High Healthcare Resource Use in Hospitalized Patients with a Diagnosis of Spinal Muscular Atrophy Type 1 (SMA1): Retrospective Analysis of the Kids’ Inpatient Database (KID)

Cardenas, Jessica; Menier, M.; Heitzer, Marjet D.; Sproule, Douglas M.

doi:10.1007/s41669-018-0093-0

Cited by 20 publications

(32 citation statements)

References 22 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: 90%

“…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: 90%

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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“…Improved functioning in SMA patients may also have positive economic effects. Our recent retrospective analysis of the Kids’ Inpatient Database (KID) database showed that the admission charges for SMA1 children were an average of $150 921 per hospitalization ($11 143 per day) . The decreased frequency of respiratory infections and hospitalizations observed following treatment with AVXS‐101 compared to untreated SMA1 patients, are likely to result in concomitant reductions in healthcare costs associated with treating SMA1 children.…”

Section: Discussionmentioning

confidence: 99%

Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy

Al-Zaidy¹,

Pickard

Kotha

et al. 2018

Pediatric Pulmonology

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135

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Background Spinal Muscular Atrophy type 1 (SMA1) is a rare genetic neuromuscular disease where 75% of SMA1 patients die/require permanent‐ventilation by 13.6 months. This study assessed the health outcomes of SMA1 infants treated with AVXS‐101 gene replacement therapy. Methods Twelve genetically confirmed SMA1 infants with homozygous deletions of the SMN1 gene and two SMN2 gene copies received a one‐time intravenous proposed therapeutic dose of AVXS‐101 in an open label study conducted between December 2014 and 2017. Patients were followed for 2‐years post‐treatment for outcomes including (1) pulmonary interventions; (2) nutritional interventions; (3) swallow function; (4) hospitalization rates; and (5) motor function. Results All 12 patients completed the study. Seven infants did not require noninvasive ventilation (NIV) by study completion. Eleven patients had stable or improved swallow function, demonstrated by the ability to feed orally; 11 patients were able to speak. The mean proportion of time hospitalized was 4.4%; the mean unadjusted annualized hospitalization rate was 2.1 (range = 0, 7.6), with a mean length of stay/hospitalization of 6.7 (range = 3, 12.1) days. Eleven patients achieved full head control and sitting unassisted and two patients were walking independently. Conclusions AVXS‐101 treatment in SMA1 was associated with reduced pulmonary and nutritional support requirements, improved motor function, and decreased hospitalization rate over the follow‐up period. This contrasts with the natural history of progressive respiratory failure and reduced survival. The reduced healthcare utilization could potentially alleviate patient and caregiver burden, suggesting an overall improved quality of life following gene replacement therapy. Trial registration http://ClinicalTrials.gov number, NCT02122952.

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“…(12) Multiple studies have found substantially higher costs for patients with SMA compared to healthy populations. (10,11,13) In addition to impairment on quality of life and social participation among patients with SMA, SMA was associated with reduced caregiver quality of life, which was related to patient burden, impaired psychosocial well-being, and loss of caregiver's productivity. (14)(15)(16) Despite the progress in SMA treatment with two important landmark treatments (approved by the Food and Drug Administration [FDA]), the disease burden and related unmet need remains signi cant for many patients with SMA and their caregivers.…”

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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High Healthcare Resource Use in Hospitalized Patients with a Diagnosis of Spinal Muscular Atrophy Type 1 (SMA1): Retrospective Analysis of the Kids’ Inpatient Database (KID)

Cited by 20 publications

References 22 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

Health outcomes in spinal muscular atrophy type 1 following AVXS‐101 gene replacement therapy

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

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