Hope Can Be Cruel

** A brief note that this will be the last blog post for a few weeks. The PAC team is taking a well-deserved break and will be back the week starting July 19 rested, refreshed, and ready to go! See you soon!**

Alzheimer’s Disease, one of the most common of over 400 types of dementia, terrifies me. I have dementia on both sides of my family, and though the majority of types of dementia are not hereditary, my emotions can’t seem to accept that.

I am not alone. In a 2016 survey out of the UK, over half of the participants  who suspected they had dementia put off going for a diagnosis. Over 60% said they thought a dementia diagnosis effectively would mean the end of their lives. I don’t blame them. I can’t let myself think knowing my mental capacity was slowly but inexorably declining while my physical self remained healthy without triggering a panic attack. When I had to designate my preferences should I be mentally, but not physically, incapacitated, I cried through the whole section. It was traumatic.

This is why I am not entirely surprised about the controversy surrounding Aduhelm, the newest treatment for Alzheimer’s. It is the first new treatment to be approved in over 15 years, and the first to actually modify the course of the disease. The theory is that it would dissipate some of the plaques that build up in the brain that are markers for Alzheimer’s, and therefore slow disease progression. Between that, the fear, and the growing population of dementia patients (as life expectancy increases, it makes sense that the number of people with dementia will, as well), certain segments of society have been growing ever more desperate for some kind of additional avenue of hope.

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According to Margot Sanger-Katz of the New Your Times and Joanne Kenen of Politico in discussion with Julie Rovner on Kaiser Health News’s What the Health? podcast, there are issues.

First, the cost of just the treatment is $56,000 per year, which, if Medicare decided to cover it, would amount to roughly $12,000 out-of-pocket per person per year. And that is just for the treatment. It’s an infusion, so it also has to be administered in a particular kind of facility by healthcare professionals who have specialized training. The treatment also requires regular brain scans. It’s the only way to take baselines and detect whether there is improvement for a condition like Alzheimer’s. All of that combined would increase everyone’s share of Medicare Part B, as well. The way Medicare Part B works is that everyone pays a percentage of the overall expenditure. Most treatments with price tags that high are prescribed to smaller populations, not enough to cause everyone’s costs to rise significantly. But with rising numbers and common prescription of this treatment, the potential is there.

Second is the way it was approved. Medications based on the same hypothesis of lowering buildup of certain plaques in the brain have been tested before and they have failed. Biogen ran two big clinical trials for Aduhelm. One trial failed and the other showed some impact, but not at the level one would hope for. In fact, before the studies even finished, the monitoring committee told Biogen to scrap it because results did not look favorable and they should stop to protect patient safety. It was the FDA that persuaded them to continue. And when the FDA approved it through the accelerated use process, which required they perform an additional clinical study after the fact to prove concrete clinical benefit, three members of the advisory committee quit.

The thing is, I trust the FDA. For the most part. They have a well-deserved, global reputation for being slow and ponderous, much more so than similar agencies in other countries. But the US version is basically a giant consumer protection agency, while many of the others were created to make inter-country commerce easier. There are times when the holdup due to red tape and internal politics is beyond frustrating and can be dangerous (right to try laws have gotten easier to use, although industry does not have to comply), but I would rather wait for FDA’s meticulous review process than be given a drug equivalent to a placebo, or even worse, one that causes detrimental issues down the line.*

I get it. After so long, we need something to hold on to as we watch this scary disease claim friends and loved ones, and fear that someday it might come for us. Considering past precedent, the perception is that the FDA is bowing to pressure from industry and patient groups. But what good is hope if it is unlikely to bear fruit, and especially if it comes with a potentially life-ruining price tag? Hope can be cruel. It can build a version of reality that is based on anything but, and it can rob us of our coping mechanisms. It can also chip away at our faith in the ones who gave it to us. Every day we wait to no avail makes us more and more cynical.

We are patients and caregivers. We are used to living without solutions. I don’t know about you, but I would rather have honesty and the space to prepare myself than tilt at medical windmills. I really hope that we find a solid treatment for Alzheimer’s, and maybe Aduhelm will turn out better than projected. But based on the evidence we have, I just don’t think so.

I don’t want to rain on anyone’s parade, so go ahead and hope if you really believe that Aduhelm is the solution it purports to be. But maybe it would be best to hope for the best and prepare for the worst.

 

*NOTE: Per the COVID-19 vaccines, the research was done in a compressed, nonlinear fashion. That is the reason the vaccines were approved so quickly, not that the research wasn’t done.