CompletedNot Applicable

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

United States4,114 participantsStarted 2024-11-06

Plain-language 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.

Who can participate

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

Number of Patients by DMT During Each Year of DMT Treatment

Timeframe: Years 1, 2, 3, 4, and 5

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

Timeframe: Up to 5 years

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

Timeframe: Years 1, 2, 3, 4, and 5

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

Timeframe: Up to 5 years

Number of Nusinersen Injections During Each Year of Treatment

Timeframe: Years 1, 2, 3, 4, and 5

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

Timeframe: Up to 5 years

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

Timeframe: Up to 4 years

Annualized Drug Costs During Each Year of Risdiplam Treatment

Trial details

NCT IDNCT07378943

SponsorNovartis Pharmaceuticals

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2025-03-30

View on ClinicalTrials.gov More Muscular Atrophy, Spinal trials

Plain-language summary

Who can participate

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Number of Patients by DMT During Each Year of DMT Treatment

Timeframe: Years 1, 2, 3, 4, and 5

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

Timeframe: Up to 5 years

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

Timeframe: Years 1, 2, 3, 4, and 5

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

Timeframe: Up to 5 years

Number of Nusinersen Injections During Each Year of Treatment

Timeframe: Years 1, 2, 3, 4, and 5

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

Timeframe: Up to 5 years

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

Timeframe: Up to 4 years

Annualized Drug Costs During Each Year of Risdiplam Treatment