The issue is not really whether these treatments work or not. Every new treatment is by necessity unproven and more risky than an established one. Some people are early adopters or terminal patients with high risk-tolerances; others would rather suffer for a while than chance a drug that might make things worse.
Both of those are fine as preferences. The trouble arises because every US citizen is forced to have the global minimum of risk-tolerances across the population. It would be as if you could not try a development version till it became user-friendly enough for your grandparents. Specifically, in the US, you can't be an early adopter: the FDA does not allow citizens to opt-out unless they leave the borders of the USA. Indeed, in Cowan vs. US (1998) it successfully sued in federal court to prevent a dying AIDS patient from trying an experimental drug:
http://www.leagle.com/xmlResult.aspx?page=4&xmldoc=19981...
Plaintiff requests that Dr. Davis be authorized to inject
Plaintiff with the with the experimental goat neutralizing
antibody drug [1] and that the FDA be enjoined from
interfering with Dr. Davis' treatment of Plaintiff. ...
The Court is sympathetic to Plaintiff's situation.
However, the law is very clear, and under the current
statutes and regulations, Plaintiff's physician may not
administer the goat neutralizing antibody drug absent
prior approval of the FDA. In Court, Plaintiff argued that
he should have the right to take whatever treatment he
wishes due to his terminal condition regardless of whether
the FDA approves the treatment as effective or safe, and
that to prohibit him from taking the treatment he wishes
violates his rights under the US Constitution. ...
This Court is in no way criticizing the intentions of
Plaintiff and his physician or the potential effectiveness
of the proposed treatment. Plaintiff's physician should
pursue approval of his Investigational New Drug
application as quickly as possible. Plaintiff's doctor
must obtain appropriate approval through the proper
regulatory authorities. As much as this Court may
empathize with Plaintiff, the authority to provide some
type of exemptions for individuals such as Plaintiff rests
with Congress and not with this Court.
Plaintiff was denied. Plaintiff died. Who knows whether the drug would have saved him, but he wasn't given the chance to try. Occasionally, if you have
tremendous political connections, you can get a waiver, like Fred Baron:
http://blogs.wsj.com/health/2008/10/17/lance-armstrong-and-b...
“We did a safety review, consulted with experts on PML,
and worked closely with FDA to come up with a risk
management program that allowed us to bring it back on the
market in a way that limited its use,” a Biogen
spokeswoman told the Law Blog. The plan prohibits giving
Tysabri for unapproved uses.
Biogen Idec is running an early-stage trial of the drug in
multiple myeloma, but Baron doesn’t meet the criteria to
participate.
Baron’s a prominent donor to the Democratic party, and
many of his powerful friends, including Lance Armstrong
and Bill Clinton, made appeals on his behalf. And the
family agreed not to sue if anything goes wrong.
Ultimately, his doctors at the Mayo Clinic worked directly
with the FDA to find a “legal basis” for giving Baron
Tysabri. The deal was announced on Baron’s son’s blog late
yesterday.
So, if you are wealthy you can travel outside the US to opt-out (though US-based companies will usually not administer treatment for fear of getting on the FDA's bad side). And if you are politically connected you can sometimes get an experimental treatment, like Baron.
But this is not really optimal. If you are an academic, you accept the concept of QC/quality checking, but you aren't stuck with just one journal to submit to. You can revise and resubmit somewhere else. And if you are an end-user, you don't have to take a reviewer's opinion into account when choosing between movies, books, bikes, or virtually any other physical good with star ratings on Amazon.com. Except for drugs. Then you, as the end-user, cannot opt-out and take the FDA's opinions with a grain of salt. In part this is because the FDA will sue you directly. In part it is because companies that even think of trying this route will get slapped by the FDA for trying to game the system, and subsequently find their approvals slowed or (nowadays) outright denied.
We need to carve out a jurisdiction where patients and entrepreneurs alike are free to take informed risks, recognizing up front that sick people do die in medicine, and also recognizing that society already allows people to take incredible risks in other contexts (joining the military, bungee jumping, walking tightropes). Whether that new jurisdiction is Singapore, or Estonia, or a seastead, or a medical cruise ship, or something else is to be determined. But that has to be the goal.
[1] In case a "goat antibody" sounds weird to you, it's a common thing in molecular biology. Google it, or see for example Thermo's web page:
http://www.pierce-antibodies.com/custom-antibodies/goat-anti...
Both of those are fine as preferences. The trouble arises because every US citizen is forced to have the global minimum of risk-tolerances across the population. It would be as if you could not try a development version till it became user-friendly enough for your grandparents. Specifically, in the US, you can't be an early adopter: the FDA does not allow citizens to opt-out unless they leave the borders of the USA. Indeed, in Cowan vs. US (1998) it successfully sued in federal court to prevent a dying AIDS patient from trying an experimental drug:
http://www.leagle.com/xmlResult.aspx?page=4&xmldoc=19981...
Plaintiff was denied. Plaintiff died. Who knows whether the drug would have saved him, but he wasn't given the chance to try. Occasionally, if you have tremendous political connections, you can get a waiver, like Fred Baron:http://blogs.wsj.com/health/2008/10/17/lance-armstrong-and-b...
So, if you are wealthy you can travel outside the US to opt-out (though US-based companies will usually not administer treatment for fear of getting on the FDA's bad side). And if you are politically connected you can sometimes get an experimental treatment, like Baron.But this is not really optimal. If you are an academic, you accept the concept of QC/quality checking, but you aren't stuck with just one journal to submit to. You can revise and resubmit somewhere else. And if you are an end-user, you don't have to take a reviewer's opinion into account when choosing between movies, books, bikes, or virtually any other physical good with star ratings on Amazon.com. Except for drugs. Then you, as the end-user, cannot opt-out and take the FDA's opinions with a grain of salt. In part this is because the FDA will sue you directly. In part it is because companies that even think of trying this route will get slapped by the FDA for trying to game the system, and subsequently find their approvals slowed or (nowadays) outright denied.
We need to carve out a jurisdiction where patients and entrepreneurs alike are free to take informed risks, recognizing up front that sick people do die in medicine, and also recognizing that society already allows people to take incredible risks in other contexts (joining the military, bungee jumping, walking tightropes). Whether that new jurisdiction is Singapore, or Estonia, or a seastead, or a medical cruise ship, or something else is to be determined. But that has to be the goal.
[1] In case a "goat antibody" sounds weird to you, it's a common thing in molecular biology. Google it, or see for example Thermo's web page: http://www.pierce-antibodies.com/custom-antibodies/goat-anti...