Gold Standard Clinical Trials are Immoral

        Clinical research in the field of rapidly fatal diseases such incurs a unique set of ethical dilemmas. One such dilemma is the conscious decision to withhold a potentially helpful therapeutic drug from an individual who would otherwise certainly die from their disease. I am of course talking about conducting a randomized, double-blinded, placebo-controlled clinical treatment trial which is the gold standard for any clinical research trial trying to determine the effectiveness of a new therapeutic. The way I phrased the second sentence makes the idea of the gold standard trial design sound heartless and immoral, but it is technically true in the placebo groups. This is the foundation for the ethical debate behind clinical research in rapidly fatal diseases. 

Right off the bat, participation in these studies has its own ethical component. Patients with a rapidly fatal disease might be more likely to try any potential treatment and subject themselves to any risk in the hopes of potentially alleviating their illness. These individuals could be classified as a vulnerable population in this regard. This is especially true in individuals who don’t have any serious illness but are considered “at-risk” such as those who are asymptomatic but are positive for a disease associated mutation. 

        An example of the lengths to which some individuals will go to receive any available potential treatment can be seen in the legal battle of two UK families in 2002 whose children had just been diagnosed with a rapidly fatal neurodegenerative disease (vCJD). The legal battle was fought and won leading to the intracranial administration of a drug that had shown minimal efficacy in a few studies in animal models (Bechtel and Geschwind, 2014). The drug was later shown to not be an effective therapeutic. This circumventing of the usual path of appropriate gold standard clinical trials is a precedent that portrays this ethical debate we face in clinical research.

        Additionally, afflicted individuals may be less likely to participate in these gold standard trials due to the nature that they might receive the placebo and receive no benefit whatsoever. This leads some individuals towards more observational research which is far less reliable and less likely to hold up when experimental trials are conducted. However, due to the nature of human trials, sometimes observational studies are the best we can do. It is this balance between the need for high quality research that benefits society as a whole and the want to do good by providing a potentially efficacious treatment at the individual level that lies at the heart of clinical research.

References: 

       Bechtel K, Geschwind MD. Ethics in prion disease. Prog Neurobiol. 2013 Nov;110:29-44. doi: 10.1016/j.pneurobio.2013.07.001. Epub 2013 Jul 29. PMID: 23906487; PMCID: PMC3818451.