• Cost of non-alcoholic steatohepatitis in Europe and the USA: The GAIN study.

      O'Hara, Jamie; Finnegan, Alan; Dhillon, Harpal; Ruiz-Casas, Leonardo; Pedra, Gabriel; Franks, Bethany; Morgan, George; Hebditch, Vanessa; Jönsson, Bengt; Mabhala, Mzwandile; et al. (2020-07-15)
      Non-alcoholic steatohepatitis (NASH) leads to cirrhosis and is associated with a substantial socioeconomic burden, which, coupled with rising prevalence, is a growing public health challenge. However, there are few real-world data available describing the impact of NASH. The Global Assessment of the Impact of NASH (GAIN) study is a prevalence-based burden of illness study across Europe (France, Germany, Italy, Spain, and the UK) and the USA. Physicians provided demographic, clinical, and economic patient information via an online survey. In total, 3,754 patients found to have NASH on liver biopsy were stratified by fibrosis score and by biomarkers as either early or advanced fibrosis. Per-patient costs were estimated using national unit price data and extrapolated to the population level to calculate the economic burden. Of the patients, 767 (20%) provided information on indirect costs and health-related quality of life using the EuroQOL 5-D (EQ-5D; n = 749) and Chronic Liver Disease Questionnaire - Non-Alcoholic Fatty Liver Disease (CLDQ-NAFLD) (n = 723). Mean EQ-5D and CLDQ-NAFLD index scores were 0.75 and 4.9, respectively. For 2018, the mean total annual per patient cost of NASH was €2,763, €4,917, and €5,509 for direct medical, direct non-medical, and indirect costs, respectively. National per-patient cost was highest in the USA and lowest in France. Costs increased with fibrosis and decompensation, driven by hospitalisation and comorbidities. Indirect costs were driven by work loss. The GAIN study provides real-world data on the direct medical, direct non-medical, and indirect costs associated with NASH, including patient-reported outcomes in Europe and the USA, showing a substantial burden on health services and individuals. There has been little research into the socioeconomic burden associated with non-alcoholic steatohepatitis (NASH). The GAIN study provides real-world data on the direct medical, direct non-medical, and indirect costs associated with NASH, including patient-reported outcomes in five European countries (UK, France, Germany, Spain, and Italy) and the USA. Mean total annual per patient cost of NASH was estimated at €2,763, €4,917, and €5,509 for the direct medical, direct non-medical, and indirect cost categories, respectively. [Abstract copyright: © 2020 The Author(s).]
    • Examining patient and professional perspectives in the UK for gene therapy in haemophilia.

      Woollacott, Ione; Morgan, George; orcid: 0000-0003-2014-3415; Chowdary, Pratima; O'Hara, Jamie; Franks, Bethany; van Overbeeke, Eline; orcid: 0000-0003-0073-9350; Dunn, Nicola; Michelsen, Sissel; Huys, Isabelle; Martin, Antony; orcid: 0000-0003-4383-6038; et al. (2022-04-19)
      With the development of gene therapy for people with haemophilia (PWH), it is important to understand how people impacted by haemophilia (PIH) and clinicians prioritise haemophilia treatment attributes to support informed treatment decisions. To examine the treatment attribute preferences of PIH and clinical experts in the United Kingdom (UK) and to develop a profile of gene therapy characteristics fit for use in future discrete choice experiments (DCEs). Semi-structured interviews were conducted with PIH (n = 14) and clinical experts (n = 6) who ranked pre-defined treatment attributes by importance. Framework analysis was conducted to identify key themes and treatment attributes; points were allocated based on the rankings. Synthesis of results by a multidisciplinary group informed development of a profile of gene therapy characteristics for use in future research. Key themes identified by PIH and clinical experts included patient relevant features and the importance of 'informed decision making'. The six top-ranked treatment attributes were 'effect on factor level' (79 points), 'uncertainty regarding long-term risks' (57 points), 'impact on daily life' (41 points), 'frequency of monitoring' (33 points), 'impact on ability to participate in physical activity' (29 points), and 'uncertainty regarding long-term benefits' (28 points). The final treatment characteristics were categorised as therapeutic option, treatment effectiveness, safety concerns, impact on self-management and quality of life (role limitations). We identified several gene therapy characteristics important to PIH and clinicians in the UK. These characteristics will be used in a future DCE to further investigate patient preferences for gene therapy. [Abstract copyright: © 2022 John Wiley & Sons Ltd.]
    • The Cost of Von Willebrand Disease in Europe: The CVESS Study

      Morgan, George; Brighton, Sarah; Laffan, Mike; Goudemand, Jenny; Franks, Bethany; Finnegan, Alan (SAGE Publications, 2022-08-17)
      Background: Von Willebrand disease (VWD) is one of the most common inherited bleeding disorders, imposing a substantial health impact and financial burden. The Cost of von Willebrand disease in Europe: A Socioeconomic Study (CVESS) characterises the socio-economic cost of VWD across Germany, Spain, Italy, France, and the UK. Methods: A retrospective, cross-sectional design captured 12 months of patient disease management, collected from August-December 2018, for 974 patients. This enabled estimation of direct medical, direct non-medical and indirect costs, utilising prevalence estimates to extrapolate to population level. Results: Total annual direct medical cost (including/excluding von Willebrand factor [VWF]) across all countries was the highest cost (€2 845 510 345/€444 446 023), followed by indirect costs (€367 330 271) and direct non-medical costs (€60 223 234). Differences were seen between countries: the UK had the highest direct medical costs excluding VWF (€159 791 064), Italy the highest direct-non medical (€26 564 496), and Germany the highest indirect cost burden (€197 036 052). Total direct medical costs per adult patient increased across VWD types with Type 1 having the lowest cost (€23 287) and Type 3 having the highest cost (€133 518). Conclusion: A substantial financial burden arises from the prevalence of VWD for the European healthcare systems considered.