Clinical trials: What are they and how do they work?
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ELI5: What are clinical trials?
Clinical trials can be thought of as “experiments” to test new medical treatments, similar to how scientists conduct experiments to test new theories.
These trials are conducted to test the safety and effectiveness of new drugs or treatments, before they are made available to the general public.
When you think of clinical trials, you might imagine scientists in white lab coats testing out new drugs on patients. But there’s actually a lot more to it than that! Clinical trials are an important part of the process of developing new medical treatments, and they involve a lot of steps to ensure that the treatments are safe and effective.
In this article, we’ll go over the basics of how clinical trials work, so you can have a better understanding of this important aspect of medical research.
Phases in clinical trials
First of all, it’s important to understand that there are different phases of clinical trials.
Phase 1 – Enrollment
Phase 1 trials are the earliest stage of testing, and they’re used to determine the safety of a new treatment.
In these trials, researchers will typically enroll a small number of volunteers (usually healthy people, rather than patients with the condition the treatment is intended for) to test the treatment and make sure it doesn’t cause any serious side effects.
Phase 2 – Receiving treatment
Phase 2 trials are the next step, and they’re used to determine whether a treatment is effective and to learn more about the side effects.
These trials typically enroll a larger number of people (around 100 to 300), and a certain amount of the participants will receive the treatment while the amount will receive a placebo (a “dummy” treatment that has no active ingredient). This allows researchers to compare the results of the treatment group to the results of the placebo group, and see if the treatment is having a real effect.
Phase 3 – Confirmation of effectiveness
Phase 3 trials are the final stage of testing before a treatment can be approved by the government.
These trials usually involve a large number of people (thousands) and are used to confirm the effectiveness of a treatment, as well as to gather more information about its safety and side effects.
The results of Phase 3 trials are often used to decide whether to seek government approval for the treatment, and if so, will begin the process of submitting their findings and data to regulatory bodies such as the Food and Drug Administration (FDA) in the United States or the European Medicines Agency (EMA). This process can take several years and is designed to ensure that the treatment is safe and effective before it is made available to the public.
Once the regulatory bodies have reviewed the data, they will decide whether or not to approve the treatment.
The approval process
If it is approved, the company will be able to market and sell the treatment to doctors and hospitals. If it is not approved, the company will have to go back to the drawing board and continue to gather data and make changes to the treatment in order to meet the regulatory requirements.
“Off-label” and “compassionate” use
It’s important to note that clinical trials are not the only way that a new treatment can be developed.
Sometimes, treatments that have been used for one condition are found to be effective for another condition as well. These treatments are known as “off-label” uses. For example, a cancer drug that is approved for one type of cancer may also be effective for another type of cancer.
In addition, the FDA has a “compassionate use” program, which allows patients with serious or life-threatening conditions to access unapproved treatments if they are unable to participate in a clinical trial and have no other treatment options available.
Summary
Overall, the clinical trial process is designed to ensure that new treatments are safe and effective before they are made available to the public. While it can be a long and tedious process, it is an important step in the development of new treatments and therapies that can improve the lives of people around the world.
It’s also worth mentioning that not every clinical trial will result in a new treatment. Some trials are conducted to determine that a treatment is not effective and therefore shouldn’t be used in a clinical setting. And it’s often that these negative results could mean that the treatment is not effective, or that it is not safe for human use, and the trial will be halted at this stage.
