BACKGROUND: Individuals with hemophilia A (HA) may be prophylactically treated with factor VIII (FVIII) or nonfactor therapies (eg, emicizumab) to prevent or reduce the fre-quency of bleeding episodes.
OBJECTIVE: To assess—using a meta-analysis (MA) on the real-world evidence—total cost of care (TCC) in the United States among noninhibitor patients with HA switching from prophylaxis with FVIII to emicizumab.
METHODS: Publications related to real-world studies reporting TCC outcomes in noninhibitor patients with HA who switched from prophylaxis with FVIII thera-pies to emicizumab were systematically reviewed in the PubMed, Embase, Cochrane Library, and EconLit data-bases. Only publications about studies conducted in the United States were eligible. No date limitations were ap-plied. Identified publications were evaluated for inclusion in the MA, which was then performed for TCC outcomes. A sensitivity analysis was also conducted to restrict pa-tient populations from overlapping between studies due to the similar databases used by some studies to obtain patient records. All effect sizes for all endpoints were calculated using standardized mean change (SMC). The consistency of data was examined using the I2 value and the P value of the chi-square test.
RESULTS: Of the 89 screened publications, 4 unique studies with a TCC endpoint were identified. Three observational studies using data from US health care claims databases were sufficiently comparable for inclusion in the primary MA and sensitivity analysis. The post-switch TCC for emi-cizumab was statistically significantly greater than the pre-switch TCC for FVIII for all studies in the MA (overall pooled SMC: 0.431; 95% CI = 0.307-0.554; P < 0.001) and for all studies in the sensitivity analysis (overall pooled SMC: 0.467; 95% CI = 0.331-0.603; P < 0.001). There was no statistically significant evidence to suggest heterogeneity across the included studies for the primary or sensitivity analyses (I2 = 0%)
CONCLUSIONS: MA indicated that the TCC post-switch for emicizumab in noninhibitor patients with HA was statisti-cally significantly greater than the TCC pre-switch for FVIII for both the primary and sensitivity analyses. In addition to the previously proven efficacy of FVIII prophylaxis in non-inhibitor patients with HA, the results of this comparison using real-world evidence suggest that FVIII prophylaxis is also less costly. The post-switch higher TCC with emi-cizumab is an important consideration for payers when determining an optimal resource allocation strategy along-side patient outcomes.
