Someone close to me has ALS and their only options seem to be in Mexico and China, not anywhere in the US.
From their research, it definitely seems like the FDA is the big bad not only in preventing treatments as a default, but also by disincentivizing companies from allowing dying people to trial unapproved treatments if their condition is far gone enough that there is a low chance of success. My understanding is that this is partially because people who take treatments and die (even if they were going to die no matter what, and did not die from the treatment itself) are used by the FDA against the biotech companies when seeking approval.
I am sorry. That is a terrible disease. To my knowledge John Hopkins currently has the most promising trials for ALS but generally speaking there is not currently a full understanding of the underlying mechanisms of that disease and little, even in the most experimental ends of the pipeline, for it. I am not aware of anything that has even the slightest promise of effectiveness that would be available in mexico or china that would not be available in the US if they met eligibility criteria for currently open trials. The ACT for ALS passed in 2021, amongst other things, specifically opens what paths are available to patients with ALS and in certain circumstances offers grants.
I am sorry for your relative, but you should be aware that in ALS (and probably other diseases) a major patient organization has a financial interest in some drugs to succeed and they do not hesitate to put a lot of pressure on social media onto the FDA. As far as I know, with the same data, this drug was not authorized in the EU.
Also in ALS, there were major manipulations on social media by companies that claimed their drug was able to cure people, and to prove it they showed on social media well-known patients going to the gym and others doing motocross!
Yet these drugs never shown any benefits in phase III clinical trials...
The point that some people miss here is that it doesn't matter. If you are going to die from ALS in 2 years, are you going to feel satisfied when the FDA says "we won't let you try this; it probably won't cure you"?
Even if the FDA is this incredibly competent organization with no corruption, people should have a right to try to live. It's the most dystopian form of the nanny state mentality, IMO.
Who is going to pay for the treatments without supporting data? I don't think people have the right to demand healthcare workers to administer whatever strikes their fancy either. I know you aren't necessarily suggesting this, but I wouldn't start an IV bleach drip on someone because they have a "right to live". Time is so limited in the clinic that I don't think there is room for trying whatever the patient wants on demand. In an ideal world there should be some room for this approach, but (at least in the US hospitals I've worked at) there just isn't the infrastructure. Something with phase 2 data, sure, that seems like a good candidate for compassionate use.
ALS is devastating, I'm sorry for you and the person you are close to.
You could probably answer all of your own questions pretty easily and recognize that I'm not talking about a bleach injection.
Maybe your gripes make more sense in a socialized medicine country. Sure, they have to sacrifice people at the edges. But in the US, there's no excuse. It's like the worst of both private and public medicine.
In terms of benefitting the collective, allowing experimentation on willing subjects with terminal illnesses could really improve medicine, too. It's really just a lose-lose-lose with the current approach. Well, some people win, who manufacture the types of drugs that don't fully cure you but keep you dependent on the prescription until you die. If you don't think that's a factor in the FDA's decisions I have a bridge to sell you.
From their research, it definitely seems like the FDA is the big bad not only in preventing treatments as a default, but also by disincentivizing companies from allowing dying people to trial unapproved treatments if their condition is far gone enough that there is a low chance of success. My understanding is that this is partially because people who take treatments and die (even if they were going to die no matter what, and did not die from the treatment itself) are used by the FDA against the biotech companies when seeking approval.