Photo: Texas Children's Hospital cancer research LAB
As patients, we expect our doctors to keep abreast of the latest medical knowledge so they can provide the best care for our condition – whether it is for something as innocuous as an annual checkup or something more serious like a life-threatening or chronic condition.
Modern medicine is constantly evolving. What was once considered the standard-of-care a few years back may not be recommended any longer, only to be replaced by a better, more effective drug or a surgical procedure. Perhaps there are even patients suffering from an illness or disease for which there were previously no treatments, now may have options.
Until the early part of 20th century, the medicine practiced relied mainly on a physician’s empirical observations and experience of what therapies worked and what did not, rather than on systematic and rigorous experimentation.
Over the last few decades, as scientists gained a better understanding of human biology, we are now “discovering” new strategies and procedures to target existing or new diseases which then have to undergo rigorous clinical testing via clinical trials. This is a crucial step before physicians and surgeons can offer these improved drugs or devices, prevention strategies and screening/diagnostic procedures to the public.
Thus, properly conducted clinical trials are essential for developing new, evidence-based therapies, drugs, surgical interventions, medical devices, and disease prevention strategies that are both safe and effective. If you have ever gone to the doctor, chances are high that some aspect of your care was tested in a clinical trial.
Since the enactment of the 1962 Drug Amendments, clinical trials are required to be reviewed and approved for their safety and efficacy by the U.S. Food and Drug Administration (FDA) before they can be marketed or sold to the public.
Almost all major advances and amazing progress in modern medicine are a result of these kinds of studies. For example, the cure rate for pediatric cancers improved from less than 10% in the 1960s to over 75% now. In fact, for some kinds of cancer, over 90% of children are cured.
How are clinical trials conducted?
Clinical trials are sponsored by pharmaceutical, biotech and medical-device companies or through governmental agencies. They come in all sizes. Depending on the product or procedure being tested and the developmental stage, they could be initial pilot studies involving a few patients in a single medical center or a large-scale study with thousands of patients being tested in multiple medical centers around the globe.
Typically, clinical trials are conducted under the supervision of physician (investigator) in academic medical centers or contract research organizations. Academic and commercial clinical study centers have institutional review boards (IRBs) that review and approve each study protocol before the study can commence. IRBs ensure the study is designed to be safe and ethical, and the participants will be properly informed of any potential risks and benefits.
The rules that protect people in research are part of U.S. federal law and are taken very seriously.
The FDA reviews and audits all clinical studies pertaining to drugs, procedures and devices in this country. Different countries have different regulatory agencies that oversee clinical trials. Usually, it is the shared responsibility of the sponsor, involved physicians, the IRBs and the regulatory agency of the country where the drug, procedure or device will be marketed/sold to ensure the safety of the subjects participating in the trial.
Patients and healthy subjects volunteer to participate in these studies via informed consent, a legal process in which subjects receive detailed information about the study, procedures that will be involved, inherent risks, and potential benefits, prior to their participation in the trial. Depending on a child’s age, parents or legal guardians can give informed consent (permission) on their child’s behalf, while children of a certain age may be required to provide informed assent (agreement).
Some people participate in clinical trials in hope of benefiting themselves or their loved ones, while others (e.g. healthy subjects) may do so because they want to contribute to the growth of medical knowledge in general.
Texas Children’s is currently recruiting patients for over 200 trials for various conditions. If you or your child is suffering from a condition, you may consider joining a clinical trial designed to learn more about that condition and/or how to treat it. Talk with your doctor if you think a clinical trial might be right for you.