Representation, Role, and ALS: Part I – Raising the Right Questions

To have ALS is to lose your voice twice – both literally and figuratively. The disease gradually robs you of the physical ability to speak.  And for most people with ALS, having a voice in ALS advocacy is a luxury they cannot afford.  They are too preoccupied with immediate physical, emotional, family, and financial concerns.

The rapid progression of ALS makes us a challenging group when it comes to patient advocacy (which I am defining in these essays very broadly, from public policy to care service priorities to clinical trial research design).  Most of us just don’t live long enough to be able to play a sustained role. For the most part, we have had to rely on others to speak for us – mainly a combination of patient advocacy specialists, medical professionals, and former caregivers and friends who have made a commitment to fight the disease in honor of a lost loved one. The staff, boards, and volunteer groups of virtually all of our ALS organizations are heavily populated with this combination of activists.  They are very committed and generally do a fantastic job, and we are very grateful.

But can people with ALS play a more prominent role? Should we? And if so, what kind of role is appropriate or even essential, and what roles are inadvisable? How can we best make ALS advocacy patient-centered and patient-led when our post-diagnosis life expectancy is generally so short? What is the most effective strategy under these circumstances?

This is an especially salient set of questions given the growing movement for patient centric healthcare and patient empowerment at all levels, including in the ALS community, where patient-centrism is now routinely invoked as a core value for clinical trials, advocacy, care services, and data collection .

I understand that patient-centrism doesn’t necessarily entail direct involvement of patients in the process – sometimes it can mean simply prioritizing patient needs and rights – but more and more of its current usage implies a direct role for patients.

I have been struggling with these questions over the past several months, trying to figure out what patient-centrism means in the ALS context, how and where people with ALS can bring a differentiating strength to policy conversations, and where we can’t.  I worry that the concept of patient-centrism in the ALS context risks becoming a fashionable but empty buzzword, or merely another box to be checked by organizations. It is easy for us to throw around expressions like “patient-led,” “patient empowerment,” and “nothing about us without us.” But what does that mean in practice?

People with ALS (pALS) can and do play an impactful advocacy role, of course. Indeed, we have been blessed with some exceptional spokespersons over the years, starting with Lou Gehrig himself.  We have some extraordinary pALs leading advocacy efforts today, as well as many other pALS playing quiet, behind-the-scenes roles on committees and working groups, while others contribute via social media.  But our roles are usually brief. We command public attention in media stories, fundraisers, and congressional hearings, until the disease runs its course.  Another group of people with ALS steps up to play the role, and then another, and another.  With the exception of a lucky few whose disease progression was or is very slow, we are shooting stars, and we know it. 

We were all reminded of this reality with the release of an October 2019 statement by the ALS Association’s Patient and Caregiver Advocacy Group, in response to the release of the FDA’s ALS Guidance for Industry. That statement was prefaced by the observation that, of the 19 PALS who were first assembled as part of the advisory group in 2015, 9 had since passed away. Almost half of the pALS who joined the preliminary conversation on the FDA Guidelines died before they could see the Guidelines released. The hard reality is this — a typical cycle for passage of a single law, or completion of a clinical trial, is the rest of a lifetime for most of us with ALS. The clock runs much faster for us than it does for our healthy friends and colleagues.

But getting to the table and then living long enough to play a sustained role is only part of the battle. Even when people with ALS are at the forefront of advocacy efforts, that does not necessarily translate into “voice” for other pALS. We may or may not be representative of the wider community of people with ALS. We certainly mustn’t assume that we are.

Who can legitimately represent people with ALS? How can we ensure that we speak for the entire ALS community?   

I am a person living with ALS, but I am also a political scientist by profession, so issues of representation and legitimacy are of special interest to me.  I have grappled with the issue of “who speaks for whom” and “who has the right to represent whom” for over three decades in the very challenging setting of Somalia, where the prolonged collapse of the state from 1991 until recently has elevated the contentious question of representation at every peace conference, every development workshop, and every attempt to form a government.

This series of essays is my reflection on the problem of and prospects for voice, representation, and role in the ALS community. I do not pretend to have all the answers, but hope to at least ask the right questions. If any of this comes across as provocative or upsetting, I apologize, that is not my intent.  My hope is that these questions serve to spark thoughtful discussion.

And it goes without saying that I am hopeful that new therapies will soon be developed to render this entire conversation irrelevant.

Prologue I: Full Disclosure

I have multiple affiliations with ALS support and advocacy groups, which is intentional.  Most of my engagement is through the ALS Association, which has generously offered me opportunities to put my energies to use on numerous committees and working groups. I am also a member of the I AM ALS group, which describes itself as a “patient centered, patient led” organization. I am a newly certified NEALS Clinical Research Learning Institute “ALS Research Ambassador” which is dedicated to empowering people with ALS to be advocates for ALS clinical research and increasing “patient centric trial designs.”  I attend an ALS clinic supported by the Muscular Dystrophy Association. I have provided material for the Joe Martin ALS Foundation website and have benefited from direct support from the group.  I am active on numerous on-line ALS support group forums. I participate in numerous research projects with groups such as the ALS Therapy Development Institute.  I participate in and will play an advisory role in the group PatientsLikeMe, which was established to “transform healthcare by putting patients back at the center of the system.”

All that to say that I am ecumenical when it comes to institutional affiliation, and generally sympathetic to the concept of patient-centric approaches.

But what I’m most interested in, to the exclusion of almost everything else, is EFFECTIVENESS. What we need is discovery of therapies to slow, halt, reverse, and cure the disease. In the meantime, we also need to provide the most effective care and support to people with ALS and their caregivers that we can muster. And we need passage of legislation and development of policies that help to advance both quality of care and pursuit of a cure.  How that gets done, who does it, who gets credit for it, and what role people with ALS play, is really of little importance.  Empowering people with ALS to play a more prominent role in ALS advocacy work is only worth pursuing if it improves our collective ability to reach these goals.  If it doesn’t, we’re wasting our time,  or worse, getting in the way.

That makes it all the more imperative to calibrate our capacities with our roles for maximum effect.

Prologue II: The ALS Advocacy Landscape

Before jumping into specific challenges of representation, voice, and role in the ALS community, it’s worth doing a quick survey of the context of ALS advocacy. Old-timers to the ALS scene will learn nothing new here (though some will disagree with my assessment), but those of you new to the ALS scene may benefit.

The ALS activist community struggles with lots of issues, several of which are of special relevance for the challenge of representation: fragmentation and contested leadership, desperation, and lack of voice. All contribute to a fundamental problem of representation – namely, that all of our representative groups are self-appointed.

Fragmentation and Contested Representation. For a “rare” disease, we have a shocking number of foundations and advocacy and care service groups. Each organization makes claims about how what they do is unique and better than anyone else. They compete for donations and reputation and good press.  Some have staying power. Others don’t last – a quick forensic search of the internet reveals plenty of old, inactive non-profits and foundations set up years ago by or in the name of a person with ALS, as well as movements that flared up and petered out.

For their part, the people with ALS who choose to engage in awareness campaigns and policy advocacy – a very small percentage of the overall patient population — tend to cluster in their own groups, based on their views on therapy access, understanding of methodologies in medical research, attitudes toward the FDA and the ALS Association, institutional affiliations, personal friendships, ideology, and other factors.  If, like me, you prefer broad coalitions and have an aversion to in-group and out-group dynamics, this does not leave many attractive options. This crowded ALS playing field has reproduced the same kinds of unfortunate levels of factionalism and distrust that exist in many other patient advocacy communities (I just finished a book on The Politics of Autism and it served as a reminder of how factionalized patient advocacy groups can be).

There is nothing wrong with having multiple groups with different perspectives working on a policy issue – in fact, healthy competition and a wide marketplace for new ideas is essential.  But too much of it becomes a problem. It makes unity of effort much more difficult, it confuses and turns off potential supporters, it produces redundant initiatives, it bewilders newly diagnosed pALS,  and it increases the amount of funds spent on administrators and overhead, as each organization typically has to have its own corps of paid staff.

The good news is that there are ways to manage fragmentation better. More on that later.

This fragmentation of the ALS community also exacerbates problems of representation, by multiplying the number of organizations, movements,  and individuals claiming to speak for us.  The biggest, the ones who have the time and inclination to dominate social media, and the ones with the best social and political network tend to get the most attention. Some are great, some are ok, others may be cause for concern. But all of them are self-appointed. I do not recall voting for any of them, and I do not accept at face value their claims to represent me, even when I find myself in agreement with them. The burden of proof is on those of us claiming to represent others.  I will come back to this challenge later. Happily, this too is a solvable problem.

Desperation.  The fast progression of ALS, the lack of a cure, and promises that therapies may be “just around the corner” breed a lot of anxietyamong pALS, for totally understandable reasons.  We are, as a group, “patients who have lost our patience.”  Some pALS lash out at the very organizations that are, whether we like it or not, our best hope – the ALS advocacy and service groups, the funders of research, the fundraisers, the researchers, and the FDA, the government agency responsible for protecting consumers against ineffective or dangerous drugs. 

The current climate of political populism in the US and globally has made this kind of visceral distrust of institutions and expertise very attractive both to elements on the right and the left of the political spectrum.   And it makes agreement on legitimate representation all the more difficult.

My experience with the ALS advocacy, service, and research organizations that I have had the chance to interact with has convinced me that this anger is misdirected.  The people I have worked with in ALS groups and the researchers I have met have been uniformly hard working and committed to the cause. The expertise of the paid staff of these groups is invaluable and hard fought. Most of these organizations operate mainly with volunteer labor on their boards, committees, and fund-raisers. They make considerable sacrifices for us, and are not in it for the money. Whether one agrees or disagrees with each policy decision they have made over the years must not obscure the fact that we need them. We cannot defeat ALS without them.

Even so, some pALS question their role as representatives of the ALS community, as is their right.

Lack of Voice. Some people with ALS chafe at having to rely on others to represent them. It doesn’t matter that those advocates are well-intentioned, expert in their field, and committed. It can be hard to have to rely on others to speak on our behalf, and frustrating when it feels like they aren’t listening, playing out old rivalries and grudges at our expense, or assuming that we are too uninformed or emotional to play a constructive role. Frustration with this lack of direct voice is one of the drivers that periodically gives birth to patient-led advocacy movements.

So, whether we’re a person with ALS, a former caregiver, a professional advocate, or a medical researcher, when it comes to speaking on behalf of people with ALS, we’re all self-appointed. That’s just one more part of the ALS landscape we can’t change.  The good news is that this is a manageable problem, and something I’d like to explore in subsequent essays

The next essay deals with the challenge of representation and exclusion in the pALS community.

Author: Ken Menkhaus

Professor of Political Science at Davidson College. Specialist on Somalia and the Horn of Africa. Interests include development, statebuilding, informal governance systems, peace and conflict studies, and political Islam. I also teach on philanthropy and the non-profit sector.

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