ALS Policy, Advocacy, Research – Page 2

ALS future scenarios: Anticipating the impact of new therapies

Summary:

The possibility of new treatments in coming years that could, slow, halt, or reverse the progression of ALS demands advance planning across a wide range of issue areas and stakeholder groups. This paper reviews three of the most likely scenarios that new therapies could produce. It anticipates the impact of each scenario on ALS demographics, early detection, physician caseload, clinics and care services, fundraising, clinical trials, insurers and other sources of coverage, and on persons living with ALS (pALS), their caregivers (cALS), and their family finances.

It concludes that therapies that slow or halt the disease will produce major but manageable challenges to organizations dedicated to ALS care services, fundraising, research, and advocacy. Some of these challenges will require effective advanced planning to mitigate. Two of the most important emerging issues will include the need to develop much better early detection capacities, and the need for much higher levels of fundraising to cope with the dramatic increase in the number of people living with ALS.

Impact of therapies that slow or halt the progression of ALS on pALS, their caregivers, and their family finances will be highly variable, resulting in very positive outcomes and options for some, and very difficult choices for others about whether the new therapies’ benefits outweigh the costs.

A scenario in which a therapy reverses the symptoms of ALS for most or all pALS is obviously the most unambiguously positive and least complicated outcome. It will highlight the need to expand access, via international aid programs, to make the therapy affordable and accessible for the large and growing caseload of pALS in poorer countries. Advanced stages of the battle to defeat ALS will be waged in the global south.

Impact inventory

With several drugs and treatments now in Phase 3 clinical trials, and more in the research pipeline, medical researchers are increasingly expressing hopes that we will soon see one or more new ALS therapies approved and available, possibly within the next two to three years.

This optimism comes with lots of anxieties and uncertainties – about which therapies or combinations of therapies will prove to be effective, when they will be available, what percentage of people with ALS (pALS) will respond well to them, how much they will cost, whether insurance companies will cover them, and what kind of impact they will have on disease progression.

The latter question is especially critical. Will the new therapies slow ALS progression? Halt it? Reverse it?

Whatever the case, more effective therapies are going to have wide-ranging impact — on pALS, on pALS’ families, and on the wider ALS support, care, advocacy, research, and coverage ecosystems. It is not too soon to start planning and preparing for all of the opportunities and challenges that new therapies will bring, even if the first new therapies are several years away.

These need to be frank discussions, because not all of the impacts of new therapies are going to be uniformly positive. We have to be ready to handle both the good and the bad.

The good news is that organizations involved in ALS work are already thinking ahead about some of these issues. The ALS Association convened a meeting in late October to bring together representatives from pharmaceutical companies, the FDA, and insurance companies – with some pALS representatives actively involved — to jumpstart discussions on questions of coverage of potential new therapies. This kind of forward thinking is crucial to help us get ahead of the curve on all of the issues that a new therapy will raise.

This paper is intended to anticipate the impacts of new therapies, based on three different scenarios. All of them over-simplify what will likely be a much messier reality. First, they assume uniform impact of new therapies, even though we know that variability of impact is much more likely, given the heterogeneity of the disease. Second, all refer to a single new therapy when we know that a more likely outcome will be an effective “cocktail of therapies” that will emerge gradually rather than all at once. And all are based on impacts in the US setting. In other countries with different health care systems and income levels, aspects of these scenarios could play out quite differently.

It should be noted that though these are presented as separate scenarios, they could, and probably will, occur sequentially.

The author welcomes suggestions for amending this first cut at an “impact inventory” and hopes to update the paper using reader feedback to improve the analysis.

Scenario 1: Slowing of progression

All of us living with ALS are hoping for new therapies that reverse the disease’s progression or even cure it. But we have to be prepared for more modest advances.

One possible outcome of this current round of new therapies could be a drug, a combination of drugs, or a gene therapy, that does not reverse the disease for most of us, but that significantly slows it down.

Let’s assume, for the sake of argument, that a therapy is approved by 2021 that on average halves the speed of progression. That will double the median, post-diagnosis life expectancy of people with ALS from 3 to 6 years. For pALS with slow progression, they could see their life expectancy leap from 7 to 14 years, or 10 to 20 years, or even more.

What are the implications?

ALS demographics. This will potentially double the number of people living with ALS. In the US, our numbers will quickly grow from 30,000 to 60,000, assuming most everyone has access to and uses the new therapy.

In fact, this could actually be a significant underestimation of the total number of cases. How so? The aging of the US (and global) population is expected to naturally increase the number of ALS cases in coming decades, even without any new therapies to slow the disease down. One recent study estimates a 69% increase in the number of people with ALS globally from 2016 to 2040 (from 223,000 to 337,000), solely based on aging of the population. Preparing for a sizable increase in the number of pALS seems prudent even if no new therapies are developed in coming years.

To borrow from the terminology of epidemiologists, ALS incidence rate (rate of occurrence of new cases, often expressed as the percentage of new cases of a disease per year per 100,000 people) will increase simply because of the aging population. The ALS prevalence rate (proportion of total cases in a population at a given time) will leap both because of the impact of the therapies that extend life expectancy, and because an aging population will produce more cases of ALS.

It is worth noting that this level of increase in the population of people with ALS in the US would not change the FDA and NIH’s categorization of ALS as a rare disease (defined as fewer than 200,000 affected by the disease), so ALS therapy development would continue to benefit from the provisions of the 1983 Orphan Drug Act.

Early detection. Drugs that significantly slow disease progression will have much more positive impact if paired with early detection, which has been a chronic problem with ALS diagnosis. The need for earlier detection will be urgent in this scenario. This will require primary physicians and neurologists to be better trained on ALS and made aware of the need for speed in diagnosis. If delayed diagnosis remains a problem, drugs that slow progression down will have limited impact, as they will simply arrive too late to extend life expectancy as much as they could. They will also miss the opportunity to extend the initial period of symptom onset, when symptoms are more mild and when pALS are most physically functional.

ALS physician caseload. For ALS neurologists, this scenario will, within four to five years, double the number of cases they have to carry. They could cope by reducing the number of annual consultations with pALS from quarterly to bi-annually. But how would that impact data collection, and quality of support to pALS, especially those who may not respond as well to the treatment? Another response could be policies to promote expansion of the number of newly trained neurologists who specialize in ALS. In the short term, do we have enough ALS specialists in the medical school pipeline to respond to heightened demand? Recent reports suggest that in the coming decade the US will actually be facing a serious shortage of doctors, so the odds of increasing the number of neurologists specializing in ALS may not be good. The physician caseload in this scenario looks to be a potentially serious bottleneck.

ALS inter-disciplinary clinics, and care and support services. For ALS interdisciplinary care and support work, this scenario will significantly increase demands on their services, doubling the requests for equipment, counseling, respiratory therapy, in-home care-giving, and other support. Rationing existing resources and care support would constitute a major drop in quality of care. Responding fully to new demands would require a major ratcheting up of fund-raising efforts, as well as an expansion of hiring of specialists and expansion of new clinics. The possibilities of harnessing new technologies to reach new pALS with telemedicine and virtual care could partially offset the spike in demand for care and support services, but would be no panacea.

Fundraising. The need to increase fund-raising for a doubling of care and support services will be acute. This will very likely exacerbate tensions between those who want to prioritize funding for research for a cure and those prioritizing fundraising for care services. Even though care services and research are much more closely interconnected than most of the public realizes, tensions over allocations of funds for research versus care services are real, and will be aggravated by a doubling in need for care services.

The solution — a successful ratcheting up of fundraising to fully meet all needs — is easier said than done. ALS competes with many other worthy charitable causes, and in the US total charitable giving has been flat despite a long period of economic expansion. Powerful public awareness campaigns will be required to explain why advances in ALS therapies are actually increasing the need for more funding.

Insurance companies, Medicare, Veteran’s Health Administration. For insurance companies, Medicare, and the Veteran’s Health Administration, the cost of new therapies and the doubling of patient life expectancy will create additional payouts, but given the relatively small number of cases this will not be a significant problem. The high cost per patient of ALS cases could, however, create pressure for cost control or create access problems. Some insurance companies could decline to cover expensive new “orphan drugs” if they have doubts about evidence of effectiveness.

Research and clinical trials. If most of the population of pALS is taking drugs that significantly slow down disease progression, how will this effect eligibility criteria for new clinical trials? Will it make it harder to discern positive impact of drugs in clinical trials? We know that slow progression cases tend to confound clinical trial results. In this scenario pretty much all pALS would become, to varying degrees, slow progression cases. This author is not expert on clinical trial design issues, so cedes the floor to others on this question. But it seems to be a question worth raising now.

The heightened importance for the need for early detection and screening will likely lead to calls for more research to advance that capacity.

Social Security disability insurance program. Our longer life expectancy could potentially raise questions as to whether newly diagnosed pALS should continue to receive expedited access to disability benefits, since at least some pALS will have the capacity to continue to work for a longer period of time (depending on their line of work and disease progression).

People with ALS, their caregivers, and their families. This is by far the biggest issue to consider.

First, the impact of this scenario on pALS, caregivers, and families will clearly be highly variable.

For some pALS in advanced stages of the disease, this scenario may be perceived to bring minimal benefits, as it will only prolong the most difficult, final phase of the disease when quality of life calculations can come into play. Some might have good reasons to want to extend their lives nonetheless — Stephen Hawking serves as a reminder that pALS who are entirely paralyzed can continue to pursue meaningful and productive lives. But some in late stages might forego treatment completely.

For most pALS and their caregivers, the benefit of living twice as long with the disease will be weighed against the doubling of the period of time of intensive, expensive home care for the pALS. This will put huge additional demands on caregivers, and huge additional expenses on family finances. A therapy that only slows the disease down could inadvertently produce a spike in ALS-related bankruptcies and could push some caregivers to the breaking point. Here we could expect to see a big split in choices based on household wealth. Those with ample resources will be in a better position to choose to extend their lives than those with modest resources.

The pALS who will stand to gain the most from this scenario are those in early stages of the disease, and those with slow progression. They will potentially be able to live many more years than they expected, and could remain in possession of most of their physical capacities for much longer. Some could live long enough to end up dying of other causes. Importantly, this scenario will also buy them more time to benefit from future medical breakthroughs that could reverse or cure ALS. But the flip side of the coin is the prospect of a very long period of partial or total incapacitation, with all of the difficulties that entails for the patient, the caregivers, and family finances. Some might opt for a two-track approach — taking the therapy during the period when they have retained most of their physical capacities, but then dropping it once they reach advanced stages of the disease.

Assessing Scenario #1. This scenario produces sizable but mostly manageable challenges for organizations involved in care, support, research on, and financial coverage of ALS. Impact on pALS and their caregivers will be more problematic and variable. The possibility of new therapies that significantly slow but do not halt or reverse ALS will be a positive development for some pALS, but a Faustian bargain for others. PALS and their caregivers and families already face very difficult and emotionally wrenching decisions on whether to use or forego procedures that extend life. This scenario will significantly increase the difficulty of weighing the costs and benefits of using therapies that extend life expectancy.

Scenario 2: Halting of progression

In this scenario, a new drug or combination of drugs effectively stops further disease progression, but does little or nothing to reverse damage already done. Everyone with ALS who gets this treatment must live with symptoms that are present at the time they start the new therapy. ALS is transformed from a fatal to a chronic disease.

What are the implications?

ALS demographics. In this scenario the total number of people living with ALS in the US will steadily increase by about 6,000 per year, less the (initially) small number who die of other causes – a number that will increase as the population of pALS ages. Moreover, as the American population ages (the total number of Americans over the age of 65 will double between now and 2060) the total number of new ALS cases is expected to rise well above 6,000 per year, increasing the number of people living with ALS even further. It would take a skilled team of demographers, epidemiologists, and statisticians to give us an accurate estimate of the total number of people living with ALS by 2040-2050 in this scenario. But we can all do a quick back-of-the-envelope rough estimate, and reach the conclusion that the number of people with ALS in the US could easily reach the hundreds of thousands.

Early detection. In this scenario early detection becomes an absolute priority. Every month of delayed diagnosis will mean permanent loss of some functionality for a person with early stage ALS, losses that could have been avoidable. Delayed diagnosis will be viewed as malpractice, not as an unfortunate reflection of the slow, “process of elimination” approach to determining if someone has ALS. Ideally, if advances in diagnostic tools are developed, detection will be done before patients even reach the diagnostic threshold, so that treatment can begin before symptoms appear or advance. Primary care physicians and other medical professionals who are often the first point of contact with people with possible ALS will need to be given much more training and guidance on referrals of possible ALS cases to specialists. Protocols might be needed to give possible but not definitive cases of ALS access to the therapy.

ALS physician caseload. The ALS caseload will explode in this scenario and will require a complete rethink of the current clinical support system. There is no way that the current system, which sees 20,000-25,000 cases, could shoulder several hundred thousand cases.

This potential crisis could be partially offset by the possibility that the new generation of “stabilized” pALS might not need to see an ALS specialist regularly, or even at all once diagnosed and given access to the new treatment. They could instead be monitored by their family physician and only referred to the ALS neurologist if their condition changed, if they experienced side effects from the therapy, or had other complications. This switch in the site of care and monitoring could erode or complicate data collection on ALS cases, and could require a rethink of the role of clinics in trials of new therapies.

ALS inter-disciplinary clinics, and care and support services. For ALS interdisciplinary care and support work, this scenario will create dramatic swings in demands for services over time. Initially, demand will be steady, since the number of ALS patients with moderate to advanced stages of the disease, now with much longer life expectancies, will go largely unchanged. Over time, the profile of the total ALS population will shift, with a declining percentage of pALS in moderate to advanced stages of the disease, and a rapidly growing percentage and number of pALS stabilized in early stages of the disease. The type of care required will shift accordingly, with more support to deal with partial loss of limb or voice strength, and far less need for support associated with advanced stages of the disease.

Fundraising. Fundraising for care services would initially need to be increased, then over time reduced, as the needs of the pALS population shifts almost entirely toward early onset issues. Fund-raising for screening and early detection services, and training of additional specialists for this task, will become a major new priority. Fundraising for research to reverse and cure the disease will also become a dominant priority. Whether fundraising becomes easier or harder in this scenario is hard to know, On the one hand, the success enjoyed by new therapies could create momentum for more support for research. On the other hand, it might become more difficult to argue that ALS is a top priority when the disease is transformed from a fatal to a chronic condition.

Insurance companies, Medicare, Veteran’s Health Administration. For insurance companies, Medicare, and the Veteran’s Health Administration, the high cost of new therapies combined with the exponential increase in people with ALS will constitute a significant expense, though the price of new therapies will presumably drop over time. Since most people with ALS in this scenario will be dealing with stable, early onset symptoms, many more will continue to be able to work, so a disproportionate percentage of the costs will fall on private insurance companies, not Medicare.

Research and clinical trials. If the population of pALS is using therapies that halt disease progression, eligibility criteria for new clinical trials will have to move away from the current practice restricting participants to those with fewer than 24 months since symptom onset. For some clinical trials, clean samples of pALS might be needed, which will pose a challenge.

Research to advance capacity for early screening and detection will be a high priority.

Social Security disability insurance program. Once pALS have a drug or drugs that stabilize their condition, and presuming most are diagnosed in time to minimize symptom onset, a much larger percentage of pALS will be able to continue to work, though some may have to find new lines of work depending on their condition. This would likely lead to pressure to eliminate the expedited access to disability benefits for pALS.

People with ALS, their caregivers, and their families. The impact on pALS and caregivers will vary dramatically by generation and income level. Those with advanced stages of disease progression at the time the new therapy is made available will only benefit if they have a powerful reason to want to continue to live despite severe physical constraints and if they have access to financial resources to cover costs of caregiving. For others with advanced progression, the therapy may not be an attractive option, though some might opt for it anyway in hopes of extending their lives long enough for a new therapy that reverses disease progression.

For pALS in early stages, suffering from only minor to moderate symptoms, this scenario is a life-saver, and almost all will seek access to the therapy.

PALS who are newly diagnosed after the new therapy is made available will constitute a new generation of pALS for whom the disease is chronic, and hopefully – if diagnoses are made early enough – featuring only mild and manageable symptoms such as foot drop or slight speech impediment. The entire profile of pALS will shift over time.

Assessing Scenario #2. The halting of ALS will lead to an explosion in the total number of people with ALS, place significant new demands on sources of health coverage, require much more capacity for early detection and diagnosis, and create changing demands over time on support and care agencies. For pALS with advanced disease progression, this scenario presents them with very difficult choices. For the rest of the pALS population, the rendering of ALS from a fatal to a chronic condition will be a welcome new lease on life, tempered only by frustrations over whatever physical abilities have been lost to the disease. Hopes and expectations that new therapies will be developed to reverse disease progression will be high.

Scenario 3. Reversal of progression

This is, of course, a happy scenario to contemplate. For pALS and their families and friends, a therapy that reverses progression to produce a (more or less) symptom-free form of ALS will be met with tremendous joy and relief. The gradual recovery of strength and physical capacity once lost to the disease will be a tremendous emotional journal for pALS (though it is possible pALS in advanced stages of the disease could suffer some irrecoverable losses in functionality).

Almost all US-based pALS, regardless of stages of progression or level of income, will benefit equally from the prospect of a therapy that reverses progression. The exception will be those without insurance, Medicare, or VA coverage, who, faced with exceptionally high costs of the therapy, could face the prospect of bankruptcy and loss of their home before qualifying for Medicaid. This is a potentially solvable problem, if the right legislation were passed or policies developed, and one that will demand close attention as a matter of equity and social justice.

What would be the wider impact of a therapy that reverses ALS progression?

ALS demographics. The population of people living with ALS in the US as a chronic, largely symptom-free condition will eventually grow to several hundred thousand.

Early detection. Early detection will no longer be as urgent, since symptoms are reversible. Delayed detection will constitute an inconvenience and annoyance for patients dealing with temporary unwanted symptoms.

ALS physician caseload. For ALS neurologists, their work will shift to confirming diagnosis and trouble-shooting cases of complications.

ALS inter-disciplinary clinics, and care and support services. Most ALS interdisciplinary care and support will gradually be rendered unnecessary. Those serving groups with other neurological conditions will simply shift their focus to those patients. The wild-card in this regard is the degree of variability of impact of new therapies. If not all pALS respond positively to the new therapies, care services will still be in demand, though at more modest levels.

Fundraising. Fund-raising might be needed to continue to support some research on the disease – mainly devoted to finding a cure — but that would be a lower priority compared to other medical research needs. Most of the organizations dedicated to fundraising for ALS will either close down or repurpose their mission, as the March of Dimes did once a polio vaccine was developed.

The wildcard in fundraising for ALS would be the possibility of a major shift in focus from domestic to international programming – an option considered below.

Insurance companies, Medicare, Veteran’s Health Administration. For insurance companies, Medicare, and the Veteran’s Health Administration, the cost of a new therapy required over the course of a lifetime, for hundreds of thousands of pALS, will be a significant new expense. Over time, presumably, the cost of the drug or therapy will drop.

Research and clinical trials. Research in pursuit of an actual cure for ALS will still be conducted, but most researchers in this scenario will move on to work on other medical research endeavors, as ALS research will likely be viewed as a less urgent priority by the NIH and other funding agencies.

Social Security disability insurance program. PALS will no longer have any need to go on long term disability, though some might require short term disability if serious symptoms presented themselves and diagnosis was slow.

Assessing Scenario #3. A cocktail of drugs and therapies that reverses ALS will produce almost all benefits and very manageable costs, mostly related to insurance coverage. This is a dream outcome, and is much less fraught and complicated than scenarios #1 and #2. Even those people who lose their source of employment in ALS fundraising, advocacy, and care service organizations and have to move on to tackle other diseases will take great satisfaction that they put themselves out of business by doing what they set out to do — defeating ALS.

But many of the current team of ALS advocates and fundraisers may not have to shift to another line of work; they will simply have to shift to a different target population. ALS is a global scourge, and will require a major global effort to treat populations in the global south who are far from medical support that can diagnose and treat ALS. The energies and expertise of people working on ALS in the American context could be put to excellent use in an expanded initiative to provide treatment globally.

Global implications

It is worth briefly anticipating the implications of new therapies globally, especially in the global south, where most of the world’s people reside and where most of the cases of ALS exist.

One study (Arthur et al 2015) estimates that 223,000 people have ALS globally at this time, and that that number will increase 69% by 2040 even in the absence of a new therapy.

Three factors will drive this trend. First, the global population is growing at a rate of just over 1% per year, or 78 million people per year, almost exclusively in the global south. Second, life expectancy has increased dramatically in almost every country in the world, so many more people are now reaching ages when ALS is more common. Third, access to better medical care is improving globally, albeit unevenly, so we can expect that a greater percentage of people with ALS in the global south will soon be diagnosed properly.

The 2015 Arthur et al study observes that, without or without any new therapies, the weight of ALS “will shift away from the developed world towards developing countries.” It concludes that “[t]here are currently no studies outlining the cost of ALS in developing countries, and while economic costs are most likely lower in these locations, the increase in ALS cases will place an immense burden on their healthcare systems in the coming decades.”

Now, consider what would happen if a new therapy were made available globally that halted or reversed disease progression, and that most pALS who needed the treatment could access it. The global population of people living with a managed or symptom free form of ALS could grow about tenfold, to 3-4 million, by 2040.

But how will pALS in poor countries get access to new ALS therapies? The hurdles are daunting.

First, accurate and speedy diagnosis will be very difficult given the limited number of neurologists in the global south and the general lack of familiarity with rare diseases such as ALS among primary physicians. The wealthy cohort of people in the global south will stand a much better chance of correct diagnosis, but the rest of the population will face the risk of dying of ALS without ever knowing the true cause.

Second, even those who are diagnosed will face costs for therapies far beyond what most can afford and beyond what poor national health care systems can provide.

Third, calls for funding and support for ALS therapies will compete with demands for many other more endemic and more urgent health needs in the global south, and may not be a sufficiently high priority for some governments’ public health strategies.

The potential solution to these bottlenecks could be a much smaller variation of the global health initiative to combat HIV/AIDS. Since 2003, the US Agency for International Development has devoted $80 billion through the PEPFAR program to provide affordable, accessible treatment for people in the global south living with HIV/AIDS. It has saved an estimated 17 million lives in the process, rendering what was a fatal disease into a chronic one.

ALS organizations could play a critical role in international aid to raise awareness about, provide diagnoses for, and provide affordable access to therapies for populations in poorer countries of the world. This would be a hugely challenging assignment, but one which warrants preliminary planning now.

Next Steps

This paper is intended to jumpstart a conversation about how best to anticipate and plan for the impact of different ALS therapy scenarios. It is only a first cut at the topic, and an invitation to start a much wider and more thorough conversation.

Next steps could include:

Collection of feedback on the draft, to revise the paper and make it into a more robust white paper on the topic for general use.
Development of specific action items culled from the analysis.
Proposals to convene stakeholders at workshops, virtual workshops, or in electronic forums, for targeted discussion of the most important action items stakeholders want to address for planning for each scenario.
Agreement on a division of labor among the many ALS organizations for who plays the role of convener for which prioritized topics, to maximize inter-agency coordination and eliminate redundant efforts.
Consideration of use of surveys as a tool to acquire better information on issue areas we feel are high priority but for which we lack solid data. This would be especially valuable in anticipating likely pALS responses to various options created by new therapies. This too would need to be a coordinated effort by ALS organizations, to avoid redundant surveys and the risk of survey fatigue among pALS and their caregivers.

Comments and suggestions are welcome – this is a living document and the author expects to update it. Send your comments to kemenkhaus@davidson.edu . The analysis presented here is the author’s alone and does not necessarily reflect the positions of any of the ALS organizations with which he is associated. Many thanks to several colleagues who agreed to review draft versions of the paper and who provided very helpful comments.

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.

Lifetime risk, 1 in 300

In the world of ALS advocacy, there is no shortage of really difficult, even intractable issues. But sometimes you spot some low-hanging fruit, offering the promise of a quick and easy win, and you wonder why we haven’t jumped on the opportunity.

A case in point: We all share the goal of making the public more aware of, and more concerned about, ALS.

We could dramatically improve the impact of public awareness campaigns if we used different, more impactful metrics to describe how common the disease is.

For some reason, the websites of almost every ALS advocacy group in the US use two perfectly accurate but very easily misunderstood epidemiology yardsticks to depict one’s odds of getting ALS. One is incidence rates, or the number of new cases per year, which is 2 in 100,000. The other is prevalence, or total number of people living with ALS. Depending on who is doing this estimation, the prevalence is described as between 15,000 to 30,000 people living with ALS in the US.

Both of these are correct, but make the disease sound really, really rare. But that’s only because the general public isn’t familiar with how to interpret this kind of statistic. The 2 in 100,000 figure is one’s risk of being diagnosed with ALS each year. If the average age of an American is around 80 years, that means one’s odds of dying of ALS are actually a whole lot higher.

A much more impactful statistic would be lifetime risk of being diagnosed with ALS. A colleague in the UK, and a person living with ALS, Lee Millard, reports on a 2006 study in the Journal of Neurology (C. Johnston et al, “ALS in an Urban Setting: A Population Study of Inner City London”) which concluded that the lifetime risk is 1 in 300. One in 300! That is a statistic that has immediate meaning, and packs a powerful punch. A general reader can easily visualize a group of 300 people — it’s the size of typical high school graduating class. If people started envisioning the risk of ALS in the kinds of tangible terms that lifetime risk figures deliver, it would, I believe, raise awareness much more effectively.

In the UK, the flagship ALS group, the Motor Neuron Disease Association, has embraced the lifetime risk statistic of 1 in 300 in its fact-sheet about the disease.

Why aren’t we doing the same in the US?

I understand that some people dispute whether 1 in 300 is accurate, and they may insist on a higher figure. It doesn’t matter. Whether 1 in 300, 1 in 500, or whatever figure the data support, lifetime risk is simply a more powerful way of conveying to the public how much more common the disease is. And it solves the puzzle of how such a seemingly rare disease can be everywhere around us; how almost everyone knows one or several people who have died of ALS.

Undercurrents

All the fragmentation and divisiveness in the ALS advocacy community can be discouraging. But the past few days have also reminded me that just beneath the choppy and roiling surface are deep currents of cooperation, coordination, and commitment to unity of purpose. At the operational level, where it matters most, the community is doing a much better job of working together.

First was the news that three ALS non-profit research funders – ALS Finding a Cure, the ALS Association (ALSA), and the Muscular Dystrophy Association (MDA) – announced a joint $2.5 million grant to support a promising multi-year ALS clinical trial. That is a great example of combined efforts to fund research, which will yield greater efficiencies and scale.

Next, I traveled to my local ALS support group in Charlotte yesterday, and there was very pleased to hear how the three main ALS service providers in the area – ALSA, MDA, and the Joe Martin ALS Foundation – routinely work together to combine resources in support of people with ALS and their families.

Finally, this afternoon I joined a conference call for a task force assembled by ALSA to help conceive and execute an important project. I was blown away by the number of people willing to volunteer significant time to this project despite having full time jobs. Many to most on the task force are people who have lost a loved one to ALS and who are giving their time and expertise to help strengthen organizations like ALSA dedicated to finding a cure. Those of us with ALS don’t thank nearly often enough those many volunteers, who really make these organizations work.