Comparison of Real-World Dose and Consumption for Two Extended Half-Life Recombinant Factor VIII Products for the Treatment of Hemophilia A in the United States

Abstract

Background

US patients with hemophilia A can receive prophylaxis with extended half-life recombinant factor VIII (rFVIII) products, including efmoroctocog alfa (fragment crystallizable fusion protein) and rurioctocog alfa pegol (antihemophilic factor [recombinant], PEGylated).

Objective

To evaluate dosing patterns and weekly consumption of extended half-life rFVIII products in the United States.

Methods

We performed a retrospective analysis using the US Specialty Pharmacy Database (2015–2018). Included patients had a diagnosis of hemophilia A, ≥2 consecutive monthly claims for efmoroctocog alfa or rurioctocog alfa pegol for prophylaxis, and weight data. Outcome measures included weekly dosing frequency and dispensed weekly dose.

Results

The analysis included 774 patients (efmoroctocog alfa, 506; rurioctocog alfa pegol, 268). Mean (SD) age was 24.2 (15.8) and 26.3 (14.9) years for patients receiving efmoroctocog alfa and rurioctocog alfa pegol, respectively; mean (SD) weight was 68.4 (36.8) and 79.8 (37.7) kg, respectively. The most frequent efmoroctocog alfa regimen was twice weekly (45.7%), followed by every 4 days (20.6%), every 3 days (9.1%), and 3 times per week (7.5%). The most frequent rurioctocog alfa pegol regimen was twice weekly (72.4%), followed by 3 times per week (8.7%), every 4 days (7.6%), and every 3 days (5.5%). The proportion of efmoroctocog alfa twice-weekly dispensing records increased from 31.5% to 50.9%, and every 4 days dispensing records decreased from 31.3% to 14.5% (2015–2018). The proportion of rurioctocog alfa pegol dispensing records remained broadly stable (2016–2018). Overall, mean (SD; median) weekly prophylactic dose was 105.4 (77.9; 92.6) IU/kg with efmoroctocog alfa, and 96.8 (41.9; 90.9) IU/kg with rurioctocog alfa pegol.

Conclusion

In this database study, the most frequently observed dosing frequency was twice weekly for patients receiving efmoroctocog alfa or rurioctocog alfa pegol. The observed mean weekly consumption was slightly higher, and variation was greater, in patients receiving efmoroctocog alfa versus rurioctocog alfa pegol.

Keywords:

• hemophilia A
• fragment crystallizable fusion protein
• efmoroctocog alfa
• antihemophilic factor (recombinant)
• PEGylated
• rurioctocog alfa pegol
• dosing patterns

Abbreviations

EHL, extended half-life; FVIII, factor VIII; HIPAA, Health Insurance Portability and Accountability Act; ICD, International Classification of Diseases; IQR, interquartile range; PEG, polyethylene glycol; rFVIII, recombinant factor VIII; SHL, standard half-life.

Data Sharing Statement

The data that support the findings of this study are available from Specialty Pharmacy Database records from individual states, but restrictions apply to the availability of these data due to applicable privacy laws and data use agreements. The data were used pursuant to data use agreements between Shire US Inc., a Takeda company, and the individual states.

Ethics Approval and Informed Consent

No institutional review board approval was required for this retrospective database analysis because only de-identified data were used. All data analyzed in the present study complied with the requirements of the Health Insurance Portability and Accountability Act (HIPAA) of 1996 for fully de-identified datasets.

Acknowledgments

Medical writing and editorial support were provided by Lisa O’Brien, PharmD, and Roz Bonomally, MSc, employees of Excel Medical Affairs (Fairfield, CT, USA) and funded by Takeda Development Center Americas, Inc., Lexington, MA, USA.

Author Contributions

All authors made a significant contribution to the conception, study design, execution, acquisition of data, analysis and/or interpretation of the manuscript. In addition, all authors took part in drafting, revising, and critically reviewing the article; gave final approval of the version to be published; have agreed on the journal to which the article has been submitted; and agree to be accountable for all aspects of the work.

Disclosure

Y. Wu and S.X. Sun are employees of Takeda Development Center Americas, Inc., Cambridge, MA, USA, and Takeda stock owners. T. Fan is an employee of Takeda Pharmaceuticals USA, Inc., Lexington, MA, USA, and Takeda stock owner. The authors report no other conflicts of interest in this work.

Additional information

Funding

This study was funded by Takeda Pharmaceutical Company Limited, Cambridge, MA, USA. The study sponsor was involved with the study design, analysis and interpretation of data, writing of the manuscript, and decision to publish the article.