Muscular Atrophy, Spinal
Conditions
Keywords
Spinal muscular atrophy, Disease modifying therapies, Real-world evidence, Dosing patterns, Treatment costs, Healthcare costs
Brief summary
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.
Interventions
None listed
Sponsors
Novartis Pharmaceuticals
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
Nusinersen and Risdiplam Cohorts: Inclusion criteria * Patients with ≥1 SMA diagnosis (International Classification of Diseases, Tenth Revision, Clinical Modification \[ICD-10-CM\] codes: G12.0, G12.1, G12.9) at any time * Patients with ≥1 record of nusinersen or risdiplam in the data based on relevant Healthcare Common Procedure Coding System (HCPCS) codes and National Drug Code (NDC) on or after December 23, 2016 * Patients aged ≥ 2 at the index date * Patients with ≥ 1 quarter of clinical activities within 1 year prior to the index quarter (i.e., the quarter containing the index date) * Patients with ≥ 2 quarters of clinical activities in the first year of the follow-up
Exclusion criteria
• None Onasemnogene Abeparvovec Intravenous Infusion (OAV) Monotherapy Cohort: Inclusion criteria * Patients with ≥1 SMA diagnosis (ICD-10-CM codes: G12.0, G12.1, G12.9) at any time * Patients with ≥1 record of OAV in the data based on relevant HCPCS codes and NDC on or after May 26, 2019 * Patients under 2 years old at the index date * Patients with ≥ 2 quarters of clinical activities in their first year of follow-up
Design outcomes
Primary
| Measure | 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 | 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 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Annualized All-cause Healthcare Costs During Each Year of OAV Follow-up | Years 1, 2, 3, and 4 | All-cause healthcare costs included costs for outpatient visits, emergency room visits, inpatient admissions, other medical visits, and pharmacy costs. |
| Annualized All-cause Healthcare Costs During the Entire OAV 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. |
| Annualized SMA-related Healthcare Costs During Each Year of OAV Follow-up | Years 1, 2, 3, and 4 | SMA-related healthcare costs included SMA-related complications, procedures, and durable medical equipment (DME). |
| Annualized SMA-related Healthcare Costs During the Entire OAV Follow-up Period | Up to 4 years | SMA-related healthcare costs included SMA-related complications, procedures, and durable medical equipment (DME). |
Countries
United States
Contacts
STUDY_DIRECTORNovartis Pharmaceuticals
Novartis Pharmaceuticals
Outcome results
None listed