Study type

Study type

Non-interventional study

Scope of the study

Effectiveness study (incl. comparative)
Safety study (incl. comparative)
Non-interventional study

Non-interventional study design

Cohort
Study drug and medical condition

Name of medicine

ROCTAVIAN

Study drug International non-proprietary name (INN) or common name

VALOCTOCOGENE ROXAPARVOVEC

Medical condition to be studied

Factor VIII deficiency

Additional medical condition(s)

Hemophilia A
Population studied

Age groups

Adults (18 to < 46 years)
Adults (46 to < 65 years)
Adults (65 to < 75 years)
Adults (75 to < 85 years)
Adults (85 years and over)

Estimated number of subjects

720
Study design details

Main study objective

To characterize the long-term effectiveness and safety outcomes of patients treated with ROCTAVIAN in a real-world setting.

Outcomes

Targeted adverse events (TAEs) of hepatoxicity, thromboembolic events, infusion reactions, new malignancies, and development of FVIII inhibitors. Suspected adverse drug reactions, bleeding events, FVIII expression, use of haemostatic medications, and patient reported quality of life.

Data analysis plan

Full statistical methods and data specifications will be detailed further in an SAP. Analysis population will include: Safety Analysis Population: patients administered ROCTAVIAN and in the WFH GTR. Outcomes Analysis Population: patients administered ROCTAVIAN in the WFH GTR, and with data collected on the specific endpoint of interest captured in the WFH GTR database. Summary statistics (annual and overall) of the proportion, event rate, and incidence rate of the TAEs, and the event rate and/or incidence of suspected ADRs among all patients administered ROCTAVIAN will be provided. Summary statistics of annualised bleeding rate for treated bleeds and percentage of patients with zero (0) bleeds by treatment duration, FVIII level change over time, use of exogenous factor and non-factor replacement treatment(s) and change in EQ-5D-5L will be described (as allowed by the data).