Are FDA Drug Approval Rules Irrational?

[Marat]

Prior to the establishment of the precursor organizations of the FDA at the beginning of the 20th century, Americans could buy any substance they wanted to treat their diseases as they chose. After the FDA came into full control of medicine, people could only access drugs and treatment modalities to ameliorate or cure disease if the FDA had already tested them first to determine whether they were 'safe and effective.' Today the process to establish safety and efficacy has become so painstaking that it usually requires 15 years of testing and about $300,000,000 of investment to bring a new drug to market.

 

But do these standards of safety and efficacy, which prohibit patients' access to these drugs on pain of criminal sanctions, make any sense for people who are either now in extreme suffering from incurable diseases or who are certain to die of their disease before the FDA approval process is finished? The FDA does grant some access to experimental, unapproved drugs on 'compassionate' grounds, but it is very slow and very stubborn in reaching these decisions, often giving in only after a long and expensive court battle. But who says that a government agency should have the right to make that decision or refuse to do so when dealing with a people who claim to be the freest in the world?

 

What if there were a drug which promised to cure all diseases and provide humans with a youthful existence for 150 years, but it would take ten billion years, longer than the human race will survive on Earth, for the FDA tests on its safety and effectiveness to be completed? Wouldn't the government override the FDA in that case, because the majority, rather than just a small minority of sick people, couldn't wait?

 

What if you were drowning in quicksand in an isolated swamp and there was only one tree branch within reach by which you could pull yourself up to save yourself? How would you react if an FDA official appeared as you were reaching up for the branch and forbade you to use it until 15 more years of testing of the branch for its safety and effectiveness were completed? Would that intervention seem rational, even if the official could demonstrate that pulling on a possibly rotten branch which collapsed might cause you to sink faster?

 

Shouldn't a free people have the right to access all medicines as long as they carry some government-certified warning about their potential risks or their unknown potential for risk? It is perfectly legal for me to run into a burning building filled with dynamite if I want to in order to save my relatives inside, but for some reason it is illegal for me to risk my health to a much smaller degree by taking some medicine the FDA has not yet approved. But what should belong to my autonomy more than my right to try to save my health and life as I see fit?

[ewmon]

It seems that, as societies become more advanced, they have more to lose, and so, the more they avert risks. America's FDA, Europe's EMA, Australia's TGA, etc do protect their societies. They seek to protect societies, not necessarily individuals. "The few must be sacrificed for the sake of the many."

 

Diseases that people were dropping like flies from decades ago are no longer unacceptable because other diseases have been cured. People see cures around them, and they yearn more for a cure. It's another version of "keeping up with the Joneses". Their disease must wait its turn, if it ever comes.

 

However, like Hugh and Chris Hempel, if you're really serious, you might find a way to follow the rules AND help your loved ones. This

is good.

[Twinbird24]

The FDA does have some aberrant rules and regulations. Here is an excerpt from a book I have (Natural Prescriptions by Dr. M. Giller):

"The supplement coenzyme Q10 (or CoQ10) has been of enormous help to my patients with congestive heart failure. (Unfortunately, CoQ10 is on the FDA's 'hit list,' not because it's dangerous, but because the FDA claims that it has no benefit."

 

I also have another book (which I can't find at the moment so I can't tell you the name of the book or provide any specific examples) that compares the prices of certain drugs and medical equipment sold in USA to other countries around the world, the prices in USA (for the same drug) are substantially higher than in other countries.

 

I also must note, Marat, that your examples are extremely over-exaggerated. There will never be a drug that cures EVERY disease and provides a youthful existence for 150 years.

Edited July 9, 2010 by Twinbird24

[Marat]

I quite agree that there will never be a drug which provides that degree of longevity and serves as a panacea. But that was only a hypothetical construct to generate a thought-experiment showing that the whole society would rise up in rebellion against the same FDA rules which now slow down access to life-saving medicines for dying patients, were the need of the whole population comparable to that of the minority of seriously ill people.

 

The example of co-enzyme Q10 you give is interesting, since the drug companies developing the new class of statin drugs recognized that these drugs would have seriously deleterious side-effects because they would deplete patients' co-enzyme Q10 levels. They proposed to market statins only in combination with co-enzyme Q10, but this never happened for a variety of reasons. The result has been that statin drugs, which are now taken by an enormous number of people, cause a huge range of severely damaging side-effects, from rhabdomyelosis to gum loss, and from renal failure to exhaustion.

[Cap'n Refsmmat]

After the FDA came into full control of medicine, people could only access drugs and treatment modalities to ameliorate or cure disease if the FDA had already tested them first to determine whether they were 'safe and effective.'

You can still get them. They just can't be labelled "medicine." See nutritional supplements and herbal medicines.

 

What if there were a drug which promised to cure all diseases and provide humans with a youthful existence for 150 years, but it would take ten billion years, longer than the human race will survive on Earth, for the FDA tests on its safety and effectiveness to be completed?

 

How exactly do you know it cures all diseases and gives humans a youthful existence if you can't test it in under ten billion years?

[Marat]

While you can get some 'medicines' which have now fallen under the classification of 'foods' or 'supplements,' this is far from being the case with many drugs. Interestingly, the restrictions on patients' access to medicines are highly culture-dependent, which argues against their being rational, since the human body is the same everywhere in the world. Thus in Germany you can get without a prescription a noradrenalin ampule for injection which you could never get near to under the paternalistic healthcare restrictions of the United States. But in contrast, Austrian culture is so suffocatingly paternalistic that you practically need a letter of permission from the health ministry to get an aspirin -- even though it was one country with Germany until 1945.

 

What counts as 'knowing' that a medicine is safe and effective for use for ordinary rationality is profoundly different from what counts as knowing this for purposes of FDA approval. Dr. Denise Faustman at Massachusetts General Hospital has recently been forced to waste two years and tens of millions of dollars proving that BCG is a safe and effective drug for treating autoimmune diabetes, but BCG has been in regular use for decades in medicine for other purposes where there was no question of its safety and effectivenes.

 

The motives for the FDA (or 'foot-dragging administration' as it is called among medical researchers) deliberately blocking medical progress are well-known. Like most federal regulators, the FDA is in bed with the institutions it is supposed to regulate, and since the big pharmaceutical companies hate competition, the FDA helps them by making the testing and approval process for new drugs so prohibitively long and expensive that nothing ever emerges to challenge the existing monopoly of the drug companies.

[Narroo]

Well, the idea of the FDA is good. The regulation laws were made because people were selling snake oil as medicine, and other things that were actually harmful. The idea was to make sure medicine was effective and unharmful, so that people can reliably access real medication, instead of bad tonics.

But~ that doesn't mean the FDA is doing their job right.

[Marat]

What if the FDA only had the authority to label all medications with the results of its safety and effectiveness tests in both medically technical terminology and layman's terminology? Then the public would be put on notice about the potential risks, but they would remain free to take those risks or not as they saw fit. Perhaps the requirement for some particularly toxic medicines could be even more stringent, such as insisting on a 15-minute talk with a pharmacist before the patient could obtain the drug. For drugs for which there was not yet sufficient information to determine whether they were safe and effective or not, there would be a requirement that this lack of data be brought to the consumer's attention before the consumer could purchase them, but still, the decision would ultimately rest with the patient. This would be more consistent with America's claim to be 'the freest country on earth' than its present restrictive practices, which make many drugs less readily available in the U.S. than they are in supposedly less free countries in Europe and South America. If it is legal for Evil Knieval to ride his motorcycle over a gorge with a high risk of death just for a stunt, then it makes no sense that it is illegal to take adrenalin without a prescription because the state decides for its citizens that it is not safe enough to use without a doctor's written permission.

[CharonY]

What is the difference to how it is now? If something is not FDA approved by not being effective could, if it is not harmful, still be sold as nutrient supplement, for instance. It just may not claim to cure anything.

 

Still, it would be required to undergo stringent testing, how else are you going to be sure that it is effective/toxic? How else are you going to make a proper risk assessment?

Now, these clinical trials are essentially what is expensive, and what makes or breaks a medication.

 

Of course you could lower the requirements and lower the testing standards. This could lower prices. And as we all saw with the BP debacle letting the industry regulate themselves is always a good idea.

 

And finally, the medication decisions is usually done with a physician. Self medication for serious conditions are usually not a good idea (which does not mean that one should not inform oneself on top of consulting specialists, preferably several).

[Marat]

The FDA can and does ban supplements just as easily as it bans medicines -- e.g., vitamin B17, also known as laetrile. The Clinton administration had to pass legislation to try to slow down the ravenous attempt of the FDA to swallow up and forbid nearly the whole supplement industry, which it hates in its role as the agent of Big Pharma and the Medical Establishment, which profit most from more expensive prescription drugs. As one FDA official once quipped, "If coffee were a newly introduced substance today, you would have to get a prescription before you could have a cup of it." The worst problem is that if anyone, even someone not associated with the manufacture of a supplement, claims that it can cure disease, then the FDA can re-classify it as a drug and ban it. The FDA even banned poor old Dr. Reich's wooden 'Orgone Boxes' and put him in federal prison for selling them, even though they were totally harmless -- just because they were ineffective and curative claims had been made for them.

 

Before you dash into a burning building to save your family from death, you don't know whether your action will be safe and effective, but you have every right to make your own judgment about whether to do it or not. The same is true of countless other risks people take every day. So if you are dying of an incurable disease and you would prefer to risk death or the worsening of your condition by taking an unproven remedy to continuing on with things as they are, then that should be your own business, not the decision of a state bureaucrat. Many people who were otherwise certain to die of a presently incurable illness would quite sensibly prefer to accept a 50% chance that they would make their condition worse or more painful by taking an unproven drug that offered them a 1% chance of cure. Yet as things are now, the federal government will step in with all its might to stop them from trying to save their lives as they see fit, and unfortunately, the courts, which usually roll over and play stupid when confronted with Medical Science, almost always give into them. See the decision in Abagail Alliance v. von Eschenbach.

[DJBruce]

Before you dash into a burning building to save your family from death, you don't know whether your action will be safe and effective, but you have every right to make your own judgment about whether to do it or not.

 

This comparison really is not a valid one. When you rush into a burning building you are only endangering your own life. However, if the FDA were just to be shut down, and people allowed to make and take whatever medicine they want our entire society would be put at danger.

 

Would you honestly feel safer knowing that their is no regulation on any medication? That product that claims to cure headaches could actually be dimethyl mercury for all you know. That pill that is going to cure your cancer could be just as effective at curing cancer as a sugar pill. Yes, the approval process for a new medication takes time, but it takes time for a reason. For without proper testing it could mean it does not actually help those it is supposed to, and that it could harm many many people.

[Marat]

I think most people are quite risk-averse, so even if there were no FDA the typical citizen would not take any drugs which were not approved by traditional medical advice. Many countries don't have anything like the FDA and yet masses of people don't die from dangerous medical experimentation on themselves, just as they did not die in the U.S. prior to the existence of such regulatory bodies.

 

I would not ban the FDA, since its provision of advice to people on the risks and benefits of various treatments would still be useful. But I would deny it the legal power to prevent people from trying whatever treatments they wanted according to their own judgment. After all, it is now legal to eat high-cholesterol foods every day, intoxicate yourself with alcohol, go motorcycle racing, work in a dangerous coal mine, and smoke constantly, and all this you are allowed to do purely for your own profit or entertainment, so why shouldn't you be able to try an unproven medicine to save your life?

[CharonY]

I think you misunderstand (deliberately or not) the role of the FDA. As others already told you, they do not prevent you from taking experimental medicine. They just limit the options of companies to sell them. You want volunteer for a trial of an experimental drug? Go ahead some people actually make a living out of it. The companies are just not allowed to put something on the market without showing the risk/benefit of it. Which, incidentally, is basically what you propose.

 

Why, do you think can you freely buy homeopathic drops, which are essentially just pure water? And more to the point, if people were good in risk/benefit assessment, why do they buy it, instead of a few gallons of water?

Edited July 11, 2010 by CharonY

[Marat]

It is not easy to get into a clinical trial of an experimental treatment, but most importantly, you have no autonomous right to participate in that treatment, and you can only get the drug if someone else -- the physicians and scientists directing the experiment -- let you. That is hardly medical liberty. But if some drug or treatment doesn't happen to be enrolling subjects into an experiment when you need it, or if you are not accepted into the trial, then the FDA will exercise the full power of the federal criminal law to stop you from accessing the therapy, which is inconsistent with the American philosophy of individual liberty. Benjamin Rush, a physician and a major force in the American Revolution, actually wrote that medical liberty -- the free access by the individual to any form of treatment he wants -- is the most essential liberty of all, and Thomas Jefferson wrote statements in agreement with him. But that was before America was taken over by monied interests, and Big Pharma has no interest in letting sick people escape the plantation on which it holds them and drains their money away for expensive treatments, nor does Establishment Medicine want people being able to access drugs without paying doctors first to get a prescription.

 

If you want to take Laetrile or undergo Ardenne treatment for cancer, have an autologous stem cell transplant for your chronic and otherwise incurable disease, or have fresh cell injections for their rejuvenative effects, the 'freest country on Earth' forbids you to do this, and your only option is to become a medical refugee from America and seek the greater medical liberty available in Europe and South America to exercise your right to manage the medical treatment of your own body as you see fit.

 

If you look at the case of Abagail Alliance v. von Eschenbach you will see how mightily the FDA struggles against Americans having the right to treat their own diseases as they choose. (You can find this case online.)

[Cap'n Refsmmat]

If you look at the case of Abagail Alliance v. von Eschenbach you will see how mightily the FDA struggles against Americans having the right to treat their own diseases as they choose. (You can find this case online.)

Most experimental medications are protected by patents. Should the company developing the medication decide not to sell it to you, why should you have a right to use it?

[CharonY]

Actually the company would sell it. And here is the problem. Normally drugs undergo three to four clinical trials, based on the outcome of the pre-clinical studies. The costs are increasing massively from step to step. Now, if people were ready to buy drugs after the early, e.g. phase I trials, why should the company do the large-scale risk assessment? Keep in mind that only in phases II and III side effects, and effectiveness are tested.

In other words, it would be beneficial to swamp the market with inefficient drugs. Even if fewer people buy it, the profit margins could still be higher.

 

And with regards to the OP, while the rules may in detail be inefficient (which is to be expected considering the broad variety of issues they have to face) but they are not irrational.

[Marat]

Since there would still be many, conservative, risk-averse patients who would only want to use drugs that had completed all four phases of the FDA certification process, it would still be worthwhile for drug companies to go through the entire process, even if patients could still access these drugs earlier.

 

The irrationality of the FDA rules is that while they might be scientific they are not existentially sensible for people who are certain to die unless they try some desperate and profoundly risky experiment which might work. If somone is in the last stages of terminal cancer, and some new and unproven drug claims to be able to rescue patients even at this stage, it would be absurd for that patient not to try it, but the FDA will still often fight that patient to prevent him from getting the drug. That is not just irrational, it is downright lunatic, and totally inconsistent with American traditions of individual liberty.