In my previous blog post, I touched upon some of the ethical concerns for clinical trials, mostly in regards into how they are typically run and the ethics of running/not running them for rare disease cases. I briefly touched upon the dilemmas surrounding running a clinical trial when the condition which you are treating is fatal, but we’re gonna delve a bit deeper into that now.
As I previously highlighted, there are many steps and checkpoints in the Clinical Trial process. Phase I tests the drug in healthy individuals to check for side effects, phase II tests for the effectiveness of the drug against the disease, and phase III confirms the phase II findings in a larger population.
In some cases however, there are exceptions to this pipeline. One example is with patients who have tried all other standard options to target a (usually terminal) disease. Typically these are cancer patients who have exhausted all of their alternatives. In these circumstances, most have deemed it ethical to go straight to cancer patients in phase I, rather than healthy individuals, because those patients have been given only a few more months left to live, so many of the health considerations that are enforced for treatments for commonplace conditions like diabetes are waived.
While there are more ethical dilemmas to unpack here, one that immediately comes to mind is the whether it is ethical to run the necessary double blind study with these patients as it that is a considered a requirement for FDA approval.
A double blind study involves the use of a sugar “placebo” pill in order to counteract the effects of placebo, which is where the patient gets better, not because the drug is actually doing anything, but because they’re told that it will. To prove the efficacy of a drug, one needs to confirm that placebo is not the cause of their success. So, to get around this, researchers have developed the double blind trial in which half of the patients are administered the actual drug, and the other half are given a placebo. To ensure that there is minimal impact of placebo, none of the patients know which group they belong to, and neither do the doctors directly administering the trial.
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While necessary from a research perspective, and to ensure in the long term that a drug is effective, a ethical dilemma arises when it comes to purposefully withholding medications from patients who could benefit from them. This issue is still being debated within the oncology community as that is the most contentious case. A research paper in 2015purposed that a double blind study in cancer patients can be considered ethical based on their findings regarding the survival rates of patients in cancer clinical trials. However, considering it’s been 9 years since that study was conducted, perhaps the field of clincal oncology has shifted since then.
Thus the debate continues: Is it worth it to prove the absolute efficacy of a drug if it means putting individuals at risk? Or is it more dangerous in the long run to utilize a therapeutic that isn’t fully proven to work?