Turning scientific knowledge into real world medical treatments is known as ‘clinical
translation’. Before being marketed or adopted as a standard of care, most medicines
are tested through clinical trials. Usually clinical trials start with a small number
of people and as the procedures are perfected and potential risks evaluated, the
number of participants are gradually increased as the effectiveness of the treatment
is more closely examined.
Once safety and effectiveness is considered to be acceptable, a drug regulatory agency
such as:
The European Medicines Agency (EMEA)
The U.S. foodstuffs and Drug Administration (FDA)
The TGA.
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 to form a clinical trial, certain treatments might be tried on
one or two those. This form of testing is sometimes referred to as innovative medicine
/ single case studies.
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 people and demonstrate
that the treatment will possibly prove beneficial.
The Role of Scientific Data
Sound scientific evidence should support the rationale of any experimental treatment
Before any treatments are tested on those a strong indication from experiments in
laboratories must indicate that the scientific reasoning is sound. This usually occurs
in two stages:
Basic research, where scientists discover the underlying scientific principles.
Pre-clinical research, where new treatments are first tested on animals.
If basic research data and pre-clinical studies indicates that it is promising the
treatment may next be tested on human subjects in clinical trials.
Publication and Peer-Review of Data
An important part of the research process is that the scientists/researcher:
Reveal their methods.
Presents all of their results / data
Present their conclusions for detailed scrutiny.
This is typically done by; publishing in a peer-reviewed journal, so that 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. Another part of making
sure the science is accurate and has been presented correctly is that over time others
can replicate and repeat the same experiments to demonstrate the same outcome. These
publications also allows other scientists and health professionals to build upon
previous discoveries.
Protecting people during Clinical Translation Process
The medical community has established a number of checks to protect the rights of
people during the process of developing new therapies through both clinical trials
and innovative medicine / single case studies.
Important elements that are fundamental to this process are:
The monitoring of experimental treatments for patient safety and ethical practice.
The oversight by a regulatory agency.
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
those, 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.
Clinical trials should be conducted in a setting that provides appropriate medical
care.
Participating people should be made fully aware of the medical procedures they are
undergoing and the potential risks and provide their consent.
The progress of the people 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 people.
Treatment should be reviewed by a medical ethics committee to make sure that the
patient is being treated in an ethical manner.
Overview by Regulatory Agencies
Drug regulatory agencies monitor the development of new treatments. The nature and
methods of regulatory agencies and their responsibilities vary from country to country.
They adopt a code of conduct or guidelines for researchers and health professionals
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.
