21 Aug, 2019

Building a Patient Registry

Neil Bennet from Action Duchenne discusses setting up a patient registry.

We recently held an event in London that brought together patient registries and charities to talk about the challenges and opportunities of doing research in genomics. One of the speakers at the event was Neil Bennett, the director of research at Action Duchenne. He discussed setting up a patient registry and how to recruit for clinical trials. If you haven’t already listened to the podcast episode of his talk, here’s what you missed:

About Action Duchenne

Action Duchenne is a UK-based charity that supports families with Duchenne muscular dystrophy. It was the first national charity solely dedicated to supporting those living with Duchenne, a condition which is caused by mutations, or changes, in the dystrophin gene. This mutation stops the body making a vital muscle protein and, over time, causes muscles to weaken and waste away. The first signs of Duchenne happen in the early years of life and the condition usually remains undiagnosed until around 4 years old. Unfortnately, Duchenne also reduces life expectancy to an average of 30 years and currently has no cure.

Action Duchenne is working towards developing effective treatments by funding research and supporting clinical trials, as well as building a more inclusive society for the people and families living with the condition. It does this by creating and maintaining a patient registry of the 2,500 people who have Duchenne muscular dystrophy in the UK and using it to help join patients and researchers to work on clinical trials.

Why set up a patient registry

The registry built by Action Duchenne was set up over 13 years ago and was one of the first of its kind. It was originally set up because the hope of better treatment finally looked as though it was becoming more realistic. Back in 2006, treatment was mostly corticosteroids, which did slow down muscle wasting and had some beneficial effects on the heart and lungs. However, these steroids didn't help the underlying genetic condition that was the root cause of the muscular dystrophy.

Action Duchenne's registry was set up by a group of parents who really wanted to drive research forward, particularly in genetic medicine. By 2005, research was already going ahead in things like exome skipping, which is a treatment that helps cells (in this case muscle cells) skip over exomes and restore a reading frame to avoid a mutation. It was clear that if these types of treatments were going to be tested in clinical trials, it would be really important to know about the patients in the UK and also understand their genetic mutations.

Since then, they've used their registry to link up researchers and patients, improve the uptake and specifications of clinical trials, complete surveys, identify participants and conduct feasibility studies. So, there are plenty of reasons why you might want to set up a registry but how do you get started?

Starting a Patient Registry

Firstly, you have to decide what you want to use your registry for; In Action Duchenne’s case, they decided the key thing they wanted to do was help get more people involved with clinical trials. They do this by asking everyone in their registry key inclusion criteria questions to quickly and efficiently sort them, so they know what medications everyone is taking, their demographic, whether they’re on any genetic treatments and even functional measures like whether they can stand or if they use a wheelchair. Knowing all of this information is key to linking people quickly with researchers for clinical trials and for being able to do feasibility studies - both of which can help improve the treatments available.

You also have to decide on your data entry model. In Action Duchenne’s case, information was previously entered by clinicians but because of time constraints, patients now enter their own data (excluding genetic data). In addition to this, be aware of data privacy and build a policy around how you will support the patients or users in your registry. Action Duchenne’s model is voluntary and anonymous so that no one is able to find out who you are just by looking at the registry. The only person who can find out is the curator; this keeps everyone’s personal data safe.

Technically, starting a patient registry isn’t difficult, so long as you have the necessary structures in place for data privacy, what information you’re taking from people, how you get that information and your protocol for sharing it. Neil says that their original database was simply built by “a parent who was good at IT”.

How to Use a Patient Registry

You want to use your registry to best facilitate research in your area or condition. To do this, it’s best to create questions that will help decide who meets inclusion criteria for trials so you can quickly and easily ask people whether they want to be involved.

You also have to decide how your users interact with your registry, in Action Duchenne’s case, they use a web-based model where users can simply log-in and update their data whenever they need to. In addtion to this, you'll need to determine how you contact users to be involved in clinical trials - do you ask for every trial? Certain trials? Or do they choose to automatically be signed up?

Initially, Action Duchenne’s registry was mostly used to recruit participants for clinical trials, however, it’s since evolved to conduct more feasibility studies. Whereby, the charity will be approached by researchers or a pharmaceutical company and be asked if it would be possible to find a certain number of participants that fit a specific demographic and criteria. Doing these kinds of studies basically enables you to get back to researchers and tell them how many people would be interested in their trial, meaning that you can help bring trials to your area and get more people involved.

You can also use your registry to send surveys from your patients to researchers and help them both interact through targeted emails etc.

Keep up to date with Neil Bennett & Action Duchenne

Be sure to visit Action Duchenne’s website to find out more about the great work they’re doing and to learn more about Duchenne muscular dystrophy. You can also follow them on Twitter and Facebook too.

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