Cost-effectiveness of newborn screening for cystic fibrosis determined with real-life data
C P B van der Ploeg1, M E van den Akker-van Marle2, A M M Vernooij-van Langen3
1Department of Child Health, TNO, Leiden, The Netherlands.
Insights
Newborn screening for cystic fibrosis (NBSCF) is cost-effective. The immunoreactive trypsinogen-testing followed by pancreatitis-associated protein-testing (IRT-PAP) strategy offers the best economic value for public health initiatives.
Area of Science:
- Public Health
- Genetics
- Medical Economics
Background:
- Previous literature indicated newborn screening for cystic fibrosis (NBSCF) is economically viable, improving health outcomes and survival.
- NBSCF is recognized as a beneficial public health initiative.
Purpose of the Study:
- To compare the cost-effectiveness of four NBSCF screening strategies using primary data.
- To evaluate strategies including IRT-PAP, IRT-DNA, IRT-DNA-sequencing, and IRT-PAP-DNA-sequencing against no screening.
Main Methods:
- A decision analysis model was utilized for NBSCF cost-effectiveness evaluation.
- Primary data from a large-scale study in The Netherlands informed the model parameters.
Main Results:
- Screening strategies ranged from €23,600 to €29,200 per life-year gained.
- The IRT-PAP strategy demonstrated the most favorable cost-effectiveness ratio.
- IRT-DNA screening yielded additional life-years at a higher cost.
Conclusions:
- NBSCF is an economically justifiable public health measure.
- The IRT-PAP strategy is the most economical choice for NBSCF implementation.
- Findings support incorporating IRT-PAP into decision-making models for NBSCF programs.
Background:
Previous cost-effectiveness studies using data from the literature showed that newborn screening for cystic fibrosis (NBSCF) is a good economic option with positive health effects and longer survival.
Methods:
We used primary data to compare cost-effectiveness of four screening strategies for NBSCF, i.e. immunoreactive trypsinogen-testing followed by pancreatitis-associated protein-testing (IRT-PAP), IRT-DNA, IRT-DNA-sequencing, and IRT-PAP-DNA-sequencing, each compared to no-screening. A previously developed decision analysis model for NBSCF was fed with model parameters mainly based on a study evaluating two novel screening strategies among 145,499 newborns in The Netherlands.
Results:
The four screening strategies had cost-effectiveness ratios varying from €23,600 to €29,200 per life-year gained. IRT-PAP had the most favourable cost-effectiveness ratio. Additional life-years can be gained by IRT-DNA but against higher costs. When treatment costs reduce with 5% due to early diagnosis, screening will lead to financial savings.
Conclusion:
NBSCF is as an economically justifiable public health initiative. Of the four strategies tested IRT-PAP is the most economic and this finding should be included in any decision making model, when considering implementation of newborn screening for CF.
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