The vast majority of new treatments fail in Phase 2 - that is, in showing effectiveness in a human model. A huge chunk of the remainder fail in phase 3.
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.
>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.
Your post is misleading. You're not providing counter-evidence for the 'failure for decades' claim, and are in fact confirming the 'one of the first' part.
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]
And even the Cancer drugs that "work" and are registered only improve stuff like PFS and OS (overall survival) a couple of months in most cases. Cancer treatments tend to work for some time until the illness finds a new pathway rendering the treatment ineffective. It's frankly pretty depressing to look at the survival curves with and without a new drug. You don't save many people anyway.
You really can't make such statements about "cancer". Stage II lung cancer? You will probably die in a few months, surviving five years isn't very likely. Stage 4b Hodgkin's lymphoma at the age of 18? You have a decent chance of getting to know your grand children.
You presume the new treatment will save more lives than the current one. Without adequate research and testing, this is an unsupported presumption. It's as likely to do more harm than good if the preliminary research isn't done.
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.
any way you slice it people are going to die and make grave errors. but open transactions between researchers and free human beings would have drugs evolving much faster than unaccountable and corruptible overlords. reputation systems could replace much of their most important functionality.
> reputation systems could replace much of their most important functionality.
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.
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?
You have cancer. You are going to die. Having a terminal illness changes the risk/reward beyond the bounds that most people can imagine. Even so, it seems that most people can make a reasonable decision, as long as the worst of the snake-oil peddlers are kept out. Here are your choices.
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.
Thanks, but I'd rather have medical research systems designed by scientists than libertarians. Free markets are not a panacea, and it's dangerous to think of them as such. In fact, the medical community used to be such a place, and it was dominated by quacks and snake oil salesmen.
you also have to consider that rushing through the trial stages can cause mistakes which leads to the treatment looking like it's ineffective. When this happens, it loses funding any may not then be developed further.
