4 ms·

I'm not entirely comfortable with allowing this by policy. I could see this affecting the interpretability of results from such trials in the long run. If ther

by maldeh 12y ago

I'm not entirely comfortable with allowing this by policy. I could see this affecting the interpretability of results from such trials in the long run. If there's a rising proportion of mega-rich people in any given study, this could introduce significant sampling biases. Ethnicity, diet, lifestyle, gender - it all factors in and will have to be controlled for appropriately. Another concern is that this could introduce incentives into the eco-system that detract from the primary goal that is to obtain a drug that would be effective for as many people as possible (setting aside drugs with specificity as a goal for a moment). Imagine there's a drug A that happens to show very promising results on caucasians but not so much for africans, and a drug B that has more general effectiveness (hackneyed I know, but please bear with me). While a more impartial set of trials might have wound up selecting drug B (albeit after some passage of time), it's now possible drug A could get fast-tracked to market by virtue of being awash with funding from affluent volunteers while drug B never generates enough monetary interest to make it.

tempestn 12y ago
I think this concern was addressed in the article. The more advanced version of the idea would have wealthy donors fund studies, then receive the drug on a compassionate basis alongside the study, rather than actually participating in it. (There was a bit more detail to give the drug companies maximum liability coverage, but that was the basic idea.)
josu 12y ago
That's easy to fix, limit the number of places on sale to x% of the total.
_benedict 12y ago
Not only has this been addressed in the article, as mentioned by another commenter, but this market place is intended to apply only to Stage I to IIa trials, and currently only non-randomized trials. These concerns, even were they not already dealt with, would be moot as phase IIb and III trials would still need to be done via the normal (even more expensive) mechanism, with larger more robust populations.
- maldeh 12y ago
  Alright, that's fair. I'm not too familiar with the design of these trials, so I concede that my worrying about sample-poisoning is likely misplaced. Still, and I realise I'm coming dangerously close to sounding like a tinfoil spouting "money corrupts" as I type this, I would still be wary about how introducing massive amounts of private donations of this sort might impact the process of bringing drugs to the market. For the moment, this sounds great as trials could now have a source of funding where there currently is none... but it seems to me that this would further exacerbate the problem with the pharma industry focusing on cash-cow meds. Things could get such that it would be more profitable to target an exotic ailment with wealthy backers than to target an affliction disproportionately affecting the plebes. Not that I'm saying that we must not explore alternatives to the current policy of denying experimental drugs to terminal patients. I'm simply cautioning that we have limited resources and researchers, so we ought to be careful how policy changes influences their priorities over the years.
healthenclave 12y ago
You are right. More importantly hand picking individuals to be part of the study could result in Selection Bias. http://en.wikipedia.org/wiki/Selection_bias http://en.wikipedia.org/wiki/Selection_bias The other logical workaround would be to give the HNI donor a trail of the drug but not include (or interpret with caution) the results of that particular individual (which can be tough for rare diseases)

by maldeh 12y ago

tempestn 12y ago

I think this concern was addressed in the article. The more advanced version of the idea would have wealthy donors fund studies, then receive the drug on a compassionate basis alongside the study, rather than actually participating in it. (There was a bit more detail to give the drug companies maximum liability coverage, but that was the basic idea.)

josu 12y ago

That's easy to fix, limit the number of places on sale to x% of the total.

_benedict 12y ago

Not only has this been addressed in the article, as mentioned by another commenter, but this market place is intended to apply only to Stage I to IIa trials, and currently only non-randomized trials. These concerns, even were they not already dealt with, would be moot as phase IIb and III trials would still need to be done via the normal (even more expensive) mechanism, with larger more robust populations.

maldeh 12y ago
Alright, that's fair. I'm not too familiar with the design of these trials, so I concede that my worrying about sample-poisoning is likely misplaced. Still, and I realise I'm coming dangerously close to sounding like a tinfoil spouting "money corrupts" as I type this, I would still be wary about how introducing massive amounts of private donations of this sort might impact the process of bringing drugs to the market. For the moment, this sounds great as trials could now have a source of funding where there currently is none... but it seems to me that this would further exacerbate the problem with the pharma industry focusing on cash-cow meds. Things could get such that it would be more profitable to target an exotic ailment with wealthy backers than to target an affliction disproportionately affecting the plebes. Not that I'm saying that we must not explore alternatives to the current policy of denying experimental drugs to terminal patients. I'm simply cautioning that we have limited resources and researchers, so we ought to be careful how policy changes influences their priorities over the years.

healthenclave 12y ago

You are right. More importantly hand picking individuals to be part of the study could result in Selection Bias. http://en.wikipedia.org/wiki/Selection_bias http://en.wikipedia.org/wiki/Selection_bias The other logical workaround would be to give the HNI donor a trail of the drug but not include (or interpret with caution) the results of that particular individual (which can be tough for rare diseases)