Blanket statements like this aren't true. With a lethal ailment, a risk analysis must be done on how many you are killing with each delay.
People whine about how slow the pace of research is, but that's not because clinical trials are so slow - it's because most drugs fail. The vast majority of drugs that work in animal models get bounced in Phase 1 (when they show unacceptable toxicity in humans so great it can't be dosed) or Phase 2 (where it just shows no effectiveness in humans at all - not even some level of effectiveness that fails in cost/benefit analysis). this is the problem with people complaining "But every delay kills people!"
No. You don't know going in whether it's going to work - that's why you're doing a trial. And from a naive position, the probability is pretty clear that it's very unlikely it's going to work. There's absolutely no reason to think any particular drug is worthwhile, and every to worry about its side-effects. With a sample size of 12, this one may yet became a last-ditch drug with toxicities worse than chem/rad. We don't know yet. Using this is an example of over-cautiousness is ridiculous.
Additionally, the slowest part here wasn't the clinical science - it was the basic science. Gene therapy has been a failure for decades, and we've been repeatedly going at it to try and get it to work. It's not as though this is the first clinical trial for a gene therapy: it's just one of the first to be worth anything.
Glybera is a gene therapy which has been approved for medical use.
Yes, it has been approved, but only very recently, in late 2012. It is also the first such drug that has been approved (and to date, the only one), and this happened under 'exceptional circumstances'. The usual standard of supporting evidence was forgone because the disease it treats is so rare. The company is explicitly required to supply further supporting evidence, and the rules for its use are very strict.[1]
We have the regulations we do because we caused harm in the past. They didn't spring fully formed from the minds of regulators, they happened because researchers killed and maimed people by not doing the necessary verification before human trials began.
I'll start believing this once you show that a "reputation system" (don't these already exist?) actually does anything to stop quacks from peddling diluted water as a cure-all.
If you relied on reputation alone to regulate the medical market, feel-good snake oil treatments would prosper at the expense of legitimate medical advancement.
The same "reputation systems" that saved us from the million miracle-cures sold on late night tv, from "green coffee diet" to "Oxygenated water"?
Do you honestly believe that people with a fatal illness, dealing with people offering a "cure" for that fatal illness, are free to make a rational choice?
Do nothing. Make your time. You are going to die pretty soon. Try to make yourself and your family comfortable.
Take what treatment your insurer allows. You die more slowly, with a small chance of remission, but also with a small chance that the treatment will kill you sooner. Maybe your won't consume your family's assets.
Take what treatment you can afford. Die even more slowly (probably), with a small (but probably better) chance of remission, but also with a small chance that the treatment will kill you sooner. You may consume all your assets, and or incur huge debt/bankrupt your family.
Get yourself into an experimental trial. There is some chance that you will be in the control group (if one is used) in which case, you will die. There is some chance to suffer some adverse reaction in which case, you may be removed from the trial, back to step 1. There is some chance to suffer some catastrophic adverse reaction, in which case you will die, and if you are unlucky the death will be worse than death by cancer/chemo. There is some remote chance the treatment will work as good or marginally better than existing treatments, you win, congratulations. There is some very extremely remote chance the treatment will work amazingly (as in fairy-tale amazing) well, epic win. It may cost little or nothing, but who cares what it cost, you're alive and you just helped save the lives of many others.