Cost-Utility Analysis of Ravulizumab Compared with Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria.

BACKGROUND: Paroxysmal nocturnal hemoglobinuria, characterized by intravascular hemolysis and venous thrombosis, can be managed with eculizumab, an inhibitor of the complement system; however, patients may periodically experience breakthrough hemolysis. Ravulizumab is a newly approved treatment for paroxysmal nocturnal hemoglobinuria that may reduce breakthrough hemolysis risk, thus improving health-related quality of life and reducing treatment costs. OBJECTIVE: The objective of this study was to compare the costs and benefit of treatment with ravulizumab vs eculizumab in adult patients with paroxysmal nocturnal hemoglobinuria, from a US payer perspective. METHODS: A cost-utility analysis was conducted using a semi-Markov model, informed by clinical experts. Lifetime costs and benefit (quality-adjusted life-years) (both discounted at 3% per annum) and incremental cost-effectiveness ratios were estimated, over a lifetime horizon. Results are reported for an entire treated population and subgroups of eculizumab treatment history. Scenario analyses were characterized by assumptions of non-inferiority between treatments, in terms of breakthrough hemolysis incidence and blood transfusion requirements, and of variations in eculizumab dosing adjustments used in response to breakthrough hemolysis. RESULTS: In the base-case analysis for the overall population, there was a positive impact on health-related quality of life (quality-adjusted life-year gain of 1.67) and costs were lower (- $1,673,465), for ravulizumab vs eculizumab. This led to a negative incremental cost-effectiveness ratio (- $1,000,818, indicating cost savings per quality-adjusted life-year gained). Health-related quality-of-life improvement and cost savings were also observed in all cohorts and scenario analyses. CONCLUSIONS: In adults with paroxysmal nocturnal hemoglobinuria, ravulizumab is associated with improved health-related quality of life and provides a large cost saving from the perspective of a US payer, when compared with eculizumab.

"My bitterness is deeper than the ocean": understanding internalized stigma from the perspectives of persons with schizophrenia and their family caregivers.

BACKGROUND: It is estimated that 8 million of the Chinese adult population had a diagnosis of schizophrenia. Stigma associated with mental illness, which is pervasive in the Chinese cultural context, impacts both persons with schizophrenia and their family caregivers. However, a review of the literature found a dearth of research that explored internalized stigma from the perspectives of both patients and their caregivers. METHODS: We integrated data from standardized scales and narratives from semi-structured interviews obtained from eight family-dyads. Interview narratives about stigma were analyzed using directed content analysis and compared with responses from Chinese versions of the Internalized Stigma of Mental Illness Scale and Affiliated Stigma Scale. Scores from the two scales and number of text fragments were compared to identify consistency of responses using the two methods. Profiles from three family-dyads were analyzed to highlight the interactive aspect of stigma in a dyadic relationship. RESULTS: Our analyses suggested that persons with schizophrenia and their caregivers both internalized negative valuation from their social networks and reduced engagement in the community. Participants with schizophrenia expressed a sense of shame and inferiority, spoke about being a burden to their family, and expressed self-disappointment as a result of having a psychiatric diagnosis. Caregivers expressed high level of emotional distress because of mental illness in the family. Family dyads varied in the extent that internalized stigma were experienced by patients and caregivers. CONCLUSIONS: Family plays a central role in caring for persons with mental illness in China. Given the increasingly community-based nature of mental health services delivery, understanding internalized stigma as a family unit is important to guide the development of cultural-informed treatments. This pilot study provides a method that can be used to collect data that take into consideration the cultural nuances of Chinese societies.