I read articles like these, where the treatment is at least somewhat successful, and I wonder how many instances there are where they tried an experimental treatment but the patient died anyway.
He needed surgery to remove small tumors on his lungs, but the drugs made his melanoma just disappear.
He was very lucky. Not only because he was offered to take part in a trial, but also because only a small number of patients had complete success stories like his.
The link below is to a very similar case from the same trial:
https://7news.com.au/lifestyle/health-wellbeing/miracle-drug...
Sure, but they could be. Easy enough to gain profit from those who get the disease at a later date. If a current standard of care treatment is used as a control for say, half of the cohort, there could be some way to charge every participant's insurance for half the cost of the standard of care (with the other half getting the experimental treatment on a blinded basis).
2) Trials are already obscenely (and increasingly) difficult to design and execute even for pure scientific ends. It’s really not trivial to add other objectives like this, even if money were totally irrelevant.
2) And thus the humanitarian part of the trial can serve dual purpose as a training exercise for the additional physicians working with the humanitarian cohort.
Depending on the rarity of the illness, this may be something that the government can easily force pharma companies to accept. They accept far more in regulatory burden and still do business.
Additionally, the pharma company wouldn't allow this because in addition to the upfront design complexity, it would put the trial itself at risk. You're not really allowed to say, "just try it in a bunch of people, it's okay if untrained folks mess it up because if they do we can just throw out the data."
Not opposed to that, but it's not a study cohort. It's just "give investigational drug away for free."
This part:
> You're not really allowed to say, "just try it in a bunch of people, it's okay if untrained folks mess it up because if they do we can just throw out the data."
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> It's just "give investigational drug away for free."
And here insurance regulation can also come in to play along with the pharma regulation.
Sure, none of this would be easy. But the status quo has issues too.
I'm grateful for the work of the doctors in the article, and very hopeful that the treatment works. A close friend of mine was on a trial vaccine for his cancer, which looked promising for a while, but suddenly stopped working - the cancer was ultimately fatal.
I think trials should be offered as potential first steps, especially for aggressive cancers. Patients know the risks. They're either 100% going to die within 6 months to cancer, or, worst case, die trying to fight it, or best case, be cured.
We need really good science and statistics, far superior to p-values, to allow this potentially life saving treatment without the inevitable deaths from people at the limits returning a "not significant" finding for something that actually works.
Maybe something like allowing people who are too far gone to qualify for treatment as part of a study to instead qualify for the experimental treatment with their results only being included as a special "humanitarian" cohort.
I'd also like studies to be able to compare a treatment group against prior control groups, so that everyone today can get the experimental treatment instead of some of them being given a placebo (if there's a lack of another decent treatment to use as a control). If there's nothing effective that can be done as a control, every one should get the treatment.
But the part of me that witnessed glioblastoma - I think when someone is facing that, it should be a VIP pass straight to the "whatever has even a remote chance of actually working" lobby. Which is basically what the article describes, so I'm optimistic for the doctor and his team.
Of course, not everyone is as risk tolerant as I am. My dad wanted to believe he'd be one of the rare "survivors" (I don't think it happens) using traditional radiation and chemo, and was still weighing pros and cons of other potential treatments. But I have the benefit of hindsight, and a lot more education now, and I think it's unethical to not immediately consider trial programs. The most common way to treat glioblastoma today (targeted radiation and chemo) one that only promises to give you more months of life, not years, is also the only treatment option covered by Medicare, the health insurance provider for most retirees in the United States. The uber-aggressive glioblastoma that my dad developed is what most people over retirement age get when they develop glioblastoma as well. Average lifespan from diagnosis to death: 6 months. That's if you surgically remove the tumors, a procedure not without its own risks. Risks that are usually worth it, since without surgically removing the tumors, lifespan drops to weeks.
What I'm saying is there's no path to trial programs for most of the people who'd benefit from those trials the most. It's not even on the map for them.