Cost-effectiveness analysis of genetic tools to predict treatment response in patients with cystic fibrosis

Yeva Sahakyan; Lusine Abrahamyan; Felix Ratjen; Christine Bear; Lisa Strug; Paul D W Eckford; John K Peel; Murray Krahn; Beate Sander

doi:10.1016/j.jcf.2023.04.013

Cost-effectiveness analysis of genetic tools to predict treatment response in patients with cystic fibrosis

J Cyst Fibros. 2023 Sep;22(5):933-940. doi: 10.1016/j.jcf.2023.04.013. Epub 2023 Apr 24.

Authors

Yeva Sahakyan¹, Lusine Abrahamyan², Felix Ratjen³, Christine Bear⁴, Lisa Strug⁵, Paul D W Eckford⁶, John K Peel⁷, Murray Krahn², Beate Sander⁸

Affiliations

¹ Toronto Health Economics and Technology Assessment Collaborative (THETA), University Health Network, Toronto, ON, Canada. Electronic address: yeva.sahakyan@theta.utoronto.ca.
² Toronto Health Economics and Technology Assessment Collaborative (THETA), University Health Network, Toronto, ON, Canada; Toronto General Hospital Research Institute, University Health Network, Toronto, ON, Canada; Institute of Health Policy, Management, and Evaluation, University of Toronto, Toronto, ON, Canada.
³ Division of Respiratory Medicine, Department of Pediatrics, Translational Medicine, Research Institute, Hospital for Sick Children, Toronto, ON, Canada; Department of Pediatrics, University of Toronto, Toronto, ON, Canada.
⁴ Molecular Medicine Programme, Hospital for Sick Children, Toronto, ON, Canada; Department of Physiology, University of Toronto, Toronto, ON, Canada.
⁵ Program in Genetics and Genome Biology, Hospital for Sick Children, Toronto, ON, Canada; Departments of Statistical Sciences and Computer Science, University of Toronto, Toronto, ON, Canada.
⁶ Molecular Medicine Programme, Hospital for Sick Children, Toronto, ON, Canada.
⁷ Institute of Health Policy, Management, and Evaluation, University of Toronto, Toronto, ON, Canada.
⁸ Toronto Health Economics and Technology Assessment Collaborative (THETA), University Health Network, Toronto, ON, Canada; Toronto General Hospital Research Institute, University Health Network, Toronto, ON, Canada; Institute of Health Policy, Management, and Evaluation, University of Toronto, Toronto, ON, Canada; Public Health Ontario, Toronto, ON, Canada; ICES, Toronto, ON, Canada.

PMID: 37100704
DOI: 10.1016/j.jcf.2023.04.013

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) Test→Treat, 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 Test→Treat 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 Test→Treat was highly cost-effective compared to Treat All in 100% of simulations at cost-effectiveness thresholds as high as $500,000 per QALY. Test→Treat 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.

Keywords: Cost-effectiveness; Cost-utility; Cystic fibrosis; Modulators; Personalized therapy; Predictive tools.

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

Canada
Cost-Benefit Analysis
Cost-Effectiveness Analysis
Cystic Fibrosis Transmembrane Conductance Regulator / genetics
Cystic Fibrosis* / diagnosis
Cystic Fibrosis* / genetics
Cystic Fibrosis* / therapy
Humans

Substances

Cystic Fibrosis Transmembrane Conductance Regulator