Introduction
The Economics and Patient Outcomes in China in Haemophilia (EPOCH) project is a global collaboration effort that aims to initiate a system to collect data to elucidate the current standard of care (SOC) and identify unmet needs of people with haemophilia (PWH) in China. EPOCH will establish a centralized research program based in China for data collection and analysis. The planned multi-centre study will run for three years, and information collected will provide evidence about the experience of living with and treating haemophilia in China that can be used to advocate for improved care and more equitable access to treatment. Data collection via a web-based platform is planned through a cooperative network of core haemophilia treatment centres (HTCs) with participation and collaboration between providers and patients across China. Local primary investigators will extract information from electronic health medical records (retrospective component), then patients will complete a standardized questionnaire that includes QoL and patient-reported outcome measures (cross-sectional component). The questionnaire synthesizes elements from currently available studies and uses validated instruments in haemophilia. The study will measure clinical outcomes, health-related quality of life (HRQoL) outcomes and health economics outcomes to understand the needs and support care provision in China. The platform will be designed to capture real-world data and observe individual patient journeys as they access treatment.
Methods
EPOCH is planned as a three-year multi-center study that includes retrospective, cross-sectional, and prospective components. The one year pilot phase is initiating soon (Figure). A China research network will be established; data will be housed in-country. Data collection is planned through a cooperative network of core hemophilia treatment centers with participation and collaboration between providers and patients across China. A web-based application for data collection (myEPOCH app) will be developed, implementing a longitudinal platform for continuing research. Local primary investigators will extract information from electronic health medical records, and then patients will complete a standardized questionnaire that includes QoL and patient-reported outcome measures (Table). The questionnaire synthesizes elements from currently-available studies and uses validated instruments in hemophilia.
Results
Expected participation in the pilot phase is 500 patients. Participants in the pilot phase will be invited to enroll in the longitudinal portion and provide updated information at each follow-up timepoint. Clinical and treatment experience data will complement the QoL and economic outcomes reported by patients to generate a 360-degree view of the hemophilia treatment and burden landscape in China. EPOCH will be a platform to capture real-world data and observe individual patient journeys as they access treatment.
Conclusion
EPOCH aims to establish a research network to expand the research capabilities of local providers and patient groups in China. A robust longitudinal dataset will provide clinical, economic, and quality of life data to support improvement in access, care, and outcomes.
TABLE. Patient-Reported Outcomes
| Outcome Category | Outcomes Collected |
|---|---|
| Quality of Life | PROBE (pain, independence, education, employment, family life, mobility) and EQ5D-5L with VAS (current health status) |
| Direct Health - Related Costs | Medical/surgical procedures, visits/consultations, hospitalizations, coagulation factor, tests and examinations, traditional Chinese medicine, other self medication (over the counter), devices and person aids, professional caregivers |
| Direct Non-Health Related Costs | Home alterations, travel costs, nonprofessional caregivers, transfer payments, alternative therapies |
| Indirect/Societal Costs | Absenteeism, work productivity impact |
FIGURE. EPOCH Study Timeline and Outcome Collection Overview
EPOCH TEAM
| Name | Occupation | |
|---|---|---|
 |
Professor Alfonso Iorio | Director of the Health Information Research Unit and the Hamilton-Niagara Regional Hemophilia Treatment Centre located at McMaster University Medical Centre (MUMC) |
 |
Paul Johnston | Qualified accountant with senior-level financial, commercial and health-sector experience involving working with the NHS, growing start-up businesses and setting up valuable partnership contracts |
 |
Jamie O'Hara | Leading HEOR researcher with expertise in haemophilia’s burden of illness and experience in advising on EU-level health policy and consulting for multi-billion dollar market access initiatives, Senior Lecturer of Health Economics at the University of Chester, Project Director for the CHESS studies |
 |
Brian O'Mahony | Chief Executive of the Irish Haemophilia Society, Vice-Chair of the Tender Commission, Chair, Research Committee,Irish Blood Transfusion Service. Assistant Professor, Department of Health Service Management, Trinity College, Dublin |
 |
Mark Skinner | President/CEO of the Institute for Policy Advancement Ltd and an Assistant Professor in the Department of Health Research Methods, Evidence and Impact at McMaster University |
 |
Elizabeth Clearfield | Senior Research Manager with the Institute for Policy Advancement Ltd, specializing in core outcome set development and patient quality-of-life studies as part of her focus on qualitative and quantitative methods in patient-centered outcomes research. |
Acknowledgements
Thank you to the professors, hematologists, and staff at the
hemophilia treatment centers.
EPOCH is an independent investigator research project
funded in part by Novo Nordisk and Sanofi.
2. Zhang W, Song X, Dou X, Poon M-C, Liu W, Chen L, Zhang L, Xue F, Yang R. Demographics, clinical profile and treatment landscape of patients with haemophilia B in China. Haemophilia. 2022.
3. World Federation of Hemophilia. Report on the Annual Global Survey 2018. Montréal, Québec: World Federation of Hemophilia, 2019