FDA Dangles Golden Ticket to Spur Drugs for Neglected Diseases
npr.org
npr.org
It seems insane that drug approvals could be safely expedited by four months, at the suggested value to companies of $150-$300 MM, and this isn't done routinely. That revenue could easily pay for the extra eyeballs at FDA -- which are certainly much cheaper than the clinical trials themselves. I think if Congress were to question the FDA's workings, this could be prima facie evidence that the pace of drug reviews isn't set by hard constraints of public safety: it's slower than it could be, and agency leaders know it.
Regulators at the FDA are capricious and malign, and there are countless stories of petty revenge via increased demands for data and enormous imposed costs in time and money. This is the way things always go in the late stages of centralized, powerful bureaucracy with a side order of regulatory capture.
The only good thing I can find to say about the bureaucrats at the FDA is that they respond to regional regulatory arbitrage - to medical tourism - by loosening their grip and letting things through. Or at least they do when said treatments are so widely available elsewhere in the world that the risk of looking bad by continuing to block them in the US is worse than the risk of looking bad because one of the outcomes of a normal, low error rate in medicine gets picked up by the press. Look at simple stem cell transplants, now available in the US these last couple of years, for a good example of this in practice.
Amusingly, the top comment on a front page HN post [1] right now concerns one of the first court cases [2] that gave FDA some real power. Before this case, companies could sell a poison like emetic tartar and market it as a hangover drug. Selling stuff like this still happens today in countries without an FDA equivalent. [3] It even happens in countries with a drug regulatory body, but one that's weak. [4]
As with all regulation, there's a balance between benefit/risk. As you point out, there is a financial and time burden of regulation. But with warts and all, the work that the FDA has done and currently does far outweighs these burdens.
> The only good thing I can find to say about the bureaucrats at the FDA is that they respond to regional regulatory arbitrage - to medical tourism - by loosening their grip and letting things through. Or at least they do when said treatments are so widely available elsewhere in the world that the risk of looking bad by continuing to block them in the US is worse than the risk of looking bad because one of the outcomes of a normal, low error rate in medicine gets picked up by the press. Look at simple stem cell transplants, now available in the US these last couple of years, for a good example of this in practice.
I'm not quite sure how to respond to this rant. But I'll point out findings from a 2012 NEJM study comparing the FDA to the EU's EMA and Canada's Health Canada: [5]
"RESULTS: There were 510 applications for novel therapeutic agents approved from 2001 through 2010 — 225 by the FDA, 186 by the EMA, and 99 by Health Canada; among the applications, there were 289 unique agents. The median length of time for completion of the first review was 303 days (interquartile range, 185 to 372) for applications approved by the FDA, 366 days (interquartile range, 310 to 445) for those approved by the EMA, and 352 days (interquartile range, 255 to 420) for those approved by Health Canada (P<0.001 for the comparison across the three agencies). The median total review time was also shorter at the FDA than at the EMA or Health Canada (P=0.002). Among the 289 unique novel therapeutic agents, 190 were approved in both the United States and Europe (either by the EMA or through the mutual recognition process), of which 121 (63.7%) were first approved in the United States; similarly, 154 were approved in both the United States and Canada, of which 132 (85.7%) were first approved in the United States."
[1] https://news.ycombinator.com/item?id=7758508
[2] https://en.wikipedia.org/wiki/United_States_v._11_1/4_Dozen_...
[3] http://ijp-online.com/article.asp?issn=0253-7613;year=2007;v...
As long as we're throwing around bullshit: false hope and pseudoscience has caused more unnecessary death at the hands of tricksters and con men in the past two thousand years than all wars humans have fought or ever will fight.
Having worked in biotech I was never a fan of some (or many) of the FDA's policies but I found this insulting. You have no clue how hard it is to develop any medicine that doesn't harm the first few patients you give it to, let alone one that actually reaches the market to help thousands or millions. You have no clue what it is like to be the only gatekeeper between a $900 billion industry working on some of the hardest scientific problems of our time and 300 million people. Almost all of whom want their cake, to eat it now, and for the calories to magically disappear from their gastrointestinal tract.
The FDA is not a product of pharmaceutical companies, it is a product of a political system made of voters like you who decide their policies based on Libertarian pamphlets, Fox News, and Reddit. A political system that allows pharmaceutical companies to selectively withhold data, that has gutted the basic scientific research institutions that allows pharma to finance billion dollar ventures, and that mercilessly punishes any mistake that it itself is supposed to catch.
You get what you vote for.
http://fdareview.org/harm.shtml
The delay and large reduction in the total number of new drugs has had terrible consequences. It is difficult to estimate how many lives the post-1962 FDA controls have cost, but the number is likely to be substantial; Gieringer (1985) estimates the loss of life from delay alone to be in the hundreds of thousands (not to mention millions of patients who endured unnecessary morbidity). When we look back to the pre-1962 period, do we find anything like this tragedy? The historical record—decades of a relatively free market up to 1962—shows that voluntary institutions, the tort system, and the pre-1962 FDA succeeded in keeping unsafe drugs to a low level. The Elixir Sulfanilamide tragedy, in which 107 people died, was the worst of those decades. Every life lost is important, but the grisly comparison is necessary. The number of victims of Elixir Sulfanilamide tragedy and of all other drug tragedies prior to 1962 is very small compared to the death toll of the post-1962 FDA.
You can talk about the economics of human lives but the reality is simple: removing the FDA won't bring on a Golden Age of medicine that will bring back the dead. Instead, the companies developing the medicine that can actually save people, companies that are now "delayed" and overburdened by regulation, will be beaten down by a market of charlatans with big marketing budgets and limited liability.
Thanks to Facebook, I know of half a dozen former classmates who have close relatives that lost years of their lives because they chose to listen to some neo naturalist instead of their oncologist. That is the alternative. It already exists and destroying the FDA would unleash the flood of crazy that it has tried, with so little actual support, to hold back for all these years.
Is it the fact that the easy fruits have all been picked, there is nothing left to be discovered because everything at market already does a good job at a much better price point than any new drug? Or is it the fact that Pharma doesn't produce on a timescale that Wall Street likes, or both? Hell, Facebook is valued like Pfizer, but Facebook gives faster returns on capital.
Your argument is completely at odds with observable facts.
Currently, marketing budgets dwarf R&D budgets at the large pharma companies, so I don't expect the situation to improve any time soon.
Sure, looser regulations might save some lives but so does not using a seat belt. The only sane way to do such comparisons is by looking at both lives saved and lost. And if your looking at the Cost/befit of something you can't use a baseline of other countries without including their costs as well.
Edit: To be clear it's a huge but subtle point. If the FDA saves 10k more lives than X, and costs a total of 20k lives you have no idea if it's better than nothing without knowing X.
Actually it looks like the developed countries with more, better treated bureaucrats are doing far better.
[1] http://www.washingtonpost.com/r/2010-2019/WashingtonPost/201...
And for the "career bureaucrats", if you want a modicum of stability you need them, you can't have elections or change the rules every other year - what you need is institutional memory and predictability.
It would be better if the FDA had more power over largely fraudulent markets like homeopathy, naturopathy, herbal medicine, etc. But for the time being those scams are largely unrestricted.
I'm alive because of experimental treatments (bone marrow transplant), monitored by the FDA and others. At the time, patients with my profile had 90% mortality.
I will tell you firsthand, and watching my hospital friends die, that in most cases, the cure is at least as bad as the disease. Which is why doctors, who would know best, often choose to go out with grace (no heroic measures).
And yet science marches on.
Today BMTs are routine and in some cases are practically outpatient treatments.
The development and approval of new drugs and therapies are exactly as fast as they should be. Want something faster, then work towards it.
In other words: less talk, more walk.
Because:
1. the $10 million would come out of the FDA's budget while the $150 million doesn't; and
2. spending $10 million to clear the queue would do nothing to incentivise drug development for neglected diseases, such as leishmaniasis.
It's biting its own tail.
Not to mention creating new drugs without implementing any rational controls over their use in humans and livestock is like pouring more gas on the fire.
But I digress about leaving science to legislators....
http://www.roche.com/media/media_releases/med-cor-2013-11-04...