Adding value in patient advocacy is obvious. Or is it?
Those involved in patient advocacy constantly ask themselves “Am I achieving anything?” . They know they are doing things, but whether those things make an impact is another matter.
Those activities are diverse and require personal skill, knowledge and motivation to do. But does good come of it? Indeed how is that good to be measured and recognised?
This applies also to groups like the aHUS alliance. The alliance was founded in 2013 ( more information here) and depends on unpaid volunteers to add value to the care of aHUS patients globally . But does it add value beyond the efforts of national aHUS advocate organisations in individual countries? Indeed is there any substance at all to the rare disease mantra “alone we are rare , together we are strong ” in actual reality when it comes to aHUS patient groups?After all that was the basic question asked of the founding affiliate countries of the aHUS alliance “ are there things that can be done better together?”. If not there is no value in such an alliance.
The alliance cannot do everything and each year it tackles different aspects of aHUS, which fall within the alliance’s mission in moving forward to realise its vision of “good will come, together”. That good can come in many forms , as all aHUS patients and their families well know; but good health is paramount.
The three themes that the alliance chose to focus on, at the outset of 2017, to add value for this year were:
- Access to complement inhibitor treatment
- Patient Engagement
- aHUS patients’ research agenda
Access to treatment for those who still cannot get access to complement inhibitors is a major challenge, and, if not met , good will certainly not have come for all.
The alliance has done much to make visible the difficulties of access in the developing world in countries like India , South Korea , South Africa and Romania. It is supportive of national organisations in countries where individual hurdles exist.
It does not have funds to help, but it has the arguments to use with intransigent health care decision makers , who are refusing funding of treatment based on false premises, if needed. It also has the global social media contacts.
Everyone knows why funding is difficult, but, until a solution is found by the supplier and healthcare providers working together for patients , all that can be done is to appeal for them to find that solution.
The alliance also scans the horizon for new complement inhibitors and has begun a “Trials Watch” series of updates about trials of potential benefit to aHUS patients, and, which can inform them enough to engage and bring such trials to the attention of their own clinicians
Encouraging patients to engage in their own heath care whether privately or in public has featured strongly in the alliance work throughout the year. The alliance website content has grown significantly in that time , providing more information and knowledge to help aHUS patients to engage more confidently with their care providers. That is where patient engagement needs to be strong. As one Doctor said at a Research conference this year it is “Flipping “what is the matter with you?” into “what matters to you?”” .
The next step is to go public as many aHUS patients and carers have done.The alliance has provided vehicles for doing that in the videos for Rare Disease Day and aHUS Awareness Day, and also in its Youtube channel for the patient’s voice.
It is worth repeating that most aHUS patients are not even aware of the voice of the aHUS patients on their behalf , and neither do they believe they have the means themselves to go public within the own country.
This is where the alliance has welcomed another affiliate organisation- aHUS Rest of World – to lead on delivering a founding aspiration of the alliance to encourage the start up and support for the development of newly emerging national patient organisations. Through that, small groups are forming in Poland, South Africa , New Zealand, Rumania United Arab Emirates and Israel. Even though we cannot provide financial support, expert knowledge of doing something like it ourselves can be of help at the start . What a delight it is to see self -determining ,self confident national aHUS patient organisations blossoming.
The alliance cannot do everything , indeed it could be said it cannot do much, so the collective effort of national organisations is of paramount importance in adding value. Any time that the individual countries can contribute to alliance efforts is a bonus; but not all are capable of doing so.
The aHUS patients research agenda has been created , at least it is in an “interim form”. The agenda is the result of work begun by the alliance working together to advise the aHUS Registry’s Scientific Advisory Board. It was built on by the 2016 aHUS Global Poll and finished off with the final piece of the jigsaw, the 2017 Rare Disease Day video. The latter was a novel ,and , probably, unique way to date in rare disease public patient engagement to produce something collectively for the aHUS patient community. It is not “official”, and individual countries may prefer their own agendas, but it is the first , it is visible and it is owned by aHUS patients everywhere.
It embodies the” its not what is the matter with you, but what matters to you” approach to patient included in research.
Furthermore after Rare Disease Day 2017, the alliance embarked on a extensive response to each participants questions, and, in doing so, provided a current status for each topic and much content for its website . All of which will not only help general readers understand aHUS, but also aid patients in their private patient engagement.
Inevitably, as with any plan during any year , something else comes along that needs to be addressed. Among the most important of these was the subject of Thrombotic Microangiopathy or TMA. It is only 65 years since the term was first coined and it has provide an immense challenge to those who try to diagnose the cause of it, particularly when it comes to diagnosing aHUS.
Understanding more about TMA even raises doubts about the real scope of aHUS patients organisations’ work as the “atypical” does not sufficiently delineate what those organisations have traditionally focused on as aHUS , not just with those with “typical” HUS,but also those with TMAs that stem from primary diseases , such as Lupus and Leukaemia, which have their own patient advocacy groups.
The alliance designed a symposium, which was held in the Conference Centre of Harvard Medical School to look at TMA “though the lens of aHUS” to reflect this realisation . Diagnosis , complement genetic mutations , pregnancy onsets and potential treatments with complement inhibitors are just some of the things that aHUS patients share with some of those with much more common diseases, but who also have experienced TMA.
The videos of the talks of the conference demonstrate too how patients are included and can publically engage with others about their disease.
The videos also featured in the alliance’s contribution to another aHUS Awareness Day in September, along with another alliance video in which aHUS patients and families got another chance to engage in public advocacy throughout the world. The alliance’s social media interactions about aHUS ,leading up to ,and on aHUS patients’ very own day, reached further than ever before. This year the events in Algeria and Brazil come to mind as an example of the ever increasing reach.
Whilst there is evidence that aHUS patient engagement has been enhanced by the alliance’s work this year ,and certainly an aHUS patients’ research agenda now exists , greater visibility of the challenge of TMA diagnosis, although raised, needs much more work . Perhaps more importantly, other than connecting people to complement inhibitor trials, the alliance has not made much progress on the challenge of access to treatment for all.
But on balance the alliance could be said to have added some value in 2017, although there will be those who might not agree with that.
Well even if we have not, we will still be around for at least one more year and hopefully will do something to make it a happier 2018 for more aHUS families around the world.
HAPPY NEW YEAR from all at the aHUS alliance
