THOMAS V. HOLOHAN, M.D., FACP
CHIEF, PATIENT CARE SERVICES
VETERANS HEALTH ADMINISTRATION
DEPARTMENT OF VETERANS AFFAIRS
COMMITTEE ON GOVERNMENT REFORM
U. S. HOUSE OF REPRESENTATIVES
February 24, 1999
Public interest in, and use of, unconventional, complementary, or alternative medical practices (CAM) is increasing. Contemporary observers have offered many reasons for the phenomenon, including dissatisfaction with limitations of conventional medicine, desire for treatment directed toward the "whole person", distrust of drugs and side effects, and awareness of linkages between disease and emotional and lifestyle factors. Added to this is the understandable frustration and search for "cure" on the part of patients afflicted with chronic or serious disorders which have not responded well to available therapy. Other factors likely include the reduction in time physicians are able or willing to spend with individual patients, as productivity concerns in medical practice assume ever greater importance.
Several recent publications have indicated that up to $27 billion are spent each year on CAM, and that a third of persons surveyed have used at least one modality that may be defined as "alternative". In 1993, the National Institutes of Health (NIH) established the Office of Alternative Medicine (OAM) to facilitate the evaluation of alternative therapies. OAM currently has an annual budget of $50 million. Interest on the part of the conventional medical community is evidenced by the fact that an entire recent issue of the Journal of the American Medical Association was devoted to CAM.
The heterogeneous treatments employed by the many schools of alternative practices cannot be encompassed within a single paradigm. When we refer to CAM, we are using a nonspecific term to describe a variable group of practices which include the use of botanical therapies, mind-body interactive approaches, nutritional interventions, various physical or manual therapies, some chemical entities or biologics which have not been evaluated by the Food and Drug Administration (FDA) on the bases of clinical trials, and similar modalities. Still others are rooted in cultural or religious belief systems that may be foreign to contemporary Western society. Their single commonality is that their underlying philosophies and the manner in which their agents and techniques are employed diverge from our mainstream medical principles and practices. However, that separation is not distinct and absolute, as we shall later discuss.
VA recently awarded a contract to survey alternative practices as they might apply to our system of healthcare. Elements of the report are to include: the current state of use and familiarity with alternative practice within our system; a review of VA’s patient database and a literature-based assessment of the applicability of alternative practices to VA patients; an evaluation of the state of alternative practices in the private sector; and an appraisal of the cost-effectiveness of alternative practices. At present, the report has not been completed. We do, however, have some preliminary information regarding the state of alternative practices in VA facilities. While knowledge, and even awareness, of alternative practices varies widely among VA providers and facilities, most facilities provide some such treatments. These have generally included modalities such as acupuncture, dietary therapy, yoga, biofeedback, relaxation, music therapy, and Native American therapies (e.g., "sweat lodges"). These practices usually reflect the presence of practitioner advocates and managerial willingness to accept the implementation of those programs. Preliminary data also show that most facility management teams were reported as pragmatically oriented, and described as having no biases for or against alternative treatments. The main concerns VA personnel expressed related to the highly variable training and credentialing of practitioners, the lack of sound scientific evidence supporting use of many alternative therapies, and uneasiness about the budgetary impact of alternative practice in an environment of constrained resources.
We would call the Committee’s attention to the fact that many practices often considered as alternative medicine have been, or are, also used in "conventional" medicine. For example, physical and manual treatments (such as massage therapy) significantly overlap with modalities widely used in the current practice of physical medicine and rehabilitation. Many nutritional therapy models have counterparts in allopathic medicine, such as the use of hyperalimentation as an adjunct to conventional cancer treatment. In addition, the mainstream medical literature contains numerous studies of vitamin C supplementation, zinc for treatment of viral respiratory infections, and the use of vitamin E as an antioxidant, among many others. Many drugs widely used by conventional practitioners are, in fact, botanical preparations which have been evaluated in clinical trials and approved for marketing by FDA. These include vincristine (from the periwinkle plant), digitalis (foxglove), and taxol (originally extracted from Pacific Yew tree bark). Many metallic elements have been employed by conventional practitioners including cis-platinum (for cancer), mercury (syphilis), magnesium (eclampsia of pregnancy), and others. Indeed, maggots have been sometimes used in lieu of surgery to successfully treat putrefying wounds and remove dead tissue in certain infections, based on observations made in the First World War that maggot infested wounds rarely became gangrenous.
Moreover, the mind-body interaction is not a phenomenon only recognized by alternative practitioners. There is a long history in medicine of appreciation of those mutual effects. A significant body of mainstream research – both old and new - has provided data that indicate the prognosis for coronary disease patients with depression is worse than for those without; that breast cancer patients who attend a support group have measurably better outcomes than those who do not; that single male cancer patients have worse prognoses than married patients; that cancer patients who are optimistic and who have stronger religious feelings tend to fare better than patients not so inclined. Indeed, it is no accident that the VA’s Chaplains’ Service is an integral part of our Office of Patient Care Services, and not attached to administrative programs. We see their ministry as part and parcel of clinical care. Further, we are currently developing a formal VA-wide strategy to fully integrate mental health and medical services throughout our system of care, based upon our belief that all diseases or disorders exist within an individual who is the unit of care, and that one treats a whole patient, not simply an organ system.
An additional example of the appreciation of mind-body interactions includes our growing understanding that the so-called placebo effect is real, and is potent. We know that patients often experience what they expect to experience. The Physician’s Desk Reference (PDR) compendium of FDA-approved drug labeling includes data on adverse effects reported by patients during trials in which drugs were compared with placebo. For pilocarpine (used to treat salivary gland dysfunction), nine percent of patients given a placebo reported increased sweating, and eight percent reported headache. For diltiazem (used to treat hypertension), of the patients given placebo and not the active drug, five percent reported headache, three percent dizziness, and 2.3% had abnormalities of their electrocardiogram (versus 1.6% of those treated with the active drug). In controlled trials of interferon, nearly 20% of placebo-treated patients reported fever, and 15% malaise. Suggestion is powerful, as has repeatedly been demonstrated since the 19th century by hypnotherapy.
At the same time, one cannot ignore the portion of the spectrum of alternative or unconventional care that is truly extreme. There are a number of therapies whose advocates have proposed unduly optimistic claims and whose treatments have been unequivocally demonstrated as ineffective and often harmful. Such treatments include immunoaugmentative therapy for cancer, offered in the Bahamas for many years. In this genre are also administrations of coffee enemas as cancer therapy, and the promotion and sale of laetrile, the latter having been legalized by the legislatures of 24 states despite the opposition of major medical professional societies, the NIH, FDA, and the American Cancer Society. Indeed, in the early 1980’s, a committee chaired by the late Congressman Claude Pepper published a lengthy, sobering yet remarkable report on the wide variety of ineffective or fraudulent treatments being sold to the public. We do not mean to imply that all unconventional treatments are ipso facto suspect. The critical point to be made is that the advocate of any treatment, conventional or unconventional, allopathic or homeopathic, surgical or psychological, has an ethical and moral obligation to provide high quality, convincing evidence that satisfactorily demonstrates the treatment is effective and that the benefit is clearly proportionate to the risk. This meets the intent of the Kefauver Commission which in 1962 mandated that pharmaceuticals must be shown by their manufacturers to be effective for their intended purpose.
Claims asserting that scientific research standards are inappropriate or irrelevant to alternative practices are flawed. Science is neither an entity nor a belief system, but merely a disciplined method of investigation. It is a tool that enables one to formulate and then test a hypothesis; as such its applicability is virtually universal. For science, data rather than opinion are determinative. It is the only instrument that permits a mathematically sound statement of the probability that a particular cause will result in a specific effect. A casual and unsystematic linkage of cause and effect is too often erroneous in biological systems. For those reasons, prudent clinicians are loath to accept anecdotal evidence, a few cases, or subjective judgements as proof of efficacy.
Treatments should be offered patients only after there has been provided convincing evidence of effectiveness and acceptable adverse effects. This is no less true of conventional than of alternative practices. Ignoring this tenet does not serve patients well. During the 1960’s an operation to prevent stroke by connecting the external carotid to the internal carotid artery (EC-IC bypass) was developed and widely applied based upon a number of reports of small series of patients. Because of the outspoken skepticism of some in the medical community, and despite the prestige of the advocates, the NIH sponsored an international, multicenter prospective randomized trial comparing the surgery with medical therapy. The results, published in 1985, demonstrated that not only did the procedure fail to prevent stroke, but also that operated patients died sooner and in greater proportion than did patients treated medically. Clinical research on pharmaceutical preparations is required by statute. Current federal law requires that the FDA approve drugs only on the basis of well-controlled clinical trials; similar requirements exist for some medical devices (those implanted or used for life-threatening conditions). There are no such standards for surgical procedures or for alternative medical therapies.
VA believes that we have a serious responsibility to demand evidence of benefit and safety for treatments we provide to veteran patients, and we have invested considerable resources to that end. We have recently established a Technology Recommendations Panel, to add to our Technology Assessment Program (a component of our Office of Research and Development), with the view of aiding such evaluations. We also believe that expert opinion or beliefs do not constitute scientific evidence; anecdotes, case reports, and studies of small series of patients represent the weakest forms of evidence and best serve to provide a hypothesis which may be tested in a well-designed clinical trial. While such positions are not in accord with the opinions of some in both the conventional and alternative medical fields, they are ratified by how most of us, tacitly or overtly, rely on the scientific method in our daily lives. When we step into an airplane, we are aware of our dependence upon the research and experimentation underlying the engineers’ theories, and upon the repeated testing of both the materials and the design of the airframe, engines, and controls. We expect the Federal Aviation Administration to provide serious oversight of aircraft manufacture, and that design and construction will not rely solely on opinions or beliefs but on the application of scientific investigation. Likewise, we expect items such as our automobile brakes and tires will be the products of materials research, carefully controlled testing, and standardized components and construction meeting standards of the Department of Transportation. In both of those venues, we also expect that the production will be accomplished by technical experts qualified by training and experience and certified as competent by reliable and responsible authorities.
It is dangerous to assume that so-called "natural" or non-pharmaceutical products are by nature safe. This month, the FDA required manufacturers to recall gamma butyrolactone (GBL), a dietary supplement widely sold in health food stores, gymnasiums, and over the Internet. Its primary commercial use is as a solvent in paint thinners and nail polish remover. Use of the product had caused comas, seizures, dramatic slowing of heart rates, respiratory arrest, and death. Those occurrences mimic those of a chemical banned in 1991, gamma hydroxybutyrate, which had been linked to deaths and use in "date rape" cases. However, after ingestion, GBL is converted in the body to the banned gamma hydroxybutyrate. The Council for Responsible Nutrition, a trade association representing supplement manufacturers, stated that GBL should never have been marketed because it had clearly been shown to be dangerous to humans and animals. Undoubtedly, most consumers made ill or killed by GBL assumed that its production and sale implied at least some research demonstrating safety, if not effectiveness. Sadly, they were mistaken.
At the present time, there exists a paucity of rigorous, reliable, and valid clinical trial data supporting many alternative interventions. Indeed, that was identified as a major concern of VA personnel in preliminary information received from our contractor. We believe such evidence is critical in our determination as to the role CAM may play in the care of our patients. To that end, the VA research and development program will continue to fund scientifically meritorious investigator-initiated research related to alternative practices at all levels, from basic biomedical trials through multicenter clinical trials, and health services outcomes studies. We will continue to encourage health professionals to submit research proposals in their areas of interest and expertise, as well as support public and private sector collaborations to pursue research on high priority health care needs of veterans. Whenever appropriate, such collaborations will include alternative or unconventional medical practices.
Additionally, inconsistent CAM provider credentialing, licensing, and regulation pose serious problems in the utilization of those practitioners and techniques. Acupuncturists are licensed in 35 states; massage therapists are licensed in 27, naturopaths in 14, and homeopaths in four states. A naturopathy-related Internet site states that "Certified Naturopaths may complete a four-year program of study or they may be someone with nothing more than a diploma from a ‘diploma mill’ or a correspondence school." Similar problems undoubtedly exist for other alternative disciplines. VA has set high standards for practitioner training, education, credentialing, and certification. All newly hired VA physicians must be licensed and board certified; all advanced practice nurses must possess licensure and a graduate degree, and all registered nurses licensure and a Bachelor’s degree. We believe that all providers in VA should meet appropriately equivalent and comparable standards, irrespective of their practice focus.
In closing, VA is investigating alternative medical practices, and is presently gathering data addressing the interest of our clinicians and extent of alternative medicine use in our system. We expect to be reviewing information developed from the literature base for alternative practices, the appropriateness of employment for our population, and information on cost and cost-effectiveness. VA expects that any treatment offered to veteran patients – conventional or alternative – and provided outside the context of a clinical trial will be chosen upon the basis of objective evidence sufficient to permit the conclusion that it is both safe and effective.