Real-world phosphorodiamidate morpholino oligomer treatment patterns in Duchenne muscular dystrophy: a claims-based analysis
Publication: Journal of Comparative Effectiveness Research
Abstract
Aim: Phosphorodiamidate morpholino oligomers (PMOs) are exon-skipping therapies administered through once-weekly intravenous infusions used to treat Duchenne muscular dystrophy (DMD). This study assessed treatment patterns among patients with DMD receiving PMOs using administrative claims data while accounting for limitations in claims data for these therapies. Materials & methods: This study used Inovalon® public and private closed claims data (1 June 2016–31 March 2024). Male patients with ≥1 claim for a PMO approved for DMD in the US (eteplirsen, casimersen, golodirsen and viltolarsen) were included. Index date was the first PMO claim. All available follow-up data were used to assess continuous PMO claims coverage, ≥60-day and ≥30-day gaps in PMO claims and PMO re-initiation after a gap. Adherence during 1 year after index was measured using proportion of days covered (PDC). Treatment patterns were also assessed in patients stratified by baseline algorithm-defined nonambulatory status (inferred from claims). Results: Among 397 patients included, median (IQR) follow-up time was 788 (484, 1109) days. Gaps in PMO claims coverage occurred in 190 (47.9%) and 254 (64.0%) patients using ≥60-day and ≥30-day gaps, respectively, among whom 110 (57.9%) and 176 (69.3%) had PMO re-initiation. Using ≥60-day and ≥30-day gap lengths, median (IQR) time to first gap in PMO claims was 25.5 (22.3, 32.9) months and 13.5 (10.2, 17.7) months, respectively and median (IQR) time to PMO re-initiation (not including gap time) was 4.4 (2.8, 8.7) months and 2.5 (1.7, 3.2) months. Median (IQR) PDC was 78.8% (38.8, 94.0) during 1 year after index. PMO treatment patterns were generally similar in patients stratified by algorithm-defined nonambulatory status. Conclusion: In an analysis of administrative claims data, adherence to PMO treatment for DMD was high. For patients with a gap in PMO claims, most subsequently re-initiated treatment, indicating lower discontinuation rates than previously reported.
Plain language summary: Phosphorodiamidate morpholino oligomers in patients with Duchenne muscular dystrophy: how long are these treatments used?
Why was this study done?
Duchenne muscular dystrophy (DMD) is a debilitating disease involving muscle weakness that starts in the first years of life and worsens over time. Most affected children lose their ability to walk by their teens. Phosphorodiamidate morpholino oligomers (PMOs) are a type of treatment used in certain patients with DMD. However, it is not well known how long patients stay on these treatments.
What did this study look at?
This study used information from health insurance claims to look at how long patients with DMD stay on PMO treatment, how many patients have a longer-than-expected gap in PMO claims and whether patients who have a gap in claims eventually re-initiate PMO treatment after a gap.
What were the results?
Nearly 400 patients were included in the study. On average, patients stayed on PMO treatment without any gaps in claims for more than 2 years. Overall, about 80% of the patients in the study either stayed on PMO treatment without a gap in claims through to the end of the study or re-initiated PMO treatment after a gap. The results were similar between patients who were not able to walk (nonambulatory patients) and those who may have been able to walk (ambulatory patients) when they started PMO treatment.
What do the results mean?
The results show that patients with DMD who are taking PMOs stay on the treatment for a long period of time and that patients with a gap in claims often continue their treatment.
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References
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Received: 18 March 2025
Accepted: 13 June 2025
Published online: 18 July 2025
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Real-world phosphorodiamidate morpholino oligomer treatment patterns in Duchenne muscular dystrophy: a claims-based analysis. (2025) Journal of Comparative Effectiveness Research. DOI: 10.57264/cer-2025-0037
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