Who should our patient advocacy organizations represent?

The answer to this question is, at first glance, blindingly obvious. The patients, of course.

But which patients? The ones who have the condition or disease and know it? Or the many many more who don’t have it yet but will?  

There are an estimated 25,000 people in the United States who  are currently living with ALS. I’m one of them.

There are an estimated 650,000 or more Americans alive today who don’t have ALS yet but who  will get the devastating diagnosis. Maybe in one year, maybe in five, maybe in 40.

Do our US-based ALS organizations have a responsibility to protect and advance the interests of the 650,000 too, or only those of us with the disease now?

It is a fundamental question, laced with moral and policy implications. Each of us volunteering or working in the ALS advocacy, research, and care service space has to answer that question for ourselves as well as demand an answer from our organizations.

If your answer is that our only task is to serve those with ALS now, then policies designed to advance short-term needs and goals should prevail.  

If your answer is that we have an obligation both to those of us diagnosed with ALS and the 650,000 Americans who will get the disease, then the calculus changes. Then the difficult challenge is to balance serving the interests of those with ALS now and the interests of the “next gen” of people with ALS. That balance can and must be achieved, but it requires us to be mindful of the many voices we can’t yet hear, all 650,000 of them.

We have to do better than this

What happened this month in the ALS advocacy space must never happen again. It’s up to those of us living with ALS and our supporters to insist that our ALS organizations do better.

What went wrong? Two of our most active ALS advocacy organizations, the ALS Association and IAMALS, were only partially in sync on two bills introduced in Congress designed to improve early access to experimental therapies.  One bill, S3872 (“Promising Pathway Act”), was enthusiastically endorsed by both organizations. But on a second bill, HR7071 (“Accelerating Access to Critical Therapies for ALS Act”), they were not in full agreement. IAMALS fully endorsed HR 7071; the ALS Association expressed reservations about the bill in its current form while expressing hope that  discussions can be held with the co-sponsors, IAMALS and other stakeholders to address those concerns and strengthen the bill. 

There is no reason that these mixed messages needed to happen after the bill was introduced.  This was avoidable, and should have been handled long before the bill saw the light of day. What was missing was communication, coordination, and trust, exacerbated by misperceptions.  I have heard enough to believe that there is plenty of blame to go around and both organizations could have done better.  I am uninterested in a blame game. What I am interested in is seeing that we do better moving forward.

Doing better means routinizing communication and coordination on public policy ideas, initiatives, and draft legislation; creating publicly accessible  forums for debates and discussion about possible advocacy ideas, so proposals get a full airing and a wide range of patient voices can weigh in on their merits or shortcomings; and building trust, without which progress on other fronts is impossible.

The good news is that in my conversations with both leaders and members of these two organizations (including many of us who are members of both!) this desire for improved coordination is  widely and deeply shared. And I strongly suspect that leaders in the many other ALS care services, research, and advocacy groups feel the same. Everyone understands that better coordination is essential for success, and is an ethical obligation to the community we serve. I am optimistic that we can make this happen. But we need lots of voices in the ALS community to keep insisting on it. As I settle into the role of Chair of the Association’s Public Policy Committee, I promise you this will be a top priority, and I will hold myself accountable to that goal.

I am also optimistic because, as I have gotten to know both volunteers and staff in our many ALS organizations, I am deeply impressed at how dedicated, smart, and hard-working they all are, and how similar our goals and values are. These are good people. They are having to work in a highly fractious ALS organizational environment, but that is something we can change, if we want to.

All this is not to say that ALS organizations must agree on everything. We have to expect differences in policy preferences, strategies, and principles. In fact, robust exchanges of views on public policy and other matters make for better policies. The key is ensuring that those exchanges occur early and often in the process, and that if organizations end up embracing different positions it can be managed in ways that are respectful and don’t overshadow all the many, many other policies, principles, and positions on which we all agree.

The problem with HR 7071

I write this in my own capacity, I am not speaking on behalf of the ALS Association.

A key provision in HR 7071 (“Accelerating Access to Critical Therapies for ALS Act”) authorizes $450 million over four years to underwrite the costs of early access to experimental therapies. This is not a simple ask.  It raises fundamental questions of principles and priorities for the ALS community, questions that deserve a full and open discussion – one that has not happened yet, but needs to happen now.

The goal of the $450 million request is straightforward. It seeks to solve one of the most frustrating impediments people with ALS face in their attempts to gain early access to therapies still in clinical trial – namely, cost. Pharmaceutical companies running clinical trials on therapies demand very high fees for access to their experimental therapies, if they agree to allow early access at all. Depending on the type of therapy, the expenses for early access can be prohibitively expensive for all but the very wealthiest patients, some reaching as high as $1 million per treatment – and they typically require recurring treatments.  The $450 million request – covering $75 million per year in years one and two, and thereafter $150 million annually in years 3 and 4 – is meant to cover costs of early access for people with ALS.

Several legitimate concerns have been raised about this proposal.

  1. It does not solve the problem it presumably sets out to solve — making early access affordable for all people with ALS. Instead, it would only allow a small fraction of Americans living with ALS to gain early access to experimental therapies. This is the case no matter how one works the math. If the most promising therapy in clinical trial happens to be one which runs close to $1 million per treatment, that would enable only 75 people, out of a US population of 25,000 with ALS, to gain access. That’s 1 in 333 of us – roughly the same odds of being diagnosed with ALS. If the treatments cost less, say $300,000 per round, that would still only cover 250 of us, or 1% of the US population of people with ALS.  Even if the cost of early access fell to as little as $10,000 – a figure some argue might be a reasonable estimate for access to a pill-form therapy in a platform trial —  that would cover 7,500 people with ALS, or less than one-third of us in the US. And such a low-ball estimate only raises another objection – if the cost of early access drops to $10,000 for some experimental therapies, why are we asking taxpayers to cover that when middle class and wealthy households could find a way to cover the costs and poorer households could be assisted by other means? 
  2. It risks creating major rifts within the ALS community. Who will choose what constitutes an eligible “promising therapy” and who decides which people with ALS get covered by the subsidy for early access?  How will the vast majority of people with ALS feel when they learn this massive appropriation of funds provides nothing for them?   Some who follow ALS research closer than I do suggest that the most promising therapies in the pipeline in coming years are those targeting familial ALS. Will that result in most or all of the $450 million covering  the 10% of the ALS population that has familial ALS, and how will the 90% with sporadic ALS react?
  3. Given the very poor track record of ALS phase three clinical trials to date, the odds of early access resulting in meaningful outcomes for the lucky few who get it are slim, making this a very high cost, high risk of failure gambit for a small number of people with ALS.  Of the dozens of ALS therapies that have made it to phase three clinical trials, only two have led to approved therapies, and both have only modest, uneven impact in slowly disease progression. This raises a legitimate cost-benefit question about the fund. Could that $450 million be put to better use than this?
  4. If the ALS community can convince Congress to devote $450 million to our disease, earmarking it for research for a cure would be a far better way to make use of the funding.   The bill stipulates that the $450 million be a separate appropriation from research funding, as a way to deflect criticism that this will divert money away from research. But that is a distinction without a difference. The reality is that interest group pressure that is able to secure this kind of funding for early access could certainly be able to do the same for research for a cure. The authorization for early access reflects a choice we as a community are making – that we prioritize gaining subsidized access to experimental therapies for a few of us, now, over seeking a cure for all. Is that what our community wants?

If our priority to to pursue treatments that stop or reverse ALS as speedily as possible for  all people with ALS, there are good reasons to question whether the $450 million for early access is a good idea, and good reasons to argue instead for lobbying for more money for research for a cure.

You may disagree with parts or all of the analysis I share above, but these are very reasonable questions and concerns, and they are being raised across the ALS community.   Fear of being attacked on social media is preventing some people with ALS from saying this publicly, but make no mistake, they are expressing these concerns in private.

What we need now, and what should have happened well before this bill was introduced, is a full, thoughtful, open discussion and debate in the ALS community about whether this bill in its current form actually reflects our priorities and principles, and if not how we can make calls to modify it to make it stronger and aligned with our principles.