Hacker News Board Hacker News, browsed like a classic message board
Live data from Hacker News

Is the FDA Too Conservative or Too Aggressive? Analysis of Clinical Trial Design

papers.ssrn.com

Post reply on HN
Print view View original

11–20 of 34 posts

Re: Is the FDA Too Conservative or Too Aggressive? Analysis of Clinical Trial Design

#11

Post by KingMob »

kesor wrote: post #2

A family member of mine has tried to participate in a clinical trial. The trial also forces him to forgo any existing medication, basically making his illness intolerable. There is no indication in the trial if he is being served with real medication or a placebo, but his life has been extremely affected just by participation. Naturally he quit the trial. How would this statistical analysis address these common behav…

There's a whole statistical literature on survival bias. The basic equations all require "independent and identically distributed" data loss. As soon as that's not the case (e.g., more placebo patients drop out than treatment patients), you have to compensate for that.

That being said, I suspect this analysis is operating at a higher level where survivor bias isn't as relevant, because it's looking across many studies at once.

Re: Is the FDA Too Conservative or Too Aggressive? Analysis of Clinical Trial Design

#12

Post by minthd »

cryoshon wrote: post #6

I've been on the science side of a number of FDA clinical trials for a few years now. The FDA is too conservative with many metrics, and too aggressive with others. The clinical trial pipeline is tortuously slow for everyone involved-- the EU equivalent agency has done a lot to accelerate the process fruitfully. Eclipsing these FDA malregulations is the problem of transparency; if the FDA forced clinical trials to pu…

Is there any realistic possibility Americans could use drugs approved in Europe ?

Re: Is the FDA Too Conservative or Too Aggressive? Analysis of Clinical Trial Design

#13

Post by tim333 »

transfire wrote: post #10

TriCorder X-Prize? FDA has done nothing but hobbled it's development. DNA screening for the masses? FDA shut down 23andMe. Terminally ill and willing to try an experimental drug? FDA says, "too bad" (and the Supreme Court agrees).

They should have a principle of maximising the public good which I feel would allow some of that stuff.

Re: Is the FDA Too Conservative or Too Aggressive? Analysis of Clinical Trial Design

#14

Post by dragonwriter »

minthd wrote: post #12
cryoshon wrote: post #6

I've been on the science side of a number of FDA clinical trials for a few years now. The FDA is too conservative with many metrics, and too aggressive with others. The clinical trial pipeline is tortuously slow for everyone involved-- the EU equivalent agency has done a lot to accelerate the process fruitfully. Eclipsing these FDA malregulations is the problem of transparency; if the FDA forced clinical trials to pu…

Is there any realistic possibility Americans could use drugs approved in Europe ?

Americans can obviously use drugs approved in Europe in Europe; drugs approved in Europe but not the USA cannot generally legally be imported, sold, or prescribed in the USA, however.

Re: Is the FDA Too Conservative or Too Aggressive? Analysis of Clinical Trial Design

#15

Post by kirsebaer »

transfire wrote: post #10

TriCorder X-Prize? FDA has done nothing but hobbled it's development. DNA screening for the masses? FDA shut down 23andMe. Terminally ill and willing to try an experimental drug? FDA says, "too bad" (and the Supreme Court agrees).

The FDA approves requests to use non-approved medications 99% of the time. When you hear about someone unable to access an experimental drug, it is almost always because the pharmaceutical company does not want to make the drug available early (for various reasons).

> At the meeting, a senior FDA representative indicated the agency has approved over 99 percent of expanded access requests submitted via single patient or emergency INDs since 2009, suggesting the regulatory agency is not a major barrier to expanded access. As such, provided the access request is reasonably related to the potential benefits of the drug, the biopharmaceutical company is almost solely responsible for the decision and liability regarding whether to grant expanded access to an individual.

http://healthaffairs.org/blog/2014/07/31/individual-patient-...

Re: Is the FDA Too Conservative or Too Aggressive? Analysis of Clinical Trial Design

#16

Post by kirsebaer »

minthd wrote: post #12
cryoshon wrote: post #6

I've been on the science side of a number of FDA clinical trials for a few years now. The FDA is too conservative with many metrics, and too aggressive with others. The clinical trial pipeline is tortuously slow for everyone involved-- the EU equivalent agency has done a lot to accelerate the process fruitfully. Eclipsing these FDA malregulations is the problem of transparency; if the FDA forced clinical trials to pu…

Is there any realistic possibility Americans could use drugs approved in Europe ?

Any American MD can ask the FDA for "expanded access" to use a medication not approved by the FDA. 99% of the time the FDA grants expanded-access requests.

However, a recent review in NEJM explained how it is time consuming and expensive to apply for expanded-access and often the pharmaceutical companies refuse to distribute the drugs even after the FDA grants expanded-access.

http://www.nejm.org/doi/full/10.1056/NEJMhle1409465

"Compassionate use" is a separate, but related concept with different rules than "expanded access". And in the EU expanded access is often called "named-patient".

Re: Is the FDA Too Conservative or Too Aggressive? Analysis of Clinical Trial Design

#18

Post by rayiner »

transfire wrote: post #10

TriCorder X-Prize? FDA has done nothing but hobbled it's development. DNA screening for the masses? FDA shut down 23andMe. Terminally ill and willing to try an experimental drug? FDA says, "too bad" (and the Supreme Court agrees).

They didn't "shut down" 23andMe. They stopped them from marketing unproven health diagnoses derived from the DNA tests.

Re: Is the FDA Too Conservative or Too Aggressive? Analysis of Clinical Trial Design

#19

Post by fedups »

rayiner wrote: post #18
transfire wrote: post #10

TriCorder X-Prize? FDA has done nothing but hobbled it's development. DNA screening for the masses? FDA shut down 23andMe. Terminally ill and willing to try an experimental drug? FDA says, "too bad" (and the Supreme Court agrees).

They didn't "shut down" 23andMe. They stopped them from marketing unproven health diagnoses derived from the DNA tests.

I think it went beyond a marketing ban, unless 'marketing' extends to selling and displaying health related information.

Re: Is the FDA Too Conservative or Too Aggressive? Analysis of Clinical Trial Design

#20

Post by vitd »

minthd wrote: post #12
cryoshon wrote: post #6

I've been on the science side of a number of FDA clinical trials for a few years now. The FDA is too conservative with many metrics, and too aggressive with others. The clinical trial pipeline is tortuously slow for everyone involved-- the EU equivalent agency has done a lot to accelerate the process fruitfully. Eclipsing these FDA malregulations is the problem of transparency; if the FDA forced clinical trials to pu…

Is there any realistic possibility Americans could use drugs approved in Europe ?

Yes. My wife was given medication not FDA approved by her doctor. She had to order it from a pharmacy in Canada, but it was all legit. The (US) doctor wrote her a script and filled out some sort of paperwork for the government, as I understand it.

And no, her doctor isn't some quack who regularly prescribes unapproved medicine. Apparently this particular drug is approved in Canada and Europe, but the company that makes it is small enough that they haven't yet decided to apply for FDA approval because of the cost. (Or at least, that's what I've been told.)

Post reply on HN