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 |
References
- Akobundu, E.; Ju, J.; Blatt, L.; Mullins, C.D. Cost-of-illness studies. Pharmacoeconomics 2006, 24, 869–890. [Google Scholar] [CrossRef] [PubMed]
- Seuring, T.; Archangelidi, O.; Suhrcke, M. The economic costs of type 2 diabetes: A global systematic review. Pharmacoeconomics 2015, 33, 811–831. [Google Scholar] [CrossRef] [PubMed] [Green Version]
- Paul, H.; Isabelle, S.; Linda, S. Measuring the societal burden of cancer: The cost of lost productivity due to premature cancer-related mortality in Europe. Int. J. Cancer 2015, 136, E136–E145. [Google Scholar]
- Ehteshami-Afshar, S.; FitzGerald, J.M.; Doyle-Waters, M.M.; Sadatsafavi, M. The global economic burden of asthma and chronic obstructive pulmonary disease. Int. J. Tuberc Lung Dis. 2016, 20, 11–23. [Google Scholar] [CrossRef] [PubMed]
- Quentin, W.; Riedel-Heller, S.G.; Luppa, M. Cost-of-illness studies of dementia: A systematic review focusing on stage dependency of costs. Acta Psychiatr. Scand. 2010, 121, 243–259. [Google Scholar] [CrossRef]
- Puig-Junoy, J.; Zamora, A.R. Socio-economic costs of osteoarthritis: A systematic review of cost-of-illness studies. Semin. Arthritis Rheum. 2015, 44, 531–541. [Google Scholar] [CrossRef]
- Oliva-Moreno, J.; Trapero-Bertran, M.; Peña-Longobardo, L.M.; Del Pozo-Rubio, R. The valuation of informal care in cost-of-illness studies: A systematic review. Pharmacoeconomics 2017, 35, 331–345. [Google Scholar] [CrossRef]
- López-Bastida, J.; Oliva-Moreno, J.; Linertová, R.; Serrano-Aguilar, P. Social/economic costs and health-related quality of life in patients with rare diseases in Europe. Eur. J. Health Econ. 2016, 17, 1–5. [Google Scholar] [CrossRef] [Green Version]
- Schieppati, A.; Henter, J.I.; Daina, E.; Aperia, A. Why rare diseases are an important medical and social issue. Lancet 2008, 371, 2039–2041. [Google Scholar] [CrossRef]
- Alías, L.; Barceló, M.J.; Bernal, S.; Martínez-Hernández, R.; Also-Rallo, E.; Vázquez, C.; Santana, A.; Millán, J.M.; Baiget, M.; Tizzano, E.F. Improving detection and genetic counseling in carriers of spinal muscular atrophy with two copies of the SMN1 gene. Clin. Genet. 2014, 85, 470–475. [Google Scholar] [CrossRef]
- López-Bastida, J.; Peña-Longobardo, L.M.; Aranda-Reneo, I.; Tizzano, E.; Sefton, M.; Oliva-Moreno, J. Social/economic costs and health-related quality of life in patients with spinal muscular atrophy (SMA) in Spain. Orphanet J. Rare Dis. 2017, 12, 141. [Google Scholar] [CrossRef] [PubMed] [Green Version]
- Talbot, K.; Tizzano, E.F. The clinical landscape for SMA in a new therapeutic era. Gene Ther. 2017, 24, 529–533. [Google Scholar] [CrossRef] [PubMed] [Green Version]
- Klug, C.; Schreiber-Katz, O.; Thiele, S.; Schorling, E.; Zowe, J.; Reilich, P.; Walter, M.C.; Nagels, K.H. Disease burden of spinal muscular atrophy in Germany. Orphanet J. Rare Dis. 2016, 11, 58. [Google Scholar] [CrossRef] [PubMed] [Green Version]
- Von Elm, E.; Altman, D.G.; Egger, M.; Pocock, S.J.; Gøtzsche, P.C.; Vandenbroucke, J.P. The strengthening the reporting of observational studies in epidemiology (STROBE) statement: Guidelines for reporting observational studies. Lancet 2007, 370, 1453–1457. [Google Scholar] [CrossRef]
- Dolan, P. Modeling valuations for EuroQol health states. Med. Care 1997, 35, 1095–1108. [Google Scholar] [CrossRef] [PubMed]
- Mahoney, F.I.; Barthel, D.W. Functional evaluation: The Barthel index. Md. State Med. 1965, 14, 61–65. [Google Scholar]
- Shah, S.; Vanclay, F.; Cooper, B. Improving the sensitivity of the Barthel index for stroke rehabilitation. J. Clin. Epidemiol. 1989, 42, 703–709. [Google Scholar] [CrossRef]
- Ruzafa, C.J.; Damian Moreno, J. Valoracion de la discapacidad fisica: El Indice de Barthel. Rev. Esp. Salud Pública 1997, 71, 127–137. [Google Scholar] [CrossRef]
- Hérbert, R.; Bravo, G.; Préville, M. Reliability, validity, and reference values of the Zarit burden interview for assessing informal caregivers of communitydwelling older persons with dementia. Can. J. Aging 2000, 19, 494–507. [Google Scholar] [CrossRef]
- Hoefman, R.J.; van Exel, J.; Brouwer, W. How to include informal care in economic evaluations. Pharmacoeconomics 2013, 31, 1105–1119. [Google Scholar] [CrossRef]
- Van den Berg, B.; Spauwen, P. Measurement of informal care: An empirical study into the valid measurement of time spent on informal caregiving. Health Econ. 2006, 15, 447–460. [Google Scholar] [CrossRef] [PubMed] [Green Version]
- Van den Berg, B.; Brouwer, W.; van Exel, J.; Koopmanschap, M.; van den Bos, G.; Rutten, F. Economic valuation of informal care: Lessons from the application of the opportunity costs and proxy good methods. Soc. Sci. Med. 2006, 62, 835–845. [Google Scholar] [CrossRef] [PubMed]
- Lopez-Bastida, J.; Monton-Alvarez, F.; Serrano-Aguilar, P.; Perestelo-Perez, L. Social economic costs and health-related quality of life in patients with degenerative cerebellar ataxia in Spain. Mov. Disord. 2008, 23, 212–217. [Google Scholar] [CrossRef] [PubMed]
- Chevreul, K.; Gandré, C.; Brigham, K.B.; López-Bastida, J.; Linertová, R.; Oliva-Moreno, J.; Serrano-Aguilar, P.; Posada-de-la-Paz, M.; Taruscio, D.; Schieppati, A.; et al. BURQOL-RD research network. Social/economic costs and health-related quality of life in patients with fragile X syndrome in Europe. Eur. J. Health Econ. 2016, 17, 43–52. [Google Scholar] [CrossRef]
- Cavazza, M.; Kodra, Y.; Armeni, P.; De Santis, M.; López-Bastida, J.; Linertová, R.; Oliva-Moreno, J.; Serrano-Aguilar, P.; Posada-de-la-Paz, M.; Taruscio, D.; et al. Social/economic costs and health-related quality of life in patients with Duchenne muscular dystrophy in Europe. Eur. J. Health Econ. 2016, 17, 19–29. [Google Scholar] [CrossRef]
- López-Bastida, J.; Perestelo-Pérez, L.; Montón-Alvarez, F.; Serrano-Aguilar, P.; Alfonso-Sanchez, J.L. Social economic costs and health-related quality of life in patients with amyotrophic lateral sclerosis in Spain. Amyotroph. Lateral Scler. 2009, 10, 237–243. [Google Scholar] [CrossRef]
- Thomas, G.P.; Saunders, C.L.; Roland, M.O.; Paddison, C.A. Informal carers’ health-related quality of life and patient experience in primary care: Evidence from 195,364 carers in England responding to a national survey. BMC Fam. Pract. 2015, 16, 62. [Google Scholar] [CrossRef] [Green Version]
- Pinquart, M.; Sörensen, S. Differences between caregivers and noncaregivers in psychological health and physical health: A meta-analysis. Psychol. Aging 2003, 18, 250–267. [Google Scholar] [CrossRef]
- Vitaliano, P.P.; Zhang, J.; Scanlan, J.M. Is caregiving hazardous to one’s physical health? A meta-analysis. Psychol. Bull. 2003, 129, 946–972. [Google Scholar] [CrossRef]
- Boumans, N.P.G.; Dorant, E. Double-duty caregivers: Healthcare professionals juggling employment and informal caregiving. A survey on personal health and work experiences. J. Adv. Nurs. 2014, 70, 1604–1615. [Google Scholar] [CrossRef] [Green Version]
- Courtin, E.; Jemiai, N.; Mossialos, E. Mapping support policies for informal carers across the European Union. Health Policy 2014, 118, 84–94. [Google Scholar] [CrossRef] [Green Version]
- Jacobs, J.C.; Van Houtven, C.H.; Tanielian, T.; Ramchand, R. Economic Spillover Effects of Intensive Unpaid Caregiving. Pharmacoeconomics 2019, 37, 553–562. [Google Scholar] [CrossRef] [PubMed] [Green Version]
- Metzelthin, S.F.; Verbakel, E.; Veenstra, M.Y.; van Exel, J.; Ambergen, A.W.; Kempen, G.I.J.M. Positive and negative outcomes of informal caregiving at home and in institutionalised long-term care: A cross-sectional study. BMC Geriatr. 2017, 17, 232. [Google Scholar] [CrossRef] [PubMed]
- Raúl, P.R.; Román, M.S.; Isabel, P.G.; Francisco, E.S. Catastrophic long-term care expenditure: Associated socio-demographic and economic factors. Eur. J. Health Econ. 2019, 20, 691–701. [Google Scholar]
- Finkel, R.S.; Mercuri, E.; Darras, B.T.; Connolly, A.M.; Kuntz, N.L.; Kirschner, J.; Chiriboga, C.A.; Saito, K.; Servais, L.; Tizzano, E.; et al. ENDEAR Study Group. Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy. N. Engl. J. Med. 2017, 377, 1723–1732. [Google Scholar] [CrossRef] [PubMed] [Green Version]
- Tizzano, E.F.; Finkel, R.S. Spinal muscular atrophy: A changing phenotype beyond the clinical trials. Neuromuscul. Disord. 2017, 27, 883–890. [Google Scholar] [CrossRef]
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
APA StylePeña-Longobardo, L. M., Aranda-Reneo, I., Oliva-Moreno, J., Litzkendorf, S., Durand-Zaleski, I., Tizzano, E., & López-Bastida, J. (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(16), 5640. https://doi.org/10.3390/ijerph17165640