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.
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.
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.
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No funding was received for this study.
Conflicts of Interest
Authors have no conflicts of interest to disclose.
This was a modelling study and did not require ethics approval.
Availability of data and material
All data inputs used to parameterize the model are available within the article and the Online Supplementary Materials. Description of the decision tree component of the model is provided in the article. The microsimulation component is adapted from the Sheffield Type 1 Diabetes Policy Model which is described in a previously published article: Thokala et al. “Assessing the cost-effectiveness of Type 1 diabetes interventions: the Sheffield Type 1 Diabetes Policy Model.” Diabetic Medicine 31.4 (2014): 477-486. The model was built based on these model descriptions and data inputs.
The model was built in TreeAge Pro software, which does not require user-specified codes.
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Consent for publication
SM conceptualized the idea. Both SM and HVN contributed to data analysis, interpretation of results and manuscript writing.
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Mital, S., Nguyen, H.V. Cost Effectiveness of Teplizumab for Prevention of Type 1 Diabetes Among Different Target Patient Groups. PharmacoEconomics (2020). https://doi.org/10.1007/s40273-020-00962-y