Welcome to Ethics Consult -- an opportunity to discuss, debate (respectfully), and learn together. We select an ethical dilemma from a true, but anonymized, patient care case, and then we provide an expert's commentary.
Last week, you voted on whether or not researchers should biopsy kids' brains with no benefit to them.
Is it ethical to involve children in this research?
Yes: 35%
No: 64%
And now, bioethicist Jacob M. Appel, MD, JD, weighs in:
One of the fundamental differences between research studies and clinical medicine is that investigative subjects should not expect to benefit from their participation in studies. In fact, subjects in scientific research, such as pharmaceutical trials, may actually be harmed.
For instance, drugmaker Eli Lilly halted late-stage trials of its promising Alzheimer's disease treatment, semagacestat, in 2010 after participants showed increased cognitive decline and higher rates of skin cancer. All six participants in a 2006 trial of the synthetic antibody TGN1412 became critically ill. Yet despite highly publicized negative outcomes, such as the death of 18-year-old Jesse Gelsinger in a University of Pennsylvania gene therapy study in 1999, many participants mistakenly believe they will benefit from participating in research.
The mistaken belief, first described by psychiatric ethicist Paul Appelbaum, is called the "therapeutic misconception." Medical sociologist Gail Henderson and her colleagues have the therapeutic misconception as occurring when "individuals do not understand that the defining purpose of clinical research is to produce generalizable knowledge, regardless of whether the subjects enrolled in the trial may potentially benefit from the intervention under study or from other aspects of the clinical trial."
Patients in research studies -- for one of many reasons -- lose sight of the fact that they might not benefit. To many laypeople, research and healthcare appear very similar -- both involve medical professionals, frequently dressed in white lab coats, providing interventions such as pharmaceuticals. Often these participants are desperate for help, having exhausted all known remedies.
Power imbalances between scientific experts, who are usually physicians, and patients, further complicate matters. Many research subjects, receiving experimental pills from doctors, simply trust these doctors (mistakenly) not to expose them to risk. In the pediatric setting, parents of subjects are vulnerable to the same misunderstandings and false optimism. One must ask to what degree the parents of potential participants in Crusher's proposed study truly understand that their own children will not benefit.
The federal government has designated children among the vulnerable populations -- which also include pregnant women, cognitively impaired people, and prisoners -- who deserve special protections as the subjects of research. In Crusher's scenario, these children will face genuine risks of medical complications at no benefit to themselves, and they are generally too young to assent meaningfully to such risks. Under these circumstances, the federal government allows institutional review boards (IRBs) to approve such studies only under narrow circumstances, including when risk is low and the study is of vital importance "for the understanding or amelioration of the subjects' disorder or condition; and adequate provisions are made for soliciting assent of the children and permission of their parents or guardians."
If the research does not meet these requirements, the U.S. Department of Health and Human Services may, under certain circumstances, commission a panel of experts to authorize the study. In practice, Dr. Crusher will have to convince an IRB that the increased risks of brain biopsies are minor to these patients and that the data she gathers will prove crucial in cancer research. IRBs can be notoriously unsympathetic audiences, however, so she may have her work cut out for her.
Jacob M. Appel, MD, JD, is director of ethics education in psychiatry and a member of the institutional review board at the Icahn School of Medicine at Mount Sinai in New York City. He holds an MD from Columbia University, a JD from Harvard Law School, and a bioethics MA from Albany Medical College.
Check out some of our past Ethics Consult cases:
Let Researcher Create Mouse-Human Brain?