The continued advancement of medical therapeutics and procedures through experimentation is vital to creating safer, more effective, and more accessible treatments for disease. Experimental medicine, in contrast to therapeutic medicine, is defined as the study of treatments currently undergoing clinical trials or other forms of testing to understand their effectiveness and safety.
As outlined by Marianne Boenink, who discusses the ethics of molecular medicine, the scientific community’s reason for developing experimental treatments exists within five core ideas. While this list is not exhaustive, the ultimate goal of experimental medicine is to save lives and improve patients’ conditions.
Core Goals of Experimental Medicine
- Diagnose disease earlier and with more accuracy
- Improve the reliability of prognoses to prevent over or under treatment
- Create more effective treatments
- Reduce the risks and invasiveness of a given treatment
- Create better methods to monitor and overview a patient’s health/condition
Experimental medicine exists in many areas of the healthcare field. One example is gene therapy, which can make edits to genes or strands of DNA that will help to prevent or treat certain diseases. For example, gene therapy trials have shown promising results in treating several types of cancer, including lung and pancreatic cancer.
Another modern example of experimental medicine is the use of bacteriophages. Bacteriophages are viruses that attack bacterial cells’ ability to reproduce, ultimately destroying the bacterial cell in the process. They are harmless to humans as Bacteriophages target specific structures on bacteria not found on human cells. Bacteriophages are currently being used as an alternative treatment for bacterial infections, especially in cases where strains of bacteria have developed wide-scale antibiotic resistance. For instance, bacteriophages have been used in clinical trials to treat antibiotic-resistant strains of Cholera, Diphtheria, and E.coli.
In order for progress to be made and for experimental therapies to be brought to market, humans must eventually participate in trials in order to prove the effectiveness and safety of a given treatment. This brings with it the possibility of harm if patients are treated unethically.
The New England Journal of Medicine highlighted two core ideals regarding experimental medicine after the end of World War II which still resonate to this day. The first is that patients who participate in any clinical trial must be appropriately informed about the risks and benefits of a procedure or treatment and the second is that there must be a reasonable balance between risks and benefits.
Informed consent is a vital part of any medical procedure and is a more thorough process than one might initially imagine. Two of the greatest challenges regarding informed consent are (1) making sure that patients understand the complex medical jargon that can be a part of the experiment’s explanation, and (2) ensuring that patients have capacity to consent to voluntary participation. The first issue may only require a detailed conversation that thoroughly explains the confusing aspects of a procedure.
Ensuring that a patient has decision-making capacity can be more challenging. For participants under 18 and those lacking capacity, a surrogate decision maker is required. A clinical trial that used gene therapy to prevent blindness caused by Leber’s congenital amaurosis ran into this issue. Researchers found that the gene therapy under investigation was most effective at preventing blindness when started at an early age. This raised questions about whether or not younger patients are able to consent for experimental treatments, or if parental consent is required. The ultimate decision was that parents reserve this right until children turn 18 years old. Situations like these can make obtaining informed consent in clinical trials difficult, but not impossible. Informed consent ultimately exists to ensure patients know what they stand to gain and lose from a given procedure.
The second idea that the New England Journal of Medicine discussed was that any experimental treatment must have a reasonable balance between the benefits gained and the risks involved. It is essential to understand that physicians, although with good intent, cannot use exceedingly dangerous or ethically wrong procedures on a patient even if it could possibly heal the patient. It is important to ensure that a patient involved in an experimental treatment/procedure does not bear an unjust amount of suffering. Physicians are bound by the principles of non-maleficence, to do no harm, and beneficence, to act for the benefit of a patient. If the risks of a procedure begin to outweigh the benefits, the principle of non-maleficence has been violated.
A third and final ethical ideal important to experimental medicine introduced by Sue V. Rosser involves the approval process of clinical trials and other experimental treatments. Dr. Rosser found that the review boards approving clinical research were made up primarily of men. This led to the approval of studies which would primarily benefit men with significantly fewer studies focusing on therapies which would benefit women. This finding is vital to the future of clinical research as areas of the world continue to address gender equity and become more ethnically diverse. To clarify, the pursuit of racial and gender equality is critical for addressing disparities disproportionately affecting various groups of people.
This is especially true within experimental medicine. Taking the time to ensure that multiple perspectives from different cultures, genders, and ethnicities are included in the approval process of clinical research is essential to reduce health disparities present in society. Failure to do so may further marginalize groups who are already underrepresented in society and exacerbate unjust health conditions present within them.
In summary, the advancement of medicine is an integral part of developing more beneficial treatments for patients. While this sounds great in theory, any real-world application of experimental medicine must ensure that trial participants are treated with a high level of respect and care. Failure to do so can create a dehumanizing environment that targets a specific group of people and further exacerbates medical disparities present in the world.