Cost-effectiveness analysis of genetic tools to predict treatment response in patients with cystic fibrosis.
J Cyst Fibros
; 22(5): 933-940, 2023 Sep.
Article
en En
| MEDLINE
| ID: mdl-37100704
ABSTRACT
BACKGROUND:
Cystic fibrosis (CF) transmembrane conductance regulator (CFTR) modulator therapies show variable efficacy for patients with CF. Patient-derived predictive tools may identify individuals likely to respond to CFTRs, but are not in routine use. We aimed to determine the cost-utility of predictive tool-guided treatment with CFTRs as add-on to standard of care (SoC) for individuals with CF.METHODS:
This economic evaluation compared two strategies using an individual level simulation (i) Treat All, where all patients received CFTRs plus SoC and (ii) TestâTreat, where patients who tested positive on predictive tools received CFTRs plus SoC and those who tested negative received SoC only. We simulated 50,000 individuals over their lifetime, and estimated costs (2020 CAD) per quality-adjusted life year (QALY) from the healthcare payer's perspective, discounted at 1.5% annually. The model was populated using Canadian CF registry data and published literature. Probabilistic and deterministic sensitivity were conducted.RESULTS:
The Treat All and TestâTreat and strategies yielded 22.41 and 21.36 QALYs, and cost $4.21 M and $3.15 M respectively. Results of probabilistic sensitivity analysis showed that TestâTreat was highly cost-effective compared to Treat All in 100% of simulations at cost-effectiveness thresholds as high as $500,000 per QALY. TestâTreat may save between $931 K to $1.1 M per QALY lost, depending on sensitivity and specificity of predictive tools.CONCLUSION:
The use of predictive tools could optimize the health benefits of CFTR modulators while reducing costs. Our findings support the use of pre-treatment predictive testing and may help inform coverage and reimbursement policies for individuals with CF.Palabras clave
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Contexto en salud:
1_ASSA2030
Problema de salud:
1_financiamento_saude
Asunto principal:
Fibrosis Quística
Tipo de estudio:
Health_economic_evaluation
/
Prognostic_studies
/
Risk_factors_studies
Aspecto:
Patient_preference
Límite:
Humans
País/Región como asunto:
America do norte
Idioma:
En
Revista:
J Cyst Fibros
Año:
2023
Tipo del documento:
Article