At what point should an experimental drug be made available for anyone to try it?
[...] "Right To Try" law [allows] a therapy to be prescribed after it's passed Phase I and is under active investigation in Phase II. [...] Insurance companies are not required to pay for these, it should be noted, nor are drug companies required to offer access.
The big underlying question here is "Who gets to make the decisions?" Right-to-try advocates would say that the patients themselves should be making those calls (presumably with some advice from their physicians). Others would say no, we need an FDA, some sort of regulatory authority to make sure that the therapies people are choosing from are actually worth choosing. Another argument is that the underlying medical and clinical issues are complex enough to make some sort of expert review worthwhile, and that we can't necessarily expect "informed consent" to always be informed enough under some of the more wide-open proposals.
[...] The Limits of Expertise
Past that, though, you run into the folks who aren't even bothering to appeal to experts at all, because they simply don't trust them much and don't believe what they have to say. Here, too, we have gradations. If we're talking about the Poincaré Conjecture, to pick an issue on one end of the scale, the only people that will be of much use while discussing the details of Grigori Pearlman's proof of it will be those who have devoted serious time to the study of topology. No one who is not comfortable dropping the phrase "Riemannian manifold" into their conversational flow can really have a seat at that table. Even world-renowned prize-winning scientists from many other fields are not going to able to pull up a chair.
Dr. Lowe breaks these types of questions into "Matter for Experts", "Just Plain Facts", "Flat-Out Unknowable", and "Matters of Opinion" and the relative value of expertise for each.
http://blogs.sciencemag.org/pipeline/archives/2016/06/30/expertology
Background on "Right To Try" laws:
http://blogs.sciencemag.org/pipeline/archives/2014/06/10/right_to_try_here_we_go
[Ed. note (martyb): This is the same Dr. Derek Lowe who authored the hilariously-written and highly-informative blogs: Things I Won't Work With and Things I'm Glad I Don't Do.]
Related Stories
https://blogs.sciencemag.org/pipeline/archives/2020/01/27/coronavirus
As the world knows, we face an emerging virus threat in the Wuhan coronavirus (2019-nCoV) outbreak. The problem is, right now there are several important things that we don't know about the situation. The mortality rate, the ease of human-human transmission, the rate of mutation of the virus (and how many strains we might be dealing with – all of these need more clarity. Unfortunately, we've already gone past the MERS outbreak in severity (which until now was the most recent new coronavirus to make the jump into humans). If we're fortunate, though, we'll still have something that will be worrisome, but not as bad as (say) the usual flu numbers (many people don't realize that influenza kills tens of thousands of people in the US each year). The worst case, though, is something like 1918, and we really, really don't need that.
[Ed note: The linked story is by Derek Lowe who writes a "commentary on drug discovery and the pharma industry". He is perhaps best known for his "Things I Won't Work With" blog entries which are as hilarious as they are... eye opening. I have found him to be a no-nonsense writer who "tells things as they are", holding no punches. The whole story is worth reading as he clearly explains what a coronavirus is, about the current one that reportedly originated in Wuhan, China, what could be done about it, how long that would likely take, and what can be done for those who have already been infected. --martyb]
Previous Stories Referencing Derek Lowe:
Machine Learning Comes to Biochemistry
Ignition! The Funniest, Most Accessible Book on Rocket Science is Being Reissued
Another Failed Alzheimer's Disease Therapy
Marathon Pharmaceuticals is Part of the Problem
Lobbying Results in FDA Approval for Controversial Drug
"Right to Try" New Experimental Medicine and the Value of Experts
Cancer Hazard vs. Risk - Glyphosate
A Terrific Paper on the Problems of Drug Discovery
Things I Won't Work With
(Score: -1, Offtopic) by Anonymous Coward on Saturday July 02 2016, @06:22AM
Things I Won't Work With
Poor people?
Things I'm Glad I Don't Do
Shoveling shit?
And you wonder why the immigants be taken yer jerbs, fucker.
(Score: 2) by quintessence on Saturday July 02 2016, @06:36AM
While I'm very sympathetic to the author's intent, to a large degree the point is moot medically when something like homeopathy is on the table. Best case scenario is you try to advocate for the patient, which is a subtle but important difference from advising the patient. Good, bad, or indifferent- it is still their life. Perfectly sane, educated, and involved people go against medical advice all the time.
In terms of politics, the nature of experts seems to be more of a ruler than a guide, which where distrust crops up, especially among the informed who look back in horror at the freedom to do stuff that sucks.
I'd rather good, even heated arguments from opposing sides than the browbeating that seems to be much of the discourse now.
(Score: 0) by Anonymous Coward on Saturday July 02 2016, @06:58AM
Going against medical advice is a sign on mental illness. Fixed that loophole for you, bitch. Now buy some overpriced shit and make big pharma some profit.
(Score: 0) by Anonymous Coward on Saturday July 02 2016, @07:19AM
Even the mentally ill have rights.
Short of determining mental status for trial, quarantine, or 48 hour observation, I am unaware of any other situation where you can be forced into medical treatment, and even then it's more of a public health concern or requiring an order from a judge.
(Score: 2) by Gravis on Saturday July 02 2016, @01:24PM
if we stick with science and the FDA, we will eventually get medicines that work. the "right to try" idea has been done before and it's how we got people selling snake oil.
(Score: 1) by khallow on Saturday July 02 2016, @02:10PM
if we stick with science and the FDA
Those two are not always compatible. The FDA among other things doesn't face consequences for creating enormous opportunity costs.
the "right to try" idea has been done before and it's how we got people selling snake oil.
Which if you look at it, is not that big a deal. We could always make fraud illegal, right?
(Score: 1) by Francis on Saturday July 02 2016, @05:46PM
The problem with the FDA is that it just shifts the type of errors between types 1 and 2. And there's little to no attention paid to the effects of drugs over time. It's why Adderall is still legally prescribed even though it's been known to cause Parkinson's disease for decades. The mechanism by which it causes the brain damage is even known.
It's better that we have the FDA than we just allow people to try any thing they want, we had a system where people could effectively try whatever they wanted and you wound up with even more mountebanks and snake-oil salespeople than we have now and the consequences were even worse.
Ultimately the FDA regulations are only as good as the doctors and scientists working to get the necessary research done, which is to say it's not terrible reliable, but a huge step up from the unregulated supplements market where the standards are pretty much a matter of dissolving and being what they say it is. No particular guarantee that it works for anybody or that it won't make you blind in the future.
(Score: 0) by Anonymous Coward on Saturday July 02 2016, @06:15PM
Citation needed for the Adderall statement.
Drugs get pulled from the market and side effect lists have to be updated.
(Score: 2) by shortscreen on Saturday July 02 2016, @07:15PM
When people vote, their bad decisions screw everybody. If we can afford to allow that, then certainly we can afford to let people make bad decisions which merely screw themselves.
(Score: 2) by DutchUncle on Tuesday July 05 2016, @01:57PM
John W. Campbell wrote an editorial in Analog SF/SF many years ago suggesting a category of "Registered Quack". The idea was that instead of the FDA blanket prohibiting things, a patient with a serious disease could do whatever they wanted as long as (1) they signed up to take responsibility for themselves and not blame anyone else if it went wrong, and (2) they signed up for meticulous record-keeping and occasional testing by normal medical practice, just on the off-chance that they actually hit upon something worthwhile. This editorial was derided as fostering anti-science attitudes, but - especially considering Campbell's other writings - I read it as suggesting that people should be allowed to take chances and try extreme and/or experimental treatments when normal medicine has already told them they have little to lose.
"Tough Medicine" (article in New Yorker) discusses how some cancer cases have been cured by using medicines at higher doses or for different uses than officially approved, and how study results that should influence standard practice are instead discounted and ignored.
There should never be experiments on people without their full knowledge; but if someone with a serious (and especially terminal) disease is willing to be the next guinea pig in the hope that it works for them, why not let them?