Trial in Progress: Quantifying the Humanistic and Socioeconomic Burden of Paroxysmal Nocturnal Hemoglobinuria in the COMMODORE Burden of Illness Study

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 a number of countries, this has resulted in negative health technology appraisals (HTAs), indicative 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 have the potential to improve how PNH is clinically treated and may have a positive effect on the economic burden and access to C5 inhibitors globally. Robust data on the real-life burden and cost of PNH are therefore needed to assess the impact of current therapies and establish a baseline for new therapeutic approaches. The data derived from this study will be used to support HTA processes and inform the value of new therapies for PNH.

Objectives

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).

Study Design and Methods

This is an international, prevalence-based, bottom-up, burden of illness 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 an electronic case record form (eCRF). 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, United Kingdom, and China will be included.

The study aims to recruit 94 physicians reporting 350 patient eCRFs with an expected return of 140 patient surveys and longitudinal data collection after 6 to 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 with country-specific unit costs. National economic burden will be extrapolated by applying national prevalence estimates of PNH. Additionally, the impact of PNH on HRQoL in patients will be assessed within this study.

Summary

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. Final results are expected be available by the second quarter of 2022.