The PREFER Project is Working to Include the Patient Voice in Mitochondrial Disease Research

PREFER

PREFER stands for Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle. It is a multi-stage project developed by Newcastle University’s Welcome Centre for Mitochondrial Research in effort to give mitochondrial disease patients and their caregivers more of a voice in the drug development process. The Lily Foundation, a UK charity organization for mitochondrial disease, has supported this project through patient recruitment as well as dissemination.

Doctor Cecilia Jimenez-Moreno has just announced that the first stage of the project is now complete.

The Patient Voice

The voice of advocates determined to prove the importance of the patient voice in research has become stronger and stronger in recent years and with good reason. However, in order to most effectively utilize patient contributions we need to understand at what point in the drug development process it is most crucial, and how to reap the most benefit from patient opinions. This is the aim of the PREFER project. It is looking at the how and the when of patient contributions in mitochondrial disease research.

Some factors being examined in this study include:

  • Perspectives and expectations of research based on their current medical needs
  • Opinion on risks/benefits of potential therapies
  • Opinion on what trade-offs they may be willing to make for a treatment (ex: side effects)
  • Perspective on how their disease impacts them in their daily lives

This study also included caregivers in the conversation. These discussions were completed using focus groups and in-person interviews across the UK.

In addition to the valuable information these discussions provided to researchers, they also proved immensely beneficial for patients. Through sharing experiences, they were able to empathize with one another and ultimately form relationships with others who have been going through the same thing. Patients were eager to participate and enthusiastic about sharing the knowledge they had. Other patients talked about the knowledge they gained through the process. One participant explained that-

“knowledge is power, and I need to empower myself… so that’s why I’m here.”  

The second stage of this study should be available by the fall of 2019. It will utilize an online “patient preference questionnaire.” Researchers hope that both patients and their caregivers across the globe will participate. The goal is for these voices to be the leading guide for shaping the future of research.

You can read more about the PREFER project here.


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