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).
Legal Notice, Privacy Notice and Data Protection Statement

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

Online registry platform. Participants join and maintain their data through a secure online portal.
Event triggering registration

Event triggering registration of a person in the data source

Other

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

New registration received (patient-initiated). Once registered, participants receive invitations every six months to review and update their data.

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

Loss to follow up
Other

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

Participant asks to be removed from the registry or provides a medical report which confirms they do not have a diagnosis of SMA.

Event triggering creation of a record in the data source

Patient-initiated (voluntary) registration.
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, pre-linked

The UK SMA Patient Registry was not created by the linkage of other data sources.

Linkage description, possible linkage

Consent is collected for data linkage between the UK SMA Patient Registry and SMA REACH UK (paediatric) and Adult SMA REACH clinical databases. Data linkage is achieved through a manual process. All three databases are independent and their IT platforms are not connected. The purpose of the linkage is to link patient-reported outcome measures (PROMs) from the patient registry with clinical data from SMA REACH.

Linked data source 1

Pre linked

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

No

Data source, other

Adult SMA REACH

Linkage strategy

Other

Linkage variable

A single variable is used, referred to as the SMA REACH reference number (e.g. SN1234). The single variable is created by the SMA REACH data sources. If a patient is participating in either of these studies, they will be allocated an SN number which they can report to the UK SMA Patient Registry.

Linkage completeness

Approximately 50% of participants in the UK SMA Patient Registry have reported an SMA REACH reference number.

Linked data source 2

Pre linked

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

No

Data source, other

SMA REACH UK

Linkage strategy

Other

Linkage variable

A single variable is used, referred to as the SMA REACH reference number (e.g. SN1234). The single variable is created by the SMA REACH data sources. If a patient is participating in either of these studies, they will be allocated an SN number which they can report to the UK SMA Patient Registry.

Linkage completeness

Approximately 50% of participants in the UK SMA Patient Registry have reported an SMA REACH reference number.
Data management specifications that apply for the data source

Data source refresh

Every 6 months

Informed consent for use of data for research

Other

Informed consent, other

There is a steering committee to evaluate requests for data access. Participants consent to the use of their data for purposes approved by the steering committee.

Possibility of data validation

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

No

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?

Yes
Common Data Model (CDM) mapping

CDM mapping

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

No