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

Dosing Patterns and Costs in Patients With Spinal Muscular Atrophy (SMA) Receiving Disease Modifying Therapies (DMT) - Real-world Analysis Using the Komodo Claims Data

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.

Study Overview

• Status: Completed
• Conditions: Muscular Atrophy, Spinal

• Study Type: Observational
• Enrollment (Actual): 4114

Contacts and Locations

Study Locations

• United States
  • New Jersey
    • East Hanover, New Jersey, United States, 07936
      • Novartis

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• 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.

Description

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

Exclusion criteria

• Treatment with nusinersen or risdiplam at any time

Study Plan

How is the study designed?

Number of groups / cohorts

3

Cohorts and Interventions

Group / Cohort
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.

What is the study measuring?

Primary Outcome Measures

Outcome 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 Outcome Measures

Outcome Measure	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	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

Collaborators and Investigators

• Sponsor: Novartis Pharmaceuticals
• Investigators: Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): November 6, 2024
• Primary Completion (Actual): March 30, 2025
• Study Completion (Actual): March 30, 2025

Study Registration Dates

• First Submitted: January 23, 2026
• First Submitted That Met QC Criteria: January 23, 2026
• First Posted (Actual): January 30, 2026

Study Record Updates

• Last Update Posted (Actual): January 30, 2026
• Last Update Submitted That Met QC Criteria: January 23, 2026
• Last Verified: January 1, 2026

More Information

Terms related to this study

• Keywords: Spinal muscular atrophy; Real-world evidence; Healthcare costs; Disease modifying therapies; Dosing patterns; Treatment costs
• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; Motor Neuron Disease; Muscular Atrophy, Spinal
• Other Study ID Numbers: COAV101A1US10

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO