Contributions
Abstract: PB1486
Type: Publication Only
Session title: Bone marrow failure syndromes incl. PNH - Clinical
Background
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease associated with major complications such as thrombotic events and impaired renal function. Prior to the introduction of C5 inhibitors in 2007, PNH had been fatal in about 35% of patients within 5 years of diagnosis; yet this fatality rate continues in countries without access to these medications. Worldwide access to the C5 inhibitor eculizumab is hindered by the unavailability of treatment, and in places where treatment is approved, cost of treatment, reimbursement issues, infrastructure, or patient restrictions may further impede access (Risitano et al, Am J Hematol. 2018; Risitano et al, Front Immunol. 2019). PNH treatment places a significant economic burden on healthcare systems. In many countries, this has resulted in negative health technology appraisals (HTAs), indicating that optimal care and resource utilization are not being achieved (Coyle et al, Med Decis Making. 2014). New therapies with more convenient modes of administration may improve how PNH is clinically treated and positively impact access to C5 inhibitors globally. Further, treatments that allow for home administration may reduce patient and healthcare systems costs. Robust data on the real-life burden and cost of PNH are needed to assess the impact of current therapies and establish a baseline for new therapeutic approaches. Data derived from this study, which to our knowledge is the first socioeconomic study in PNH, will be used to support HTA processes and inform the value of new PNH therapies.
Aims
The COMMODORE burden of illness (BOI) study will quantify the direct medical costs (eg, treatment and hospitalization), direct nonmedical costs (eg, travel), and indirect costs (eg, impact on work productivity and family burden) associated with PNH for patients and care providers and determine the impact of PNH on health-related quality of life (HRQOL).
Methods
This is an international, prevalence-based, bottom-up, BOI study containing both retrospective and prospective data collection. The study will be overseen by an expert reference group consisting of multidisciplinary stakeholders. The study protocol and materials will be submitted for ethical approval to the University of Chester in the United Kingdom. Physicians will provide information on sociodemographic, clinical, and medical resource utilization using electronic case record forms (eCRFs). Through patient and public involvement and engagement, patients, after giving informed consent, will provide further information on the economic and HRQOL impact of PNH by completing patient-reported outcome surveys. Patients from France, Germany, the United Kingdom, and China will be included.
The aim is to recruit 94 physicians who will report 350 patient eCRFs with an expected return of 140 patient surveys and longitudinal data collection after 6-12 months for each patient. Mean per-patient costs, including direct medical and nonmedical costs, and indirect resource utilization will be calculated by multiplying the individual resource utilization by country-specific unit costs. National economic burden will be extrapolated by applying national prevalence estimates of PNH. Additionally, the impact of PNH on patient HRQOL will be assessed.
Results
Final results are expected in spring 2022.
Conclusion
The COMMODORE BOI study aims to characterize current PNH treatment via quantification of the humanistic and socioeconomic burden at the patient, healthcare system, and societal level to enhance the evidence base for treatment and decision-making in this community.
Keyword(s): Complement, Health care, Paroxysmal nocturnal hemoglobinuria (PNH), Quality of life
Abstract: PB1486
Type: Publication Only
Session title: Bone marrow failure syndromes incl. PNH - Clinical
Background
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease associated with major complications such as thrombotic events and impaired renal function. Prior to the introduction of C5 inhibitors in 2007, PNH had been fatal in about 35% of patients within 5 years of diagnosis; yet this fatality rate continues in countries without access to these medications. Worldwide access to the C5 inhibitor eculizumab is hindered by the unavailability of treatment, and in places where treatment is approved, cost of treatment, reimbursement issues, infrastructure, or patient restrictions may further impede access (Risitano et al, Am J Hematol. 2018; Risitano et al, Front Immunol. 2019). PNH treatment places a significant economic burden on healthcare systems. In many countries, this has resulted in negative health technology appraisals (HTAs), indicating that optimal care and resource utilization are not being achieved (Coyle et al, Med Decis Making. 2014). New therapies with more convenient modes of administration may improve how PNH is clinically treated and positively impact access to C5 inhibitors globally. Further, treatments that allow for home administration may reduce patient and healthcare systems costs. Robust data on the real-life burden and cost of PNH are needed to assess the impact of current therapies and establish a baseline for new therapeutic approaches. Data derived from this study, which to our knowledge is the first socioeconomic study in PNH, will be used to support HTA processes and inform the value of new PNH therapies.
Aims
The COMMODORE burden of illness (BOI) study will quantify the direct medical costs (eg, treatment and hospitalization), direct nonmedical costs (eg, travel), and indirect costs (eg, impact on work productivity and family burden) associated with PNH for patients and care providers and determine the impact of PNH on health-related quality of life (HRQOL).
Methods
This is an international, prevalence-based, bottom-up, BOI study containing both retrospective and prospective data collection. The study will be overseen by an expert reference group consisting of multidisciplinary stakeholders. The study protocol and materials will be submitted for ethical approval to the University of Chester in the United Kingdom. Physicians will provide information on sociodemographic, clinical, and medical resource utilization using electronic case record forms (eCRFs). Through patient and public involvement and engagement, patients, after giving informed consent, will provide further information on the economic and HRQOL impact of PNH by completing patient-reported outcome surveys. Patients from France, Germany, the United Kingdom, and China will be included.
The aim is to recruit 94 physicians who will report 350 patient eCRFs with an expected return of 140 patient surveys and longitudinal data collection after 6-12 months for each patient. Mean per-patient costs, including direct medical and nonmedical costs, and indirect resource utilization will be calculated by multiplying the individual resource utilization by country-specific unit costs. National economic burden will be extrapolated by applying national prevalence estimates of PNH. Additionally, the impact of PNH on patient HRQOL will be assessed.
Results
Final results are expected in spring 2022.
Conclusion
The COMMODORE BOI study aims to characterize current PNH treatment via quantification of the humanistic and socioeconomic burden at the patient, healthcare system, and societal level to enhance the evidence base for treatment and decision-making in this community.
Keyword(s): Complement, Health care, Paroxysmal nocturnal hemoglobinuria (PNH), Quality of life