Massive claims study reveals Real-World SMA treatment patterns and costs
NCT ID NCT07378943
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study analyzed insurance claims from over 4,000 people with spinal muscular atrophy to see how three approved treatments (Zolgensma, Spinraza, and Evrysdi) are actually used in daily practice and what they cost over time. Researchers looked at dosing patterns, yearly drug costs, and overall healthcare expenses using data from 2016 to 2024. The goal was to understand real-world treatment habits and financial burden, not to test a new therapy.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Participants
-
4,114 people
The number who actually took part.
- Started
-
Nov 2024
- Finished
-
Mar 2025
- Lead sponsor
-
A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
SMA patients with data in the KRD+ between 01 January 2016 and 31 October 2024 who received OAV, nusinersen, or risdiplam.
- Ages
-
Children (under 18), adults (18 to 64) and older adults (65 and over)
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Muscular atrophy, spinal are added.
Genom att skicka in godkänner du våra Användarvillkor
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Novartis
East Hanover, New Jersey, 07936, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a muscle-boosting antibody help people with spinal muscular atrophy over the long haul?
- Can a nationwide registry unlock the secrets of adult spinal muscular atrophy?
- Can a spinal injection safely slow spinal muscular atrophy? a real-world study in korea seeks answers.
- New drug BIIB115 aims to build on gene therapy for spinal muscular atrophy
- New hope for SMA babies: boosting gene therapy with a Follow-Up drug
- Real-World data reveals treatment patterns for kids with SMA