Placebo-Controlled Clinical Trials. Are They Still Relevant?


Dr Michael Silverman of BioStrategics Consulting Ltd raises the question and discusses the relevance of placebo-controlled clinical studies in medicine and drug development today.

Of course a placebo-controlled study is far superior in addressing the question of does your drug work and is it safe, but this not about answering a simple question. The real issue is an ethical question: whether it is fair to deny a patient with a disease the benefit of an effective intervention by treating them with a placebo instead of active treatment.

In life-threatening or highly morbid conditions in which active therapy exists, biomedical researchers agree that placebo controls are not acceptable. In contrast to this, in the complete absence of known treatment, biomedical researchers would accept the ethics of including a placebo control group in a clinical study of a new intervention. But where is the line drawn for each indication and each intervention? When is a placebo control group ethically acceptable or not? Where is the so-called “middle ground”.

In this short blog, I will try to summarize the benefits and arguments for using placebo controls and the arguments against their use:

Arguments for using placebo controls:

  • Eliminates the risk, in the treated group, that effects result from the act of simply being treated
  • Allows unbiased determination of treatment efficacy
  • Allows unbiased determination of treatment safety
  • Provides a control group if no alternative therapy is available
  • Controls for the risk of increased patient attention as a result of the clinical trial
  • FDA requires that adequate and well-controlled trials are required to support the claims of effectiveness of new drugs (does not place an emphasis on placebo-controlled)
  • Minimizes exposure to investigational agents before properly tested

Arguments for NOT using placebo controls:

  • It is unethical to not treat a patient with a disease if a treatment exists
  • Patients should receive the best treatment available as control
  • New agents should demonstrate superiority to existing treatments
  • Informed consent does not sanction the use of placebos
  • Requirements by regulatory agencies for absolute, not relative, efficacy represents flawed public policy.
  • The risk of unblinding who receives the placebo

I would be delighted to hear your thoughts on these issues, your approaches, or anecdotes about decisions you have faced in similar circumstances. To make comments or ask questions, click on this link.

For more information: go to my website or send me an email at msilverman@biostrategics.com

Copyright © 2011 BioStrategics Consulting Ltd.

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About BioStrategics Consulting Ltd

BioStrategics Consulting Ltd provides a range of management-level clinical development consulting services to the pharmaceutical and biotechnology industries
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