Public Attitudes Regarding the Appropriate Use of Placebos in Clinical Trials: A Qualitative Study

Executive Summary

March 2002

COMPAS Inc.
Multi-Audience Research
Ottawa, Toronto and Winnipeg

Background and Methods

• Health Canada commissioned COMPAS to conduct a set of focus groups to explore the attitudes of Canadians towards the use of placebos in clinical trials. The main purpose was to determine public perceptions of placebo-controlled clinical trials to inform the current placebo debate. Issues that were explored include levels of understanding of clinical trials and placebos, issues related to the use of placebos in clinical trials (including withholding of treatment), the issue of informed consent, and issues of risk (e.g. assessments of risk, communicating about risk). This research focused on three audiences: the general public, people with Type 2 diabetes, and people with depression/anxiety disorder. Health Canada submitted the research design to a Research Ethics Board (REB) and received REB approval. 
  
• In total, seven focus groups were conducted: three groups with the general public, and two with each of the 'patient' groups. Groups were held in Toronto, Winnipeg and Montreal (French). Written information was shared with participants during the focus groups, once people had expressed their initial views, so that subsequent discussions had the benefit of exposure to issues and information that are part of the current debate on placebo use (written information is appended).
  
• This research was qualitative in nature, not quantitative. As such, the results provide an indication of participants' views about the issues explored, including insight into how people think about the issues, but cannot be generalized to the full population of Canadians or of people with Type 2 diabetes or who suffer from anxiety/depression.
  
  

Level of Understanding About Clinical Trials & Placebos

• Participants exhibited relatively low levels of awareness and knowledge about both clinical trials and placebos. Some people in each group were unaware or uncertain about one or both of these. Among those who were aware, the level of knowledge appeared to be quite rudimentary. Clinical trials were equated with the testing of new medications - very little information beyond this was offered. Placebos were often referred to as 'sugar pills'. However, the concept of no 'active' ingredients in placebos, and their use as a control measure, were largely understood by people who claimed to be aware.
  
• Most participants have had little exposure to clinical trials. Relatively few knew anyone who has ever taken part in a clinical trial. Consequently, participants' knowledge of clinical trials is very general and lacking in details. It was clear that this issue is not top-of-mind, nor previously considered by most participants.
  
  

Initial Perceptions of Placebo Use in Clinical Trials

• After being provided with a brief verbal description of placebo use in clinical trials, most participants expressed support for their use. Indeed, a majority of participants in all but one group felt that placebo use was appropriate, as long as safeguards accompany this.
  
• When asked to explain why they thought placebo use was appropriate, participants focused on placebo-controlled trials being acknowledged as the best method to evaluate new medications, and on the need to test new medications on humans, not just animals, before they become available to the general public.
  
• There was a strong desire for sufficient safeguards to be in place to govern the use of placebos in clinical trials. Many suggestions were offered, including pre-screening applicants (i.e. only accepting those with low risk), careful monitoring of patient's health during the clinical trial, the ability to withdraw from a clinical trial at any time, and compensation for clinical trial participants if health is irreversibly impaired (i.e. a safety net if things "go bad"). Note that many of the suggested safeguards are already in place, something participants were not aware of.
  
  

Perceptions of Placebo Use Following Review of Written Information

• Following this initial discussion, participants read a two-page document that provided information on the use of placebos in clinical trials. This information was designed to present a neutral, balanced view of issues in the placebo debate, including the pros and cons. The intent was to increase participants' knowledge of the issues in order to generate more informed discussion. Exposure to the information did further engage participants in the issues.
  
• After reading the material, participants routinely noted that the ideas identified in the written note (both pros and cons) had already been raised in the group discussion. This served as affirmation for participants that the initial group discussion was on the mark in terms of the types of issues requiring consideration.
  
• Review of the written information tended to firm up participants' perspectives, rather than change their minds. Very few people changed their view about placebo use as a result of the information. Of those who did, some became supportive, but no one became opposed.
  
• After having reviewed the written information and hearing the views of others, participants were asked one last time whether or not they support the use of placebos in clinical trials. There was a virtual consensus in support of placebo use. Only one-in-ten expressed opposition, while all others expressed support in varying degrees. This was modestly higher than the initial levels of support. Once again, however, participants identified safeguards to govern the use of placebos, including situations where it was not considered to be appropriate.
  
• Circumstances where placebo use was not considered to be appropriate included:
  • Serious or fatal illnesses (e.g. AIDS or cancer).
  • Where risk is too great if medication is not continued.
  • If people have not been properly screened (i.e. to ensure that they are healthy enough to take part).
  • People suffering from mental illness, particularly where there is a real risk of harm to the 'patient' or to others.
  • Children or the elderly (i.e. people seen to be particularly vulnerable). Some people disagreed with this, feeling that special rules might be needed, but that such trials should still be allowed.

• The participation of certain groups was seen to require a separate process, closer scrutiny or simply more safeguards. This was seen to apply to children and to mental health patients in general, but particularly to people who are mentally challenged. The reason offered is that these groups cannot truly provide informed consent (only via a guardian), and so extra safeguards should be in place to protect their interests.
  
• Most participants were open to personal participation in a placebo-controlled clinical trial. Risk tolerance appeared to decrease when participants focused on their own children and, to a lesser degree, other family members. Decision-making factors include the type of illness or condition, the severity of the illness or condition, the state of personal health and degree of vulnerability, the consequences of non-treatment, quality of life issues, and the availability of other options. In general, the more that a clinical trial involves a medication for a serious illness, and the more that other options have been exhausted, the more open the participants are to participation in a placebo-controlled trial. However, it is important to note that potential concerns focused mainly on clinical trials themselves, not on the use of placebos in such trials.
  
  

Informed Consent & Related Issues

• Participants were generally unable to identify what would be an acceptable level of risk (other than the need to avoid irreversible harm). However, people were much more able to identify what they saw as key requirements for informed consent and for the appropriate communication of risk. Moreover, their views about informed consent and communication of risk were very consistent across the different groups.
  
• Many elements were seen to be required to achieve informed consent. This includes the provision of both written and verbal information, the use of 'plain language' in all information (i.e. the KISS principle), the use of visuals aids (e.g. videos, graphs), and access to others, such as to a neutral medical professional to discuss benefits/risks, to people who previously participated in a placebo-controlled clinical trial, or to a support group (e.g. on-line chat groups). Many other suggestions were also offered.
  
• Participants' expressed a high level of trust in doctors, particularly their own family doctor (and especially if they have gone to him/her for many years). There was a clear preference for doctors to provide both information and advice in discussions of clinical trials with patients. People wanted both objective information and the advice of their physician, who was seen to better understand their health than others. Despite the fact that people were looking for advice, few participants indicated that they would feel obliged to participate in a clinical trial even if their doctor recommended it.
  
• There was no consensus on the dual role of doctors in clinical trials - that of the protector vs. that of the researcher. This was of real concern to some people, but not to others. Most appeared to be relatively comfortable with this. That said, the level of concern increased when participants were informed that doctors receive payment for recruiting patients for clinical trials. This was not previously known and was generally not supported.
  
• Participants saw the government's role in the process centred on protection. The protection role includes ensuring that there are appropriate rules and standards to govern placebo-controlled clinical trials, monitoring performance and adherence to these rules, and ensuring that there is no undo risk for patients. Some people prefer a minimalist role for government (i.e. less is best).
  
  

Conclusions

• The ethical use of placebos in clinical trials is not an issue that the Canadian public has given much thought to. Nevertheless, participants quickly became engaged in the subject and offered thoughtful, consistent and value-based feedback on related issues.
  
• There was strong support for placebo use. However, support is conditional on the need for adequate safeguards, not only to govern the clinical trial process itself, but also to ensure that the level of risk is effectively communicated and that informed consent is actually achieved. People also identified circumstances where placebo use was not judged to be appropriate.
  
• The level of risk was seen to be difficult to assess and communicate, but people want the government, doctors and researchers to be thorough in this area, and in dialogue with patients and their families. Participants ultimately view themselves as self-agents in this area - able to make an informed decision - as long as they have been armed with clear, balanced information about the potential risks and benefits.