During the most recent kerfuffle about whether or not Evidence-Based Medicine can legitimately claim to be science-based medicine, it became clear to me that a whole, new round of discussion and documentation is necessary. This is frustrating because I’ve already done it several times, most recently less than a year ago. Moreover, I’ve provided a table of links to the whole series at the bottom of each post…Never mind, here goes, and I hope this will be the last time it is necessary because I’ll try to make this the “go to” series of posts for any future such confusions.  

The points made in this series, most of which link to posts in which I originally made them, are in response to arguments from statistician Steve Simon, whose essay, Is there something better than Evidence Based Medicine out there?, was the topic of Dr. Gorski’s rebuttal on Monday of this week, and also from several of the comments following that rebuttal. Mr. Simon has since revised his original essay to an extent, which I’ll take into account. I’ll frame this as a series of assertions by those who doubt that EBM is deficient in the ways that we at SBM have argued, followed in each case by my response.

First, a disclaimer: I don’t mean to gang up on Mr. Simon personally; others hold opinions similar to his, but his essay just happens to be a convenient starting point for this discussion. FWIW, prior to this week I perused a bit of his blog, after having read one of his comments here, and found it to be well written and informative.  

 What’s in a Name?

 One of Mr. Simon’s objections, in his revision, is this:

What is SBM? Here’s a definition found on the opening entry in the SBM blog:

“the use of the best scientific evidence available, in the light of our cumulative scientific knowledge from all relevant disciplines, in evaluating health claims, practices, and products.” http://www.sciencebasedmedicine.org/?p=1

But how does this differ from David Sackett’s definition of EBM?

“the conscientious, explicit, and judicious use of current best evidence in making decisions about the care of individual patients.” http://www.bmj.com/content/312/7023/71.full

The only substantial difference I see is the adjective “scientific” that appears twice in the definition of SBM. The claim on the SBM blog is that EBM ignores scientific plausibility. Actually, ignores is too strong a word.

“EBM ‘levels of evidence’ hierarchy renders each entry sufficient to trump those below it. Thus a ‘positive’ clinical trial is given more weight than ‘physiology, bench research or “first principles”,’ even when the latter definitively refute the claim.”  http://www.sciencebasedmedicine.org/?p=42

 (I agree that “ignore” is too strong a word, but I didn’t actually write it that way, as Dr. Gorski pointed out and as I think Mr. Simon was acknowledging above.)

A difference between Sackett’s definition and ours is that by “current best evidence” Sackett means the results of RCTs. I realize that this assertion requires documentation, which will come below. A related issue is the definition of “science.” In common use the word has at least three, distinct meanings: 1. The scientific pursuit, including the collective institutions and individuals who “do” science; 2. The scientific method; 3. The body of knowledge that has emerged from that pursuit and method (I’ve called this “established knowledge”; Dr. Gorski has called it “settled science”).

I will argue that when EBM practitioners use the word “science,” they are overwhelmingly referring to a small subset of the second definition: RCTs conceived and interpreted by frequentist statistics. We at SBM use “science” to mean both definitions 2 and 3, as the phrase “cumulative scientific knowledge from all relevant disciplines” should make clear. That is the important distinction between SBM and EBM. “Settled science” refutes many highly implausible medical claims—that’s why they can be judged highly implausible. EBM, as we’ve shown and will show again here, mostly fails to acknowledge this fact.

Finally, Mr. Simon has misinterpreted our goal at SBM:

But if someone wants to point out that EBM needs work, I’m fine with that. I dislike that they think that EBM needs to be replaced with something better.

You see EBM as being wrong often enough that you see value in creating a new label, SBM. I see SBM as being that portion of EBM that is being done thoughtfully and carefully, and don’t see the need for a new label.

I generally bristle when people want to create a new and improved version of EBM and then give it a new label.

I am as harshly critical of the hierarchy of evidence as anyone. I see this as something that will self-correct over time, and I see people within EBM working both formally and informally to replace the rigid hierarchy with something that places each research study in context. I’m staying with EBM because I believe that people who practice EBM thoughtfully do consider mechanisms carefully. That includes the Cochrane Collaboration.

Mr. Simon, we agree! Yes, we are pointing out that EBM needs work. Yes, SBM is that (tiny) portion of EBM that is being done thoughtfully and carefully, and if it were mainly done that way there would be no need to call attention to the point. Our goal is not to change the name of EBM (“give it a new label”). Our goal is to convince EBM to live up to its current name. Yes, it may self-correct over time, but we are trying to shorten that time. Bad things have unnecessarily happened, in part due to EBM’s scientific blind spot: As currently practiced, it doesn’t rationally consider all the evidence. We don’t see much evidence that people at the highest levels of EBM, eg, Sackett’s Center for EBM or Cochrane, are “working both formally and informally to replace the rigid hierarchy with something that places each research study in context.”

We chose to call our blog “science-based medicine” only because the term “evidence-based medicine” had already been taken, and we needed to distinguish ourselves from the inaccurate use of the word “evidence” in “EBM.” I’ve written about this before, and have made the point utterly clear:

These are the reasons that we call our blog “Science-Based Medicine.” It is not that we are opposed to EBM, nor is it that we believe EBM and SBM to be mutually exclusive. On the contrary: EBM is currently a subset of SBM, because EBM by itself is incomplete. We eagerly await the time that EBM considers all the evidence and will have finally earned its name. When that happens, the two terms will be interchangeable.

Plausibility Misinterpreted

Mr. Simon’s interpretation of our view of plausibility, like that of many others, is wrong:

I would argue further that it is a form of methodolatry to insist on a plausible scientific mechanism as a pre-requisite for ANY research for a medical intervention. It should be a strong consideration, but we need to remember that many medical discoveries preceded the identification of a plausible scientific mechanism.

I think, from his revision, that Mr. Simon understood Dr. Gorski’s explanation of why this was wrong, but I’m not certain. The misrepresentation of scientific plausibility is an issue that I’ve faced for years, as explained previously here:

Plausibility ? Knowing the Mechanism

Let’s first dispense with a simple misunderstanding: We, by which I mean We Supreme Arbiters of Plausibility (We SAPs) here at SBM, do not require knowing the mechanism of some putative effect in order to deem it plausible. This seems so obvious that it ought not be necessary to repeat it over and over again, and yet the topic can’t be broached without some nebbishy South Park do-gooder chanting a litany of “just because you don’t know how it works doesn’t mean it can’t work,” as if that were a compelling or even relevant rebuttal. Let’s get this straight once and for all: IT ISN’T.

Steve Novella explained why at the Yale conference and again here. We talked about it at TAM7 last summer. For a particularly annoying example, read the three paragraphs beginning with “Mr. Gagnier’s understanding of biological plausibility” here.

OK, I’ll admit that I’m beginning to learn something from such frustration. Perhaps we’ve not been so good at explaining what we mean by plausibility. The point is not that we don’t know a particular mechanism for homeopathy, for example; the point is that any proposed mechanism would necessarily violate scientific principles that rest on far more solid ground than any number of equivocal, bias-and-error-prone clinical trials could hope to overturn. The same is true for “energy medicine” and for claims based on non-existent anatomical structures (iridology, reflexology, auricular acupuncture, meridians, chiropractic “subluxations”), non-existent physiologic functions (“craniosacral rhythms“), or non-existent anatomic-physiologic relations (“neurocranial restructuring,” “detoxification” with coffee enemas, dissolving tumors with orally administered pancreatic enzymes). The spectrum of implausible health claims euphemistically dubbed “CAM” is full of such nonsense.

Reader daedalus2u proposed a useful way to clarify the point:

I think the idea of prior plausibility should actually be reframed into one of a lack of prior implausibility. It isn’t that one should have reasons to positively think that something is plausible before testing it, but rather that one should not be able to come up with reasons (actually data) why it is fatally implausible.

Some of what We deem implausible will not be fatally so, of course. Implausibility can be based not only on established physical and biological knowledge, but also on studies, as is the case for sticking needles into people, injecting them with chelating agents, or claiming that autism is caused by childhood immunizations.

EBM, Basic Science, and RCTs

Steve Simon wrote, “I have not seen any serious evidence of EBM relying exclusively on RCTs. That’s certainly not what David Sackett was proposing in the 1996 BMJ editorial…” And: “No thoughtful practitioner of EBM, to my knowledge, has suggested that EBM ignore scientific mechanisms.”

Want serious evidence? Consider these quotations from Cochrane reviews, originally posted here:

In view of the absence of evidence it is not possible to comment on the use of homeopathy in treating dementia.

There is not enough evidence to reliably assess the possible role of homeopathy in asthma. As well as randomised trials, there is a need for observational data to document the different methods of homeopathic prescribing and how patients respond.

There is currently little evidence for the efficacy of homeopathy for the treatment of ADHD. Development of optimal treatment protocols is recommended prior to further randomised controlled trials being undertaken.

Though promising, the data were not strong enough to make a general recommendation to use Oscillococcinum for first-line treatment of influenza and influenza-like syndromes. Further research is warranted but the required sample sizes are large.

Yes, EBM undervalues basic science and overvalues RCTs when the former is sufficient to reject a claim. EBM also undervalues experimental evidence other than RCTs when the former is sufficient to reject a claim, as will be discussed. Here is how a truly evidence-based review might conclude a discussion of homeopathy for dementia:

The probability that homeopathy is specifically therapeutic for dementia is, for all practical purposes, zero.

The following is from my first post on the topic, in which I reviewed the overwhelming evidence—from basic science and pre-clinical research—that homeopathic ‘remedies’ have no, specific therapeutic actions, and wondered why the most esteemed exponents of EBM have written that such treatments are “promising” and that “further randomized trials are needed.” I included the Center for Evidence-based Medicine’s formal “Levels of Evidence” scheme (not copied here), the pertinent quotation from Sackett’s 1996 editorial, my opinion that this failure of EBM was initially unintended, how Sackett et al eventually did address “CAM,” and the Cochrane abstracts quoted above:

It wasn’t meant to be like this. When I first discussed with my fellow bloggers the curious absence of established knowledge in the EBM “levels of evidence” hierarchy, at least one insisted that this could not be true, and in a sense he was correct. David Sackett and other innovators of EBM do include basic science in their discussions, but they recommend invoking it only when there are no clinical trials to consider:

Evidence based medicine is not restricted to randomised trials and meta-analyses. It involves tracking down the best external evidence with which to answer our clinical questions…And sometimes the evidence we need will come from the basic sciences such as genetics or immunology. It is when asking questions about therapy that we should try to avoid the non-experimental approaches, since these routinely lead to false positive conclusions about efficacy. Because the randomised trial, and especially the systematic review of several randomised trials, is so much more likely to inform us and so much less likely to mislead us, it has become the “gold standard” for judging whether a treatment does more good than harm.

That statement is consistent with EBM’s formal relegation of established knowledge to “level 5,” as seen in the Figure. I am not a historian of EBM and don’t care to be, but I suspect that the explanation for this choice is that “they never saw ‘CAM’ coming.” In other words, it probably didn’t occur to Sackett and other EBM pioneers that anyone would consider performing clinical trials of methods that couldn’t pass the muster of scientific plausibility. Their primary concern was to emphasize the insufficiency of basic science evidence in determining the safety and effectiveness of new treatments. In that they were quite correct, but trials of “CAM” have since reminded us that although established knowledge may be an insufficient basis for accepting a treatment claim, it is still a necessary one.

Take note: Sackett wrote, “we should try to avoid the non-experimental approaches, since these routinely lead to false positive conclusions about efficacy.” My point is that pre-RCT evidence does not routinely (if ever) lead to false negative conclusions. In that passage, moreover, Sackett seems to suggest that the only alternative to a “non-experimental approach” is an RCT; yet there are often other types of experiments that can definitively refute treatment claims, as will be discussed. Eventually Sackett et al did catch wind of “CAM,” but they got it exactly wrong:

Lacking that perspective, Sackett’s Center for Evidence-Based Medicine promulgates an “Introduction to evidence-based complementary medicine” by “CAM” researcher Andrew Vickers. There is not a mention of established knowledge in it, although there are references to several claims, including homeopathy, that are refuted by things that we already know. Vickers is also on the advisory board of the Cochrane CAM Field, along with Wayne Jonas and several other “CAM” enthusiasts.

In another post I cited the 2006 Cochrane Review of Laetrile:

A 2006 Cochrane Review of Laetrile for cancer would, if its recommendations were realized, stand the rationale for RCTs on its head:

The most informative way to understand whether Laetrile is of any use in the treatment of cancer, is to review clinical trials and scientific publications. Unfortunately no studies were found that met the inclusion criteria for this review.

Authors’ conclusions

The claim that Laetrile has beneficial effects for cancer patients is not supported by data from controlled clinical trials. This systematic review has clearly identified the need for randomised or controlled clinical trials assessing the effectiveness of Laetrile or amygdalin for cancer treatment.

Why does this stand the rationale for RCTs on its head? A definitive case series led by the Mayo Clinic in the early 1980s had overwhelmingly demonstrated, to the satisfaction of all reasonable physicians and biomedical scientists, that not only were the therapeutic claims for Laetrile baseless, but that the substance is dangerous. The subjects did so poorly that there would have been no room for a meaningful advantage in outcome with active treatment compared to placebo or standard treatment… The Mayo case series “closed the book on Laetrile,” the most expensive health fraud in American history at the time, only to have it reopened more than 20 years later by well-meaning Cochrane reviewers who seemed oblivious of the point of an RCT.

Is that review not serious evidence that the Cochrane Collaboration overvalues RCTs? In this case, moreover, it wasn’t only basic science that Cochrane ignored, but a definitive piece of clinical research that was not an RCT. Sure, I know that Cochrane is not the only pinnacle of EBM, but it’s one of them.

In both that post and another, I called attention to a statement that Edzard Ernst, the most prolific EBM-style “CAM” researcher of the past 20 years, had made in 2003:

A couple of years ago I was surprised to find that one of the authors of [the Cochrane Laetrile] review was Edzard Ernst, a high-powered academic who over the years has undergone a welcomed transition from cautious supporter to vocal critic of much “CAM” research and many “CAM” methods. He is now a valuable member of our new organization, the Institute for Science in Medicine, and we are very happy to have him. I believe that his belated conversion to healthy skepticism was due, in large part, to his allegiance to the formal tenets of EBM. I recommend a short debate published in 2003 in Dr. Ernst’s Focus on Alternative and Complementary Therapies (FACT), pitting Jacqueline’s countryman Cees Renckens against Dr. Ernst himself. Dr. Ernst responded to Dr. Renckens’s plea to apply science to “CAM” claims with this statement:

In the context of EBM, a priori plausibility has become less and less important. The aim of EBM is to establish whether a treatment works, not how it works or how plausible it is that it may work. The main tool for finding out is the RCT. It is obvious that the principles of EBM and those of a priori plausibility can, at times, clash, and they often clash spectacularly in the realm of CAM.

I’ve discussed that debate before on SBM, and I consider it exemplary of what is wrong with how EBM weighs the import of prior probability. Dr. Ernst, if you are reading this, I’d be interested to know whether your views have changed. I hope that you no longer believe that human subjects ought to be submitted to a randomized, controlled trial of Laetrile!

Uh, talk about “suggesting that EBM ignore scientific mechanisms”! When the principles of EBM and those of a priori plausibility clash spectacularly in the realm of CAM, it is a priori plausibility that should take precedence—not merely because the latter renders RCTs unnecessary, but because for such questions RCTs tend to confuse rather than clarify, as will be discussed further in the next part of this series.

I am happy to report that Dr. Ernst wrote me privately about that passage, with the answer that I’d mostly hoped for:

Have I changed my mind? I am not as sure as the sceptics seem to be that I ever was a supporter of CAM and I am still a bit sceptic about the sceptics [which perhaps makes me the "ueber-sceptic"]. Would I argue for more Laetrile studies? NO.

Even more to the point, perhaps, is a recent editorial by Dr. Ernst in which he calls homeopathy “absurd” and compares it to other, obvious absurdities, which I doubt he’d have done only a few years ago:

Should we keep an open mind about astrology, perpetual motion, alchemy, alien abduction, and sightings of Elvis Presley? No, and we are happy to confess that our minds have closed down on homeopathy in the same way.

This kind of clear thinking, as easy as it ought to be for intelligent people, seems oddly difficult for those steeped in EBM. I’ll offer another example in part 2, as part of my response to Mr. Simon’s assertion that “There is some societal value in testing therapies that are in wide use, even though there is no scientifically valid reason to believe that those therapies work.”

This post is intended to illustrate a bit about how medicine, including alternative medicine, is defined and limited legally by state licensure. This is, of course, an enormous topic, especially given the variety of laws and regulations among the 50 states and District of Columbia, and the many, often mutually inconsistent, court decisions interpreting them. A comprehensive survey would resemble Gibbon’s history of Rome and would likely be out-of-date the moment it was finished. My more modest goal here is to highlight a few of the ways in which licensure and scope of practice laws intersect the practice of CAM and give a few representative examples. 

The Rise of Medical Licensure

In the 19th century, a bewildering variety of different approaches to maintaining health and treating disease competed for the trust, and dollars, of prospective patients (or their owners, in the case of animal patients). Caveat emptor was the rule in an unregulated medical marketplace. Mainstream medicine was a competitor in this marketplace, though it was hardly science-based to any great extent compared to conventional medical practices today. Homeopathy was another pretty big player, along with osteopathy and numerous other more or less organized schools, as well as many individual snake oil salesmen, faith healers, local providers of folk remedies, and so on.^1,2

Throughout the 19th and early 20th centuries, state legislatures passed medical practice acts defining the practice of medicine, the criteria for medical licensure, and the criminal penalties for the unlicensed practice of medicine. The American Medical Association, founded in 1846, played a powerful role in driving and shaping these early enactments of the state police authority to regulate medicine. The AMA-sponsored Flexner Report on medical education released in 1910 did much to shape the criteria states used to award licensure, and thus to shape the content of accepted medical practices. Veterinary medicine lagged a bit behind in this initial licensure movement, with California apparently being the first state to license vets in 1893, but the general legal trend has been much the same as for regulation of human medicine.^1,3

What is the Practice of Medicine?

There is some variation in the details of how the practice of medicine is defined in different state practice acts, but all the definitions are quite broad. They give physicians great latitude in the therapies they provide, but  they also leave some uncertainty as to what actually constitutes practicing medicine, and there is room for the courts to interpret and clarify the law.

In New York, for example, the law reads:

The practice of medicine is defined as diagnosing, treating, operating or prescribing for any human disease, pain, injury, deformity or physical condition.⁴

In California, the definition is even broader:

[A]ny person who practices or attempts to practice, or who advertises or holds himself or herself out as practicing, any system or mode of treating the sick or afflicted in this state,  or who diagnoses, treats, operates for, or prescribes for any ailment, blemish, deformity, disease, disfigurement, disorder, injury, or other physical or mental condition of any person…⁵

State veterinary practices acts are also very broad, sometimes even more so than medical practice acts. For example, in California:

A person practices veterinary medicine, surgery, and dentistry, and the various branches thereof, when he or she does any of the following:
(a) Represents himself or herself as engaged in the practice of veterinary medicine, veterinary surgery, or veterinary dentistry in any of its branches.
(b) Diagnoses or prescribes a drug, medicine, appliance, application, or treatment of whatever nature for the prevention, cure or relief of a wound, fracture, bodily injury, or disease of animals.
(c) Administers a drug, medicine, appliance, application, or treatment of whatever nature for the prevention, cure, or relief of a wound, fracture, bodily injury, or disease of animals…
(d) Performs a surgical or dental operation upon an animal.
(e) Performs any manual procedure for the diagnosis or pregnancy, sterility, or infertility upon livestock or Equidae.
(f) Uses any words, letters or titles in such connection or under such circumstances as to induce the belief that the person using them is engaged in the practice of veterinary medicine…⁶

With such general legal language, it might seem that physicians and veterinarians could do almost anything and call it practicing medicine. And it might also seem dangerous for people who are not licensed to practice medicine to do anything at all to a sick person or animal for fear of violating their state’s medical practice act. However, the states and the courts have created numerous exceptions and limitations to these very general standards. 

Some common exceptions are quite sensible and obvious. For example, nurses and other healthcare providers working under physician supervision, physicians working for federal agencies, properly supervised students, lay people providing reasonable first aid or lifesaving care or using widely accepted over-the-counter remedies, and many other similar activities are defined as legitimate and not the unlawful practice of medicine.¹ 

Other exceptions are sometimes less obvious, and they may provide loopholes for alternative medical providers to ply their trades. For example, animals are considered property, not persons, under the law, so their owners and anyone they authorize can do almost anything they like to treat them, within only the limitations of animal cruelty laws, which have very high standards of proof and lax enforcement.³ Some states, such as California, also provide other specific exemptions, such as this one:

Nothing in this chapter [the Medical Practice Act] shall.…regulate, prohibit, or apply to any kind of treatment by prayer, nor interfere in any way with the practice of religion.⁷

In addition to defining the practice of medicine, the state practice acts also define the criteria required to obtain and maintain a medical license. These often include graduation from an approved educational program with coverage of specified subject matter, practical clinical training, a passing score on licensing examinations, and ongoing professional education. In the early days of medical licensure, it was the development of these criteria that had the greatest impact in terms of promoting scientific medicine and limiting the ability of practitioners of alternative approaches to continuing practicing their forms of medicine. 

Licensure of Non-Physicians

In addition to physicians, states also license a number of other medical professions, including providers of alternative therapies. The relevant legislative acts define the scope of practice and criteria for licensure for these providers just as they do for physicians and veterinarians, though the scope of acceptable activities is often far more limited and narrowly defined. 

The major CAM methods are the most commonly licensed. Chiropractors are licensed in all 50 states, non-M.D. acupuncturists are licensed in 37 states, naturopaths are licensed in 17 states (though they are specifically prohibited from practicing in 2 states), and “homeopathic physicians” are licensed in 3 states.⁸ States that do not license such providers do sometimes still explicitly regulate the more common alternative methods, such as acupuncture, within other health or professions statues. 

Laws licensing alternative therapists are often very specific, while still leaving a surprising amount of room for subsequent interpretation and controversy. In California, for example, the Acupuncture Licensure Act defines acupuncture directly while also managing to fit in a much broader ideological statement:

In its concern with the need to eliminate the fundamental causes of illness, not simply to remove symptoms, and with the need to treat the whole person, the Legislature intends to establish in this article, a framework for the practice of the art and science of Asian medicine through acupuncture…

“Acupuncture” means the stimulation of a certain point or points on or near the surface of the body by the insertion of needles to prevent or modify the perception of pain or to normalize physiological functions, including pain control, for the treatment of certain diseases or dysfunctions of the body and includes the techniques of electroacupuncture, cupping, and moxibustion.⁹

The relevant legislation in California concerning chiropractic licensure defines the scope of practice for chiropractors very specifically:

(1) A duly licensed chiropractor may manipulate and adjust the spinal column and other joints of the human body and in the process thereof a chiropractor may manipulate the muscle and connective tissue related thereto. 

(2) As part of a course of chiropractic treatment, a duly licensed chiropractor may use all necessary mechanical, hygienic, and sanitary measures incident to the care of the body, including, but not limited to, air, cold, diet, exercise, heat, light, massage, physical culture, rest, ultrasound, water, and physical therapy techniques in the course of chiropractic manipulations and/or adjustments. 

(3) Other than as explicitly set forth….a duly licensed chiropractor may treat any condition, disease, or injury in any patient, including a pregnant woman, and may diagnose, so long as such treatment or diagnosis is done in a manner consistent with chiropractic methods and techniques and so long as such methods and treatment do not constitute the practice of medicine by exceeding the legal scope of chiropractic practice as set forth in this section. 

(4) A chiropractic license issued in the State of California does not authorize the holder thereof: 
(A) to practice surgery or to sever or penetrate tissues of human beings, including, but not limited to severing the umbilical cord; 
(B) to deliver a human child or practice obstetrics; 
(C) to practice dentistry; 
(D) to practice optometry; 
(E) to use any drug or medicine included in materia medica; 
(F) to use a lithotripter; 
(G) to use ultrasound on a fetus for either diagnostic or treatment purposes; or 
(H) to perform a mammography.

(5) A duly licensed chiropractor may employ the use of vitamins, food supplements, foods for special dietary use, or proprietary medicines, if the above substances are also included in section 4057 of the Business and Professions Code, so long as such substances are not included in materia medica as defined in section 13 of the Business and Professions Code…

(6) Except as specifically provided in section 302(a)(4), a duly licensed chiropractor may make use of X-ray and thermography equipment for the purposes of diagnosis but not for the purposes of treatment. A duly licensed chiropractor may make use of diagnostic ultrasound equipment for the purposes of neuromuscular skeletal diagnosis. 

(7) A duly licensed chiropractor may only practice or attempt to practice or hold himself or herself out as practicing a system of chiropractic….A chiropractor may not hold himself or herself out as being licensed as anything other than a chiropractor or as holding any other healing arts license or as practicing physical therapy or use the term “physical therapy” in advertising unless he or she holds another such license.¹⁰

The Devil in the Details

Despite the specificity of such laws and regulations, there is a fair bit of ambiguity as to exactly who is allowed to do what, and state attorneys general and the courts are frequently called upon to interpret these laws. In some states, physicians have been held to be legally permitted to practice alternative therapies even without specific licenses in those approaches, under the general provisions of medical practice. Other states, however, have required physicians to be licensed in these approaches before offering them. And, of course, given the controversial nature of many CAM therapies, physicians run some risk of being sanction for “unprofessional conduct” by their state medical board for offering some unconventional therapies. 

In one well-known example, a North Carolina doctor was disciplined by the state medical board or administering homeopathic treatments. The board concluded that since homeopathy “does not conform to the standards of acceptable and prevailing medical practice,” using it constituted unprofessional conduct. After this sanction was reversed by two lower courts (one asserting there was not sufficient evidence to support the board’s conclusions about homeopathy, and the other arguing that it didn’t matter because homeopathy was harmless), the state supreme court upheld the board’s decision.¹¹

The majority argued, quite sensibly, that “a general risk of endangering the public is inherent in any practices which fail to conform to the standards of ‘acceptable and prevailing’ medical practice” regardless of whether the specific treatment was directly harmful. The justices also recognized that “certain aspects of regulating the medical profession plainly require expertise beyond that of a layman” and that “while questions as to the efficacy of homeopathy….may be open to debate among members of the medical profession….the courts are not the proper forum for that debate.”¹² 

Unfortunately, the legislature of North Carolina took a different view, preferring to protect consumer choice and the autonomy of individual providers, and the law was subsequently amended to specifically protect physicians who offer treatments that are “experimental, nontraditional, or that [depart] from acceptable and prevailing medical practices” unless they can be clearly shown to be more harmful than conventional treatments.¹²

A variety of legal opinions and rulings also exist that clarify (or sometimes obscure) the limits of acceptable therapies licensed alternative practitioners can provide as well. For example, in some states chiropractors can legally provide colonic irrigation, perform pelvic and rectal exams, perform electrocardiograms, provide herbal remedies or nutritional supplements, or perform acupuncture under generous interpretations of the definition of chiropractic.¹³ However, other states have ruled it a violation of their scope of practice limitations for chiropractors to utilize herbal or nutritional substances in treating patients, perform pelvic exams, perform or order diagnostic blood or urine tests, and engage in other such practices deemed outside of their licensed activities.¹³ There are similar inconsistencies in the interpretation of what therapies are permissible for other licensed alternative providers as well.

Faith Healing and Secular Spiritual Practices

A particularly interesting area of medical jurisprudence is the relationship of licensing laws to spiritual and religious practices. The tension between individual liberty and freedom of economic activity on the one hand and the role of government in protecting the public health on the other is weak tea compared to the conflict between government police powers and the legal and cultural imperatives in the U.S. to protect religious freedom. Religion may be specifically excluded from medical regulation, as in the California code above or in states like Minnesota and Ohio, which specifically exempt Christian Science faith healing from the definition of medical practice for example.¹⁴ And even in the absence of specific exemptions for religious activity, medical regulations are often required to meet a very high standard of justification if they are perceived to interfere with religious practices. The decisions made by courts and state legislatures on such matters often hinge more on the issue of religious liberty versus state police powers than on the question of whether spiritual healing practices have any medical benefit.

This controversy also has implications for so-called “secular spiritual” approaches, such as the various energy therapies like Reiki, Thought Field Therapy, Healing Touch, some meditation or mind-body therapies, and so on. Are such methods the practice of medicine? Are they religious practices and so protected to some extent from regulation? Does it matter if they work? Or if they are harmless? These are questions that have been raised by advocates of these approaches and legal scholars but which have not often been directly or clearly addressed in law or judicial rulings.

In cases involving ostensibly religious healing practices, courts have both supported and overruled restrictions imposed by medical regulations. In one Florida case, a man who claimed to heal through prayer and laying on hands was accused of practicing medicine. He claimed his activity was protected under the provision indicating the state practice act did not apply to “the practice of the religious tenets of any church.” The state supreme court ruling in the defendant’s favor did point out that his actions did not “invade the province of the medical profession and assume the ability to diagnose diseases and prescribe drugs or other medical or surgical or mechanical means to restore the health” of his clients. However, the bulk of the opinion was concerned with the issue of religious liberty and with the apparent lack of any direct harm done by the healing practice, as well as the more general epistemological argument that science doesn’t know everything:

…from ancient times down to this modern and so called materialistic age, there have always been quite a large percentage of people who believed in the efficacy and availability of Divine power, not only to save the souls but also the bodies and lives of men and to heal all the ills that flesh is heir to….And if this class of people hear and believe that some person can and does invoke the power of Most High to heal people of their ills, or that in his own person such individual possesses some strange mental, magnetic or psychic power to banish disease from the human body….[they] will seek him out. And it is not the policy of our laws to prevent them; nor to punish those to whom they go, and who endeavor to heal the ills of men by such mental or spiritual means…

The reason for this policy is founded upon the liberty of the individual citizen under our bill of rights, and the fact that so long as these faith healers or spirit mediums rely upon their power, by prayer or faith, to invoke the exercise of the power of the Almighty, if indeed they fail to cure, they at least can do no harm…

Now this appellant testified that the power which he invoked was not his own, but that it was the power of God. And if some of the uncontradicted witnesses are to be believed, he was instrumental in accomplishing some remarkable cures….Now, to most of us, this matter of healing ‘by faith and the laying on of hands,’ ancient as it is, is still beyond us. But according to Shakespeare’s Hamlet, ‘There are more things in heaven and earth, Horatio, than are dreamt of in your philosophy.’ And in that magnificent speech of St. Paul’s before King Agrippa, he said to the king: ‘Why shuld it be thought a thing incredible with you, that God should raise the dead?’ So the legislature and the courts might well accord our citizens the liberty to decide such questions as these for themselves.”¹⁵

Other rulings on the question of whether religious practices are subject to the regulations that govern the practice of medicine have been more pragmatic and less prolix. A California case concerned a healer who imposed on people not only prayer and laying on of hands but strict fasting and dietary requirements and a prohibition on conventional medical diagnostics or treatment. He attempted to defend his practices as religious rather than medical, even after the death of one person and significant injury to others following his approach. Despite the state’s exemption of religious practices from the medical practice act, the court ruled that the methods used went well beyond protected religious activity and invaded the domain of secular medicine. 

There is no question that the described activities constitute the practice of medicine; that the Board has a substantial interest in preventing such activities, which are demonstrably harmful on this record; …[and] there is [no] serious argument made that the injunction infringes appellants’ constitutional rights of free exercise of religion under the First Amendment. Cases are legion which hold that freedom of religious belief may be absolute but freedom of action is not.[citations omitted] The state may legitimately regulate dangerous conduct regardless of religious content. It is therefore universally held that in the interest of protecting its citizens’ health, the state may regulate health treatments which are potentially dangerous to the patient….In reaching this decision we do not deem it necessary to question the bona fides of appellant Andrews’ religious faith; that fact is not relevant…¹⁶

Of course, there are salient differences in the facts in these cases, as well as the historical moments in which they were heard. The practical question of whether direct harm is done by a spiritual healing approach is weighed by the courts in adjudicating such cases. However, as is frequently the case, the reasoning that informs the courts’ decisions often fail to address the scientific question of the efficacy of such treatments or the potential indirect harm they may do in discouraging effective medical care. And the issues of individual and religious liberty are often given as much or more weight than the question of whether the interventions are demonstrably effective or not.

Is Medical Licensure Fair and Does It Protect the Public?

Medical licensure is widely accepted as a legitimate use of state authority to protect the public health by preventing people from being exposed to dangerous and ineffective therapies in an unrestrained medical market. Proponents of CAM, as well as opponents of government regulation generally, sometimes cast the promulgation of medical licensing laws as a straightforward protectionist campaign by “allopathic” doctors to wipe out the competition. While it would be disingenuous to suggest that professional organizations such as the AMA or the American Veterinary Medical Association (AVMA) have no political or economic agendas beyond the protection of the public good, it is a convenient but inaccurate exaggeration to say that concerns for territory or income have been the prime motivators of efforts to license and regulate medicine. Concerns for the actual, verifiable scientific truth behind medical practices and the welfare of patients have always been a genuine and important reason for encouraging government to regulate healthcare. 

Even in the notorious antitrust case Wilk v. American Medical Association, in which chiropractors succeeded in using anti-trust laws and allegations of protectionism to weaken the ability of the AMA, and other professional organizations, to marginalize unscientific medical practices, the court was “persuaded that the dominant factor [in the AMA's efforts] was patient care and the AMA’s subjective belief that chiropractic was not in the best interests of patients.”¹⁷

Licensure and scope of practice limitations do leave enormous room for physicians and others to engage in ineffective and dangerous medical practices, and the spirit and letter of the law is subject to wide-ranging interpretations in different states and courts. And while licensing CAM professions arguably gives the state some ability to enforce reasonable standards of training and practice, there is an element of Tooth Fairy Regulation in this (with apologies to Dr. Hall). 

For example, the California law regulating acupuncturists requires a minimum of 2250 hours of clinical training and 1548 hours of theoretical and didactic training to apply for a license. Some of this, such as how to avoid transmitting infectious diseases with needles, is legitimate training that protects public health. But it is debatable how helpful it is to require many hours of study of Qi Gong, Traditional Oriental Medicine Theory, Moxibustion, Ear Acupuncture theory, and so on.
And, of course, licensure creates a perception of legitimacy and accuracy to the claims of CAM providers in the minds of the public, who generally don’t appreciate the extent to which decisions about medical regulation are made less on the basis of scientific facts than on the basis of political and philosophical issues. 

On balance, the regulation of conventional medical practice, and to a lesser extent alternative therapies, probably is reasonably effective in protecting the public. The popularity and availability of dangerous and clearly ineffective approaches is certainly less than it was during the age of medical anarchism, and such laws have doubtless played a role in this. 

Why We Should Understand Medical Licensing Laws

While we must always maintain our emphasis on verifiable scientific facts about the safety and efficacy of proposed therapeutic approaches, those of us dedicated to science-based medicine may also be able to play a role as a constituency in shaping the writing and interpretation of medical laws and regulation to better protect the public. And we must certainly be aware of what our own state’s laws are, and participate in seeing that they are properly executed. 

In researching this subject, for example, I became aware that the California veterinary practice act has very strict requirements for veterinary supervision of chiropractic applied to animals, and also a requirement that “the veterinarian shall obtain as part of the patient’s permanent record, a signed acknowledgment from the owner of the patient or his or her authorized representative that [musculoskeletal manipulation] is considered to be an alternative (nonstandard) veterinary therapy.”¹⁸ I am certain most of the vets I know who refer patients for chiropractic treatment do not comply with these guidelines, and if wider compliance can be achieved it would likely reduce the utilization and potential harm of this unproven, and possibly dangerous, therapeutic approach. 

A familiarity with the laws govern medical practice is an important element in advocating for good quality scientific medicine and discouraging unproven or unsafe interventions. Part of my goal in this series is to encourage such familiarity. The references I cite in these posts are a good starting point, though they have their limitations and biases. Most relevant state laws and regulations are easily accessible on the internet. 

Proponents of alternative therapies are aware of the importance of understanding and helping to shape medical laws and regulations, and they explicitly encourage CAM practitioners to be knowledgeable and involved (see, for example,  these resources for acupuncturists, naturopaths, and chiropractors). Since professional organizations such as the AMA and AVMA are limited by political and legal considerations from aggressively working to shape legislation and public policies that discourage alternative therapies, those of us who would promote science-based medicine would do well to be as familiar with medical laws and the agendas that influence them as we try to be with the scientific facts concerning questionable medical practices. 

References

1. Jesson LE, Tovino SA. Complementary and alternative medicine and the law. Durham (NC), USA: Carolina Academic Press, 2010.  Return to text.
2. Ramey DW, Rollin BE. Untested therapies and medical anarchism. In: Complementary and alternative veterinary medicine considered. Ames (IA), USA: Iowa State Press, 2004. p.168-9. Return to text.
3. Wilson JF, Rollin BE, Garbe, JAL. Law and ethics of the veterinary profession.Morrisville (PA), USA: Priority Press Ltd, 1993. Return to text.
4. N.Y. Educ. Law § 6521. Cited in Jesson LE, Tovino SA. Complementary and alternative medicine and the law. Durham (NC), USA: Carolina Academic Press, 2010. p. 36. Return to text.
5. Cal. Bus. & Prof. Code § 2052(a). Cited in Jesson LE, Tovino SA. Complementary and alternative medicine and the law. Durham (NC), USA: Carolina Academic Press, 2010. p. 36. Return to text.
6. Cal. Bus. & Prof. Code § 4826. Cited In: California Veterinary Medicine Practice Act. 2010 Ed. Charlottesville (VA), USA: LexisNexis, Matthew Bender & Company, Inc, 2010. p. 5-6. Return to text.
7. Cal. Bus. & Prof. Code § 2063. Retrieved Sept. 9, 2010 from California Law Website: http://www.leginfo.ca.gov/cgi-bin/displaycode?section=bpc&group=02001-03000&file=2050-2079 Return to text.
8. Jesson LE, Tovino SA. Complementary and alternative medicine and the law. Durham (NC), USA: Carolina Academic Press, 2010. p.56. Return to text.
9. Cal. Bus. & Prof. Code § 4026-4027. Retrieved Sept. 9, 2010 from California Law Website: http://www.leginfo.ca.gov/cgi-bin/displaycode?section=bpc&group=04001-05000&file=4925-4934.2 Return to text.
10. Cal. Admin. Code tit. 16, § 302 Retrieved Sept. 9, 2010 from webite: http://weblinks.westlaw.com/result/default.aspx?cite=16CAADCS302&db=1000937&findtype=L&fn=%5Ftop&ifm=NotSet&pbc=4BF3FCBE&rlt=CLID%5FFQRLT2612239251299&rp=%2FSearch%2Fdefault%2Ewl&rs=WEBL10%2E08&service=Find&spa=CCR%2D1000&sr=TC&vr=2%2E0 Return to text.
11. Guess v. Board of Medical Examiners 393 S.E.2d 833 (1990). Cited in Jesson LE, Tovino SA. Complementary and alternative medicine and the law. Durham (NC), USA: Carolina Academic Press, 2010. p. 42-44. Return to text.
12. Jesson LE, Tovino SA. Complementary and alternative medicine and the law. Durham (NC), USA: Carolina Academic Press, 2010. p.50. Return to text.
13. Jesson LE, Tovino SA. Complementary and alternative medicine and the law. Durham (NC), USA: Carolina Academic Press, 2010. p.87-88. Return to text.
14. Jesson LE, Tovino SA. Complementary and alternative medicine and the law. Durham (NC), USA: Carolina Academic Press, 2010. p.61-62. Return to text.
15. Curley v. State of Florida Supreme Court of Florida, en Bacn 16 So. 2d 440 (1943). Cited in Jesson LE, Tovino SA. Complementary and alternative medicine and the law. Durham (NC), USA: Carolina Academic Press, 2010. p. 58-60. Return to text.
16. Board of Medical Quality Assurance v. Arthur Andrews Court of Appeal, Sixth Distr., California 211 Cal. App. 3d 1346 (1989). Cited in Jesson LE, Tovino SA. Complementary and alternative medicine and the law. Durham (NC), USA: Carolina Academic Press, 2010. p. 60-63. Return to text.
17. Wilk v. American Medical Association 671 F. Supp 1465 (N.D. Ill.) 1987. Cited in: Jesson LE, Tovino SA. Complementary and alternative medicine and the law. Durham (NC), USA: Carolina Academic Press, 2010. p. 230-241. Return to text.
18. Cal. Code of Regulations § 2038. Cited In: California Veterinary Medicine Practice Act. 2010 Ed. Charlottesville (VA), USA: LexisNexis, Matthew Bender & Company, Inc, 2010. p173. Return to text.

One of the recurrent themes of science-based medicine is that any medical intervention that can plausibly cause physiological benefit can also plausibly cause physiological harm.  There is no such thing as “it can’t hurt.” Sometimes the risk may be minuscule – but we should never assume that it is zero. Being “natural” or “holistic” or being blessed with some other alleged marketable virtue does not affect the risk vs benefit calculation of an intervention.

Vitamins are an excellent example. There is widespread sentiment that vitamins are harmless, and that supplementing with vitamins is therefore a no risk-possible benefit scenario. It is certainly reasonable to conclude from the evidence that vitamins (at usual supplemental levels) are low risk, compared to many other types of medical interventions. High doses, or megadoses, of vitamins, however, risk toxicity and this risk increases with the dose.

But even at sub-toxic doses vitamins should not be assumed to be risk free. This is especially true when we take a public health perspective – what is the net effect of large scale supplementation on the population? A new meta-analysis looking at the net effects of Vitamin E supplementation on stroke risk reinforces this caution.

Vitamin E has received a lot of attention recently because of its antioxidant effects. Oxidative stress plays a role in tissue damage, aging, and various disease processes, and so supplementing with anti-oxidants seems like an obvious treatment to mitigate this damage. However, biology is complex, and oxygen free radicals also play a role in cell signaling, for example, so that exogenously suppressing them may have negative unintended consequences.

For example, the emerging research regarding Vitamin E and heart disease is mixed and complex. Vitamin E supplements actually seem to increase total mortality and heart failure. However, observational studies show a decreased risk of cardiac disease in those who take Vitamin E. The difference may be that foods rich in Vitamin E come with a health benefit (which may be from the foods that are not eaten with such a diet), but vitamin pills do not convey this benefit. There may also be a difference between primary prevention (in those without prior cardiac events) where there is a net benefit and secondary prevention (in those who have suffered a cardiac event) where there is net risk. There may be subpopulations, like diabetics, who benefit more.

The bottom line at this time is that eating a healthful diet rich in fruits and vegetables is consistently associated with decreased risk of various diseases, including heart disease. However taking vitamin E supplements may not have this same benefit, and in fact may come with a net risk of increased heart disease and mortality.

What about stroke risk? While there are differences, stroke is also a vascular disease, like myocardial infarction, and the risk factors tend to be similar. In the new meta-analysis the researchers found:

In this meta-analysis, vitamin E increased the risk for haemorrhagic stroke by 22% and reduced the risk of ischaemic stroke by 10%. This differential risk pattern is obscured when looking at total stroke. Given the relatively small risk reduction of ischaemic stroke and the generally more severe outcome of haemorrhagic stroke, indiscriminate widespread use of vitamin E should be cautioned against.

Again we see a bottom line caution against supplementing with vitamin E. It is important to note that even a small increase in net stroke incidence has a huge effect on the general population. Stroke is a debilitating disease, potentially fatal, and a huge financial burden on the health care system. Even small percentage increases therefore have a huge societal effect. A 22% increased in hemorrhagic stroke is a very large clinical effect.

Conclusion

The research on vitamins in general and vitamin E in particular is messy and complicated. My overall impression of this research is that there is no consistent signal of net benefit for routine supplementation. There are many specific conditions in which specific supplementation is of benefit, but not routine supplementation for general health.

At the same time there is a consistent signal of benefit to having a healthful diet, the primary feature of which is to have a diet rich in fruits and vegetables. So in the end, after decades of research, what your mother always told you turns out to be the best advice – eat your vegetables.

Melanie Thernstrom has written a superb book based on a historical, philosophical, and scientific review of pain: The Pain Chronicles: Cures, Myths, Mysteries, Prayers, Diaries, Brain Scans, Healing, and the Science of Suffering. Herself a victim of chronic pain, she brings a personal perspective to the subject and also includes informative vignettes of doctors and patients she encountered at the many pain clinics she visited in her investigations. She shows that medical treatment of pain is suboptimal because most doctors have not yet incorporated recent scientific discoveries into their thinking, discoveries indicating that chronic pain is a disease in its own right, a state of pathological pain sensitivity.

Chronic pain often outlives its original causes, worsens over time, and takes on a puzzling life of its own… there is increasing evidence that over time, untreated pain eventually rewrites the central nervous system, causing pathological changes to the brain and spinal cord, and that these in turn cause greater pain. Even more disturbingly, recent evidence suggests that prolonged pain actually damages parts of the brain, including those involved in cognition.

Sometimes the original problem creates new ones as the patient distorts posture and avoids exercise in an attempt to reduce the pain.  In chronic pain, the protective mechanism of avoidance becomes maladaptive. Muscles atrophy from disuse and new sources of pain develop. Jerome Groopman, MD, in The Anatomy of Hope, told how he conquered years of chronic back pain by realizing that his pain was not a warning to avoid further damage but a false message that he could refuse to listen to; with exercise and physical therapy he rebuilt his muscles and became pain-free.

Dr. John Sarno believes that chronic musculoskeletal pain is a manifestation of “tension myositis syndrome” due to repressed negative emotions. He recommends renouncing all treatments, accepting that pain is only in the mind, and resuming normal activities. I don’t accept his psychosomatic premise, but there is a grain of truth in his method. If patients can re-frame their thinking and resume normal activities despite the pain, they are more likely to improve than if they maintain the self-image of a sick, disabled victim.

Distraction is effective in removing the awareness of pain. Thernstrom tells us that as she got better,

I wasn’t aware of being in pain all the time, but whenever I thought about whether I had pain, I always did. There were pain-free moments owing to my being preoccupied — happily or unhappily — with something else, but I was never able to “catch” a pain-free moment and enjoy it, which meant that, in some sense, I was always in pain.

Pain perception in the brain involves two different pain systems: one of pain perception and one of pain modulation. Acute injuries always hurt more later as the modulation effects diminish and the brain releases neurotransmitters into the spinal cord that amplify incoming signals and augment pain. This serves the adaptive purpose of enabling flight at first and then enforcing rest. It is possible to induce complete analgesia in humans and animals by electrically stimulating pain-modulating areas of the brain. Various cognitive and affective states activate the two systems, especially attention and expectation. Simply asking patients to think about their pain activates the pain-perception circuits. Anticipation of a placebo effect causes the pain-modulating release of endorphins in the brain.

One medication requires the placebo effect for all of its effectiveness. An intriguing 1995 clinical trial proved an analgesic called proglumide to be a more effective pain reliever than a placebo when both groups were told they were being given an exciting new painkiller. But then subjects were slipped proglumide without their knowledge, thus ensuring they had no placebo effect, they felt no relief at all. None.

It turns out proglumide enhances the endorphin response by blocking cholecystokinin receptors.  Thernstrom speculates that drugs could be designed to enhance or create a placebo effect. Hmm… what would medical ethicists have to say about that? For that matter, how can a treatment still be called a placebo if it is shown to have the effect of producing endorphins in the brain?

Opioids relieve pain, but they are both under-used and over-used. If acute pain were better controlled, fewer patients would develop chronic pain. On the other hand, many chronic pain patients develop opioid-induced hyperalgesia, where their body becomes more sensitive to pain stimuli or even ordinary stimuli; they develop pain in parts of their bodies remote from the original injury site.

Caution is required. Relieving pain sometimes causes harm. A phase 3 study of tanezumab was recently halted by the FDA. Although the drug relieved the pain of osteoarthritis, it also resulted in more joint failure, presumably because there was more wear and tear on the joints when pain was absent.

The Pain Chronicles is a fascinating glimpse into the world of pain sufferers as well as a good overview of our current scientific knowledge. It suggests avenues of investigation that may vastly improve our management of pain. I highly recommend it to anyone who wants to know more about any aspect of the pain experience and the science.

When I write or talk about the scientific evidence against particular alternative medical approaches, I am frequently asked the question, “So, if it doesn’t work, why is it legal?” Believers in CAM ask this to show that there must be something to what they are promoting or, presumably, the government wouldn’t let them sell it. And skeptics raise the question often out of sheer incredulity that anyone would be allowed to make money selling a medical therapy that doesn’t work. It turns out that the answer to this question is a complex, multilayered story involving science, history, politics, religion, and culture. 

While we science types tend to be primarily interested in what is true and what isn’t, that is a sometimes surprisingly minor factor in the process of constructing laws and regulations concerning medicine. What I hope to do in this series of essays is look at some of the major themes involved in the regulation of medical practice, particularly as they relate to alternative medicine. I will begin by touching on some of the general philosophical and legal issues that have defined the debate among the politicians and lawyers responsible for shaping the legal environment in which medicine is practiced. The I will review some of the specific domains within this environment, including: medical licensure and scope-of-practice laws; malpractice law; FDA regulation of drugs, homeopathic remedies, and dietary supplements; truth-in-advertising law; and anti-trust law.

But first…

The Disclaimer

Obviously, an exhaustive and comprehensive look at the Byzantine and unstable landscape of medical law is beyond the scope of both this blog and my own knowledge and expertise. I am no lawyer, and for the details of the laws and judicial opinions concerning this subject I must rely on sources whose accuracy I am not qualified to verify independently. Much of the published material I have found on CAM and the law seems written from a political and ideological perspective sympathetic to the postmodernist notion of multiple equally legitimate “ways of knowing,” and also to a laissez-faire approach to regulation generally. So clearly the details provided and the interpretations given in such writings may not fairly represent the legal or regulatory environment. In any case, while I hope to provide some useful insight into how CAM fits into the system of medical law and regulation in the United States,  nothing I say should be taken as the definitive word on the law or as legal advice.  

Caveat Emptor v. Caveat Venditor

There is a deep ideological divide in America on the subject of who is responsible for ensuring that the products we buy are safe and perform as advertised, and the area of medicine is not exempt from this political debate. On one extreme is the self-identified “health freedom” lobby, which argues that the consumer and the market should be the only forces to regulate healthcare products and services. As an example, economist Randall Holcombe has written:

An auto mechanic does not have to be a medical expert to use market information to find good health care, any more than a doctor has to be an automobile expert to find a good car…Deregulation not only provides incentives for patients to look for, and physicians to offer, better care, it permits all parties concerned the freedom to decide what better care is. For instance, in the debate over alternative medicine, such as herbal treatments, chiropractors, acupuncture, and so on, the question is not only whether alternative medicine is effective, but whether people should be allowed to use these alternatives even if their physical health may not improve or may even suffer….In a free country, people should be free to choose whatever health care options they want for whatever purpose…even if healthcare professionals believe that care is substandard.¹

Those more sympathetic to laws and regulations intended to protect consumers from unsafe and ineffective therapies argue against this concept of “medical anarchism:”

Why not let the market decide? Why not trust the citizenry to sort out what works from what doesn’t work in medicine as we do in other aspects of life?

The answer has to do with knowledge and risk. People do let the market decide with regard to goods like ice cream cones and baseball bats, and services like travel booking. If the ice cream is not good, people won’t buy it; if the service is defective, people will go elsewhere. However, in such situations, people are easily able to evaluate the quality and value of the goods and services they receive…Nor are such services administered under duress, nor are they represented as necessary for one’s health or well-being…

But in the area of medicine, too much is at stake. If one chooses the wrong therapeutic modality, once can lose health, life, and limb. Furthermore, few individuals are sufficiently wealthy, educated, or possessed of the resources to test putative medical therapies. In fact, there are so many putative therapies, that it is impossible for an individual to try them all. When people are ill, they do not have time to test even a handful.²

These arguments tend to run in parallel, and to be only tenuously connected, with the usual focus of this blog; the question of how one evaluates medical therapies and what the evaluation indicates about safety and efficacy. Of course, many proponents of  CAM who invoke the “health freedom” position do actually believe the therapies they promote are beneficial. But the fundamental position itself does not hinge on this, since from a perspective such as Dr. Holcombe’s people should be free to choose even therapies that are ineffective or harmful without “burdensome” government regulatory interference. The self-evident notion that it is the role of government to protect the public from quackery turns out not to be self-evident to many Americans, and thus demonstrating that a given approach is quackery may not be sufficient to convince them that it should be prohibited or even officially discouraged. 

The Right to Privacy v. State Police Powers

In the legal arena, the political conflict between those favoring or opposing aggressive consumer protection regulations in the area of healthcare takes the form of statutes and judicial opinions balancing the competing constitutional principles of an individual right to privacy and a governmental authority, or even mandate, to protect the public health. Neither a right to privacy or absolute authority over one’s own body nor a government role in regulating healthcare are specifically mentioned in the U.S. Constitution, but both are held to exist by long-standing interpretation. A right to privacy, including control over one’s own body and the care of it, is generally believed to be established by a broad reading of the 14th Amendment, though there is some controversy about this as about most areas of constitutional law. The authority of the state to abrogate this right in the process of protecting the public health is usually understood to be based in the “police powers” established by the 10th Amendment.

In 1824, the Supreme Court made reference to “health laws of every description” as encompassed within the “state police powers,” those powers not specifically delegated to the federal government nor prohibited to the States which are thus held, under the Tenth Amendment, to be the prerogative of the individual states.³ The court cited and expanded this opinion in a subsequent case in 1905, in which a state mandate to protect the public health was held to override, at least in some circumstances, the individual right to control one’s own body. The case involved a man prosecuted for refusing a mandatory smallpox vaccination. The opinion stated:

The authority of the state to enact this statute is to be referred to what is commonly called the police power…this court …distinctly recognized the authority of a state to enact quarantine laws and “health laws of every description…”

The defendant insists that his liberty is invaded when the state subjects him to fine or imprisonment for neglecting or refusing to submit to vaccination…and that the execution of such a law…is nothing short of an assault upon his person. But the liberty secured by the Constitution of the United States…does not import an absolute right in each person to be, at all times and in all circumstances, wholly freed from restraint. There are manifold restraints to which every person is necessarily subject for the common good.⁴

The court went on to specifically balance the “liberty secured by the 14th Amendment,” including “the control of one’s body” against “the power of the public to guard itself against imminent danger” and concluded that under at least some circumstances the authority to protect the public health trumps he right of an individual to control his or her own body. 

This precedent was further developed and expanded in subsequent cases to validate the state’s authority to define and regulate medical practices, to control what practices could be offered and by whom via licensing and scope-of-practice laws, and to prohibit individual’s from choosing specific medical treatments if these were considered to be ineffective or dangerous. I will discuss the specifics of these cases in subsequent posts. But for now I simply want to illustrate that the legal basis for the regulations of medical practice which today pertain to CAM, as well as scientific medicine, is generally seen by the courts as a balance between the individual right to privacy and the state authority to protect public health.^1,5

Just the Facts, Ma’am?^*

I feel it is important to emphasize again that the question of the medical facts in such cases, and how these are established, are not always seen by the courts to be as relevant as the legal or political issues. For example, in Jacobson v. Massachusetts the court specifically addressed the factual claims by the defendant that the vaccine was ineffective and unsafe. The court’s reasoning will seem familiar, and disturbing, to those of us dealing with the anti-vaccination movement today:

The appellant claims that vaccination does not tend to prevent smallpox, but tends to bring about other diseases, and that it does much harm, with no good. It must be conceded that some laymen, both learned and unlearned, and some physicians of great skill and repute, do not believe that vaccination is a preventative of smallpox. The common belief, however, is that it has a decided tendency to prevent the spread of this fearful disease…While not accepted by all, it is accepted by the mass of the people, as well as by most members of the medical profession…A common belief, like common knowledge, does not require evidence to establish its existence, but may be acted upon without proof by the legislature and the courts…The fact that the belief may be wrong, and that science may yet show it to be wrong, is not conclusive; for the legislature has the right to pass laws which, according to the common belief of the people, are adapted to prevent the spread of contagious diseases. In a free country, where the government is by the people, through their chosen representatives, practical legislation admits of no other standard of action, for what the people believe is for the common welfare must be accepted as tending to promote the common welfare, whether it does in fact or not. Any other basis would conflict with the spirit of the Constitution, and would sanction measures opposed to a Republican form of government.⁴

While the decision in this case, to support the authority of the state to enforce mandatory vaccination as a public health measure, might be welcomed by supporters of science-based public health policy, the decision itself was by no means based in science or scientific reasoning. 

The laws and judicial opinions which govern the practice of medicine may sometimes support and sometimes oppose legitimate, science and evidence-based medicine. But the legislators, lawyers, and judges responsible for these laws and opinions are not scientists, and their reasoning about scientific and medical issues often has a philosophical and epistemological basis often incompatible with the scientific approach. Such policy mistakes as DSHEA and NCCAM are much easier to understand, and hopefully prevent, if we clearly understand this.

If we are to be effective at promoting scientific medicine and containing unscientific approaches and ineffective or unsafe therapies, we must be aware of the limitations of scientific and fact-based arguments in persuading legislators and judges, as well as the general public. Though science and facts derived from scientific knowledge and investigation must be the foundation of our medical approach, they are not always the most effective means of making the case for this approach, even with our colleagues much less with the citizens, politicians, and legal professionals who ultimately control what sort of influence and oversight government has on medicine. Non-scientists tend to view debates about regulation of CAM in terms of individual rights, consumer protection, truth-in-advertising, fair competition in the marketplace, and other such political and philosophical frames which are as important, or even more important, to them as the issue of what is factually true about CAM and whether particular therapies help or harm. 

In this series of essays, I will look at laws and regulations concerning CAM primarily from these perspectives. The kinds of questions that arise in this process may initially seem odd to those of us accustomed to a straightforward emphasis on the relevant facts and evidence. Are doctors allowed to offer unproven or even clearly bogus therapies? Are they required to offer them if a patient wants them? Can a mainstream doctor, be sued for providing or failing to provide an alternative therapy? Can an alternative practitioner be sued for providing, or failing to provide, mainstream scientific medical care? Can and should patients have whatever care they want regardless of whether science supports it? And from my perspective as a veterinarian, since pets are legally property not persons, is there any legal or regulatory control over alternative veterinary medicine at all? Such questions and the reasoning behind asking and answering them, shapes the landscape within which we operate as healthcare providers and advocates for science-based medicine, so I hope an examination of them will be interesting and useful.

* Our friends at snopes.com tell me that Joe Friday never actually said this, but due to its cultural resonance I choose to invoke the phrase anyway. Oh, I hope all this exposure to legal argument and reasoning hasn’t damaged my respect for actual facts! Return to text.

References

1. Jesson LE, Tovino SA. Complementary and alternative medicine and the law. Durham (NC), USA: Carolina Academic Press, 2010. p. 279. Return to text.
2. Ramey DW, Rollin BE. Untested therapies and medical anarchism. In: Complementary and alternative veterinary medicine considered. Ames (IA), USA: Iowa State Press, 2004. p.168-9. Return to text.
3. Gibbons v. Ogden, 22 U.S. 1, 78 (1824). cited in Jesson LE, Tovino SA. Complementary and alternative medicine and the law. Durham (NC), USA: Carolina Academic Press, 2010. p. 26. Return to text.
4. Jacobson v. Massachusetts, 197 U.S. 11 (1905). cited in Jesson LE, Tovino SA. Complementary and alternative medicine and the law. Durham (NC), USA: Carolina Academic Press, 2010. p. 26-29. Return to text.
5. Cohen MH. Legal issues in alternative medicine: A guide for clinicians, hospitals, and patients. Victoria (BC), Canada: Trafford Publishing, 2003. Return to text.

We advocate science-based medicine (SBM) on this blog. However, from time to time, I feel it necessary to point out that science-based medicine is not the same thing as turning medicine into a science. Rather, we argue that what we do as clinicians should be based in science. This is not a distinction without a difference. If we were practicing pure science, we would be theoretically able to create algorithms and flowcharts telling us how to care for patients with any given condition, and we would never deviate from them. It is true that we do have algorithms and flowcharts suggesting guidelines for care for a wide variety of conditions, but there is wide latitude in them, and often a physician’s “judgment” still ends up trumping the guidelines. While it is also true that sometimes physicians have an overinflated view of the quality of their own “clinical judgment,” sometimes to the point of leading them to reject well-established science, as Dr. Jay Gordon frequently does, what I consider to be physician’s judgment is knowing how to apply existing medical science to individual patients based on their circumstances and, yes, even desires and values.

Indeed, if there’s one area where SBM has all too often fallen short in the past, it’s in taking into account the patient’s experience with various treatments. What got me thinking (again) about this issue was an article by Dr. Pauline Chen in the New York Times last Thursday entitled Listening to Patients Living With Illness. She begins her article with an anecdote:

Wiry, fair-haired and in his 60s, the patient had received a prostate cancer diagnosis a year earlier. When his doctors told him that surgery and radiation therapy were equally effective and that it was up to him to decide, he chose radiation with little hesitation.

But one afternoon a month after completing his treatment, the patient was shocked to see red urine collecting in the urinal. After his doctors performed a series of tests and bladder irrigations through a pencil-size catheter, he learned that the bleeding was a complication of the radiation treatment.

He recalled briefly hearing about this side effect three months earlier, but none of the reports he had been given or collected mentioned it, and once he had recovered from the angst of the emergency room and the doctor’s office visits and the discomfort of the clinical work-up, he didn’t give it more thought — until a few weeks later, when he started bleeding again.

By the time I met him, he was in the middle of his third visit to the hospital. “I feel like I’m tied to this place,” he said. He showed me a plastic jug partly filled with urine the color of fruit punch, and he described a post-treatment life marked by fear of going to the bathroom and discovering blood. “If I had known that my life would be like this after radiation,” he sighed, “I would have chosen the surgery.”

To this, I’ll add a little random bit of personal experience of my own. No, I wasn’t a patient who had to face something like this patient, but I do see something similar in my patients. Back when I was in my surgical oncology fellowship — and before that, in my general surgery fellowship — I was always taught that lumpectomy was preferable to mastectomy because it saves the breast and most women want to save their breasts. After all, lumpectomy plus radiation therapy results in the same chance of survival as mastectomy; so we should offer lumpectomy whenever tumor characteristics (the main one being size relative to the rest of the breast) permit it. Yet this assessment often neglects to acknowledge that, for some women, undergoing six or seven weeks of radiation is horribly inconvenient, and that there are often complications. It also often neglects to acknowledge that there is a price for saving the breast besides having to undergo radiation therapy: there’s the possibility of more surgery to achieve clear surgical margins, not to mention a higher risk of local recurrence in the breast. For some women, this latter possibility is a deal-breaker. Even though they acknowledge that their chances of survival would be the same with lumpectomy or mastectomy, the thought of an approximately 8% local recurrence rate eats at them to the point that they opt for mastectomy.

Then there is the issue of chemotherapy. We frequently recommend cytotoxic chemotherapy for women with relatively early stage breast cancer, even though the addition of chemotherapy in such patients only increases the chance of survival by perhaps 2–3% on an absolute basis, depending upon the tumor. Of course, as I’ve pointed out before, the benefits of chemotherapy are more marked in more advanced operable tumors, but in early stage tumors they are rather modest. This is therapy that causes hair loss, increased risk of infections, and can cause damage to the heart, but it is the standard of care. Most women are willing to undergo this sort of therapy, too; I can’t locate the study, but I’ve seen one survey where women respond that they would be willing to undergo chemotherapy for a 1% increased chance of survival.

The point is that these sorts of questions are value judgments that often depend upon what patients consider important. The patient described by Dr. Chen, for instance, would apparently prefer risks of surgery rather than peeing blood all the time and having to go back to the doctor’s office and hospital time and time again for this problem. Science can tell a physician and patient like this that radiation or surgery will produce an equivalent chance of surviving his cancer. It can tell them what the complications of each choice are likely to be, and what the odds are of each complication. That’s part of what I mean when I refer to science-based medicine. What it can’t tell the patient and doctor is which constellation of risks would be more easily bearable by the patient. The same is true for whether to choose mastectomy or radiation or whether to opt for chemotherapy after breast cancer. Science provides the numbers and the “price” of each choice, but it can’t — nor should it — tell the patient what to value. Moreover, what the patient values may not be what the physician values. As Dr. Chen points out:

Whether conducted at a laboratory bench or in clinical trials, medical research has long been driven by a single overriding goal — the need to find a cure. Usually referred to more modestly as a search for “the most effective treatment,” this standard has served as both a barometer of success and a major criterion for funding. Most published studies are marked by a preponderance of data documenting even minor blips in laboratory values or changes in the size of a spot of cancer or area of heart muscle damage on specialized X-rays. Some studies bolster the apparent success of their results with additional data on societal effects like treatment costs or numbers of workdays missed.

Few studies, however, focus on the patient experience.

She then refers to a study by Dr. Albert W. Wu, lead author and a general internist and professor of health policy and management at the Johns Hopkins Bloomberg School of Public Health in Baltimore published in the journal Health Affairs entitled Adding The Patient Perspective To Comparative Effectiveness Research. In this study, Wu et al argue for the inclusion of the patient’s perspective in comparative effectiveness research. What this involves is patient-reported outcomes. To illustrate the concept, Wu et al use this chart for patients with chronic obstructive pulmonary disease (COPD).

These sorts of measures are particularly appropriate for comparative effectiveness research (CER). For the reason, consider what CER is: basically CER compares existing treatment modalities already determined to be effective in prior clinical trials in order to determine which is more effective. Other important measures include cost-effectiveness. However, although some efforts go into assessing patient-reported quality of life outcomes of the sort listed above, all too often it’s hit-or-miss whether these sorts of measurements are included in clinical trials. One initiative that this article describes is the Patient-Centered Outcomes Research Institute, whose mandate is to:

• Establish an objective research agenda;
• Develop research methodological standards;
• Contract with eligible entities to conduct the research;
• Ensure transparency by requesting public input; and
• Disseminate the results to patients and healthcare providers.

Wu et al suggest that the PCORI can only realize its potential if it supports initiatives that integrate measures of patient experience into not just research but into routine clinical care. A number of possibilities are suggested, including how to integrate general and disease-specific tools into clinical trials in order to measure patient-reported outcomes. Also suggested are various means of integrating these tools not just into clinical research but into routine clinical care, including using them in administrative claims data, linking this data to electronic medical records, and even promoting the collection of such data as being required for reimbursement.

One problem I can perceive immediately in trying to use the PCORI is that it has no real power. In fact, the health insurance reform bill known as the Patient Protection and Affordable Care Act (PPACA), which mandated the creation of the Patient-Centered Outcomes Research Institute, provides no power to it. Indeed, its main charge is to assess “relative health outcomes, clinical effectiveness, and appropriateness” of different medical treatments, both by evaluating existing studies and conducting its own. Even given that huge mandate, the law also states that the PCORI does not have the power to mandate or even endorse coverage rules or reimbursement for any particular treatment. Indeed, so toothless is the PCORI, at least in its present form, that it has been disdainfully described as being like the UK’s NICE but without any teeth, which is all too true. Basically, the law says that Medicare may take the institute’s research into account when deciding what procedures it will cover, as long as the new research is not the sole justification and the agency allows for public input. Moreover, if the political reaction to the USPSTF’s revision of the guidelines for mammographic screening last year is any indication, if politicians don’t like a PCORI recommendation, you can be quite sure that they’ll behave similarly. After all the ranting about “rationing” that was used to attack the PPACA, it was not politically feasible to make the PCORI a government agency or to imbue it with any real authority.

Politics aside, let’s get back to the sorts of initiatives suggested by Wu et al. One that in particular interests me is the concept of using patient portals to collect this information. Patient portals are websites that offer a variety of services to patients, including secure e-mail communication with the clinician, the ability to schedule appointments and request prescription refills, as well as the opportunity to complete intake and other forms that used to be completed on paper in the office. The authors propose using such portals to collect patient-centered quality of life measurements and give an example of how this might be done in the case of a hypothetical breast cancer patient:

In one possible scenario, a woman with breast cancer is being followed by an oncologist who would like to know how she is doing on the chemotherapy regimen she is receiving. The oncologist logs on to PatientViewpoint.org, enters the patient’s number, and orders the BR-23 Breast Cancer–Specific Quality of Life Questionnaire for her to complete online before her next visit. The patient receives an e-mail notification to do this, logs on to PatientViewpoint.org, and completes the survey.

The patient’s results are automatically calculated and are made available both on the website and within the hospital’s electronic health record alongside all of her other laboratory test results. At the visit, the oncologist pulls up the results and asks the patient about an increase in her depression scores. It would also be possible to aggregate all of the patient’s questionnaire results with those of other patients receiving chemotherapy for similar breast cancer cases and to use these data to help compare the effectiveness of different regimens.

Dr. Wu’s site is currently only set up to accommodate breast and prostate cancer patients, but it could be expanded. There now exist a large number of tools like the BR-23 to assess quality of life, and, with what appears to be the nigh inevitable infiltration of the electronic medical record into medicine over the next several years, integrating such tools into routine clinical care should become increasingly easy and inexpensive. On the other hand, one problem with such tools is that clinicians are already buried in “information overload.” Whether they would actually read and use the results of such studies outside the context of clinical trials is not assured, at least not if there is no incentive to do so. If this sort of approach is going to work, the government and insurance companies are going to have to pony up. Another problem is that a lot of doctors don’t like this sort of measurement. They consider it unscientific and “squishy” or they don’t know what to do with the information. Whether these attitudes will change or not as CER becomes increasingly embedded in clinical research is impossible to say.

Dr. Wu’s article leads me to reflect upon two things. First, it’s important to remember that the reason these “softer,” “squishier” measures are becoming more important is precisely because SBM has been so successful. Diseases that were once fatal are now chronic. A prime example is HIV/AIDS. Back when I was in medical school, HIV was invariably fatal. AIDS patients died rapidly — and in most unpleasant ways. Thanks to SBM, which developed the cocktails of antiretroviral drugs, HIV/AIDS has become a chronic disease, so much so that babies born with HIV are now approaching adulthood. What this success means is that, although not completely, by and large mortality is no longer the be-all and end-all of HIV treatment. Now, we are seeing quality of life issues coming to the fore. The same is true for some cancers, and it’s certainly true for diabetes and heart disease. As Wu et al point out:

Patient-reported outcomes directly support the primary goal of much of health care: to improve health-related quality of life, particularly for people with chronic illnesses. No one can judge this better than the patient. For example, the main objective of hip replacement surgery is to reduce pain and improve the capacity to get around. The main goal of cataract extraction is to improve visual functioning—that is, the ability to perform activities that require eyesight, such as reading, walking without falls, and working on a computer.

In addition, there are often trade-offs between the length and quality of life. Important considerations are the side effects of treatment of HIV disease, the temporary diminution of functioning after coronary bypass surgery, or fatigue resulting from cancer chemotherapy. Even for life-saving treatments, this kind of trade-off can influence a patient’s decision making among alternative courses of care.

Once again, these decisions and the trade-offs patients decide to accept should be informed by the science. The options presented to the patient and their cost in terms of potential complications and impact on the patient’s ability to go about his daily activities and in essence live his life must be based on science. However, that does not mean that the final determination will always be purely based on estimates of efficacy. If the patient decides, for instance, that the survival advantage that chemotherapy will provide after her breast cancer surgery is not sufficient to be worth months of hair loss, fatigue, and the risk of heart damage, then that is her choice. The key is that we as clinicians must make sure that she has accurate, science-based information upon which to base that choice. Informed consent must be based on sound, scientifically verified information. Anything else, such as the sorts of “informed consent” advocated by “health freedom” groups is in reality misinformed consent. It is our responsibility as science-based practitioners to do our best to make sure that the treatments we offer our patients are based in science and that the information about the relative benefits, risks, and costs about these treatments is also based in science.

The second thing that comes to my mind is the complete contrast between the sorts of efforts that Wu et al are undertaking and what purveyors of unscientific so-called “complementary and alternative medicine” (CAM) do. SBM is, through CER, undertaking systematic measurements of quality of life measures, and the use of genetic tests that provide information about prognosis and predict response to therapy, making its first real steps towards truly “personalized” medicine. Yes, these steps are halting — stumbling at times, even — but they are steps towards the day when SBM can offer patients treatment options based on science and personalized to the characteristics of the biology of their disease that are unique to them, all while taking the patients’ own values and desires into account. Contrast that to so-called CAM, where “personalized medicine” basically means making it up as the practitioner goes along, and I think you’ll see what I mean. Whatever the deficiencies and faults of SBM (and it’s impossible not to concede that there are many), SBM is far closer to true “personalized medicine” than any CAM, and it is using CER to come even closer still. CAM has nothing to compare.

Attacks on science-based medicine (SBM) come in many forms. There are the loony forms that we see daily from the anti-vaccine movement, quackery promoters like Mike Adams and Joe Mercola, those who engage in “quackademic medicine,” and postmodernists who view science as “just another narrative,” as valid as any other or even view science- and evidence-based medicine as “microfascism.” Sometimes, these complaints come from self-proclaimed champions of evidence-based medicine (EBM) who, their self-characterization otherwise, show signs of having a bit of a soft spot for the ol’ woo. Then sometimes there are thoughtful, serious criticisms of some of the assumptions that underlie SBM.

The criticism I am about to address tries to be one of these but ultimately fails because it attacks a straw man version of SBM.

True, the criticism of SBM I’m about to address does come from someone named Steve Simon, who vocally supports EBM but doesn’t like the the criticism of EBM implicit in the very creation of the concept of SBM. Simon has even written a very good deconstruction of postmodern attacks on evidence-based medicine (EBM) himself, as well as quite a few other good discussions of medicine and statistics. Unfortunately, in his criticism, Simon appears to have completely missed the point about the difference between SBM and EBM. As a result, his criticisms of SBM wind up being mostly the application of a flamethrower to a Burning Man-sized straw man representing what he thinks SBM to be. It makes for a fun fireworks show but is ultimately misdirected, a lot of heat but little light. For a bit of background, Simon’s post first piqued my curiosity because of its title, Is there something better than Evidence Based Medicine out there? The other reason that it caught my attention was the extreme naiveté revealed in the arguments used. In fact, Simon’s naiveté reminds me very much of my very own naiveté about three years ago.

Here’s the point where I tell you a secret about the very creation of this blog. Shortly after Steve Novella invited me to join, the founding members of SBM engaged in several e-mail frank and free-wheeling exchanges about what the blog should be like, what topics we wanted to cover, and what our philosophy should be. One of these exchanges was about the very nature of SBM and how it is distinguished from EBM, the latter of which I viewed as the best way to practice medicine. During that exchange, I made arguments that, in retrospect, were eerily similar to the ones by Simon that I’m about to address right now. Oh, how epic these arguments were! In retrospect, I can but shake my head at my own extreme naiveté, which I now see mirrored in Simon’s criticism of SBM. Yes, I was converted, so to speak (if you’ll forgive the religious terminology), which is why I see in Simon’s article a lot of my former self, at least in terms of how I used to view evidence in medicine.

The main gist of Simon’s complaint comes right at the beginning of his article:

Someone asked me about a claim made on an interesting blog, Science Based Medicine. The blog claims that Science Based Medicine (SBM), that tries to draw a distinction between that practice and Evidence Based Medicine (EBM). SBM is better because “EBM, in a nutshell, ignores prior probability (unless there is no other available evidence and falls for the p-value fallacy; SBM does not.” Here’s what I wrote.

No. The gist of the science based medicine blog appears to be that we should not encourage research into medical therapies that have no plausible scientific mechanism. That’s quite a different message, in my opinion, that the message promoted by the p-value fallacy article by Goodman.

First off, Simon’s complaint makes me wonder if he actually read Dr. Atwood’s entire post. To show you what I mean, I present here the whole quote from Dr. Atwood in context:

EBM, in a nutshell, ignores prior probability† (unless there is no other available evidence) and falls for the “p-value fallacy”; SBM does not. Please don’t bicker about this if you haven’t read the links above and some of their own references, particularly the EBM Levels of Evidence scheme and two articles by Steven Goodman (here and here). Also, note that it is not necessary to agree with Ioannidis that “most published research findings are false” to agree with his assertion, quoted above, about what determines the probability that a research finding is true.

Simon, unfortunately, decides to bicker. In doing so, he builds a massive straw man. I’m going to jump ahead to the passage the most reveals Simon’s extreme naiveté:

No thoughtful practitioner of EBM, to my knowledge, has suggested that EBM ignore scientific mechanisms.

Talk about a “no true Scotsman” fallacy!

You know, about three years ago I can recall writing almost exactly the same thing in the aforementioned epic e-mail exchange arguing the very nature of EBM versus SBM. The problem, of course, is not that EBM completely ignores scientific mechanisms. That’s every bit as much of a straw man characterization of SBM as the characterization that Simon skewered of EBM being only about randomized clinical trials (RCTs). The problem with EBM is, rather, that it ranks basic science principles as being on either very lowest rung or the second lowest rung on the various hierarchies of evidence that EBM promulgates as the way to evaluate the reliability of scientific evidence to be used in deciding which therapies work. The most well-known of these is the that published by the Centre for Evidence-Based Medicine, but there are others. Eddie Lang, for instance, places basic research second from the bottom, just above anecdotal clinical experience of the sort favored by Dr. Jay Gordon (see Figure 2). Duke University doesn’t even really mention basic science; rather it appears to lump it together at the very bottom of the evidence pyramid under “background information.” When I first started to appreciate the difference between EBM and SBM, I basically had to be dragged, kicking and screaming, by Steve and Kimball, to look at these charts and realize that, yes, in the formal hierarchies of evidence used by the major centers for EBM, basic science and plausible scientific mechanisms do rank at or near the bottom. I didn’t want to accept that it was true. I really didn’t. I didn’t want to believe that SBM is not synonymous with EBM, which would be as it should be in an ideal world. Simon apparently doesn’t either:

Everybody seems to criticize EBM for an exclusive reliance on randomized clinical trials (RCTs). The blog uses the term “methodolatry” in this context. A group of nurses who advocate a post-modern philosophical approach to medical care also criticized EBM and used an even stronger term, micro-fascism, to describe the tendency of EBM to rely exclusively on RCTs.

But I have not seen any serious evidence of EBM relying exclusively on RCTs. That’s certainly not what David Sackett was proposing in the 1996 BMJ editorial “Evidence based medicine: what it is and what it isn’t”. Trish Greenhalgh elaborates on quite clearly in her book “How to Read a Paper: The Basics of Evidence Based Medicine” that EBM is much more than relying on the best clinical trial. There is, perhaps, too great a tendency for EBM proponents to rely on checklists, but that is an understandable and forgivable excess.

I must to admit to considerable puzzlement here. EBM lists randomized clinical trials (RCTs) and meta-analyses or systematic reviews of RCTs as being the highest form of evidence, yet Simon says he sees no serious evidence of EBM relying exclusively on RCTs. I suppose that’s true in a trivial sort of way, given that there are conditions and questions for which there are few or no good RCTs. When that is the case, one has no option but to rely on “lower” forms of evidence. However, the impetus behind EBM is to use RCTs wherever possible in order to decide which therapies are best. If that weren’t true, why elevate RCTs to the very top of the evidence hierarchy? Simon is basically misstating the the complaint anyway. We do not criticize EBM for an “exclusive” reliance on RCTs but rather for an overreliance on RCTs devoid of scientific context.

Simon then decides to try to turn the charge of “methodolatry,” or as revere once famously called it, the profane worship of the randomized clinical trial as the only valid method of investigation, against us.This misinterpretation of what SBM is leads Simon, after having accused SBM of leveling straw man attacks against EBM, to building up that aforementioned Burning Man-sized straw man himself, which he then begins to light on fire with gusto:

I would argue further that it is a form of methodolatry to insist on a plausible scientific mechanism as a pre-requisite for ANY research for a medical intervention. It should be a strong consideration, but we need to remember that many medical discoveries preceded the identification of a plausible scientific mechanism.

While this is mostly true, one might point out that, once the mechanisms behind such discoveries were identified, all of them had a degree of plausibility in that they did not require the overthrow of huge swaths of well-settled science in order to be accepted as valid. Let’s take the example of homeopathy. I use homeopathy a lot because it is, quite literally, water and because its proposed mechanism of action goes against huge swaths of science that has been well-characterized for centuries. I’m not just talking one scientific discipline, either. For homeopathy to be true, much of what we currently understand about physics, chemistry, and biology would have to be, as I am wont to say, not just wrong, but spectacularly wrong. That is more than just lacking prior plausibility. It’s about as close to being impossible as one can imagine in science. Now, I suppose there is a possibility that scientists could be spectacularly wrong about so much settled science at once. If they are, however, it would take compelling evidence on the order of the mass of evidence that supports the impossibility of homeopathy to make that possibility worth taking seriously. Extraordinary claims require extraordinary evidence. RCTs showing barely statistically significant effects do not constitute extraordinary evidence, given that chance alone will guarantee that some RCTs will be positive even in the absence of an effect and the biases and deficiencies even in RCTs. Kimball explains this concept quite well:

When this sort of evidence [the abundant basic science evidence demonstrating homeopathy to be incredibly implausible] is weighed against the equivocal clinical trial literature, it is abundantly clear that homeopathic “remedies” have no specific, biological effects. Yet EBM relegates such evidence to “Level 5”: the lowest in the scheme. How persuasive is the evidence that EBM dismisses? The “infinitesimals” claim alone is the equivalent of a proposal for a perpetual motion machine. The same medical academics who call for more studies of homeopathy would be embarrassed, one hopes, to be found insisting upon “studies” of perpetual motion machines. Basic chemistry is still a prerequisite for medical school, as far as I’m aware.

Yes, Simon is indeed tearing down a straw man. As Kimball himself would no doubt agree, even the most hardcore SBM aficianado does not insist on a plausible scientific mechanism as a “pre-requisite” for “ANY” research, as Simon claims. Rather, what we insist on is that the range of potential mechanisms proposed do not require breaking the laws of physics or that there be highly compelling evidence that the therapy under study actually has some sort of effect sufficient to make us doubt our understanding of the biology involved.

Simon then appeals to there being some sort of “societal value” to test interventions that are widely used in society even when those interventions have no plausible mechanism. I might agree with him, except for two considerations. First, no amount of studies will convince, for example, homeopaths that homeopathy doesn’t work. Witness Dana Ullman if you don’t believe me. Second, research funds are scarce and likely to become even more so over the next few years. From a societal perspective, it’s very hard to justify allocating scarce research dollars to the study of incredibly implausible therapies like homeopathy, reiki, or therapeutic touch. (After all, reiki is nothing more than faith healing based on Eastern mystic religious beliefs rather than Christianity.) Given that, for the foreseeable future, research funding will be a zero sum game, it would be incredibly irresponsible to allocate funds to studies of magic and fairy dust like homeopathy, knowing that those are funds that won’t be going to treatment modalities that might actually work.

When it all comes down to it, I think that Simon is, as I was, in denial. When confronted with the whole concept of SBM compared to EBM, I denied what I didn’t want to believe. To me, it seemed so utterly obvious that the scientific plausibility of the hypothesis under study has to be taken into account in evaluating the evidence. I just couldn’t imagine that any system of evaluating evidence could be otherwise; it made no sense to me. So I imposed this common-sense view on EBM, and I rather suspect that many other advocates of EBM like Simon labor under the same delusion I did. The problem is, though, that critics of EBM are basically correct on this score. Still, realizing it or admitting it did not come easy. For me to accept that EBM had a blind spot when it came to basic science, it took having my face rubbed in unethical and scientifically dubious trials like that of the Gonzalez therapy for pancreatic cancer or chelation therapy for cardiovascular disease. Let’s put it this way. To be willing to waste money studying something that is nothing but water and has as its “scientific basis” a hypothesis that is the equivalent of claiming that a perpetual motion machine can be constructed tells me that basic science basically means close to nothing. Ditto wasting money on studying a therapy whose major component is coffee enemas used to treat a deadly cancer. Simon cheekily suggests at the end of his post that “maybe we should distinguish between EBM and PIEBM (poorly Implemented Evidence Based Medicine). The problem is, trials of therapies like the Gonzalez regimen, homeopathy, and reiki are a feature of, not a bug in EBM. In fact, I challenge Simon to provide a rationale under EBM as it is currently constituted to justify not having to do a clinical of these therapies. There is none.

I realize that others have said it before here (and probably said it better than I), but we at SBM are not hostile to EBM at all. Rather, we view EBM as incomplete, a subset of SBM. It’s also too easily corrupted to provide an air of scientific legitimacy to fairy dust like homeopathy and reiki. These problems, we argue, can be ameliorated by expanding EBM into SBM. Personally, I suspect that the originators of EBM, as I do (and, I suspect, Simon does), never thought of the possibility of EBM being applied to hypotheses as awe-inspiringly implausible as those of CAM. It simply never occurred to them; they probably assumed that any hypothesis that reaches a clinical trial stage must have good preclinical (i.e., basic science) evidence to support its efficacy. But we know now that this isn’t the case. I can’t speak for everyone else here, but after agreeing with Kimball that EBM ought to be synonymous with SBM I also express the hope that one day there will be no distinction between SBM and EBM. Unfortunately, we aren’t there yet.

NOTE: There will be one more post later today; so don’t go away just yet.