What is a clinical trial?
A clinical trial is a study that evaluates the safety and effectiveness of an experimental medical intervention in human volunteers. Clinical trials are used to test new ways to prevent, diagnose or treat disease.
Researchers must follow strict rules to protect participants. Typically, treatments are tested in lab animals before being studied in people.
In a clinical trial, participants may be assigned to receive an experimental treatment, such as a medication, surgical procedure or medical device. Researchers measure specific outcomes to determine whether the treatment is safe and effective.
Another type of clinical study, called an observational study, doesn't involve assigning participants to a specific treatment. Instead, participants receive routine care while researchers collect and analyze data over time.
How do clinical trials work?
Clinical trials are done in phases. Researchers try to answer a different question in each phase.
Phase 1: Is it safe?
In phase 1, researchers test a treatment in a small group of people for the first time. The main goal is to assess the treatment's safety. Participants may be healthy or may have the condition that the treatment is intended to help.
Phase 2: Does it work?
Once researchers determine the treatment is safe, they test it in a larger group of people to make sure it is effective.
Phase 3: Is it better than what we have now?
In phase 3, researchers compare the new treatment with one or more standard treatments to see which works better and is safer. This phase usually involves a large number of participants.
If the treatment proves to be safe and effective, the Food and Drug Administration (FDA) reviews it for approval. If approved, it becomes available to the public.
Phase 4: What else can we learn?
After FDA approval, researchers may conduct a phase 4 study to see whether the treatment works for other conditions or leads to problems in the long run.
Who can join a clinical trial?
People of all ages, genders and ethnicities can participate in clinical trials. Each trial has its own criteria, which may include age, sex, disease type and stage, previous treatments and other factors.
Participants may be healthy volunteers or "patient volunteers" – people with the condition being studied.
What are the benefits of joining a clinical trial?
Clinical trials are vital for medical progress. They help researchers develop better treatments and improve outcomes for many patients.
Healthy volunteers may join because they want to help others and contribute to science. Patient volunteers may also be motivated by the chance to try a promising new treatment.
However, joining a clinical trial involves some risks. The experimental treatment may not work as well as standard care or may cause side effects. Participation may also require more doctor visits and additional tests.
Participating is a personal choice. If you're considering a clinical trial, make sure you understand what's involved, ask any questions you have, and weigh the potential benefits and drawbacks.
Here are some questions to consider:
- Do I meet the eligibility criteria?
- How might I benefit from participating?
- What are the possible side effects?
- What if I'm placed in the control group that doesn't receive treatment?
- What happens if I leave or am removed from the trial?
- What if my condition worsens during the trial?
If you don't qualify for a trial, you may still be able to access the experimental treatment through a program called expanded access, also known as compassionate use. This allows companies to offer investigational treatments to people with serious illnesses who don't meet the trial's criteria but may still benefit.
