Editor’s Note: Over the next several months, Mad in America is publishing a serialized version of Les Ruthven’s book, Much of U.S. Healthcare is Broken: How to Fix It. In this blog, he addresses off-label prescribing as well as physicians’ anti-science pushback against the use of well-conducted clinical trials. Each Monday, a new section of the book is published, and all chapters are archived here.
Training in the Physical Sciences vs. Training in Science
Most people, including many healthcare providers, confuse the above two similar sounding terms, but the two terms are quite different and the distinction is central to my thesis that the major key to affordable healthcare is to improve the quality and appropriateness of current healthcare through application of the scientific method to determine questions of efficacy and safety.
First, physicians in their undergraduate courses and medical school take perhaps 90 percent of their courses in the biological and physical sciences (as it should be) but this is not training in how to conduct sound scientific research or to evaluate the research of others. The latter occurs at the PhD graduate school level in many of the disciplines in the biological, physical, and social sciences. Some few physicians have both the MD and PhD and are trained in both, with the latter focusing on how to conduct research and review the research of others.
This distinction is important because the MD is basically a practitioner degree (which is appropriate for the practitioner) and not a research degree, which unfortunately has shaped the quality of healthcare in the 20th century and beyond. This has resulted in both good and poor healthcare. Both practitioners and health researchers shape the kind of healthcare delivered to patients but, unfortunately, practitioner influence has been substantially greater than the scientific health research influence in shaping clinical practice. As a result, belief and anecdotal “evidence” rather than science has shaped much of the poor-quality healthcare that we have today.
Moreover, the non-scientist MD practitioner is vulnerable to naively accepting the research of others as truth even when the study does not meet acceptable scientific standards or contain adequate scientific controls. One would hope that the FDA clinical trials would be free of bias and scientific error in their research design, and that practitioners and payers of healthcare could depend on FDA approval to guide sound clinical practice, but, unfortunately, we will find that this FDA “gold standard” is all too often not valid in a number of drug and non-drug treatments.
I will point out many examples of poor-quality healthcare research in all sections of healthcare (not only in mental health but also “medical” care as well), but I find in my review of research that industry-sponsored research (e.g., pharmaceutical companies and medical device companies) are especially prone to producing very poor quality and biased research. Having reviewed many of these industry-sponsored studies, I believe substantially better and more cost-effective healthcare would result if all physicians completely ignored such propaganda acting under the guise of science.
Many popular therapies of today—often therapies that generate high revenues—are based on research studies that do not support the therapy at all and are in fact contradicted by the research data in the study!
I can think of no better way to achieve affordable healthcare than by insisting that a particular drug or therapy is only reimbursable if the treatment has been proven to be substantially effective (and safe) by sound research and not just because practitioners believe the treatment is effective or that the treatment is FDA-approved.
Currently, health insurance companies and Managed Care Organizations (MCOs) are allowing payment for health services that are either not supported by the best science available or, many times, payment is allowed which is contrary to the available scientific research. Typically, health insurance companies and their MCOs on paper will not authorize payment for drugs or treatments that are said to be “experimental” in nature but, despite this spoken standard, we will find that in many of these pages these insurance entities do in fact authorize payment for a great deal of healthcare that is really “experimental” and mostly unproven or wrong.
A prime example is, as I have mentioned, the off-label prescribing of an FDA-approved drug for one health problem for a different health problem, a practice devoid of any evidence to support it. I am not sure of this but I suspect that in everyday clinical practice more drugs are prescribed off-label by your physician than FDA approved drugs! Let’s insist that off-label practices must pass scientific standards for efficacy and safety and, until then, do not authorize payment for such health services that are of a speculative and unproven nature. I am sure that some off-label drug uses are indeed effective for another health problem, but we have no way of knowing this without submitting the drug-therapy to a scientific test in the form of a randomized, placebo-controlled trial.
One must accept the fact that all drugs have unwanted and adverse side effects and that the benefits of the drugs must be considered with the risks involved in taking the drug. For example, uncontrolled grand mal seizures are a very major, even life-threatening health problem in many cases. The health and quality of life benefits of an effective drug to treat them are worth the risk of very serious adverse side effects such as a stroke or even death. Considering the severity of the health problem such as seizures, the very serious risks of the drug are indeed justified. I criticize the FDA for not insisting on a higher standard of efficacy for drug (or medical device) approval, but I am also critical of some FDA practices in dealing with safety issues. Despite the very serious adverse side effects of anti-seizure drugs, these risks are warranted but should these necessary high-risk drugs be also FDA-approved for more minor and less serious health problems? I don’t believe so but such a practice is unfortunately not an isolated event and patients are not informed that a high-risk drug for a possible life-threatening disease is being offered to treat a more minor health problem!
When drugs with very severe side effects are prescribed for more minor health problems, these patients are not informed about the high risks involved with the proposed medication. This terrible practice of withholding vital information from the patient occurs in dozens of healthcare areas and is a practice that must be stopped. As an example of this practice a neurologist offered me an off-label seizure medication for my left leg nerve pain, but I declined the offer because I like my brain too much!
While I have been finding fault with a number of FDA practices, I would like to point out that FDA approval should not only deal with the treatment’s efficacy and safety but, in these days of high healthcare costs, the approval process should take into consideration the cost of the treatment in making approval decisions, including the availability of effective low-cost alternatives! The FDA approval process seems to have no concerns about the relative cost of two or more approved treatments for the same health problems when the cost of one improved treatment may be 200 times the cost of the other approved treatment!
We need an entirely new entity that will make payment decisions not only on the basis that the treatment must be of proven effectiveness and relatively safe but also, when there are several treatments for the same health problem, payment should be restricted to the most cost-effective treatment of those available. For example, insomnia can be treated very competently and cost effectively by one specialist and there is no proof offered that the disorder requires treatment in a sleep center with one or two overnight stays in the hospital or clinic with EEG monitoring of sleep and non-sleep segments.
I have noticed in recent years that necessary scientific standards such as FDA randomized, placebo-controlled, drug and medical device clinical trials have been subject to increasing attack by much of the medical community, as mentioned previously. As you will read, I, too, am very critical of these same FDA clinical trials because they are often biased in favor of finding a treatment effect (and are therefore granted approval) even if the treatment superiority over inert placebo is clinically negligible!
I argue for eliminating the biases in the FDA clinical trials that favor a drug effect but not eliminating these placebo-controlled trials, which an increasing number in the medical community recommend because these physicians are aware that in these trials the superiority of the drug over placebo is far from impressive. If we eliminate scientifically sound clinical trials (which many physicians recommend) we are left with clinical opinion to determine efficacy and safety.
I think questioning the advisability and worth of such FDA randomized placebo-controlled trials by health practitioners reflects a growing anti-science movement in medicine and perhaps the broader culture as well. National Public Radio (NPR) reported a recent research study in which airline passengers completed a survey asking who would be interested in participating in a randomized, parachute/placebo-controlled (no parachute) trial of jumping from an airplane at cruise altitude!
This was an actual series of published survey studies (I have not as yet obtained the reference) and the obvious intent was to ridicule one of our best and at times only means of evaluating the true efficacy of any treatment. Many individual physicians (psychiatrists and non-psychiatrists alike) have denied the necessity of such clinical trials “when we all know the treatment in question is effective.”
Many physicians have argued that these placebo-controlled trials are unethical in knowingly withholding an effective treatment such as an antidepressant drug from depressed clinical trial patients in the placebo group! However, we are not told by these individuals what other scientific tests can we use to determine treatment efficacy if FDA clinical type trials are excluded?
I think their only real fallback position is “Because the doctor from his/her clinical experience knows and says the treatment is effective.” Without randomized, placebo-controlled trials we are back to pre-20th century physician “proof” such as “I gave this snake oil to Mary and it cured her rheumatism,” which unfortunately continues to be the source of large segments of poor medical care today. There are several dozens of prescription drugs and medical procedures (see Chapters 2, 3, 4, and 5) that continue to be delivered in which there are serious questions about either their efficacy or safety (e.g., whether or not some cancer drugs cause damage to the brain).
There are opinions on both sides with regard to questions of efficacy and safety of these drugs and medical procedures; the obvious answer is to apply the scientific method to discover the truth about these matters, but I have found that rarely do physicians turn to science as the final arbiter.
As we will observe, there are dozens of popular treatments and procedures that raise questions about efficacy and safety; some of these discussions have been going on for ten years or more, yet there seems little or no interest in applying the scientific method to determine the truth. In many of these questionable practices, there are sound studies proving the lack of efficacy and safety, but somehow these studies are “buried” and have no effect on current clinical practice. This must change.
Mad in America hosts blogs by a diverse group of writers. These posts are designed to serve as a public forum for a discussion—broadly speaking—of psychiatry and its treatments. The opinions expressed are the writers’ own.
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