3 ms·

First, do no harm. This is a foundational tenant that permeates healthcare. As a former health system executive I have been in positition to influence decision

First, do no harm. This is a foundational tenant that permeates healthcare. As a former health system executive I have been in positition to influence decision making about high risk experimental treatments in certain cases. In the US currently, in most states, you as a patient have a more or less unlimited right to try experimental treatments if you are facing a reasonable likelihood of death otherwise. The FDA plays a very limited role in one-off situations though they are frequently made to play the villain. The reality is that there are legal avenues available much of the time that providers and companies are loathe to employ. Much of the time drug companies and doctors have lots of reasons not to want to roll the dice if they think the odds of success are low enough. The risk/reward is a very different outlook for the patient than the providers and companies that may need to be a party to the treatment. There are reprecussions to all patient deaths as a result of an experimental treatment however well intended and consented. A complicating factor that weighs over everything is that there is a long history of terrible abuse of informed consent with less privileged patients, puerto rico is a trainwreck of abuse going back a century but there are many more. As an administrator I always felt that patients who are freely giving informed consent always have the right to try, however slim the chance of success. But, it can be brutally difficult to parse out those instances where influence, coercion or even misrepresentation are at play with a particular patient and family. Family dynamics of course can play a large role too.

fzeroracer 3y ago
Yeah, I agree with this. Ultimately the danger is that right to try essentially leads patients to deny proven methods of treatment due to coercion or being misled. If a patient refuses the usual cancer treatment and demands instead a regime of herbs and folk medicine, how do you handle that? Especially when the family then turns around and blames you. In this scenario where you have an expert in their field knowing that the outcome for the experimental treatment could make things far worse but is willing to contribute the data to further the field anyways I think the answer is far easier to parse.
lend000 3y ago
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.
- duffpkg 3y ago
  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.
- JPLeRouzic 3y ago
  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...
  - lend000 3y ago
    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.

fzeroracer 3y ago

Yeah, I agree with this. Ultimately the danger is that right to try essentially leads patients to deny proven methods of treatment due to coercion or being misled. If a patient refuses the usual cancer treatment and demands instead a regime of herbs and folk medicine, how do you handle that? Especially when the family then turns around and blames you. In this scenario where you have an expert in their field knowing that the outcome for the experimental treatment could make things far worse but is willing to contribute the data to further the field anyways I think the answer is far easier to parse.

lend000 3y ago

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.

duffpkg 3y ago
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.
JPLeRouzic 3y ago
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...
- lend000 3y ago
  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.