The process used to turn scientific knowledge into real world medical
treatments is called clinical translation.
Before being marketed or adopted as standard of care, most medicines are tested through clinical trials. Usually clinical trials start with a small number of people to test safety. As the procedures are perfected and the risks evaluated, the number of participants is gradually increased and the effectiveness of the treatment is more closely examined. Once safety and effectiveness have been established, a regulatory agency such as the European Medicines Agency (EMA) or the U.S. Food and Drug Administration (FDA) may approve the treatment for particular diseases or conditions.
Not all development takes place in clinical trials. Sometimes, in attempting new surgical techniques or where the disease or condition is rare and does not have a large enough group of people to form a clinical trial, certain treatments might be tried on one or two people. This form of testing is sometimes referred to as innovative medicine.
Whether through a formal clinical trial or a single attempt to treat just one person, there should be a number of checks in place to protect the rights of patients and show that the treatment will possibly prove beneficial.
Before any treatments are tested on people there should be a strong indication from experiments in laboratories that the scientific reasoning is sound. This usually occurs in two stages: basic research, where scientists discover the underlying scientific principles; and preclinical research, where new treatments are first tested on animals. If basic research data and preclinical studies look promising a new treatment may next be tested on human subjects in clinical trials.
A vital part of the research process is that scientists open up their methods, results and conclusions to the scrutiny of other experts. This is typically done by publishing in a peer-reviewed journal, meaning the work is first read and critiqued by others working in the field to make sure it was done well and accurately discusses the scientific discoveries. An important part of making sure the science is accurate is that over time other scientists can replicate and repeat the same experiments and have the same outcome. Publication also allows other scientists and doctors to build upon previous discoveries.
The medical community has established a number of checks to protect the rights of patients during the process of developing new therapies through both clinical trials and innovative medicine. Among the many criteria that are important for developing new therapies, the ISSCR identified two elements that are fundamental to this process; monitoring of experimental treatments for patient safety and ethical practice, and oversight by a regulatory agency such as the European Medicines Agency (EMA) or the U.S. Food and Drug Administration (FDA).
1. Monitoring of Experimental Treatments for Patient Safety and Ethical Practice
Clinical trials are conducted before a treatment becomes available to the general public. Before a trial begins, it should first be carefully reviewed by a group of people, who together have broad expertise and experience in research, medicine and ethics. These groups, often called Institutional Review Boards (IRBs) or medical ethics review committees, evaluate a number of factors including the potential risks weighed against the potential benefit. Trials should be conducted in a setting that provides appropriate medical care. Participating patients should be made fully aware of the medical procedures they are undergoing and the potential risks and provide their consent. The progress of the patients in the trial should be continuously monitored by medical practitioners who are both knowledgeable in the field and who have the authority to stop the trial for the benefit of the patients. Even if a single patient is involved, the treatment should be reviewed by a medical ethics committee to make sure that the patient is being treated in an ethical manner.
Read more about How Clinical Trials Work
2. Oversight by Regulatory Agencies
National oversight agencies, such as the European Medicines Agency (EMA) or the U.S. Food and Drug Administration (FDA) monitor the development of new treatments. The nature of regulatory agencies and their responsibilities varies significantly from country to country. Generally they adopt a code of conduct or guidelines for researchers and clinicians to follow to promote safe and effective medical practice. These agencies judge the scientific principles, the level of danger to the patient, and potential benefits of the proposed therapy at several stages in the clinical translation process. Once a treatment has been shown to be both safe and effective, the national agency approves the treatment for the market and it becomes available to the public.
The principles outlined here developed over the period of several decades, and largely in response to instances where people were harmed during human experimentation. Find out more about How Medical Safeguards Evolved.