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

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.

The Association Between Persistence and Adherence to Disease-Modifying Therapies and Healthcare Resource Utilization and Costs in Patients With Multiple Sclerosis.

Background: Persistence and adherence to disease-modifying therapies (DMTs) affects treatment efficacy and economic outcomes, both of which contribute to overall patient disease burden. Current literature suggests that patients with multiple sclerosis (MS) who adhere to DMT for 12 months have fewer relapses and reduced MS-related healthcare resource utilization (HCRU) and medical costs than nonadherent patients. Objective: To expand on previous research by estimating the association of persistence and adherence with all-cause and MS-related HCRU and non-DMT costs of patients with MS across 12 and 24 months of therapy use. Methods: This study was a retrospective analysis of adult patients with MS in the IBM MarketScan Commercial and Medicare Supplemental databases using claims data between April 2016 and December 2019. The index date was defined as the initiation of the DMT. Patients were required to have ≥12 months' continuous enrollment pre-index and ≥12 or ≥24 months' continuous enrollment post-index. Persistence was defined as no gap in DMT supply for ≥60 days within the post-index period or switch to another DMT. Adherence was calculated using the proportion of days covered (for this study, number of days covered by the DMT was 365 or 730 days), with ≥80% proportion of days covered considered adherent. Multivariable analyses were conducted to estimate total and individual components of non-DMT costs by persistence and adherence while controlling for baseline differences. Results: Patients who were persistent with medication for 12 months showed a reduction in mean total non-DMT medical costs of $10 022 compared with nonpersistent patients; these savings nearly doubled ($19 230) after 24 months of persistence. A similar pattern was observed for adherent vs nonadherent patients (reduction in costs at 12 months, $8543; at 24 months, $16 091). The largest reduction in all-cause HCRU costs was observed in the inpatient setting, while the largest reduction in MS-related costs was observed in the outpatient setting. Discussion: Patients with MS who were persistent and adherent to medication had substantially lower all-cause and MS-related non-DMT medical costs compared with those who were nonpersistent or nonadherent. Conclusions: These findings further support the importance of persistence and adherence to DMTs in patients with MS.

Adherence to and Persistence with Disease-Modifying Therapies for Multiple Sclerosis Over 24 Months: A Retrospective Claims Analysis.

INTRODUCTION: We sought to assess adherence to and persistence with ocrelizumab (OCR) compared with other disease-modifying treatments (DMTs), by route of administration (RoA), for multiple sclerosis (MS) after 24 months in the United States. METHODS: This retrospective claims analysis of MS patients initiating a new DMT was conducted using the IBM MarketScan Commercial and Medicare Supplemental databases between April 2016 and December 2019. Continuous enrollment of ≥ 12 months before and up to 24 months after initiating the index DMT was required. Adherence was assessed based on proportion of days covered (PDC) in the follow-up period with values ≥ 80% considered adherent. Persistence was defined as no evidence of switching to another DMT or no gap ≥ 60 days in DMT coverage. RESULTS: A total of 1710 patients with ≥ 24 months of follow-up (OCR, n = 524; oral, n = 701; injectable, n = 365; other intravenous [IV], n = 120) were included. Patients initiating OCR had higher adherence (80% vs. 55%, 35%, and 54% for oral, injectable, and other IV, respectively) and persistence (75% vs. 54%, 33%, and 55%, respectively) at 24 months. Relative risks (RRs) of 24-month non-adherence for those initiating orals, injectables, and other IVs were 2.2 (95% CI, 1.7-2.9), 3.0 (95% CI, 2.2-4.0), and 2.2 (95% CI, 1.5-3.3), respectively, compared to those initiating OCR. Similarly, patients receiving orals, injectables, and other IVs had RR of 1.9 (95% CI, 1.4-2.4), 2.5 (95% CI, 1.9-3.4), and 1.8 (95% CI, 1.2-2.6) for 24-month discontinuation, respectively. Similar patterns were observed at 12 and 18 months. CONCLUSIONS: Patients initiating OCR in a real-world setting achieved higher rates of adherence and persistence at 24 months compared with those initiating other DMTs, consistent with published literature showing similar results at 12 and 18 months. Optimizing medication adherence and persistence is fundamental to MS care, so clinicians should consider all elements of DMTs that may improve compliance.

Cardiovascular Outcomes Among Patients with Type 2 Diabetes Newly Initiated on Sodium-Glucose Cotransporter-2 Inhibitors, Glucagon-Like Peptide-1 Receptor Agonists, and Other Antidiabetic Medications.

BACKGROUND: Cardiovascular disease (CVD) remains the most prevalent cause of morbidity and mortality in patients with type 2 diabetes (T2D) and is a primary driver for health care costs associated with diabetes management. Sodium-glucose cotransporter-2 inhibitors (SGLT-2is) and glucagon-like peptide-1 receptor agonists (GLP-1 RAs) have demonstrated significant reductions in cardiovascular endpoints in clinical trials compared with placebo. However, it is uncertain whether these findings can be applied to the broader T2D population because these trials specifically included high-risk patients with established CVD. OBJECTIVE: To evaluate and compare cardiovascular outcomes among adults with T2D newly initiated on SGLT-2is, GLP-1 RAs, and other antidiabetic medications (oADMs) in a real-world setting. METHODS: This retrospective new-user cohort study used administrative claims and electronic health record data from an integrated delivery network in Texas. Patients aged ≥18 years with T2D and ≥1 prescription claim for an SGLT-2i, a GLP-1 RA, or an oADM filled between April 2013 and December 2018 were included. Patients were divided into three 1:1 propensity-matched groups according to index medication identified. Primary outcomes were heart failure hospitalization and a composite end-point of myocardial infarction, stroke, unstable angina, or coronary revascularization. Cox proportional hazards regression was used to compare cumulative incidence of all outcome variables. RESULTS: Among 9,477 patients, 1,134 were initiated on SGLT-2is, 1,072 on GLP-1 RAs, and 7,271 on oADMs. Patients initiating SGLT-2is versus oADMs had significantly lower risk of the composite endpoint (HR = 0.64, 95% CI = 0.46-0.90), heart failure hospitalization (HR = 0.56, 95% CI = 0.39-0.81), and unstable angina requiring hospitalization (HR = 0.56, 95% CI = 0.39-0.81). Patients initiating GLP-1 RAs compared with oADMs had significantly lower risk of the composite endpoint (HR = 0.71, 95% CI = 0.52-0.98) and unstable angina requiring hospitalization (HR = 0.60, 95% CI = 0.41-0.86). No differences in cardiovascular outcomes were found between SGLT-2is and GLP-1 RAs. CONCLUSIONS: Both SGLT-2is and GLP-1 RAs showed significant reductions in the composite outcome and unstable angina requiring hospitalization versus oADMs. However, only SGLT-2is were associated with a lower risk for heart failure hospitalizations. Nevertheless, cardiovascular outcomes were similar between SGLT-2is and GLP-1 RAs. DISCLOSURES: No outside funding supported this study. The authors have no conflicts of interest to report.