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Examining Real-World Adherence to Nusinersen for the Treatment of Spinal Muscular Atrophy Using Two Large US Data
Bora Youn1, Crystal M Proud2, Nasha Wang3
1Biogen, Cambridge, MA, USA.
Advances in Therapy
|January 16, 2023
Summary
Most patients with spinal muscular atrophy (SMA) received nusinersen on schedule. Real-world data confirm adherence to the nusinersen dosing regimen, highlighting the importance of data accuracy in treatment evaluations.
Area of Science:
- Neurology
- Pharmacology
- Real-world evidence
Background:
- Spinal muscular atrophy (SMA) is a rare, progressive neuromuscular disease impacting muscle strength and function.
- Nusinersen represents the first approved therapeutic intervention for SMA, administered via a specific dosing schedule.
- Understanding real-world adherence to nusinersen's complex dosing is crucial for optimizing patient outcomes.
Purpose of the Study:
- To evaluate real-world adherence to the prescribed nusinersen administration schedule in patients with SMA.
- To assess the accuracy of capturing nusinersen dosing in large administrative healthcare databases.
- To analyze patient adherence patterns during both the loading and maintenance phases of nusinersen treatment.
Main Methods:
- Retrospective analysis of two large US databases: electronic health records (EHR) and healthcare claims (MarketScan).
- Identification of patients with SMA treated with nusinersen between 2017 and 2020.
- Summarization of baseline demographics, on-time nusinersen administrations, and inter-dose intervals.
Main Results:
- A total of 358 patients were included across the EHR (n=67) and claims (n=291) databases.
- High overall adherence was observed, with 93.9% of doses on time in EHR and 80.5% in claims data.
- Claims data suggested lower adherence during the loading phase (71.1%) compared to maintenance (90.6%), while EHR data showed high adherence in both phases (95.5% and 92.6%).
Conclusions:
- The majority of nusinersen doses were administered consistent with the recommended treatment schedule in real-world settings.
- Discrepancies between EHR and claims data highlight potential challenges in accurately capturing all nusinersen administrations, particularly loading doses.
- Methodological considerations are essential when utilizing administrative databases to evaluate treatment patterns for nusinersen in SMA.
