At what point should an experimental drug be made available for anyone to try it? The usual answer is "Unless you're enrolled in a clinical trial, then not until it's no longer an experimental drug". There's always compassionate use, but that's a hard topic to deal with, and one that has a different answer for every drug. Otherwise, the regulatory position is that volunteers take unproven drugs, and paying patients take the ones that have been through testing and review.
Colorado would like to try something different. They've passed a "Right To Try" law, which allow a therapy to be prescribed after it's passed Phase I and is under active investigation in Phase II. (Arizona, Louisiana, and Missouri are heading in the same direction). Insurance companies are not required to pay for these, it should be noted, nor are drug companies required to offer access. But if there are willing patients and a willing company, they can work together. The patient has to be suffering from a terminal illness, and has to have exhausted all approved therapies (if any).
As my fellow science-blogger David Kroll notes, though, this doesn't seem to add much past what was already allowed by the FDA:
I submit that this seemingly well-meaning but meaningless Colorado act does nothing but create a sense of false hope for similar families. The act does nothing more than assuage the concerns of lawmakers that they haven’t done enough to help their constituents. Instead, they’ve done a disservice.
At Science-Based Medicine there are similar thoughts from oncologist David Gorski. He goes into the details of the law that's under consideration in Arizona, and worries that it has such broad definitions that it opens the door to unscrupulous operators. I've worried about that as well. The Arizona law also allows the companies (at their discretion) to charge for providing the drugs (Colorado's allows for at-cost charging). The fear is that some unscrupulous operators could run the lightest, breeziest "Phase I" trial they possibly could, and then settle down into a long, lucrative spell of milking desperate patients while their "Phase II' trials creep along bit by bit. I realize that that's not a very nice thing to assume about people, but as a character in The War of the Worlds says about a similar predatory proposal, there are those who would do it cheerful. In fact, we already have evidence of people working in just this fashion.
These arguments have come up around here before, when Andy Grove (ex-Intel) proposed changing the structure of clinical trials (more here), and when Andy Eschenbach (ex-FDA) proposed something similar himself. Balancing these things is very hard indeed, and anyone who says it isn't either hasn't thought about the situation enough or is eager to sell you something. We've come to Chesterton's Gate again:
There exists in such a case a certain institution or law; let us say, for the sake of simplicity, a fence or gate erected across a road. The more modern type of reformer goes gaily up to it and says, “I don’t see the use of this; let us clear it away.” To which the more intelligent type of reformer will do well to answer: “If you don’t see the use of it, I certainly won’t let you clear it away. Go away and think. Then, when you can come back and tell me that you do see the use of it, I may allow you to destroy it.
Gorski (in that Science-Based Medicine link above) has many other good points, but there's one more that I'd like to emphasize. Some of these laws seem to be based on the idea that there are all sorts of wonderful cures out there that for some reason are tied up in sloth and red tape. It isn't so. Clearing out bureaucratic obstacles, while no picnic, is still a lot easier than discovering drugs. And allowing patients access after a Phase I does not offer very good odds, considering that almost all clinical failures take place later than that. Plenty of tox gets discovered later than that, too - only the fast and nasty stuff gets picked up in Phase I.
So overall, I think that these laws offer, for the most part, chances for people to feel good about themselves for voting for them, and chances for patients to get their hopes up, likely for no reason. Even with that, I don't see them doing much harm compared to the existing regulatory regime, except for the provisions that offer companies the chance to charge money. Those give the added bonus of opening the door to unscrupulous quacks, some of whom might have very creative ideas of what "at cost" might mean.
According to Biocentury, a Colorado company is already planning to offer access through this program:
Neuralstem Inc. (NYSE-M:CUR) plans to take advantage of Colorado's right-to-try law to offer patients access to an experimental, unapproved human neural stem cell (hNSC) therapy (NSI-566) to treat amyotrophic lateral sclerosis. President and CEO Richard Garr told BioCentury the company will not apply for an IND or other permission from FDA, noting that "the Colorado right-to-try law allows a company to prescribe for a fatal disease a therapy that has passed a Phase I safety trial and is being actively pursued in a Phase II trial." Garr said Neuralstem's hNSC ALS therapy meets these criteria, and the company plans to start a Phase III trial next year.
Neuralstem is in the process of training surgeons and identifying a hospital and neurologists in Colorado to administer the hNSC therapy. The therapy will be administered with the identical procedure, cells and training as a clinical trial, but without FDA oversight and without "the artificial limitations built around a trial," said Garr. "The whole point of right-to-try is it sits parallel to the clinical trial process, it is not instead of clinical trials." Neuralstem has not determined whether it will charge Colorado patients.
I know nothing about Neuralstem or their therapy, so I'll defer comment until I learn more. Looks like we're going to see how this works, whether we're ready for it or not.