Table 1.

Key model inputs

ParameterValueRange considered in sensitivity analysisOriginal model inputs References
Percentage of patients with severe phenotype 35% 20%-60% 25% (10%-40%) 8,9  
Baseline annual expenditure for patient with SCD  $51 700 $21 830-$85 680 $48 900 ($19 900-$78 100) 13,14  
Cost of single gene therapy treatment per person (million) $2.29 $1.78-$3.19 $2.267 ($1.769-$2.659) 7,11,12  
Annual market diffusion rate for gene therapy 7% 2%-15% 7% (2%-15%) Assumed 
ParameterValueRange considered in sensitivity analysisOriginal model inputs References
Percentage of patients with severe phenotype 35% 20%-60% 25% (10%-40%) 8,9  
Baseline annual expenditure for patient with SCD  $51 700 $21 830-$85 680 $48 900 ($19 900-$78 100) 13,14  
Cost of single gene therapy treatment per person (million) $2.29 $1.78-$3.19 $2.267 ($1.769-$2.659) 7,11,12  
Annual market diffusion rate for gene therapy 7% 2%-15% 7% (2%-15%) Assumed 

Costs adjusted from 2019 to 2024 US dollars.

Average of estimates in associated reference and adjusted to 2024 US dollars.

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