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

Novartis

Status

Completed

Conditions

Muscular Atrophy, Spinal

Study type

Observational

Funder types

Industry

Identifiers

NCT07378943

COAV101A1US10

Details and patient eligibility

About

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.

Enrollment

4,114 patients

Sex

All

Volunteers

No Healthy Volunteers

Inclusion and exclusion 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

Exclusion criteria

• Treatment with nusinersen or risdiplam at any time