TREAT-NMD Core Datasets
What is the purpose of the registry? Is there already an existing registry that could serve the same purpose?
Deciding on the purpose of the registry is an important first step as it will guide how the registry is designed. Many of the national registries that have been set-up for neuromuscular conditions have been done so with the objective of clinical trial readiness in mind. This means that the dataset that is collected is very concise but captures the important aspects of the inclusion criteria required for clinical trials. This is called a “mandatory dataset”. Collecting an agreed set of data allows the data to be comparable and anonymously aggregated which ensures a coordinated approach to clinical trial readiness. All national registries that wish to participate in the TREAT-NMD Global DMD/SMA registries are required to collect the “mandatory dataset”. Other diseases, such as FSHD and myotonic dystrophy, also have internationally agreed mandatory datasets.
- Duchenne Muscular Dystrophy
- Facioscapulohumeral muscular dystrophy
- Myotonic dystrophy
- Spinal Muscular Atrophy
Many registries also collect additional data on top of the mandatory dataset, such as items relating to the natural history of the disease or quality of life of the patient. Whatever the main purpose of the registry it is important to make sure that the registry is designed appropriately, the relevant consent has been sought from the patients and that all necessary approvals are in place.
Where a disease is very rare, and national registries are not feasible, then an international registry should be considered. In these instances it is important to be very clear about the objective(s) of the registry and why it is being set-up as it may try to serve too many purposes and not be successful. This has been achieved successfully for rare FKRP-related conditions with the setup of the Global FKRP Registry.
If you are thinking about setting up a registry in your country then please make sure that there is not already an existing registry that could serve the same purpose. It is important to avoid having competing registries where possible as this only duplicates efforts.