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. 2021 Mar 22;175(6):1–7. doi: 10.1001/jamapediatrics.2020.7140

Table 1. Key Model Inputs.

Parameter Value Range considered in sensitivity analysis Source
Patients with SCD with severe phenotype, % 25 10-40 Assumed
Baseline annual expenditure for patient with SCD, $ 42 200a 17 100-67 250 Kauf et al,13 Arnold et al17
Cost of single gene therapy treatment per person, $ 1 850 000 1 440 000-2 170 000 Beasley and Mathias18
Annual market diffusion rate for gene therapy, % 7 2-15 Assumed

Abbreviation: SCD, sickle cell disease.

a

Mean of estimates in associated reference.