Patient registries

Patient registries are databases that people living with a particular condition (or their parents/carers) can add their information to. This includes information about their symptoms, and if possible, what their genetic diagnosis is. Generally, each registry is designed for one specific condition. 

The aim of many registries is to help researchers running clinical trials to find people who want to take part in them, but they can have other purposes.  

The information registries hold can help clinicians develop care standards for a condition. People can sometimes access information about their condition or the latest research.  

Registries are usually set up at a national level, gathering information on people living with a particular condition in one country. However, there are some international registries for people living with rarer conditions, or conditions with many different subtypes (such as congenital muscular dystrophies). 

Patient registries are mainly designed to help match clinical trials with people who want to take part in them. By adding your details to a patient registry, you could have access to more opportunities to take part in clinical trials for your condition.  

Clinical trials and patient registries contribute towards research and could help to improve treatment and diagnosis in the future for people with the condition. Learn more about clinical trials for muscular dystrophies. 

Some patient registries also send regular updates to people on their registry, to keep them informed about the latest research into their condition.  

Researchers, including people working at pharmaceutical companies, developing treatments for neuromuscular conditions ask for help from registries for two main reasons.  

Firstly, when they are planning their trial, researchers can use data from registries to understand how many people might meet the eligibility criteria for the trial. This helps them decide how many countries and how many hospitals in each of those countries need to take part in the trial. This helps them make sure they can recruit enough participants for the trial in a reasonable timescale. 

Secondly, when they come to recruit people for the trial, they can ask the registries to contact all potentially eligible people on their behalf. Normally, only one or two hospitals in every country will be running a trial, but because each hospital only knows the people who are normally seen in that hospital, without the involvement of the registries, people elsewhere in the country may miss out. 

If recruitment into the clinical trial is too slow, researchers can use international registries to help them recruit additional participants. This can speed up the recruitment of participants, meaning the trial can start sooner. 

Registries are already in place for several neuromuscular conditions, and anyone who has one of the conditions listed can register. If you are the parent of a child with the condition, you can register on their behalf. 

Joining the registry starts with filling out an online form, answering questions about yourself and how the condition affects you.  

If someone wants to join but doesn’t have access to the internet, they can usually contact the registry by phone and be sent a paper version of the registration form. 

The registries are managed by a curator who monitors whether the information is entered correctly.  

You might be asked to give your consent for the curator to approach your treating clinician(s) for more specific clinical or genetic information.

All information you give to the registries is stored on a secure server, protected in a similar way to online bank accounts, which only specially appointed staff have access to. Information that is entered online is encrypted while being transferred, so that it cannot be intercepted.  

You can view the data the registry has about you at any time, including the information added by your clinician(s). At all times the data remains your property, and you have the right to withdraw it from the registry. 

Each registry is governed by an oversight committee, which includes medical experts and patient representatives. It is their responsibility to check that the registry is run correctly.  

Registries that can demonstrate high data quality and safety, and appropriate consent processes, can join the TREAT-NMD Global Registry Network.  

The main purpose of patient registries is to help researchers and professionals in the pharmaceutical industry find people to take part in trials. To do this, the registry acts as a trusted mediator between researchers and people living with the condition.  

A company or researcher can contact the registry with a specific request – for example: “we need to recruit 15 people in the UK who have this specific genetic change, are in this particular age range, are taking these drugs, and are able to walk without assistance” – and the registries have all this information immediately to hand for the people who have registered. 

First, the oversight committee for the registry checks that the pharmaceutical company or research team have all the necessary ethical approvals to run the trial. If the oversight committee approves the request, then the registry contacts everyone who meets the criteria for the trial.  

When a researcher is allowed access to the data in the registry, personally identifiable information (e.g. your name, address etc.) does not go with it. The data about your condition is identified only by a code. This means you can be sure that your information is safe and nobody unauthorised can access it. 

If you were contacted by the registry, you would be told that there is a trial you might be eligible for and given the details on how you could take part. Usually this would involve you contacting the researchers running the trial to arrange an appointment to discuss the trial and what it involves. You would only need to do anything if you were interested in taking part – your personal details are never given to the company or researchers without your permission.