BACKGROUND: Ocrelizumab (OCR) is the only disease-modifying therapy (DMT) for both relapsing and primary progressive forms of multiple sclerosis (MS). OCR is given by intravenous (IV) infusion twice a year, which may improve adherence to the dosing schedule relative to other MS DMTs that require more frequent administration. Real-world evidence on the persistence and adherence of patients with MS to OCR compared with other DMTs is limited.

Background The extent to which different US private insurers require their enrollees to meet the same coverage criteria before gaining access to treatment is unclear. Our objective was to scrutinize the patient access criteria imposed by US private insurers for a set of rare neuromuscular disease (NMD) disease-modifying therapies (DMTs). Methods We examined coverage policies issued by 17 large US private insurers for the following NMD treatments: nusinersen and onasemnogene abeparvovec for spinal muscular atrophy, edaravone for amyotrophic lateral sclerosis, and eteplirsen for Duchenne muscular dystrophy. We reviewed the plans’ coverage policies and identified the patient access criteria, including clinical prerequisites, step therapy protocols, and prescriber requirements. We compared the plans’ patient access criteria with the therapies’ US Food and Drug Administration (FDA)-labeled indications. Results The included insurers issued 65 coverage policies for the included therapies. Plans imposed coverage restrictions beyond the FDA-approved indications in 60 coverage policies; plans did not cover eteplirsen in five policies. No therapy was covered the same way by all insurers. Plans applied clinical criteria beyond the FDA label indication in 56 policies and step therapy protocols in three policies. Plans required that a neurologist prescribe the therapy in 37 policies, 22 of which required the neurologist to have expertise in the particular disease. Plans often required patients to suffer from symptoms of particular severity; e.g. for eteplirsen, plans differed in their 6-min walk test requirements; for edaravone, some plans required that patients had normal respiratory function, while others required only that patients did not require ventilation; for nusinersen and onasemnogene abeparvovec, plans differed in the number of SMN2 gene copies they required patients to have (SMN2 copy number is correlated with disease severity). Conclusions The evaluated large US private insurers tended to impose coverage restrictions beyond the FDA label indication for the included set of rare NMD DMTs. Plans rarely applied the same patient access criteria in their coverage policies for the same products. Inconsistent coverage criteria mean that patients with different insurers have variable access to the same therapies across insurers.

Implementation of an institutional guideline and order set for hyperglycemic emergencies decreased ICU LOS and time to anion gap closure, with no difference in rates of hypoglycemia.

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 C 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 followup period with values C 80% considered adherent. Persistence was defined as no evidence of switching to another DMT or no gap C 60 days in DMT coverage.Results: A total of 1710 patients with C 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 realworld 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.

Background Spinal muscular atrophy (SMA) is a genetic debilitating disease affecting approximately 10,000 individuals in the United States. Individuals with SMA frequently require caregiver support and care. Through a partnership with a large patient organization, we surveyed caregivers of individuals with SMA below 18 years of age to understand the impact of SMA on caregivers with respect to their daily activities and health-related quality of life (HRQoL). In addition to structured questions, a standardized HRQoL instrument, the EQ-5D-5L and visual analogue scale, were administered. Results The caregiver sample consisted of 45 unpaid caregivers of individuals with SMA. Of them, 22% reported that they were sole caregivers that received no additional caregiving support and 98% were parents of an affected individual. The majority of caregivers cared for individuals with type 2 (58%), followed by type 1 (38%) and type 3 (4%) SMA. Sixty-four percent of the individuals with SMA were able to sit without support or better, while 31% had some motor function and 5% reported no motor function. On average, caregivers reported spending 136 hours/month managing the overall treatment, care, and support for the affected individual. Most of the individuals (91%) were reported to have received nusinersen; caregivers reported spending 29 hours managing treatment in a typical month. Caregiver time investment correlated directly with disease severity measured by both SMA type and patient motor function level. On average, caregivers rated their overall health as 76 on a scale of 0-100 using the EQ-5D-5L HRQoL visual analogue scale. Specifically, 42% of caregivers reported any inability to do their usual activities and 73% reported any anxiety or depression. Conclusions SMA negatively affects caregiver’s daily activities and HRQoL and represents a substantial burden. Disease severity is associated with an increasing amount of time required for care and support for patients with SMA and caregiver’s own HRQoL. As treatments become available, economic evaluation of these treatments should include effects on the family as well.