The Economic Impact and Health-Related Quality of Life of Spinal Muscular Atrophy. An Analysis across Europe
Abstract
:1. Introduction
2. Methods
2.1. Study Population
2.2. Health Outcomes for Patients and Caregivers
2.3. Treatment of Costs
3. Results
4. Discussion
5. Conclusions
Author Contributions
Funding
Conflicts of Interest
Availability of Data and Materials
Appendix A
Category Cost | UK | France | Germany | |||
---|---|---|---|---|---|---|
N=34 | N=27 | N= 25 | ||||
Mean in PPP | % | Mean in PPP | % | Mean in PPP | % | |
Drugs | 530.94 | 1.03 | 12.87 | 0.04 | 34.76 | 0.07 |
Medical tests | 828.65 | 1.61 | 353.10 | 1.20 | 156.93 | 0.30 |
Medical visits | 4.33 | 0.01 | 1,719.54 | 5.84 | 1940.75 | 3.76 |
Hospitalizations | 2.10 | 0.00 | 1,130.11 | 3.84 | 3148.51 | 6.10 |
GP & Emergency | 798.31 | 1.55 | 132.41 | 0.45 | 612.82 | 1.19 |
Medical material | 1.86 | 0.00 | 910.34 | 3.09 | 1369.65 | 2.65 |
Healthcare transport | 54.99 | 0.11 | 37.70 | 0.13 | 0.00 | 0.00 |
Direct healthcare costs | 10,506.01 | 20.41 | 4296.09 | 14.58 | 7263.43 | 14.07 |
Social services | 2073.52 | 4.03 | 946.21 | 3.21 | 4350.31 | 8.43 |
Direct non-healthcare formal costs | 2073.52 | 4.03 | 946.21 | 3.21 | 4350.31 | 8.43 |
Main informal carer | 25,610.35 | 49.75 | 16,062.53 | 54.52 | 27,250.02 | 52.78 |
Other informal carers | 12,814.66 | 24.89 | 7,495.17 | 25.44 | 12,405.33 | 24.03 |
Direct non-healthcare informal costs | 38,423.11 | 74.64 | 23,557.70 | 79.95 | 39,655.36 | 76.81 |
Non-healthcare transport a | 475.00 | 0.92 | 663.91 | 2.25 | 361.53 | 0.70 |
Direct non-healthcare costs | 40,971.63 | 79.59 | 25,167.82 | 85.42 | 44,367.20 | 85.93 |
TOTAL COST | 51,477.64 | 100.00 | 29,463.91 | 100.00 | 51,630.62 | 100.00 |
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Characteristics | UK | France | Germany |
---|---|---|---|
N (%) | N (%) | N (%) | |
Patients | n = 34 | n = 27 | n = 25 |
Type I | 7 (20.59) | 5 (18.52) | 11 (44.00) |
Type II | 20 (58.82) | 13 (48.15) | 12 (48.00) |
Type III | 7 (20.59) | 9 (33.33) | 2 (8.00) |
Gender (female) | 17 (50.00) | 16 (59.26) | 18 (72.00) |
Age (mean, SD) | 5.55 (4.79) | 6.19 (6.13) | 9.52 (6.19) |
Education | |||
Educated at an ordinary school | 10 (29.41) | 5 (18.52) | 12 (48.00) |
Educated at an ordinary centre with special sessions | 6 (17.65) | 8 (29.63) | 4 (16.00) |
Educated at a special education centre | 2 (5.88) | 1 (3.70) | 4 (16.00) |
Home-schooled | 1 (2.94) | 1 (3.70) | 0 |
Nursery school | 5 (14.71) | 5 (18.52) | 5 (20.00) |
No education | 7 (20.59) | 4 (14.81) | 0 |
NA | 3 (8.82) | 3 (11.11) | 0 |
Barthel index (mean, SD) | 35.93 (28.29) | 34.33 (30.11) | 47.66 (22.90) |
Caregivers | n = 11 | n = 16 | n = 14 |
Gender (female) | 7 (63.64) | 15 (93.75) | 11 (78.57) |
Age (mean, SD) | 41.09 (11.56) | 36.13 (9.15) | 42.53 (10.57) |
Caregiving time (daily hours) (mean, SD) a | 12.50 (5.96) | 9.31 (8.44) | 10.65 (5.45) |
Zarit caregiver’s burden, (mean, SD) | 26.63 (13.39) | 40.37 (16.10) | 21.33 (18.33) |
Risk of burnout (Zarit scale) (n, %) b | 0 | 1 (12.50) | 0 |
UK | France | Germany | |
---|---|---|---|
n = 34 | n = 27 | n = 25 | |
N (%) | N (%) | N (%) | |
Patients | |||
HRQOL (TTO social tariff score), mean (SD) | 0.167 (0.277) | 0.116 (0.285) | 0.532 (0.335) |
To be confined to bed | 11 (32.35) | 5 (18.52) | 5 (20.00)) |
Unable to wash or dress himself/herself | 14 (41.18) | 12 (44.44) | 8 (32.00) |
Unable to perform usual activities | 8 (23.53) | 7 (25.93) | 3 (12.00) |
Extreme pain/discomfort | 1 (2.94) | 1 (3.70) | 0 |
Suffer anxiety/mid depression | 0 | 2 (7.41) | 0 |
HRQOL (VAS score), mean (SD) | 75.44 (19.36) | 59.15 (29.84) | 69.76 (13.42) |
Caregivers | UK n = 11 | France n = 16 | Germany n = 14 |
HRQOL (TTO social tariff score), mean (SD) | 0.852 (0.155) a | 0.396 (0.468) a | 0.800 (0.298) a |
Unable to walk | 0 | 0 | 2 (15.38) |
Unable to wash or dress himself/herself | 0 | 4 (25.00) | 1 (7.69) |
Unable to perform usual activities | 0 | 4 (25.00) | 0 |
Extreme pain/discomfort | 0 | 1 (6.25) | 0 |
Suffer anxiety/mid depression | 0 | 2 (12.50) | 0 |
HRQOL (VAS score), mean (SD) | 80.36 (17.01) | 62.12 (33.41) | 71.92 (14.20) |
UK | France | Germany | ||||
---|---|---|---|---|---|---|
N = 34 | N = 27 | N = 25 | ||||
Mean (SD) | % | Mean (SD) | % | Mean (SD) | % | |
Drugs | 560 (2085) | 1.03% | 14 (42) | 0.04% | 35 (106) | 0.07% |
Medical tests | 874 (1223) | 1.61% | 384 (1375) | 1.20% | 158 (252) | 0.30% |
Medical visits | 4569 (9908) | 8.42% | 1870 (2463) | 5.84% | 1954 (3656) | 3.76% |
Hospitalizations | 2219 (5490) | 4.09% | 1229 (5260) | 3.84% | 3170 (4769) | 6.10% |
GP and Emergency | 842 (3130) | 1.55% | 144 (274) | 0.45% | 617 (1966) | 1.19% |
Medical material | 1958 (2428) | 3.61% | 990 (1770) | 3.09% | 1379 (1648) | 2.65% |
Healthcare transport | 58 (270) | 0.11% | 41 (152) | 0.13% | 0 | 0.00% |
Direct healthcare costs | 11,081 (10,764) | 20.41% | 4672 (7219) | 14.58% | 7313 (8636) | 14.07% |
Social services | 2187 (6197) | 4.03% | 1029 (2312) | 3.21% | 4380 (8044) | 8.43% |
Direct non-healthcare formal costs | 2187 (6197) | 4.03% | 1029 (2312) | 3.21% | 4380 (8044) | 8.43% |
Main informal carer | 27,012 (43,826) | 49.75% | 17,468 (22,550) | 54.52% | 27,436 (30,060) | 52.78% |
Other informal carers | 13,516 (25,151) | 24.89% | 8151 (16,583) | 25.44% | 12,490 (17,050) | 24.03% |
Direct non-healthcare informal costs | 40,526 (60,016) | 74.64% | 25,619 (35,263) | 79.95% | 39,926 (42,047) | 76.81% |
Non-healthcare transport a | 501 (1053) | 0.92% | 722 (1733) | 2.25% | 364 (381) | 0.70% |
Direct non-healthcare costs | 43,214 (61,139) | 79.59% | 27,370 (35,383) | 85.42% | 44,670 (45,063) | 85.94% |
TOTAL COST | 54,295 (68,431) | 100.00% | 32,042 (38,303) | 100.00% | 51,983 (47,662) | 100.00% |
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Peña-Longobardo, L.M.; Aranda-Reneo, I.; Oliva-Moreno, J.; Litzkendorf, S.; Durand-Zaleski, I.; Tizzano, E.; López-Bastida, J. The Economic Impact and Health-Related Quality of Life of Spinal Muscular Atrophy. An Analysis across Europe. Int. J. Environ. Res. Public Health 2020, 17, 5640. https://doi.org/10.3390/ijerph17165640
Peña-Longobardo LM, Aranda-Reneo I, Oliva-Moreno J, Litzkendorf S, Durand-Zaleski I, Tizzano E, López-Bastida J. The Economic Impact and Health-Related Quality of Life of Spinal Muscular Atrophy. An Analysis across Europe. International Journal of Environmental Research and Public Health. 2020; 17(16):5640. https://doi.org/10.3390/ijerph17165640
Chicago/Turabian StylePeña-Longobardo, Luz María, Isaac Aranda-Reneo, Juan Oliva-Moreno, Svenja Litzkendorf, Isabelle Durand-Zaleski, Eduardo Tizzano, and Julio López-Bastida. 2020. "The Economic Impact and Health-Related Quality of Life of Spinal Muscular Atrophy. An Analysis across Europe" International Journal of Environmental Research and Public Health 17, no. 16: 5640. https://doi.org/10.3390/ijerph17165640