Article No. 429
23 April 2021
The aHUS alliance reputation for its 360 degree view of the rare disease aHUS continues to grow. Support of international aHUS researchers is built into our DNA. That support is reflected in the way it features up to date research on our platform and gives recognition to the aHUS research teams around the world
No one rivals what the aHUS alliance is doing. But it does more than passively reflect the developing knowledge about a life threatening ultra rare disease.
Although aHUS Global Action is limited in resource and capacity, it also undertakes its own research to gain knowledge about aHUS.
Even in the first week its website appeared, aHUS Global Action reported on research that had taken place. The alliance had been conducting its second Global aHUS Poll. It had done so in 2014 in partnership with Rare Connect, but the 2016 poll was its own. Bigger and more ambitious as the article about it at the time shows.
At the same time the 2016 Global Poll unfolded, work was also in hand on another research project. In this research aHUS Global Action was getting an answer for “what research is a priority for aHUS patients”. Done in partnership with the aHUS Registry, the results of the research were published in the Orphanet Journal of Rare Diseases. An article about the publication featured on our website.
The subject of aHUS Expert Centres has been a regular topic on our website. So aHUS Global Action decided to study what they did and used the EURORDIS and RDUK list of roles and criteria for such centres to “score” four European aHUS centres of excellence while visiting them to understand their work. The results of the research were presented on our website.
Another research project for Global Action was again linked to the aHUS Registry. It was about “patient reported outcome” feedback compliance by aHUS patients participating in that Registry. From data supplied by the Registry about reporting over a six year period, a four part article featured on our web site which explained the importance and value of patient feedback but showed the reality of what happens over time.
Another form of research is a literature review about a specific topic of interest. aHUS Global Action did that to produce the biggest 2020 horizon scan of new therapeutics for Complement conditions , including aHUS. The results were reported on our website.
In the same year, aHUS alliance Global Action attempted a challenging novel project by inviting aHUS patients with experience of both eculizumab and ravulizumab technology to share their views for a comparison of experiences.The resultant piece of qualitative research provided powerful and persuasive evidence for use in any health policy decisions about the drugs around the world..
And finally onto the latest direct research action which is still in progress. An even more ambitious and insightful study of aHUS. It is about the collective recalled experiences and perception of patients themselves about their aHUS Diagnosis Process. An enormous amount of data was provided and has been analysed and first draft report is nearing completion.
Research can take many forms whether it is to produce a patients aHUS research agenda from hundreds of patients suggestions
or an aHUS Patients’ Vision
What matters is what matters to aHUS patients and their families.
Each year Findacure a global rare disease organisation runs a competition for medical students to write essays about rare diseases. In one of these essays* by Caitlyn Taylor, the following comment resonated
“Patient organisations such as the Addison’s Disease Self Help Group are key in coordination.
Prior to speaking to D, I was of the misconception that patient organisations were (as the
name suggests) for patients and were only of limited value to clinicians. Now I see that a
group of patients working together is extremely powerful, as patient experience is central to
the coordination of the diagnosis and management of rare disease.”
All in all our direct research action, which is performed by patient advocates themselves exemplifies the maturing scope and direction of aHUS patient advocacy going forward. Above all, it is about gathering and giving a coherent and authoritative patient voice on what really matters to them.
*full article HERE.