No abstract

BACKGROUND: Darolutamide, a structurally distinct androgen receptor inhibitor approved for the treatment of men with nonmetastatic castration-resistant prostate cancer (nmCRPC), has been shown to increase metastasis-free survival among men with nmCRPC compared with placebo. This treatment has a novel chemical structure that may also have safety, tolerability, and efficacy advantages for men with nmCRPC. OBJECTIVE:To estimate the projected budget impact of including darolutamide on a U.S. payer formulary as a treatment option for men with nmCRPC.

Aim Haemophilia A (HA) is a male‐predominant disorder, yet women and girls can have factor VIII (FVIII) deficiency with bleeding events requiring treatment. This study aimed to identify and characterize female patients with HA. Methods Administrative claims dated 01 January 2012‐31 July 2016 were accessed for patients with 18 months’ coverage by commercial or Medicare Advantage with Part D insurance. Patients were included by HA diagnoses or treatments and/or bleeding‐related diagnoses or procedures, and excluded by haemophilia B or qualitative platelet disorder diagnoses. A sample of charts was examined for bleeding history, HA therapies and bleeding treatments. All‐cause healthcare utilization and costs were also described. Results Among 353 patients meeting initial inclusion criteria, 86 charts were procured, with 8 patients identified as having HA. Their mean age was 60 ± 17 years and most were Medicare‐insured. The mean Charlson Comorbidity Index score was 2.50 ± 2.56; the most prevalent comorbid conditions involved coagulation/haemorrhage, fluid/electrolyte balance and non‐traumatic joint disorders. Over 18 months, a mean of 54 ambulatory visits and 120 pharmacy fills were observed; mean medical costs were $86 694 and pharmacy costs were $25 396. Conclusions Identifying females with HA is challenging using healthcare claims, because diagnostic nomenclature is unclear for female patients treated for bleeding events. Although chart abstraction enhanced claims data, very few female patients were identified with HA. Nevertheless, even in a small sample, sizeable burden in comorbidity and healthcare use was observed. Improved nomenclature and coding for HA diagnoses for women and girls is key to improving research and treatment.

S105VALIDATION OF THE FINE-CKD MODEL placebo. 8 Based on the FIDELITY results, on top of the maximum tolerated renin-angiotensin system inhibitor (RASi) treatment, finerenone showed substantial benefits for a range of CV and renal outcomes. 11

Introduction: It is important to achieve good persistence and adherence to disease-modifying therapies (DMTs) to achieve the best outcomes in chronic diseases such as multiple sclerosis (MS). The BETACONNECT device is an electronic auto-injector for the DMT interferon beta-1b (Betaseron), designed to improve patients' injection experience and to monitor adherence. This observational study aimed to assess patient adherence to and persistence with interferon beta-1b therapy as well as patientreported satisfaction in a US population. Methods: A prospective, observational, multicenter study was conducted in 146 adult patients with relapsing-remitting MS or clinically isolated syndrome, newly prescribed or currently established on interferon beta-1b therapy and naı ¨ve to the BETACONNECT device, and followed up during a 6-month observation period. Results: Among the 91 patients who completed the study, the overall mean adherence rate was 82.5%, with 65.9% of patients adherent for at least 80% for the duration of the 6-month period. At 6 months, 98.9% of patients had less than a 60-day gap in therapy. Of the 115 patients who provided satisfaction data, 90.5% of patients were either very satisfied or satisfied with the BETACONNECT device. Conclusion:This study shows that the BETA-CONNECT device was associated with high adherence to interferon beta-1b therapy in patients with MS. Patients also reported high degrees of satisfaction with the device. Therefore, this may be a viable delivery option to help with adherence and persistence, potentially leading to improved clinical outcomes.