Q: Can I carry out an interventional study on human subjects even though I don't have enough convincing evidence from animal and in vivo studies?
I am currently writing a research protocol on using an oral medication in type 1 diabetes. The medication has not been tried in humans in this context but is given for other conditions. There has been only one animal study on the medication with promising results. From a legal/ethical perspective, can I still carry on with the randomized control trial (RCT), or should I begin with another study design such as a case series in an attempt to increase evidence?
There are seven ethical requirements for an RCT as per a report published in the Journal of American Medical Association. One of them is favorable risk-benefit ratio, which involves determining that the potential benefits for individuals and society at large are greater than the associated risks. In case the risks are deemed greater, the trial cannot be justified.
Also, the design must first be reviewed by your institutional review board (IRB) or ethics committee (before you can proceed with registering the protocol). To receive the committee approval, you need to provide sufficient rationale to conduct the RCT. Without sufficient justification, the IRB is unlikely to provide its approval.
Based on the above points and given the information you have provided, an RCT would not seem justifiable here.
As your alternative is a case series, note that this too requires the IRB approval, and more importantly, informed consent from participants.
Hope that helps. You may also find the following resources useful:
This content belongs to the Conducting Research Stage