EPOCH-Economics and Patient Outcomes in China in Haemophilia (EPOCH)

The overarching goal of the EPOCH project is to:

• Support a broad and robust needs assessment in the haemophilia space in China
• Measure the variability in the needs and care provision
• Generate outcome data (health economic outcomes and supporting clinical and PRO data) to enable measuring the impact of different treatment modalities and different levels of treatment access PRIMARY OBJECTIVES
• Determine the burden of haemophilia in China, and the determinants of its variability
• Determine the costs of haemophilia care in China, and the determinants of its variability SECONDARY OBJECTIVES
• Measure treatment patterns and their variability
• Measure levels of access to care
• Estimate the impact of haemophilia and its treatment of patients reported outcomes
• Understand consistency of care by centres/geography/demographics

Study Overview

• Status: Active, not recruiting
• Conditions: Hemophilia

• Study Type: Observational
• Enrollment (Estimated): 500

Contacts and Locations

Study Locations

• China
  • Tianjin Municipality
    • Tianjin, Tianjin Municipality, China, 300020
      • Instiute of Hematology and Blood Diseases Hospital CAMS＆PUMC

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: Yes

• Sampling Method: Non-Probability Sample

Study Population

Male patients, aged ≥12 years at the index date

Description

Inclusion Criteria:

• Male patients, aged ≥12 years at the index date
• Patients must be diagnosed with non-acquired severe haemophilia A or B prior to the previous 12 months
• Patients who are able to understand and assent / consent to the study

Exclusion Criteria:

• Patients who cannot, for any reasons (language barriers, mental incapacity, etc.) understand and answer the questions of the study questionnaires
• Patients that cannot access internet connection or download the application

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Successfully registered research centers	Successfully registered research centers in Trial Phase	Baseline until week 52
Number of enrolled patients	Number of enrolled patients in Trial Phase	Baseline until week 52
Proportion of completed questionnaires	Proportion of completed questionnaires for the Trial Phase	Baseline until week 52
Proportion of missing data	Proportion of missing data for the Trial Phase	Baseline until week 52
Qualitative information on study barriers and facilitators	Qualitative information on study barriers and facilitators for the Trial Phase	Baseline until week 52
Diagnostic and treatment burden for hemophilia patients	Diagnostic and treatment burden for hemophilia patients for the Implementation Phase	Baseline until week 52
Costs of hemophilia diagnosis and treatment	Costs of hemophilia diagnosis and treatment for the Implementation Phase	Baseline until week 52

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Measure treatment patterns and their variability	Measures of treatment patterns will be defined by type of treatment product, dose and frequency of administration. These measures will be considered in relation to severity of disease and eventually presence of inhibitors and relevant comorbidities. This measure will be collected both cross-sectionally and prospectively and reported directly by the patients. By organizing patient-level data and applying statistical analysis methods, we calculated the total cost of hemophilia diagnosis and treatment in China and specific regions.	Baseline until week 52
Measure levels of access to care	The access to care will be calculated considering the mean number of visits/consultations/follow-ups and hospitalizations for patient and stratified in relation to severity of disease, frequency of bleeding and presence of inhibitors/comorbidities and haemophilia related complications or health problems.	Baseline until week 52
Estimate the impact of haemophilia and its treatment on patients reported outcomes	General health problems are defined by determinants like use of mobility aids and assistive devices, use of pain medication, presence of and how acute pain and chronic pain interfere with daily life, difficulty with daily activities, surgical history and concurrent medical problems and work/school life. Haemophilia-related health problems refer to disease severity, inhibitor status and history, bleeding frequency and history, treatment regimen and history and presence of target joint(s)	Baseline until week 52

Collaborators and Investigators

• Sponsor: Institute of Hematology & Blood Diseases Hospital, China
• Collaborators: EPOCH Study, Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): November 14, 2024
• Primary Completion (Estimated): December 31, 2028
• Study Completion (Estimated): December 31, 2028

Study Registration Dates

• First Submitted: June 5, 2025
• First Submitted That Met QC Criteria: March 3, 2026
• First Posted (Actual): March 6, 2026

Study Record Updates

• Last Update Posted (Actual): March 6, 2026
• Last Update Submitted That Met QC Criteria: March 3, 2026
• Last Verified: March 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; Hemophilia A
• Other Study ID Numbers: QTJC2024006; EPOCH Study, Ltd. (Other Grant/Funding Number: EPOCH Study, Ltd.)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No