Real-world Adherence to Nusinersen in Adults with Spinal Muscular Atrophy in the US: A Multi-site Chart Review Study

Elman, Lauren; Youn, Bo-Ra; Proud, Crystal; Frey, Margaret R.; Ajroud‐Driss, Senda; McCormick, Mary; Michelson, David; Cartwright, Michael S.; Heiman‐Patterson, Terry; Choi, Joseph M.; Chandak, Aastha; Khachatryan, Artak; Martínez, Marta; Paradis, Angela D.

doi:10.3233/jnd-210768

Cited by 6 publications

(19 citation statements)

References 23 publications

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“…Elman et al conducted an analysis of a retrospective chart review using a limited set of Electronic Medical Record (EMR) data from nine Muscular Dystrophy Association (MDA) clinics and used the same grace periods as this study. In contrast to our findings, Elman et al found that 58 patients (67% of cohort) were adherent at 24 months and 92% of doses were administered on schedule when doses were considered cumulatively across all patients [24]. Certain study design aspects of the analysis by Elman et al may have contributed in part to the differences in the adherence observed compared with this analysis.…”

Section: Discussioncontrasting

confidence: 99%

“…Elman et al found that 92% of patients with SMA were persistent with nusinersen treatment at 24 months [24]. In contrast, and similar to the present study results, Gauthier-Loiselle et al [25] reported that \ 50% of patients, regardless of SMA type, remained on treatment 2 years after starting nusinersen.…”

Section: Discussionsupporting

confidence: 88%

“…There are additional studies in SMA that showed comparatively lower discontinuation rates (5-10%) during the first 12--24 months of treatment, with relatively high adherence [31][32][33][34][35]. However, these studies were center based and either did not include or did not have as rigid definitions of adherence and persistence, as these were not the main outcomes for their analyses when compared with Elman et al [24], Gauthier-Loiselle et al [25], or this study.…”

Section: Discussionmentioning

confidence: 73%

“…*Per product label and grace period (7 days for loading doses, 28 days for maintenance doses). All patients represents the total number of patients with the corresponding follow-up time [24,25]. In this base-case persistence analysis, a decrease in persistence before 6 months (67%) and further decline at 1 (57%) and 2 years (55%) was observed.…”

Section: Discussionmentioning

confidence: 86%

“…This decrease in persistence may better reflect the actual rate of discontinuation due to adverse events or lack of efficacy. The sustained persistence observed between 6 months and 2 years may have been attributed to the longer dosing intervals and grace periods after 24 weeks, which required less This study used the same discontinuation definition as Elman et al [24], and the present base-case persistence estimates may reflect the larger sample size of this study. Furthermore, the present analysis used real-world data from adjudicated insurance claims across the USA, whereas Elman et al included EMR data from 86 patients from nine pre-selected MDA-associated clinics.…”

Section: Discussionmentioning

confidence: 87%

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Adherence and Persistence to Nusinersen for Spinal Muscular Atrophy: A US Claims-Based Analysis

Fox¹,

To²,

Seetasith³

et al. 2022

Adv Ther

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Introduction: Spinal muscular atrophy (SMA) is a genetic, neuromuscular disease caused by deletions and/or mutations in the survival of motor neuron 1 (SMN1) gene leading to reduced SMN protein levels. Nusinersen, an intrathecally administered antisense oligonucleotide therapy that increases SMN protein levels, is approved for use in adult and pediatric patients with SMA. Data to inform real-world patient adherence and persistence to nusinersen are limited, with disparities in the population with SMA, study design, and results. The objective of this study is to characterize real-world nusinersen adherence and persistence in patients with SMA. Methods: This retrospective study examined nusinersen adherence and persistence over a 2-year period in patients with SMA in the USA from the IQVIA PharMetrics Plus claims database. Patients were followed from the date

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Section: Discussioncontrasting

confidence: 99%

Section: Discussionsupporting

confidence: 88%

Section: Discussionmentioning

confidence: 73%

Section: Discussionmentioning

confidence: 86%

Section: Discussionmentioning

confidence: 87%

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Adherence and Persistence to Nusinersen for Spinal Muscular Atrophy: A US Claims-Based Analysis

Fox¹,

To²,

Seetasith³

et al. 2022

Adv Ther

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Examining Real-World Adherence to Nusinersen for the Treatment of Spinal Muscular Atrophy Using Two Large US Data Sources

et al. 2023

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Introduction Spinal muscular atrophy (SMA) is a rare neuromuscular disease characterized by progressive muscular atrophy and weakness. Nusinersen was the first treatment approved for SMA. Per the US label, the nusinersen administration schedule consists of three loading doses at 14-day intervals, a fourth loading dose 30 days later, and maintenance doses every 4 months thereafter. Using two large US databases, we evaluated real-world adherence to nusinersen with its unique dosing schedule among generalizable populations of patients with SMA. Methods Patients with SMA treated with nusinersen, likely to have complete information on date of treatment initiation, were identified in the Optum ® de-identified electronic health records (EHR) database (7/2017–9/2019), and in the Merative™ MarketScan ® Research Databases from commercial (1/2017–6/2020) and Medicaid claims (1/2017–12/2019). Baseline demographics, number of nusinersen administrations on time, and distribution of inter-dose intervals were summarized. Results Totals of 67 and 291 patients were identified in the EHR and claims databases, respectively. Most nusinersen doses were received on time (93.9% EHR, 80.5% claims). Adherence was higher during the maintenance phase (90.6%) than the loading phase (71.1%) in the claims analysis, in contrast with the EHR analysis (95.5% and 92.6%, respectively), suggesting that not all loading doses of nusinersen may be accurately captured in claims. Inter-dose intervals captured in both databases aligned with the expected dosing schedule. Conclusion Most nusinersen doses were received on time, consistent with the recommended schedule. Our findings also highlight the importance of careful methodological approaches when using real-world administrative databases for evaluation of nusinersen treatment patterns. Supplementary Information The online version contains supplementary material available at 10.1007/s12325-022-02414-9.

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Adherence and Persistence Among Risdiplam-Treated Individuals with Spinal Muscular Atrophy: A Retrospective Claims Analysis

Pineda,

To,

Dickendesher

et al. 2024

Adv Ther

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Introduction Spinal muscular atrophy (SMA) is a neuromuscular disease caused by deletions and/or mutations in the survival of motor neuron 1 ( SMN1 ) gene. Risdiplam, the first and only oral SMN2 pre-mRNA splicing modifier, is US Food and Drug Administration-approved for the treatment of pediatric and adult patients with SMA. For patients with SMA, long-term adherence to and persistence with an SMA treatment may be important for achieving maximum clinical benefits. However, real-world evidence on patient adherence to and persistence with risdiplam is limited. Methods This retrospective study examined real-world adherence and persistence with risdiplam from a specialty pharmacy in patients with SMA over a 12-month period. Adherence was estimated by using proportion of days covered (PDC) and was calculated over variable (time between first and last fill) and fixed (time from first fill to study period end) intervals. Persistence was defined as no gap in supply ≥ 90 days. Patients were included if the time between the index date and study observation period was ≥ 12 months, if they initiated risdiplam between August 2020 and September 2022, received ≥ 2 risdiplam fills, and had an SMA diagnosis associated with a risdiplam fill. Subgroup analyses of risdiplam adherence and persistence were performed by age and primary payer type. Results The proportion of patients ( N = 1636) adherent at 12 months based on variable and fixed interval PDC was 93% and 79%, respectively. Adherence was high among patients on commercial insurance, Medicaid, or Medicare (range 86–96%). Mean persistence was 330.4 days. The highest proportion of patients who were persistent were on Medicaid (81%). Conclusion These findings demonstrate that patient adherence to and persistence with risdiplam treatment were high, including across all subgroups tested. Supplementary Information The online version contains supplementary material available at 10.1007/s12325-024-02850-9.

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Real-world Adherence to Nusinersen in Adults with Spinal Muscular Atrophy in the US: A Multi-site Chart Review Study

Cited by 6 publications

References 23 publications

Adherence and Persistence to Nusinersen for Spinal Muscular Atrophy: A US Claims-Based Analysis

Adherence and Persistence to Nusinersen for Spinal Muscular Atrophy: A US Claims-Based Analysis

Examining Real-World Adherence to Nusinersen for the Treatment of Spinal Muscular Atrophy Using Two Large US Data Sources

Adherence and Persistence Among Risdiplam-Treated Individuals with Spinal Muscular Atrophy: A Retrospective Claims Analysis

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