What are patient registries?
Patient registries are databases that contain information about individuals affected by a particular condition. Most registries focus on the information that is needed to find patients eligible for clinical trials, but they have many other benefits. The information they hold can help clinicians develop care standards for a particular condition. Patients can link to the research community and have the opportunity to access information directly relevant to their condition. The registries are generally designed for one specific condition. They include information about the symptoms of the people registered and if possible, what their genetic diagnosis is.
Registries are usually set up at a national level and then the data combined into one international registry. For example, there are now registries for Duchenne muscular dystrophy in over 40 countries and data from these registries is combined into one international registry. For some rarer conditions, or conditions that have many different subtypes such as congenital muscular dystrophies, international registries have been set up without the intermediate step of national registries.
Who can register and how do you do it?
Registries are already in place for several neuromuscular diseases 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.
All registries involve filling in a form on the internet and if you do not have access to the internet you can make contact by phone and be sent a paper version of the registration form.
The registries are managed by a curator who monitors whether the data are entered correctly. You are normally asked a number of questions about yourself and how the condition affects you. You might also be asked to give your consent for the curator to approach your treating clinician(s) for more specific clinical or genetic information. You can view your data at any time, including the information added by your clinician(s).
You can find the web addresses of the currently available registries here.
Is my data safe?
All data you give to the registries are 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.
If the registry is run by a patient organisation they are required to adhere to the ‘TREAT-NMD Registries Charter‘. This document sets out the recommended best practice for running a registry and provides guidelines to protect your data. At all times the data remains your property and you have the right to withdraw it.
When your data is transferred to an international registry or when a researcher is allowed access to the data in the registry, personally identifiable information such as 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 details are safe and nobody unauthorised can access them.
Each registry is governed by an oversight committee which includes medical experts and patient representatives. It is their responsibility to monitor that the registry is appropriately run and to review any request for data, for example a company planning a clinical trial might request information from the registry. The committee will then decide whether the request should be granted.
If you don’t give my details away, how will I be contacted about trials?
The main point of the registries is to be able to find patients for trials and the registry acts as a kind of ‘trusted intermediary’. A company or researcher can come to a registry with a specific request – like “we need to recruit 15 patients in the UK who have this specific mutation, 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.
First it is checked that the company has all the necessary ethical approvals to run the trial. If the oversight committee for the registry approves the request then everyone in the registry who meets the criteria for the trial is contacted. 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 clinic 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 details are never given to the company.
How have registries helped in the development of treatments?
Companies developing treatments for neuromuscular conditions have asked for help from the registries in two main areas. Firstly, when they are in the planning stages of their trial, they can use the registries to find out statistical data about the numbers of patients who might meet the criteria for the trial. This helps them decide on the number of countries and the number of centres in each of those countries they need to include in order to make sure they can recruit sufficient patients quickly enough.
Secondly, when they come to recruit patients for the trial, they can ask the registries to contact all potentially eligible patients on their behalf. This is particularly helpful because usually only one or two centres in every country will be running the trial. As those centres only know the patients who are normally seen in that centre, without the involvement of the registries patients elsewhere in the country may miss out .
When pharmaceutical companies find recruitment into their particular clinical trial too slow, they can use international registries to help them recruit their remaining patients. This can really speed up the recruitment of participants and the trials can start more quickly.