Clinical studies 101
You’ve probably heard a little bit about clinical studies. They’re part of the process researchers use to establish the safety profile and effectiveness of drugs before they’re approved by the FDA (U.S. Food and Drug Administration) and made available to patients. But have you ever wondered what really goes into them? Or why they can take so long and seem so complicated?
Let’s start with the basics
The idea for a clinical trial often starts in the lab. After researchers test new treatments or procedures in the lab and in animals, the most promising treatments are moved into clinical trials. As new treatments move through a series of steps called phases, more information is gained about the treatment, its risks, and its effectiveness.
A clinical study involves research with human volunteers (or participants) in order to add to medical knowledge.
Studies have specific goals or outcomes in mind when they begin—lowering blood pressure, for example. Or, in the case of multiple sclerosis, seeing how many relapses a person might have while on treatment compared to someone who received only a placebo. Studies may be sponsored by many different entities including drug companies, the federal government, or universities.
Sometimes, people living with a particular condition are recruited; other times, healthy people participate. Either way, once the person is screened to make sure they fit the protocol, they need to give their informed consent to begin. That means that they understand the risks and potential benefits of the study, how long it’s going to take, and what is expected of them. Even after they agree, people who participate are free to leave the study at any time.
Stage by deliberate stage
Clinical trials are conducted in a series of steps called phases. Each phase has a different purpose and helps researchers answer different questions.
The stages include:
- Phase 1 focuses on a drug’s safety. The goal is to determine the most frequent and serious side effects. These trials usually involve a small number of participants and are often conducted in healthy volunteers.
- Phase 2 gathers preliminary data on whether a drug works in people who have a certain condition or disease. Safety continues to be evaluated.
- Phase 3 looks at a larger group of people to learn more about safety and effectiveness
- Phase 4 happens after the FDA has approved a drug and gathers more information about safety, effectiveness, or optimal use
Each stage will have its own process and protocol. Before a drug can be approved by the FDA, all of the results need to be analyzed and reviewed. That’s a big reason why it can take years for a drug to be approved! But all that time is important so you can feel confident that the efficacy and safety profile of the drug are well established.