There's obviously potential downside, but for people with serious illnesses there's potential upside.
For example: If you've got a disease that's going to kill you and unlikely helped by current medication/therapies, then dying a bit early is a small downside, not dying for however many more decades, on the other hand, is a huge upside.
In this case, the upside/downside is asymmetrical in favour of the upside! It would be logical to take 'under-tested, possibly lethal, drugs'.
> not dying for however many more decades, on the other hand, is a huge upside.
Looking at modern controversial cancer drugs we see that people get maybe six months of extra life, at very great expense, and these are the good drugs. The not so good drugs are even more expensive and provide perhaps two weeks extra life.
You missed the point that the very best newest cancer drugs are really expensive and if they're good give you an extra six months of life, and if they're not good give you perhaps an extra fortnight of low quality life.
There are multiple checks to ensure prudence of the use of development stage drugs. First of all a physician has to be convinced that the development stage drug could benefit his patient. If that’s the case, the physician can prescribe it to his patient. After that health authorities must grant permission before the physician can start treating his patient. Health authorities only allow treatment with such a development stage drug if it is considered safe and if it concerns a patient with a serious or life threatening disease who has no registered medicinal treatment alternatives left.
But what happens when physicians' criteria don't align with the priorities of a patient?
Take this hypothetical example: you wake up with a dozen marble-sized tumors in your brain. A combination of BRAF and PD-1 inhibitors has a small chance of curing you. This is backed by science, this is already a known result. Without the treatment you are dead within six months. With the treatment, you have a small chance of being cured.
However, due to potential known complications of PD-1, it is not yet approved as first-line for patients with brain mets. So the doctor says you must first go through whole brain radiation.
Your oncologist's only goal is to extend your life, he doesn't care about what you value and what kind of life you want. Therefore WBR and then immune therapy is the logical choice.
But maybe you don't just care about not dying, maybe you expect a certain quality of life and are willing to take the risk of foregoing whole brain radiation, because life after whole brain radiation is not a life you'd want to live.
What right does anyone have to tell you you are not allowed to take that risk?
These pure theoretical reasons for not giving patients the right to choose are so far removed for the day-to-day reality of someone dying of cancer, it's ridiculous. We're not dealing with abstract data with the goal of reaching some life-expectancy maxima using one-size-fits all treatment. We're dealing with people who have different priorities, and maybe care about more than optimizing for the treatment that is statistically most likely to squeeze an extra month of existence out of them.
"We focus on disease areas with unmet needs; oncology, neurology, psychiatry and rare diseases." This is from their main site and is the key to your question. If I were to have only 3 months left to live using conventional medicine, I would most likely try a drug that could prolong my life expectancy. I see it as a service for terminal ill patients.
The way things currently are, people are dying without access to drugs that are known to work. The approval process is painfully slow.
As a concrete example, there's a new class of immune therapy drugs used in the treatment of melanoma, among them PD-1 inhibitors and Ipilimumab. The data for these has been around for years, they are known to offer some kind of response in about a quarter of patients. In a small percentage of patients, there has been complete response and they've been disease free for years. This is unheard of in metastatic melanoma (stage IV has a 10% 5-year survival rate).
Dying of melanoma, by the way, is not a pretty death, there's nothing dignified or natural about how melanoma kills. It's something out of a horror movie. Dying early of liver failure would is a walk in the park compared to what a terminal patient has to look forward to.
In any case, the point is moot, because not only are PD-1 and its ilk more effective, but the side effects in trial patients have been minimal compared to the destructive known effects of chemo and radiation (or golf-ball sized bleeding tumors cropping up in your intestines).
I invite you to visit any melanoma forum and read the backlogs. You can read through years of people going through horrific treatments, all of which, again, are known to not be effective (biochemo, interferon, surgeries, whole brain radiation once the cancer spreads to the brain), while desperately applying for studies for drugs that are out there and already showing positive results. Then they suffer. A lot. Then they die.
Trials often have very strict exclusionary criteria, leaving many people out - often those who need it most:
> Auden said he was told that in order to be accepted into a clinical trial for the anti-PD-1 drug, he would need to have either no brain tumors or brain tumors that were at least no longer growing.
Even once they are approved, they might not be immediately approved as first-line treatment, meaning you still have to go through the old standard of treatment, wait until it doesn't work (meaning: put your body through the wringer, then wait for the cancer to come back again -- hopefully nowhere important like your brain or your lungs), and only then you have access to the new miracle drugs.
It's easy to make internet comments over some theoretical "gotcha" if you've never been on the receiving end of the stick. But the current system is broken and real people are suffering needlessly.
How in any way does it negate the scientific process to allow people who have nothing but months of pain and suffering ahead of them to give an experimental drug a shot?
You usually don't have only 3 moths left when you have a psychiatric disease, so waiting looks like a better option.
I can't find a well known horror story of experimental drugs just now. But if you want to read a horror story of experimental surgery you can start with HM http://en.wikipedia.org/wiki/Henry_Molaison .
I don't understand how a startup will help here. The problem is regulatory hurdles (good or bad): there are laws that prevent patients from buying medicine before it's been thoroughly vetted. Will this company lobby to change those laws? Or is this an Uber situation where the fact that it's a VC-funded startup means that regulations magically don't apply.
What could go wrong?