Drug Evaluation Committee 2008-54 Examples of non-therapeutic clinical trials
Related classification: Other
First published: Jul. 2009
Question
In GCP Article 50, Paragraph 4, Operational Notification 2, the following statement is made.
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(1)In the case of a non-therapeutic clinical trial, if all of the following items (1) through (4) are satisfied, a clinical trial may be conducted with the consent of a surrogate of the person who is to be the subject."
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(2)Such trials should be conducted in patients with the disease or condition for which the subject is intended to be indicated, unless an exception is justified."
We would like to provide specific examples when explaining this section. （As an example of (1), you mention pharmacokinetic studies in children. Are there any other appropriate examples?
（I am not sure of the specific examples of "exceptions" when the exception in (2) is justified. For example, I assume that you are referring to "the above study in the case of a rare disease with extremely few patients".
Opinion of the Pharmaceutical Manufacturers Association of Japan (PMAJ)
The "exception" referred to in GCP Article 50, Paragraph 4, Guidance 2 refers to "non-therapeutic clinical trials conducted with the consent of a surrogate", so examples of such trials would include "pharmacodynamic studies in children" and "pharmacodynamic studies in dementia", in addition to "pharmacokinetic studies in children".
The contents of GCP Article 50 include the following: i) The general principle is that consent should be obtained from the subject himself/herself; ii) When it is difficult to obtain consent from the subject himself/herself, the participation in a clinical trial by a substitute is permitted; iii) In non-therapeutic clinical trials, consent by the subject himself/herself is required in principle; and iv) In non-therapeutic clinical trials, consent by a substitute may be permitted. In non-therapeutic clinical trials, consent by the subject himself/herself is required in principle. Furthermore, it states that ⅴ) non-treatment clinical trials by a surrogate may be permitted in exceptional cases for patients other than those with the intended indication. Of these, ⅳ) and ⅴ) are considered to be provisions based on the assumption of extremely rare cases, and are considered to be very rare cases in clinical trials to develop ordinary drugs, and there are no specific examples that can be provided.
Even in the case of a "rare disease with extremely few patients," as you pointed out, we believe that clinical trials should be conducted on patients with the disease as much as possible.