First Patient Enrolled in Pilot Phase of the Economics and Patient Outcomes in China in Hemophilia (EPOCH) Study

Speaker(s)

Yang R1, Sun J2, Wang S3, Runhui W4, Zhang X5, Xue F1, Clearfield E6, Skinner M7, O'Mahony B8, O'Hara J9, Iorio A10
1Institute of Hematology and Blood Diseases Hospital, Tianjin, Tianjin, China, 2Department of Hematology, Nanfang Hospital, Southern Medical University, Guangzhou, Guangdong, China, 3Peking Union Medical College Hospital, Beijing, Hebei, China, 4Beijing Children’s Hospital, Capital Medical University, Beijing, Hebei, China, 5Shandong Blood Center, Jinan, Shandong, China, 6Institute for Policy Advancement, Ltd, Washington, DC, USA, 7Institute for Policy Advancement, Ltd, Washington, D.C., DC, USA, 8Irish Haemophilia Society, Dublin, Ireland, 9University of Chester, Chester, UK, 10McMaster University, Hamilton, ON, Canada

Presentation Documents

OBJECTIVES: EPOCH is a global collaboration initiating a patient register to observe current standards and costs of care, quality of life (QoL), and unmet needs for people with hemophilia (PWH) in China. Health economic and outcomes research (HEOR) data collected includes direct and indirect costs and supporting clinical and patient reported outcomes (PRO) to measure the impact of different treatment modalities and varying levels of treatment access. EPOCH establishes a centralized data collection/analysis program based in China.

METHODS: EPOCH is designed with two phases: the pilot phase (one year), will assess the feasibility of the project, and the implementation phase (two years), will collect data to understand the treatment landscape in China. A cooperative network of hemophilia treatment centers across China will enroll participants. Data is collected on a web-based platform that links provider-patient pairs. Providers extract information from medical records; patients complete a questionnaire with QoL measures synthesizing elements from currently-available studies with validated instrument: the Patient Reported Outcomes, Burdens, and Experiences (PROBE) Study, a hemophilia-specific QoL questionnaire. Socioeconomic insight will be derived from the register to form comparison with CHESS (an ongoing survey in Europe/Americas quantifying humanistic and economic burden of hemophilia) and PROBE surveys conducted in other parts of the world.

RESULTS: The web-based platform has been developed and deployed for hosting on a server based in China. Ethical approval has been obtained and the first patient is expected to be enrolled in July 2024 . EPOCH expects to enroll 500 and 1500 PWH in the pilot and implementation phases, respectively.

CONCLUSIONS: The EPOCH platform is designed to capture real-world data and observe individual patient journeys as they access treatment. Information collected will provide evidence about the experience of living with and treating hemophilia in China that can be used to advocate for improved care and more equitable access to treatment.

Code

RWD150

Topic

Clinical Outcomes, Economic Evaluation, Patient-Centered Research, Study Approaches

Topic Subcategory

Clinician Reported Outcomes, Patient-reported Outcomes & Quality of Life Outcomes, Registries

Disease

Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)