Healthcare resource use and direct costs in severe aplastic anemia (SAA) patients before and after treatment with eltrombopag

Abstract

Purpose: This study evaluated healthcare resource utilization (HCRU), and direct costs among severe aplastic anemia (SAA) patients treated with eltrombopag (EPAG) using US claims data.

Methods: This retrospective, real-world claims database study identified SAA patients aged ≥2 years treated with EPAG who initiated any SAA treatment between 1 July 2014 and 31 December 2017 (identification period) using the Truven MarketScan databases. A subset of 82 patients treated with EPAG during the identification period were evaluated for all-cause and SAA-related HCRU and direct costs as well as blood transfusion 1 month before EPAG initiation (baseline) and at Month 6 after EPAG initiation (follow-up period).

Results: The average patient age was 50.8 (SD = 20.6) years old, predominantly female (n = 43, 52.4%), and had a mean CCI at baseline of 1.1 (SD = 1.7). Hospitalizations, and ER, office, and outpatient visits were significantly lower at Month 6 after EPAG initiation compared with 1 month before EPAG initiation (p < .05 for all four all-cause HCRU and SAA-related hospitalizations). An almost two-fold decrease in reliance on biweekly blood transfusions was observed: 1.0 at weeks 1–2 to 0.5 at Month 6 after EPAG initiation. Although prescription costs (mean [SD]) were significantly higher at Month 6 after EPAG initiation compared with 1 month before EPAG initiation (difference of $11,045 USD [SD = $18,801]), these increases were offset by savings in direct costs. Overall, a mean reduction in total all-cause costs of $29,391 USD [SD = $137,770] was reported at Month 6 after EPAG initiation due to substantial reductions in hospitalization ($40,060 USD [SD = $123,198]) and outpatient visits ($2,043 USD [SD = $25,264]).

Conclusion: All-cause and SAA-related HCRU were reduced following EPAG treatment. Prescription costs were higher following treatment; however, these costs were generally offset by reductions in direct costs. These results provide real-world evidence around the role of EPAG in SAA treatment.

Keywords:

• Severe aplastic anemia
• eltrombopag
• blood transfusion
• healthcare resource utilization
• direct costs
• immunosuppressive therapy

JEL CLASSIFICATION CODES:

• I10
• I19

This article is related to:

Healthcare costs and resource utilization in patients with severe aplastic anemia in the US

Transparency

Declaration of funding

Funding for this study was provided by Novartis Pharmaceuticals Corporation.

Declaration of financial/other relationships

BC was a full-time employee of Novartis Pharmaceuticals Corporation when this study was conducted. QS was a full-time employee of Novartis Pharmaceuticals Corporation when this study was conducted. XL was an employee of KMK Consulting Service and worked at Novartis Pharmaceuticals Corporation as a contracted consultant when this study was conducted. FYL was a full-time employee of Novartis Pharmaceuticals Corporation when this study was conducted. SA was a full-time employee of Novartis Pharmaceuticals Corporation when this study was conducted.

A peer reviewer on this manuscript has disclosed that they receive support from, and consult for, Novartis. A peer reviewer on this manuscript has disclosed that they have been a consultant for Novartis, Amgen, Dova, Sysmex, Octapharma, and Shionogi all of whom have products in development or FDA approved for ITP. They have also served on advisory boards for Novartis, Amgen and Dova and have received research funding from Novartis, Amgen, Rigel, and Sysmex. A peer reviewer on this manuscript has disclosed that they have been a speaker for, and acted in an advisory capacity, for Novartis. This manuscript has been additionally reviewed by an Editorial Board Member for an impartiality verification. The Board Member has disclosed that they hold equity in Matrix45, which has provided consultancy services for Novartis and some of its subsidiaries. By Matrix45 company policy, equity holders cannot hold equity in sponsor/client organizations, nor provide services independently to or receive compensation independently from sponsor/client organizations. The peer reviewers on this manuscript have no other relevant financial relationships or otherwise to disclose.

Acknowledgements

We would like to thank Write All, Inc for the writing and editorial assistance.

Author contributions

BC, QS, SA, XL, and FL were involved in the conception and design, or analysis and interpretation of the data; the drafting of the paper or revising it critically for intellectual content; and the final approval of the version to be published. All authors agree to be accountable for all aspects of the work.