Patient registries: use by industry

  • Contacts
  • Nathalie Goemans
    Nathalie Goemans
    Dr Nathalie Goemans (M.D. PhD) is a paediatrician and child neurologist, with certification in rehabilitation...
  • Hugh Dawkins
    Hugh Dawkins
    Professor Dawkins leads the OPHG in the development and management of policies, plans and statewide...
  • Rebecca Leary
    Rebecca Leary
    Becca Leary is a member of the TREAT-NMD Secritariat and responsible for the Global SMA...

In collaboration with clinicians and patient advocacy groups across the world, TREAT-NMD has created national patient registries containing the information needed to establish whether a particular patient might be eligible for a trial, together with the means of contacting them.

The global registries for patients with DMD and SMA are already running and accepting enquiries, and registries for other conditions are also in preparation. The global registries are safe for patients since they comply with all EU and national data protection legislation and have full ethical approval, but the information they contain makes them valuable tools that will save the pharmaceutical industry time and resources when identifying and recruiting patients for clinical trials.

Key benefits of the TREAT-NMD registries:

  • One single entry point for access to patient data worldwide
  • Registries contain accurate, verified genetic diagnosis together with key clinical data items including medication use and ambulation status
  • Unlike pre-existing data collections, the TREAT-NMD registries are not anonymous: they contain detailed information on individual patients and thus are not simply a statistical tool but a recruitment tool
  • Patient data is updated at least once a year
  • Powerful feasibility tool: can filter patients by precise mutation, age, ambulation status, medication type and location
  • Powerful recruitment tool: patients have consented to being contacted about trials for which they may be eligible

This is a unique resource and one that is revolutionizing the way patients are recruited for trials in neuromuscular diseases. Recruiting through the patient registries in parallel with recruitment directly by trial sites saves valuable time and resources and helps meet recruitment targets far more quickly and easily.

Information about what is collected by the global registry is available below.

DMD Core dataset

SMA Core dataset

To register your interest in using the global DMD or SMA registry please use the links below.

DMD interest form

SMA interest form

12 Apr 2017