Pathetic level of slavery to evidence

| 63 Comments

Andrew Weil practices “alternative” medicine, that is, medicine for which there is no evidence of efficacy. Now, according to an article in the Los Angeles Times, he recommends that evaluations of the efficacy of a treatment include “[p]atient factors – including how patients felt about the treatment, whether they can afford it and any evidence of a placebo response” (the words are those of the reporter, not Weil).

Some of Weil’s other recommendations, such as consideration of the funding source and possible unintended consequences, make sense, but “how patients felt about the treatment” is an invitation to peddle snake oil - maybe we should perform all clinical trials using red pills to get the best outcome.

Paraphrasing Weil, the article goes on to say,

Medicine has become enslaved to “evidence-based” approaches that rely on randomized, clinical trials as the only measure of whether a treatment is valuable[.]

Enslaved to evidence-based approaches? Reminds me of nothing more than William Dembski’s pathetic level of detail.

63 Comments

“Medicine has become enslaved to “evidence-based” approaches … “

Just like engineering, architecture, aviation, agriculture and seismology are ‘enslaved’ to “evidence-based” approaches. Evidence-based approaches have served these fields well–why abandon them in the case of medicine?

And, of course, the law … “your honor, I would like to offer ‘non-materialistic’ evidence in favor of my client.” “Denied.”

Not to mention businesses, emergency response organizations, and absolutely the military: a warfighter that doesn’t care about the real facts is going down in flames.

And don’t get me started on how enslaved to evidence-based approaches the judiciary is.

Although, I always like to imagine IDiots and people like Weil being consistent in their anti-evidence shtick when accused of some serious crime. Do you think that any of them will clamor for the use of “revelation” or “it looks like he’s guilty” when their own freedom is on the line?

Somehow, I think that they’ll challenge every “non-materialistic” and “non-evidence-based” claim made against them, although they might very well try to use nonsense as a defense.

Nothing new there, though. They always demand rigorous evidence from others, while they feel exempt from the demands of scientific rigor themselves. They’ll hold us to sound standards, it’s just that they have “higher ways of knowing” than their opponents.

Glen Davidson

Glen Davidson said:

And don’t get me started on how enslaved to evidence-based approaches the judiciary is.

Although, I always like to imagine IDiots and people like Weil being consistent in their anti-evidence shtick when accused of some serious crime. Do you think that any of them will clamor for the use of “revelation” or “it looks like he’s guilty” when their own freedom is on the line?

Somehow, I think that they’ll challenge every “non-materialistic” and “non-evidence-based” claim made against them, although they might very well try to use nonsense as a defense.

Nothing new there, though. They always demand rigorous evidence from others, while they feel exempt from the demands of scientific rigor themselves. They’ll hold us to sound standards, it’s just that they have “higher ways of knowing” than their opponents.

Glen Davidson

Unless they could be assured that the entire court was in their pockets, I think Intelligent Design proponents, the more cunning ones, at least, would avoid the courtroom like it was the radioactive plague.

After all, you remember how Bill Dembski wanked on and on how he hoped and prayed to get the chance to put all those Evilutionists on trial, only for him to hide under a rock when the opportunity in Dover came knocking.

I think for any practical endeavor any complaints about “materialism” are silly: “Ya’ll lost that battle a long time ago, people.”

On that same coin, I do get a little weary of overblown claims for “Science” with a capital “S” … is it really any more or less “evidence-based” than any other practical exercise? And this commitment to evidence is what exercises the critics. Again: “You’ve lost. You can complain forever but you can’t win. Get some sense and give it up.”

The people critical of “evidence-based approaches” can never answer the big question:

If you were wrong, how would you know it?

They simply assume that their internal truth detector is infallible. Gee, now where on earth do you suppose they were first introduced to that concept? So much for the “what’s the harm” arguments.

I suspect Weil would have the same problem defending his position in court that Behe had, when Behe proposed a broader definition of science. The opposing lawyer would get up, point out that sacrificing chickens on an altar could count under Weil’s definition of “effective treatment,” and that would be the end of it.

Weil is eccentric but should not be confused with dangerous quacks. In no way does he try to replace or prevent mainstream therapy.

He openly recommends mainstream medicine for serious complaints.

Even where he recommends what he calls “integrative” or “alternative” stuff, it is often just evidence-based mainstream stuff.

His diet and exercise advice is 100% mainstream.

This is a guy who recommends eating lots of fruit and vegetables and fish oil, exercising, and managing stress, and using mainstream medicine if you get sick anyway.

He may do it in a funny robe with a big bushy beard, and he may charge tens of dollars for books that peddle glorified common sense and other arguably useless but fairly harmless products, and he may have made a very silly statement here, but he really is not a harmful figure.

If anything, he weans a lot of well-meaning but confused people off of harmful woo.

One can’t help thinking of homeopathic dilutions that go far beyond the number of molecules that would have to be removed from a water solution in order to leave its “essence” imprinted in the water.

There is only one motto that fits that “science:” Nothing works.

harold said: but he really is not a harmful figure.

I disagree. Weil quote from the article:

“The individual’s belief system will affect the value of a treatment significantly and cannot be ignored.”

I think Weil’s statement is harmful to the public’s understanding of science and medicine, because it teaches the opposite of what is true. What enlightenment science has discovered is that, to a very large extent, the individual’s belief system can be ignored and will not affect the value of the treatment.

And to the extent that Weil’s statment is true, he is basically recommending doctors lie to their patients. That is really the only way to get the placebo effect to work for you - deceive the patient about what it is.

eric -

We’ll have to partly agree and partly disagree.

I think Weil’s statement is harmful to the public’s understanding of science and medicine, because it teaches the opposite of what is true. What enlightenment science has discovered is that, to a very large extent, the individual’s belief system can be ignored and will not affect the value of the treatment.

I agree with you but perceive a difference between having made this statement, and being harmful. I don’t agree with everything Weil says but I do agree with his preventative and therapeutic recommendations - in fact, I have to, because for all his talk about “belief systems”, his actual medical recommendations are supported by the literature.

If a patient goes to Weil, they will receive a recommendation that they eat what is strongly supported as a healthy diet, and undertake healthy exercise. If they are ill, he will recommend standard therapy. Eccentricity in personal beliefs is not grounds to label a physician harmful to the public.

If there was any evidence that Weil recommended not giving appropriate therapy to patients with certain “belief systems”, I would massively condemn him, but he does not cross that line (or even come close to doing so).

And to the extent that Weil’s statment is true, he is basically recommending doctors lie to their patients. That is really the only way to get the placebo effect to work for you - deceive the patient about what it is.

If Weil recommended knowingly lying to patients, that would be a very serious breach of ethics. I have no reason to think he does that. The only medically indicated use of placebos is in controlled studies, and the patients know that they may be getting the placebo in those studies, and understand the reason for the randomization.

Stanton said:

Glen Davidson said:

And don’t get me started on how enslaved to evidence-based approaches the judiciary is.

Although, I always like to imagine IDiots and people like Weil being consistent in their anti-evidence shtick when accused of some serious crime. Do you think that any of them will clamor for the use of “revelation” or “it looks like he’s guilty” when their own freedom is on the line?

Somehow, I think that they’ll challenge every “non-materialistic” and “non-evidence-based” claim made against them, although they might very well try to use nonsense as a defense.

Nothing new there, though. They always demand rigorous evidence from others, while they feel exempt from the demands of scientific rigor themselves. They’ll hold us to sound standards, it’s just that they have “higher ways of knowing” than their opponents.

Glen Davidson

Unless they could be assured that the entire court was in their pockets, I think Intelligent Design proponents, the more cunning ones, at least, would avoid the courtroom like it was the radioactive plague.

After all, you remember how Bill Dembski wanked on and on how he hoped and prayed to get the chance to put all those Evilutionists on trial, only for him to hide under a rock when the opportunity in Dover came knocking.

And still pocketed what $20K is consulting fees?

Dembski will swallow his pride for money.

harold said:

Weil is eccentric but should not be confused with dangerous quacks. In no way does he try to replace or prevent mainstream therapy.

He openly recommends mainstream medicine for serious complaints.

Even where he recommends what he calls “integrative” or “alternative” stuff, it is often just evidence-based mainstream stuff.

His diet and exercise advice is 100% mainstream.

This is a guy who recommends eating lots of fruit and vegetables and fish oil, exercising, and managing stress, and using mainstream medicine if you get sick anyway.

He may do it in a funny robe with a big bushy beard, and he may charge tens of dollars for books that peddle glorified common sense and other arguably useless but fairly harmless products, and he may have made a very silly statement here, but he really is not a harmful figure.

If anything, he weans a lot of well-meaning but confused people off of harmful woo.

Isn’t this the sap who is responsible for a drop in the number of kids getting immunized because of a bogus claim that compounds in vaccinations caused autism?

He’s a weapon of mass destruction.

Stuart Weinstein said:

harold said:

Weil is eccentric but should not be confused with dangerous quacks. In no way does he try to replace or prevent mainstream therapy.

He openly recommends mainstream medicine for serious complaints.

Even where he recommends what he calls “integrative” or “alternative” stuff, it is often just evidence-based mainstream stuff.

His diet and exercise advice is 100% mainstream.

This is a guy who recommends eating lots of fruit and vegetables and fish oil, exercising, and managing stress, and using mainstream medicine if you get sick anyway.

He may do it in a funny robe with a big bushy beard, and he may charge tens of dollars for books that peddle glorified common sense and other arguably useless but fairly harmless products, and he may have made a very silly statement here, but he really is not a harmful figure.

If anything, he weans a lot of well-meaning but confused people off of harmful woo.

Isn’t this the sap who is responsible for a drop in the number of kids getting immunized because of a bogus claim that compounds in vaccinations caused autism?

He’s a weapon of mass destruction.

Oopps spoke to soon. Wrong dude. Apologies to Dr. Weil

Stuart

Stuart Weinstein -

I believe you’re thinking of this repugnant slimeball.

Weil is a horrifying charlatan pretending that patient beliefs change the outcome of cancer treatments:

Finally, two of the more pernicious proposals in Weil’s paper are related. First, he repeats his explicit argument for favoring placebo medicine:

Given the history and philosophical preferences of allopathic medicine, it should come as no surprise that the factors defining the healing response become minimized or ignored in current practice. This failing must be remedied, as these factors account for a huge component of how humans heal and recover. The healing relationship has taken a much larger role in IM as practitioners in nearly all CAM modalities place a much higher emphasis on it. The importance of these issues can be demonstrated most clearly in psychiatry research, where the placebo response plays a huge role accounting for as much as 40%–90%+ of the total response.26

Significant placebo and expectancy responses inhabit other areas of medical practice, such as dealing with pain and even life expectancy in patients with terminal cancer. Clearly, patient factors must compose a significant part of all treatment selection processes. Ideally, every treatment should be matched to the individual’s belief system to reach the highest level of response possible.

Aside from stating, “CAM is placebo medicine, and we should use placebo medicine when it fits in with patient beliefs,” a more explicit admission that what is being proposed in this opinion piece is placebo medicine I cannot imagine. Worse, it’s based on misinformation, the most egregious of which is Weil’s claim that placebo and expectancy effects can increase life expectancy in patients with terminal cancer. Would that it were true, but unfortunately it’s not, either in late stage cancer or early stage cancer. Nor is there any evidence that it has an effect on recurrence.

Weil exposed

Not surprising, given his idiotic attack upon “‘evidence-based’ approaches.”

Glen Davidson

I wonder how Dr. Weil knows when to put fuel in his car? Does he do it when it feels like the right time, or is he enslaved to an evidence-based approach that says looking at the fuel gauge is the only way to tell if his car needs gas?

harold | May 16, 2011 2:34 PM | Reply | Edit

Weil is eccentric but should not be confused with dangerous quacks. In no way does he try to replace or prevent mainstream therapy.

He may do it in a funny robe with a big bushy beard, and he may charge tens of dollars for books that peddle glorified common sense and other arguably useless but fairly harmless products, and he may have made a very silly statement here, but he really is not a harmful figure.

Encouraging people to spend money out of their health care budget on useless products is harmful.

…and you can’t wean people off of more harmful kinds of woo by telling them that woo works.

This topic is right up my alley as I teach EBM to medical students and GP registrars.

On reading the article in LA Times, I think it is fair to say that the only objectionable statements were made by the reporter rather than quotes from Weil. For instance:

“Medicine has become enslaved to ‘evidence-based’ approaches that rely on randomized, clinical trials as the only measure of whether a treatment is valuable, Weil and his co-authors wrote.”

The original paper (Scott Shannon, Andrew Weil, Bonnie J. Kaplan. Alternative and Complementary Therapies. April 2011, 17(2): 84-91. doi:10.1089/act.2011.17210 – as the reporter couldn’t be bothered to provide a proper reference) says nothing of the sort. It does make some (IMO very dubious) claims about over-emphasis of randomised control trials, but the paper recommends a grading scale of evidence for effectiveness in which the top 3 levels are based on RCTs.

The LA Times article also fails to recognise that the standard medical tiers of evidence already include non-RCT studies, such as case-control and cohort studies. The paper that first linked tobacco to lung cancer was a famous 1950 case-control study by Doll and Hill (they thought that tarmac or motor vehicle exhaust fumes caused lung cancer and discovered to their surprise that the only strong association was with cigarette smoking) and supported by the 1954 British Doctors Study, a cohort study. No RCTs in sight.

There are still many statements in the paper that I would dispute, but they are not on the same scale as the LA Times article suggests. In fact, the article pretty much misses the point of the paper, which calls for a different approach to weighing evidence, while the reporter has reconstructed it as a “mainstream evidence is bunkum” narrative.

The first post doctoral proposal I wrote was for a study of the ethnopharmacology of coca use in Peru. That was in 1976, and I became acquainted with Andy Weil, and some others like Tim Plowman at Harvard, who were of the opinion that the “war on drugs” was really a war on ourselves, and particularly a war on minorities. The DEA was then, as now, merely an armed branch of the political and religious far-right. DEA “enforcement” was then just moving into armed foreign operations in South America targeting “communists” whether or not they were involved with drug production.

The DEA became particularly enraged with Weil when he pointed out that the “war” was already lost when the quality illegal drugs like heroin went up, and the street price went down. Andy also observed anyone in the USA who had the least interest in taking heroin, cocaine, or any other drug could find a ready supply anywhere! As an MD, he was particularly vulnerable to DEA pressure. He would not retract his (obviously true) statements that drug use, drug abuse, and drug addiction were not the same thing, and that this is a medical question that cannot be “treated” with guns and prisons. (That was true 35 years ago, and it is true today). The DEA retaliation was to deny Weil a schedule II drug license so that he was effectively prevented from practicing medicine.

My opinion was that this led Andy to two rather profound questions; “What is medical practice beyond giving people shots and pills?” and “What can I do to earn a living?”

The answer to both questions was that he teach people that prevention is better than any medicine by writing books, giving talks and to sell this with a degree of flamboyance.

Some of the specific misunderstandings about medical practice, “evidence based” or not, that I have seen in the posts so far will wait until after my dinner.

Weil and co-authors illustrate the simple point that medical research, like science generally, is always an interested endeavor. Always conducted in social and cultural context (Laura Nader 1996 Naked Science). I have intimate experience with a close family member diagnosed with Multiple Sclerosis. The treatments for MS (Rebif and Copaxone) have proven largely ineffective in controlling both the occurrence of her lesions and the symptomology of the disease. We have pursued information on other treatments, but very little is establishment-sanctified. Big Pharma is the interested actor here, which pays for and conducts almost all the cited research on MS treatments. There are few “scientific” studies, for instance, on the effect of diet on MS, despite strong anecdotal evidence that diet can play a significant role in both lesion count and symptomology. See Terry Wahls’ work and her book Minding my Mitochondria if you are interested. Why would a pharmaceutical company conduct or fund a study on the effects of diet on MS when none of their products would be used, and in which a treatment could be found that would actually subvert their own research-based products? They wouldn’t, and no one would expect them to. However, as patients, our family has grown increasingly frustrated with seemingly narrow self-interested approaches to the MS research problem by a mainstream science that cannot provide answers to easy and basic questions. Our specialist does not dismiss the efficacy of diet as treatment for MS. But when we ask him about it he says he doesn’t know much about it. There is little accepted clinical research on diet and MS, and little of what there is makes it to the literature with which he is familiar. I agree with Lawson above that the LATimes reporter recontextualizes the article in a standard narrative format and misses the more interesting point of the article on an alternative system of weighing evidence of efficacy that includes funding, accessibility of the treatment, and other patient factors. Medicine and the scientific research upon which it is based is today too much a closed paradigmatic corporate loop in the worst Kuhnean sense. A critique of science and medicine as socially interested and invested in certain kinds of (pharmacological) results is one part of a healthy understanding of contemporary medicine and science.

rrich -

Weil and co-authors illustrate the simple point that medical research, like science generally, is always an interested endeavor. Always conducted in social and cultural context (Laura Nader 1996 Naked Science).

Okay, that’s a reasonable statement.

I have intimate experience with a close family member diagnosed with Multiple Sclerosis.

Best wishes. That’s a tough disease to deal with.

The treatments for MS (Rebif and Copaxone) have proven largely ineffective in controlling both the occurrence of her lesions and the symptomology of the disease.

That sucks. Modern therapy has benefited many patients, but there’s still a lot to learn.

We have pursued information on other treatments, but very little is establishment-sanctified. Big Pharma is the interested actor here, which pays for and conducts almost all the cited research on MS treatments. There are few “scientific” studies, for instance, on the effect of diet on MS, despite strong anecdotal evidence that diet can play a significant role in both lesion count and symptomology.

I don’t follow your logic. You seem to think that because pharmaceutical companies do research on their own products, no-one else can ever do any other type of research. That doesn’t make sense to me.

See Terry Wahls’ work and her book Minding my Mitochondria if you are interested. Why would a pharmaceutical company conduct or fund a study on the effects of diet on MS when none of their products would be used, and in which a treatment could be found that would actually subvert their own research-based products? They wouldn’t, and no one would expect them to.

1) Terry Wahls certainly isn’t being censored or suppressed, or prevented from seeking funding for a clinical study. She’s on faculty at a highly prestigious medical school.

2) Her dietary advice is probably beneficial for overall health, for most people, at least based on current understanding of diet and health. Whether it does any good for MS I don’t know. It might. MS is a notoriously waning and waxing disease, as I’m sure you already know. I’m sure you can see how that could lead to false attributions of improvement to something random, when they were just the natural course of the disease. The next step is a controlled trial. Of course, there can’t be a placebo in a trial that tests a diet, and if whoever is testing the diet is convinced that it works, they might feel that it was unethical to even have a control group. Within those limitations, though, I don’t see any rationale for failing to do some kind of controlled, organized scientific study.

However, as patients, our family has grown increasingly frustrated with seemingly narrow self-interested approaches to the MS research problem by a mainstream science that cannot provide answers to easy and basic questions.

I don’t think of MS or any other autoimmune disease as posing “easy” or “basic” questions. But if you think the questions are so easy and basic, why don’t you just answer them yourself?

Meanwhile, Wahl does make her dietary recommendations very explicit, and although I have no idea whether they help MS or not, they’re certainly unlikely to be harmful. So if you think she’s right, why don’t you just do what she recommends? My understanding, and please correct me if I’m wrong, is that she doesn’t try to replace conventional therapy, but does suggest that a dietary strategy, one that is compatible with what we currently believe to be a healthy diet, can help.

Our specialist does not dismiss the efficacy of diet as treatment for MS. But when we ask him about it he says he doesn’t know much about it. There is little accepted clinical research on diet and MS, and little of what there is makes it to the literature with which he is familiar.

That’s all true, but you don’t need a prescription at the grocery store. If you think that a nutrient rich diet will help, eat one. And tell your specialist when you start, so that if unusual benefit is noted, he can report the anecdote. Sometimes that’s how research gets started.

I agree with Lawson above that the LATimes reporter recontextualizes the article in a standard narrative format and misses the more interesting point of the article on an alternative system of weighing evidence of efficacy that includes funding, accessibility of the treatment, and other patient factors.

I sort of agree here, except that I believe that rich societies like the US should assure that every man, woman and child has access to the indicated treatment for every disease.

Medicine and the scientific research upon which it is based is today too much a closed paradigmatic corporate loop in the worst Kuhnean sense.

Here I disagree. There is plenty to criticize, but buzzwords and conspiracy theories don’t add anything.

A critique of science and medicine as socially interested and invested in certain kinds of (pharmacological) results is one part of a healthy understanding of contemporary medicine and science.

Yes it is indeed.

Yet a persistent claim of harmful if not predatory quacks (and I don’t mean Weil, nor Wahl, who I believe is at worst sincere in a mistaken belief that a diet that is overall beneficial will help MS, and may even be partly correct), is that some kind of perfectly healthy diet or lifestyle will perfectly prevent, or, in the case of real criminal predators, cure, clinical disease. Often, insultingly simplified stereotypes of hunter gatherer societies or others are created in support of such claims. To which I reply -

1) Yes, it is true that “western medicine” has historically focused on “treatment”, rather than prevention. There is a reason for this - at any given time, there are millions of people who need the treatment. Furthermore, much of the prevention is extremely well known, yet there is nothing the medical profession can do to force people to prevent.

2) Fortunately, there are people who do try to lead healthy lifestyles, although in my view, many of them go overboard into obsession and well past the point of diminishing returns. Plenty of them get sick anyway. Less often, overall, than of the people who don’t take care of themselves, but it’s a question of statistical advantage, and there are many other factors.

3) Everyone who claims to prevent or cure all diseases, or even a particular disease, is free to do everything in their power to objectively test their claims. Vast numbers of people seek to make such claims, yet evade such testing.

Good luck with the MS. I hope I don’t come across as rude or hostile, that’s not my intent.

Well, Andy’s employment problem was resolved at the U. of Arizona. The question of how do we measure patient outcome visa-vis drug trials was not so easily resolved.

Let me give a personal experience: some years ago I was prescribed a medicine by a urologist to prevent vomiting during treatment for a kidney disease. The drug had passed all kinds of trials with flying colors- except it did not prevent me from vomiting. And, when I did, it was a particularly nasty tasting blue vomit, much worse than the ordinary unmedicated stuff.

This is the difference between an “evidence-based” double-blind drug trial, and a clinical result.

rrich said:

Medicine and the scientific research upon which it is based is today too much a closed paradigmatic corporate loop in the worst Kuhnean sense. A critique of science and medicine as socially interested and invested in certain kinds of (pharmacological) results is one part of a healthy understanding of contemporary medicine and science.

There are other complicating factors. Many similar diseases - like Gaucher’s disease or Pompe’s disease, or ALS, or Friedreich’s Ataxia – are often referred to as “orphan diseases” because the research on them not only involves relatively small populations, but the development of drugs to slow or stop the progress of these diseases is agonizingly difficult and expensive.

Clinical trials with drugs under development involve extremely complicated protocols of bringing people from around the globe to a relatively few locations where they can be given treatments and evaluated on the effects of those treatments. It means setting up teams of investigators, evaluators, and coordinators who make sure research protocols are followed and patients are protected.

Companies such as Genzyme take a huge financial risk in developing drugs, become a sole source (until some other company steps in and takes similar risks), and then has a disastrous contamination problem that shuts them down for months while patients desperately try to hang on.

Understanding diseases is not accomplished by simply scatter-shot approaches and anecdotal reports and testimonials. The best these can do is in providing leads; but more often than not, they send people off on wild goose chases.

It is an extremely complex process of not only identifying the disease, but sorting through literally hundreds of complicating factors that vary greatly from individual to individual.

I get first hand reports of this from a close relative who is a clinical research coordinator in the clinical research trials on a couple of these diseases.

Mike Elzinga, you support you promotion of “factual,” “empirical,” “scientific” medicine with “first hand reports of this from a close relative?”

This is not the “factual,”empirical,” or “scientific” evidence that the Holy Church of the Double-Blind accepts. These “first hand reports of this from a close relative” are exactly the anecdotal data that purely “scientific” medicine rejects, exactly like to many “scientific” doctors reject their patient’s actual clinical experiences. (You cannot die, I used medicine to cure you!) Are we to practice medicine as physicians, or statistics undergrads?

Damn! I hate typos, even with the preview they slip by. Then seconds later, they are glaring, mocking me.

you(r)

to(o)

Gary Hurd said:

Mike Elzinga, you support you promotion of “factual,” “empirical,” “scientific” medicine with “first hand reports of this from a close relative?”

This is not the “factual,”empirical,” or “scientific” evidence that the Holy Church of the Double-Blind accepts. These “first hand reports of this from a close relative” are exactly the anecdotal data that purely “scientific” medicine rejects, exactly like to many “scientific” doctors reject their patient’s actual clinical experiences. (You cannot die, I used medicine to cure you!) Are we to practice medicine as physicians, or statistics undergrads?

It’s not just the relative. I’ve personally met many of the patients; many of whom are college educators, lawyers, engineers, and researchers themselves. I have met them on several occasions and in two different cities.

They are a well-organized and well-informed community of people who have been in these studies for a number of cycles now. They know the principle investigators, the companies and company executives; and they have suffered through the problems of depending on a single company that ran into production problems and bungled its relationships with the investigators, coordinators, and patients.

I’ve followed some of the issues in the press as well as from other inside sources. And the patients have been very open about their experiences, frustrations, and personal feelings.

The studies are all double blind and have to meet federal regulatory standards and frequent reviews; and the patients understand that. We have had discussions about protocols and some of the problems that people in the companies have in understanding and building such protocols. It is often a mess because of the shortage of qualified people who understand the processes of setting up research protocols.

That doesn’t mean I or the patients sympathize with the companies and their profit motives. Nevertheless, given the nature of their diseases, most patients recognize the difficulties and risks that the companies take when plunging into the development of drugs for orphan diseases. These patients also work with the research coordinators and investigators to encourage other companies to get involved.

It has been only recently that the much larger companies that have huge research budgets have gotten into the field for these diseases. Until about 4 or 5 years ago, only the smaller start-ups with promising results have been working on these and applying for clinical trials. More companies are now getting into the act.

So what I see in this picture is not a one-sided picture of companies and doctors screwing patients for profit. It is messy, involving the screw-ups of shipping companies who have nothing to do with the research but who misrepresent their abilities to get vital, perishable test results, supplies, and medications to the proper locations on time.

It involves insurance companies with bureaucrats and drones who can’t get paperwork done on time or who balk at insuring patients who get into these kinds of studies. It involves personnel turnover, changes in the laws, inexperience and incompetence, and overworked staff in many organizations that have to work together, and who all have other things going on simultaneously. It involves inefficient record keeping and the lack of interlinked computer systems and electronic data records.

The cartoon Dilbert could apply to some of what goes on; but the process of trying to keep all this mess functioning is pretty much like what we find in many companies, universities, legislative bodies, churches, military organizations, and governments.

It’s people attempting to do too much with too little, and trying to do it in the face of constantly changing rules and regulations and budget problems. But from what I can tell, the people directly involved with the patients really do care.

One of the most inspiring things I learned from the patients is that it really helps to be involved in a network of other patients with the same disease. That way they keep each other informed and become active in not only keeping the researchers, coordinators, federal monitors, and companies on their toes, but who also make sure other patients get new information as soon as it becomes available.

And this is also part of a coordinator’s job; to not only refine protocols and coordinate with the government regulators and monitors, but to keep in close touch with the patients and see that their individual needs are met.

As for the patients, that relative I mentioned also makes sure that any new people who enter studies are put in contact with those patient organizations. It helps to keep them informed and brings them into a community of others who have also lived with the disease.

And that is generally a recommendation for anyone who suffers from any form of disease.

Harold, I think you do a great job of responding to to rrich. I’d like to add a little something though:

Harold said:
1) Yes, it is true that “western medicine” has historically focused on “treatment”, rather than prevention. There is a reason for this - at any given time, there are millions of people who need the treatment. Furthermore, much of the prevention is extremely well known, yet there is nothing the medical profession can do to force people to prevent.

In fact, vaccination is arguably an excellent example of a highly successful preventative medical programme. In many cases, children will only be accepted into an education system if they have an up-to-date vaccination schedule. Whether it’s the ‘medical profession’ who forces participation in this is arguable, but I for one think it’s a very good thingTM.

This is a guy who recommends eating lots of fruit and vegetables and fish oil, exercising, and managing stress, and using mainstream medicine if you get sick anywa

Yes, and so does my doctor, but she doesn’t peddle nonsense on top of it.

eric said:

Unfortunately, submission of results is entirely voluntary and thus open to abuse. A company can conduct 10 trials, get positive results in only 1 of them, and then submit only that trial’s results to the FDA - making the drug appear far more effecacious than it is.

This is not true. Sponsors are required to submit the results of all clinical trials when they seek approval for a new drug.

Personally, I’d like to see the FDA institute two rules: (1) the FDA will only consider results of trials registered with them before the trial is conducted, and (2) the FDA will count non-submission of a registered trial’s results (after some reasonable time to complete it) as a negative result.

The first part of your proposal has been in effect for quite a few years already. Any clinical trial (other than Phase I trials) that a sponsor intends to use in support of drug approval has to be registered in advance at clinicaltrials.gov. The results of unregistered trials cannot be used to support drug approval (but they can still be used to deny approval - e.g. if they contradict other positive trials or reveal safety issues).

Relating this back to Weil’s comments, while RCT’s probably always will be (and should be) the gold standard, there is no reason why companies couldn’t submit other types of studies if an RCT is impossible.

This happens in rare cases, but in general, the company would have to convince FDA in advance that blinded RCTs weren’t possible, and that they could still establish efficacy in some other way.

qetzal said:

The first part of your proposal has been in effect for quite a few years already. Any clinical trial (other than Phase I trials) that a sponsor intends to use in support of drug approval has to be registered in advance at clinicaltrials.gov. The results of unregistered trials cannot be used to support drug approval (but they can still be used to deny approval - e.g. if they contradict other positive trials or reveal safety issues).

I’m glad somebody said that. Ben Goldacre raised a fuss about the supposed failure to register trials in his otherwise creditable book of essays on bogus medicine.

I think I’d be more comfortable going to a vet that to Mr Weil.

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This page contains a single entry by Matt Young published on May 16, 2011 12:38 PM.

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