Study identification

EU PAS number

EUPAS1000000748

Study ID

1000000748

Official title and acronym

Description and assessment of fitness-for-purpose of real-world data (RWD) sources on Duchenne Muscular Dystrophy for regulatory decision-making

DARWIN EU® study

No

Study countries

Denmark

Study description

Duchenne Muscular Dystrophy (DMD) is a severe and rare progressive inherited neuromuscular disorder for which there is currently no cure. Several new types of therapies have recently achieved conditional approval.
Therefore, there is a need to supplement the clinical trial data with long-term safety and effectiveness evidence to support regulatory decisions.
The aim of the current study is thus to map exiting data sources within DMD for future real-world evidence (RWE) studies.
This mapping will include the following key elements:
• Establishment of a list of core variables required for future RWE studies in DMD disease.
• A structured inventory of registries and other data sources relevant to DMD.
• Assessment of longitudinal completeness, core variable availability (e.g., genotype, functional
scores, medication use), and outcome definitions.
• Evaluation of completeness, consistency, and timeliness of data, as well as mechanisms for
ongoing data curation and quality assurance.
• A review of access procedures, legal and ethical considerations, and technical feasibility of
linkage with other data sources.
Finally, we will describe the overall findings and considerations in a manuscript for publication including recommendations on important operational, methodological and technical elements to consider when considering using real-world data (RWD) to address any research questions of interest.

Study status

Ongoing
Research institutions and networks

Institutions

The Danish Rehabilitation Centre for Neuromuscular Diseases (RCFM)

Networks

Contact details

Tine Iskov Kopp 0000-0003-3729-4420

Primary lead investigator
ORCID number:
0000-0003-3729-4420

Study timelines

Date when funding contract was signed

Actual:

Study start date

Planned:
Actual:

Data analysis start date

Actual:

Date of interim report, if expected

Planned:

Date of final study report

Planned:
Sources of funding
EMA
Regulatory

Was the study required by a regulatory body?

No

Is the study required by a Risk Management Plan (RMP)?

Not applicable