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Fighting mito,
finding hope.

Celebrating 15 years of… patient advocacy


17 April 2023

One of the reasons The Lily Foundation exists is to help give a voice to mito patients and their families, to enable them to have a say in the services and support available. A lot has changed in the last 15 years, and we’re proud of the progress we’ve made, but we don’t intend to rest on our laurels, because understanding mitochondrial disease is the key to unlocking effective treatments and that eventual cure.

A couple are seated in front of a medic in an office chatting

Patient advocacy is important in all areas of healthcare, but particularly so in the complex field of mitochondrial medicine, where symptoms are often so rare and unique to each individual. There can be a real lack of understanding, even within the medical world, about the impact of mito on people’s lives. We know mitochondrial specialists are aware of this, because they often comment that their patients are the real experts who know more about their disease than their doctors. We pride ourselves on giving patients and their families a platform to have their voices heard, and here are just some of the ways we’re achieving this.

We’re working with researchers

In recent years the landscape around scientific research has changed a lot. Researchers are doing an incredible job seeking treatments and that all-important cure for mito, but there’s a disparity between someone in a white coat in a lab, and a mito patient struggling to get out of bed each morning because of their condition. We’ve worked hard to bring the two parties together, so that researchers can understand directly from patients the symptoms of their disease, and the challenges they bring.

a women with dark hair in a science laboratory is working at a bench with various machines and instruments for scientific assessment

One of these researchers is Katherine, who is currently working on a collaborative project within the National Disease Registration Service to create a mitochondrial disease registry. She ran a focus group at our Family Weekend last year to find out how patients and their relatives feel about the use of patient data, and which research questions are important to them. Katherine spoke positively of her experience: “The participants were supportive of our research. They were happy for data to be linked together for research within the NHS. They helped me to understand which aspects of mitochondrial disease and its treatment might not be captured by our research using hospital codes… I found the group so useful that my team went on to arrange a similar focus group with adults affected by mitochondrial disorders… It was very useful to explain the research to people living with mitochondrial disease and get their feedback and ideas, outside of a hospital environment.”

We’re working with drug companies too

The approach from the big pharmaceutical companies has also shifted. There’s a growing consensus across the industry that it’s no longer enough to just go where the science takes them – they also have to understand what patients need and what’s really going to make a difference to their quality of life. Patient perspectives are especially important with a complex condition like mitochondrial disease, where experiences can be so varied.

As a patient organisation representing a rare disease, we have the opportunity to be actively involved in facilitating faster access to new drugs, and a key part of this is about engaging directly with drug companies and representing the patient voice. We’ve been working with various global pharmaceutical companies for several years now, and these relationships help to educate those working in the field and improve their understanding of the disease, as well as giving us an insight into drug development pathways so we can manage patients’ expectations when it comes to accessing new medicines.

A group of 3 Doctors made up of one woman and 2 men stand behind a smiling male doctor wearing glasses and blue scrubs with his arms folded in front of him

“Charity involvement is a safeguard to ensure that research does in fact reflect the needs and priorities of the people who will end up using the products,” explains Alison, our Head of Research and Finance. “For a rare disease like mito, where there is hardly any natural history of published data, this kind of direct patient input can make the difference between a trial succeeding or failing. It’s a very important part of our approach.”

And then there’s the Innovative Licensing and Access Pathway (ILAP), which aims to smooth the complex approval process drug companies have to follow to bring new treatments from ‘bench to bedside’. We were one of several patient-facing charities who were invited to help steer decision-making at the initial stages of the process and ensure patient needs were given priority. You can find out more about that initiative here.

Our Patient Information Days

These informal settings give people affected by mito the chance to have their voices heard away from the sterile and sometimes unsettling environment of a doctor’s office or hospital ward. The feedback we collect from these gatherings is used to inform our charity’s policy and aims, as well as those of national and international bodies involved in the fight against mitochondrial disorders. This information is then used to help direct research, design clinical trials, develop potential treatments and create clinical care guidelines.

It was good to get updates on the latest research and listen to all the other talks. I enjoyed speaking with the Oxford team, The Lily Foundation and meeting some of the other patients who are dealing with Mito.” – Clare, young adult mito patient

The UK Mito Patient Registry

The Lily UK Mito Patient Registry, launched last year to coincide with our 15th anniversary, is a powerful online tool that lets people with a diagnosis of mitochondrial disease log details about their condition and how it affects their daily life. That information can then be used to help direct research, improve understanding about mito and facilitate patient access to clinical trials. It was developed with input from patient groups to ensure outcomes that put the patient first, and over time will help to build a more accurate picture of people’s lived experiences of the disease. Ultimately, it should help us move closer to finding a cure.

And, of course, there’s Katie!

A headshot of a  woman with a brown bob wearing glasses  smiling at the camera

Our very own Katie joined the team last year as Science and Patient Engagement Officer. She’s a qualified children’s nurse who understands the importance of research in providing hope for families affected by rare diseases, and part of her role is to work closely with our mito community, listening to their needs and helping to ensure they have a voice in research and care provision. “I love having the opportunity to raise awareness of mito,” Katie explained. “I work so closely with patients and their families to understand their needs, and then I communicate this to scientists, researchers and industry.”

By listening to patients and their families, we’ve helped ensure that the services provided by our charity and partner organisations (including the NHS) are appropriate, effective and ethical. Patients have helped inform our policy making at every turn, from the support services we provide to new diagnostic techniques and research into potential treatments. As a result, the patient voice has never been louder in the field of mitochondrial medicine. Many doctors, clinical specialists, researchers, drug companies and healthcare providers all now include patient advocacy groups in their policy-making.

But this can only happen while mito patients’ voices continue to be heard. The more we listen to them, the closer we can get to uncovering new treatments and that all-important cure. And that’s the reason why patient advocacy has been at the heart of our charity since its inception. Help us to keep it that way.

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