Is an aHUS patient research agenda worthwhile?

aHUS patients have a global research agenda.

Work began on it in 2015 and after three iterations involving more than 200 patients and carers the current one was published on Rare Disease day 2019*, almost five years ago.

And so it has been since then. Many in the aHUS community probably may not recall it and most are probably not aware of it, including some patient advocates. Some may even think they can do better but offer no alternative.

For better or worse this one exists. On the landing page for the aHUS Patients’ Global Research Agenda, viewable at you can view the document in 3 languages (EN, FR, ES) as well as its evolution and importance to the aHUS community.

Rare Disease Organisations aspire to having a patients research agenda, most don’t have one, some spend thousands of dollars creating one.

The aHUS research agenda cost nothing just the time of unpaid volunteers with research and project management skills and input from patients to bring it to fruition.

Maybe its lack of cost might be regarded as of low worth to the community.

So we asked an expert we know with a knowledge and understanding about patient research agendas whether the research agenda created of topics which are important to patients is worthwhile having? The expert told us :

Yes, having an aHUS research agenda created by the aHUS patient community can be incredibly valuable. Patient-centered research agendas are essential for several reasons:

  1. Patient Priorities: Patients have unique insights into their condition, experiences, and the challenges they face. Their research agenda reflects the topics and questions that matter most to them.
  2. Research Relevance: A patient-driven research agenda ensures that research projects and studies are focused on addressing the issues that have the most significant impact on patients’ lives.
  3. Empowerment: Involving patients in setting research priorities empowers them to actively participate in the research process, making them partners in advancing knowledge and care.
  4. Improved Outcomes: Research aligned with patient priorities is more likely to lead to interventions and treatments that better meet the needs of those living with aHUS.
  5. Community Engagement: A patient-driven research agenda can strengthen the aHUS community, foster collaboration, and raise awareness among healthcare providers and researchers.

While traditional medical and scientific research remains essential, patient-centered research agendas provide a complementary and invaluable perspective. Patients and their families play a crucial role in advocating for and guiding research efforts related to aHUS. Such initiatives are worthwhile and can drive positive change in research and care.

So given the positive and supportive view of this expert we asked for the expert’s view of each of the questions in the aHUS patients research agenda.

There are 15 topics of importance to the community. The expert has answered them all for us. There is far too much to read all at once so Global Action will spread them out , publishing them over the coming weeks so that each question gets a spotlight on it.

This is the direct link HERE to the first question which is about genetic and triggering causes of aHUS.

Article No. 615

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