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

Study type

• Study topic:
  Disease /health condition

  Other
• Study topic, other:
  Real World Data feasibility study
• Study type:
  Not applicable
• Scope of the study:
  Feasibility analysis

  Validation of study variables (exposure outcome covariate)
• Data collection methods:
  Secondary use of data

Study drug and medical condition

• Medical condition to be studied:
  Duchenne muscular dystrophy

Population studied

• Short description of the study population:
  Patients with Duchenne muscular dystrophy registered in a disease register (world-wide)
• Estimated number of subjects:
  30000

Study design details

• Study design:
  na
• Main study objective:
  To identify a list of core variables to be used for future RWD studies in DMD patients with focus on the four regulatory research questions provided by EMA.
• Summary results:
  The final core variable list for the fit-for-purpose assessment includes variables that are both scientifically ; and clinically relevant to collect in real-world data sources, particularly disease registries. A “question-led”; approach was used to identify variables of interest enabling selection of variables ensuring focus on the ; regulatory research question of interest.