Representation, Role, and ALS: Part III — The Steep Learning Curve

People living with ALS who are seeking to join ALS advocacy efforts face the daunting challenge of getting smart fast on multiple issue areas, some of which are exceedingly complex. We need to quickly acquire an understanding of, among other things:

  • the disease itself;
  • basic protocols of scientific research and clinical trials;
  • debates over new clinical trial designs and research protocols;
  • statistics, basic and advanced;
  • state of the art survey techniques;
  • biology, bio-chemistry, neurology, and genomics;
  • the medical research currently being undertaken to develop therapies for ALS;
  • the organizational landscape of actors working in and on ALS;
  • the history of ALS advocacy, conflicts, therapies, and legislation;
  • the politics, public policy, and legislation related to ALS, including preferred access and Right to Try;
  • the policy making process related to legislation relevant to ALS;
  • the regulatory role of the FDA;
  • trends and best practices in patient advocacy and empowerment;
  • funding modalities for ALS research;
  • coordination initiatives in ALS research and data collection;
  • coordination issues and opportunities within the ALS advocacy and fundraising community;
  • the functioning of non-profits, including standard fund-raising and endowment practices and the role of boards;
  • partnership arrangements between government agencies, non-profit funders, non-profit research institutes, and pharmaceutical companies;
  • the interests, risk calculations, and drug development strategies of pharmaceutical companies;
  • the role and impact of third party interest groups on ALS-related legislation;
  • new developments in assistive technology;
  • medicare and insurance issues related to pALS;
  • The Veterans Health Administration and its support to veterans with ALS;
  • wider issues related to the US health care system as it impacts pALS;
  • financial and care support issues for families caring for a pALS;
  • public policy and health care systems in other countries and implications for care; services and therapy access for pALS outside the US;

and so on… you get the picture.

Some of these topics can be mastered relatively quickly. But most cannot. Most are fields of knowledge that involve years or decades of advanced education and/or work experience. Most people working on ALS are specialists in one or a few of these issue areas, and have spent a professional lifetime working on them. The knowledge base needed to engage on these issues constructively is just not something one can casually pick up on the internet or at a weekend training workshop, though those can be good points of departure.

So pALS aspiring to play an advocacy role face an obvious problem. In order to equip ourselves with the necessary knowledge to play a constructive role, we need time – lots of time – to immerse ourselves in one or more of these issue areas. Yet time is the commodity we are most short of.  By the time we are minimally competent to weigh in on some of these issues, many to most of us are dealing with advanced disease progression that makes it hard to play a role.

In some cases, pALS already possess professional qualifications that give them a big leg up in understanding one or more of the issues listed above. We have pALS who are or were physicians, medical researchers, statisticians, biologists, economists, nurses, executives in pharmaceutical companies, health insurance executives, physical therapists, policy specialists, and a variety of other relevant professional backgrounds.  They are a great asset, and can quickly be put to excellent use.  Some share their expertise on blogs that are great guides for the rest of us. But this is a small sub-set of us, and even with these skills, applying them to the specific case of ALS can take additional time and training.

I’ll take myself as an example. As a political scientist with decades of experience in policy work, you might think that at least the policy-oriented issues would be in my wheelhouse and easily mastered. Wrong. I have worked on policy issues related to foreign policy and international development, not health care. They are worlds apart. When I told a colleague I was hoping to shift my professional energies to ALS policy work, her eyes got wide and said something to the effect of, good luck, health care policy is a lifetime specialization. I have books on my desk that I am plowing through to try to acquire basic competency in the field.  One, an award-winning book on the FDA, is 802 pages long (and devotes exactly one page to ALS, which is a very sobering reminder of where we stand in the big wide world of medical research). And the FDA is only one part of a much bigger galaxy of actors on the ALS scene. I have a lot of homework.

“A little learning is a dangerous thing.” We all know this quote and appreciate its meaning. I have spent the better part of a year trying to learn as much as I can about most of the issues listed above, and freely confess that I am now at that point where “the more I read, the less I know.” That is, I’m getting just smart enough about each ALS issue to appreciate how little I actually know, and how naive it would be for me to wade into some of the most complex policy and medical research design debates.  

Does this mean pALS should abandon the quest to play a role in ALS advocacy and research debates, and just leave it to the experts? Absolutely not. As I will argue in a subsequent essay, I think we have lots to offer, and absolutely need to be at the table in discussions about ALS research, public policies, care services, and the position ALS organizations take.

I will also argue that for many of these issue areas we don’t need to be expert, we just need to meet the “good enough” criteria – we need to know enough to play a constructive role. We don’t necessarily need to be in a position to provide answers — we just need to know enough to ask the right questions, or mobilize to get an issue on the front-burner for policy-makers and then let experts take over from there. We also need to know enough not to get played by parochial interest groups looking to use us to advance their own agendas or line their own pockets.

But we need to be very clear-eyed, precise, and modest about what we do and do not know, what the knowledge threshold is for basic competence, and what roles we should and should not play.  We need to treat the concept of patient-centrism pragmatically, not dogmatically. Above, all, we need to enter into these discussions with a “healthy” respect for science and expertise (healthy meaning critical but not dismissive).

I say this because some ALS advocates appear to want to solve the “steep learning curve” problem by simply minimizing the importance of expertise at all, or by arguing that the requisite expertise is something that pALS and others can acquire quickly. Those are assumptions that worry me because I believe they set us up for failure and embarrassment, and risk reinforcing the view among some experts that our involvement creates more problems than it solves.  If we’re going to engage, we have to get it right.  And getting it right involves doing a lot of homework.

In the next essay I try to unpack the concept of “patient centrism” and try to identify areas where pALS have essential or useful roles and where we may not.

Representation, Role, and ALS: Part II — Our Silent Majority

All advocacy groups, even mass movements, face challenges of exclusion and elitism to varying degrees. So do we.

I contend that the problem of exclusion and marginalization in ALS advocacy is acute, and generally goes unacknowledged.

If we are going to claim to represent the ALS community in its entirety, it’s critical to come to terms with the fact that we have a “silent majority” and to devise strategies to better represent them.

What specifically is the problem?  The small circle of us pALS who are active on ALS awareness campaigns, policy advocacy, and research protocol debates are drawn disproportionately from pALS who are (1) slow progression cases; (2) the professional and/or relatively wealthier sub-set of the population; and (3) American. This is an observation based on repeated participant-observation at ALS workshops and meetings, and from review of the profiles of pALS activists in ALS organizations and on social media. To be sure, people with normal progression and/or people of more modest income levels take part in our work and our meetings, and some important contributions are being made by pALS from outside the United States, but they are a small part of the activist community.

How exclusive are we as a group? I did some back-of-the envelope calculations and came up with this set of figures. First, we know that 20% of people with ALS live five years or more with the disease, and if we count that cohort as slow progressors, that gives us an 80%-20% ratio of normal progression versus slow progression cases.

As for wealth and professional status, this is trickier, but if we use $100,000 in annual household income as the threshold dividing wealthier US households from the rest (this varies according to location – you can calculate your own with this tool), we can see that roughly 30% of the US population enjoys an annual household income of $100,000 or more, and 70% fall below that figure. 

I applied these percentages to the matrix below and the results are sobering.

Typology of (US) pALS activists

Poor to average income 70% Wealthier and/or professional class 30%
Normal progression 80% The core of the silent majority,  56% Occasionally powerful, but short term roles  24%
Slow progression 20% Modest role, mainly in social media 14% Dominant role  6%

The quadrant of slow progression, wealthier, typically professional pALSs  from which most pALS activists come constitute just 6% of all pALS, if you accept how I defined and measured the variables. But even if you dispute the metrics I used (and I accept that they are imperfect), the general point I’m trying to make is still valid – those of us pALS who serve as representatives or opinion leaders in ALS organizations and on social media represent a very small slice of the community.

Our problem of representation gets worse when we take race and ethnicity into account. To the extent that race and ethnicity in the US are strongly associated with income levels (a correlation that has been amply demonstrated in countless studies), we should expect low levels of involvement of pALS who are also people of color. That, indeed, turns out to be the case.  Meetings involving pALS are overwhelmingly white.  To cite just one example, check out the 13 years of group photos of NEALS CRLI participants on the CRLI website and see if you can spot any or many people of color (For the record, this is not a criticism of CLRI! They are working with the pALS who nominate themselves).

I know that ALS occurs somewhat more frequently among Caucasians than among other ethnic groups, but even taking that into account, people of color are still quite under-represented in our ranks.

Finally, the pALS activist community is dominated by American voices to a surprising degree. It makes for a very insular conversation, one focused entirely on policy and access issues that are often of very limited relevance to the hundreds of thousands of pALS living outside the US. We do sometimes hear from pALS in more affluent countries of the world. But when it comes to PALS living in the global south, who may well outnumber the rest of us, they are almost entirely voiceless.

We are, in sum, not a very diverse group. The typical profile of a pALS activist is white, American, professional, relatively financially well off, and slow progression.

So what? Is that a problem? Not necessarily. Not if we take the time to listen and understand, and then have a commitment to represent the voices of those who lack the ability, time, resources, or desire to play a part in ALS advocacy.

But I am unconvinced that we have in fact done that work. For one thing, we lack the tools to connect with the silent majority, even those in the US. Many in the ALS community never make themselves known to the rest of us. They may be less likely to continue to attend clinics because the clinics are too far away and too hard to get to, or because they don’t see the point. They are less likely to register with the national ALS registry. They are less likely to indulge in twitter or facebook or start their own blogs. If they engage with the rest of us at all it is most likely to be on forums designed to share advice on coping with the disease. Those with fast or normal progression may simply want to spend their remaining time doing things they love with those they love, and not get bogged down in advocacy work.  

We can reach this silent majority, but to do so will take a level of effort and commitment, and an active strategy of outreach, that we have not attempted to date.

Importantly, the organizations that have done the most to reach out to our silent majority (at least in the US) are the care and service outreach groups – the interdisciplinary ALS clinics supported by ALSA and MDA, the on-line support group forums, and the host of local and regional ALS support groups across the country – like Team Gleason, the Les Turner ALS Foundation, and the Joe Martin ALS Foundation. We do a better job of inclusion in our care and support outreach than we do with our advocacy. I feel much closer to the voice of the average pALS when I exchange ideas on on-line support groups like ALS Forums than when I follow ALS twitter wars or attend an ALS workshop.

The absence of voice of that silent majority matters because those of us with the combination of slow progression and more privileged backgrounds have – I contend — very different interests than the other 94%. As slow progressors, we stand a better chance of surviving long enough for therapies to be developed to stop or reverse the disease. And as more affluent pALS, we may be a bit less worried about the enormous financial impact of the disease on our families. We might, therefore, be a constituency with a far greater interest in devoting funding toward a cure than toward care services. By contrast, families with more modest means, and pALS whose progression is rapid, may be less likely to focus on advances in therapies from which they have little hope of benefiting, and may be much more likely to prioritize essential care services and support.

This is just informed speculation on my part.  Only very good survey data can help us answer these questions authoritatively (Spoiler alert – “very good survey data” is going to be my answer to many of the problems this series of essays addresses….).

We need to be mindful of the fact that the group of us playing roles in ALS advocacy are not necessarily speaking for the whole group at this time. And those of us who are relatively privileged in our disease progression and assets have a powerful ethical responsibility to ensure that we take into account how policies and funding allocations impact the majority of pALS, who are either faster progression cases, or who are from households with modest financial assets, or both. If we are going to claim to represent pALS, we must be sure to represent ALL pALS.

This same logic applies to all of the ALS organizations and activists that advocate on our behalf.

The next essay explores “the steep learning curve” — the problem of acquiring competence to engage on ALS advocacy issues.

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.

Health update, late October 2019

I’ve gotten a number of inquiries from friends asking how I’m doing, so I thought I should share a quick update.

I just had a quarterly consultation with my ALS clinic this week, and it’s again mostly good news. My vitals, my breathing, and my core and upper body strength are all great. My right leg is maintaining its strength. The setback I had starting in summer — a much weakened left foot — remains a problem, but does not appear to have gotten worse since the summer. So, I’m a bit slower and more wobbly on my feet than 6 months ago, but otherwise stable.

I’m still doing most of what I want to do. I can still walk 1-2 miles at a time, and recently played a couple of rounds of golf at the beach without trouble (in fact, Tom Kazee and I tied for first in that member-guest golf tournament, earning each of us gift cards for redemption at the Caledonia golf pro shop, so I got some nice Caledonia golf swag! Thank God for high handicaps). I also traveled all the way to Kenya for a workshop and managed the airports fine. I went to a Clemson game with brother Pete, and visited my sister Susan for her birthday in CT and did one mile of the local Walk to Defeat ALS with her, our parents, and some of Susan’s friends. My family and I are planning a big “bucket list” trip this winter to tour the Grand Canyon and southern Utah, which I’ve always wanted to see.

Realistically, this is about the slowest disease progression I could hope for. With each passing month I have more hope that I will be a true “slow progression” case. Time will tell.

I am now using a cane, just as a precaution against falling, and have been fitted for foot braces (AFOs) and will start to use those to reduce the risk of tripping because of foot drop. So far, no falls, but I definitely notice that I’m working harder to maintain my balance, a bit like one feels on a ship at sea.

Getting to work each morning got a lot easier thanks to my brother Pete and sister in law Stephanie, who loaned me their golf cart, which is fitted out with everything to drive legally on streets with speed limits 25MPH or less. That allows me to park right up against my classroom building at Davidson College and has been a godsend.

Karin and I are doing some early planning to get ahead of the curve for when the time comes that we need to make bigger adjustments to my declining mobility.

Meanwhile, I continue to teach full-time at the college. I’m staying very engaged in ALS advocacy work with the ALS Association and am starting to feel a bit better informed and better-positioned to start to weigh in on a variety of ALS policy matters. Karin and I traveled to an ALS training workshop in Florida in October and I have committed to serving as an “ALS research ambassador” for the group (Northeast ALS Consortium) that generously provided the training. I’ll be posting a series of essays on the challenges of ALS advocacy on this blog soon. Stay tuned!

And, as always, thanks for asking how I’m doing.