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Economic Analysis for Adding Newborn Screening for ALD
John D. Thompson, PhD MPH MPA
Washington State Department of Health
Washington Criteria for NBS
1. Early identification benefits the newborn
2. Treatment is available
3. Nature of the condition justifies population-based screening
4. A good screening test exists
5. The benefits justify the costs of screening
Strategy
• Decision Tree
– Compares status quo v. screening model
• Data from
– Primary literature (including pilot studies)
– Reports from NY NBS program
– Expert opinion
• Sensitivity analysis – vary assumptions
– High and low estimates for parameters
No Screening Decision Tree
# babies with ALD
Early ID –family history
Late ID –clinical symptoms
AI
CALD
AI
CALD
Early MRI
Early MRI
Late MRI
Adrenal crisis
Adrenal crisis
23%
77%
80%
35%
80%
35%
0.1%
1%
11%
100%
Mortality
11%25%
100%75%
NBS Decision Tree
# babies with ALD
Early ID –true positive NBS
Late ID –false negative NBS
AI
CALD
AI
CALD
Early MRI
Early MRI
Late MRI
Adrenal crisis
Adrenal crisis
99.5%
0.5%
80%
35%
80%
35%
0.1%
1%
11%
100%
Mortality
false positive NBS
0.002%
11%25%
100%75%
Parameters• Birthrate
• Prevalence of ALD
• % of cases with family history of ALD
• Morbidity rates (adrenal, CALD)
• Mortality rates (adrenal, CALD)
• Screening test performance
• Costs of newborn screening
• Costs of early v. late treatment
• Costs of serial testing
• Value of a life
Base-Case: Morbidity & Mortality
Base-Case: Shift
Base-Case: Shift
Benefit/Cost Ratio
Net Benefit
• Birthrate
• Prevalence of ALD
• % of cases with family history of ALD
• Morbidity rates (adrenal, CALD)
• Mortality rates (adrenal, CALD)
• Screening test performance
• Costs of newborn screening
• Costs of early v. late treatment
• Costs of serial testing
• Value of a life
1:11,000 – 1:17,000
30% to 90% (late)
$5 - $11 per baby
$7 - $11 million
Parameters Range
Model built in MS Excel
Formula driven – assumptions can be tailored by individual programs
Recent Models Available
X-ALD (2015) – b/c ratio = 5.83
MPS-I (2017) – b/c ratio = 0.88
Pompe disease (2017) – b/c ratio = -18.02
Acknowledgements
Economics
• Scott Grosse (CDC)
Neurology
• Gerald Raymond (U of Minnesota)
• Jennifer Kwon (U of Rochester)
Newborn Screening
• Joe Orsini (NY NBS)
• Beth Vogel (NY NBS)
Parent Advocates
• Brad and Nancy Zakes
Lesson Learned
2017 update – significant analysis
• Prompted further conversations with neurologists
• Current clinical practice differs from published literature
– Clinically identified cases with Loes scores > 10 no longer transplanted
• b/c ratio improved from 3.95 to 5.83
Washington State Newborn Screening
www.doh.wa.gov/nbs
(206) 418-5410
or
1-866-660-9050
