Mitochondrial patients and caregivers have been integral in helping guide the development of potential treatments for mitochondrial disease as part of the first phase of the PREFER study. But what does this mean and what happens next? Dr Cecilia Jimenez-Moreno, the PREFER clinical case study lead based in the Wellcome Centre for Mitochondrial Research at Newcastle University, tells us more.

By Lyndsey Butterworth.

What is PREFER?

PREFER is a project that aims to investigate how and when is best to study patient preferences and support decisions in the process of drug development. In our case study, we are seeking patient perspectives and expectations based on current medical needs that they would consider a priority. We are particularly interested in the views of both patients and carers regarding the potential risks and benefits of any new treatments for mitochondrial disease.

What do you want to find out?

We want to know what people with mitochondrial disease think about different hypothetical treatment scenarios and explore what ‘trade-offs’ they might be willing to make. A ‘trade-off’ is the act of balancing a negative against a positive, for example, balancing the side effects or risks of a treatment against the benefits it may offer and taking a decision based on this. The information will be used to help pharmaceutical companies design more patient-centric treatments and clinical trials.

What has happened so far?

We have conducted a number of face-to-face interviews and focus groups with mitochondrial patients and caregivers across the UK. This has included focus groups at the recent Newcastle Patient Information Day that took place earlier this year (photograph below). During these focus groups, participants discussed the impact of mitochondrial disease on their daily lives and the symptoms that currently give them the greatest difficulty or concern. They also talked about what they would expect from a treatment if one suddenly became available and what risks they would be most worried about or not willing to take.

Did the focus groups go well?

The focus groups were a chance for patients to meet one another and share their experiences. I think these types of discussion give participants the opportunity to listen to different stories and empathise with each other. We had some very helpful discussions, and everyone was very enthusiastic and keen to contribute. When asked about their reasons to take part, one patient said, “knowledge is power, and, I need to empower myself… so that’s why I’m here”.   Another patient said, “I’m just looking at if there’s anything that can help anything and that can improve anyone, especially the younger generation”.

What will happen next?

The first phase of the PREFER study is now complete and we are currently in the process of compiling all the data obtained from the focus groups. After this, we will be ready to move onto the next phase of the study, which will use the results obtained to produce an online patient preference questionnaire. It is likely that this will be ready for autumn 2019 and we will be encouraging mitochondrial patients and caregivers from around the world to take part and help shape the future of mitochondrial research.

Dr Cecilia Jimenez-Moreno would like to thank all the mitochondrial patients and caregivers who gave their time and support for the PREFER project. Cecilia said, “I am very grateful for the trust shown by all participants and for the stories shared without hesitation”. A special thank you also goes to The Lily Foundation, the UKs leading mitochondrial disease charity. Cecilia said, “Every day more and more researchers and industry relay on the support and engagement of patient organisations to deliver successful trials. In this case, The Lily Foundation is continuing to support the PREFER study with patient engagement, recruitment and dissemination”.

The Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle (PREFER) project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No 115966. This Joint Undertaking receives support from the European Union’s Horizon 2020 research and innovation programme and EFPIA.

Disclaimer: This website and its contents reflects the PREFER project’s view and not the view of IMI, the European Union or EFPIA