Home Health & Wellness Creating a Future, Together, for Rare-Disease Research

Creating a Future, Together, for Rare-Disease Research

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Patients with rare diseases have traditionally been the subjects of medical research. However, in recent years, their role has begun to shift from “research participants” to “experts with a lived experience”, with some being involved in study planning, design, and interpretation. Additionally, they may soon be involved in helping pick the most important areas to prioritise for research.

In a study published last month in the journal Research Involvement and Engagement, researchers from Osaka University created an online space, referred to as the “Evidence-generating Commons”, for conversation, collaboration, and knowledge sharing among patients with rare diseases, family members, researchers, and former policymakers.

The researchers had two important goals: To generate evidence that could contribute to new policies in the field of rare diseases, and to explore ways that stakeholders could be involved in the process.

To do this, they designed a series of in-person and online workshops that focused on understanding the difficulties faced by patients with rare diseases and their families, deciding on criteria that could be used to set research priorities, and then setting research priorities by applying these criteria. The researchers were flexible in their process, allowing the discussion points to be modified as the workshops went along to make sure the opinions and ideas that emerged in previous meetings were captured.

“In the first stage, participants were given 10 minutes to brainstorm difficulties faced as well as solutions, before coming together as a group to discuss and organise these ideas,” explained lead author of the study Atsushi Kogetsu. “In the second and third stages, participants engaged in workshops on specific themes that had come up and worked together to build a set of research priorities.”

The “Commons” was found to have a surprisingly positive impact on participants. “Everyone who participated in the project was able to grow and learn from each other, and several patients voiced their appreciation at having the opportunity to be part of the conversation and have their voices heard,” said Kazuto Kato, senior author.

The results showed that patients with rare diseases faced a wide range of issues including impacts on daily life, family problems, and social issues. From an initial set of 228 sticky notes, the project members identified 33 core themes that could be organised into 10 categories. They also identified seven topics that were considered high priority, including financial burden, impacts on daily life, anxiety, and the challenges of hospital visits. The achievements of the study were published in a research paper, with several patients acting as co-authors.

Policymakers are increasingly aware that evidence comes from a variety of sources and that the unique perspectives of patients and their families is one such critical source. Yet, there are still far too few examples of in-depth collaboration between patients and professionals. By bringing these voices together to create a set of research priorities, this study takes an important step toward more inclusive, collaborative, and effective healthcare.

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