Randomized Controlled Trials: The Golden Calf?

Practitioners of the Homeopathic Art of Healing are cautioned to stay clear from hypotheses concerning life sciences.(1)Hahnemann S: Footnote to the 1st Aphorism – Organon of Medical Art, 6e, Ed. O’Reilly WB, Birdcage, 1996 Indeed, our understanding of life, health and disease have evolved from a simple homeostatic model, to a resilient system and now we are moving towards a more comprehensive complex-adaptive model. This means that as Healers, we must accept and respect the cyclic nature of growth, adaption, transformation, and, in the end, collapse.(2)Holling, C. S. 2004. From complex regions to complex worlds. Ecology and Society 9(1): 11. Disease is not an external entity that is to be destroyed. It is an expression of the innovation and adaptation of the Life Force of an individual in distress.

Complexity and Life Systems

Complexity in any system results from the inter-relationship, inter-action and inter-connectedness of elements within a system and between a system and its environment. Only in recent times are we learning about the interactions and inter-relationships between ecology, weather, cultural attitudes, immunology, evolution and a whole slew of other factors. The complex-adaptive system is characterized by a state of paradox – a dance of stability and instability, a play between competition and cooperation, and coexistence of order and disorder. In other words, we must move away from the comfort zone of inadequate and simplistic cause-and-effect theorems.

From Art to Science

The practice of healing has undergone a sea change over the centuries. Once considered an Art, it is now firmly in the domain of Science. Indeed, in the past two centuries or so, science has expanded our knowledge of the body and pathological conditions that occur in disease. However, in practice, the person-in-disease does not respond to treatment the way the physicians want or expect.

Now consider this. On the one hand we have a silent killer, known as iatrogenesis, or death-by-medicine,(3)Null Gary PhD, Dean C MD, et al: Death by Medicine, Life Extension Magazine March 2004 which is the third leading cause of death in treatment of disease. On the other hand we have an even more embarrassing situation: oftentimes patients will respond to a placebo, a non-medicinal sugar pill, which should logically produce no physiological effects at all! Thus, a truly rational therapeutic model still eludes us. Simply put, we have to accept that medicines can kill while placebos can cure.

Randomized Controlled Trial: The Golden Calf or A Gold Standard?

To overcome this dichotomous situation, researchers have evolved a system of evaluating a treatment, medicinal, surgical or otherwise, known as the Double-Blind Randomized Controlled Trial (RCT).

Briefly, the RCT procedure is conducted as follows:

  1. A number of similar people are randomly assigned to two (or more) groups to test a specific drug or treatment
  2. One group (the experimental group) receives the treatment being tested
  3. The other (the comparison or control group) receives an alternative treatment, a dummy treatment (placebo) or no treatment at all
  4. The groups are followed-up to see how effective the experimental treatment was
  5. Outcomes are measured at specific times and any difference in response between the groups is assessed statistically
  6. The trial is deemed double-blind because neither the participants nor the evaluators know who is getting the treatment and who is being given the placebo

RCTs are claimed to be “true experiments” conducted with scientific rigor, and are considered to be the gold standard and demonstrate a cause-and-effect relationship between an intervention and an outcome. RCT are the bedrock on which present day clinical practice of Evidence Based Medicine (EBM) is supposed to rest. However, it is being increasinly accepted that there are some fundamental shortcomings in the assumptions on whic RCTs are based.(4)Ventegodt S, Andersen NJ, et al: Evidence-Based Medicine: Four Fundamental Problems with the Randomized Clinical Trial (RCT) used to document chemical medicine. International Journal of  Adolescent Medicine & Health. 2009 Oct-Dec;21(4):485-96

How does it work out in the clinic?

Lancet, the world’s leading peer-reviewed medical journal admits that RCTs most often lack evidence that the treatment under investigation will prove of any use to a real patient seen by a doctor in the clinic.(5) Rothwell PM: External validity of randomized controlled trials: “To whom do the results of this trial apply?”. Lancet 2005; 365: 82–93 Indeed, RCTs have been criticized a lot in recent times. It seems a community of researchers have fallen prey to  a ritualistic worship of the metaphorical Golden Calf of RCT. Loes Knappen of McGill University, Canada states that we have reached a stage where, “The legitimacy of EBM relies neither on experts nor numbers, but on distinct procedures for handling (non) Evidence, reflecting its ‘regulatory objectivity’”.(6)Knappen L: Being evidence based in the absence of evidence: The management of non-evidence in guideline development. Social Studies of Science October 2013, vol. 43 no. 5 pp. 681-706

In the latest edition of a standard textbook on RCTs, authors Jadad and Enkin admonish us “to stop worshiping the randomized controlled trial as if it were a talisman that would guarantee objectivity”.(7) Jadad AR & Enkin MW: Randomized Controlled Trials: Questions, Answers and Musings, second edition, BMJ Books/Blackwell Publishing; 2007 The authors point out that RCTs do a good job to solve simple or even complicated problems, but have very limited use in complex problems. They advocate a multi-disciplinary approach. To cite an example, raising a child and treating dementia would hardly lend itself to RCTs.

In the course of his research, another respected researcher, Austin Bradford Hill, who authored Principles of Medical Statistics, lamented that the influence of statistical method (read RCT) on clinical practice has reduced research from one of assured certainty to one merely of modest advantage. He said, “At its best the RCT shows what can be accomplished with a medicine under careful observation and certain restricted conditions. The same results will not invariably or necessarily be observed when the medicine passes into general use”.(8)Quoted by Horton R: Common sense and figures: the rhetoric of validity in medicine – Bradford Hill Memorial Lecture 1999. Statistics in Medicine, 2000 Dec 15;19(23):3149-64.

So what do RCT do?

To be fair, RCTs do answer small questions that are only part of the complex-adaptive puzzle. Qualitative data pertaining to feelings of well-being, impact of a treatment on social life, using many interventions together to affect rapid healing, are but a few examples that point to the shortcomings of RCTs. RCTs in their present avatar are data driven, so subjective feelings have little influence in measuring outcomes.

Another area of research that is often ignored are “Single-Case Experiments”.  This is another important source of evidence that is most often overlooked.(9) Kazdin, A.E : Single-Case Research Designs: Methods for Clinical and Applied Settings, 2nd edition. New York: Oxford University Press, 2010 In contrast to RCTs, which involve many subjects and few observations, single-case designs involve many observations but often few subjects.

Is Homeopathy any different?

Every practicing homeopath has had to bear the insults of the worshipers of the Golden Calf who state that Homeopathy does poorly in RCTs and that its remedies are no more than placebos. However, Homeopathy fairs no better or worse in RCTs than conventional medicine. By the end of 2010 RCTs of homeopathy 41% had a balance of positive evidence, 7% had a balance of negative evidence, and for 52% no conclusions could be drawn either way(10)http://twinhealers.in/homeopathy. RCTs of conventional medicine, indicate that 44% of the reviews concluded the interventions studied were likely to be beneficial (positive), 7% concluded that the interventions were likely to be harmful (negative), and 49% reported that the evidence was non-conclusive(11)El Dib RP, Atallah AN, Andriolo RB: Mapping the Cochrane evidence for decision making in health care. Journal of Evaluation in Clinical Practice 2007;13:689–692.

Sadly, it is not realized that in the past two decades Homeopathy has consistently met the standards of RCT protocols, even in healing conditions that are considered “incurable” by modern medicine. And it does so without increasing virus and bacterial resistance while also improving immunity, mental and psychological well-being, raising happiness and most of all, doing no harm. At the same time homeopaths are open and inclusive as they realize that health and sickness are a part of a complex-adaptive system and only an integrative approach will help to heal.

Most critics of Homeopathy fail to realize that our Materia Medica  has been collated over more than two centuries after each remedy has been tested for producing symptoms in persons-in-health and have been confirmed in the clinic to heal persons-in-disease.

Dr. Bernard Lown, renowned cardiologist and healer echoes the sentiment of Hahnemann when he states that, “The real crisis in medicine today is not about economics, insurance, or managed care—it’s about the loss of the fundamental human relationship between doctor and patient. The art of healing does not mean abandoning the spectacular advances of modern science, but rather incorporating them into a sensitive, humane, enlightened approach to medical care”.(12)Lown, Bernard MD: The Lost Art of Healing, Balantine Books, 1996

References   [ + ]

1. Hahnemann S: Footnote to the 1st Aphorism – Organon of Medical Art, 6e, Ed. O’Reilly WB, Birdcage, 1996
2. Holling, C. S. 2004. From complex regions to complex worlds. Ecology and Society 9(1): 11.
3. Null Gary PhD, Dean C MD, et al: Death by Medicine, Life Extension Magazine March 2004
4. Ventegodt S, Andersen NJ, et al: Evidence-Based Medicine: Four Fundamental Problems with the Randomized Clinical Trial (RCT) used to document chemical medicine. International Journal of  Adolescent Medicine & Health. 2009 Oct-Dec;21(4):485-96
5. Rothwell PM: External validity of randomized controlled trials: “To whom do the results of this trial apply?”. Lancet 2005; 365: 82–93
6. Knappen L: Being evidence based in the absence of evidence: The management of non-evidence in guideline development. Social Studies of Science October 2013, vol. 43 no. 5 pp. 681-706
7. Jadad AR & Enkin MW: Randomized Controlled Trials: Questions, Answers and Musings, second edition, BMJ Books/Blackwell Publishing; 2007
8. Quoted by Horton R: Common sense and figures: the rhetoric of validity in medicine – Bradford Hill Memorial Lecture 1999. Statistics in Medicine, 2000 Dec 15;19(23):3149-64.
9. Kazdin, A.E : Single-Case Research Designs: Methods for Clinical and Applied Settings, 2nd edition. New York: Oxford University Press, 2010
10. http://twinhealers.in/homeopathy
11. El Dib RP, Atallah AN, Andriolo RB: Mapping the Cochrane evidence for decision making in health care. Journal of Evaluation in Clinical Practice 2007;13:689–692
12. Lown, Bernard MD: The Lost Art of Healing, Balantine Books, 1996

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