• Adherence and a Potential Trade-Off Currently Faced in Optimizing Hemophilia Treatment

      Burke, Tom; Asghar, Sohaib; Misciattelli, Natalia; Kar, Sharmila; Morgan, George; Dhillon, Harpal; O'Hara, Jamie (American Society of Hematology, 2020-11-05)
      INTRODUCTION Severe hemophilia, i.e., <1% normal FVIII level (A) or FIX level (B), are congenital bleeding disorders characterized by uncontrolled bleeding. The clinical benefits of prophylactic FVIII/IX replacement therapy are well understood, but require adherence to a schedule of routine infusions. Optimal adherence is associated with better joint outcomes and lower rates of chronic pain. Nonetheless a lack of patient-reported data has to date limited our understanding of the patient burden associated with adherence to treatment, and the relationship between adherence and the ability to work, among people living with hemophilia in the US. Data from the Bridging Hemophilia B Experiences, Results and Opportunities into Solutions (B-HERO-S) study reported a high proportion of adults with hemophilia B receiving routine infusions (at least one infusion per month), showing a negative impact on their ability to work, and people receiving routine infusions were more likely than people treated on-demand to report an inability to work in most situations. The ability of people living with hemophilia to participate in the labor force, without barriers to job choice or working hours, is a key outcome in the drive to achieve health equity. The objective of the analysis is to examine the relationship between adherence and the labor force participation of people with severe hemophilia in the US. METHODS This analysis draws data from a patient-reported study, the 'Cost of Severe Hemophilia Across the US: A Socioeconomic Survey' (CHESS US+). Conducted in 2019, the CHESS US+ study is a cross-sectional patient-centered study of adults with severe hemophilia in the US. A patient-completed questionnaire collected data on clinical, economic, and humanistic outcomes, for a 12-month retrospective period. This analysis examines labor force participation and employment status (full-time, part-time, unemployed, retired) and chronic pain categorized by 'none', low-level ('1-5'), and high-level ('6-10'). The analysis was stratified by adherence to treatment, self-reported on a 1-10 scale, from "not at all" to "fully", categorized into low (1-6), moderate (7-9) and full (10) adherence. Results are presented as mean (standard deviation) or N (%). RESULTS The analysis comprised 356 people with severe hemophilia A (73%) and B (27%) who participated in CHESS US+ study. In Table 1, the baseline characteristics of the study population are stratified by full adherence (N = 119), moderate adherence (N=134) and low adherence (N=103). Having no chronic pain was most prevalent in the full adherence group (37.7%), compared to moderate (8.3%) or low (13.9%) adherence cohorts. Chronic pain, both low- and high-levels were least prevalent among people with full adherence. Moreover, people with low adherence were disproportionately more likely to have high-levels of chronic pain relative to moderate adherence or full adherence (Table 1). Unemployment, however, was highest in full adherence (21.1%), and people with full adherence were also least likely to be in full-time employment (42%). The full-time employment rate decreased as adherence declined from full to moderate (Table 1), and was comparable in people with low adherence (57.3%) or moderate adherence (54.5%). CONCLUSIONS This analysis of CHESS US+ examined the complex relationship between labor market outcomes and adherence to treatment, among adults with severe hemophilia in the US. Adherence was associated with lower rates of chronic pain, representing the importance of achieving an optimal treatment strategy. Nonetheless, patients achieving optimal adherence were less likely to be in full-time employment, and more likely to be part-time or unemployed, comparatively. Together, these data characterize a trade-off in clinical outcomes versus workforce participation, and suggest that the goal of achieving health equity may currently still be unmet. Disclosures Burke: HCD Economics: Current Employment; University of Chester: Current Employment; F. Hoffmann-La Roche Ltd: Consultancy. Asghar:HCD Economics: Current Employment. Misciattelli:Freeline: Current Employment, Current equity holder in publicly-traded company. Kar:Freeline: Current Employment, Current equity holder in publicly-traded company. Morgan:uniQure: Consultancy; HCD Economics: Current Employment. Dhillon:HCD Economics: Current Employment; F. Hoffmann-La Roche Ltd: Other: All authors received editorial support for this abstract, furnished by Scott Battle, funded by F. Hoffmann-La Roche Ltd, Basel, Switzerland. . O'Hara:HCD Economics: Current Employment, Current equity holder in private company; F. Hoffmann-La Roche Ltd: Consultancy.
    • Adherence and a Potential Trade-Off Currently Faced in Optimizing Hemophilia Treatment

      Burke, Tom; Asghar, Sohaib; Misciattelli, Natalia; Kar, Sharmila; Morgan, George; Dhillon, Harpal; O'Hara, Jamie (Elsevier, 2021-08-03)
      INTRODUCTION Severe hemophilia, i.e., <1% normal FVIII level (A) or FIX level (B), are congenital bleeding disorders characterized by uncontrolled bleeding. The clinical benefits of prophylactic FVIII/IX replacement therapy are well understood, but require adherence to a schedule of routine infusions. Optimal adherence is associated with better joint outcomes and lower rates of chronic pain. Nonetheless a lack of patient-reported data has to date limited our understanding of the patient burden associated with adherence to treatment, and the relationship between adherence and the ability to work, among people living with hemophilia in the US. Data from the Bridging Hemophilia B Experiences, Results and Opportunities into Solutions (B-HERO-S) study reported a high proportion of adults with hemophilia B receiving routine infusions (at least one infusion per month), showing a negative impact on their ability to work, and people receiving routine infusions were more likely than people treated on-demand to report an inability to work in most situations. The ability of people living with hemophilia to participate in the labor force, without barriers to job choice or working hours, is a key outcome in the drive to achieve health equity. The objective of the analysis is to examine the relationship between adherence and the labor force participation of people with severe hemophilia in the US. METHODS This analysis draws data from a patient-reported study, the ‘Cost of Severe Hemophilia Across the US: A Socioeconomic Survey’ (CHESS US+). Conducted in 2019, the CHESS US+ study is a cross-sectional patient-centered study of adults with severe hemophilia in the US. A patient-completed questionnaire collected data on clinical, economic, and humanistic outcomes, for a 12-month retrospective period. This analysis examines labor force participation and employment status (full-time, part-time, unemployed, retired) and chronic pain categorized by ‘none’, low-level ('1-5'), and high-level ('6-10'). The analysis was stratified by adherence to treatment, self-reported on a 1-10 scale, from “not at all” to “fully”, categorized into low (1-6), moderate (7-9) and full (10) adherence. Results are presented as mean (standard deviation) or N (%). RESULTS The analysis comprised 356 people with severe hemophilia A (73%) and B (27%) who participated in CHESS US+ study. In Table 1, the baseline characteristics of the study population are stratified by full adherence (N = 119), moderate adherence (N=134) and low adherence (N=103). Having no chronic pain was most prevalent in the full adherence group (37.7%), compared to moderate (8.3%) or low (13.9%) adherence cohorts. Chronic pain, both low- and high-levels were least prevalent among people with full adherence. Moreover, people with low adherence were disproportionately more likely to have high-levels of chronic pain relative to moderate adherence or full adherence (Table 1). Unemployment, however, was highest in full adherence (21.1%), and people with full adherence were also least likely to be in full-time employment (42%). The full-time employment rate decreased as adherence declined from full to moderate (Table 1), and was comparable in people with low adherence (57.3%) or moderate adherence (54.5%). CONCLUSIONS This analysis of CHESS US+ examined the complex relationship between labor market outcomes and adherence to treatment, among adults with severe hemophilia in the US. Adherence was associated with lower rates of chronic pain, representing the importance of achieving an optimal treatment strategy. Nonetheless, patients achieving optimal adherence were less likely to be in full-time employment, and more likely to be part-time or unemployed, comparatively. Together, these data characterize a trade-off in clinical outcomes versus workforce participation, and suggest that the goal of achieving health equity may currently still be unmet. Disclosures Burke: HCD Economics: Current Employment; University of Chester: Current Employment; F. Hoffmann-La Roche Ltd: Consultancy. Asghar: HCD Economics: Current Employment. Misciattelli: Freeline: Current Employment, Current equity holder in publicly-traded company. Kar: Freeline: Current Employment, Current equity holder in publicly-traded company. Morgan: uniQure: Consultancy; HCD Economics: Current Employment. Dhillon: HCD Economics: Current Employment; F. Hoffmann-La Roche Ltd: Other: All authors received editorial support for this abstract, furnished by Scott Battle, funded by F. Hoffmann-La Roche Ltd, Basel, Switzerland. . O'Hara: HCD Economics: Current Employment, Current equity holder in private company; F. Hoffmann-La Roche Ltd: Consultancy.
    • Evidence of a Hemophilia Employment Gap: Comparing Data from CHESS US+ and the 2019 Current Population Survey

      Asghar, Sohaib; Burke, Tom; Misciattelli, Natalia; Kar, Sharmila; Morgan, George; O'Hara, Jamie (Elsevier, 2021-08-03)
      INTRODUCTION Severe hemophilia A (<1% normal FVIII activity) and B (<1% normal FIX activity) are congenital bleeding disorders characterized by uncontrolled bleeding, either spontaneously or in response to trauma or surgery. Recent commentary has identified a number of patient-important and patient-relevant outcomes that have been understudied, namely the challenges faced by people living with hemophilia to participate in the labor force. The socio-economic impact of hemophilia is comparatively less well understood than clinical outcomes and therapy-related costs. Under-employment and under-utilization have long-term consequences to individuals' job prospects and psychosocial health, as well as an economic cost to the society. The objective of the analysis is to compare labor market participation, among people with severe hemophilia from the US and the general population. This analysis draws on household data derived from the 2019 Current Population Survey (CPS), and on patient-reported data from a patient-centric study conducted in 2019 of people with severe hemophilia, in the US: the ‘Cost of Severe Hemophilia Across the US: A Socioeconomic Survey’ (CHESS US+). METHODS A patient-centric framework informed the design of CHESS US+ a retrospective (12 months prior to study enrollment), cross-sectional dataset of adults with severe hemophilia in the US. Conducted in 2019, the study used a patient-completed questionnaire to collect data on patient-relevant clinical, economic, and humanistic outcomes. This analysis examines labor market participation (full-time, part-time, unemployed), and corresponding general population data derived from the 2019 Current Population Survey (CPS). Data on the general population were sourced from the 2019 CPS ‘Employment status of the civilian noninstitutional population’. Persons ‘not in the labor force’ in the 2019 CPS and retired persons in CHESS US+ were not included in the analysis. We present data on the civilian labor force, in CHESS US+ and in the 2019 CPS. Results are presented as mean (standard deviation) or N (%). RESULTS Of 356 patients profiled in the CHESS US+ study, 97 (27%) had severe hemophilia B and 257 (73%) had severe hemophilia A. Mean age and weight (kg) of the cohort was 34.99 (12.15) and 85.71 (22.81), respectively. The labor force participation rates of non-retired people with severe hemophilia in CHESS US+ (N = 340) and the general population (161,458) are described in Table 1. Examining aggregate data on employment status observed a higher proportion of people with severe hemophilia in part-time employment (24.4% vs. 15.7%). Differences in the labor force participation of people living with severe hemophilia compared to the general population were most pronounced in the full-time employment rate and the unemployment rate. Compared to 80.7% of the general population (Table 1), only 53.5% of people with severe hemophilia in CHESS US+ had a full-time job. Moreover, the unemployment rate (Table 1) in the 2019 CPS compared with the rate observed in CHESS US+ (3.7% vs. 22.1%) provides a stark contrast in the employment experiences of people living with severe hemophilia relative to the general population. CONCLUSIONS This analysis of CHESS US+ illustrates the impact of severe hemophilia on labor force participation. People with severe hemophilia were more likely than the general population to be unemployed, or in part-time employment. A notable contrast was observed in the rate of full-time employment and unemployment, among the general population compared to people living with severe hemophilia. These data illustrate the need to quantify the impact of hemophilia using a holistic approach that considers the cost of involuntary illness-related part-time and unemployment. Disclosures Asghar: HCD Economics: Current Employment. Burke: HCD Economics: Current Employment; F. Hoffmann-La Roche Ltd: Consultancy; University of Chester: Current Employment. Misciattelli: Freeline: Current Employment, Current equity holder in publicly-traded company. Kar: Freeline: Current Employment, Current equity holder in publicly-traded company. Morgan: HCD Economics: Current Employment; uniQure: Consultancy. O'Hara: F. Hoffmann-La Roche Ltd: Consultancy; HCD Economics: Current Employment, Current equity holder in private company.