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 Type
OBSERVATIONAL
Enrollment
500
Instiute of Hematology and Blood Diseases Hospital CAMS＆PUMC
Tianjin, Tianjin Municipality, China
Successfully registered research centers
Successfully registered research centers in Trial Phase
Time frame: Baseline until week 52
Number of enrolled patients
Number of enrolled patients in Trial Phase
Time frame: Baseline until week 52
Proportion of completed questionnaires
Proportion of completed questionnaires for the Trial Phase
Time frame: Baseline until week 52
Proportion of missing data
Proportion of missing data for the Trial Phase
Time frame: Baseline until week 52
Qualitative information on study barriers and facilitators
Qualitative information on study barriers and facilitators for the Trial Phase
Time frame: Baseline until week 52
Diagnostic and treatment burden for hemophilia patients
Diagnostic and treatment burden for hemophilia patients for the Implementation Phase
Time frame: Baseline until week 52
Costs of hemophilia diagnosis and treatment
Costs of hemophilia diagnosis and treatment for the Implementation Phase
Time frame: Baseline until week 52
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.
Time frame: 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.
Time frame: 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)
Time frame: Baseline until week 52
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