By now followers of the Reluctant Advocate will no doubt have grasped the expansion of work that naturally occurs when running a charity to help people get the best of health. Sometimes this work has a global impact.
Apart from creating awareness about rare diseases, a greater understanding was emerging of the range of issues that rare diseases patients face, from diagnosis to research and registries for their disease.
In April 2014 I attended a second meeting of the Rare Disease Group for aHUS, a part of the Rare Renal initiative of the Renal Association funded by major Kidney Patient Organisations like Kidney Research UK . Although I used the meeting to brief other rare kidney organisations about what had been learned from our experience of NICE, something else caught my eye.
This meeting coincided with the annual UK Kidney Week conference that year held in Glasgow, Scotland. We were given a Conference pack, and, for the first time I realised what was involved in such conferences. In particular that in addition to lectures there were what were known as “poster presentations” , in which hundreds of individuals in an exhibition room put up posters to explain what had been found in some research that they had done.
The pack included a memory stick containing files of the Kidney Week programme and I did a search for anything related to “aHUS” or “eculizumab”. Among several search results I noticed a presentation by Dr Sally Johnson on behalf of something called the “The Academy of Complement Inhibition” about a Global aHUS Registry. The research was about the characteristics of just over 200 aHUS patients who had been enrolled in the Registry by then.
As Rare Disease Patients frequently face having no patient Registry for their Disease , here was one I knew nothing about for aHUS . So surprised and pleased to find it I wrote about it on the aHUS UK website having researched more about it: Probably the first aHUS patient organisation to do so.
“This registry sometimes referred to as an Alexion Registry has an official title of An Observational, Non-Interventional, Multi-Center, Multi-National Study of Patients With aTypical Hemolytic-Uremic Syndrome (aHUS Registry) and is officially a clinical trial listed by ClinicalTrials.gov. It was established in 2012 and will continue to recruit until 2023 and the final outcome will be published in 2025. By that time it is expected there will be 2000 patients registered. Although sponsored by Alexion the data is being stored by Ohio State University*. Full information can be read about it on ClinicalTrials.gov website by clicking here. The registry is being overseen by a Scientific Advisory Board of International aHUS clinical experts.
* this was a misinterpretation of the website information about where data was being collected as the website entry had not included all the enrollment sites.
And that was that as far as I was concerned. I knew the Registry would be operating until 2025 and so I expected no more than to see updates of what it discovers.
Then in June 2014 I got an e mail. It was from someone who was managing the Registry. The Scientific Advisory Board of the Registry had discussed and agreed at its May meeting that it should have patient input to what it was doing. Orphanet and EURORDIS/ Rare Connect had referred the Registry official to aHUSUK.
After the e mails and a telephone conversation .followed by a face to face meeting I agreed with the Registry officials that it should be the aHUS alliance ( of which they had no knowledge) and not just aHUSUK which provides a patient representative; but I could not make that decision. It would have to be agreed by alliance affiliates at their next meeting, planned for November.
At its first meeting in Barcelona the alliance had identified working with international aHUS Research Networks as one of its aspirations. It now had the opportunity to do just that. It could provide expert advice for healthcare and research for the benefit of aHUS patients globally.
Meanwhile there was the small matter of the NICE evaluation of eculizumab in the UK.
Read more about the Reluctant Advocate story HERE.
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Len Woodward is a member of the aHUS Registry Scientific Advisory Board to provide advice to the Board on global patient issues on behalf of the aHUS alliance. If any aHUS patient or carer needs to contact him on matters relating to participation in the Registry, he is contactable at firstname.lastname@example.org