on this manuscript has disclosed that they have received honoraria for participation in disease training videos and advisory boards for Recordati. The peer reviewers on this manuscript have no other relevant financial relationships or otherwise to disclose.

Author contributions

BB: study design, data analysis, drafting of the manuscript. JE: study design, data interpretation, and revision of the manuscript. PS: study design, data interpretation, review of the manuscript, and study supervision.

Previous presentations

A first look at results were shared in a poster presentation at the International Congress on Porphyrins and Porphyrias (Milan, Italy; September 8–11, 2019).

Acknowledgements

No assistance in the preparation of this article is to be declared.