Global Registry Enquiries
The TREAT-NMD Global Registries (also known as the ‘Global Database(s)’) are recognised as a leading source for trial planning and recruitment at an international level. In addition, these registries can help researchers to answer questions about global disease prevalence. The TREAT-NMD Global Registries can also be used to support other activities to improve patient care, such as the assessment of care standards in different countries.
There have been over 20 enquiries to the TREAT-NMD Global Registries so far. These have included DMD, SMA and DM1 enquiries, and have included feasibility enquiries for clinical programmes looking at exon skipping and nonsense mutation as well as recruitment for clinical trials and a multinational study focusing on health economics. All of this work is undertaken with the aim of ensuring that promising new treatments can be brought to patients as quickly as possible.
Pharmaceutical companies interested in locating patients for a clinical trial are able to request information from the TREAT-NMD Global Registries in a secure fashion that means that patients’ details are never disclosed directly to the company, but companies can speedily find the information they need. Enquiries are also encouraged from academics and patient organisations, please contact us for information on how we can support these.
All TGDOC Registries adhere to the disease-specific mandatory core data sets listed below, created with clinical trial readiness in mind. Global enquiries can be completed into any of the items in these datasets, any may also include additional non-dataset items.
The types of enquiries that are usually received by the TREAT-NMD Global Registries are:
- Feasibility enquiries – general data on patient numbers within particular countries (or worldwide), that meet certain criteria, such as age range, ambulation status, etc. Usually requested in the planning phases of a clinical trial.
- Assistance with recruitment into a clinical trial – patients can be contacted through their national registry if they meet the clinical trial inclusion criteria and are then asked to contact their local trial site. Patients not seen at a particular site, and who wouldn’t otherwise be known to staff at that site, can also be informed about the trial.
The process for requesting data from the TREAT-NMD Global Registries is outlined below: