(232) Impact of emicizumab on real-world healthcare resource utilization in the US

Background: : Clinical trial and real-world data have shown that emicizumab prophylaxis significantly improves bleeding outcomes in people with hemophilia A (PwHA). Additional real-world evidence on the impact of emicizumab on healthcare resource utilization (HCRU), as a result of fewer bleed events, would further support its value.

Objective: : To evaluate changes in real-world HCRU in PwHA in the US pre- and post-emicizumab prophylaxis initiation.

Methods: : This retrospective, observational cohort study used US adjudicated health plan claims data from IQVIA PharMetrics® Plus. PwHA were included if they had ≥1 emicizumab prescription filled between Nov 1, 2017, and Jun 30, 2023 (index date defined as first fill); continuous medical and pharmacy benefits for ≥12 months pre- and post-index; and ≥1 inpatient or ≥2 outpatient International Statistical Classification of Diseases, Tenth Revision hemophilia A (hereditary Factor [F]VIII deficiency) diagnosis codes pre-index or on the index date. PwHA were excluded if they were diagnosed with hemophilia B or other rare bleeding disorders during the study period. HCRU outcomes were evaluated during the 12 months pre- and post-emicizumab initiation and included all-cause hospitalizations; outpatient hospital, emergency room (ER) and physician office visits; and FVIII prescription fills. Pre- and post-emicizumab outcomes in the same PwHA were compared using descriptive statistics and χ2 and Wilcoxon signed-rank tests.

Results: : Among 472 PwHA, mean age was 24.7 years (SD, 17.3) and 98.7% were male. Most PwHA were from the South (37.3%) or Midwest (26.2%) and had commercial (60.2%) or self-funded (21.2%) insurance. In the 12 months pre-emicizumab, 53.7% of PwHA had claims for FVIII and/or bypassing agents. Following emicizumab initiation, fewer PwHA had any hospitalization (11.2% pre-emicizumab vs 5.5% post-emicizumab; P=0.001), outpatient hospital visit (86.7% vs 76.7%; P< 0.001) or ER visit (37.1% vs 24.8%; P< 0.001). Per PwHA, fewer average hospitalizations (mean [SD], 0.6 [3.9] vs 0.4 [3.6]; median, 0 vs 0; P=0.002) and ER visits (mean [SD], 0.8 [2.5] vs 0.4 [1.4]; median, 0 vs 0; P< 0.001) were observed post-emicizumab. Physician office HCRU did not change. FVIII prescription fills were significantly reduced post-emicizumab (mean [SD], 6.0 [9.7] vs 1.8 [3.7]; median [IQR], 1 [0-13] vs 0 [0-2]; P< 0.001).

Conclusions: : Based on real-world data, initiating emicizumab was associated with significantly decreased FVIII prescription fills, hospitalizations and ER visits among PwHA in the US.

Sponsorship:: F. Hoffmann-La Roche Ltd.