Comment by mchusma

3 years ago

This brings up again the sorry state of medicine being "default banned". Should this treatment be banned for patients? Absolutely not.

Is it a small, underpowered study that needs to be replicated? Absolutely.

Could it warrant banning in the future? Sure.

Should it be covered by insurance? Complicated.

We need to unbundle these things.

70% of Phase 2 trials fail and 50% of Phase 3 trials fail[1]. Why should the default to be to approve drugs in the early stage of human experimentation?

Here are some interesting case studies of drugs graduating from Phase 2 trials only to fail Phase 3 trials on efficacy and safety grounds: https://www.fda.gov/media/102332/download

[1] https://www.parexel.com/application/files_previous/5014/7274...

  • Because (at least) for people with a high risk of dying, and operating under well-informed consent, that decision should be between patients and their doctors alone.

    • If we allowed any type of experimentation on dying patients as long as there was consent, dying patients would end up getting used as a Guineapigs by companies trying to get a lucky indication. It would certainly benefit the families left behind financially, but it would not be pretty. “Here’s a million bucks, please take these 40 drugs that will certainly kill you, but we’ll learn which one we should continue with”. And why even do toxicity studies or phase 1 and 2 trials? It’s cheaper to pay of poor people who are on the verge of dying and just pushing them over the edge.

      The decision should be just because a patient and a doctor, because the consequences of such decisions are more far reaching than just those two. We have a well established framework for what a drug needs to prove before they can reasonable be allowed to be given to any human being, no matter their situation, and that barrier of entry is there for a multitude of good reasons. Discarding it just because a specific patient is at risk of dying or because you can get consent is not helping patients.

      6 replies →

    • There's definitely a case to be made for early access to drugs for certain terminal illnesses in cases where there is compelling evidence that the drugs will pass Phase 3 trials. But, that should hardly be the 'default' as OP suggests.

    • Because it would degenerate into pharmaceuticals selling false hope in exchange for lab rats that are too far along to be saved even if the drugs work.

      That being said I disagree with prescription pads. I should buy antibiotics for my kid without waiting four, five hours to be seen and processed

      14 replies →

    • > Because (at least) for people with a high risk of dying

      and who decides that?

      Can a drug company give "gifts" to doctor who then recommend the patient use that company's drugs when he/she is dying?

  • After Phase 2, the drug has been proven safe.

    In a sane and humane system, sick people would then be allowed to try it.

  • I mean if they've already tried everything else and nothing on the horizon and I just heard 12 of 12 people with same cancer as me had gone into remission after treatment? I'd probably be more than happy to become #13

In the clinical trials I have been a part of the manufacturer pays for the drug and all related study costs. They even pay you a stipend for commuting, lunch, etc.