"I can't  imagine anything more heart-wrenching than believing that you or a  family member could be helped by something out there but you can't get  it," says Weinstock. The FDA is in a difficult situation, he adds,  because "they want to be as aggressive as possible in allowing people to  have access to as many drugs as possible."
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ExpandThere's a  misconception that the FDA is a bunch of bureaucrats, making arbitrary  decisions based on politics — but Weinstock says it's actually "people  like me, academics and scientists and clinicians, who get asked to serve  on committees... and the overwhelming majority of the time, the FDA  goes along with [our] decisions." Image via Nephron.
There are  plenty of drugs that do a great job of shrinking tumors in mice, but  turn out not to be suitable for humans, adds White. "Obviously you're  excited [when a drug works on mice]. But the problem is that 95 percent  of those new drug applications fail. They don't get FDA approval. 70  percent of them fail in that Phase One, which is the toxicity study.  That leaves with you with 30 percent. And then 60 percent of those fail,  and [most] of the time, it's because they don't work. That's nobody's  fault. It isn't surprising that progress is slow."
"It's a  tragedy when the drug is toxic and hurts people, and it's almost as  tragic when the drug doesn't work," says Weinstock. "We've seen that  recently with Avastin," which had its approval for breast cancer  revoked.
"Very good  people and very smart people spend their life trying to beat this  disease," says White. "So every time you see a glimmer of hope, you hope  that this is the great breakthrough... and obviously that gets hyped."  Part of this is because you constantly have to seek more funding for  your research, and everyone from members of Congress to your peers want  to fund research that looks exciting. And then, of course, there's the  media — and we certainly tend to get over-excited as well.
 
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