Access and validation

Governance details

Documents or webpages that describe the overall governance of the data source and processes and procedures for data capture and management, data quality check and validation results (governing data access or utilisation for research purposes).
https://www.dropbox.com/s/sy6j4xs60vxpfr2/5.%20WFH%20GTR%20Governance_22June2022.pdf?dl=0

Biospecimen access

Are biospecimens available in the data source (e.g., tissue samples)?

No

Access to subject details

Can individual patients/practitioners/practices included in the data source be contacted?

Yes

Description of data collection

Data will be collected by physicians and entered in the electronic Data Capture Tool. Patients will provide patient-reported outcome data via the patient engagement tool, myGTR.
Event triggering registration

Event triggering registration of a person in the data source

Start of treatment

Event triggering de-registration of a person in the data source

Death
Loss to follow up
Other

Event triggering de-registration of a person in the data source, other

Request to de-register

Event triggering creation of a record in the data source

If a patient receives gene therapy for hemophilia they are elligibile to participate in the registry and will be invited. Data will be collected at months 3, 6, 9, 12, 18, 24, and annually thereafter. Adverse events can be entered in the database at any time.
Data source linkage

Linkage

Is the data source described created by the linkage of other data sources (prelinked data source) and/or can the data source be linked to other data source on an ad-hoc basis?

Yes

Linkage description, possible linkage

Through a data linkage protocol. The data source does not link different records for patients already in the system (myGTR). New patients, not otherwise included in the system, can be included through data transfer from their national registry.

Linked data source 1

Pre linked

Is the data source described created by the linkage of other data sources?

No

Data source, other

Any national hemophilia registry collecting data on patients who receive gene therapy can link with the GTR.

Linkage strategy

Other

Linkage variable

Unique patient identifiers will be used to ensure no duplication of patients.

Linkage completeness

Completeness of linkages cannot be provided at this moment. The data source expects a high percentage for completeness as the data set was shared with several national registries who will transfer data.
Data management specifications that apply for the data source

Data source refresh

Yearly

Informed consent for use of data for research

Other

Informed consent, other

There is a committee to evaluate requests for data access

Possibility of data validation

Can validity of the data in the data source be verified (e.g., access to original medical charts)?

Yes

Data source preservation

Are records preserved in the data source indefinitely?

Yes

Approval for publication

Is an approval needed for publishing the results of a study using the data source?

No
Common Data Model (CDM) mapping

CDM mapping

Has the data source been converted (ETL-ed) to a common data model?

No