Epidemiology, healthcare resource utilization and healthcare costs for spinal muscular atrophy in Alberta, Canada

Abstract

Aims

Spinal muscular atrophy (SMA) is a progressive neuromuscular disease associated with the degeneration of motor neurons in the brainstem and spinal cord. Studies examining the epidemiology and economic impact of SMA are limited in Canada. This study aimed to estimate the epidemiology as well as healthcare resource utilization (HRU) and healthcare costs for children with SMA in Alberta, Canada.

Materials and methods

We conducted a retrospective study using anonymized data from administrative healthcare databases provided by Alberta Health. Data from 1 April 2010 to 31 March 2018, were extracted for patients <18 years of age identified with SMA. Five-year incidence and prevalence were calculated for cases identified between 1 April 2012 and 31 March 2017. HRU and healthcare costs were assessed one year after SMA diagnosis, including hospitalizations, physician visits, ambulatory care visits and long-term care admissions.

Results

The five-year incidence and prevalence of pediatric onset SMA were 1.03 per 100,000 person-years and 9.97 per 100,000 persons, respectively. General practitioner, specialist, and ambulatory care visits were common among children with SMA in the first-year post-diagnosis. The mean (SD) total annual direct cost per patient in the first-year post-diagnosis was $29,774 ($38,407); hospitalizations accounted for 41.7% of these costs ($12,412 [$21,170]), followed by practitioner visits at 32.3% ($9,615 [$13,054]), and ambulatory care visits at 26.0% ($7,746 [$9,988]).

Conclusions

Children with SMA experience substantial HRU, particularly for hospitalizations and practitioner visits, following diagnosis. Given the high costs of SMA, timely access to effective treatment strategies, such as the novel survival motor neuron (SMN)-restoring treatments recently approved for use, are needed to improve health outcomes and HRU.

Keywords:

• Spinal muscular atrophy
• cost-of-illness
• neuromuscular disease
• health services research
• epidemiology

JEL classification codes:

• I10
• I1
• I
• Z00
• Z

Transparency

Declaration of funding

Funding for this project was provided by Hoffmann-La Roche Limited, Mississauga, Ontario, Canada.

Declaration of financial/other interests

GC is a consultant for Medlior Health Outcomes Research Ltd. He has also received research funding from the Canadian Institutes for Health Research. BS, BG, MSF, and TC are employed by Medlior Health Outcomes Research Ltd. CC, JWW, BM, and KKP are employed by Hoffmann-La Roche Limited who funded this study and hold Hoffmann-La Roche Limited stock. JKM received a consultant fee and research grant from Hoffmann-La Roche Limited.

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Author contributions

GC provided support with statistical analysis. BS provided support with economic analysis. BG and MSF provided project management support and medical writing support. TC provided project management support. CC, JWW, BM and KKP provided project management and editing support. JKM provided clinical expertise and editing support.

Acknowledgements

This study is based on data provided by Alberta Health and Alberta Precision Laboratories. The interpretation and conclusions contained herein are those of the researchers and do not necessarily represent the views of the Government of Alberta. Neither the Government of Alberta nor Alberta Health expresses any opinion in relation to this study. We would also like to thank Wayne Khuu for his support with medical writing and analysis.

Notes

i SAS version 9.4 is a registered trademark of SAS Institute, Cary, NC, USA.