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

Novartis Pharmaceuticals4,114 enrolled

Overview

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 Type

OBSERVATIONAL

Enrollment

4,114

Conditions

Muscular Atrophy, Spinal

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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

Locations (1)

Novartis

East Hanover, New Jersey, United States

Outcomes

Primary Outcomes

Number of Patients by DMT During Each Year of DMT Treatment

Time frame: Years 1, 2, 3, 4, and 5

Number of Patients Using Nusinersin During the Entire Treatment Follow-up Period

Time frame: Up to 5 years

Annualized Drug and Drug Administration Costs During Each Year of Nusinersen Treatment

Time frame: Years 1, 2, 3, 4, and 5

Annualized Drug and Drug Administration Costs for Nusinersen During the Entire Treatment Follow-up Period

Time frame: Up to 5 years

Number of Nusinersen Injections During Each Year of Treatment

Time frame: Years 1, 2, 3, 4, and 5

Number of Nusinersen Injections During the Entire Treatment Follow-up Period

Time frame: Up to 5 years

Number of Patients Using Risdiplam During the Entire Treatment Follow-up Period

Time frame: Up to 4 years

Annualized Drug Costs During Each Year of Risdiplam Treatment

Time frame: Years 1, 2, 3, and 4

Annualized Drug Costs for Risdiplam During the Entire Treatment Follow-up Period

Time frame: Up to 4 years

Annualized Risdiplam Costs Among Patients With More Than 90 Days of Supply in Year 1

Time frame: Year 1

Mean Prescribed Risdiplam Dosage During Each Year of Treatment

Time frame: Year 1, 2, 3, and 4

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.

Secondary Outcomes

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.

Time frame: 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.

Time frame: 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).

Time frame: 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).

Time frame: Up to 4 years