A Study of Dosing Patterns and Costs in Patients With Spinal Muscular Atrophy Receiving Disease Modifying Therapies

NCT07378943 | Completed

• 1 other identifier: COAV101A1US10
• Study Type: observational
• Target: 4,114
• Locations: 1 country
• Sites: 1

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.

Conditions

Muscular Atrophy, Spinal

Keywords

Spinal muscular atrophy; Disease modifying therapies; Real-world evidence; Dosing patterns; Treatment costs; Healthcare costs

Outcome Measures

Primary Outcomes (16)

• 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 Outcomes (4)

• Annualized All-cause Healthcare Costs During Each Year of OAV Follow-up

  Years 1, 2, 3, and 4

• Annualized All-cause Healthcare Costs During the Entire OAV Follow-up Period

  Up to 4 years

• Annualized SMA-related Healthcare Costs During Each Year of OAV Follow-up

  Years 1, 2, 3, and 4

• Annualized SMA-related Healthcare Costs During the Entire OAV Follow-up Period

  Up to 4 years

Study Arms (3)

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.

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

SMA patients with data in the KRD+ between 01 January 2016 and 31 October 2024 who received OAV, nusinersen, or risdiplam.

You may qualify if:

• 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

You may not qualify if:

• Onasemnogene Abeparvovec Intravenous Infusion (OAV) Monotherapy Cohort:
• 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
• Treatment with nusinersen or risdiplam at any time

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (1)

Novartis

East Hanover, New Jersey, 07936, United States

Location

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; Neuromuscular Diseases

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 23, 2026
• First Posted: January 30, 2026
• Study Start: November 6, 2024
• Primary Completion: March 30, 2025
• Study Completion: March 30, 2025
• Last Updated: January 30, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)