Where and what next?

Article No. 297
15 November 2019


There is a saying in “management theory” that in the development of a “people organisation”  that it will go through phases  “Forming – Storming-  Norming –  Reforming. This is true of the aHUS Alliance as it is of organisations like Alexion.

Sometime after Alexion Pharmaceuticals was created (UDEC Pharmaceuticals registered 1992 in Delaware) it had one drug with some ideas of what it could be used for. It grew to be successful and around 25  years after it formed  it hit a stormy patch. Many of the original people lost out in the storm and a new organisation settled down again without them. But Alexion has not settled  for a norm and  is embarking on reform. That is evident in its communications.
In a recent article*, Alexion’s Chief Patient and Employees Experience Officer Anne-Marie Law explained that patients will be central to a cultural change within the company. A reformation.
Briefly some extracts from that article show that Alexion view “people with rare and devastating diseases are our inspiration” with a focus that there “will be a strategic focus on the patients we serve and our employees”, and that Alexion will be more than “delivering just our valued, effective medicines” by developing a business wide, “a renewed sense of purpose and greater empathy; inspiring and motivating us to think, act and deliver results differently”.  In the end Alexion’s “goal is to reshape how we approach our work and restate our responsibilities within a rare disease patient’s journey”.
As always these things are a journey and take time to do, but do give a  vision and sense of mission along the way.
For those with a rare disease it is something to be applauded. Something someone with any disease would applaud.
Simply hearing and acting on the aHUS patient voice is all that is asked.
Unlike ten or more years ago, Alexion now has different patients in mind when it talks about patients. aHUS Patients  were not its first market, as PNH patients came before them, and still do. Having invested so much in treatments for patients with other conditions it will be important to Alexion’s future that these are a success. So the aHUS voice is heard among other voices.
So what next for aHUS patients’ organisations?
aHUS patient organisation interactions with Alexion have not been a prime activity. When the early aHUS advocacy began, Alexion were well ahead in the development and application of eculizumab but those aHUS  pioneers brought about greater information and awareness.
It Is well known that traditionally patient organisations receive grants from Pharma to create awareness and the ability to do their voluntary work. It is something that has to be approached with great care,  unconditionally and maintaining independence. There is connection, there is an exchange of information. Done in a friendly and respectful way, but not as friends.
Around all Pharma (like any corporations)  there is forcefield of statutory regulations which must be complied with. Regulations established to ensure that no favouritism is shown nor  unfair promotion  impact on  competition and sales,  and no undue influence on heath decision makers. Great care is needed by patient groups who must be mindful of the duty of care and compliance they are obligated to fulfill through being a registered non profit / charitable organisation.
So any collaboration ,not just engagement. with Pharma  must be a transparent partnership  and solely to benefit patients. It must not seem, nor feel, that patients are being used.
Basically, that will mean things that can be done by  aHUS patients and families which matter to aHUS patients and families themselves. That will be a powerful force for good.
The Global aHUS Patients Research Agenda has been an example of turning “what is the matter with aHUS patients” into “what matters to aHUS patients?” What is relevant and valued by them.
An excellent starting point for Alexion to listen. But there are likely to be 20,000 aHUS patients around the world , how do their needs get articulated and captured.
For organisations like the alliance with an outlook which  embraces  countries in which Alexion has no marketing presence yet, a balance is demanded between the aHUS issues of the first world and those of the developing world, and what realistically it can contribute.
Like Alexion’s challenge for holistic organisational change so too will a reformation of global aHUS patient advocacy focus be needed to deliver what comes next.
Readers of this article, who are aHUS patients, or maybe just related, may well now be thinking about “ if and when the call comes” what will they do as self advocates for themselves or for others.

Where to, and what next might happen? A decision may be needed.


* The full article by Anne-Marie Law, Alexion.
Listening and Learning from the Rare Disease Patient
In the world of rare diseases, where so many conditions remain undiagnosed and untreated, we take seriously the opportunity to really listen to and learn from patients and caregivers and their healthcare providers. At Alexion, innovation includes really focusing our collective efforts to strive toward understanding patients’ needs, including meaningful therapies and beyond. We are focused on innovating in all we do to improve the day-to-day experience of people living with a rare disease.
This is particularly evident in our most recent FDA approval for the first medicine for neuromyelitis optica spectrum disorder (NMOSD), a rare, devastating and chronic autoimmune disorder of the optic nerve and spinal cord.
Our innovation in NMOSD first started in 2009 with a group of dedicated researchers and brave patients who paved the way. We are deeply thankful for all of the patients who participated in the clinical trials.
Truly transforming patient experience with this disease means delivering a medicine and more. It means actively listening to them, their caregivers and their healthcare providers to deliver services and support to address their needs.
Recently, we hosted Sumaira Ahmed, founder of the Sumaira Foundation, which is dedicated to “Illuminating the darkness of NMOSD.” Sumaira shared her story with our employees, including what the healthcare community needs to understand about a person’s experience after receiving this diagnosis.
Sumaira makes an impression on you from the moment you meet her: she’s a self-described fashionista with a vibrant personality and dreams of becoming a Bollywood actress. When I met her, I was both blown away and humbled by her energy and drive.

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When Sumaira started having vision issues for which she had no answer she visited multiple specialists. As her vision worsened Sumaira faced a hard truth: she was seriously ill, scared and needed answers. After additional evaluations, she learned she had NMOSD. True to her nature, Sumaira channeled this diagnosis into new purpose.
Sumaira shared her emotional journey with us, from the frustration of the initial diagnosis and worry about her future, to the fight for answers, the fatigue and isolation of learning how to manage the disease, and the desire to be a part of the decision-making process. She felt there was limited support or information for people living with the disease and took matters into her own hands by launching her own foundation to provide resources for the patient community.
We learned a lot listening to Sumaira and other NMOSD patients as part of our ownAlexion journey. We know that living with a disease like NMOSD is not just about the medicine. As a leading rare disease partner, we can and should step up with support services that not only help people living with the disease manage through the day, but help them live their best lives. And so, as we partner with NMOSD community, we are committed to continued collaboration to build innovative tools and networks that answer their call.
Thanks so much for reading my latest article from my #RareInspirationChangingLives LinkedIn series. I look forward to sharing more in the months to come. “

Note : The author of the alliance article, Len Woodward, has received payment, in accordance with statutory regulations, from Alexion for talks about aHUS advocacy.

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