The economic burden of spinal muscular atrophy

Armstrong, Edward P.; Malone, Daniel C.; Yeh, Wei-Chang; Dahl, G; Lee, Rees L.; Sicignano, Nicholas

doi:10.1080/13696998.2016.1198355

Cited by 42 publications

(60 citation statements)

References 13 publications

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“…In accordance with findings from the current study, previous studies have consistently found SMA to be associated with high HRU and healthcare costs [11][12][13][14][15][16][17][18][19] , including a few recent claims-based studies that evaluated the economic burden of SMA [12][13][14] . For example, Dabbous et al 13 conducted a retrospective analysis of patients with SMA1 using the Quintiles IMS's PharMetrics Plus Health Plan Claims Database (February 2011-November 2016).…”

Section: Discussionsupporting

confidence: 91%

“…who likely had SMA1) had particularly high healthcare costs that amounted to $159,227 PPPY, those aged 2-18 years had PPPY costs of $105,206, and those aged >18 years had PPPY costs of $39,355. Similarly, Armstrong et al 12 conducted a retrospective analysis of pediatric patients (<18 years) with SMA using US Department of Defense Military Healthcare System (MHS) data contained within the MHS Data Repository (2003-2012). The authors reported that PPPY healthcare costs were significantly higher for patients diagnosed in their first year of life (i.e.…”

Section: Discussionmentioning

confidence: 99%

“…Assessments of the real-world economic burden of SMA, including the healthcare resource utilization (HRU) and healthcare costs associated with the disease, remain limited in the US [11][12][13][14][15][16][17][18][19] . In a study conducted by Dabbous et al 13 , which evaluated the economic burden of patients with SMA1 in the US, healthcare costs in the first month following SMA diagnosis averaged $27,063 per patient compared to $274 among matched patients without SMA.…”

Section: Introductionmentioning

confidence: 99%

“…This figure was notably high in patients aged <2 years old, for whom it reached $159,227 PPPY 14 . However, prior studies included data collected before the approval of nusinersen and, thus, did not capture the impact of this recent development on the disease burden [11][12][13][14][15][16][17][18][19] . Therefore, the primary objective of this study was to provide contemporary estimates of the HRU and healthcare costs among patients with SMA1 in realworld US practice, overall and following the initiation of nusinersen among treated patients.…”

Section: Introductionmentioning

confidence: 99%

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Economic burden of spinal muscular atrophy in the United States: a contemporary assessment

Droege

Sproule

Arjunji

et al. 2019

Journal of Medical Economics

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Aims: To estimate healthcare resource utilization (HRU) and costs among patients with spinal muscular atrophy (SMA) type 1 (SMA1) in real-world practice, overall and among patients treated with nusinersen. As a secondary objective, HRU and costs were estimated among patients with other SMA types (i.e. 2, 3, or 4 combined), overall and among patients treated with nusinersen. Materials and methods: Patients with SMA were identified from the Symphony Health's Integrated Dataverse (IDV) open claims database (September 1, 2016-August 31, 2018) and were classified into four cohorts based on SMA type and nusinersen treatment (i.e. SMA1, SMA1 nusinersen, other SMA, and other SMA nusinersen cohorts). The index date was the date of the first SMA diagnosis after December 23, 2016 or, for nusinersen cohorts, the date of nusinersen initiation. The study period spanned from the index date to the earlier among the end of clinical activity or data availability. Results: Patients in the SMA1 (n ¼ 349) and SMA1 nusinersen (n ¼ 45) cohorts experienced an average of 59.4 and 56.6 days with medical visits per-patient-per-year (PPPY), respectively, including 14.1 and 4.6 inpatient days. Excluding nusinersen-related costs, total mean healthcare costs were $137,627 and $92,618 PPPY in the SMA1 and SMA1 nusinersen cohorts, respectively. Mean nusinersen-related costs were $191,909 per-patient-per-month (PPPM) for the first 3 months post-initiation (i.e. loading phase) and $36,882 PPPM thereafter (i.e. maintenance phase). HRU and costs were also substantial among patients in the other SMA (n ¼ 5,728) and other SMA nusinersen (n ¼ 404) cohorts, with an average of 44.5 and 63.7 days with medical visits PPPY and total mean healthcare costs (excluding nusinersenrelated costs) of $49,175 and $76,371 PPPY, respectively. Limitations: The database may contain inaccuracies or omissions in diagnoses, procedures, or costs, and does not capture medical services outside of the IDV network. Conclusions: HRU and healthcare costs were substantial in patients with SMA, including in nusinersen-treated patients.

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Section: Discussionsupporting

confidence: 91%

Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Economic burden of spinal muscular atrophy in the United States: a contemporary assessment

Droege

Sproule

Arjunji

et al. 2019

Journal of Medical Economics

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“…(8,9) Given the rarity of SMA, the direct and indirect costs per capita are close to $200,000. (10,11) The latest treatment patterns and economic study on patients with SMA found signi cantly higher healthcare utilization and costs than the general population. (12) Multiple studies have found substantially higher costs for patients with SMA compared to healthy populations.…”

Section: Introductionmentioning

confidence: 99%

Treatment Preference Among Patients With Spinal Muscular Atrophy (SMA): A Discrete Choice Experiment

Monnette

Er²,

Hong

et al. 2020

Preprint

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OBJECTIVE: To examine patient/caregiver preference for key attributes of treatments for spinal muscular atrophy (SMA). BACKGROUND: In the rapidly evolving SMA treatment landscape, it is critically important to understand how attributes of potential treatments may impact patient/caregiver choices.DESIGN/METHODS: A discrete choice experiment survey was developed based on qualitative interviews. Patients with SMA (18 years) and caregivers of patients were recruited through a U.S. patient organization. Respondents made choices in each of 12 sets of hypothetical treatments. The relative importance of five treatment characteristics was compared (measured by regression coefficients [RC] of conditional logit models): (1) improvement or stabilization of motor function, (2) improvement or stabilization of breathing function, (3) indication for all ages or pediatric patients only, (4) route of administration (repeated intrathecal [IT] injections, one-time intravenous [IV] infusion, daily oral delivery) and (5) potential harm (mild, moderate, serious/life threatening).RESULTS: Patient ages ranged from less than 1 to 67 years (n=101, 65 self-reported and 36 caregiver-reported) and 64 were female. Total SMA subtypes included: type 1 (n=21), type 2 (n=48), type 3 (n=29), other (n=3). Prior spinal surgery was reported in 47 patients. Nusinersen and onasemnogene abeparvovec-xioi use were reported in 59 and 10 patients, respectively. Improvement in motor and breathing function was highly valued (RC: 0.65, 95% confidence interval [CI]: 0.47–0.83 and RC: 0.79, 95% CI: 0.60–0.98, respectively). Oral medication and one-time infusion were strongly preferred over repeated IT injections (RC: 0.80, 95% CI: 0.60–0.98 and RC: 0.51, 95% CI: 0.30–0.73, respectively). Patients least preferred an age-restricted label/approved use ( 2 years of age) (RC: -1.28, 95% CI: -1.47 to -1.09). Cross-attributes trade-off decision suggested a lower willingness for a high-risk therapy despite additional efficacy gain. For some patients, there may be willingness to trade off additional gains in efficacy for a change in route of administration from repeated intrathecal administration to oral medication.CONCLUSIONS: Improvements in motor/breathing function, broad indication, oral or one-time infusion, and minimal risk were preferred treatment attributes. Treatment decisions should be made in clinical context and be tailored to patient needs.

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Significant healthcare burden and life cost of spinal muscular atrophy: real-world data

Chan

Wong

et al. 2022

Eur J Health Econ

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Objectives The aim of this study is to quantify the mortality rate, direct healthcare costs, and cumulative life costs of pediatric patients with spinal muscular atrophy (SMA) type 1, type 2, and type 3 born in Hong Kong. Methods Data were collected from genetically confirmed SMA patients born in or after 2000 from the Hospital Authority medical database. Patients were followed up from birth until they died, left Hong Kong, reached 18 years, or initiated disease-modifying treatment. Study outcomes included incidence risks of mortality, cumulative direct medical costs—attendances of special outpatient clinics, emergency department, allied health services, and mean length of stay in hospitals over time. Total direct medical costs were calculated as unit costs multiplied by utilization frequencies of corresponding healthcare services at each age. Results Seventy-one patients with SMA were included. Over a median follow-up period of 6 years, the overall incidence rate of death was 5.422/100 person-years (95%CI 3.542–7.945/100 person-years). 67.7% and 11% of deaths occurred in SMA1 and SMA2 groups, respectively. The median age of death was 0.8 years in SMA1 and 10.9 years in SMA2. The mean cumulative direct medical costs in overall SMA, SMA1, SMA2 and SMA3 groups per patient were US$935,570, US$2,393,250, US$413,165, and US$40,735, respectively. Interpretation: Our results confirmed a significantly raised mortality and extremely high healthcare burden for patients with SMA especially SMA type 1 and 2 without disease-modifying treatment. Study evaluating health and economic impact of newborn screening and early treatment is needed.

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The economic burden of spinal muscular atrophy

Abstract: Individuals with SMA have a high degree of morbidity, particularly those diagnosed during infancy. SMA patients have significant medical expenditures and high utilization of healthcare services.

Cited by 42 publications

References 13 publications

Economic burden of spinal muscular atrophy in the United States: a contemporary assessment

Economic burden of spinal muscular atrophy in the United States: a contemporary assessment

Treatment Preference Among Patients With Spinal Muscular Atrophy (SMA): A Discrete Choice Experiment

Significant healthcare burden and life cost of spinal muscular atrophy: real-world data

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