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Tuesday, April 16, 2024

What are Clinical Trials and Why are They Necessary?


Clinical trials are formal scientific studies that determine the safety and effectiveness of new medications and medical devices before they can receive approval for use by patients. In short, they are science projects just like most of us completed in middle school.

Clinical trials use the “scientific method” to test a hypothesis rigorously, typically comparing a new medication or device to an approved drug or device or a placebo (non-medicine that looks like the medication being studied but doesn’t have any “active ingredients”). Clinical trials are the “gold standard” for testing whether a medication or device is effective and safe in a particular population. Clinical trials may involve just a few patients with an uncommon or rare disease or include tens of thousands of patients with a common condition.

As mandated by federal law, clinical trials are required by the Food and Drug Administration (FDA) for new medications to gain approval.

They typically occur after years of laboratory research to understand how the proposed medication acts in cells and small animals, identify any potential side effects and profile its interaction with other medicines.

Often, a potential medication may do one thing in a test tube or a small animal, making it seem promising for use in humans.

However, it fails to gain FDA approval because it doesn’t have the desired effect when studied in a clinical trial with human patients.

Medications may also fail to gain FDA approval due to safety issues such as causing unexpected “offtarget” effects on the kidneys, liver, heart or other parts of the human body. Safety is always the most critical part of the FDA evaluation because once a drug receives FDA approval, it can be prescribed and used without the rigorous oversight of a clinical trial.

Just because the FDA approves a medication or device doesn’t guarantee that it will be covered for payment by an insurance company. A new drug may not be the best choice for treating a particular disease, but it may be another “tool in the toolbox” for use when preferred medications fail or have side effects for a specific patient. Once a drug or device receives FDA approval, physicians may be able to use it for patients who are different from the populations for which it was approved. This is called “off-label” use and may or may not be paid for by insurance companies.

Participation in clinical trials is an inherently generous act in that the patient may not actually benefit from participation in the study and always accepts some potential risk of a known or unknown consequence of the study medication. In a clinical trial, the participant may get the new or comparison medication (standard of care medication or a placebo). Which medication a person receives in a clinical trial is decided randomly and in a blinded fashion (neither the study team nor the patient knows which medication they receive until the end of the study).

What is learned in a clinical trial may be that the medication under study is not effective or has some safety issues that would only have been known with the clinical trial being conducted. As such, participation in a clinical trial may benefit others in the future more so than the participant.

Clinical trials are part of a careful scientific process to improve health outcomes and assure the safety of those receiving a particular medication or device after FDA approval. Ultimately, biomedical research, including clinical trials, provides hope that better solutions to disease and suffering are possible and will be found. The physicians, scientists, nurses and support staff who spend their lives performing research and conducting clinical trials are dedicated to improving health for all. You can count on it! Learn more about clinical trials and clinical trials in our region at www.research.lsus.edu/tools/clinicaltrials-office.

John A. Vanchiere, MD, Ph.D., is the associate vice chancellor for clinical research, and Erin Yates, RN, BSN, is the director of the clinical trials office at LSU Health Shreveport.


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