https://orcid.org/0000-0002-8479-2775
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Abstract
Aim
Gaucher disease (GD) is a rare autosomal recessive condition. Type 1 GD (GD1) is the most prevalent form of GD in Western countries; enzyme replacement therapy (ERT) is a treatment option for patients with GD1. To understand the economic value of the GD1 ERT velaglucerase alfa, a budget impact model (BIM) was developed from a United States (US) payer perspective.
Methods
We estimated the budget impact of velaglucerase alfa for a 10-million-member US health plan by comparing the annual total costs of therapy between a scenario using current velaglucerase alfa uptake to a projected scenario with increased velaglucerase alfa uptake. Total drug costs for both scenarios were estimated as the sum of the product of the number of eligible patients on each treatment and the annual per-patient cost of each medication. Average per-patient costs for ERTs were calculated by adding the yearly drug acquisition, drug administration, and site-of-care markup costs. The budget impact was measured over years 1–3.
Results
An estimated 65 patients would receive velaglucerase alfa treatment in year 1, increasing to 90 patients by year 3. Across analyses, cost savings were realized with velaglucerase alfa compared with imiglucerase ($115,909) and taliglucerase alfa ($80,401). An annual total budget savings of $8.67 million could be realized for a hypothetical 10-million-member US health plan with increased velaglucerase alfa uptake. The per-member per-month costs decreased by $0.0241 across years 1–3.
Conclusions
BIM results show that increased velaglucerase alfa uptake for GD1 treatment is cost-saving for US health plans.
PLAIN LANGUAGE SUMMARY
Type 1 Gaucher disease (GD1) is a rare inherited condition. Long-term enzyme replacement therapy (ERT) can reverse and prevent complications. Imiglucerase, taliglucerase alfa, and velaglucerase alfa are 3 ERTs used to treat GD1. In this study, we estimated how increasing uptake of velaglucerase alfa vs. the other ERTs would impact the budget of a hypothetical US healthcare plan. The results show that increased uptake of velaglucerase alfa is cost-saving for US health plans.
Transparency
Declaration of funding
Takeda provided funding for this study.
Declaration of financial/other relationships
SF is a full-time employee of Takeda and a stockholder of Takeda Pharmaceuticals Company Limited. Takeda provided medical writing support in relation to this work.
TJI is a former full-time employee of OPEN Health. OPEN Health received funding from Takeda for medical writing support in relation to this work.
GP is a former full-time employee of OPEN Health. OPEN Health received funding from Takeda for medical writing support in relation to this work.
EW is a full-time employee of Takeda and a stockholder of Takeda Pharmaceuticals Company Limited. Takeda provided medical writing support in relation to this work.
RRP is a full-time employee of Takeda and a stockholder of Takeda Pharmaceuticals Company Limited. Takeda provided medical writing support in relation to this work.
MB is a full-time employee of Takeda and a stockholder of Takeda Pharmaceuticals Company Limited. Takeda provided medical writing support in relation to this work.
A reviewer on this manuscript has disclosed that they have acted as an adviser and speaker for all three companies but have no formal or ongoing affiliations. Peer reviewers on this manuscript have no other relevant financial relationships or otherwise to disclose.
Author contributions
SF, TJI, and MB: concept and design. SF, TJI, GP, EW, RRP, and MB: analysis and interpretation of the data, drafting of the paper or revising it critically for intellectual content, and final approval of the version to be published. All authors agree to be accountable for all aspects of this work.
Acknowledgements
Medical writing support was provided by Beth Lesher, PharmD, BCPS, from OPEN Health, Bethesda, MD, and funded by the study sponsor.