• Adult lifetime cost of hemophilia B management in the US: payer and societal perspectives from a decision analytic model

      Li, Nanxin; Sawyer, Eileen K.; Maruszczyk, Konrad; orcid: 0000-0002-0173-5020; Guzauskas, Greg; orcid: 0000-0002-9095-1672; Slomka, Marta T.; Burke, Tom; Martin, Antony P.; orcid: 0000-0003-4383-6038; O’Hara, Jamie; orcid: 0000-0001-8262-034X; Stevenson, Matt; orcid: 0000-0002-3099-9877; Recht, Michael; orcid: 0000-0002-2805-1016 (Informa UK Limited, 2021-03-02)
    • Adult lifetime cost of hemophilia B management in the US: Payer and societal perspectives from a decision analytic model

      Li, Nanxin; Sawyer, Eileen K.; Maruszczyk, Konrad; orcid: 0000-0002-0173-5020; Guzauskas, Greg; orcid: 0000-0002-9095-1672; Slomka, Marta T.; Burke, Tom; Martin, Antony P.; O’Hara, Jamie; Stevenson, Matt; Recht, Michael (Informa UK Limited, 2021-02-16)
    • Adult lifetime cost of hemophilia B management in the US: payer and societal perspectives from a decision analytic model

      Li, Nanxin; Sawyer, Eileen K.; Maruszczyk, Konrad; orcid: 0000-0002-0173-5020; Guzauskas, Greg; orcid: 0000-0002-9095-1672; Slomka, Marta T.; Burke, Tom; Martin, Antony P.; orcid: 0000-0003-4383-6038; O’Hara, Jamie; orcid: 0000-0001-8262-034X; Stevenson, Matt; orcid: 0000-0002-3099-9877; Recht, Michael; orcid: 0000-0002-2805-1016 (Informa UK Limited, 2021-03-02)
    • Clinical, humanistic, and economic burden of severe haemophilia B in adults receiving factor IX prophylaxis: findings from the CHESS II real-world burden of illness study in Europe

      Burke, Tom; Asghar, Sohaib; orcid: 0000-0001-8276-0131; O’Hara, Jamie; Chuang, Margaret; Sawyer, Eileen K.; Li, Nanxin; email: n.li@uniqure.com (BioMed Central, 2021-12-20)
      Abstract: Background: Real-world studies of the burden of severe haemophilia B in the context of recent therapeutic advances such as extended half-life (EHL) factor IX (FIX) products are limited. We analysed data from the recent CHESS II study to better understand the clinical, humanistic, and economic burden of severe haemophilia B in Europe. Data from male adults with severe haemophilia B receiving prophylaxis were analysed from the retrospective cross-sectional CHESS II study conducted in Germany, France, Italy, Spain and the United Kingdom. Inhibitors were exclusionary. Patients and physicians completed questionnaires on bleeding, joint status, quality of life, and haemophilia-related direct and indirect costs (2019–2020). All outcomes were summarised using descriptive statistics. Results: A total of 75 CHESS II patients were eligible and included; 40 patients (53%) provided self-reported outcomes. Mean age was 36.2 years. Approximately half the patients were receiving EHL versus standard half-life (SHL) prophylaxis (44% vs 56%). Most patients reported mild or moderate chronic pain (76%) and had ≥ 2 bleeding events per year (70%), with a mean annualised bleed rate of 2.4. Mean annual total haemophilia-related direct medical cost per patient was €235,723, driven by FIX costs (€232,328 overall, n = 40; €186,528 for SHL, €290,620 for EHL). Mean annual indirect costs (€8,973) were driven by early retirement or work stoppage due to haemophilia. Mean quality of life (EQ-5D) score was 0.67. Conclusions: These data document a substantial, persistent real-world burden of severe haemophilia B in Europe. Unmet needs persist for these patients, their caregivers, and society.
    • Clinical, humanistic, and economic burden of severe hemophilia B in the United States: Results from the CHESS US and CHESS US+ population surveys

      Burke, Tom; Asghar, Sohaib; orcid: 0000-0001-8276-0131; O’Hara, Jamie; Sawyer, Eileen K.; Li, Nanxin; email: n.li@uniqure.com (BioMed Central, 2021-03-20)
      Abstract: Background: Hemophilia B is a rare congenital bleeding disorder that has a significant negative impact on patients’ functionality and health-related quality of life. The standard of care for severe hemophilia B in the United States is prophylactic factor IX replacement therapy, which incurs substantial costs for this lifelong condition. Accurate estimates of the burden of hemophilia B are important for population health management and policy decisions, but have only recently accounted for current management strategies. The ‘Cost of Severe Hemophilia across the US: a Socioeconomic Survey’ (CHESS US) is a cross-sectional database of medical record abstractions and physician-reported information, completed by hematologists and care providers. CHESS US+ is a complementary database of completed questionnaires from patients with hemophilia. Together, CHESS US and CHESS US+ provide contemporary, comprehensive information on the burden of severe hemophilia from the provider and patient perspectives. We used the CHESS US and CHESS US+ data to analyze the clinical, humanistic, and economic burden of hemophilia B for patients treated with factor IX prophylaxis between 2017 and 2019 in the US. Results: We conducted analysis to assess clinical burden and direct medical costs from 44 patient records in CHESS US, and of direct non-medical costs, indirect costs, and humanistic burden (using the EQ-5D-5L) from 57 patients in CHESS US+. The mean annual bleed rate was 1.73 (standard deviation, 1.39); approximately 9% of patients experienced a bleed-related hospitalization during the 12-month study period. Nearly all patients (85%) reported chronic pain, and the mean EQ-5D-5L utility value was 0.76 (0.24). The mean annual direct medical cost was $614,886, driven by factor IX treatment (mean annual cost, $611,971). Subgroup analyses showed mean annual costs of $397,491 and $788,491 for standard and extended half-life factor IX treatment, respectively. The mean annual non-medical direct costs and indirect costs of hemophilia B were $2,371 and $6,931. Conclusions: This analysis of patient records and patient-reported outcomes from CHESS US and CHESS US+ provides updated information on the considerable clinical, humanistic, and economic burden of hemophilia B in the US. Substantial unmet needs remain to improve patient care with sustainable population health strategies.
    • Evidence of a disability paradox in patient‐reported outcomes in haemophilia

      O’Hara, Jamie; Martin, Antony P.; Nugent, Diane; Witkop, Michelle; orcid: 0000-0003-0758-286X; Buckner, Tyler W.; Skinner, Mark W.; orcid: 0000-0002-0934-0680; O’Mahony, Brian; Mulhern, Brendan; Morgan, George; orcid: 0000-0003-2014-3415; Li, Nanxin; et al. (Wiley, 2021-02-17)
    • Evidence of a disability paradox in patient‐reported outcomes in haemophilia

      O’Hara, Jamie; Martin, Antony P.; Nugent, Diane; Witkop, Michelle; orcid: 0000-0003-0758-286X; Buckner, Tyler W.; Skinner, Mark W.; orcid: 0000-0002-0934-0680; O’Mahony, Brian; Mulhern, Brendan; Morgan, George; orcid: 0000-0003-2014-3415; Li, Nanxin; et al. (Wiley, 2021-02-17)
    • Evidence of a disability paradox in patient‐reported outcomes in haemophilia

      O’Hara, Jamie; Martin, Antony P.; Nugent, Diane; Witkop, Michelle; orcid: 0000-0003-0758-286X; Buckner, Tyler W.; Skinner, Mark W.; orcid: 0000-0002-0934-0680; O’Mahony, Brian; Mulhern, Brendan; Morgan, George; orcid: 0000-0003-2014-3415; Li, Nanxin; et al. (Wiley, 2021-02-17)
    • Patient preferences and priorities for haemophilia gene therapy in the US: A discrete choice experiment

      Witkop, Michelle; Morgan, George; orcid: 0000-0003-2014-3415; email: george.morgan@hcdeconomics.com; O'Hara, Jamie; Recht, Michael; Buckner, Tyler W.; Nugent, Diane; Curtis, Randall; orcid: 0000-0002-6859-6432; O'Mahony, Brian; orcid: 0000-0001-9780-6972; Skinner, Mark W.; orcid: 0000-0002-0934-0680; Mulhern, Brendan; et al. (2021-07-26)
      Abstract: Introduction: Gene therapy has shown promise in clinical trials for patients with haemophilia, but patient preference studies have focused on factor replacement treatments. Aim: We conducted a discrete choice experiment (DCE) to investigate the relative importance and differential preferences patients provide for gene therapy attributes. Methods: We surveyed male adults with haemophilia in the United States recruited from patient panels including the National Hemophilia Foundation Community Voices in Research platform using an online survey over 4 months in 2020/21. Participants indicated preferences for gene therapy attributes including dosing frequency/durability, effect on annual bleeding, uncertainty related to side effects, impact on daily activities, impact on mental health, and post‐treatment requirements. The relative importance of each attribute was analysed overall and for subgroups based on haemophilia type and severity. Results: A total of 183 males with haemophilia A (n = 120) or B (n = 63) were included. Half (47%) had severe haemophilia; most (75%) were White. Overall, participants gave effect on bleeding rate the greatest relative importance (31%), followed by dose frequency/durability (26%), uncertainty regarding safety issues (17%), and impact on daily activities (11%). Dose frequency/durability had the greatest importance for those with haemophilia B (35%). Conclusion: People with haemophilia prioritised reduced bleeding and treatment burden; the former was more important in haemophilia A and the latter in haemophilia B, followed by safety and impact on daily life in this DCE of gene therapy attributes. These findings and differences can inform clinical and health policy decisions to improve health equity for people with haemophilia.
    • Patient preferences and priorities for haemophilia gene therapy in the US: A discrete choice experiment

      Witkop, Michelle; Morgan, George; orcid: 0000-0003-2014-3415; O'Hara, Jamie; Recht, Michael; Buckner, Tyler W.; Nugent, Diane; Curtis, Randall; orcid: 0000-0002-6859-6432; O'Mahony, Brian; orcid: 0000-0001-9780-6972; Skinner, Mark W.; orcid: 0000-0002-0934-0680; Mulhern, Brendan; et al. (Wiley, 2021-07-26)
    • Patient preferences and priorities for haemophilia gene therapy in the US: A discrete choice experiment

      Witkop, Michelle; Morgan, George; orcid: 0000-0003-2014-3415; O'Hara, Jamie; Recht, Michael; Buckner, Tyler W.; Nugent, Diane; Curtis, Randall; orcid: 0000-0002-6859-6432; O'Mahony, Brian; orcid: 0000-0001-9780-6972; Skinner, Mark W.; orcid: 0000-0002-0934-0680; Mulhern, Brendan; et al. (Wiley, 2021-07-26)