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The aim of this study was to assess real-world dosing patterns, long-term healthcare costs, and characteristics of SMA patients who received treatment with DMTs. DMTs included onasemnogene abeparvovec, nusinersen, and risdiplam. This study was conducted using both open and closed claims data from the Komodo Health Research Database (KRD+) between 01 January 2016 and 31 October 2024.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Nusinersen Cohort | Patients with SMA aged two years or older who received nusinersen. | ||
| Risdiplam Cohort | Patients with SMA aged two years or older who received risdiplam. | ||
| Onasemnogene Abeparvovec Intravenous Infusion (OAV) Monotherapy Cohort | Patients with SMA who were under two years of age and received OAV monotherapy. |
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| Measure | Description | Time Frame |
|---|---|---|
| Number of Patients by DMT During Each Year of DMT Treatment | Years 1, 2, 3, 4, and 5 | |
| Number of Patients Using Nusinersin During the Entire Treatment Follow-up Period | Up to 5 years | |
| Annualized Drug and Drug Administration Costs During Each Year of Nusinersen Treatment | Years 1, 2, 3, 4, and 5 | |
| Annualized Drug and Drug Administration Costs for Nusinersen During the Entire Treatment Follow-up Period | Up to 5 years | |
| Number of Nusinersen Injections During Each Year of Treatment | Years 1, 2, 3, 4, and 5 | |
| Number of Nusinersen Injections During the Entire Treatment Follow-up Period | Up to 5 years | |
| Number of Patients Using Risdiplam During the Entire Treatment Follow-up Period | Up to 4 years | |
| Annualized Drug Costs During Each Year of Risdiplam Treatment | Years 1, 2, 3, and 4 | |
| Annualized Drug Costs for Risdiplam During the Entire Treatment Follow-up Period | Up to 4 years | |
| Annualized Risdiplam Costs Among Patients With More Than 90 Days of Supply in Year 1 |
| Measure | Description | Time Frame |
|---|---|---|
| Annualized All-cause Healthcare Costs During Each Year of OAV Follow-up | All-cause healthcare costs included costs for outpatient visits, emergency room visits, inpatient admissions, other medical visits, and pharmacy costs. | Years 1, 2, 3, and 4 |
| Annualized All-cause Healthcare Costs During the Entire OAV Follow-up Period |
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Nusinersen and Risdiplam Cohorts:
Inclusion criteria
Exclusion criteria • None
Onasemnogene Abeparvovec Intravenous Infusion (OAV) Monotherapy Cohort:
Inclusion criteria
Exclusion criteria
• Treatment with nusinersen or risdiplam at any time
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SMA patients with data in the KRD+ between 01 January 2016 and 31 October 2024 who received OAV, nusinersen, or risdiplam.
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis | East Hanover | New Jersey | 07936 | United States |
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| ID | Term |
|---|---|
| D009134 | Muscular Atrophy, Spinal |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
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| Year 1 |
| Mean Prescribed Risdiplam Dosage During Each Year of Treatment | Year 1, 2, 3, and 4 |
| Mean Number of Days of Supply of Risdiplam During Each Year of Treatment | Year 1, 2, 3, and 4 |
| Mean Daily Dosage of Risdiplam During Each Year of Treatment | Year 1, 2, 3, and 4 |
| Mean Prescribed Risdiplam Dosage During the Entire Treatment Follow-up Period | Up to 4 years |
| Mean Number of Days of Supply of Risdiplam During the Entire Treatment Follow-up Period | Up to 4 years |
| Mean Daily Dosage of Risdiplam During the Entire Treatment Follow-up Period | Up to 4 years |
All-cause healthcare costs included costs for outpatient visits, emergency room visits, inpatient admissions, other medical visits, and pharmacy costs. |
| Up to 4 years |
| Annualized SMA-related Healthcare Costs During Each Year of OAV Follow-up | SMA-related healthcare costs included SMA-related complications, procedures, and durable medical equipment (DME). | Years 1, 2, 3, and 4 |
| Annualized SMA-related Healthcare Costs During the Entire OAV Follow-up Period | SMA-related healthcare costs included SMA-related complications, procedures, and durable medical equipment (DME). | Up to 4 years |
| D019636 | Neurodegenerative Diseases |
| D009468 | Neuromuscular Diseases |