Greg Conko laments an increasingly cautious Food & Drug Administration.
The FDA approved just 16 new drugs last year, and is on pace to approve only 18 this year. That's down from a high of 53 in 1996 and 39 in 1997.
After a few high-profile drug scares, such as the 2004 withdrawal of Vioxx from the market, FDA officials have become gun-shy about approving new products. After all, the agency receives scathing criticism from Congress and the press when an approved drug turns out to be more risky than expected — but rarely for keeping beneficial ones off the market.
Last year alone, the FDA rejected five new cancer drugs, including a breakthrough treatment for prostate cancer called Provenge. A panel of cancer experts that advises the FDA on new drug approvals unanimously agreed that Provenge was safe, and voted 13-4 that it was effective enough. But the FDA demanded still more testing that may delay approval for three years.
As Conko explains, this excess of caution means that terminally ill patients are blocked from obtaining treatments that could save their lives. When the FDA plays it "safe," it can cost people their lives.
Seems this cycle has gone around a few times for the FDA in my 56 years.
And we know this figure with precision exactly how?
Perhaps ıt shouldn`t be the FDA`s call. Perhaps the FDA should stıll *approve* drugs, but that approval shouldn`t be a precondıtıon to use of the drug.
On a more pragmatıc note, low-probabılıty problems wıth drugs often don`t come out untıl the drug ıs wıdely used; the FDA could approve these drugs subject to contınued FDA-supervısed data collectıon. Best of both worlds.
Just because fewer drugs are being approved does not necessarily mean that the FDA is getting "gun shy". It may simply be that drug manufacturers are submitting fewer drugs for approval. The editorial provides very little substantive evidence that the FDA is dragging its heels.
To make it easier for customers to understand each label can be given a number. e.g. 7 is more approved than 3, zero would have been a banned product.
But doesn't the FDA regulations stop drugs that would kill people? Not by more than an order of magnitude. The Vioxx debacle that the FDA was so criticized about had led to the death of less than 100 people, while providing pain relief not otherwise obtainable to millions. Thalidomide led to a few thousand birth deformities. As with most other regulations, the cost does not justify the benefit. Pharmaceutical companies do not make profits by killing their customers.
it could also mean that drug companies are wildly over optimistic about the effectiveness of the drugs they manufacturer.
Most graduate from law school with a base of knowledge. They're overworked. The information they get on new drugs is provided (surprise!) by drug reps. While the law has cut down on the the amount of trips to Cancun, the number of "seminars" and free lunches (literally) is staggering, and this is just to get the doctors to prescribe to increase market share and to encourage off-brand uses. Sometimes it becomes a scandal (see, e.g., neurontin). Usually it goes unnoticed.
Or do you think drug comapanies pay out the vast sums to young, attractive drug reps out of the goodness of their hearts? You know, welfare for the beautiful?
Anyway, this is only part of the problem. As soon as doctors are allowed to be the gateway, then patients are allowed to pressure dotors into prescribing the medication. And drug companies can 'market' (either directly if allowed, or indirectly by putting the information on the internet for patients) the information to patients, who will then pressure the doctors.
If long-term studies haven't been done to show a risk, then the doctor may just prescribe the drug rather than deal with a 15 minute badgering session with the patient.
So it goes.
Oh yeah.... allowing a medication to combat *nausea* versus thousands of birth deformities. Hmmm... that's a great example.
(BTW, later it was found that thalidomide might have uses for things such as leprosy . . . but that was found later, and it is now given only when there are asurances the patient is not, and will not become pregnant)
Let the FDA (and every other drug and diet regulatory agency of government) pontificate all they want about the safety and efficacy of any substance on the planet, naturally occurring or artificially manufactured. Let them commandeer advertising time and space from every communication medium to shout from the rooftops that l-tryptophan causes auto-immune disease, or that sassafras tea causes cancer, or that Linus Pauling was just an ignorant cow college graduate.
Then we, the
peoplehopelessly ill-educated and stupid subjects of infinitely wise and benevolent rulers, can make our own dietary and pharmaceutical choices, thoroughly informed of all dangers, real and imaginary, seen by our omniscient government overlords.Just don't let them ban anything.
Can you explain why you view any of this as a problem? It all sounds like a pretty good system to me.
No, it doesn't. There are a number of reforms to be made (for example, loosening the rules for terminally ill patients, increased resources for the FDA, changing the mechanism for approval) that would increase the availability of drug and solve the problems that we have while not greatly increasing the many problems that we already have.
A little knowledge is a dangerous thing. Studies have shown how bad *doctors* are at understanding the statistics related to pharmaceuticals. And you want the patients to do it. There's a difference between the government telling you what food you can eat (I can haz cheezburgerz?!?!) and the government testing potentially harmful drugs before they enter market. If you can't see that, I don't know what to do for you.
Just because fewer drugs are being approved does not necessarily mean that the FDA is getting "gun shy". It may simply be that drug manufacturers are submitting fewer drugs for approval. The editorial provides very little substantive evidence that the FDA is dragging its heels.
Hm, even if true, why could it possibly be that “drug manufacturers are submitting fewer drugs for approval”? Could it be that the oh-so popular social disapproval of the “evil” pharmaceutical corporations is having some kind of (gasp) effect?
As HIV-positive blogger and columnist Andrew Sullivan wrote a while back: “Thank God for the evil pharmaceutical companies. One day, when the history of this period is written, I have a feeling we will look back with astonishment as we recognize that advances in medical science, particularly pharmaceuticals, were arguably one of the most significant developments of this era. And yet the people who pioneered these breakthroughs were… demonized and attacked. Baffling and bizarre. I'm merely grateful the attacks haven't stopped the research progress. They've merely slowed it.”
"Oh yeah.... allowing a medication to combat *nausea* versus thousands of birth deformities. Hmmm... that's a great example."
My point is that the WORST example people can think of, to wit Thalidomide, only harmed (severely, though did not kill) thousands of people. Tragic. Yet the regulations to prevent that routinely lead to MILLIONS of premature deaths from the delays in getting drugs to market. This has been well documented in the economics literature for more than 4 decades.
The problem (if you can call it that) is twofold:
1. First, a great deal of the primary research for the medications that the pharmaceutical companies make is done on the gummint dime.
2. Second, they are corporations. There is nothing wrong with that, but as corporations, they seek to maximize profit -- not serve the public. If you are were the director of random BigPharma, would you want to develop the new class of antibiotics (low profit- administered short time, rare repeat, resistance developed) or the new lifestyle drug (anti-depressant, for example) that will be expensive and have a repeat customer for a long time? This creates misaligned incetives between the public health and the corporations, which is what people sense when they are "unfairly maligned". They are simply doing what corporations should do- maximize shareholder wealth. If this means spending more on marketing than R&D, then so be it. But it explains why there is the perception, and why other entities (private, like BG Foundation, or Government) need to step in to fill in the voids.
1. Please cite your sources. It couldn't have been well documented "for more than four decades" because our current regime is barely four decades old, and while some arguments are made, the cost-benefit analysis is notoriously tricky (if a drug gets yanked early, how do you extrapolate the costs of long-term use?). In short, prove it.
2. Thalidomide is not the only drug that has been kept off the market. It happened to be the most well-known one yanked, and the rallying cry after it led to original legislation for drug testing in '62.
Thalidomide is not the only drug that has been kept off the market. It happened to be the most well-known one yanked…
As pointed out above, thalidomide is not “off the market.” It has been approved by the FDA for several purposes (though precautions against any resulting births are mandated) and is being studied for others.
Thus, once again (much like the case with DDT) the “Ban it!” crowd are proved wholly too simplistic.
Perhaps because I pointed it out.
(BTW, later it was found that thalidomide might have uses for things such as leprosy . . . but that was found later, and it is now given only when there are asurances the patient is not, and will not become pregnant)
Pity they couldn't have tested it first and found those pregnancy problems *before* putting it on the market. If only there was.... some sort of agency... that, uh, tested it? Before approving it for morning sickness . . .
BTW, not such a great hit job, that.
At least for the major dıseases, dedıcated research ınstıtutıons regularly publısh (ın extensıve detaıl) the scıence-based best-known-course of treatment. I would poınt you to the one for bre*st cancer, but the fılter on thıs computer ıs blockıng ıt. Where doctors (and thus patıents) get ın trouble ıs when small-town docs devıate from the treatment guıdelınes publıshed by those who DO have tıme to do the readıng. For the less common dıseases, one should probably vısıt a specıalıst anyway.
Part of the solution, as others have pointed out, is to turn FDA into an information agency, with required labeling.
For many years, addicted smokers wanted nicotine gum but the FDA forbade them from getting it. If that isn't pernicious, I don't know what is!
Peltzman, Sam (1973), ‘The Benefits and Costs of 1962 Amendments to the 1938 Food, Drug andCosmetic Act’, in Landau (ed.), Regulating New Drugs, University of Chicago Center for Policy Study.
Peltzman, Sam (1973), ‘An Evaluation of Consumer Protection Legislation: The 1962 Drug Amendments’, 81 Journal of Political Economy, 1049-1091.
Regulation of Pharmaceutical Innovation: The 1962 Amendments (Evaluative Studies 15) American Enterprise Institute for Public Policy Research (1974)
Now Loki is quite correct that 1973 was not 40 years ago. My apologies to the group for gross exaggeration. The yearly excess deaths in the United States from FDA regulation have only been known for 35 years. I wonder if this is more or less than Loki's entire life...
Both are bad.
The question is how you strike the balance.
This is nothing new.
"We are shown again, that there exists two types of error, Type I and Type II error . . .
Both are bad.
The question is how you strike the balance.
This is nothing new."
This is not exactly right. The more fundamental question is who gets to strike the balance. Why should my preference for, say, type II error prohibit your preference for type 1 error? The fundamental problem with the FDA is not that it makes the wrong choice, but that it mandates that choice for everyone, well informed or not.
O RLY? Sam Peltzman twice and an article from the AEI? Ummm.... all three articles before the FDA included cost-benefit analysis?
There are (good) arguments to be made for certain reformations to the FDA approval system.
(BTW- by the Peltzman effect analysis, we should all be driving with a big spike pointed at us in our steering wheel and no seatbelts)
"while some arguments are made, the cost-benefit analysis is notoriously tricky"
Those arguments, and others, led to the inclusion of the CBA in the FDA.
(also, I missed a line- while some good arguments are made for certain reformations of the FDA approval system, I haven't seen a credible argument for dismantling the gateway system)
Drug makers have an option: To pursue FDA approval or not.
If the drug maker seeks and receives FDA approval, through the typical onerous process, the drug maker is immune to suit from product liability suits related to the drug's safety/efficacy.
If the drug maker chooses not to receive FDA approval, the maker can distribute the drug, but is subject to the full panoply of suits and concomitant risk.
That will encourage makers to seek FDA approval, and ensure that if makers elect not to pursue FDA approval, that they will be held liable by the market for that decision in private suits.
As a medical student, i can't even count how many times i've been told this is wrong. most of these recommendations are written entirely or in part by pharmaceutical companies who have an obvious incentive to push "science based" recommendations that favor their new still patented,and expensive, drugs over older generic drugs that may be as, if not more, effective.
And as someone who has spent time as a (sorta) doctor in a hospital, i can't even count the number of times that the "best-known-course" of treatment (i don't think you are using the correct term here) is too vague because there just isn't enough research on a certain subgroup of patients to make evidence based recommendations or the best available research is conflicting.
and i'm not sure what you mean by "extensive detail"...guidelines are often quite vague and open to interpretation.
Take a look at Beta Glucan sometime and ask yourself why you've never heard of it.
i take you have never heard of orphan drugs.
1. I wonder how many of these new drug applications are actually for new drugs, and how many are for "me too" versions of existing drugs.
2. I wonder if Mr. Peltzman also took into consideration the excess deaths caused by the pre-1962 regulatory regime. The articles are not available on-line to non-subscribers, so I can't see for myself, but maybe a kind soul out there has access and can answer the question.
3. The no free lunch principle applies here- some regulation is indeed needed (unless you want to return to pre-1932 days, where drug companies were pretty free to do as they pleased, subject to the discipline of the marketplace- in practice none), but which kind?
you need fda approval for genric as well as new formulations and new slats esters combinations etc..and to use an already approved drug for new purposes.
the author is only counting drugs that are brand new drugs that are classified by the FDA as new molecular entities, (class 1 New drug approval) not generics and not new combinations or esters or off label uses.
A few things...
From a patent perspective, a drug involving Beta Glucan could certainly be patentable: one may patent the procedure for concentrating the molecule, engineering a time-release delivery, etc. Process patents could be very effectual with any therapy that uses a naturally-occurring substances.
As for your rhetorical question: my physician, who specialises in breast cancer, has done research on high-fiber diets and the effects on breast abnormalities. So some of us have heard of this... from our own doctors.
And that makes sense...why? The government will gladly assume I have the good sense not to try to treat my cancer by going out in the back yard and making a tea of some random root, on the advice of my Mayan-ancestry neighbor, but I can't be trusted to make up my own mind whether the risk of heart problems is worth taking this or that pill from Pfizer?
It's bizarre logically inconsistent crap like this that makes ordinary people want to hang lawyers and purge all their wretched works from the memory of man.
I'm happy with the FDA reviewing drugs, and publishing lots o' handy-dandy consumer guides, like the EPA publishing mileage figures, and there's nothing wrong with requiring warning and information labels on drugs, like the nutrition labels you have to put on junk food. That's a good role for government: spread information that the public needs to make good choices, and the provision of which may conflict with the interests of private enterprise.
But this business of forbidding people to take a flyer on maybe dangerous drugs even if, you know, they're already dying, is not merely stupid -- it's inhuman.
Pity they couldn't have tested it first and found those pregnancy problems *before* putting it on the market. If only there was.... some sort of agency... that, uh, tested it? Before approving it for morning sickness . . .
But, see, there isn't one. You don't think the FDA actually does the testing, do you?
From http://www.fda.gov/cder/regulatory/applications/
It is the responsibility of the company seeking to market a drug to test it and submit evidence that it is safe and effective. (See "Testing Drugs in People" in the July-August 1994 FDA Consumer.) A team of CDER physicians, statisticians, chemists, pharmacologists, and other scientists reviews the sponsor's new drug application (NDA) containing the data and proposed labeling.
You got me. You're right, the FDA doesn't *in fact* do the testing; the companies test it (under a set of regulations promulgated by the FDA) and then the FDA approves the drug. While I was careless in my language, I'm sure most of the people here are relatively certain that the FDA barely has the employees to look at the applications, and certainly not to do the testing. Also, the US doesn't have 57 states. Happy? In addition, I incorrectly used my ellipses.
DMN,
The snarky response is that I'm sure you have your ticket paid to Somali, where there is much less government interference. The longer answer (which would take too long to go into here) invvolves the delicate balance between free markets (GOOD!) and market failure (BAD!). Or perhaps the tension upon classical and neo-classical economic theory upon which most libertarian belief relies and behavioral economics (aka observations in the real world) upon which practical applications are built. And I get aggravated by those theorists who insist on tearing down that which works in practice (as opposed to working to improve it) based upon pithy ideals of theory. Today's libertarians are yesterday's Marxists, ignoring the realities of the world and peculiarties of people to advance their own utopia, both of which would lead back to the stone age.
As a French Engineer said to an American Engineer after seeing a bridge buit, "Yes, it works great in practice, but how does it work in theory????"
As for your pithy line.... why do you hate life?
As for "working in practice," that's the whole bone of contention.
The point of contention is whether, in my mind, is whether there should be a gatekeeeper or not. In other words, ddo we amend the system, or allow people to take anything they want to, whenever they want to. While I am generally amenable to the idea of allowing people to screw up their own lives, I think the specific conjuction of sick, desperate people combined with companies beholden to the profit motive (again, not a bad thing generally) combined with the well-documented studies that show how bad people are measuring statistics in general and risk specifically argue for a regulatory regime in this case. If you're arguing for a change of the FDA's 'fast track' implementation for the chronically ill, I'd love to hear it. Your pithy quote did not indicate it.
Also, I do not equate your situation to Rosa Parks (is there a libertarian equivalent to Godwin's law?). Simply noting that the great examples of 'failed states' (aka no government) don't work so well. To which libertarians often argue for their own brand of more limited government, often from a position at a public university.
Don't be so one-sided. If any drug that the company claim that it's safe get approved just on their words, we'll reduce the population of USA very quickly. Especially when patients don't have the right to sue a company for a drug that has been approved by FDA.
The author's use of the term "new" actually says very little. It is my understanding that the repackaging of an existing product, in a different strength, for example, rewsults in a "new" drug application. Same for relabeling an existing product that will now be approved for children (normally, initial approvals are only for adult use).
The FDA's website indicates that a "New Drug Application" is required for all of these cases, except for generics (which only require an "Abreviated New Drug Application").
A doctor once prescribed me an antibiotic the day the news came out it caused severe liver damage, and should be used only in extreme situations. I did not take any. Later, my wife was part of a test program for an NSAID which eventually was not released. Both antibiotic and NSAID have common, safe alternatives, and did not "need" approval.
Actually, all antibiotics need alternatives. Blame overprescribing doctors. Blame obnoxious patients/parents (but my son has an earache, can't you do SOMETHING??!!!). Blame BigAgra (shovelling the antibiotics down the livestock). Truth is, bugs are getting resistant faster than the drugs are coming down the pipeline now. Absent a radical breakthrough- we'll be in a pre-sulpha, pre-penicillin world.
The very drug panic central to legislation that empowered the current FDA testing regime, the Thalidomide panic, is a case in point. Thalidomide's anti-angiogenesis properties caused terrible birth defects. In response, panic driven legislation empowered the FDA to ban any drug for all purposes, not just for situations where its only harm was known.
It took over 30 years for even a scintilla of common sense to find its way into FDA policy on Thalidomide. That's 30 years during which the drug that actually cures some cancers (by much the same mechanism that it caused harm) was forbidden for any purpose, including curing cancer.
How many MDs, PhDs and MPHs does it take to produce an atom of common sense? In 1962 it was possible for even a plebe to reason that since the only known harm was in utero, then a limited ban on prescription to pregnant women would prevent the known harm. But none of those learned but government empowered physicians and scholars, or not enough, did so. The FDA banned the drug for all purposes, for all patients, and insofar as was possible, for all time.
How many people have to die just so some apparently well educated bureaucrats can pretend that ham handed stupidity is doing the right thing?
As Thomas Jefferson remarked in Notes on the State of Virginia, Query XVII:
Why not play it really safe, and not approve any drugs at all?
As for thalidomide, while it did take the FDA to long to approve the new uses, it is also true that these new uses were not discovered until after the drug was banned. It was being used, inter alia, to treat morning sickness. Not too bright. And now it is only used when the person is not, and could not be, pregnant. This argues for a reform of the appproval process, not for getting rid of the approval process altogether.
Finally, many of the posts make me a little crazy. They go to the belief that somehow, somewhere, there are all these mystical cures that would be available if only "the gummint" wasn't keeping them from us. This "Natural Cures" conspiracy-style belief is extremely hard to debunk, similar to the belief in flying saucers.
rfg - "Me too drugs" are also beneficial. They have slightly different effects and side effects, and it gives doctors and patients more choices to find a drug that works well in a particular case. Even if they didn't have slightly different effects they would create competition and help control costs.
What "reform of the approval process" could possibly prevent that? A rule against being stupid, craven or cruel? A rule against bureaucrats not giving a damn who suffers as long as they can cover their posteriors by distorting some kind of "precautionary principle" beyond all human reason?
You keep digging your hole. The reason there were fewer birth defects in the US than the rest of the world was because, um, an INTRANSIGENT FDA administrator used his wiles to keep the drug off the market as long as he could using the 1938 Drug &Cosmetic Act. Other, not as cautious countries, reaped the whirlwind of that drug tragedy. And that was an easy one to spot, as it manifested quickly, ass opposed to the more long-term problems we see associated with some pharmaceuticals.
What a horrible guy. Using, like, caution and stuff. The pregnant women in the USA should have just relied on the pharamceutical company's marketing materials.
Why do you hate American kids? Or, why do you like flippers?
http://leda.law.harvard.edu/leda/data/351/Lutz.html
Don't you get it? People with training are ELITISTS! We can't have ELITISTS like doctors and scientists telling us what drugs are safe! Hell, I don't want trained pilots flying planes, there should be lotteries so anyone, even Fub, could be the pilot. And if there's a plane crash, well, freedom isn't free, y'know.
I'm very, very anti-euthanasia... which may be some of the reason that I disagree with the FDA's policies.
Incidentally, I'm assuming that you are against chemotherapy. Many cancer deaths are a result, not of the disease itself, but the toxicity of the treatment or the side effects of it. Is chemo the functional and moral equivalent of euthanasia, in your mind?
As it is now, the FDA - not doctors - decide whether or not a patient, even a terminally ill one, may take certain medications, even life-saving medication. We do not live in a world in which patients may prescribe themselves medication, nor a world in which a primary care physician would be treating advanced pancreatic cancer.
Or are you denouncing doctors as untrained and lacking in medical knowledge?
There have been revisions to the fast-track system. If you are arguing that the rules should be amended (they were a little over two decades ago to include cost-benefit for example), I would agree. If you want further revisions to the fast-track, I could agree with you on that as well. But the belief that there is a *wonder drug* that will solve all the problems just waiting on the wings is a myth. As for doctors- most are overworked and don't have the time to fully digest the best available efficacy studies, which aren't always on the up-and-up. Hence the different in techniques, proceedures, and outcomes based on location.
You want to speed drugs down the pipeline? Clear up the divided property rights / IP problem that has been documented in drug development.
FDA shouldn't do anything more then set standards to approve medicine, and follow them. And should something get missed, improve for the next time.
Oh, and let's turn off the extraordinary profit margins made in the U.S. on Prescription Drugs. If congress wants to provide funding to any research groups, then how about retaining rights of patent in such cases, since in all reality, the people did pay for the research.
Besides, I don't see any constitutional powers that allow regulation of anything beyond commerce between the states, and commerce between foreign nations, and the U.S. So why do we allow them to price fix?
Why? So pharmaceutical companies will stop making them and go into a more profitable industry?
A better question: how much is it worth to you to be alive and have a good quality of life? IMHO, prescription drugs are a bargain, at any price.
Loki - wasn't saying that there is a miracle drug or anything like that. Not into mythology... just into allowing dying patients to try whatever they can.
I don't think we're in disagreement that reforms are needed to allow patients who are already diagnosed as terminal access to medications that may help, even if not approved (it's not like the side effects are going to worry them that much). The countervailing issue is that these patients are, understandably, desperate, and we don't to turn their desperation into a free-for-all. I think there's a middleground between completely unfettered access and our current system for dying patients.
Yet the FDA chose to ban the drug entirely.Denying all use of the drug, even to cure serious and life threatening
diseases where the risk of the known harm is not present, is not caution, but at best irrationality.
However, "stuff" is at best a euphemism. Even a fully effective ban on the drug to treat serious or life threatening diseases in men could not possibly prevent the known harm. Men do not become pregnant. To call that ban prevention of the known harm is just bull "stuff".Pregnant women in the USA should, like everyone else, be allowed to rely upon the advice of their physicians informed by science.
Physicians and women patients with serious and life threatening diseases are capable of weighing the known risks of the drug in the context of their specific medical situation, and preventing the known harm by not using it in specific circumstances, far more effectively than any broad ban preventing adequate treatment of those diseases. Even the least skilled physician can read and understand a drug label's specific contraindications.I decline to repeat your sick taunting of those who have suffered harm.
Intentionally causing some to suffer simply because others have suffered is neither good medicine nor science, but a tit for tat political game played with contempt for all who suffer. You have just amply demonstrated your true intentions.