A Cost-Effectiveness Analysis of Newborn Screening for Severe Combined Immunodeficiency in the UK
Abstract
1. Introduction
2. Materials and Methods
3. Results
4. Discussion
5. Conclusions
Supplementary Materials
Author Contributions
Funding
Acknowledgments
Conflicts of Interest
References
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Parameter | Mean (95% Confidence Interval) | Reference |
---|---|---|
Number of births (UK) | 780,835 | [17,18,19] |
Incidence of SCID | 1:49,000 (1:39,857, 1:61,527) | [20] |
Incidence of undiagnosed SCID | 1:521,000 (1:167,052, 1:7,236,800) | [20] |
Incidence of syndromes | 1:45,000 (1:24,390, 1:110,606) | [21] |
Incidence of secondary conditions | 1:130,000 (1:50,686, 1:782,506) | [21] |
Incidence of idiopathic TCL | 1:99,000 (1:42,255, 1:432,482) | [21] |
Incidence of positive TREC in pre-terms | 1:99,000 (1:42,255, 1:432,482) | [21] |
Presumptive positives (20 copies/µL) | 0.041% (0.0035%, 0.1018%) | [22] |
Sensitivity for SCID | 0.99 (0.985, 0.998) | [23] |
Proportion of SCID patients with a family history | 0.30 (0.21, 0.41) | [20] |
Proportion of SCID that is ADA-SCID | 0.17 (0.1, 0.26) | [20] |
Proportion of SCID patients with a matched family donor available | 0.25 (0.07, 0.5) | [20] |
Pre HSCT mortality (late diagnosed) | 35.3% (22.8%, 49.3%) | [6] |
Pre HSCT mortality (early diagnosed) | 1.68% (0.11%, 7.63%) | [6] |
HSCT mortality (late diagnosed) | 38.7% (22.4% 56.3%) | [6] |
HSCT mortality (early diagnosed) | 8.48% (1.79%, 23.4%) | [6] |
Number of days HSCT | 54.0 | [24] |
Early diagnosis—Total days non-critical care | 82.6 (50.3, 122.8) | [24] |
Early diagnosis—Total days critical care | 3.96 (0.15, 8.41) | [24] |
Late diagnosis—Total days non-critical care | 144 (108.6, 184.3) | [24] |
Late diagnosis—Total days critical care | 8.19 (3.72, 14.4) | [24] |
QALYs—early diagnosis (1979–2015 cohort, base case values) | 0.95 | [25] |
QALYs—late diagnosis (1979–2015 cohort, base case values) | 0.82 | [25] |
QALYs—early diagnosis (2000–2015 cohort) | 0.96 | [25] |
QALYs—late diagnosis (2000–2015 cohort) | 0.87 | [25] |
Cost HSCT (early diagnosed) | £128,363 | [24,26] |
Cost HSCT (late diagnosed) | £231,186 | [24,26] |
Cost death prior to HSCT | £43,368 | [24,26] |
Presumptive positive cost | £276 | [24,26] |
Diagnosis SCID | £711 | [26,27] |
Diagnosis idiopathic SCID and syndromes | £1,551 | [26,27] |
Idiopathic SCID follow up (5 years discounted) | £20,142 | [26,28,29,30] |
Syndromes 4 year follow up | £4,872 | [24,26] |
Follow up preterm & secondary to other conditions | £533 | [24,26] |
Screening | 95% CI | No Screening | 95% CI | Incremental | 95% CI | ||
---|---|---|---|---|---|---|---|
Outcomes | SCID cases diagnosed symptomatically | 0.2 | (0.1, 0.3) | 11.0 | (8.4, 14.1) | −10.9 | (−13.9, −8.2) |
SCID cases diagnosed via a family history | 0 | (0, 0) | 4.9 | (3.1, 7.0) | −4.9 | (−3.1, −7.0) | |
SCID cases not diagnosed | 0 | (0, 0) | 1.5 | (0.1, 4.8) | −1.5 | (−0.1, −4.8) | |
SCID cases screen detected | 17.2 | (13.5, 21.7) | 0 | (0, 0) | 17.2 | (13.5, 21.7) | |
ADA SCID | 2.9 | (1.6, 4.7) | 2.9 | (1.6, 4.7) | 0 | (0.0, 0.0) | |
SCID mortality | 1.7 | (0.6, 4.0) | 8.1 | (5.3, 12.0) | −6.3 | (−9.7, −4) | |
Screening outcomes | Non SCID TCL | 31.1 | (16.3, 50.1) | 0 | (0, 0) | 31 | (16.3, 50.1) |
Pre-term | 8.0 | (1.9, 18.7) | 0 | (0, 0) | 8 | (1.9, 18.7) | |
Total presumptive positives | 322.1 | (79.5, 852.9) | 0 | (0, 0) | 0 | (79.5, 852.9) | |
Costs | Direct screening costs | £3.04m | (£2.97m, £3.19m) | £0.00m | (£0.00m, £0.00m) | £3.04m | (£2.97m, £3.19m) |
Diagnosis and follow up pre-terms | £4,394 | (£1,029.36, £10,311) | £0 | (£0.00m, £0.00m) | £4,394 | (£1,029.36, £10,311) | |
Diagnosis and follow up non SCID TCL | £0.27m | (£0.12m, £0.51m) | £0.00m | (£0.00m, £0.00m) | £0.27m | (£0.12m, £0.51m) | |
Diagnosis costs SCID | £14,235 | (£11,112, £17,967) | £13,023 | (£13,023, £15,954) | £1,212 | (£61.7, £3,919) | |
SCID treatment up and including HSCT | £3.35m | (£2.20m, £4.83m) | £3.63m | (£3.63m, £2.65m) | −£0.28m | (−£1.10m, £0.62m) | |
SCID long-term costs | £2.03m | (£1.20m, £3.12m) | £1.30m | (£1.30m, £0.78m) | £0.72m | (£0.23m, £1.37m) | |
Totals | Total costs | £7.30m | (£5.96m, £9.06m) | £3.96m | (£3.96m, £2.92m) | £3.34m | (£2.36m, £4.47m) |
Total QALYs | 410.1 | (308.3, 527.3) | 226.9 | (226.9, 164.9) | 183.17 | (109.3, 267.2) | |
ICER | £18,222 | (£12,013, £27,763) |
Sensitivity Analysis | Screening | No Screening | Incremental | Probability Cost-Effective at Threshold | ||||||
---|---|---|---|---|---|---|---|---|---|---|
Costs | QALYs | Costs | QALYs | Costs | QALYs | ICER | £20k | £30k | ||
Base case | £7.30m | 410 | £3.96m | 227 | £3.34m | 183 | £18,222 | 65% | 99% | |
Incidence | Halved | £5.49m | 223 | £2.01m | 113 | £3.48m | 110 | £31,647 | 2% | 35% |
Doubled | £10.97m | 785 | £7.89m | 453 | £3.07m | 332 | £9,266 | 100% | 100% | |
TREC cut off | Additional false positives | £7.53m | 412 | £3.99m | 227 | £3.54m | 185 | £19,137 | 56% | 98% |
Increase proportionately | £7.44m | 409 | £3.98m | 227 | £3.46m | 182 | £18,983 | 56% | 98% | |
Family History | 10% family history | £7.32m | 411 | £4.06m | 183 | £3.26m | 229 | £14,237 | 95% | 100% |
50% family history | £7.30m | 410 | £3.89m | 269 | £3.42m | 141 | £24,208 | 14% | 84% | |
Test cost | £1.50 | £5.75m | 410 | £3.98m | 227 | £1.77m | 183 | £9,674 | 100% | 100% |
£2.50 | £6.55m | 413 | £3.99m | 228 | £2.56m | 184 | £13,876 | 95% | 100% | |
£4.50 | £8.10m | 410 | £3.98m | 227 | £4.11m | 183 | £22,471 | 25% | 92% | |
Discount rate | 1.5% both costs and health benefits | £8.82m | 702 | £5.02m | 391 | £3.80m | 311 | £12,219 | 99% | 100% |
1.5% health benefits & 3.5% costs | £7.32m | 703 | £4.00m | 392 | £3.32m | 311 | £10,680 | 100% | 100% | |
QALYs | 2000–2015 cohort QALYs used | £7.32m | 415 | £3.98m | 235 | £3.34m | 180 | £18,588 | 61% | 98% |
Pre-transplant mortality | Early diagnosed mortality (OR) | |||||||||
8.15%(0.23) | £7.12m | 381 | £3.67m | 206 | £3.45m | 175 | £19,691 | 51% | 97% | |
29.40%(0.83) | £6.44m | 294 | £3.48m | 182 | £2.95m | 113 | £26,237 | 12% | 68% | |
Post-transplant mortality | Early diagnosed mortality (OR) | |||||||||
17.42%(0.45) | £7.16m | 369 | £3.68m | 203 | £3.48m | 166 | £20,915 | 39% | 96% | |
36.77%(0.95) | £6.79m | 283 | £3.58m | 178 | £3.21m | 105 | £30,746 | 3% | 43% |
Healthy at Birth Disbenefit (QALY) | Cost per TREC Test £3.50 | |
---|---|---|
False-Positive Disbenefit Threshold (Quality-Adjusted Days) | ||
Discounting 3.5% | Discounting 1.5% | |
Mean (95% CI) | Mean (95% CI) | |
0 | 17 (0, 22) | 171 (1,113, 91) |
1 | 6 (0, 14) | 159 (1,069,83) |
2 | 0 (0, 6) | 148 (1,025, 75) |
3 | 0 (0, 0) | 136 (980, 67) |
4 | 0 (0, 0) | 125 (936, 59) |
10 | 0 (0, 0) | 55 (672, 11) |
20 | 0 (0, 0) | 0 (230, 0) |
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Bessey, A.; Chilcott, J.; Leaviss, J.; de la Cruz, C.; Wong, R. A Cost-Effectiveness Analysis of Newborn Screening for Severe Combined Immunodeficiency in the UK. Int. J. Neonatal Screen. 2019, 5, 28. https://doi.org/10.3390/ijns5030028
Bessey A, Chilcott J, Leaviss J, de la Cruz C, Wong R. A Cost-Effectiveness Analysis of Newborn Screening for Severe Combined Immunodeficiency in the UK. International Journal of Neonatal Screening. 2019; 5(3):28. https://doi.org/10.3390/ijns5030028
Chicago/Turabian StyleBessey, Alice, James Chilcott, Joanna Leaviss, Carmen de la Cruz, and Ruth Wong. 2019. "A Cost-Effectiveness Analysis of Newborn Screening for Severe Combined Immunodeficiency in the UK" International Journal of Neonatal Screening 5, no. 3: 28. https://doi.org/10.3390/ijns5030028
APA StyleBessey, A., Chilcott, J., Leaviss, J., de la Cruz, C., & Wong, R. (2019). A Cost-Effectiveness Analysis of Newborn Screening for Severe Combined Immunodeficiency in the UK. International Journal of Neonatal Screening, 5(3), 28. https://doi.org/10.3390/ijns5030028