PMID- 32960433 OWN - NLM STAT- MEDLINE DCOM- 20210917 LR - 20210917 IS - 1179-2027 (Electronic) IS - 1170-7690 (Linking) VI - 38 IP - 12 DP - 2020 Dec TI - Cost Effectiveness of Teplizumab for Prevention of Type 1 Diabetes Among Different Target Patient Groups. PG - 1359-1372 LID - 10.1007/s40273-020-00962-y [doi] AB - 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. FAU - Mital, Shweta AU - Mital S AD - School of Pharmacy, Memorial University of Newfoundland, 300 Prince Philip Drive, St. John's, NL, A1B 3V6, Canada. FAU - Nguyen, Hai V AU - Nguyen HV AD - School of Pharmacy, Memorial University of Newfoundland, 300 Prince Philip Drive, St. John's, NL, A1B 3V6, Canada. hvnguyen@mun.ca. LA - eng PT - Journal Article DEP - 20200922 PL - New Zealand TA - Pharmacoeconomics JT - PharmacoEconomics JID - 9212404 RN - 0 (Antibodies, Monoclonal, Humanized) RN - 0 (HLA-DR3 Antigen) RN - S4M959U2IJ (teplizumab) MH - Alleles MH - Antibodies, Monoclonal, Humanized MH - Cost-Benefit Analysis MH - *Diabetes Mellitus, Type 1/drug therapy/genetics MH - HLA-DR3 Antigen/genetics MH - Humans EDAT- 2020/09/23 06:00 MHDA- 2021/09/18 06:00 CRDT- 2020/09/22 12:13 PHST- 2020/08/31 00:00 [accepted] PHST- 2020/09/23 06:00 [pubmed] PHST- 2021/09/18 06:00 [medline] PHST- 2020/09/22 12:13 [entrez] AID - 10.1007/s40273-020-00962-y [pii] AID - 10.1007/s40273-020-00962-y [doi] PST - ppublish SO - Pharmacoeconomics. 2020 Dec;38(12):1359-1372. doi: 10.1007/s40273-020-00962-y. Epub 2020 Sep 22.