

Sick kids, desperate parents, and the battle for experimental drugs - Mz
http://www.bostonglobe.com/magazine/2015/07/15/sick-kids-desperate-parents-and-battle-for-experimental-drugs/Hle3CTwriFfTXoOvQ7TbZP/story.html#

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Animats
_" Even some of the drugs showing early promise and that are fast-tracked for
approval by the FDA end up failing."_

Which is the problem. Most new drugs don't work. Some work in some animals but
not in humans. Some have side effects in humans. Some seem to work at first,
but the effect doesn't last. Some have long-term side effects. Some are
harmful to some part of the population. That's why so much clinical testing is
required to find the ones that do work.

The gold standard of clinical testing is "better off 10 years later". Drugs
have made it all the way to approval and later been pulled because they failed
that test.

~~~
gwern
That's what scares me about the parents the article describes. They are
exactly the sort of people who would gladly help dismantle RCTs and try to
return drug and medical treatment development to a pre-scientific process. The
usage of RCTs is already fragile and threatened - I remember during the Ebola
outbreak, there was a ton of pressure to use limited supplies of Zmapp on
patients as fast as possible and the suggestions to make the most of the
opportunity by allocating scarce doses via lottery were, as far as I know,
ignored. That in a situation where it was vital to know as soon as possible
whether we had a useful Ebola treatment or not in case it went pandemic!

~~~
JoshTriplett
If you're evaluating something for use to keep healthy people healthy, then
sure, you want to know they'll still be healthy after 10 years and not
afflicted by some terrible long-term side effect. The potential downside of
taking it may outweigh the downside of not taking it.

On the other hand, if you're evaluating something to keep people from dying,
then "not dead" is sufficient in the short-term _while_ continuing to watch
for long-term effects. The downside of taking it cannot possibly outweigh the
downside of not taking it. (Assuming some reasonable baseline expectation that
it treats the condition it intends to treat.)

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Thriptic
One of the big impediments against more companies engaging in compassionate
use is required adverse event reporting to the FDA. To my knowledge
(clinicians / clinical research specialists please correct me here if I'm
wrong) companies are required to report any adverse events experienced by
patients granted early access to a drug under compassionate use. Any adverse
event reported may make it harder for them to bring a drug to market. In
addition, it is very difficult for companies to screen these patients or
guarantee that they are adhering to regimens as they would in a clinical
trial. This biases companies against providing the drugs, even though many
would want to. In my opinion, the FDA should drop this requirement and allow
for universal access to medications for terminal patients under compassionate
use.

~~~
DanBC
What's the advantage of stopping reporting of adverse events? Doesn't that
just make it easier for dangerous / ineffective (but fantastically expensive)
drugs to come to market?

~~~
Thriptic
In very sick patients (those generally filing for compassionate use) it's
sometimes difficult to pin down whether the drug actually caused a problem or
death. Were the patients properly adherent? Did they have comorbidities? Were
they simply too sick and died?

A few deaths might cause the FDA to order additional trials which may actually
not be necessary, to the tune of millions of dollars. If you were a company
that spent years and hundreds of millions trying to bring a product to market,
would you want to take that risk?

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themartorana
This seems like a simple equation to me. The kid needs to be without further
recourse, and the drug company _needs_ to be indemnified. After that, what's
wrong with it? A last ditch effort is better than no effort, at least in the
hearts of the parents that are about to suffer the worst fate that can befall
a human being - losing a child.

(I know it's not "simple.")

~~~
erikpukinskis
You've basically got it, except if you have a ton of super sick people taking
the drugs it can mess up the results (when you're that close to death all kids
of unrelated stuff starts shutting down).

If you allow the drug companies to omit those people from the results, that
can be exploited. Drug companies could give risky drugs to a bunch of really
sick people and then use _those_ results to tailor the real trial to try to
hide side effects. The point of "report everything" is they can't monkey
around with the experiments.

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markvdb
Artificially expensive cures against rare diseases. Company blackmailing the
government to pay:

[http://www.standaard.be/tag/medicijnen-voor-
viktor](http://www.standaard.be/tag/medicijnen-voor-viktor) (Dutch)

