Cost Effectiveness of Teplizumab for Prevention of Type 1 Diabetes Among Different Target Patient Groups
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ORIGINAL RESEARCH ARTICLE
Cost Effectiveness of Teplizumab for Prevention of Type 1 Diabetes Among Different Target Patient Groups Shweta Mital1 · Hai V. Nguyen1 Accepted: 31 August 2020 © Springer Nature Switzerland AG 2020
Abstract Objective Teplizumab was recently shown to be the first-ever drug to prevent or delay type 1 diabetes mellitus onset in at-risk individuals, especially those with certain genetic and antibody characteristics. However, its potentially high price may pose challenges for coverage and reimbursement for payers and policymakers. Thus, it is critical to investigate the cost effectiveness of this drug for different target individuals. Research Design and Methods Using Markov microsimulation modeling, we compared the cost effectiveness of five options for choosing target individuals (i.e., all at-risk individuals, individuals without human leukocyte antigen (HLA)-DR3 or with HLA-DR4 allele, individuals without HLA-DR3 and with HLA-DR4 allele, individuals with anti-zinc transporter 8 (ZnT8) antibody negative, and no provision at all) at different possible prices of teplizumab. Effectiveness was measured by quality-adjusted life-years. Costs were estimated from a health system perspective. Results If the price of teplizumab is below US$48,900, treating all at-risk individuals is cost effective. However, it will be cost effective to treat only individuals without HLA-DR3 or with HLA-DR4 alleles for prices between US$48,900 and US$58,200, only individuals both without HLA-DR3 and with HLA-DR4 alleles for prices between US$58,200 and US$88,300, and only individuals with negative ZnT8 antibody status for prices between US$88,300 and US$193,700. Conclusions Cost-effective provision of teplizumab to target individuals depends on the price of teplizumab and genetic and antibody characteristics of treated individuals. As the drug makes its way to the market, findings from this study will help inform policymakers and payers on cost-effective ways to provide this innovative but expensive drug to at-risk individuals.
1 Introduction Type 1 diabetes mellitus is an auto-immune disorder that occurs due to the destruction of insulin-producing beta cells in the islet of Langerhans region of the pancreas [1]. The disease affects nearly 1.25 million children and adults in the United States (US) [2]. Furthermore, first-degree relatives of type 1 diabetes patients have a 15-fold increased risk of developing type 1 diabetes themselves [3]. Treatment for type 1 diabetes involves lifelong dependence on external insulin, which not only causes the inconvenience of daily insulin injections, but also imposes severe economic burden Electronic supplementary material The online version of this article (https://doi.org/10.1007/s40273-020-00962-y) contains supplementary material, which is available to authorized users. * Hai V. Nguyen [email protected] 1
School of Pharmacy, Memorial University of Newfoundland, 300 Prince Philip Drive, St. John’s, NL A1B 3V6, Canada
on patients and health care systems. In the US, an
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