Positive Health Online

RCTs and the Placebo Effect

In the past fifty years, the world of medicine has been swept by the Evidence-Base Medicine revolution (EBM). Based strongly on empirical thought and systematic clinical rigour, enormous advances have been made in our understanding of disease, diagnosis and treatment. Inspired by this progress, I decided to join scientific forces and contribute in breaking the frontiers of medical knowledge and advance health care. I enthusiastically signed up for a PhD in Health Sciences and Clinical Evaluation at the University of York. Here I learned about one of the landmarks of this revolution, the double-blind randomized controlled trial (RCT).

Large samples of patients with similar characteristics are often randomized either to a treatment or to something that looks just like it but has none of its specific therapeutic properties. Placebos help separate the effects of the real treatment from the effects of beliefs and expectations. If patients receiving the therapy have significantly better health outcomes than patients who receive the placebo, then the therapy can be prescribed and marketed.

A significant proportion of patients respond to placebos, and for a variety of conditions. Evidence for this comes from research designs that hierarchically sit at an even higher place than the RCT: systematic reviews of RCTs.[1],[2],[3] The placebo effect has received a lot of bad press. It has been seen as 'noise' and a nuisance, causing a lot of frustration to investigators who find it difficult to publish negative findings, and to companies or scientists who developed the therapy. It has often been discounted as 'only a placebo effect', possibly demonstrating that the patient's illness, was only 'in their head', that it wasn't 'real'.

Increasingly, research is demonstrating that this effect is very real. Neuroimaging studies are showing that patients with severe depression respond to placebos with an activation of the pre-frontal cortex,[4] patients with Parkinson's react to placebos with an increase of dopamine,[5],[6] and patients with ostheoarthritis of the knee can have remarkable improvements in both pain and function lasting for at least two years following placebo surgery.[7]

This phenomenon may be the key to understanding how self-healing responses can be activated by factors such as beliefs, expectations, and health care interactions. It highlights the potential that individuals have to heal and reminds us that while there are often average responses to specific therapies, the range, depth and course of a healing response can differ widely among people.

In an attempt to understand how healing responses can be activated during a consultation, I systematically reviewed all RCTs where study participants were randomized either to a drug or to a placebo and to different ways of receiving one of the two treatments. Patients may get a friendly and warm doctor who told them that the treatment being investigated was 'extremely effective and safe', or they may get a formal, detached doctor who told them that the treatment being investigated 'may or may not work'.[8]

We found at least some evidence to suggest that raising expectations and interacting with patients in a personal and social way was actually therapeutic. This finding was published in the Lancet.[9] From this review I could recommend that medical students be trained in being warm and friendly with their patients and raise their expectations, but would this be practical or ethical? Being over-friendly could be misinterpreted as not taking the patient seriously and raising beliefs around a treatment may cause the patient to have unrealistic expectations, possibly leading to disappointment. Rather than simply being 'friendly and warm', what matters in a healthcare interaction is about developing trust, building a relationship, an alliance or a partnership.

Consultation Research

Considering that the average consultation time is 10 minutes,[10] and that our health care system is highly specialized and focused on large patient turnover, is it possible do develop a good patient-practitioner relationships? What is a 'good' relationship?

Researchers in the UK have been exploring ways to measure the quality of a consultation using a number of tools. These include assessing whether following a clinical encounter patients felt more 'enabled' by their doctor. Patient enablement is described as feeling better able to understand and cope with the illness, and feeling more confident about their health.[11]

Considering the time constraint, it is perplexing how healthcare professionals can explore the source of patients' illnesses and help to empower them in single consultations. Many illnesses are caused or at least influenced by a variety of factors; during a visit, particularly with patients suffering from chronic conditions, it is important to take a holistic approach.

In a survey of 3,713 general practitioners, nearly nine out of ten felt that a holistic approach was essential to providing good healthcare, but only one in 15 thought that the current organization of primary care services made this possible, because of time.[12]

Would allowing healthcare professionals to spend more time with patients increase the quality of the consultation and thus harness healing?

This really depends on how time is spent. Medical education is largely based on anatomy, physiology, pharmacology, surgery and diagnostic tools and, to a much lesser extent on humanistic skills. Medical training is also increasingly based on EBM, consisting largely of findings from clinical trials and systematic reviews. These studies are powerful but extremely limited as they are able to provide average response rates from large samples but they fail to point to individual responses.

When an individual becomes ill, and is given a serious diagnosis such as cancer, she may want to know the statistics, such as what is the average life expectancy for people with the same diagnosis, how effective are the treatment options, and what are their side-effects. The patient may also want to know what is possible beyond statistics, based more on case studies. Biomedicine can only provide a small picture of the human potential. Because of its focus on organic conditions and physical interventions, it has tended to ignore the therapeutic power of psychosocial factors such as optimism, laughter, faith, love, creativity and expression.

Inspiring Healing

As a reaction against the time limited and highly reductionistic approach of medicine, increasingly patients are seeking care from complementary and alternative medical (CAM) practitioners.[13] This is also to find other sources of hope, to ways to find balance and to lift the spirit from painful suffering. Stories of spontaneous remissions and miraculous cures give hope to anyone facing life-challenging conditions. They may not be able to give specific advice, but they can inspire. Rachael Naomi Remen, Berni Siegel, Norman Cousins are a few examples of wonderful healers who present medicine as a healing art and provide stories to encourage physicians and patients to wonder and be in awe of the healing potential.

David Reilly is another of these remarkable healers. He is the lead Consultant at the Glasgow Homeopathic Hospital (GHH), a unique holistic and integrative hospital offering a range of therapies for patients with complex illnesses who have failed to progress with more orthodox care. The mission of this hospital is 'to help people self-heal – if possible from their disease, but always from their suffering. We wish to create a space, a place, an atmosphere, an approach and an experience that helps this healing happen. We strive to treat each patient as unique, individual, and whole person, recognising their inner and outer life and have a significant impact on the processes of disease and healing'.[14]

The average consultation time is 56 minutes for new patients and 19 minutes for follow-up patients.[15] The length of time spent with patients appears to be providing outstanding results. Following admissions to the GHH, 88 per cent of patients perceived benefits in general well-being. A total of 90 per cent rated the care at the GHH to be better or much better than conventional care, with 81 per cent rating overall care as excellent. In a follow-up of 230 patients attending the GHH the overall average enablement score in this setting was found to be 50 per cent higher than the average in primary care.[12] Patients explained how they valued 'time', the 'whole-person' approach, 'being treated as an individual', that their story was being listened to (often for the first time), and that their symptoms were taken seriously. They felt that the doctors were compassionate and positive, often engendering hope. Equality of relationship was found to be a major theme, with a strong sense of mutual respect.[16]

Wanting to understand how healing could be activated in health care consultations, I spent time observing dozens of patients consulting with Dr Reilly. I noted how a number of these patients experienced a personal transformation or an awakening following their consultation. This awakening has been defined as a moment of clarity where a new insight or understanding is gained and new possibilities of growth to new levels of psychological and spiritual maturity are opened. Maslow describes it as profound moments in a person's life, in which there is a "feeling of ecstasy and awe… with the conviction that something extremely important and valuable has happened…"[17]

These moments have important and lasting effects on individuals who are likely to see themselves and others in a more spontaneous and healthier way. This shift in perception may be very sudden and is experienced as a flash of insight, a sense of 'aha' that occurs when the misleading set is finally broken. According to Rogers, a proper emotional climate is needed and this climate includes empathic understanding and being genuine.[18] Empathy and compassion are valued extremely highly at the GHH. One of the leading researchers there, Dr Mercer, actually developed an empathy measure and discovered that empathy was actually crucial for patient enablement.[12],[15]

Some of the changes I observed at the GHH were beyond the practice and expectation of clinical science. Patients are encouraged not to try any treatment (conventional or alternative), but to simply realize that healing can come from within. They are also encouraged to come off anti-depressants and painkillers and to engage in new ways of dealing with their suffering by being encouraged to keep a 'creative process going', having a 'heart-to-heart' with someone they could trust, keeping a diary, playing music, dancing, and being gentle with themselves.

The EBM revolution in its focus on evaluating the effectiveness of therapies is failing to capture some of the amazing aspects of healing, and in continuing to do so, may actually remove the likelihood for us to fill this gap. In 2002, an Effective Care Bulletin presented an overview of four systematic reviews of homeopathy and concluded that: "there is insufficient evidence of effectiveness either to recommend homeopathy as a treatment for any specific condition, or to warrant significant changes in the current provision of homeopathy".[18] Finding little evidence for the effectiveness for homeopathy the Scottish NHS recently announced a plan to cut the in-patient unit at the GHH, as part of a savings strategy.

Conclusion

EBM is only allowing us to touch the tip of the iceberg as RCTs and systematic reviews do not capture the real value and effectiveness of holistic and patient-centered approaches and fail to provide a complete picture of the therapeutic effects of care that takes place. Instilling hope and faith is more an art than a science and requires a great deal of sensitivity, connection and genuine trust, using a language that speaks directly to the individual patient. Like mistaking the hand that is pointing to the moon for the moon, we are incorrectly concluding that the pill (drug, acupuncture, psychotherapy, etc.) is failing to prove its worth, when the value and effectiveness of the treatment do not simply reside only in the properties of the medication, but also in the health care setting and in the type of healthcare relationship that is provided. Therapies have enormous values, certainly, but so do our own personal self-healing mechanisms. We just need to understand ways of tapping into these. In failing to do so, we are underestimating the individual potential we have to heal and be healed.

References

1. Turner JA, RA Deyo et al. The importance of placebo effects in pain treatment and research. JAMA. 271(20): 1609-14. 1994.
2. Kirsch I and G Sapirstein. Listening to Prozac but Hearing Placebo: A Meta-Analysis of Antidepressant Medication. Prevention & Treatment 1(Article 0002A.): 1998. Available on the World Wide Web: http://journals.apa.org/prevention/volume1/pre0010002a.html
3. Crow R, T Gage et al. The role of expectancies in the placebo effect and their use in the delivery of care: a systematic review. Health Technology Assessment. 3(3): 1999.
4. Leuchter AF, IA Cook et al. Changes in brain function of depressed subjects during treatment with placebo. Am J Psychiatry. 159(1): 122-9. 2002.
5. de la Fuente-Fernandez R, M Schulzer et al. The placebo effect in neurological disorders. Lancet Neurol. 1(2): 85-91. 2002.
6. de la Fuente-Fernandez R and AJ Stoessl. The placebo effect in Parkinson's disease. Trends Neurosci. 25(6): 302-6. 2002.
7. Moseley JB, K O'Malley et al. A controlled trial of arthroscopic surgery for osteoarthritis of the knee. N Engl J Med. 347(2): 81-8. 2002.
8. Gryll SL and M Katahn. Situational factors contributing to the placebos effect. Psychopharmacology (Berl). 57(3): 253-61. 1978.
9. Di Blasi Z, E Harkness et al. Influence of context effects on health outcomes: a systematic review. Lancet. 357(9258): 757-62. 2001.
10. Deveugle, Derese et al. 2002. Consultation length in general practice: Cross sectional study in six European countries. BMJ. 325 (7362): 472. 2002.
11. Howie JG, DJ Heaney et al. Quality at general practice consultations: cross sectional survey. BMJ. 319(7212): 738-43. 1999.
12. Mercer SW, D Reilly et al. The importance of empathy in the enablement of patients attending the Glasgow Homoeopathic Hospital. Br J Gen Pract. 52(484): 901-5. 2002.
13. Eisenberg DM, RB Davis et al. Trends in alternative medicine use in the United States, 1990-1997: results of a follow-up national survey. JAMA. 280(18): 1569-75. 1998.
14. Reilly D. 1999 quoted in: Hays M. Glasgow Homeopathic Hospital. Architectural Competition. UK City of Architecture and Design. Glasgow Homeopathic Hospital and Glasgow. 1999.
15. Mercer SW, GC Watt et al. Empathy is important for enablement. BMJ. 322(7290): 865. 2001.
16. Mercer SW and D Reilly. A qualitative study of patient's views on the consultation at the Glasgow Homoeopathic Hospital, an NHS integrative complementary and orthodox medical care unit. Patient Educ Couns. 53(1): 13-8. 2004.
17. Maslow. 1970. Religions, Values and Peak Experiences. New York. Harper and Row. 1970.
18. O'Meara S, P Wilson et al. Homeopathy. Effective Health Care Bulletin. 7(3): 1-12. 2002.