Registries tool kit
Things to consider when setting up a registry
TREAT-NMD, along with many patient organizations, are experienced in the creation and implementation of registries for neuromuscular conditions.
All registries are tailored to not only the disease they cater for but also the location in which they operate, making the creation of each registry a unique process.
A registry can vary by purpose and design and can be a very simple collection of data to an elaborate database using bespoke software.
That said, many common factors still exist and we have tried to outline some of the fundamental things that should be considered when setting up any type of rare disease registry.
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.
How will the registry be developed and information collected?
There are several different ways in which a registry can be set-up. It is important to take into account any country specific, disease specific or cultural issues which may hinder the success of the registry and the ability to capture the required data.
Types of registry design:
- Patient self-reported: only patients are asked to provide information – generally through a publically available website but this can also involve patients providing data on paper and registry staff entering the data into the registry
- Professional reported: only doctors provide information – these registries generally do not have a publically available website
- Combination of patient self-report and professional report: some information is provided by both patients and their doctor – generally has a publically available website
If you are setting up a registry for a disease where there is currently no mandatory dataset agreed then it may be useful to get in touch with TREAT-NMD to check if there is one in the pipeline, or if you would like help to gather the opinions of the Key Opinion Leaders in the field.
There are many different software options, including freely available software, that can be used depending on the requirements of the registry and funding available. TREAT-NMD has helped to set-up many registries and can provide information and advice on a number of software options.
The registry is only as good as the data that is held within it so if it proves impossible to capture the necessary data then the registry itself is not fit for purpose. The design of the registry should be continually reviewed and changed if necessary, even after it has been established.
What documentation is required?
There are various documents that are required for any registry, regardless of the registry’s purpose and design.
The types of documents that are typically required by registries are:
- consent forms (and in some countries, assent forms for children)
- patient information sheets (appropriately worded and adapted for different age ranges- check for national guidance on the age ranges you should use)
- Standard operating procedures (covering daily management of the database, data quality control and communications plan)
All of these documents will probably need to be reviewed by a local ethics board. It can be useful to do this at the same time as the registry software is being developed as it can take some time and may cause unnecessary delays to the registry being launched. Ethics boards will usually wish to see evidence of patient involvement in the creation of patient facing documentation so it is important to attempt to engage with the patient community at an early stage in the creation of a registry.
How will the registry activities be governed?
Registries should have a governing body (sometimes called a ‘steering committee’) in place that will ensure the registry is working towards its objectives. The function of a governing body can be varied and will depend ultimately on what the purpose of the registry is. The list below outlines some of the many functions a governing body can have:
- Data access and use by researchers
- Database content and research objectives
- Financial and administrative issues
- Ethical and legal issues
- Communication with funding sources, healthcare providers, etc.
It can be useful to have a document outlining what the function of the governing body is and its composition. This can sometimes be referred to as “Terms of Reference” but whatever the document is called it is important that all members of the governing body agree to it. Governing bodies will usually have patient representation as well as representation from expert doctors, patient organisations and other key opinion leaders. It is advisable to have a confidential disclosure agreement (CDA) in place with members of the governing body, to protect and support registry discussions. If you are part of an academic institution, your legal department should be able to assist you in the set-up of this.
All the national registries involved in the TREAT-NMD global registry agree to the TREAT-NMD Global Registries Charter, this can be used as an example document of best practice.
Who will approve the registry and review all of the documentation?
The ethical and legal requirements for a registry will vary depending on the country in which it is being set-up and so specific details can’t be given.
Registries collect potentially sensitive and valuable data about patients. It is therefore important to comply with local data protection regulations (e.g. In Europe General Data Protection Act (GDPR)). Even if such legislation is not available in your country it is still important to consider the safety and security of the data you are collecting. If you are setting up the registry in an academic institution, there may be an institutional ethics board which can assess your registry projects. In the UK, the Health Research Authority (HRA) research ethics committee (REC) can assess registry projects and in most cases, REC approval covers the whole of the UK (unless you are considering collecting data from adults who lack the capacity to consent for themselves). In the UK, if you are setting up a project within a hospitals trust, you may alsio need to approach your Trust R&D department for approval.
Other approvals may be required and it is important to check with local governing bodies. Seeking approvals can often be a lengthy process so it is worth seeking these out in parallel to the development of the registry to ensure there are no unnecessary delays.
Do not start to collect any information from patients until you have the necessary approvals in place.
Who is responsible for the data?
In most cases, a registry requires a Principal/Chief Investigator who is responsible for the overall management of the database. They will need to ensure that only responsible individuals are on the registry team (In most cases, background checks should be carried out) and have access to the database. The registry will usually also require a ‘lead organisation’ (an academic institution or hospital trust for example) which has the necessary insurances in place should a problem occur.
There are various aspects to sustainability, with funding being of key importance to most registries. Funding can be sought from various sources, including local government and patient organisations, not only for the initial set-up of the registry but also for an on-going support.
TREAT-NMD is not a funding organization and so is unable to support any registry initiatives on a monetary level but does have a wealth of expertise and experience in this field and is able to offer help and advice to anyone wishing to set up their own registry.
Once a registry has received all of the necessary approvals and is ready for patients to participate then it is important to make sure that the right people know that the registry exists! You may wish to create a website for the registry to provide information about the registry and to display registry news and basic statistics. Conferences are great ways to let a lot of people know all at once. Patient and family conferences can make a big impact on the success of the registry if patients are to self-report. Patient organisations are an execellent source of help and support as they can advertise the registry on their website and newsletters, and send information about the registry to their mailing lists and explain to patients the need for a registry. Many patient organisations are listed under the disease specific pages of this website.
Leaflets are also a good way of passing on information about registries and can be disseminated through patient organisations and at clinical centres. Any doctors that will be involved in the care of the patients should be contacted and provided with information about the registry.
You could also contact diagnostic laboratories which conduct gene testing for your condition of interest, to ask them if they would be willing to add a sentence about the registry on positive confirmatory reports.
TREAT-NMD should also be made aware of any new registry initiatives so that it can be listed on the website and in the newsletter.
Disclaimer: TREAT-NMD is not liable for any registry that is set-up with the help of the information made available on this website. The information is intended to be used only as a guide and should not be classed as a definitive resource.